Stem Cell Clinic

Neural Stem Cell Transplantation Crawls Toward the Clinic – The Scientist

Pelizaeus-Merzbacher disease is a genetic malady that leaves neurons without their myelin coating. This deficit has devastating consequences for the boysits X-linkedwho have it. These children have severe developmental delay, so they have inability to walk, inability to talk and perform self-care, says Nalin Gupta, a professor of neurological surgery and pediatrics at the University of California, San Francisco (UCSF). Their neurologic function typically does not improve, and usually they actually die during childhood.

About a decade ago, the biotech firm StemCells Inc. was looking for a neurosurgeon to try out an intervention that might finally offer some help for these children. Because Gupta had experience conducting surgical clinical trials in kids with disabilities, the company approached him to see if he could transplant neural stem cells into the brains of boys with Pelizaeus-Merzbacher disease (PMD)an approach that researchers had considered promising for a range of conditions, but which had yet to be proven effective in a clinical trial for any disease. He agreed.

In 2012, Gupta and colleagues reported that four boys with PMD who had received pluripotent neural stem cells in a Phase 1 clinical trial tolerated the procedure, and imaging techniques that indirectly detect myelin indicated they may have had myelination in their brains one year following the transplant. This August, the researchers reported the results of a long-term follow-up study of those patientsall four are still alive at ages 10, 11, 12, and 13. Patients like these who have symptoms of the disease starting at birth typically die in their teens.

Although the researchers could not directly examine myelinationthat would require autopsiesthe imaging evidence is promising. There were some clinical improvements, too, although with such a small number of patients and no control group in a trial designed to examine safety, its hard to know whether they are attributable to the transplant.

We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease.

Nalin Gupta, UCSF

Guptas study is the latest report in a series of clinical trials on neural stem cell transplantation, in which pluripotent neural cells taken, in most cases, from the brains of aborted fetuses are expanded in the lab and then injected into the brains or spinal cords of patients with incurable neurological disorders. These include stroke, multiple sclerosis, ALS, spinal injury, and Parkinsons disease. But for all the effort that has gone in to testing these cells, none have been able to work themselves out of trials and into clinical practice.

When asked which of the human clinical trials have been most successful, Steven Goldman, a professor of neurology and neuroscience at the University of Rochester, replies, So far, none of them, right? To date, no Phase 2 trial to evaluate the efficacy of a neural stem cell treatment has been completed, he points out. And scientists, Gupta included, are less-than-ecstatic about the methods and outcomes of the clinical trials that have been done so far.

Goldman, who was not involved in the PMD experiment, calls it by far the most rigorous and well controlled. But that trial can go no further.

Gupta says he and his colleagues felt that there was sufficient evidence from the Phase 1 trial to justify a Phase 2. They cant proceed, however, because StemCells, the company that funded the research and provided the cells, closed in 2016. We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease, he says.

Research on other potential uses for neural stem cells are also affected by a lack of momentum. Theres somewhat of a pause in what people are doing in terms of stem cell therapeutics, says Gupta. Treatments for conditions such as spinal cord injury and stroke hold the most interest for their potential societal benefit, he says, but the complexity of the changes that occur when the brain or spinal cord are injuredmeaning regions composed of multiple cell types and networks of connections are just wiped outmake for a challenging repair. Were probably a long way from being able to transplant a structure that will recapitulate the three-dimensional organization and structure of the brain and spinal cord, he says. Trials for diseases with more specific defects might be more successful, he adds, such as multiple sclerosis, which like PMD involves demyelination.

According to Evan Snyder, the director of the Center for Stem Cells and Regenerative Medicine at the Sanford Burnam Prebys Medical Discovery Institute in La Jolla, California, there havent been enough trials, and certainly not enough under ideal circumstances, to know whether neural stem cell transplantation can be an effective treatment in humans. I think the field is too young to know right now if theyre effective. I think the field can just say that theyre safe, says Snyder. To be able to know whether the cells are effective, you really need to be able to put them into an optimal setting where their mechanism of action is optimal, and that kind of trial has never been done yet, he adds.

The animal research that laid the foundation for the PMD study, and other studies on diseases involving the loss or absence of myelination, took place in a mouse model called shiverer. These animals have a mutation that prevents their oligodendrocytes from making myelin, such that their neurons are badly insulated and cannot efficiently conduct electrical signals. The shiverer mice have problems with motor functions and self-care along with seizures. They also have a tremor, hence their name.

In 1999, Snyders lab reported in PNAS that injecting mouse neural stem cells into the brains of shiverer mice led to the remyelination of neurons as well as some tremor reductionmeasured by dipping each mouses tail in ink and noting the size of the stain it left on a piece of graph paper.

Using the model, Goldmans team later transplanted human glial progenitor cellswhich are derived from neural stem cellsinto shiverer mice, generating chimeras in which mouse neurons became insulated with human myelin. The chimeric mice, as Goldman reported in Cell Stem Cell in 2008, survived longer and had improved neurological phenotypes, including fewer seizures, compared with untreated controls. In 2012, StemCells Inc., in collaboration with researchers at Oregon Health & Science University and elsewhere, reported in Science Translational Medicine that transplanting shiverer mice with human neural stem cells resulted in remyelination in the brain. Also in that issue, Gupta and StemCells described the one-year results from the PMD trial, which used the same cells for transplantation.

Neurons (green) and glia (red) differentiated from human neural stem cells in culture. Nuclei are stained blue.

Evan Snyder

The PMD study was not the first trial launched by StemCells Inc. In 2006, the company launched a Phase 1 trial of neural stem cell transplantation in children with Batten disease, a fatal condition in which children are missing a lysosomal storage enzyme. That study was the first study authorized by the FDA for transplantation with neural stem cells into the brain, says Stephen Huhn, a biotech consultant and the former chief medical officer of the company.

The trial, which was completed in 2009, revealed the treatment to be safe, the authors reported in Journal of Neurosurgery: Pediatrics. Autopies on the brains of several kids who died of the disease during the study suggested that in some patients donor cells had both survived and migrated away from the subcortical and ventricular injection sites and into the basal ganglia, among other locations, Huhn says.

The fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.

Stephen Huhn, formerly of StemCells Inc.

The stem cells used for this and other StemCells trials were isolated from the brain of a single aborted fetus, expanded as balls of cells called neurospheres, and frozen for later use. Before injection into patients, the cells were thawed, cultured for two weeks, and dissociated, so that what was injected was no longer a neurosphere but a cluster of cells, according to Huhn. Because the neural stem cells were donor-derived, patients were given immunosuppressant drugs for several months following the transplant to prevent rejection.

Using the same procedure and stock of cells, Gupta and colleagues transplanted neural stem cells into the brains of the four boys with PMD in a Phase 1 trial that began in 2009 and ran through 2012the same trial whose long-term follow-up results came out this summer. One year after transplantation, diffusion tensor imagingan MRI-based technique that lets researchers indirectly observe myelinated axonsof the boys brains suggested that myelination had occurred.

From 2012 to 2015, the company ran a Phase 1/2 trial of neural stem cell transplantation for age-related macular degeneration. The treatment proved safe, and there was also evidence of a treatment effecta slowing of the retinal damage called geographic atrophy and improvements in visual functionin some patients, says Huhn.

At the same time, the firm was engaged in a Phase 1/2 trial of stem cell transplantation for patients with injuries to the thoracic region of the spine. The treatment proved safe, and Huhn notes that several participants seemed to have sensory improvement below the level of injury, which would imply that the stem cells were having a treatment effect.

But the companys run of auspicious results did not last forever: Its Phase 2 trial of neural stem cells to treat cervical spinal cord injury, which began in 2014, terminated two years later after an independent review of the emerging data found that the study was unlikely to show a statistically significant treatment effect, Huhn says. For that same reason, a follow-up study on the same patients also ended in 2016, he adds.

At that point, StemCells Inc. shut down. STAT reported that the reason was disappointing results from the spinal cord study.

Despite stopping, the companys work was not in vain, says Huhn, as it demonstrated that the approach is safe and might be worth pursuing. These are challenging disorders, Huhn says, adding that the fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.

According to Snyder, who was not involved in the work, the PMD trial suffered from the limitations of the clinical trials system. The unfortunate thing is the way clinical trials are designed, you only get a patient who has failed every other intervention, is very deep into the disease, and almost has no chance of anything changing the course, he says. This problem is not unique to the PMD trial but applies to all neural stem cell clinical trials to date, Snyder says. In the Batten disease trial, for instance, the patients had little hope of recovery, Snyder notes. Three of the six participants had died of their disease by the time the researchers stopped collecting data. Where stem cells are going to be most useful, ultimately, is going to be the early stages of a disease where there are regions that can be rescued, and where the cells are placed in a position where they can distribute themselves throughout the region that needs to be fixed. And no clinical trial has ever met those [criteria].

Although the follow-up PMD study revealed some myelination, there was not a lot of it, notes Goldman, who was not involved in the work. Theres some evidence for local remyelination around the region of the transplants, but there was nothing that was dispersed or broad, and these patients need really widespread remyelination, he says.

Goldman says he believes that there was not more widespread and robust myelination in the PMD patients because of the cell type used. While neural stem cells can give rise to oligodendrocytes, astrocytes, and neurons, they not very efficient at making oligodendrocytes, he says. And, he adds, they do not migrate much, which is necessary for them to have widespread effects. In contrast, human glial progenitor cells, which are produced from neural stem cells and give rise to both oligodendrocytes and astrocytes, are more migratory, says Goldman, and for this reason, the field has shifted away from neural stem cells and toward glial progenitor cells for transplantation. Goldman has trials of his own in the works using a neural stem cell derivative to treat multiple sclerosis and PMD through a company he cofounded, Oscine Therapeutics.

Other trials are currently underway. Researchers at Emory University and the University of Michigan, with funding from the company Neuralstem, have completed a Phase 1 study of neural stem cells to treat ALS and, according to ClinicalTrials.gov, a Phase 2 clinical trial is ongoing. Theres a Phase 2/3 trial of nasally delivered neural stem cells to treat Parkinsons disease enrolling in China. And theres an active Phase 1 trial for Parkinsons disease in Australia using human parthenogenetic neural stem cells derived from unfertilized eggs, rather than fetal tissue.

This year, Snyder received a California Institute for Regenerative Medicine (CIRM) grant to do work leading up to cell-based therapies for babies who are at risk for developing cerebral palsy due to perinatal asphyxia, or oxygen and blood deprivation in the womb, he says. Within the first few days of life, the researchers plan to do brain imaging to identify babies with regions of the brain where cells are injured but not dead, he says, then transplant neural stem cells. The injurys still very fresh and cells are sort of teetering on a knife edge. They can either go on to die or they can go on to live, and the [transplanted] stem cells make factors that push them in the direction to live, Snyder says. If that happens, the prediction is the babies will do much better.

Theres only a short window, when cells are damaged but not dead, during which a neural stem cell transplant can work, he adds. Other trials in older patients with more advanced disease, he suggests, may have missed their optimal treatment windows. Snyder predicts that if the right patients are transplanted with the right neural stem cells at the right time, I think then, under those circumstances, now youre going to start seeing not just safety but real efficacy.

Ashley P. Taylor is a New Yorkbased freelance reporter. Follow her on Twitter@crenshawseedsand read her work atashleyptaylor.com.

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Neural Stem Cell Transplantation Crawls Toward the Clinic - The Scientist

Exosome Procedures Now Being Included With the R3 Stem Cell Regenerative Aesthetics Training Course – Yahoo Finance

R3 Stem Cell, the nation's leader in regenerative medicine training, is now including exosome procedures in its regenerative aesthetics courses. There are still spots remaining for the November 15-16th, 2019 course in Scottsdale, Arizona.

SCOTTSDALE, Ariz., Oct. 31, 2019 /PRNewswire-PRWeb/ --R3 Stem Cell, the nation's leader in regenerative medicine training, is now including exosome procedures in its regenerative aesthetics courses. There are still spots remaining for the November 15-16th, 2019 course in Scottsdale, Arizona. Visit https://stemcelltrainingcourse.org/aesthetics or call (844) GET-STEM to register.

Exosomes are a huge buzzword in regenerative medicine and with good reason. Scientists and clinicians have found them to be extremely powerful at harnessing the body's repair processes.

At the R3 Stem Cell Regenerative Aesthetics Course, providers learn leading techniques for hair restoration, facial rejuvenation and ED. The training is hands on, including real patients and real biologics such as PRP therapy, stem cells and exosomes as well.

According to R3 CEO David Greene, MD, MBA, "Exosome therapy has been a very exciting addition to regenerative medicine. At our courses providers get hands on experience using them and also having procedures performed on them. That's the key in becoming the local leader in regenerative aesthetics!"

R3 Stem Cell has first rate trainers who have performed thousands of regenerative cases for hair restoration, facial rejuvenation and sexual health. In conjunction with fillers and PDO Threadlifts, the procedures have been amazing for patients looking and feeling younger.

Added Dr. Greene, "Compared with a surgery such as tummy tuck, facelift or hair grafting, these procedures involve no downtime, minimal risk and are much more cost effective. But they do require hands on training, and our courses provide a first rate experience!"

For the past eight years, R3's network of practices nationally have performed over 12,000 stem cell procedures and over 50,000 PRP therapies. The training courses have incorporated the best practice protocols and first rate biologics so providers receive training that immediately translates into practice.

There are presentations on sales and marketing along with "need to know" information on stem cells, exosomes and PRP biologics and how they work. Everything necessary to acquire patients, convert them to procedures and perform them is taught.

Spots are limited at the regenerative aesthetics training courses. Providers that will benefit from the training include MD, DO, RN, PA, NP and aesthetician injectors. Call (844) GET-STEM for registration, which is currently $1000 off.

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Exosome Procedures Now Being Included With the R3 Stem Cell Regenerative Aesthetics Training Course - Yahoo Finance

New NCCN Guidelines Debut to Manage Complications and Improve Readiness for Stem Cell Transplant Recipients – P&T Community

National Comprehensive Cancer Network expands resources to meet growing utilization of cell-based cancer treatments

PLYMOUTH MEETING, Pennsylvania, Oct. 30, 2019 /PRNewswire/ --Today, the National Comprehensive Cancer Network (NCCN) published new NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Hematopoietic Cell Transplantation (HCT), also known as stem cell transplant or historically as bone marrow transplant. This new resource provides step-by-step information on best practices in evaluating patients for hematopoietic cell transplantation and managing complications afterwards. This type of specialized treatment is increasingly common, occurring approximately 22,000 times a year in the United States in people with various malignancies, most commonly for blood-related cancers.1

"Establishing NCCN Guidelines for Hematopoietic Cell Transplantation is a key accomplishment in the management of blood cancers," said Ayman A. Saad, MD, Professor of Clinical Medicine, The Ohio State University Comprehensive Cancer Center James Cancer Hospital and Solove Research Institute, Chair of the NCCN Guidelines Panel for HCT. "The current version of the guidelines addresses both pre-transplant evaluation and the management of a common complication: graft versus host disease (GVHD). Given the diversity of practice and expertise, we believe these guidelines will provide a pivotal tool for learning about the continuously updated therapy landscape in HCT. We hope this will help streamline clinical practices and educate new generations of physicians-in-training."

The guidelines provide recommendations on how to evaluate a potential transplant recipient to determine if the patient is an appropriate candidate for the procedure, and how to best manage different manifestations of post-transplant GVHD. They reflect the latest evidence and consensus from foremost experts across the 28 leading academic cancer centers that comprise NCCN, including hematologists/oncologists, transplant-specific practitioners, and infectious disease specialists.

"These guidelines provide an algorithmic pathway for a systematic approach to allogeneic (donor) transplantation across several different cancer types including leukemia, lymphoma, and multiple myeloma," explained Marcos de Lima, MD, Professor of Medicine, Case Comprehensive Cancer Center and University Hospitals of Cleveland, Vice-Chair of the NCCN Guidelines Panel for HCT. "Thankfully, the number of blood and bone marrow donors has increased substantially in just the past decade. When you combine the National Marrow Donor Program registry adult donors with cord blood donors and relatives (matched and mismatched), we are now able to perform this potentially cancer-curing procedure on significantly more patients than we could in the past. That's why it's so important to set standards for preventing and treating common adverse events and infections."

"Early referral for consideration of HCT can be life-saving, so we strongly encourage all oncologists to take a look at these guidelines and refer any possible candidates to transplant centers for evaluation," said Alison W. Loren, MD, MSCE, Director, Blood & Marrow Transplant, Cell Therapy & Transplant Program, Abramson Cancer Center of the University of Pennsylvania, Member of the NCCN Guidelines Panel for HCT. "We also urge oncologists who may be caring for patients after HCT to familiarize themselves with the varied manifestations of GVHDa very common and significant post-transplant complicationand to consult with transplant providers to optimize their ongoing care. The guidelines explain how to diagnose and treat this condition in order to achieve the best possible outcomes."

The NCCN Guidelines for Hematopoietic Cell Transplantation are available free-of-charge for non-commercial use at NCCN.org and via the recently improved Virtual Library of NCCN Guidelines App for smartphone and tablet. NCCN will continue expanding blood cancer resources through continuous updates to the HCT guidelines, along with upcoming new NCCN Guidelines for Histiocytosis, Myeloid/Lymphoid Neoplasms, Pediatric B-Cell Lymphomas, and Pediatric Hodgkin Lymphoma.

About the National Comprehensive Cancer NetworkThe National Comprehensive Cancer Network (NCCN) is a not-for-profit alliance of 28 leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, efficient, and accessible cancer care so patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. By defining and advancing high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers around the world.

The NCCN Member Institutions are: Abramson Cancer Center at the University of Pennsylvania, Philadelphia, PA; Fred & Pamela Buffett Cancer Center, Omaha, NE; Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; City of Hope National Medical Center, Duarte, CA; Dana-Farber/Brigham and Women's Cancer Center | Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA; Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT; Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Mayo Clinic Cancer Center, Phoenix/Scottsdale, AZ, Jacksonville, FL, and Rochester, MN; Memorial Sloan Kettering Cancer Center, New York, NY; Moffitt Cancer Center, Tampa, FL; The Ohio State University Comprehensive Cancer Center - James Cancer Hospital and Solove Research Institute, Columbus, OH; O'Neal Comprehensive Cancer Center at UAB, Birmingham, AL; Roswell Park Comprehensive Cancer Center, Buffalo, NY; Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO; St. Jude Children's Research Hospital/The University of Tennessee Health Science Center, Memphis, TN; Stanford Cancer Institute, Stanford, CA; UC San Diego Moores Cancer Center, La Jolla, CA; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA; University of Colorado Cancer Center, Aurora, CO; University of Michigan Rogel Cancer Center, Ann Arbor, MI; The University of Texas MD Anderson Cancer Center, Houston, TX; University of Wisconsin Carbone Cancer Center, Madison, WI; Vanderbilt-Ingram Cancer Center, Nashville, TN; and Yale Cancer Center/Smilow Cancer Hospital, New Haven, CT.

Clinicians, visit NCCN.org. Patients and caregivers, visit NCCN.org/patients. Media, visit NCCN.org/news. Follow NCCN on Twitter @NCCN, Facebook @NCCNorg, and Instagram @NCCNorg.

1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides. 2018. Available at: https://www.cibmtr.org/ReferenceCenter/SlidesReports/SummarySlides/pages/index.aspx.

Media Contact: Rachel Darwin267-622-6624darwin@nccn.org

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New NCCN Guidelines Debut to Manage Complications and Improve Readiness for Stem Cell Transplant Recipients - P&T Community

Briggs undergrad researcher wins award at international conference – MSUToday

For senior Hasaan Hayat, a Lyman Briggs student with dual majors in neuroscience and human biology, the opportunity to work in a cutting-edge laboratory as an undergraduate researcher both confirmed his interests in technology and medicine and helped illuminate his career path.

For about a year, Hayat has been contributing to research in the lab of Ping Wang, an affiliate with MSUs Precision Health Program, or PHP. Precision medicine, a component of PHP, is a fairly recent field of biomedicine. This field develops personalized, patient-specific therapies and treatments, often incorporating tools like molecular imaging, nanoparticle technology and artificial intelligence to produce better outcomes for patients.

Through research like that of Wang, tools and technologies can be developed to detect disease sooner and treat it earlier, achieving better outcomes and reducing healthcare costs. PHP at MSU aims to transform the approach to healthcare from reactive to proactive by focusing on disease prediction, prevention and early detection.

Hayat has been interested in technology and human biology for as long as he can remember. After he joined Wangs lab, he became especially intrigued by the use of artificial intelligence, or AI, in the field of precision medicine.

As a child, I only dreamed of working on such technology myself due to its complexity and mass potential, but I also feared it, thanks to dystopian films such asTerminatorandiRobotwhere the sentient machine is always portrayed as the bad guy, he said. However, I find that AI can be a crucial, beneficial tool for analysis and monitoring of patients in a more modern field of medicine, specifically in oncology, radiology and stem-cell transplants.

Researching in Wangs lab has provided Hayat a unique platform to investigate the intersection of technology and biology. One specific study involved the application of deep learning in non-invasive imaging for monitoring tumor response to chemotherapy.

With help from Wang and Moore, Hayat put together an abstract of his work titled, Molecular imaging and analysis of uMUC1 expression levels in response to chemotherapy in an orthotopic murine model of ovarian cancer, and submitted it to the World Molecular Imaging Congress 2019, or WMIC 2019, in Montreal, Canada.

The WMIC 2019 program committee invited Hayat to present this research as an oral presentation, which is a high honor for the attendees. Hayats paper was one of the highest-rated abstracts at the conference and he won the Student Travel Award.

Hayat was grateful and energized by the experience of presenting at an international research conference.

The congress was phenomenal. I was able to hear about some amazing research and innovations in the field of medicine and molecular imaging/biology, he said. Networking with knowledgeable individuals from top institutions all over the world was a highlight of the event, and I am thankful to PHP and MSU for this opportunity.

Hayat was originally drawn to MSU for its many research opportunities, and specifically to Lyman Briggs College, because of its solid foundations in science.

I admire Lyman Briggs for its creative and innovative approach to STEM fields, and its focus on preparing students for success in graduate school, he said. The faculty at Lyman Briggs are very supportive and ensure that students have a clear understanding of core scientific concepts.

As for the future, his work with the Precision Health Program is inspiring him to go to medical school.

I aim to pursue an M.D.-Ph.D. after I graduate, a decision that has been heavily reinforced by the research I am doing at the Precision Health Program, and my mentor and PI, Dr. Wang, who himself is an M.D.-Ph.D. I salute the cutting-edge work that is performed here, he said. In the future, it is a dream and vision of mine to bring novel, innovative therapies and technologies such as AI and nanomedicine to the clinic in order to provide tools for physicians to use and to improve patient outcomes.

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Briggs undergrad researcher wins award at international conference - MSUToday

Delivering the promise of regenerative medicine – PMLiVE

The question remains unanswered as to whether a peer-to-peer collaborative model will prosper where medtech companies that are in some instances one step ahead of big pharma in terms of drug development are happy to be a third- party provider to big pharma that have the budgets and networks to truly deliver the regenerative medicine revolution.

In a recent document published by the UK government in response to the Regenerative Medicine Inquiry by the House of Commons Science and Technology Committee, policymakers stressed the importance of commercialising new therapies to meet the changing needs of the health sector.

In the UK, the Regenerative Medicine Expert Group (RMEG) has been tasked with developing an NHS regenerative medicine strategy to ensure the NHS is fully prepared to deliver innovative treatment and that regulations support and not hinder its delivery.

The Cell and Gene Therapy Catapult is also continuing to work to bridge the gap between translational research and commercialisation.

However, for the UK to be well-positioned to offer safe and effective regenerative therapies, a strategy is needed that covers the whole value chain from academic research, commercial development and clinical application.

The effect of Brexit on the UKs regenerative medicine sector remains unclear, but the UK has the opportunity to develop an independent framework outside the EU regulatory system to accelerate the development of new therapies and its economic potential while upholding the highest patient safety standards.

In any case, EU and UK regulators need to prioritise the standardisation of regulations governing manufacturing, quality control and the supply chain to keep up with advancements made by the FDA in the US.

Establishing an efficient supply chain for regenerative medicine

The promise of regenerative medicine requires an innovative look at the complete product life cycle, including the development of an efficient distribution network.

Once these novel drugs become mainstream, the entire healthcare ecosystem will have to adapt. Regulatory approval for any drug relies on it safely and successfully fulfilling its medical intent.

As such, information about supply chain management needs to be submitted to the regulator after the completion of phase 3 clinical trials, including packaging, labelling, storage and distribution.

The clinical supply chains required to deliver these therapies are arguably the most complex the industry has seen so far. Regenerative medicine is either personalised or matched to the donor-recipient. They are also highly sensitive to exogenous factors like time and temperature.

Advanced IT solutions and monitoring systems are being developed and employed to ensure end-to-end traceability. These are giving clinicians access to view the progress of therapies and their distribution in real-time and allow users to automatically schedule or amend material collections in line with manufacturing capacity, helping to keep the supply chain as agile as possible.

The live tissues and cells which form the basis of regenerative medicine products are highly sensitive and some have a shelf life of no more than a few hours.

Therefore, materials need to be transported from the site of harvest to manufacturing facilities, and from manufacturing facilities to medical institutions under strictly controlled conditions, within certain times and temperatures, according to cell and tissue requirements.

Temperature-controlled logistics solutions are vital to ensure a safe, effective and financially viable supply chain network for these high-value shipments. Cryopreservation is one technique increasingly being used to deliver medicines at optimum temperature using vapour phase nitrogen; however, many clinical settings remain ill-equipped to handle such equipment.

On-site production is an alternative manufacturing arrangement, particularly for autologous products which are derived from a patients own cells.

However, this throws up a number of compliance and infrastructure challenges, as the hospital would need to comply with a host of regulations including installing a Good Manufacturing Practice (GMP)-licensed clean room.

As a first-generation technology, stakeholders will have a greater tolerance for higher pricing... but only for a limited time period. By streamlining the currently very expensive manufacturing process and improving supply chain management, yields will automatically get larger and costs will slowly come down.

While there are many challenges in the road ahead, 2019 certainly appears to be the start of regenerative medicines move to the big time.

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Further Debate on T3 + T4 Combination Therapy at ATA Meeting – Medscape

CHICAGO The 89th American Thyroid Association (ATA) Annual Meeting gets underway this week, offering a 5-day dive into all things thyroid, ranging from a broad selection of debates to plenary sessions covering some of the most pressing and controversial issues facing the field, as well as a first-of-its-kind joint session with leading European thyroid groups.

The latter will cover the ongoing debate regarding combination therapy with levothyroxine [T4] and triiodothyronine [T3] for hypothyroid patients.

"The ATA meeting continues to grow: this year we have set a new abstract submission record, with 560 scheduled presentations from 39 countries," Program Committee Cochair Antonio Di Cristofano, PhD, told Medscape Medical News.

Cristofano is a professor in the Department of Developmental andMolecular Biologyat the Albert Einstein Cancer Center in New York City.

The highlighted oral abstractsession will feature research selected by the program committee as being the most important and will feature topics including CTNNB1, which "Inhibits Progression of BrafV600E-Induced Papillary Thyroid Cancer and Promotes Re-Differentiation and Iodine Uptake"; clinical trial results on the "Efficacy of Teprotumumab, an Insulin-Like Growth Factor-1 Receptor Antagonist Antibody, in Patients with Active Thyroid Eye Disease"; and "Heat-Inducible Mesenchymal Stem Cell (MSC)-Mediated Sodium Iodide Symporter (NIS) Gene Therapy."

And on Saturday afternoon, the ATA will pay tribute to Leslie De Groot, MD, praised as a leader in the field of thyroid medicine, who recently passed away. The session will focus on De Groot's many contributions to thyroidology.

The work of two prominent women will be profiled during the Clark T. Sawin Historical Vignette Women in Thyroidology plenary session on paradigm-shifting scientific contributions.

"I am very excited that we will be announcing at that session the creation of the first ATA lectureship to be named after a woman thyroidologist," ATA president Elizabeth Pearce, MD, told Medscape Medical News.

Other plenary sessions will include a lecture by Terry Fry, MD, during which he will discuss the hot topic of "CAR T-Cell Therapy in Blood Malignancies."

Fry, a professor of pediatrics, hematology, and immunology at Children's Hospital Colorado, in Denver, specializes in cell-based immunotherapy for pediatric leukemia.

And in another plenary lecture, Gil Mor, MD, will talk about "Pregnancy A Unique Immunological and Microbial Condition."

Mor is the John M. Malone Jr, MD, endowed professor, Department of Obstetrics and Gynecology at Wayne State University School of Medicine, Detroit, Michigan.

The meeting will also feature a first-of-its-kind joint symposium by the ATA, European Thyroid Association (ETA), and British Thyroid Association (BTA) that will link experts in Chicago and London by video to take on "Evidence Based Use of T3 + T4 Combinations," a topic of ongoing debate.

"The goal for this session is to explore the current evidence for benefit of combination therapy with both levothyroxine and T3 [triiodothyronine] for hypothyroid patients, an area of considerable controversy, and then to define the optimal parameters for a clinical trial to determine whether combination therapy is superior to levothyroxine alone in some or all hypothyroid patients," Pearce explained.

"I am very excited about the first joint ETA/BTA/ATA symposium," added Program Committee Cochair Mona M. Sabra, MD.

"This is obviously a very hot topic that willdiscuss optimal thyroid replacement strategies in the setting of renewed interest in combination T4 and T3 therapy, new research efforts, and data," she told Medscape Medical News.

"And a new formulation for this combination therapy...is apparently now available or soon to be available in the European market," said Sabra, a professor of clinical medicine, Endocrine Service, Department of Medicine, Memorial Sloan Kettering and Weill Cornell School of Medicine, New York City.

Pearce noted that among the most anticipated sessions at the meeting is the Arthur Bauman Clinical Symposium, which will feature a preview of new anaplastic thyroid cancer guidelines, including talks on ethical, surgical, and medical considerations in the management and treatment of this rare but aggressive cancer.

"Newly updated anaplastic thyroid cancer guidelines will be issued in late 2019 or early 2020. This session will provide an overview of the new guidelines," Pearce said.

Meanwhile, other symposia will address a number of important issues in the management of other thyroid cancers.

An Early Riser symposium on Friday, entitled "Cutting Edge Concepts in the Surgical Management of Thyroid Disease: The Future Is Here," will tackle topics including "Transoral Thyroid Surgery: State of Affairs 2019," "Radiofrequency Ablation (RFA) for Thyroid Nodules and Thyroid Cancer: Pipedream or Disruptive Technology?" and "Nerve Monitoring in Remote Access Surgery: Why It's an Imperative."

Another clinical symposium focusing on thyroid cancer will feature talks on "Low Risk Differentiated Thyroid Cancer (DTC) Patients: Who to Treat and Who to Watch," "Local Therapy (Laser, RF, MW, ETOH) for Recurrent Nodal DTC," and "TVDT as a Predictor of Prognosis and Treatment Response in Metastatic DTC."

And a symposium on the "Surgical Management of Difficult Multinodular Goiters: International Perspectives" will cover challenges and recommendations in their treatment.

There will also be a talk on the compelling scenario of "Thyroid Surgery By Flashlight: Situations in Low-Resource Environments."

Other sessions include "Thoracotomy in Substernal Goiter Always Necessary? Fact or Fiction?" "Minimizing Nervousness About the Nerve: Preservation and Monitoring in Goiter Surgery," and "There Are No Difficult Patients Only Difficult Patient Interactions."

Taking on the pressing issue of thyroid disease in children will be the "Pediatric Thyroid Forum Satellite Symposium 2019: Update on Pediatric Thyroid Disease." The hottest topics and controversies facing the thyroid field will be hashed out in a variety of debates, including "Controversies in Identifying RAI Refractory Thyroid Cancer" and "Remote Access Thyroid Surgery Update 2019: When? Why? How?"

Throughout the meeting, attendees can expect to network with as many, if not more, colleagues than ever, Cristofano said.

Although the finally tally is not in, "We are close to matching (or breaking) the all-time attendance record set last year in Seattle," he said.

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Further Debate on T3 + T4 Combination Therapy at ATA Meeting - Medscape

Cesca Therapeutics To Change Name And Ticker Symbol To Reflect New Strategic Focus – P&T Community

RANCHO CORDOVA, Calif., Oct. 31, 2019 /PRNewswire/ --Cesca Therapeutics Inc.(Nasdaq: KOOL), a market leader in automated cell processing for regenerative medicine, today announced that the company will change its name to ThermoGenesis Holdings, Inc. ("ThermoGenesis") in order to better reflect its new strategic focus on becoming a key solution provider for cell manufacturing tools and services in the cell and gene therapy markets. In conjunction with the name change, the company will begin trading under the new Nasdaq ticker symbol, THMO, effective Friday, November 1, 2019. In addition, the company's common stock will trade under a new CUSIP number, 88362L100, beginning on Friday, November 1, 2019.

ThermoGenesishas been a well-respected brand for automated cell separation and cryostorage technologies in the stem cell and cord blood industry for nearly three decades. In recent years, the company has successfully launched a series of new devices, including the CAR-TXpress cellular processing platform. Recognizing the potential of its proprietary technologies, the company is committed to further expanding the development of tools and services in the global cell and gene therapy market. The company has also decided to terminate its current clinical programs in the regenerative medicine field in order to focus exclusively on becoming a preferred solution provider for the global cell and gene therapy market. In doing so, the company has most recently entered a definitive agreement with HealthBanks Biotech (USA) to form a joint venture named ImmuneCyte Life Sciences Inc. to initiate immune cell banking services in the U.S. using the company's proprietary CART-Xpress platform.

"ThermoGenesis will continue to take advantage of its proprietary technology in the automated cell processing field and focus on developing the next generation of CMO/CDMO services," said Dr. Chris Xu, Chairman and Chief Executive Officer of the company. "Our name and ticker change reflect the company's streamlined business focus toward addressing critical unmet manufacturing needs in the global cell and gene therapy market.

"Since its inception in 1986, the company has developed and commercialized novel cell processing systems and devices that have provided global researchers, biomanufacturing organizations and clinicians with highly efficient cell processing devices that have helped them to deliver better outcomes in various applications in the cell and gene therapy field," said Phil Coelho, the company's original Founder and Chief Technology Officer. "Our present focus will allow us to continue to be at the forefront to help drive future development of CAR-T cell therapeutics and other cell and gene therapy products and make them more accessible to the patients."

About ThermoGenesis Holdings, Inc.ThermoGenesis Holdings, Inc., formerly known as Cesca Therapeutics Inc.,develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. The company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally closed CAR-TXpressplatform, which streamlines the manufacturing process for the emerging CAR-T immunotherapy market. For more information about ThermoGenesis, pleasevisit: http://www.cescatherapeutics.com.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz,Rx Communications917-322-2216pschwartz@rxir.com

View original content:http://www.prnewswire.com/news-releases/cesca-therapeutics-to-change-name-and-ticker-symbol-to-reflect-new-strategic-focus-300948861.html

SOURCE Cesca Therapeutics Inc.

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Cesca Therapeutics To Change Name And Ticker Symbol To Reflect New Strategic Focus - P&T Community

Stem Cell And Regenerative Therapy Market Size by Type, Product, Application & Market Opportunities 2023 – Guru Online News

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The global stem cell and regenerative medicines market should grow from $21.8 billion in 2019 to reach $55.0 billion by 2024 at a compound annual growth rate (CAGR) of 20.4% for the period of 2019-2024.

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. The current report offers a detailed analysis of the stem cell and regenerative medicines market.

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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.

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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.

Report Analysis@https://www.trendsmarketresearch.com/report/analysis/BCC/global-stem-cell-and-regenerative-therapy-market

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Stem Cell And Regenerative Therapy Market Size by Type, Product, Application & Market Opportunities 2023 - Guru Online News