Category Archives: Stem Cell Treatments

Steven Hodi Jr., the i3 Centers other PI, and director of Melanoma Center and the Center for Immuno-Oncology at Dana-Farber, and professor of medicine at Harvard Medical School (HMS), is leading the clinical cancer vaccine trial. He has been at the forefront of developing cancer immunotherapies using immune checkpoint inhibitors, a class of drugs able to re-activate tumor-destroying T cells that are muted in the tumor microenvironment. The funding for this center provides a unique opportunity to unite key investigators for translating fundamental advancements in immunology and biomedical engineering into highly synergistic approaches to improve the treatments for cancer patients, said Hod

Using both in vivo and ex vivo biomaterials-based approaches, the i3 Center aims to boost tumor-specific activities of cytotoxic T cells, by boosting different stages of the normal process by which T cells develop, and acquire anti-cancer activity. T cells normal development starts in the bone marrow where hematopoietic stem cells generate T cell progenitor cells. These migrate to the thymus to differentiate into nave T cells, which then travel further to lymph nodes. There, they encounter cancer-derived antigens presented to them by specialized antigen-presenting cells (APCs) that can activate T cells to recognize and eliminate cancer cells.

In relation to adoptive T cell therapies in which T cells are given to patients to fight their cancers, one team at the i3 Center will be led by Dana-Farber researchers Catherine J. Wu and Jerome Ritz, who along with Mooney, will develop and test biomaterials that can better mimic normal APCs in activating and directing the function of patient-derived T cells outside the human body, prior to their transplantation. Wu is chief of the Division of Stem Cell Transplantation and Cellular Therapies, and Ritz is executive director of the Connell and OReilly Families Cell Manipulation Core Facility at Dana-Farber.

We need to make efforts to enhance the ability of theimmune systemto recognizetumor cells. One directionmylaboratoryis taking makes use of innovative biomaterialsto help us to efficiently expandpolyclonaltumor-specificfunctionally-effectiveT cellsex vivoin a way that can be readily translated to theclinical setting. In our studies, we are currently focusing on melanoma and acute myeloid leukemia, said Wu, whose research interests include understanding the basis of effective human anti-tumor responses, including the identification and targeting of the tumor-specific antigens.

A second project explores the use of DNA origami, biocompatible nanostructures composed of DNA, to create cancer vaccines. DNA origami could provide significant advantages in presenting tumor-specific antigens and immune-enhancing adjuvants to APCs because the concentrations, ratios, and geometries of all components can be modulated with nano-scale precision to determine configurations that are more effective than other vaccination strategies. The project will be run by Wyss Institute Core Faculty member William Shih, Derin Keskin, lead immunologist at Dana-Farbers Translational Immunogenomics Lab, and Mooney.

In a third project, David Scadden, professor at Harvards Department of Stem Cell and Regenerative Biology, will collaborate with Mooney to build on their previous work. They will engineer biomaterials that recreate key features of the normal hematopoietic stem cell niche in the bone marrow. Such implantable biomaterials could help rapidly amplify T cell progenitor cells, and enhance T cell-mediated anti-cancer immunity. Scadden also is the Gerald and Darlene Jordan Professor of Medicine at Harvard University, and co-director of the Harvard Stem Cell Institute.

The i3 Centers investigators anticipate that it will stimulate additional cross-disciplinary concepts and research, due to the culture of continuous interactions, sharing of findings, data and samples between all investigators, as well strong biostatistical expertise provided by Donna Neuberg, a senior biostatistician broadly involved with exploring immune-modulating cancer interventions at the Dana-Farber.

This new i3 Center for cancer immunotherapy innovation really embodies how the Wyss Institute with its unparalleled capabilities in bioengineering and serving as a site for multidisciplinary collaboration, and can liaise with clinicians and researchers at our collaborating institutions to confront major medical problems and bring about transformative change, said Wyss Founding Director Donald Ingber. He is also theJudah Folkman Professor of Vascular Biologyat HMS and the Vascular Biology Program at Boston Childrens Hospital, and Professor of Bioengineering at SEAS.

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NIH-funded i3 Center formed to advance cancer immunotherapy - Harvard Gazette

LEXINGTON, MA / ACCESSWIRE / March 4, 2020 / Keros Therapeutics, Inc., a biotechnology company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet medical need, today announced the close of its $56 million Series C financing, bringing its total venture funding to $78.5 million to date. The Series C financing was led by new investors Foresite Capital, OrbiMed, Cowen Healthcare Investments and Venrock. Certain of Keros' existing investors also participated including Pontifax, Arkin Bio Ventures, Partners Innovation Fund, Global Health Sciences Fund and Medison Pharma.

As part of the Series C financing, Nima Farzan, MBA, an experienced biopharmaceutical executive, and Carl Gordon, Ph.D., CFA, Managing Partner at OrbiMed, will be joining the Board of Directors of Keros.

Proceeds from this financing will be used to advance Keros's pipeline of product candidates through multiple clinical data readouts. KER-050, our lead clinical-stage protein therapeutic product candidate, is currently being developed to correct cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis. KER-047, our lead clinical-stage small molecule product candidate, is being developed for treatment of anemias resulting from high hepcidin levels and for fibrodysplasia ossificans progressiva, or FOP, a rare genetic disease where muscle and connective tissues gradually become replaced by bone.

"The Keros team has a deep understanding of the biology relevant to the role of members of the TGF- family of proteins in the development of blood cells, muscle and bone, and we have leveraged that knowledge to advance two product candidates through Phase 1 clinical trials," said Jasbir S. Seehra, Ph.D., President and CEO of Keros. "This additional funding will allow us to rapidly advance our lead programs through Phase 2 clinical trials to provide proof-of-concept for novel treatments that could potentially address the limitations of current therapeutic approaches in diseases linked to the dysfunction of TGF- signaling pathways."

"We are excited to lead this important financing to advance Keros's lead programs through Phase 2 proof-of-concept studies. We believe Jas and his team are world leaders in the field TGF- biology," said Jim Tananbaum, CEO and Managing Director of Foresite Capital.

"We are thrilled to welcome these highly respected investors and look forward to working with our newest Board members", said Ran Nussbaum, Managing Partner of Pontifax and Chairman of the Keros Board of Directors. "With this financing, Keros is in a strong position to advance its technology centered on the role of members of the TGF- family of proteins and bring new treatments to patients suffering from hematologic and musculoskeletal disorders."

Our Biological Focus

Keros focuses on the role of members of the TGF- family of proteins in the development of blood cells, muscle and bone. Aged and damaged cells are routinely replaced by new cells in normally functioning organs. These new cells are derived from stem cells that have the ability to differentiate into cells with specialized function when appropriate signals are provided to maintain the homeostatic state of the tissue. Members of the TGF- family of proteins, including activins and bone morphogenetic proteins, provide the necessary signals for this process of self-renewal and repair.

Product Candidates

Our lead, clinical-stage protein therapeutic product candidate, KER-050, is an engineered ligand trap comprised of a modified ligand-binding domain of the TGF- receptor known as activin receptor type IIA that is fused to the portion of the human antibody known as the Fc domain. KER-050 is designed to increase red blood cell and platelet production by inhibiting the signaling of a subset of the TGF- family of proteins to promote hematopoiesis, and is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with MDS, and in patients with myelofibrosis.

Our lead, clinical-stage small molecule product candidate, KER-047, is designed to selectively and potently inhibit activin receptor-like kinase-2, or ALK2, a TGF- receptor. We believe that KER-047 has the potential to ameliorate excessive ALK2 signaling, and Keros is developing KER-047 for the treatment of anemia resulting from high hepcidin levels as a direct consequence of elevated ALK2 signaling, including our initial target, iron-refractory iron deficiency anemia, or IRIDA. We are also developing KER-047 as a treatment for FOP, a rare genetic disease resulting from mutations in the ALK2 receptor.

Story continues

About Keros Therapeutics

Keros is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematologic and musculoskeletal disorders with high unmet medical need. We are a leader in understanding the role of the Transforming Growth Factor-Beta family of proteins that is a master regulator of red blood cell and platelet production as well as the growth, repair and maintenance of muscle and bone. Our lead protein therapeutic product candidate, KER-050, is being developed for the treatment of cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and myelofibrosis. Our lead small molecule product candidate, KER-047, is being developed for the treatment of anemia resulting from elevated levels of hepcidin, the key regulator of iron absorption and recycling, as well as for the treatment of fibrodysplasia ossificans progressiva. For more information on Keros Therapeutics, visit http://www.kerostx.com

Keros Therapeutics Investor Contacts:

Julia Balanovajbalanova@soleburytrout.com646 378 2936

SOURCE: Keros Therapeutics

View source version on accesswire.com: https://www.accesswire.com/579058/Keros-Therapeutics-Closes-56-Million-Series-C-Financing-to-Advance-its-Programs-in-Hematologic-and-Musculoskeletal-Disorders

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Keros Therapeutics Closes $56 Million Series C Financing to Advance its Programs in Hematologic and Musculoskeletal Disorders - Yahoo Finance

When I got off the phone with the National President of the Hadassah, the worlds largest Zionist womens organization who had called to invite me to serve on the board of this amazing nonprofit, I was filled with a sense of resolve, renewal and responsibility I was also a bit overwhelmed.

Of course, I knew about the work of Hadassah the creation of two hospitals in Jerusalem, Israel, that were known as bridges to peace in the Middle East and were world-famous for their medical miracles, like stem cell treatments that reversed the effects of MS, and I was eager for my first board meeting. Id served on other boards and I thought I was experienced enough for my new role, but I quickly realized that to truly be effective and not to feel like deadwood on a board, which I detested from my days as a director of a nonprofit, I couldnt go it alone.

Its taken me many years (and many failures) to realize that asking for help is a sign of strength, not of weakness. I wish I could go back in time and convince my younger self this.

Id never had a mentor. Maybe because it didnt occur to me in my youthful ignorance that I needed one, but more likely because as a professional in a male-dominated field, no one had ever offered or felt particularly approachable. The corporate culture I was most familiar with was sink or swim and youd better do it quickly and youd better be tough.

Fortunately for me, by the time I was mature enough to realize I would benefit from a mentor, I was already a dedicated volunteer with my local Hadassah chapter. I was surrounded by smart, compassionate, accomplished women who were typically twenty years my senior. These women were not interested in taking me down but in lifting me up.

One of these women was also on the Hadassah National Board Liz Alpert. Liz is whip-smart, bold, adventurous and to my delight, lots of fun. Liz is also generous with sharing her time with me. Because of Lizs decades of Hadassah national leadership, Liz was like my personal encyclopedia of Hadassah. She could fill in the blanks, read between the lines and give me the historical perspective that wasnt possible to glean no matter how thoroughly I would prepare for the board meetings. Sometimes we disagreed on matters, but I always respected her well-reasoned opinion.

Somewhere between meetings and conferences, Liz and I became great friends. We recently led a Fashion, Food, Wine & Design travel tour to Israel, where I depended on Lizs detail-oriented personality more times than I can count. Weve had fun together at Hadassah conferences across the U.S. and I discovered from our walks and talks along the beach in Santa Cruz that Liz has more hustle than Ill ever have.

Recently, Liz and I found ourselves early on a beautiful day in Oakland, walking around Lake Merritt (well, Liz was walking, I was perpetually hurrying to catch up to her). The lake was like a mirror and particularly tempting as we rounded past the boathouse. Lets rent a rowboat! I said, Liz asked if Id rowed before. Yes, I replied, and off we went. In the middle of the lake after Id lost the oar (yet again) Liz realized that she should have asked me not whether Id done this before, but whether I knew how to row, which I did not. Of course, Liz knew how to row from a childhood summer camps. Liz spent the rest of the morning patiently coaching me and taught me how to row (at least well enough to get back to the dock).

I think the rowboat adventure (or misadventure, depending who you ask) is a good analogy for our mentor/mentee relationship. I had the passion, but I didnt know what I didnt know. Luckily, Liz was there to teach me how to safely navigate.

Our mentoring relationship is a wonderful mlange of mother-daughter, sisters and friends. Its women supporting and nurturing women with love and wisdom and it creates a family bond without biology. I have experienced this in one other area of my life, with Sammie.

Sammie Rogers came into my life as a teenager when I became her quasi-foster mother and over the years we too have evolved into this same wonderful mlange. It seems that the key to creating this is for both partners to be open and take a leap of faith. Without the competitiveness of a work environment or the invariable drama involved in mother-daughter relationships, something special but unnamed is created.

I can offer to help Sammie navigate lifes challenges without the subtext of the frequent mother-daughter condemnation and hurt feelings. And Sammie offers me love and inclusion that helps fill my heart. Its a wonderful feeling as a mentor to root for your mentee and celebrate in her successes and in the way she confronts lifes challenges and loss and to watch her persevere and thrive.

Mentoring is a celebration of strength of both the mentee and the mentor and of the bond with them. While I am confident in Sammies strength and perseverance, she is confident in mine and we rely on each other. I brought Sammie to Israel for a vacation, but she brought me up to the top of Masada to see the sun rise. She led the way, she encouraged me, she stayed with me while I caught my breath. Neither of us could accomplish what we did without the other and we had fun doing it and such a sense of renewal, resolve and responsibility.And thats what mentoring brings to both partners.

Read more here:
Asking for Help is a Sign of Strength, Not of Weakness - Thrive Global

The first symposium of the South West Brain Tumour Centre was held on Thursday at Derriford Hospital in Plymouth. During a fascinating and very well attended event, topics covered included the mechanism of tumour development, new drug targets, new biomarkers and brain tumour imaging. The South West Brain Tumour centre is of course one of the UK Centres of Excellence funded by Brain Tumour Research.

A really big cancer wide story this week is here Immune discovery 'may treat all cancer' applicable to some solid tumours but not yet brain it really shows the direction of travel toward immunotherapy I have recommended this book before but if interested please do read The Breakthrough by Charles Graeber it is available on Amazon and you can read reviews here - http://www.charlesgraeber.com.Researchers uncover novel drug target for glioblastoma by revealing a cellular pathway that appears to contribute to glioma stem cell spread and proliferation. This pathway shows that glioma stem cells ability to access key nutrients in their surrounding microenvironment is integral for their maintenance and spread. Finding a way to interrupt this feedback loop will be important for treating glioblastoma.

An intelligent molecule could significantly extend the lives of patients with glioblastoma, research finds. The molecule, called ZR2002, which can be administered orally and is capable of penetrating the blood-brain barrier, could delay the multiplication of glioblastoma stem cells resistant to standard treatment. According to scientists in the Metabolic Disorders and Complications Program at the Research Institute of the McGill University Health Centre (RI-MUHC) the ZR2002 molecule is designed to kill two birds with one stone: on top of attacking the tumour, it destroys its defence system.

Researchers find clues to drug resistance in medulloblastoma subtype.US scientists have identified specific types of cells that cause targeted treatment to fail in a subtype of medulloblastoma. They found while the majority of cells responded to treatment, diverse populations within the tumour continue to grow leadingto treatment resistance. They concluded that the diversity of cells within tumours allow them to become rapidly resistant to precisely targeted treatments," and that due to this tumour cell diversity, molecularly precise therapies should be used in combinations to be effective."

Nanoparticles deliver 'suicide gene' therapy to paediatric brain tumours growing in mice So-called "suicide genes" have been studied and used in cancer treatments for more than 25 years. Researchers report here that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a ''suicide gene'' to paediatric brain tumour cells implanted in the brains of mice.

According to a study that uncovers an unexpected connection between gliomas and neurodegenerative diseases a protein typically associated with neurodegenerative diseases like Alzheimers might help scientists explore how gliomas become so aggressive. The new study, in mouse models and human brain tumour tissues, was published in Science Translational Medicine and found a significant expression of the protein TAU in glioma cells, especially in those patients with better prognoses. Patients with glioma are given a better prognosis when their tumour expresses a mutation in a gene called isocitrate dehydrogenase 1 (IDH1). In this international collaborative study led by the Instituto de Salud Carlos III-UFIEC in Madrid, Spain, those IDHI mutations stimulated the expression of TAU. Then, the presence of TAU acted as a brake for the formation of new blood vessels, which are necessary for the aggressive behaviour of the tumours.

'Innovative research award' helps Colorado scientists block brain cancer escape routes Cancers used to be defined by where they grow in the body - lung cancer, skin cancer, brain cancer, etc. But work in recent decades has shown that cancers sharing specific genetic changes may have more in common than cancers that happen to grow in an area of the body. For example, lung cancers, skin cancers, and brain cancers may all be caused by mutation in a gene called BRAF. Drugs targeting BRAF have changed the treatment landscape for melanoma, an aggressive form of skin cancer, and are also in use against lung cancers and brain cancers with BRAF mutations. It is really worth clicking through to read more on this and the ultimate goal of identifying new potential targets for combination therapy and new agents that could be added to BRAF inhibiting drugs in brain cancer to keep the cancer from developing resistance.

Related reading:

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Weekly pick of brain tumour research news from around the world - Brain Tumour Research

For the first time, scientists have produced snake venom toxins in the lab, opening up a much-needed path for developing drugs and venom antidotes that doesn't involve having to breed and milk real-life snakes.

The toxins have been produced through mini glands called organoids, following a process adapted from growing simplified human organs something that is already helping in a wide range of scientific and medical research projects.

In the case of the snakes, researchers were able to blow organoids matching the Cape coral snake (Aspidelaps lubricus cowlesi) and seven other snake species, and they say this new approach is a welcome upgrade on current methods of farming snakes to extract their venom.

"More than 100,000 people die from snake bites every year, mostly in developing countries," says molecular biologist Hans Clevers, from Utrecht University in the Netherlands. "Yet the methods for manufacturing antivenom haven't changed since the 19th century."

By tweaking the recently developed process for growing human organoids including reducing the temperature to match reptiles rather than mammals the researchers were able to find a recipe that supports the indefinite growth of tiny snake venom glands.

Tissue was removed from snake embryos and put into a gel mixed with growth factors, but access to stem cells which is how human and mouse organoids are usually developed wasn't required.

The cells quickly began dividing and forming structures, giving the team hundreds of growing samples in the space of a couple of months, and producing small white blobs from which venom toxins could be harvested.

Al least four distinct types of cell were identified by the researchers within the artificially grown venom glands, and they were also able to confirm that the venom peptides produced were biologically active, closely resembling those in live snake venom.

Snake venom gland organoids. (Ravian van Ineveld/Princess Mxima Center)

"We know from other secretory systems such as the pancreas and intestine that specialised cell types make subsets of hormones," says developmental biologist Joep Beumerfrom Utrecht University.

"Now we saw for the first time that this is also the case for the toxins produced by snake venom gland cells."

The use of snake venom toxins to develop medicines and treatments has been going on since the time of ancient Greece. In the modern age, drugs fighting everything from cancer to haemorrhages have been developed with the help of toxins we find in snake venom.

Having faster and more controlled access to these toxins could mean these treatments can be developed more easily and on a shorter time scale, say the researchers.

Besides drug development, these organoid venom glands should make it easier and faster to develop antivenoms and with so many people suffering deaths, injuries or disabilities because of snake bites, that will make a considerable difference.

"It's a breakthrough," snake venom toxicologist Jos Mara Gutirrez from the University of Costa Rica, told Science.

"This work opens the possibilities for studying the cellular biology of venom-secreting cells at a very fine level, which has not been possible in the past."

The research has been published in Cell.

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Scientists Have Grown Snake Venom Glands in The Lab. Here's Why That's Awesome - ScienceAlert

Every year, medical technology further evolves, and new discoveries are made. This brings hope to those suffering from grave medical conditions. Health24 covers these advances on an ongoing basis, and the following are a few of the biggest breakthroughs of the decade in a nutshell:

The past decade has seen a number of medical headlines involving 3D-organ-printing, up to the point where, recently, researchers managed to create living skin, complete with blood vessels, as well as hearts. While many of these advancements need more research before they can be used in a clinical setting, 3D-printing is set to become more prevalent over the next the next decade, which will make transplanting easier for those in need.

Health24 published several stories about gene therapy over the last ten years. And while there were restrictions placed on gene therapy research in the early 2000s, there's been a strong resurgence, as illustrated by this study focusing on gene therapy in the fight against leukaemia.

Despite a number of setbacks, there were some successes that could translate to treatments in the future. One of the most recent development involves the first clinical trial of its type. Researchers used CRISPR to edit the DNA of peoples immune systems to help treat certain cancers.

While only a small number of patients were involved in the Stage 1 clinical trial, experts believe that this was an important step, in that it proved that the technique is safe to use.

Read more about gene therapy here.

The focus on gut health and our microbiome (the collection of bacteria in the gut) has never been stronger. In the past, researchers didnt pay much attention to the role of the bacteria in our gut, and it's been mainly during the past 15 years that researchers have been studying this concept.

According to the BMJ, the gut microbiota is crucial for essential processes in the body, such as the fermentation of non-digestible dietary fibres. It does more than that, though, and plays a role in many key areas of human health, from our immunity and appetite to the way we digest our food.

This helped researchers to explore the role of gut bacteria in areas like depression. An article in the BMJ reports on changes in the gut microbiota in the case of not only obesity, diabetes, and liver disease, but also cancer and even neurodegenerative diseases.

In fact, a study covered by Health24 links gut microbes to chronic fatigue syndrome, a condition that has been baffling experts for decades.

HIV and Aids remain important public healthcare topics in South Africa. During the past decade, antiretroviral treatment has improved and become more readily available. In fact, the virus is currently controlled so well that the viral load in many patients' blood has become virtually undetectable.

According to Pharmaceutical Technologies, various studies over the past decade found that treatment with antiretroviral therapy has also reduced the risk of spreading the infection to HIV-negative partners in both homosexual and heterosexual couples.

A few months ago, Health24 published a story about a man simply known as the London patient, who became entirely free from HIV following stem cell treatment for Hodgkin's Lymphoma. He was the second patient to demonstrate this phenomenon.

In 2017, a man known as the Berlin patient had two copies of the CCR5-delta32 genetic mutation. The patient stopped his ART 16 months following a bone marrow transplant, and his blood viral load was still undetectable 18 months later.

Canceris one of the leading causes of morbidity and mortality worldwide, with approximately 14 million new cases reported annually, according to the World Health Organization. Experts say immunotherapy is a promising new development, and ongoing research has been conducted over the past decade.

In one of the latest studies, Dr Christopher E. Rudd, a researcher at the Centre de Recherche de l'Hpital Maisonneuve-Rosemont (CR-HMR) and Universit de Montral, discovered a new cell therapy approach that boosts the immune response of T lymphocytes to malignant tumours. The results of the study were recently published in the respected journal Nature.

Image credit: iStock

Compiled by Marelize Wilke

See more here:
5 of the biggest medical advances of the past decade - Health24

Global Cell Therapy Technologies Marketwas valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

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Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

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Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South America

Key players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at:https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

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Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2018-2026) - Expedition 99

Right now, your bodys stem cells are working hard replacing your skin every two weeks, creating new red and white blood cells and completing thousands of other tasks essential to life. They are your own personalized fountain of youth.

Scientists generally agree that a stem cell should be able to do both of the following:

One theory of ageing suggests that between the ages of 30 and 50, our stem cells reach a turning point and start to decline in number and function. This results in the typical features associated with ageing.

There does not seem to be a single discoverer of stem cells. Accounts date back to the 1800s and even further, but the first successful medical procedure was a bone marrow transfusion in 1939. Advances in immunology led to donor matching, initially via siblings and close relatives. Unrelated donor matching flourished in the 1970s, alongside donor registries.

In the 1980s, scientists identified embryonic stem cells in mice, leading to the 1997 cloning of Dolly the Sheep. This created immense interest for human and medical applications and a backlash in the US as federal R&D funding was essentially halted in 2001.

In 2012, a Nobel Prize was awarded for the earlier discovery of induced pluripotent stem cells (iPS). Essentially, they return potency and self-renewal properties to mature non-stem cells, essentially making them act like stem cells again.

In the decade between 2010 and 2019, the first wave of stem cell start-ups emerged, alongside R&D programmes at many large pharmaceutical companies, leading to innovation and the first human clinical trials for iPS and other related therapies.

According to Q3 2019 data from the Alliance for Regenerative Medicine, there are 959 regenerative medicine companies worldwide sponsoring 1,052 active clinical trials; 525 of these companies are in North America, 233 in Europe and Israel, and 166 in Asia. In aggregate, $7.4 billion has been invested in regenerative medicine companies in 2019; $5.6 billion of which has been dedicated to gene and gene-modified cell therapy, $3.3 billion in cell therapy, and $114 million in tissue engineering.

Overview of the cancer stem cells market

Perhaps most excitingly, curative therapies are hitting the market and the results are astonishing: 60% of Acute Lymphoblastic Leukemia patients taking Novartis Kymirah showed a complete response (no traces of cancer) and were declared in full remission. Meanwhile, 75% of patients with Transfusion-Dependent -Thalassaemia treated with bluebird bios Zynteglo achieved independence from transfusions. Perhaps most astonishingly, 93% of spinal muscular atrophy patients treated with Novartis Zolgensma were alive without permanent ventilation 24 months after treatment. We should expect more medical breakthroughs in the coming years.

New science, new start-ups: several companies in the sector have gone public or been acquired. These exits led to the recycling of talent and capital into new companies. Because the science and commercial systems have also advanced, the companies in the next wave are pursuing bigger challenges, driving innovation, with even greater resources.

Patients are eager: the current market for stem cell therapies is growing at 36% per year, though it will rapidly expand when a breakthrough occurs toward the treatment of a non-communicable disease (such as cancer, diabetes, heart disease) or a lifestyle factor (for example, growing hair in the correct places, expanding cognitive abilities or increasing healthy lifespan).

New R&D models: funding is flowing into the sector from large companies, VC funds, and institutions such as the California Institute for Regenerative Medicine (CIRM) and New York State Stem Cell Science programme (NYSTEM). Some of the leading university R&D platforms include the Center for the Commercialization of Regenerative Medicine in Toronto, the Stanford Institute for Stem Cell Biology and Regenerative Medicine, the Oxford Stem Cell Institute, and most notably, the Harvard Stem Cell Institute (HSCI).

Founded in 2004, HSCI has established a phenomenal track record. It provided the first $200,000 in funding to Derrick Rossis lab, which inspired the largest biotech IPO to date. HSCI scientists were also co-founders or principals in the three most prominent gene-editing companies (CRISPR Tx, Intellia and Editas), the combined $1.55-billion True North/iPierian acquisitions and the recent $950-million acquisition of Semma Tx, Frequency Tx, Fate Tx, Epizyme Inc., and Magenta Tx.

For the casual investor, Evercore ISI is building a Regenerative Medicine Index, which may be the simplest way to build a portfolio. For institutions and those with deeper pockets, regenerative medicine funds are forming, including the Boston-centric Hexagon Regenerative Medicine Fund, which aims to create companies out of the Harvard Stem Cell Institute.

Caveat emptor. Though patients needs are immediate, those seeking treatments should think very carefully about the risks. There are many dubious clinics touting expensive stem cell treatments and some patients have experienced horrifying complications. Dr. Paul Knoepfler of UC-Davis has written a practical and scientifically accurate guide, a strongly recommended read if you or a family member are considering treatment or a clinical trial.

The leading causes of death in 1900 were mostly infectious/communicable diseases. While the prevalence of most causes has diminished, the largest increases include heart disease (+40%) and cancer (+300%). Granted, this is partly due to doubling life expectancy and a lack of death from other causes. However, given time and resources, scientists and physicians may cure these challenging diseases.

Total disease burden by disease or injury

Today, six of the seven leading causes of death are non-communicable diseases (heart disease, stroke, lung diseases, cancer, Alzheimers disease and diabetes). Based on the early promise mentioned above, regenerative medicine may be our best hope to solve the great non-communicable diseases of our time, and perhaps the single most transformative medical innovation in a century.

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The views expressed in this article are those of the author alone and not the World Economic Forum.

Original post:
Why stem cells could be the medical innovation of the century - World Economic Forum

People with multiple sclerosis have been waiting for this: A full-scale clinical trial testing the effectiveness of stem cell transplantation as an MS treatment. The trial is being conducted by the U.S. National Institutes of Health, and its enrolling people with MSat several centers in the United States and one in the United Kingdom.

The U.S. has been behind the curve when it comes to approving stem cell treatments for people with MS. Autologous hematopoietic stem cell transplantation (aHSCT) has been available in Mexico and Russia for several years, but isnt widely available in the U.S.

In the procedure, doctors collect a patients blood-forming stem cells, give the patient high-dose chemotherapy to deplete the immune system, and then return the patients own stem cells to rebuild the immune system. The system, hopefully, returns free of cells believed to be involved with MS.

Three years ago, following a promising aHSCT study, Dr. Anthony Fauci was optimistic but cautious. Fauci is the director of the National Institute of Allergy and Infectious Diseases. He said, These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS. But Fauci emphasized that a larger trial was still necessary. It appears that trial has finally arrived.

The Phase 3 trial, called BEAT-MS (NCT04047628), will put aHSCT head-to-head against the top-line disease-modifying therapies (DMTs) available in the U.S. to treat MS.

It will enroll 156 adults, ages 18 to 55, with a diagnosis of relapsing-remitting MS (RRMS) at 21 sites 20 in the U.S. and one in the U.K. Participants will be randomly assigned to receive either aHSCT or one of the high-efficacy MS treatments currently in use: Ocrevus (ocrelizumab), Lemtrada/Campath (alemtuzumab), Tysabri (natalizumab), or Rituxan (rituximab). The participants, who wont know which treatment theyre receiving, will be followed for six years.

In a news release, investigators said they want to compare how much time elapses between the start of the treatment and an MS relapse. Theyll also compare the newly developing immune systems of the participants who receive aHSCT with the immune systems of those who receive a DMT.

Finally, theyll compare the effects of the two treatment types on other measures of disease activity and severity, their cost-effectiveness in terms of healthcare costs and productivity, and participants quality of life. We hope that BEAT-MS will clarify the best way to treat people with relapsing MS, said Dr. Jeffrey Cohen, the trials leader.

I hope so, too. A study such as this is long overdue. But I think I echo the feelings of many people with MS when I express dismay that were probably still at least six years away from approval of aHSCT in the U.S.

As I wrote three years ago, [S]cientists have been studying stem cell treatments for years and it sure seems as if were still crawling when we should be cruising. Also, why limit this study to people with RRMS? Why not include those with primary and secondary progressive MS? Am I wrong to think that they would benefit from aHSCT as much, if not more, as those with relapsing MS?

What do you think?

Starting this month, Multiple Sclerosis News Today columnist Jennifer Powell and I have begun recording MS news and feature stories in audio format. Jennifers audio reports are available on SoundCloud every Monday, and mine are available every Thursday. Click here to give us a listen.

Youre invited to visit my personal blog at http://www.themswire.com.

***

Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

Ed Tobias is a retired broadcast journalist. Most of his 40+ year career was spent as a manager with the Associated Press in Washington, DC. Tobias was diagnosed with Multiple Sclerosis in 1980 but he continued to work, full-time, meeting interesting people and traveling to interesting places, until retiring at the end of 2012.

Read more:
The Clinical Trial that MS Patients Have Been Waiting For - Multiple Sclerosis News Today

News

by DAVID JENSEN posted 01.13.2020

One of the nations leading regenerative medicine industry groups is touting multi-billion dollar savings that may be achieved with the type of stem cell and gene therapies that are being developed with cash from Californias financially beleaguered stem cell program.

The industry group is the Washington, D.C-based Alliance for Regenerative Medicine (ARM). It is tackling one of the major issues facing development of commercial stem cell therapies sticker shock at their expected prices, running upwards of a $1 million or more.

The ARM study predicted cost savings of as much as $33.6 billion over about a decade in connection with three afflictions: sickle cell disease (SCD),multiple myeloma (MM) andhemophilia A (Hem A).

Without a willingness from health care insurers to cover the costs and provide a pathway to profit, it is unlikely that the biotech industry will embrace production of the therapies.

In a study released Friday, the group said:

Advances in molecular biology and genetics are leading to new treatments for rare diseases that require new ways of assessing value. CGTs (cell and gene therapies) are directed at the underlying cause of a condition and offer durable, potentially curative, or near-curative benefits. These transformative therapies create challenges for current reimbursement frameworks as they (the therapies) require significant upfront costs but are expected to provide a lifetime of benefits. The recurring treatment costs of chronically-managed patients can be greatly reduced and even eliminated with a one-time administration or short course of these novel therapies.

As CGTs arrive on the market, payers need new models for assessing their value. These treatments could potentially end the patients burden of illness, resulting in cost offsets (eliminating or reducing the need for long-term treatment, hospitalizations, and other care) and productivity gains that span a lifetime. Manufacturers incur a high per-patient development cost for these therapies and payers who bear the cost of treatment may not realize the long-term financial benefits due to health plan switching.

The ARM study predicted cost savings of as much as $33.6 billion over about a decade in connection with three afflictions: sickle cell disease (SCD), multiple myeloma (MM) and hemophilia A (Hem A).

The discussion of the costs of stem cell therapies has special resonance in the Golden State where voters are likely to be asked next fall to give $5.5 billion more to its stem cell agency.

Californias stem cell agency was not mentioned in the study, but it has funded research in all three areas. The agency is a member of ARM.

The study, backed by ARM and performed bythe Marwood Group, said, Access to CGTs for even a modest number of patients with MM, SCD, and Hem A each year can reduce overall disease costs by nearly 23% over a 10-year period. The savings from lowering healthcare costs and raising productivity are considerable, approaching $34 billion by 2029. Of the savings, $31 billion are from a reduction in healthcare costs and $3 billion are from productivity gains.

The model used by ARM assumed CGT prices as high as $2 million. The study said, The model has tested more than 180 different prices across the three potential CGTs that ranged from a minimum test price of $150,000 and up to a maximum price test of $2,000,000. The prices entered into the model created 60 different cost savings curves for all three of drugs in this model. Prices were distributed with more than 50% of test prices in the $100,000-$600,000 price per administration range.

The discussion of the costs of stem cell therapies has special resonance in the Golden State where voters are likely to be asked next fall to give $5.5 billion more to its stem cell agency, known formally as theCalifornia Institute for Regenerative Medicine (CIRM).

The agency, funded with $3 billion in 2004, is down to its last $27 million. A new, proposed ballot initiative is focusing hard on affordability of stem cell treatments. The initiative has no specific solutions but stipulates that a new version of CIRM if the ballot measure is approved should devise some ways to come up with answers for insurers who are not likely to warm easily to $1 million therapies.Eds Note: DavidJensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters in the past 15 years.

Originally posted here:
Stem cell agency indirectly boosted by national industry group - Capitol Weekly