Category Archives: Cell Medicine

R3 Ultrasound Injection Training Course - All Attendees Receive a FREE Exosome Procedure! (844) 438-7836

LAS VEGAS (PRWEB) December 05, 2019

The nations leader in regenerative medicine training, R3 Stem Cell, announced that it has opened registration for its new musculoskeletal ultrasound injection training course January 26, 2020 at the M Resort in Las Vegas, Nevada. Providers will learn the most modern techniques for both diagnostic evaluation and injections for all types of joints in the body.

The ultrasound injection training course is being held at the M Resort in Las Vegas, Nevada. The instructors have over 40 years of combined experience in ultrasound training. Several types of ultrasound machines will be utilized at the training, so providers can get a feel for what they like to use.

The R3 Ultrasound Course is different than most, as providers get to work on real patients along with other attendees (not cadavers). In addition, each provider receives a free regenerative injection with exosomes too. The course is exceptional for MD, DO, NP, NMD, PA and RNs as well.

Ultrasound is a very convenient option for image guidance and a great way to ensure accuracy for joint injections. Ultrasound machines do not take up a lot of space, are cost effective, and patients appreciate it being used. Most providers perform injections blindly and often miss the target, leading to suboptimal outcomes.

According to R3 CEO David Greene, MD, MBA, Ultrasound for injections is much more convenient than a huge C-Arm, but it takes practice. This course is completely hands on with real patients receiving real biologics, and each provider gets to receive a procedure with exosomes too!

Registration is limited and will fill up fast, so it is recommended for providers to secure their spot asap. Signups for the Las Vegas Ultrasound course can be completed at http://stemcelltrainingcourse.org/ultrasound or by calling (844) GET-STEM.

Share article on social media or email:

Read more:
R3 Stem Cell Announces Comprehensive Ultrasound Injection Training Course January 26, 2020 - PR Web

Mount Sinai Medical Center has joined withBrainStorm Cell Therapeutics to explore the safety and efficacy of NurOwn as a potential treatment for progressive multiple sclerosis (MS) in an ongoing Phase 2 trial.

The New York center is the fourth clinical site participating in the trial, in addition to Keck School of Medicine of The University of Southern California (USC), Stanford University School of Medicine, and the Cleveland Clinic in Ohio.

Mount Sinai is ready to start enrolling patients under the supervision of neurologistFred Lublin, MD, and his clinical team at The Corinne Goldsmith Dickinson Center for Multiple Sclerosis.

We are happy to be a part of this exciting study to determine if neurally-directed stem cells can be a therapeutic approach to treating MS, Lublin said in a press release.

NurOwn is a cell-based therapy that uses the patients own bone marrow-derived mesenchymal stem cells (MSC) to promote and support the repair of nerve cells.

Patients MSCs are modified in the lab to secrete growth factors that are believed to protect nerve cells from damage, to promote the repair of the protective myelin sheath in nerve cells (which is destroyed in MS), and potentially slow or halt disease progression.

The open-label Phase 2 clinical trial (NCT03799718) will enroll and treat up to 20 adults with either primary progressive MS (PPMS) or secondary progressive MS (SPMS).

All participants will undergo a bone marrow biopsy to collect MSCs, which will later on be injected back to the patient through three intrathecal administrations injected directly into the cerebrospinal fluid over 16 weeks.

During this time, and for the following 12 weeks, researchers will evaluate the safety of the procedure, as well as the neuromodulatory effect of the modified MSCs.

To confirm that NurOwn cells are delivering neurotrophic factors and immunomodulatory signaling molecules as expected, the research team will look for an increase in the amount and type of these biomarkers in patients cerebrospinal fluid following the cell transplants.

BrainStorm looks forward to partnering with and supporting Dr. Lublin and the dedicated clinical trial team at the Mount Sinai Hospital to quickly advance the Phase 2 progressive MS clinical trial, said Ralph Kern, MD, MHSc, BrainStorms chief operating officer and chief medical officer.

For more information about the trial, including its sites and contacts, please visit this link.

NurOwn has been tested in animal models for various neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), where it showed a good safety profile and promising efficacy signs.

An ongoing Phase 3 trial (NCT03280056) testing NurOwn in people with ALS is expected to conclude in December 2020.

Total Posts: 1,053

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

Go here to see the original:
Mount Sinai Joins Progressive MS Trial of NurOwn Cell Therapy - Multiple Sclerosis News Today

Results of a new study in mice suggest that the body may be able to defeat the influenza virus if a person has the right sort of diet a ketogenic, or keto, diet.

Infection with the influenza virus, better known as the flu, has accounted for 12,00061,000 deaths every year since 2010 in the United States, with an annual economic burden of $87.1 billion.

The introduction of the flu vaccine has greatly improved infection and morbidity rates. However, there is still currently no cure for the illness.

Healthcare professionals and scientists alike are continuing the search for novel therapeutics to combat the flu, yet the key may lie within the body's own immune system. Moreover, it may be activated by the keto diet.

Following the keto diet involves eating foods that are high in fat and low in carbohydrates. Meals tend to consist of a variety of meat, fish, poultry, and non-starchy vegetables.

According to the findings of a new study, appearing in the journal Science Immunology, when mice fed a keto diet were injected with the flu virus, their survival rates were much higher than those of mice fed a diet high in carbohydrates.

The main reason for this, the researchers believe, is that a keto diet blocks the formation of inflammasomes, which are multiunit protein complexes that the immune system activates.

Inflammasomes can also cause harmful immune system responses in the host. This triggers the release of gamma delta T cells.

Gamma delta T cells are responsible for producing mucus in the linings of the lungs, which helps the body get rid of infectious agents. The mucus is then wafted up the airways and coughed out.

The joint senior authors of the study are Prof. Akiko Iwasaki and Prof. Vishwa Deep Dixit, both of the department of immunobiology at the Yale School of Medicine, in New Haven, CT.

The objective of the study was to determine how the keto diet affects host defense against a lethal flu virus infection.

The researchers randomly assigned the mice to diet groups 1 week before they induced the infection. Next, they monitored the rodents for signs of infection and assessed their immune responses.

The team found that keto diet feeding confers protection against the flu virus in mice by increasing the number of gamma delta T cells in the airways.

This response occurred relatively late after the infection in the mice, due to their dependence on T cell receptors on other cells. But in humans, this response is much quicker, as gamma delta T cells can expand independently.

In addition, previous research in mice has shown that a specific subset of gamma delta T cells can efficiently induce the cytolytic killing of flu-infected airway cells.

In the current study, the expansion of gamma delta T cells resulted in lower viral titer measurements in the mice that had received a ketogenic diet.

The team also investigated the potential for changes in the levels of genetic activity using RNA sequencing, a technique that can measure the levels of transcription across the genome.

This showed that although a keto diet could impact the expansion of gamma delta T cells, this was not associated with any changes in the activity of genes involved with cytotoxicity.

Interestingly, when mice were bred without the gene that encodes for gamma delta T cells, the keto diet provided no protection against the flu virus.

Commenting on this result, Prof. Iwasaki says, "This was a totally unexpected finding."

"This study shows that the way the body burns fat to produce ketone bodies from the food we eat can fuel the immune system to fight flu infection."

Prof. Vishwa Deep Dixit

How do gamma delta T cells protect the host in response to a keto diet? As the researchers report, the current theory is that the expansion of these cells in response to ketogenic feeding leads to more efficient killing of the flu virus.

This, in turn, results in much lower viral titers and better preservation of the cells lining the airways.

Experts believe that the gamma delta T cells induced by the keto diet may enhance the barrier and innate defense systems of airway-lining cells at baseline, thereby allowing for a better response to the flu virus.

These results demonstrate that the answer to combatting the flu virus does not necessarily lie in producing drugs to relieve flu symptoms and that changing the diet can have a dramatic effect on how the body responds to infection.

The results also suggest that if the flu can be tackled in this way, there is the potential for changes in diet to help the body more effectively fight other viral infections.

This type of research is in its infancy, and much more will be needed to elucidate exactly how the keto diet may help combat the flu.

Read more:
Can the keto diet help beat the flu? - Medical News Today

Emerging medical research and cutting-edge technology will dramatically increase human life expectancy and quality of life in the near future, according to a recent fireside chat titled How Do We Make 100 Years Old Our New 60? hosted by Bob Reby, Ambassador of the Fairfield- Westchester Chapter of Singularity University and CEO of Reby Advisors, with special guest Sam Gandy, MD, Ph.D., a prominent internationally recognized expert in neurology and psychology.

Anyone interested in learning more about these medical breakthroughs may watch a video of the event for free on the Reby Advisors website: http://www.rebyadvisors.com/live-events-videos

Dr. Gandy, Chairman Emeritus of the National Medical and Scientific Advisory Council of the Alzheimer's

Association shared new research on human stem cells, genetic codes and the complex hereditary nature of Alzheimers Disease, among other topics.

With regard to stem cell research, Dr. Gandy explained, Its possible now to restore sight and hearing in certain conditions. This was not possible before. These are people who were deaf and blind, doomed to being deaf and blind lifelong.

He continued, Stem cells are the primordial type of cell that can ultimately be differentiated or specialized to form any type of cell in the body. If you have a stem cell from someone, you can then recreate the heart cells or lung cells or brain cells that a particular person has. It can really [lead to] person-based medicine.

Reby also brought up the topic of CRYSPR Genome Editing, and the potential of this research to be used for both good and harm.

CRYSPR is basically gene editing, which means that you can go into the DNA and make changes, edits. If you want to eradicate genetic diseases, it's possible to use this technology to go into an egg, or a sperm, and correct the mutation. So, you could edit out a hereditary disease.

As futuristic as these advancements in medical technology and genetic engineering may be, finding the cure for some complex diseases, like Alzheimers, remains a major challenge.

Most people with Alzheimer's Disease, it's not that simple. The challenge is to find an intervention that we can use beginning in midlife that is safe and will prevent Alzheimer's. Some of the ways that we have of intervening now are not perfectly safe and would not be things that you'd want to give people for 50 years.

The fireside chat was the first event for the Fairfield-Westchester Chapter of Singularity University, a global learning and innovation community using exponential technologies to tack the worlds biggest challenges and build a better future for all.

According to Reby, future events will focus on artificial intelligence, robotics and other exponential technologies. He explained, The reason I like [Singularity University] is their faculty is made up of a lot of business owners, so theyre not just talking about it. Theyre doing it as well.

Community leaders, business owners and technology enthusiasts are encouraged to contact Reby

Advisors if they would like to participate in the Fairfield-Westchester Chapter of Singularity University.

To watch the video of this first event, go to: http://www.rebyadvisors.com/live-events-videos

Link:
Will We Live to Age 120? International Expert Weighs in at Danbury Event - HamletHub

We have a mutualistic but complicated relationship with the collection of microbes in our gut, known as the intestinal microbiome. This complex community of bacteria breaks down different food components, and releases nutrients such as vitamins and a plethora of other factors that control functions in tissues way beyond the intestinal tract. However, the sheer numbers of microbes also present a threat as they can trigger inflammation, which is thought to be at the root of many intestinal diseases, including inflammatory bowel disease, radiation-induced intestinal injury, and some cancers.

To allow the uptake of beneficial substances from the gut lumen, and at the same time prevent gut microbes from contacting the intestinal epithelial tissue surface, specialized cells called goblet cells continuously produce mucus, the slimy goo-like substance that coats the entire intestinal surface. Mucus thus far has been notoriously difficult to study: its structure quickly disintegrates in surgically removed sections of the gut, the system most often used to study mucus, and no in vitro culture system has been able to reconstitute an in vivo-like mucus layer with the natural structure seen in living intestine outside the human body. Adding to these difficulties, mucus also differs between humans and other species, different sections of the intestinal tract, and even different individuals.

Now, focusing on the large intestine or colon which houses the greatest number of commensal microbes and has the thickest mucus layer, a team of tissue engineers at Harvards Wyss Institute for Biologically Inspired Engineering has developed a colon-on-a-chip (Colon Chip) microfluidic culture device lined by patient-derived colon cells that spontaneously accumulates a mucus layer with the thickness, bi-layered structure, and barrier functions typically found in normal human colon. The mucosal surface in their model also responds to the inflammatory mediator prostaglandin E2 (PGE2) by mounting a rapid swelling response. Their findings are published in Cellular and Molecular Gastroenterology and Hepatology.

Our approach provides researchers with the opportunity to find answers to questions about normal and disease-associated mucus biology, such as its contributions to intestinal inflammatory diseases and cancers, and complex host-microbiome interactions, said Founding Director Donald Ingber, M.D., Ph.D., who is the senior investigator on the study. Importantly, we use patient-derived cells to line these devices and so this represents an entirely new approach for personalized medicine where it can be possible to study how mucus functions or dysfunctions in a particular patient, and to tailor therapy accordingly.

Ingber is also the Judah Folkman Professor of Vascular Biology at Harvard Medical School and the Vascular Biology Program at Boston Childrens Hospital, as well as Professor of Bioengineering at Harvards John A. Paulson School of Engineering and Applied Sciences. His team is part of a multi-institutional collaboration supported by a Cancer Research UK Grand Challenge grant in which his Wyss team investigates how inflammation-related changes contribute to formation of cancers, including colon cancers. The Grand Challenge is an ambitious international cancer research initiative, supporting world-leading teams of scientists to take on some of the toughest challenges in cancer, and giving them the freedom to try novel approaches at scale.

The teams approach starts out with patient-derived colon cells from colon resections and endoscopic biopsies that are first grown as organoids, tiny organized balls of colon tissue that contain mainly epithelial stem cells. After fragmenting the organoids, their cells are used to populate the upper of a two parallel channels of a microfluidic chip that are separated by a porous membrane. Simply by perfusing the channels continuously with nutrient medium, the colon stem cells grow into a continuous sheet and form highly functional goblet cells that secrete mucus.

Growing the cells on-chip under flow results in about 15% of epithelial cells spontaneously differentiating into goblet cells. Distributed throughout the epithelium, these produce an in vivo-like mucus layer, said first-author Alexandra Sontheimer-Phelps, a graduate student from the University of Freiburg, Germany, working in Ingbers group. At the same time, other epithelial cells that keep dividing also replenish the goblet cell population just like in living colon, which means that the chip can be maintained in steady-state conditions for more than two weeks, which makes it highly useful for longer-term studies.

The Wyss team showed that the colon epithelium in the chip is fully polarized with distinct markers restricted to its lumen-exposed, mucus-secreting side and its opposite membrane-binding side. Its goblet cells secrete the major mucus protein mucin 2 (MUC2), which when linked to complex chains of sugar molecules, assembles into multi-molecular network or gel that takes up water. Our approach actually produces the bi-layered structure of normal colon mucus with an inner dense layer that we show is impenetrable to bacteria-mimicking particles flowed through the intestinal channel, and a more loose outer layer that allows particles to enter. This has never been accomplished before in vitro, said Sontheimer-Phelps.

To investigate the functionality of the mucus, she and her co-workers exposed the chip to the inflammatory mediator PGE2. The mucus underwent rapid swelling within minutes and independent of any new mucus secretion, and this process of mucus accumulation can be visualized in living cultures by viewing the chips from the side with dark field illumination. This dynamic response could be blocked by inhibiting one particular ion channel, which pumps ions into the colon epithelium and passively allow water molecules to follow and apparently, this drives mucus swelling when stimulated by signals such as PGE2.

Mucus has long been thought to be a passive, host barrier, but it is becoming increasingly clear that microbial species affect its structure and function in addition to feeding on its carbohydrates as an energy source. Our in vitro system brings us one step closer to figuring out how individual bacterial species and more complex microbial communities can affect mucus and vice versa, as well as how this complex interplay impacts development of intestinal diseases. We also now have a testbed to discover new therapeutic drug and probiotic strategies that might prevent or reverse these diseases said Ingber.

Reference:Sontheimer-Phelps, A., Chou, D. B., Tovaglieri, A., Ferrante, T. C., Duckworth, T., Fadel, C., Ingber, D. E. (2019). Human colon-on-a-chip enables continuous in vitro analysis of colon mucus layer accumulation and physiology. Cellular and Molecular Gastroenterology and Hepatology. https://doi.org/10.1016/j.jcmgh.2019.11.008

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Read more from the original source:
Investigating the Human Intestinal Mucus Barrier Up-close and Personal - Technology Networks

DetailsCategory: AntibodiesPublished on Wednesday, 04 December 2019 10:05Hits: 285

Approval based on the Phase III IMpower130 study showing the Tecentriq plus chemotherapy combination demonstrated a significant overall survival and progression-free survival benefit

SOUTH SAN FRANCISCO, CA, USA I December 03, 2019 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) approved Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [paclitaxel protein-bound; nab-paclitaxel] and carboplatin) for the initial (first-line) treatment of adults with metastatic non-squamous non-small cell lung cancer (NSCLC) with no EGFR or ALK genomic tumor aberrations.

We are pleased to offer this Tecentriq-based combination as a new treatment option that can provide a clinically meaningful survival benefit for people with non-squamous non-small cell lung cancer, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Todays approval offers another opportunity to help prolong the lives of people with this type of the disease.

This approval is based on results from the Phase III IMpower130 study, which showed Tecentriq in combination with chemotherapy helped people live significantly longer compared to chemotherapy alone (median overall survival [OS] = 18.6 versus 13.9 months; hazard ratio [HR] = 0.80; 95% CI: 0.640.99; p=0.0384) in the intention-to-treat wild-type (ITT-WT) population. The Tecentriq-based combination also significantly reduced the risk of disease worsening or death (progression-free survival; PFS) compared with chemotherapy alone (median PFS=7.2 versus 6.5 months; HR=0.75; 95% CI: 0.630.91; p=0.0024) in the ITT-WT population.

Safety for the Tecentriq plus chemotherapy combination appeared consistent with the known safety profiles of the individual medicines, and no new safety signals were identified with the combination. Grade 3-4 treatment-related adverse events were reported in 73.2% of people receiving Tecentriq plus chemotherapy compared with 60.3% of people receiving chemotherapy alone.

In lung cancer, Tecentriq is also approved in combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of adults with metastatic non-squamous NSCLC with no EGFR or ALK genomic tumor aberrations. Additionally, Tecentriq is approved by the FDA to treat adults with metastatic NSCLC who have disease progression during or following platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for NSCLC harboring these aberrations prior to receiving Tecentriq. Tecentriq is also approved in combination with carboplatin and etoposide (chemotherapy) for the initial (first-line) treatment of adults with extensive-stage small cell lung cancer (ES-SCLC).

Genentech has an extensive development program for Tecentriq, including nine Phase III studies underway across different types of lung cancer, and multiple ongoing and planned Phase III studies across genitourinary, skin, breast, gastrointestinal, gynecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

For those who qualify, Genentech offers patient assistance programs for people taking Tecentriq through Genentech Access Solutions. Doctors can contact Genentech Access Solutions at (866) 422-2377. More information is also available at http://www.Genentech-Access.com.

About the IMpower130 study

IMpower130 is a Phase III, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq in combination nab-paclitaxel and carboplatin versus chemotherapy (nab-paclitaxel and carboplatin) alone for chemotherapy-nave patients with stage IV non-squamous NSCLC. The study enrolled 724 people, of whom 681 were in the ITT-WT population and were randomized (2:1) to receive:

During the treatment-induction phase, people in Arm A received Tecentriq and carboplatin on day 1 of each 21-day cycle, and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until loss of clinical benefit, whichever occurred first. People in Arm A received Tecentriq during the maintenance treatment phase until loss of clinical benefit was observed.

During the treatment-induction phase, people in Arm B received carboplatin on day 1 and nab-paclitaxel on days 1, 8 and 15 of each 21-day cycle for 4 or 6 cycles or until disease progression, whichever occurred first. People in Arm B received best supportive care during the maintenance treatment phase. Switch maintenance to pemetrexed was also permitted. People who were consented prior to a protocol revision were given the option to crossover to receive Tecentriq as monotherapy until further disease progression.

The co-primary endpoints were:

About lung cancer

According to the American Cancer Society, it is estimated that more than 228,000 Americans will be diagnosed with lung cancer in 2019, and NSCLC accounts for 80-85% of all lung cancers. It is estimated that approximately 60% of lung cancer diagnoses in the United States are made when the disease is in the advanced stages.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Abraxane is a registered trademark of Abraxis Bioscience, LLC, a wholly owned subsidiary of Celgene Corporation.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

About Genentech in personalized cancer immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, were investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is studying more than 10 cancer immunotherapy medicines across 70 clinical trials alone or in combination with other medicines. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

SOURCE: Genentech

See more here:
FDA Approves Genentech's Tecentriq Plus Chemotherapy (Abraxane and Carboplatin) for the Initial Treatment of Metastatic Non-Squamous Non-Small Cell...

Soft tissue sarcoma cells stop a key metabolic process which allows them to multiply and spread, and so restarting that process could leave these cancers vulnerable to a variety of treatments. The enzyme that controls the process is called FBP2, and researchers from theAbramson Cancer Center, whodetailed their findingsinCell Metabolism, also showed that manipulating sarcoma cells to ramp up FBP2 expression slows or even stops their growth entirely. This ultimately leaves them susceptible to targeted therapies and potentially takes away their ability to develop treatment resistance.

Soft tissue sarcoma is actually a collection of distinct, rare cancer types affecting tissues that connect and surround other parts of the body, including muscle, fat, tendons, nerves, and blood vessels. While they can grow anywhere, the arms, legs, chest, and stomach are the most common sites. Because these cancers appear in so many different places in the body, their biology is incredibly diverse, making it difficult to develop one targeted treatment that can be broadly effective for all patients. Currently, the best options for treatment are surgerywhich may involve amputationchemotherapy, and radiation.

While other cancer types associated with high mutational burden have benefitted from the development of immunotherapies, the diversity and low frequency of genetic mutations in soft tissue sarcomas have made them more difficult to treat, which is why our identification of a broadly expressed metabolic approach is potentially so exciting, says the studys senior authorM. Celeste Simon, the Arthur H. Rubenstein, MBBCh Professor of Cell and Developmental Biology at the Perelman School of Medicine and scientific director of the Abramson Family Cancer Research Institute. The studys lead author isPeiwei Huangyang, who performed the work while obtaining her Ph.D. in Simons lab.

Read more at Penn Medicine News.

Excerpt from:
Researchers identify method to slow down cancer cells efforts to multiply - Penn: Office of University Communications

The National Institutes of Health and the Bill and Melinda Gates Foundation will together invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV with an attribute even rarer in the world of genetic medicine than efficacy, the groups announced on Wednesday: The cures, they vowed, will be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

The effort reflects growing concerns that scientific advances in genetic medicine, both traditional gene therapies and genome-editing approaches such as CRISPR, are and will continue to be prohibitively expensive and therefore beyond the reach of the vast majority of patients. Spark Therapeutics Luxturna, a gene therapy for a rare form of blindness, costs $425,000 per eye, for instance, and genetically engineered T cells (CAR-Ts) to treat some blood cancers cost about the same.

With CRISPR-based treatments already being tested in clinical trials for sickle cell disease, the blood disorder beta thalassemia, and another form of blindness, and with additional CRISPR treatments in development, scientists, ethicists, and health policy experts have grown increasingly concerned that the divide between haves and have-nots will grow ever-wider.

advertisement

Gene-based treatments are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries, the NIH said in a statement. To help right that, its collaboration with the Gates Foundation aims to develop curative therapies that can be delivered safely, effectively and affordably in low-resource settings.

Scientists whose research focuses on gene-based cures welcomed the infusion of funding and the recognition that genetic cures are on track to be unaffordable to the majority of patients. But they noted one irony. The most effective sickle cell drug, hydroxyurea, has hardly even been studied in sub-Saharan Africa, let alone made widely available. Yet a 2019 study found that giving children the drug cut their death rate by two-thirds and halved the pain crises that are common in sickle cell disease, caused by misshapen red blood cells that cannot flow through blood vessels.

The NIH-Gates collaboration is tremendously exciting and has the potential to have a great impact on sickle cell disease in sub-Saharan Africa, said Dr. Vijay Sankaran of the Dana-Farber/Boston Childrens Cancer and Blood Disorders Center, who has done pioneering research on genetic cures for the disease. But my hesitation is that even the inexpensive therapies we have today, such as hydroxyurea, are largely unavailable there. The question is, how do we best approach this disease, with therapies that are working today or with genetic therapies that might work?

The same concerns surround HIV. Very inexpensive less than $100 per year in the U.S. antiretroviral drugs can keep the virus in check, but only 67% of HIV-positive adults and 62% of HIV-positive in children in east and southern Africa are estimated to be on antiretroviral treatment.

The new collaboration aims to move gene-based cures into clinical trials in the U.S. and countries in sub-Saharan Africa within the next seven to 10 years, and to eventually make such treatments available in areas hardest hit by sickle cell disease and HIV/AIDS. The idea is to focus on access, scalability, and affordability to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, NIH Director Francis Collins said in a statement. We aim to go big or go home. But the challenge is enormous, he told reporters on Wednesday: Im not going to lie. This is a bold goal.

An estimated 95% of the 38 million people with HIV live in the developing world, with 67% in sub-Saharan Africa. Up to 90% of children with sickle cell disease in low-income countries die before they are 5 years old. In the U.S., the life expectancy for people with sickle cell disease is in the low 40s.

The NIH and the Gates Foundation will fund research to identify potential gene-based cures for sickle cell and HIV, and also work with groups in Africa to test those cures in clinical trials.

The science of genetic cures for both diseases is within reach, experts say. CRISPR Therapeutics and Vertex (VRTX) are already running a clinical trial for sickle cell disease, using the CRISPR genome editor to do an end-run around the disease-causing mutation in the hemoglobin gene: The therapy releases the brake on red blood cells production of fetal hemoglobin, whose production shuts off in infancy but which does not have the sickling damage of adult hemoglobin.

Developing effective, safe genetic cures for sickle cell and HIV would be only a first step, however. As currently conceived, such therapies require advanced medical facilities to draw blood from patients, alter their cells genomes in a lab, give the patients chemotherapy to kill diseased blood-making cells, and then perform whats essentially a bone marrow transplant, followed by monitoring patients in a hospital for days to prevent infection and provide intensive medical support, said Dr. Dan Bauer, a sickle cell expert at Boston Childrens.

He called the NIH-Gates effort terrific, but cautioned that delivering advanced gene therapies requires tremendous effort, extended hospitalization, and large supplies of blood products. All of those requirements mean that even if a CRISPR-based cure for sickle cell disease or HIV were provided at cost, there will still be barriers to access.

Recognizing that, Collins said, a genetic cure would have to be given directly into patients (in vivo), presumably through an infusion, rather than by treating blood or other cells removed from patients and genetically transformed in a lab (ex vivo). That could avoid the resources needed for and the complications that can occur with ex vivo therapies, said Sankaran, who has discussed the approach with Gates officials.

This story has been updated with additional comments.

Read this article:
NIH, Gates Foundation aim to bring genetic cures to the poor - STAT

MIAMI - October 24, 2019 - ( Newswire.com )

The AASCP has recently created guidelines thatare current safety recommendations given to physicians who are using biologics in their medical practice. A highly anticipated and sought after Safety StandardsPanel session, hosted by AASCP on Nov. 2, 2019, will be moderated by The Alliance for Cell Therapy Now,with President Ms. Janet Marchbrody.The sessions normally are closed to the public but this particular SafetyStandard Panel discussion will be open to the public, covering the growing safety concerns of the industry.

Alliance for Cell Therapy Now is a coalition of organizations representing patients, health care providers and the academic and scientific community, who are working together to advance safe and effective regenerative cell therapies. The mission is to advance the development, manufacturing and delivery of safe and effective regenerative cell therapies through policy development, consensus and advocacy. Alliance for Cell Therapy Now is bringing together experts and stakeholders to gain consensus on and advocate for policies that will advance the science and the field, including those focused on promoting clinical research, assuring the adoption of consensus standards to promote safety and quality, building capacity and expertise within the workforce, and establishing a national outcomes database to advance the science, promote improvements in quality and safety, and inform regulatory, paymentand patient decision-making.

Alliance for Cell Therapy Now is guided by an Advisory Board comprised of leaders in the scientific, academicand patient communities; Ms. Janet M. Marchibroda President, Alliance for Cell Therapy Now Fellow, Bipartisan Policy Center Senior Vice President, Health Policy, Bockorny Group, has agreed to join theAASCP as a moderator for their SafetyPanelat The Hyatt Regency in Miami. This particular coveted safetypanel session will be open to the public and broadcast live on YouTube at 3:00 p.m. on Nov. 2, 2019.

According to AASCP, if you are using biologics in your practice, whether you are using SVF, PRP, bone marrow, UCB, amniotic products,exosomes,xenografts, or peptides, there are key considerations to take into account to achieve the best safety for your patients. The AASCP also recommends communication with the Chief Scientific Officer from the laboratory you work with.AASCP advises that just talking to a sales agent is not sufficient enough when determining the quality of products for your patients. Sales agents typically do not have a medical or scientific background.

The spokesman for the AASCP, Dr. AJFarshchian,said earlier: The American Academy of Stem Cell Physicians is a group of physicians, scientists and researchers who collectively represent the most authoritativenon-federal group advocating for guidelines and education on stem cell therapy and regenerative medicine. AASCP members are experts within all fields of stem cell therapy from: SVF, BM, UCB, Exosomes, Peptides, Xenografts, Allografts and Amniotic Fluids and are considered the most experienced leaders for proper advocacy in the field. The AASCP is involved directly with other authorities within the field and seeks only to bring knowledge and awareness for the ever growing regenerative medicine industry.My hope is that the SafetyPanel discussion on Nov.2, 2019, is to help get rid of the bad actors that are damaging the field for everyone.

AASCP is hosting their medical conference in Miami on Nov. 1-3 , 2019. Sessions are normally closed to the public and, therefore, require registration. The conference is taking place at the downtown MiamiHyatt Regency, located at 400 SE 2nd Ave, Miami, FL 33131.Becauseof limited seating, we encourage everyone to please RSVP ataascp.net andto register.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatmentand prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientistsand the public in diseases that can be caused by physiological dysfunction that areameliorableto medical treatment.

For further information, please contact Marie Barbaat AASCP 305-891-4686 and you can also visit us at http://www.aascp.net.

Related Links AASCP Safety guidelinesAASCP website / registration

Press Release Service by Newswire.com

Original Source: American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public

Originally posted here:
American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public - Valdosta Daily Times

Report Scope: The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries.

New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell and Regenerative Therapy Market" - https://www.reportlinker.com/p05791357/?utm_source=GNW The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography.Based on product the market is segmented into therapeutic products, cell banking, tools and reagents.

The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes: - 28 data tables - An overview of global markets for stem cell and regenerative medicines - Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 - Details of historic background and description of embryonic and adult stem cells - Information on stem cell banking and stem cell research - A look at the growing research & development activities in regenerative medicine - Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals - Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024.

Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018.The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period.

The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications.New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future.

The biopharmaceutical companies are investing significantly in cell-based therapeutics.The government organizations are funding research and development activities related to stem cell research.

These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments.The market is impacted through adoption of stem cell therapy.

The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.Read the full report: https://www.reportlinker.com/p05791357/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Story continues

Clare: clare@reportlinker.comUS: (339)-368-6001Intl: +1 339-368-6001

See the article here:
Global Stem Cell and Regenerative Therapy Market - Yahoo Finance