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Synthetic Biology Market Size, Scope and Forecast | Cyrus Biotechnology Inc. (US), Intrexon Corporation (US), GenScript Biotech Corporation (China),…

Posted: June 13, 2022 at 2:19 am

New Jersey, United States The reports goal is to provide a detailed analysis of the Synthetic Biology Market. The study involves a study of complicated data in simple manner, as well as the industrys past and current state, as well as anticipated market size and trends. The report examines all areas of the industry, with a focus on significant companies such as big players, followers, and newcomers.

The research includes a PORTER, PESTEL analysis as well as the possible influence of microeconomic market elements. External and internal factors that are expected to make a positive or negative impact on the industry have been examined, providing decision-makers with a clear futuristic vision of the industry.

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Key Players Mentioned in the Synthetic Biology Market Research Report:

Cyrus Biotechnology Inc. (U.S.), Intrexon Corporation (U.S.), GenScript Biotech Corporation (China), Integrated DNA Technologies, Inc. (U.S.), New England Biolabs (U.S.), Thermo Fisher Scientific Inc. (U.S.), Merck KGaA (Germany), Synthetic Genomics, Inc. (U.S.), Codexis, Inc. (U.S.), Synthego Corporation (U.S.) among others.

By studying market segments and evaluating worldwide Synthetic Biology market size, the research also aids in understanding the Synthetic Biology market dynamics and structure. The research will be a full guide with a clear picture of competition analysis of key competitors in the worldwide Synthetic Biology market by product, price, cash flows, product line, strategic initiatives, and regional presence.

The research also provides profiles of prominent companies in the Synthetic Biology industry, as well as their SWOT analyses and market strategies. Furthermore, the research focuses on key market players, providing details such as company biographies, components and services provided, financial records, and so on.

Synthetic BiologyMarket Segmentation:

Synthetic Biology Market, By Product

Core Products Enabling Products

Synthetic Biology Market, By Technology

Gene Synthesis Genome Engineering Cloning Sequencing Site-directed Mutagenesis Measurement and Modeling Microfluidics Nanotechnology Bioinformatics

Synthetic Biology Market, By Application

Medical Application Pharmaceuticals Drug Discovery and Therapeutics Artificial Tissue and Tissue Regeneration Industrial Applications Biofuel and Renewable Energy Food & Agriculture Others

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Synthetic Biology Market Report Scope

Key questions answered in the report:

1. Which are the five top players of the Synthetic Biology market?

2. How will the Synthetic Biology market change in the next five years?

3. Which product and application will take a lions share of the Synthetic Biology market?

4. What are the drivers and restraints of the Synthetic Biology market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Synthetic Biology market throughout the forecast period?

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Spectrometry Market will grow at a CAGR of 6.4 % by 2028, Owing to Rising Demand from the Pharmaceutical and Biotechnology Industries; Fact. MR…

Posted: June 13, 2022 at 2:19 am

Increase in the Use of Spectrometry Devices in Various End-Use Industries and Rise in Technological Advancements in the Field Of Spectrometry Are Driving Market Growth, Reveals Fact.MR

Growing demand for new and enhanced instruments, increased focus on research and development in the healthcare industry, and rise in the number of contract research organizations (CROs) are some of the factors expected to boost spectrometry market growth over the coming years.

Fact.MR, a Market Research and Competitive Intelligence Provider - Fact.MR, a market research and competitive intelligence provider, has conducted a comprehensive analysis on the global spectrometry market, which is expected to rise at a CAGR of 6.4% over the forecast period (2021 to 2028).

Spectrometry instruments are used for a variety of applications in a wide range of end-use industries, including environmental testing, research, and the food and beverage industry. As a result, increased demand for these devices across various sectors is likely to propel the worldwide spectrometry market forward throughout the forecast period.

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Government organizations across the world have laid down strict rules for monitoring water and soil samples. This is contributing to increased sales of spectrometry devices, which is driving the global spectrometry market.

Because of their capacity to provide several benefits, such as increased imaging quality, portable and handheld spectrometry equipment are gaining enormous popularity in a wide range of end-use sectors. Furthermore, these devices are becoming more widely used since they help with on-site sample analysis. Growing demand for spectrometry instruments is supported by the strict regulatory environment governing the monitoring of soil and water samples.

What are the Reasons for the Rapid Growth of the Spectrometry Market?

Continuous Innovation in Spectrometry Technique to Augment Market Growth

Spectrometry techniques make use of cutting-edge technology. The innovation in handheld and portable instruments necessary for spectrometry processes is driving the worldwide spectrometry market. These devices have better imaging quality and can be used in sample analysis. It saves the time and effort required to carry heavy and bulky samples to laboratories for chemical examination.

Drug testing and drug discovery, food quality control, carbon dating, environmental testing, and defence industries all use these handheld and portable spectrometry equipment. The global spectrometer market is expected to increase because of the increasing use of spectrometry equipment in clinical research, protein identification, tissue analysis, proteomics, and toxicology.

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Key Takeaways from Spectrometry Market Study

Competitive Landscape

Key players in the spectrometry industry are focusing on offering improved solutions to maintain growth. Companies have a diverse and technologically advanced product portfolio as well as a wide global presence.

For instance,

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Winning Strategy

Players are putting more emphasis on R&D in order to increase the analytical efficiency of their product offerings. Companies in the worldwide spectrometry market are putting in more money into the development of technologically advanced products.

A large number of market participants also use tactics such as agreements, collaborations, and acquisitions to expand their product portfolios and geographic presence in the market.

New product releases, product approvals, and product upgrades are the core growth strategies employed by major players to increase their positions in the global spectrometry industry.

For instance,

Fact.MRs Domain Expertise in Healthcare Sector

Our healthcare consulting team guides organizations at each step of their business strategy by helping you understand how the latest influencers account for operational and strategic transformation in the healthcare sector. Our expertise in recognizing the challenges and trends impacting the global healthcare industry provides indispensable insights and support - encasing a strategic perspective that helps you identify critical issues and devise appropriate solutions.

Insulin Delivery Devices Market- The global insulin delivery devices market is expected to foray ahead at a healthy CAGR of 7% over a forecast period of 2020-2030. Analyzing through the market lens, it is expected to climb up in terms of revenue, to US$ 14 billion by the end of FY 2020.

Injection Pen Market- The sales of injection pens have considerably picked up in the last couple of years, as they have become a widely accepted treatment method to address a broad spectrum of medical considers.

Swab and Viral Transport Medium Market- Demand in the swab and viral and transport medium market has witnessed remarkable upsurge in the recent past, as the number of tests and laboratory experiments are registering an unprecedented high during the COVID-19 pandemic.

Medical Gases and Equipment Market- The global medical gasses and equipment market will continue exhibiting a steady growth. The demand is poised to surge even as COVID-19 outbreak tightens its noose around the world.

Medical Suction Devices Market- According to the US Census Bureau, the geriatric population is increasing at an accelerated rate. This is anticipated to act as a key driver for growth of the global medical suction devices market, which is projected to exhibit a CAGR of around 4% over the forecast period 2020-2030.

Contraceptives Market- Demand for contraceptives to register a robust 6.4% CAGR during the forecast period (2020-2025). The global contraceptives market shall surpass a valuation of US$ 37 Billion by the end of 2025.

Dental Restoratives Market- The dental restoratives market is projected to expand at a robust CAGR of 6.8% during the forecast period. The prevalence and severity of dental decay has waned over the past few years. Dental care needs have engendered a significant shift in the nature of dental care services- from conventional restorative care to cosmetic and preventive services.

Ornithine Transcarbamylase Deficiency Treatment Market- Ornithine transcarbamylase (OTC) deficiency is a genetic disorder arising due to inadequate levels of OTC, an enzyme that helps decompose and eliminate unwanted nitrogen from the human body. This process is also known as the urea cycle.

Medical Holography Market- The medical holography market was valued at US$ 447.8 Million in 2019 and is projected to reach a value pool US$ 3.5 Billion by 2026, registering a stellar CAGR of 34.0% throughout the forecast period. This is in response to increasing demand for medical holography imaging.

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Spectrometry Market will grow at a CAGR of 6.4 % by 2028, Owing to Rising Demand from the Pharmaceutical and Biotechnology Industries; Fact. MR...

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Digital Genome Engineering Market Size and Industry Analysis 2022 : Biotechnology Revolution to Boom the Digital Genome Business with Latest Research…

Posted: June 13, 2022 at 2:19 am

Digital Genome Engineering Market Size and Share is Estimated to Growth by US$ 60.35 at CAGR 11.1% during Forecast period 2028

This press release was orginally distributed by SBWire

Londan, United kingdom (SBWIRE) 06/06/2022 Digital Genome Engineering is a comprehensive digital set of genetic material that occurs in a cell or an organism. It allows experts to get a nearer look at genetic disorders, with instant access to sequence data and tools to resolve unending custom queries. Thus, the aforementioned parameters will augment the market growth. Digital Genome Engineering deals with genes & their functions and assists in finding causes behind chronic disorders and also to resolve them. This technology has sparked a revolt in invention-centred research & systems biology to expedite insight of the most complex genetic systems.

The global Digital Genome Engineering market research report provides an in-depth analysis of the existing and future state of the industry. The study comprises all market data and is based on extensive primary and secondary research. Statistics by type, industry, channel, and other parameters are included in the analysis, as well as market volume and value for each category. The coronavirus pandemic has an influence on the global economy. Several market conditions have shifted. The market is fast evolving, according to the study report, and its influence is being studied both now and in the future.

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The study offers exact figures for the industry's market size, share, production capacity, demand, and growth for the anticipated year. This is the most recent market effect analysis research for COVID-19. The Digital Genome Engineering market research looks at the market's top businesses, distributors, and the entire structure of the industrial chain. It also evaluates the aspects and criteria that may have an impact on market expansion.

The Major Key Company Profiles included in Digital Genome Engineering market study are:

-Abbott Laboratories-Agilent Technologies Inc-Becton, Dickinson and Company-Biomerieux SA-Bio-rad Laboratories Inc-Roche-Illumina Inc-Inscripta-Oxford Nanopore Technologies Ltd-Pacific Bioscience-Perkinelmer Inc-Qiagen NV-Regeneron Pharmaceuticals-Thermo Fisher Scientific Inc

Market Segmentation

The research looks on the industry's growth goals, cost awareness, and manufacturing procedures. Market segmentation by product type, application, end-user, and geography is discussed in the Digital Genome Engineering research report. A basic industry overview, as well as categorization, definition, and, as a result, the supply and demand chain structure, are included in the market study. Global research includes global marketing data, competitive climate surveys, growth rates, and critical development status information.

Russia-Ukraine War Impact on Digital Genome Engineering Market

The impact of the Russia-Ukraine conflict on the worldwide market is covered in the research study. While tensions between Russia and Ukraine have been increasing for years, the present military action heightens fears of a long-term conflict within Ukraine, as well as market and global economic implications. The market research report covers whether this ongoing conflict is making any of on target market or not.

The Digital Genome Engineering Market Segments and Sub-Segments are Listed Below:

Segmentation by type:

-Miss Target Assessment-Target Efficiency Prediction-Typing Efficiency Prediction-Measurement of Editing Efficiency of Target Genome-Other

Segmentation by application:

-Hospital-Research Institute-Institutions of Higher Learning

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Regional Analysis

From production and consumer ratios to market size and market share, import and export ratios, supply and demand, consumer demand ratios, technological advancements, research and development, infrastructure development, economic growth, and a strong market presence in every region, research covers everything. Geographic study will assist players in discovering profitable markets where they may capitalize on fresh opportunities. The Digital Genome Engineering market is divided into five regions: North America, Latin America, Europe, Asia Pacific, and the Rest of the World.

Competitive Outlook

The research report covers financial conditions, global positioning, product portfolios, income and gross profit margins, as well as technology and research breakthroughs. The Digital Genome Engineering market research focuses on the industry's most notable acquisitions, collaborations, and product launches. To provide deeper insights into key players, the study report incorporates modern research approaches such as SWOT and Porter's Five Forces analysis. The study provides a comprehensive assessment of the global competitive climate as well as critical insights into the major competitors and their expansion plans.

Table of Contents Major Key Points

1 Scope of the Report2 Executive Summary3 Digital Genome Engineering Market Size by Player4 Digital Genome Engineering by Regions5 Americas6 APAC7 Europe8 Middle East & Africa9 Market Drivers, Challenges and Trends10 Global Digital Genome Engineering Market Forecast11 Key Players Analysis12 Research Findings and Conclusion

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Intelligence Market Report includes a comprehensive rundown of statistical surveying reports from many distributers around the world. We brag an information base traversing basically every market classification and a much more complete assortment of statistical surveying reports under these classifications and sub-classifications.

Intelligence Market Report offers premium reformist factual looking over, statistical surveying reports, investigation and gauge information for businesses and governments all throughout the planet.

For more information on this press release visit: http://www.sbwire.com/press-releases/digital-genome-engineering-market-size-and-industry-analysis-2022-biotechnology-revolution-to-boom-the-digital-genome-business-with-latest-research-development-1358631.htm

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Digital Genome Engineering Market Size and Industry Analysis 2022 : Biotechnology Revolution to Boom the Digital Genome Business with Latest Research...

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Research-based biopharmaceutical industry on the TRIPS waiver discussions at WTO Ministerial Conference (MC12) – IFPMA

Posted: June 13, 2022 at 2:19 am

Published on: 09 June 2022

The associations representing the global research-based biopharmaceutical industry reaffirm that weakening the intellectual property (IP) framework as proposed in the Quad compromise is unnecessary and harmful to innovation. An IP waiver does not address inequitable access to COVID-19 vaccines and will put global health security at risk. It will undermine innovation and industrys ability to partner, invest at risk, and respond quickly to future pandemics.

Industry has worked around the clock, taking a business NOT as usual approach to tackling the COVID-19 pandemic, focusing significant resources to push the boundaries of science, develop workable solutions and secure the capacity to scale up manufacturing once solutions were found, while at the same time ensuring patients would continue to receive existing medicines. This resulted in the fastest ever vaccine development and authorization (in just 326 days).

Despite these strides in science and manufacturing, for the past 18 months, the World Trade Organization (WTO) Member States have been discussing various ways to weaken the IP framework. To this day, there is no evidence that IP has been a barrier to COVID-19 vaccine production or access, nor acknowledgment of the critical role IP has played in the research, development, and production of novel, safe, and effective vaccines and therapeutics.

From the outset of the pandemic, industry knew that it would need to scale up quickly, seeking to build capacity before approvals were granted, partnering wherever possible, all while pledging to not compromise on quality, safety and efficacy[1]. To date, industry has entered into 381 partnerships for COVID-19 vaccines and 150 for COVID-19 therapeutics, of which over 88% and 79%, respectively, involve technology transfer. IP protections allowed for years of research and investment at risk to bear fruit and today we have 11 vaccines with WHO Emergency Use Listing and 36 therapeutics approved across the world.[2] Biopharmaceutical companies continue to research and invest. Academia and industry have 659 (192 in clinical phase) vaccine candidates and 1,706 (885 in clinical phase) therapeutic candidates in the pipeline.[3]

By May 2021, less than six months after the first vaccine authorization, monthly production output was close to a billion vaccine doses;[4] enough to vaccinate the world if countries were willing and able to share. At that time, industry called on governments to remove trade restrictions, share doses and prepare health systems to roll out vaccinations (5 steps to urgently advance COVID-19 vaccine equity), a message repeated in March 2022 when critical bottlenecks in vaccine delivery and administration, often linked to weak healthcare systems, were becoming even more evident (Three Priorities to Urgently Increase COVID-19 Vaccine Access). Today, it is universally recognized that COVID-19 vaccine supply is not the barrier to access with global vaccine production capacity vastly exceeding demand and doses needed to inoculate and provide boosters to the world.

During this pandemic, there have been many lessons learned that can improve equitable access for future pandemics.[5] We must redouble our collective efforts to achieve health equity, while ensuring health systems and delivery infrastructure are strengthened. COVID-19 vaccines and treatments were only possible because of decades-long investment in research and an enabling IP framework that encouraged swift, voluntary partnerships across the private, public, and academic sectors.

The TRIPS waiver discussion lacks evidence and the IP framework has fallen victim of political posturing. This week, as the WTO meets for the 12th Ministerial Conference, leaders must keep in mind that weakening the IP framework will jeopardize global health security. A much better approach is to focus on the real challenges to COVID-19 vaccine access including removing trade barriers, addressing distribution challenges, strengthening healthcare systems, and partnering to drive innovation and access.

Contact:

Guilherme Cintra, Director, Innovation Policy: g.cintra@ifpma.org

Komal Kalha, Associate Director, Intellectual Property & Trade Policy: k.kalha@ifpma.org

Abigail Jones, Director, Communications: a.jones@ifpma.org

###

About

The Association of the British Pharmaceutical Industry (ABPI) exists to make the UK the best place in the world to research, develop and use new medicines. We represent companies of all sizes who invest in discovering the medicines of the future.Our members supply cutting edge treatments that improve and save the lives of millions of people. We work in partnership with Government and the NHS so patients can get new treatments faster and the NHS can plan how much it spends on medicines.Every day, we partner with organisations in the life sciences community and beyond to transform lives across the UK.

TheBiotechnologyInnovationOrganization(BIO) is the worlds largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations.

The European Federation of Pharmaceutical Industries and Associations(EFPIA) represents the biopharmaceutical industry operating in Europe.Through its direct membership of 36 national associations, 39 leading pharmaceutical companies and agrowingnumber of small and medium-sized enterprises (SMEs), EFPIAs mission is to create a collaborative environment that enables our members to innovate, discover, develop and deliver new therapies and vaccines for people across Europe, as well as contribute to the European economy.

Innovative Medicines Canada is the national association representing the voice of Canadas innovative pharmaceutical industry. The association advocates for policies that enable the discovery, development, and delivery of innovative medicines and vaccines to improve the lives of all Canadians and supports the members commitment to being a valued partner in the Canadian healthcare system. The association represents 49 companies who invest nearly $1.4 billion in R&D annually, fueling Canadas knowledge-based economy, while contributing $8 billion to Canadas economy. Guided by the Code of Ethical Practices, all members work with governments, private payers, healthcare professionals, and stakeholders in a highly ethical manner.

The International Council of Biotechnology Associations (ICBA) is a coalition of nonprofit, national biotechnology trade associations formed to promote public understanding of, and to advocate for, public policies that support the growth of the innovative biotechnology industries. The ICBA represents the global voice of the industry in international fora with the goal of promoting continued innovation in the human health, agriculture, and industrial and environmental sectors.

The International Federation of Pharmaceutical Manufacturers and Associations(IFPMA) represents research-based pharmaceutical companies and associations across the globe. The research-based pharmaceutical industrys 2 million employees research, develop and provide medicines and vaccines that improve the life of patients worldwide. Based in Geneva, IFPMA has official relations with the United Nations and contributes industry expertise to help the global health community find solutions that improve global health.

TheJapan Pharmaceutical Manufacturers Association (JPMA)is a voluntary association comprising 73 research-oriented pharmaceutical companies. JPMA has been contributing to advancing global healthcare through the development of innovative ethical drugs, facilitating sound development of the pharmaceutical industry through proactively establishing policies and recommendations in response to globalization and enhancing public understanding of pharmaceuticals.

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the countrys leading innovative biopharmaceutical research companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier and more productive lives. Since 2000, PhRMA member companies have invested more than $1 trillion in the search for new treatments and cures, including $91.1 billion in 2020 alone.

Vaccines Europewas created in 1991 to provide a voice for the vaccine industry in Europe. The group represents vaccine companies of all sizes operating in Europe, and currently includes all the major global innovative and research-based vaccine companies, including small and medium-sized enterprises.

[1] https://www.ifpma.org/wp-content/uploads/2020/09/COVID-19_Vaccine_Maker_Pledge.pdf

[2] https://covid19.trackvaccines.org/agency/who/

[3] https://www.efpia.eu/media/637039/efpia-vaccines-infographic-may-2022-1.pdf

[4] Note: Global monthly production of COVID-19 vaccines reached 0.83 bn doses in May 2021, 1.2 bn doses in June 2021, 1.2 bn doses in July 2021. Source: IFPMA analysis based on Airfinity data, available at: https://science.airfinity.com/

[5] https://www.ifpma.org/wp-content/uploads/2022/05/IFPMA_COVID-19_Pandemic_Lessons_Learned_May_2022.pdf

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Research-based biopharmaceutical industry on the TRIPS waiver discussions at WTO Ministerial Conference (MC12) - IFPMA

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Fasting triggers stem cell regeneration of damaged, old immune system …

Posted: June 13, 2022 at 2:16 am

In the first evidence of a natural intervention triggering stem cell-based regeneration of an organ or system, a study in the June 5 issue of the Cell Stem Cell shows that cycles of prolonged fasting not only protect against immune system damage a major side effect of chemotherapy but also induce immune system regeneration, shifting stem cells from a dormant state to a state of self-renewal.

In both mice and a Phase 1 human clinical trial involving patients receiving chemotherapy, long periods of not eating significantly lowered white blood cell counts. In mice, fasting cycles then flipped a regenerative switch, changing the signaling pathways for hematopoietic stem cells, which are responsible for the generation of blood and immune systems, the research showed.

The study has major implications for healthier aging, in which immune system decline contributes to increased susceptibility to disease as people age. By outlining how prolonged fasting cycles periods of no food for two to four days at a time over the course of six months kill older and damaged immune cells and generate new ones, the research also has implications for chemotherapy tolerance and for those with a wide range of immune system deficiencies, including autoimmunity disorders.

We could not predict that prolonged fasting would have such a remarkable effect in promoting stem cell-based regeneration of the hematopoietic system, said corresponding author Valter Longo, Edna M. Jones Professor of Gerontology and the Biological Sciences at the USC Davis School of Gerontologyand director of the USC Longevity Institute. Longo has a joint appointment at the USC Dornsife College of Letters, Arts and Sciences.

When you starve, the system tries to save energy, and one of the things it can do to save energy is to recycle a lot of the immune cells that are not needed, especially those that may be damaged, Longo said. What we started noticing in both our human work and animal work is that the white blood cell count goes down with prolonged fasting. Then when you re-feed, the blood cells come back. So we started thinking, well, where does it come from?

Prolonged fasting forces the body to use stores of glucose, fat and ketones, but it also breaks down a significant portion of white blood cells. Longo likens the effect to lightening a plane of excess cargo.

During each cycle of fasting, this depletion of white blood cells induces changes that trigger stem cell-based regeneration of new immune system cells. In particular, prolonged fasting reduced the enzyme PKA, an effect previously discovered by the Longo team to extend longevity in simple organisms and which has been linked in other research to the regulation of stem cell self-renewal and pluripotency that is, the potential for one cell to develop into many different cell types. Prolonged fasting also lowered levels of IGF-1, a growth-factor hormone that Longo and others have linked to aging, tumor progression and cancer risk.

PKA is the key gene that needs to shut down in order for these stem cells to switch into regenerative mode. It gives the OK for stem cells to go ahead and begin proliferating and rebuild the entire system, explained Longo, noting the potential of clinical applications that mimic the effects of prolonged fasting to rejuvenate the immune system. And the good news is that the body got rid of the parts of the system that might be damaged or old, the inefficient parts, during the fasting. Now, if you start with a system heavily damaged by chemotherapy or aging, fasting cycles can generate, literally, a new immune system.

Prolonged fasting also protected against toxicity in a pilot clinical trial in which a small group of patients fasted for a 72-hour period prior to chemotherapy, extending Longos influential past research.

While chemotherapy saves lives, it causes significant collateral damage to the immune system. The results of this study suggest that fasting may mitigate some of the harmful effects of chemotherapy, said co-author Tanya Dorff, assistant professor of clinical medicine at the USC Norris Comprehensive Cancer Center and Hospital. More clinical studies are needed, and any such dietary intervention should be undertaken only under the guidance of a physician.

We are investigating the possibility that these effects are applicable to many different systems and organs, not just the immune system, said Longo, whose lab is in the process of conducting further research on controlled dietary interventions and stem cell regeneration in both animal and clinical studies.

The study was supported by the National Institute of Aging of the National Institutes of Health (grant numbers AG20642, AG025135, P01AG34906). The clinical trial was supported by the V Foundation and the National Cancer Institute of the National Institutes of Health (P30CA014089).

Chia Wei-Cheng of USC Davis was first author of the study. Gregor Adams, Xiaoying Zhou and Ben Lam of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC; Laura Perin and Stefano Da Sacco of the Saban Research Institute at Childrens Hospital Los Angeles; Min Wei of USC Davis; Mario Mirisola of the University of Palermo; Dorff and David Quinn of the Keck School of Medicine of USC; and John Kopchick of Ohio University were co-authors of the study.

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Quizartinib Plus Chemotherapy Significantly Improved Overall Survival Compared to Chemotherapy in Patients with Newly Diagnosed FLT3-ITD Positive…

Posted: June 13, 2022 at 2:15 am

TOKYO & BASKING RIDGE, N.J.--(BUSINESS WIRE)--Positive results from the global pivotal QuANTUM-First phase 3 trial of Daiichi Sankyos (TSE:5468) quizartinib combined with standard induction and consolidation chemotherapy and then continued as a single agent demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) in adult patients aged 18-75 with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML) compared to standard chemotherapy alone. The data were featured as part of the press program and presented during the Presidential Symposium (#S100) at the European Hematology Association (#EHA2022) Congress.

AML is one of the most common leukemias in adults with an estimated five-year survival rate of approximately 30.5%.1,2 Of all newly diagnosed cases of AML, 25% carry the FLT3-ITD gene mutation, which is associated with particularly unfavorable prognosis including increased risk of relapse and shorter overall survival.3

Quizartinib combined with standard induction and consolidation chemotherapy and then continued as a single agent demonstrated a 22.4% reduction in the risk of death compared to standard chemotherapy alone (HR = 0.776 [95% CI: 0.615-0.979; 2-sided p=.0324]) in patients with newly diagnosed FLT3-ITD positive AML. After a median follow-up of 39.2 months, median OS was more than double at 31.9 months for patients receiving quizartinib (95% CI: 21.0-NE) compared to 15.1 months for patients receiving chemotherapy (95% CI: 13.2-26.2).

The safety of quizartinib combined with intensive chemotherapy and as continuation monotherapy in QuANTUM-First was generally manageable, with no new safety signals observed. Rates of grade 3 or higher treatment emergent adverse events (TEAEs) were similar for both study groups and the most common grade 3 or higher TEAEs occurring in 10% of patients were febrile neutropenia (43.4% quizartinib; 41.0% placebo), neutropenia (18% quizartinib; 8.6% placebo), hypokalemia (18.9% quizartinib; 16.4% placebo) and pneumonia (11.7% quizartinib; 12.7% placebo). Rates of TEAEs associated with fatal outcomes were 11.3% for quizartinib versus 9.7% for chemotherapy alone and were mainly due to infections.

QTcF > 500 ms occurred in 2.3% of patients receiving quizartinib and 0.8% of patients discontinued quizartinib due to QT prolongation. Ventricular arrhythmia events with quizartinib were uncommon. Two (0.8%) patients experienced cardiac arrest with recorded ventricular fibrillation on ECG (one with fatal outcome) both in the setting of severe hypokalemia.

The QuANTUM-First results show that adding quizartinib to standard chemotherapy significantly improved overall survival in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia, said Harry P. Erba, MD, PhD, Instructor, Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke Cancer Institute. There is great interest in the increased use of targeted therapies to improve outcomes for patients with AML, particularly those with the FLT3-ITD subtype, which is one of the most common, aggressive and difficult-to-treat.

We are proud that another one of our medicines has demonstrated a significant survival advantage, as our goal is to leverage innovative science to change the way cancer is treated, said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. Adding targeted treatment with quizartinib, a potent and selective FLT3 inhibitor, to standard chemotherapy resulted in a doubling of median overall survival in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia compared to standard chemotherapy alone. Based on these positive QuANTUM-First results, we have initiated global regulatory filings in order to bring quizartinib to patients as quickly as possible.

The OS improvement with quizartinib was also supported by a sensitivity analysis censoring for the effect of allogenic hematopoietic stem cell transplant (HSCT) (HR = 0.752; [95% CI: 0.562-1.008]).

Additional secondary and exploratory analyses provide further understanding and some supporting evidence for improved OS in patients receiving quizartinib combined with chemotherapy in the trial.

The primary event-free survival (EFS) analysis (with induction treatment failure (ITF) defined as not achieving complete remission (CR) by day 42 of the last induction cycle), did not show a statistically significant difference between the two study arms; two pre-specified sensitivity analyses on EFS (the first one defining ITF as not achieving CR by the end of induction; the second one defining ITF as having not achieved composite complete remission (CRc) by the end of induction) showed HR = 0.818 [95% CI: 0.669, 0.999] and HR = 0.729 [95% CI: 0.592-0.897], respectively.

The CRc rate was numerically higher for patients receiving quizartinib compared to chemotherapy alone (71.6% versus 64.9%), and rates of CR were similar for the two study arms (54.9% and 55.4%). The median duration of CR was 38.6 months for quizartinib (95% CI: 21.9-NE) and 12.4 months for chemotherapy (95% CI: 8.8-22.7).

The median relapse-free survival (RFS) for patients who achieved CR was 39.3 months for quizartinib and 13.6 months for placebo, representing a 38.7% relative risk reduction of relapse or death (HR = 0.613 [95% CI: 0.444-0.845]).

Summary of QuANTUM-First Results for OS

Efficacy Measure*

Quizartinib +ChemotherapyN=268

Placebo +ChemotherapyN=271

HR(95% CI)

OS Primary Analysis

Median OS (95% CI)

31.9 months

(21.0 NE)

15.1 months

(13.2 26.2)

HR = 0.776

(0.615 0.979)

2-sided p = 0.0324

*A hierarchical testing procedure was used to test the primary endpoint OS, followed by EFS, CR and CRc. Formal statistical testing was stopped after EFS as its result was not statistically significant.Data cut-off: August 13, 2021Abbreviations: HR = Hazard ratio; NE = not estimable; OS = overall survival

About QuANTUM-First

QuANTUM-First is a randomized, double-blind, placebo-controlled global phase 3 study evaluating quizartinib in combination with standard induction and consolidation chemotherapy and then as continued single agent therapy in adult patients (aged 18-75) with newly diagnosed FLT3-ITD positive AML. Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo combined with anthracycline- and cytarabine-based regimens. Eligible patients, including those who underwent allogeneic HSCT, continued with single agent quizartinib or placebo for up to 36 cycles.

The primary study endpoint was OS. Secondary endpoints include EFS, post-induction rates of CR and CRc, and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, also were evaluated. QuANTUM-First enrolled 539 patients at 193 study sites across Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About Acute Myeloid Leukemia (AML)

More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.4 AML is one of the most common types of leukemia in adults, representing about one-third of all cases, and the average age of diagnosis is 68 years old.1 The five-year survival rate for AML is 30.5%, the lowest by far among the major leukemia subtypes, and is 9.4% for patients aged 65 and older.5,6,7 The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients.8 The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes.9

About FLT3-ITD

FLT3 (FMS-like tyrosine kinase 3) is a tyrosine kinase receptor protein normally expressed by hematopoietic stem cells that plays an important role in cell development, promoting cell survival, growth and differentiation through various signaling pathways.3 Mutations of the FLT3 gene, which occur in approximately 30% of AML patients, can drive oncogenic signaling.3 FLT3-ITD (internal tandem duplication) is the most common type of FLT3 mutation in AML, occurring in about 25% of all newly diagnosed patients, and is associated with increased risk of relapse and shorter overall survival.3

About Quizartinib

Quizartinib is an oral, highly potent and selective type II FLT3 inhibitor currently in clinical development for the treatment of FLT3-ITD positive AML.3 In addition to QuANTUM-First, the quizartinib development program includes a phase 1/2 trial in pediatric and young adult patients with relapsed/refractory FLT3-ITD AML in Europe and North America. Several phase 1/2 combination studies with quizartinib are also underway at The University of Texas MD Anderson Cancer Center as part of a strategic research collaboration focused on accelerating development of Daiichi Sankyo pipeline therapies for AML.

Quizartinib has received Fast Track Designation from the U.S. Food and Drug Administration for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive, in combination with standard cytarabine and anthracycline induction and cytarabine consolidation. Orphan Drug Designation has been granted to quizartinib for the treatment of AML in Europe, Japan and the U.S.

Quizartinib is currently approved for use in Japan under the brand name VANFLYTA for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan.

About Daiichi Sankyo

Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose to contribute to the enrichment of quality of life around the world. In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an Innovative Global Healthcare Company Contributing to the Sustainable Development of Society. For more information, please visit http://www.daiichisankyo.com.

References

1 American Cancer Society. Key Statistics for Acute Myeloid Leukemia. Updated January 2020.2 National Cancer Institute SEER Program. Cancer Stat Facts: Acute Myeloid Leukemia 3 Daver N et al. Leukemia. (2019) 33:299312.4 Global Cancer Observatory. Population Fact Sheet: World. Updated November 2020.5 National Cancer Institute SEER Program. Cancer Stat Facts: Acute Myeloid Leukemia: 5-Year Survival Rates 6 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival 7 National Cancer Institute SEER Explorer. Acute Myeloid Leukemia: 5-Year Survival Rates by Age 8 Daver et al. Blood Cancer J (2020) 10(10):1079 Short et al. Cancer Discov. (2020);10:50625.

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Quizartinib Plus Chemotherapy Significantly Improved Overall Survival Compared to Chemotherapy in Patients with Newly Diagnosed FLT3-ITD Positive...

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Pediatric Urologist Dr. Anthony Atala to Receive 2022 Jacobson Innovation Award of the American College of Surgeons for Pioneering Work in…

Posted: June 13, 2022 at 2:14 am

Newswise CHICAGO (June 10, 2022): Anthony Atala, MD, FACS, Winston-Salem, North Carolina, will be presented with the 2022 Jacobson Innovation Award of the American College of Surgeons (ACS) at a dinner held in his honor this evening in Chicago. He is currently the George Link, Jr. Professor and Director of the Wake Forest Institute for Regenerative Medicine (WFIRM) and the W. H. Boyce Professor and Chair of Urology at the Wake Forest University School of Medicine.

The international surgical award from the ACS honors living surgeons who are innovators of a new development or technique in any field of surgery. It is made possible through a gift from Julius H. Jacobson II, MD, FACS, and his wife Joan. Dr. Jacobson is a general vascular surgeon known for his pioneering work in the development of microsurgery.

Dr. Atala is a pediatric urologist, researcher, professor, and mentor who is renowned for developing foundational principles for regenerative medicine research, which holds great promise for people who require tissue substitution and reconstruction. Dr. Atala and his team successfully implanted the worlds first laboratory grown bladder in 1999.

Dr. Atalas remarkable work has expanded, and today, WFIRM is a leader in translating scientific discovery into regenerative medicine clinical therapies. He currently leads an interdisciplinary team of more than 450 researchers and physicians. Beyond many other world firsts, WFIRM has also developed 15 clinically used technology-based applications, including muscle, urethra, cartilage, reproductive tissues, and skin. Currently, the Institute is working on more than 40 tissues and organs.

Through Dr. Atala's vision, ingenuity, and leadership, the WFIRM team has developed specialized 3-D printers to engineer tissues. This work is accomplished by using cells to create various tissues and organs, including miniature organs called organoids to create body-on-a-chip systems. Dr. Atala and his team also discovered a stem cell population derived from both the amniotic fluid and the placenta, which are currently being used for clinically relevant research applications.

Dr. Atala's theory is that every cell within the human body should be capable of regeneration. What reproduces naturally inside the body should also have the same capabilities of reproduction outside of the body. According to Dr. Atala, the key benefit to the approach of cell and tissue regeneration is that a patient will not reject their own cells or tissue, which is always a concern related to traditional organ match transplantation.

Honors and awards Dr. Atalas innovative work has been recognized as one of Time magazine's Top 10 Medical Breakthroughs in 2007, Smithsonian's 2010 Top Science Story of the Year, and U.S. News & World Report's honor as one of 14 top Pioneers of Medical Progress in the 21st Century. He has been named by Scientific American as one of the world's most influential people in biotechnology, by Life Sciences Intellectual Property Review as one of 50 Key Influencers in the Life Sciences Intellectual Property arena, and by Nature Biotechnology as one of the top 10 Translational Researchers in the World.

Dr. Atala was elected to the Institute of Medicine of the National Academies of Sciences (now the National Academy of Medicine) in 2011 and inducted into the American Institute for Medical and Biological Engineering. In 2014, he was inducted into the National Academy of Inventors as a Charter Fellow and has been a strong and thoughtful contributor to the ACS Surgical Forum and Surgical Research Committee. He presented the prestigious Martin Memorial Named Lecture during the ACS Clinical Congress in 2010 entitled, Regenerative Medicine: New Approaches to Health Care.

Other honors include being the recipient of the U.S. Congress-funded Christopher Columbus Foundation Award, which is bestowed on a living American that currently is working on a discovery that will significantly affect society; the World Technology Award in Health and Medicine for achieving significant and lasting progress; the Edison Science/Medical Award; and the Smithsonian Ingenuity Award.

A national leader in regenerative medicine Throughout his distinguished career, Dr. Atala has led or served on several national professional and government committees, including the National Institutes of Health Working Group on Cells and Developmental Biology, the National Institutes of Health Bioengineering Consortium, and the National Cancer Institute's Advisory Board. He is a founder of the Tissue Engineering Society, the Regenerative Medicine Society, the Regenerative Medicine Foundation, the Alliance for Regenerative Medicine, the Regenerative Medicine Development Organization, the Regenerative Medicine Manufacturing Society, and the Regenerative Medicine Manufacturing Consortium.

A prolific author and inventorDr. Atala is the editor in chief of Stem Cells-Translational Medicine and BioPrinting. He is an author or coauthor of more than 800 journal articles and has applied for or received over 250 national and international patents.

# # #

About Anthony Atala, MD, FACS

Dr. Atala was born in Lima, Peru, and moved to the United States with his family when he was a young boy. He earned a Bachelor of Arts degree from the University of Miami before attending medical school at the University of Louisville, where he also completed his surgical residency training. Near the end of his residency, he applied for a pediatric urology fellowship at Boston Children's Hospital, which was transitioning from a one-year to a two-year program to include a year of research prior to the clinical year. He embarked on a fellowship there in its new form with encouragement from Alan B. Retik, MD, FACS, founder of Boston Childrens first department of urology. Dr. Atala arrived in Boston and began attending seminars, which led him to explore whether uroepithelial cells could be grown and expanded ex vivo, comparable to skin. This additional year of research sparked what has become his career of transformational research, discovery, and innovation with his work focused on growing human cells, tissues, and organs.

Dr. Atala spent the first portion of his academic career at Harvard Medical School before being recruited in 2004 as professor and chair of the department of urology at Wake Forest School of Medicine. After moving his laboratory from Boston, he became the founding Director of the Wake Forest Institute for Regenerative Medicine, where his research and work has produced extraordinary results for nearly two decades.

About the American College of Surgeons The American College of Surgeons is a scientific and educational organization of surgeons that was founded in 1913 to raise the standards of surgical practice and improve the quality of care for all surgical patients. The College is dedicated to the ethical and competent practice of surgery. Its achievements have significantly influenced the course of scientific surgery in America and have established it as an important advocate for all surgical patients. The College has more than 84,000 members and is the largest organization of surgeons in the world. "FACS" designates that a surgeon is a Fellow of the American College of Surgeons.

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How psilocybin, the psychedelic in mushrooms, may rewire the brain to ease depression, anxiety – WYFF4 Greenville

Posted: June 13, 2022 at 2:14 am

Shrooms, Alice, tweezes, mushies, hongos, pizza toppings, magic mushrooms -- everyday lingo for psychedelic mushrooms seems to grow with each generation. Yet leading mycologist Paul Stamets believes it's time for fans of psilocybin mushrooms to leave such childish slang behind."Let's be adults about this. These are no longer 'shrooms.' These are no longer party drugs for young people," Stamets told CNN. "Psilocybin mushrooms are non-addictive, life-changing substances."Small clinical trials that have shown that one or two doses of psylocibin, given in a therapeutic setting, can make dramatic and long-lasting changes in people suffering from treatment-resistant major depressive disorder, which typically does not respond to traditional antidepressants.Based on this research, the U.S. Food and Drug Administration has described psilocybin as a breakthrough medicine, "which is phenomenal," Stamets said.Psilocybin, which the intestines convert into psilocin, a chemical with psychoactive properties, is also showing promise in combating cluster headaches, anxiety, anorexia, obsessive-compulsive disorder and various forms of substance abuse."The data are strong from depression to PTSD to cluster headaches, which is one of the most painful conditions I'm aware of," said neurologist Richard Isaacson, director of the Alzheimer's Prevention Clinic in the Center for Brain Health at Florida Atlantic University."I'm excited about the future of psychedelics because of the relatively good safety profile and because these agents can now be studied in rigorous double-blinded clinical trials," Isaacson said. "Then we can move from anecdotal reports of 'I tripped on this and felt better' to 'Try this and you will be statistically, significantly better.'"Your brain on mushroomsClassic psychedelics such as psilocybin and LSD enter the brain via the same receptors as serotonin, the body's "feel good" hormone. Serotonin helps control body functions such as sleep, sexual desire and psychological states such as satisfaction, happiness and optimism.People with depression or anxiety often have low levels of serotonin, as do people with post-traumatic stress disorder, cluster headaches, anorexia, smoking addiction and substance abuse. Treatment typically involves selective serotonin reuptake inhibitors, or SSRIs, which boost levels of serotonin available to brain cells. Yet it can take weeks for improvement to occur, experts say, if the drugs even work at all.With psychedelics such as psilocybin and LSD, however, scientists can see changes in brain neuron connectivity in the lab "within 30 minutes," said pharmacologist Brian Roth, a professor of psychiatry and pharmacology at the University of North Carolina at Chapel Hill."One of the most interesting things we've learned about the classic psychedelics is that they have a dramatic effect on the way brain systems synchronize, or move and groove together," said Matthew Johnson, a professor in psychedelics and consciousness at Johns Hopkins Medicine."When someone's on psilocybin, we see an overall increase in connectivity between areas of the brain that don't normally communicate well," Johnson said. "You also see the opposite of that -- local networks in the brain that normally interact with each other quite a bit suddenly communicate less."It creates a "very, very disorganized brain," ultimately breaking down normal boundaries between the auditory, visual, executive and sense-of-self sections of the mind -- thus creating a state of "altered consciousness," said David Nutt, director of the Neuropsychopharmacology Unit in the Division of Brain Sciences at Imperial College London.And it's that disorganization that is ultimately therapeutic, according to Nutt: "Depressed people are continually self-critical, and they keep ruminating, going over and over the same negative, anxious or fearful thoughts."Psychedelics disrupt that, which is why people can suddenly see a way out of their depression during the trip," he added. "Critical thoughts are easier to control, and thinking is more flexible. That's why the drug is an effective treatment for depression."The growth of brain cellsThere's more. Researchers say psychedelic drugs actually help neurons in the brain sprout new dendrites, which look like branches on a tree, to increase communication between cells."These drugs can increase neuronal outgrowth, they can increase this branching of neurons, they can increase synapses. That's called neuroplasticity," Nutt said.That's different from neurogenesis, which is the development of brand new brain cells, typically from stem cells in the body. The growth of dendrites helps build and then solidify new circuits in the brain, allowing us to, for example, lay down more positive pathways as we practice gratitude."Now our current thinking is this neuronal outgrowth probably doesn't contribute to the increased connectivity in the brain, but it almost certainly helps people who have insights into their depression while on psilocybin maintain those insights," Nutt said."You shake up the brain, you see things in a more positive way, and then you lay down those positive circuits with the neuroplasticity," he added. "It's a double whammy."Interestingly, SSRIs also increase neuroplasticity, a fact that science has known for some time. But in a 2022 double-blind phase 2 randomized controlled trial comparing psylocibin to escitalopram, a traditional SSRI, Nutt found the latter didn't spark the same magic."The SSRI did not increase brain connectivity, and it actually did not improve well-being as much as psilocybin," Nutt said. "Now for the first time you've got the brain science lining up with what patients say after a trip: 'I feel more connected. I can think more freely. I can escape from negative thoughts, and I don't get trapped in them.' "Taking a psychedelic doesn't work for everyone, Johnson stressed, "but when it works really well it's like, 'Oh my god, it's a cure for PTSD or for depression.' If people really have changed the way their brain is automatically hardwired to respond to triggers for anxiety, depression, smoking -- that's a real thing."How long do results last? In studies where patients were given just one dose of a psychedelic "a couple of people were better eight years later, but for the majority of those with chronic depression it creeps back after four or five months," Nutt said."What we do with those people is unknown," he added. "One possibility is to give another dose of the psychedelic -- we don't know if that would work or not, but it might. Or we could put them on an SSRI as soon as they've got their mood improved and see if that can hold the depression at bay."There are all sorts of ways we could try to address that question," Nutt said, "but we just don't know the answer yet."What about microdosing?Stamets, who over the last 40 years has discovered four new species of psychedelic mushrooms and written seven books on the topic, said he believes microdosing is a solution. That's the practice of taking tiny amounts of a psilocybin mushroom several times a week to maintain brain health and a creative perspective on life.A typical microdose is .01 to .03 grams of dried psilocybin mushrooms, as compared to the 25-milligram pill of psilocybin that creates the full-blown psychedelic experience.Stamets practices microdosing, and has focused on a process called "stacking," in which a microdose of mushrooms is taken with additional substances believed to boost the fungi's benefits. His famous "Stamets Stack" includes niacin, or vitamin B3, and the mycelium, or rootlike structure, of an unusual mushroom called Lion's mane.Surveys of microdosers obtained on his website have shown significantly positive benefits from the practice of taking small doses."These are self-reported citizen scientists projects and we have now around 14,000 people in our app where you register yourself and report your microdose," Stamets told an audience at the 2022 Life Itself conference, a health and wellness event presented in partnership with CNN."I'm going to say something provocative, but I believe it to my core: Psilocybin makes nicer people," Stamets told the audience. "Psilocybin will make us more intelligent, and better citizens."Video below: Psychedelic drugs may improve your cardiovascular healthScientific studies so far have failed to find any benefits from microdosing, leaving many researchers skeptical. "People like being on it, but that doesn't validate the claims of microdosing," Johnson said. "People like being on a little bit of cocaine, too."Experimental psychologist Harriet de Wit, a professor of psychiatry and behavioral science at the University of Chicago, was excited to study microdosing because it solves a key problem of scientific research in the field -- it's hard to blind people to what they are taking if they begin to trip. Microdosing solves that problem because people don't feel an effect from the tiny dose.De Wit specializes in determining whether a drug's impact is due to the drug or what scientists call the "placebo effect," a positive expectation that can cause improvement without the drug.She published a study in early 2022 that mimicked real-world microdosing of LSD, except neither the participants nor researchers knew what was in the pills the subjects took."We measured all kinds of different behavioral and psychological responses, and the only thing we saw is that LSD at very low doses produced some stimulant-like effects at first, which then faded," de Wit said.The placebo effect is powerful, she added, which might explain why the few additional studies done on it have also failed to find any positive results."I suspect microdosing may have an effect on mood, and over time it might build up resilience or improve well-being," Nutt said. "But I don't think it will rapidly fragment depression like macrodosing and going on a trip."A need for cautionObviously, not all hallucinogenic experiences are positive, so nearly every study on psychedelic drugs has included therapists trained to intercede if a trip turns bad and maximize the outcome if the trip is good."This is about allowing someone access into deeper access into their own mental processes, with hopefully greater insight. While others might disagree, it does seem very clear that you need therapy to maximize the benefits," Johnson said.There are also side effects from psychedelics that go beyond a bad trip. LSD, mescaline and DMT, which is the active ingredient in ayahuasca tea, can increase blood pressure, heart rate, and body temperature, according to the National Institute on Drug Abuse. Ayahuasca tea can also induce vomiting. LSD can cause tremors, numbness and weakness, while the use of mescaline can lead to uncoordinated movements. People hunting for psychedelic mushrooms can easily mistake a toxic species for one with psilocybin, "leading to unintentional, fatal poisoning."Another issue: Not everyone is a candidate for psychedelic treatment. It won't work on people currently on SSRIs -- the receptors in their brains are already flooded with serotonin. People diagnosed with bipolar disorder or schizophrenia, or who have a family history of psychosis are always screened out of clinical trials, said Frederick Barrett, associate director of the Center for Psychedelic and Consciousness Research at Johns Hopkins."If you have a vulnerability to psychosis, it could be that exposing you to a psychedelic could unmask that psychosis or could lead to a psychotic event," Barnes said.Then there are the thousands of people with mental health concerns who will never agree to undergo a psychedelic trip. For those people, scientists such as Roth are attempting to find an alternative approach. He and his team recently identified the mechanisms by which psychedelics bond to the brain's serotonin receptors and are using the knowledge to identify new compounds."Our hope is that we can use this information to ultimately make drugs that mimic the benefits of psychedelic drugs without the psychedelic experience," Roth said."What if we could give people who are depressed or suffer from PTSD or anxiety or obsessive-compulsive disorder a medication, and they could wake up the next day and be fine without any side effects? That would be transformative."

Shrooms, Alice, tweezes, mushies, hongos, pizza toppings, magic mushrooms -- everyday lingo for psychedelic mushrooms seems to grow with each generation. Yet leading mycologist Paul Stamets believes it's time for fans of psilocybin mushrooms to leave such childish slang behind.

"Let's be adults about this. These are no longer 'shrooms.' These are no longer party drugs for young people," Stamets told CNN. "Psilocybin mushrooms are non-addictive, life-changing substances."

Small clinical trials that have shown that one or two doses of psylocibin, given in a therapeutic setting, can make dramatic and long-lasting changes in people suffering from treatment-resistant major depressive disorder, which typically does not respond to traditional antidepressants.

Based on this research, the U.S. Food and Drug Administration has described psilocybin as a breakthrough medicine, "which is phenomenal," Stamets said.

Psilocybin, which the intestines convert into psilocin, a chemical with psychoactive properties, is also showing promise in combating cluster headaches, anxiety, anorexia, obsessive-compulsive disorder and various forms of substance abuse.

"The data are strong from depression to PTSD to cluster headaches, which is one of the most painful conditions I'm aware of," said neurologist Richard Isaacson, director of the Alzheimer's Prevention Clinic in the Center for Brain Health at Florida Atlantic University.

"I'm excited about the future of psychedelics because of the relatively good safety profile and because these agents can now be studied in rigorous double-blinded clinical trials," Isaacson said. "Then we can move from anecdotal reports of 'I tripped on this and felt better' to 'Try this and you will be statistically, significantly better.'"

Classic psychedelics such as psilocybin and LSD enter the brain via the same receptors as serotonin, the body's "feel good" hormone. Serotonin helps control body functions such as sleep, sexual desire and psychological states such as satisfaction, happiness and optimism.

People with depression or anxiety often have low levels of serotonin, as do people with post-traumatic stress disorder, cluster headaches, anorexia, smoking addiction and substance abuse. Treatment typically involves selective serotonin reuptake inhibitors, or SSRIs, which boost levels of serotonin available to brain cells. Yet it can take weeks for improvement to occur, experts say, if the drugs even work at all.

farmer images

With psychedelics such as psilocybin and LSD, however, scientists can see changes in brain neuron connectivity in the lab "within 30 minutes," said pharmacologist Brian Roth, a professor of psychiatry and pharmacology at the University of North Carolina at Chapel Hill.

"One of the most interesting things we've learned about the classic psychedelics is that they have a dramatic effect on the way brain systems synchronize, or move and groove together," said Matthew Johnson, a professor in psychedelics and consciousness at Johns Hopkins Medicine.

"When someone's on psilocybin, we see an overall increase in connectivity between areas of the brain that don't normally communicate well," Johnson said. "You also see the opposite of that -- local networks in the brain that normally interact with each other quite a bit suddenly communicate less."

It creates a "very, very disorganized brain," ultimately breaking down normal boundaries between the auditory, visual, executive and sense-of-self sections of the mind -- thus creating a state of "altered consciousness," said David Nutt, director of the Neuropsychopharmacology Unit in the Division of Brain Sciences at Imperial College London.

And it's that disorganization that is ultimately therapeutic, according to Nutt: "Depressed people are continually self-critical, and they keep ruminating, going over and over the same negative, anxious or fearful thoughts.

"Psychedelics disrupt that, which is why people can suddenly see a way out of their depression during the trip," he added. "Critical thoughts are easier to control, and thinking is more flexible. That's why the drug is an effective treatment for depression."

There's more. Researchers say psychedelic drugs actually help neurons in the brain sprout new dendrites, which look like branches on a tree, to increase communication between cells.

"These drugs can increase neuronal outgrowth, they can increase this branching of neurons, they can increase synapses. That's called neuroplasticity," Nutt said.

That's different from neurogenesis, which is the development of brand new brain cells, typically from stem cells in the body. The growth of dendrites helps build and then solidify new circuits in the brain, allowing us to, for example, lay down more positive pathways as we practice gratitude.

"Now our current thinking is this neuronal outgrowth probably doesn't contribute to the increased connectivity in the brain, but it almost certainly helps people who have insights into their depression while on psilocybin maintain those insights," Nutt said.

"You shake up the brain, you see things in a more positive way, and then you lay down those positive circuits with the neuroplasticity," he added. "It's a double whammy."

Interestingly, SSRIs also increase neuroplasticity, a fact that science has known for some time. But in a 2022 double-blind phase 2 randomized controlled trial comparing psylocibin to escitalopram, a traditional SSRI, Nutt found the latter didn't spark the same magic.

"The SSRI did not increase brain connectivity, and it actually did not improve well-being as much as psilocybin," Nutt said. "Now for the first time you've got the brain science lining up with what patients say after a trip: 'I feel more connected. I can think more freely. I can escape from negative thoughts, and I don't get trapped in them.' "

Taking a psychedelic doesn't work for everyone, Johnson stressed, "but when it works really well it's like, 'Oh my god, it's a cure for PTSD or for depression.' If people really have changed the way their brain is automatically hardwired to respond to triggers for anxiety, depression, smoking -- that's a real thing."

How long do results last? In studies where patients were given just one dose of a psychedelic "a couple of people were better eight years later, but for the majority of those with chronic depression it creeps back after four or five months," Nutt said.

"What we do with those people is unknown," he added. "One possibility is to give another dose of the psychedelic -- we don't know if that would work or not, but it might. Or we could put them on an SSRI as soon as they've got their mood improved and see if that can hold the depression at bay.

"There are all sorts of ways we could try to address that question," Nutt said, "but we just don't know the answer yet."

Stamets, who over the last 40 years has discovered four new species of psychedelic mushrooms and written seven books on the topic, said he believes microdosing is a solution. That's the practice of taking tiny amounts of a psilocybin mushroom several times a week to maintain brain health and a creative perspective on life.

Courtesy Pamela Kryskow

A typical microdose is .01 to .03 grams of dried psilocybin mushrooms, as compared to the 25-milligram pill of psilocybin that creates the full-blown psychedelic experience.

Stamets practices microdosing, and has focused on a process called "stacking," in which a microdose of mushrooms is taken with additional substances believed to boost the fungi's benefits. His famous "Stamets Stack" includes niacin, or vitamin B3, and the mycelium, or rootlike structure, of an unusual mushroom called Lion's mane.

Surveys of microdosers obtained on his website have shown significantly positive benefits from the practice of taking small doses.

"These are self-reported citizen scientists projects and we have now around 14,000 people in our app where you register yourself and report your microdose," Stamets told an audience at the 2022 Life Itself conference, a health and wellness event presented in partnership with CNN.

"I'm going to say something provocative, but I believe it to my core: Psilocybin makes nicer people," Stamets told the audience. "Psilocybin will make us more intelligent, and better citizens."

Video below: Psychedelic drugs may improve your cardiovascular health

Scientific studies so far have failed to find any benefits from microdosing, leaving many researchers skeptical. "People like being on it, but that doesn't validate the claims of microdosing," Johnson said. "People like being on a little bit of cocaine, too."

Experimental psychologist Harriet de Wit, a professor of psychiatry and behavioral science at the University of Chicago, was excited to study microdosing because it solves a key problem of scientific research in the field -- it's hard to blind people to what they are taking if they begin to trip. Microdosing solves that problem because people don't feel an effect from the tiny dose.

De Wit specializes in determining whether a drug's impact is due to the drug or what scientists call the "placebo effect," a positive expectation that can cause improvement without the drug.

She published a study in early 2022 that mimicked real-world microdosing of LSD, except neither the participants nor researchers knew what was in the pills the subjects took.

"We measured all kinds of different behavioral and psychological responses, and the only thing we saw is that LSD at very low doses produced some stimulant-like effects at first, which then faded," de Wit said.

The placebo effect is powerful, she added, which might explain why the few additional studies done on it have also failed to find any positive results.

"I suspect microdosing may have an effect on mood, and over time it might build up resilience or improve well-being," Nutt said. "But I don't think it will rapidly fragment depression like macrodosing and going on a trip."

Obviously, not all hallucinogenic experiences are positive, so nearly every study on psychedelic drugs has included therapists trained to intercede if a trip turns bad and maximize the outcome if the trip is good.

"This is about allowing someone access into deeper access into their own mental processes, with hopefully greater insight. While others might disagree, it does seem very clear that you need therapy to maximize the benefits," Johnson said.

There are also side effects from psychedelics that go beyond a bad trip. LSD, mescaline and DMT, which is the active ingredient in ayahuasca tea, can increase blood pressure, heart rate, and body temperature, according to the National Institute on Drug Abuse. Ayahuasca tea can also induce vomiting. LSD can cause tremors, numbness and weakness, while the use of mescaline can lead to uncoordinated movements. People hunting for psychedelic mushrooms can easily mistake a toxic species for one with psilocybin, "leading to unintentional, fatal poisoning."

Another issue: Not everyone is a candidate for psychedelic treatment. It won't work on people currently on SSRIs -- the receptors in their brains are already flooded with serotonin. People diagnosed with bipolar disorder or schizophrenia, or who have a family history of psychosis are always screened out of clinical trials, said Frederick Barrett, associate director of the Center for Psychedelic and Consciousness Research at Johns Hopkins.

"If you have a vulnerability to psychosis, it could be that exposing you to a psychedelic could unmask that psychosis or could lead to a psychotic event," Barnes said.

Then there are the thousands of people with mental health concerns who will never agree to undergo a psychedelic trip. For those people, scientists such as Roth are attempting to find an alternative approach. He and his team recently identified the mechanisms by which psychedelics bond to the brain's serotonin receptors and are using the knowledge to identify new compounds.

"Our hope is that we can use this information to ultimately make drugs that mimic the benefits of psychedelic drugs without the psychedelic experience," Roth said.

"What if we could give people who are depressed or suffer from PTSD or anxiety or obsessive-compulsive disorder a medication, and they could wake up the next day and be fine without any side effects? That would be transformative."

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How psilocybin, the psychedelic in mushrooms, may rewire the brain to ease depression, anxiety - WYFF4 Greenville

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Gene therapy to cure leukemia at one-tenth global costs in the works – Business Standard

Posted: June 13, 2022 at 2:13 am

ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price.

We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient, explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.

He claims that the results start showing in around one weeks time. Moreover, this is a one-time treatment that would cost around Rs 20-30 lakh per patient, as compared to Rs 4 crore or so in the US, Purwar adds.

It takes around two weeks to prepare the customised cells to be transfused into the patient, and thereafter this is like a live drug working inside the patients body, he says.

Are there any side-effects of this treatment? Purwar says none like what we witness in chemotherapy, but sometimes there is cytokine release syndrome because of dead cancer cells in the body, which can be managed. So far, we have not seen any patient with an uncontrolled cytokine release syndrome.

The therapy has completed its phase 1 or safety trials, and is now all set to go into phase two trials here. ImmunoACT expects that it would be ready for a market launch in around 18-months time subject to regulatory approvals.

Laurus Labs has picked up 26 per cent stake in this biotech start-up, and its promoters have picked up around 5-6 per cent in personal capacity, informed Laurus Labs CEO Satyanarayana Chava.

He said that as a part of policy, they have decided to pump in 10 per cent of their annual profits into such healthcare start-ups with breakthrough technology.

I feel there is a lot of promise this therapy offers in India, and especially the price is way below the global standards. We have spoken to several oncologists and they are waiting for this tool to be available to them. Several NGOs also work in this space that help patients with treatment costs, Chava said.

In the US, the insurance companies cover immunotherapy costs. In India once this product is available, one would have to work out these nuances.

ImmunoACT is also working on two more cancer products using this technology one for neuroblastoma and another for brain cancer.

Purwar says that the technology has no limitations and can be adapted for various kinds of cancers. At the moment they have set up a GMP facility in Mumbai, which can process H-CAR T -19 for 100 patients a month or around 1,200 a year.

As demand rises, they plan to add capacity at the existing site as well as add sites in new locations. There are approximately 50,000 leukemia patients diagnosed in India every year, and many of them are put on oral drugs and chemotherapy. The success rate of chemotherapy is approximately 60 percent, Purwar adds.

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Gene therapy to cure leukemia at one-tenth global costs in the works - Business Standard

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Gene Therapy Market is Estimated to Reach US$ 5.3 Bn By 2028, Increase in Incidence of Cancer to Drive the Market – BioSpace

Posted: June 13, 2022 at 2:13 am

Wilmington, Delaware, United States: According to the report, the global gene therapy market was valued at US$ 1.3 Bn in 2020 and is projected to expand at a CAGR of 19.8% from 2017 to 2028. Gene therapy is a way of fixing genetic disorders by introducing a normal and healthy gene in place of the defective one in a cell to prevent or cure different types of genetic and chronic disorders for which no cure has been developed. Gene therapy is considered an important mode of treatment, as it helps eliminate the usage of drugs, surgery, or other procedures, which can have side effects. Several clinical research studies are being conducted to develop effective gene therapy products for the treatment of diseases related to ophthalmology, oncology, and ADA-SCID.

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Gene therapy in ophthalmology includes the injection of fully functional genes into the retinal cells in order to allow restoration of normal gene expression. Different gene therapy approaches are being studied in the fields of oncology, such as pro-drug activating suicide gene therapy, anti-angiogenic gene therapy, oncolytic virotherapy, and gene therapy-based immune modulation. The adenosine deaminase (ADA)deficient severe combined immunodeficiency (SCID) is a very rare form of congenital disorder of the immune system.

Promising Therapeutic Outcomes to Drive Market

Gene therapy has considerable potential to eliminate and prevent several genetic disorders and numerous life-threatening disorders, especially cancer, heart diseases, AIDS, cystic fibrosis, and age-related disorders. Gene therapy provides a complete cure to patients affected with genetic disorders, rather than ease symptoms with other therapeutic treatments. The first gene therapy product Gendicine was developed by Shenzhen, China-based SiBiono GeneTech Co. Ltd. in 2003 and was commercialized in 2004 for the treatment of head and neck cancer. Since 2004, more than 30,000 patients have been treated with Gendicine in China. Gendicine has exhibited notable safety results, and when this gene therapy is combined with other treatment forms, such as chemotherapy and radiotherapy, the overall outcome outperforms other standard therapies alone.

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Patients treated with Novartis AGs gene therapy product Kymriah have exhibited a remission rate of 81% with three months of treatment, and all the patients who exhibit a positive response to treatment were not detected with any minimal residual disease. Hence, the overall promising results of gene therapy fuels the demand for gene therapy products.

New Product Approvals & Commercialization to Propel Market

The years 2016, 2017, and 2018 were key milestones in the history of the gene therapy market in the U.S. and Europe because around four gene therapy products have been approved and commercialized. These products are currently in a nascent stage of commercialization, and have exhibited highly positive therapeutic outcomes. Increase in competition among the leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs to gain the first mover advantage is likely to boost the growth of the gene therapy market during the forecast period.

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Increase in Number of Gene Therapy Treatment Centers to Augment Market

New gene therapy product approvals and potential success rates of gene therapy have led to an increased demand for more number of treatment centers in the U.S. and Europe. The companies, after initial success rates of gene therapy, strive to increase the number of treatment centers in order to gain access to a large patient pool requiring gene therapy treatments.

The gene therapy product Kymriah, which was approved for the treatment of patients up to the age of 25 years affected with large B-cell lymphoma, has only 41 treatment centers in the U.S., which limits a large number of patients from receiving gene therapy treatment.

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Promising Product Pipeline and High Investment in R&D

Significant investments in gene-related research & development by various biopharmaceutical companies, governments, and research institutes presents an opportunity to gain the first mover advantage in the gene therapy market. A large number of gene therapy products are under different stages of clinical studies, and the number of gene therapy candidates is projected to rise during the forecast period. High investment in gene therapy research and large number of late stage gene therapy candidates in the oncology domain and other genetic disorders are projected to fuel the demand for gene therapy products during the forecast period.

Competition Landscape

The global cell culture market is consolidated in terms of number of players. Key players in the global market includes Novartis AG, Orchard Therapeutics Limited, Celgene Corporation, Spark Therapeutics, Inc., Gilead Sciences, Inc., Sibiono GeneTech Co. Ltd., and Other prominent players.

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Gene Therapy Market is Estimated to Reach US$ 5.3 Bn By 2028, Increase in Incidence of Cancer to Drive the Market - BioSpace

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