American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND – Yahoo Finance

The Company and the Principal Investigator announce the addition of sub-investigators to the clinical trial team with extensive experience assessing and treating athletes and military personnel suffering from concussion injury, traumatic brain injury and Post Concussion Syndrome

EATONTOWN, NJ / ACCESSWIRE / October 3, 2019 / American CryoStem Corporation (OTC PINK:CRYO) a leading strategic developer, marketer and global licensor of patented adipose tissue-based cellular products and technologies for the Regenerative and Personalized Medicine industries, today announced the selection of three new Investigators to assist the Principal Investigator with CRYO's Phase I clinical study of ATcell ("Investigational Drug") as part of a single center study under a protocol entitled: ATcell Expanded Autologous, Adipose-Derived Mesenchymal Stem Cells Deployed via Intravenous Infusion for the Treatment of Post Concussion Syndrome (PCS) in Retired Military and Athletes.

Dr. Tal David, a Sport Medicine specialist and former NFL Head Team Physician for the San Diego Chargers, is going to be the Co-Principal investigator for the study. Sub-Investigators, Dr. Jason Bailie, PhD, Senior Clinical Research Director at the Defense and Brain Injury Center (DVBIC) at the Naval Hospital Camp Pendleton, CA and Dr. Ettenhofer, Director of Research Operations, Defense and Veterans Brain Injury Center (DVBIC) Naval medical Center, San Diego, CA were the key designers of the evaluation aspect of the study. This included advising on the pre-treatment evaluations to rule out complex psychological co-pathology as well as the outcome measures to follow the efficacy of the treatments, since the chronic injuries seen in athletes mirrors those issues found in the military with multiple concussions and blast injuries.

Under the direction of Dr. Hanson and Dr. David, the sub-investigators will be responsible for assessing and screening all applicants for participation and completing the ongoing assessment of each participant. These evaluations include patient physical and neuropsychological assessment, testing and screening, preparation of source documentation and collection of assessment results, and assistance with completion of the final study reports and publications. The surgical tissue collection, ATcell treatments and follow up clinical visits will be conducted at BioSolutions Clinical Research Center facility in Le Masa, CA. The Company is pleased to present a world class team with significant experience in chronic concussion syndromes.

PRINCIPAL INVESTIGATOR: Dr. Peter Hanson, MD, as previously announced on September 11, 2019, has been appointed as the Principal Investigator, for the Company's study. Dr. Hanson is also Medical Director of BioSolutions Clinical Research Center the clinical research facility engaged by the Company to conduct the Study. During Dr. Hanson's career he has participated in approximately 41 clinical studies of which he led 24 as principal investigator. His clinical studies have been sponsored by many of the biggest names in the pharmaceutical and biotech industry such as Pfizer, Sanofi, Bristol Meyers Squibb, Regeneron, Cytori and InGeneron.

CO-PRINCIPAL INVESTIGATOR: Dr. Tal David, M.D. is a board-certified orthopedic surgeon specializing in arthroscopic and sports medicine surgery, is certified in both Orthopedic Surgery and Orthopedic Sports Medicine. He practices in San Diego, CA as part of the Synergy Specialists Medical Group and the San Diego Stem Cell Treatment Center. He is a clinical faculty member of Orthopedic Surgery at UC San Diego and a faculty member of the San Diego Arthroscopy and Sports Medicine Fellowship. He has cared for injured athletes for 15 years and has served on the medical staff of various professional sports teams including more than 11 years as an NFL team physician. In addition to his private practice, he served as the former Head Team Physician for the San Diego Chargers NFL football team and is Medical Director for the San Diego Gulls, AHL hockey team.

SUB-INVESTIGATOR: Jason M. Bailie, Ph.D, is a neuropsychologist who serves as Senior Clinical Research Director of the Defense and Veterans Brain Injury Center (DVBIC), Naval Hospital in Camp Pendleton, CA. Dr. Baille currently directs research, clinical and educational activities and supervises research staff. He is responsible for clinical research program development, experimental design, human-subject recruitment, experimental procedural implementation, data acquisition, and statistical analysis for local and multi-site research studies. Additional duties include oversight of research compliance with Institutional Review Board for the Department of Navy, Department of Defense clinical research and human subject research policies; and facilitating grant acquisitions and management. He supervises a clinical staff of social workers and psychologists, educational outreach personnel and provides educational presentations and grand rounds to providers and service members in collaboration with DVBIC Regional Education Coordinator.

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SUB-INVESTIGATOR Mark L. Ettenhofer, Ph.D is a neuropsychologist and Director of Research Operations American Hospital Services Group (AHSG), Defense and Veterans Brain Injury Center (DVBIC), Naval Medical Center, San Diego (NMCSD): San Diego, CA.Dr. Ettenhofer's primary research focuses on the development and evaluation of novel approaches for neurocognitive assessment and rehabilitation of traumatic brain injury (TBI) for the US Navy. Dr. Ettenhofer currently directs DVBIC supported traumatic brain injury (TBI) research at NMCSD, including 8 approved protocols evaluating novel technologies for TBI assessment, methods for remediation of TBI-related impairment, natural history of TBI, and guidelines for progressive return to activity after injury. Additionally he provides programmatic review for DVBIC national research portfolio, supervises and mentors eight full-time staff members in research activities and is well published.

Overall, it is estimated that the cost of traumatic brain injuries (TBI) in the United States weighs in at $48.3 billion annually of which $31.7 billion is spent on hospitalization costs; an additional $16.6 billion is associated with costs attributed to fatalities. According to the Center for Disease Control, acute care and rehabilitation of brain injury patients in the United States costs about $9 billion to $10 billion per year. This does not include indirect costs to society and family caretakers due to lost productivity, work time and earnings, as well as costs linked to providing social services.

Brain Injury Cost

The Company stated that "The successful assembly of this expert team of investigators who are currently treating athletes and military personnel suffering from Concussive and Traumatic Brain Injury is a major step towards properly implementing, monitoring and effectively evaluating the treatment of the participants with ATcell. Working with this expert team will expand the Company's knowledge for treating concussion injury, traumatic brain injury and Post Concussion Syndrome and accelerate its ability to complete this Phase 1 study and provide the necessary input to continue our work toward final FDA approval of ATcell for Post Concussion Syndrome.

For further detailed Corporate or Regenerative Medicine information please visit:

http://www.americancryostem.com, request by email at info@americancryostem.com or gathering phone 732-747-1007

This press release may contain forward-looking statements, including information about management's view of American CryoStem Corporation's ("the Company") future expectations, plans and prospects. In particular, when used in the preceding discussion, the words "believes," "expects," "intends," "plans," "anticipates," or "may," and similar conditional expressions are intended to identify forward-looking statements. Any statements made in this press release other than those of historical fact, about an action, event or development, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other factors, which may cause the results of the Company, its subsidiaries and concepts to be materially different than those expressed or implied in such statements. Unknown or unpredictable factors also could have material adverse effects on the Company's future results. The forward-looking statements included in this press release are made only as of the date hereof. The Company cannot guarantee future results, levels of activity, performance or achievements. Accordingly, you should not place undue reliance on these forward-looking statements. Finally, the Company undertakes no obligation to update these statements after the date of this release, except as required by law, and also takes no obligation to update or correct information prepared by third parties that are not paid for by American CryoStem Corporation.

SOURCE: American CryoStem Corporation

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American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND - Yahoo Finance

Tenaya Therapeutics bags $92M to develop triple threat for heart disease – FierceBiotech

Tenaya CEO Faraz Ali(Tenaya Therapeutics)

Although heart disease remains the leading cause of death worldwide, its an area that hasnt seen as much interest or investment as other areas, and its treatments still focus on dealing with symptoms. With a multipronged approach and a fresh infusion of $92 million, Tenaya Therapeutics is trying to change that.

The heart is a complicated organ, and it can go wrong in different ways. Part of what weve learned the hard way is that prior approaches are not working, Tenaya CEO Faraz Ali told FierceBiotech.

Founded in 2016 by scientists from the Gladstone Institute in California and the University of Texas Southwestern Medical Center, Tenaya is going after the underlying causes of heart disease to head off heart failure. It raised $50 million in its series A round from The Column Group, which also pitched into its $92 million B round alongside the likes of Casdin Capital and GV.

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RELATED: GV leads $58.5M round for Verve, a startup looking to pit gene editing against heart attacks

In an age where companies are forming themselves around one platform thats disease-agnostic and can be applied to many different areas, South San Francisco-based Tenaya is doing the opposite. It's working on three different platforms for heart disease:regenerative treatments, gene therapies and precision medicines.

The company was founded with a big, bold mission to follow the science and use the right tool for the job, Ali said. If the problem is loss of cardiomyocytes (the muscle cells that make the heart beat), such as after a heart attack, we can look for a way to generate new myocytes, create new tissue and improve the ability of the heart to contract that way.

Thats where regenerative treatments come in. Tenaya's approach delivers transcription factors that can nudge heart fibroblasts, cells that play a role in scar formation after a heart attack, to become heart muscle.

If the problem stems from geneticsif a faulty gene doesnt cause heart muscle to die, but leads to an arrhythmia or scarring, stopping it from working properlythe solution is not to create new muscle, but to get the muscle thats there to work, Ali said. And thats where gene therapy, adding a healthy copy of a defective gene, comes in.

Finally, Tenayas taking a leaf out of the book of cancer drug developers: Its become quite the norm to look for therapies that work in a particular genetic background, he said. The companys precision medicine platform uses stem cell-derived heart muscle cells as disease models to identify new targets for heart failure and screen new drugs. Its first focus is on small molecules for the treatment of dilated cardiomyopathiesa group of conditions in which an enlarged heart chamber makes it less efficient at pumpingin genetically defined patient groups.

What the funding allows us to do is advance multiple promising projects from each of the platforms out of the pure research stage and into the clinic, Ali said. Tenaya hopes to move to human studies over the next few years.

RELATED: Renovacor bags $11M to push precision medicine for rare heart disease

Though the company isnt divulging just yet which targets its going after, Ali did say some of its programs are chasing more prevalent conditions such as heart attacks while others are looking at smaller, more defined populations.

Once we get a signal of efficacy and safety and advance into later-stage clinical development, we could potentially expand into larger, more prevalent indications, he said.

Tenaya has about 45 staffers who are mostly focused on research, early development and manufacturing. If all goes to plan, the company plans to double its size by the end of 2021, adding more employees in development and manufacturing.

Two-thirds of our platforms that were working on [gene therapy and regenerative treatments] are heavily dependent on viral vectors, adeno-associated viruses Everyone learned in the last decade or so that manufacturing is the Achilles heel of the gene therapy spaceit's difficult to do and highly technical, its nothing like small molecule manufacturing, Ali said. We made the decision to invest early, and well ahead of being in the clinic, to invest in manufacturing.

Tenaya isnt just doing it because other gene therapy players have proved it necessary. If it wants to go after larger heart disease populations rather than the smaller groups affected by rare disease, its going to need a lot of viral vector to deliver its treatments.

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Tenaya Therapeutics bags $92M to develop triple threat for heart disease - FierceBiotech

UW Medicine receives $50 million donation to start brain institute that will study addiction, Alzheimers and other brain disorders – Seattle Times

A pair of philanthropists from Bellevue have given $50 million to UW Medicine to create an institute focused on developing treatments for brain disorders such as addiction, depression and Alzheimers disease.

The money, donated by Lynn and Mike Garvey, will kickstart the development of the Garvey Institute for Brain Health Solutions.

This will be the second neuroscience-focused institute in Seattle. The Allen Institute for Brain Science examines how the brain works, while the new Garvey Institute will take a more clinical approach to brain health. The work done at each could mesh nicely together, said Dr. Jrgen Untzer, a professor and chair of UWs psychiatry and behavioral sciences department, which will be home to the Garvey Institute.

Garvey Institute researchers will build on work already being done at UW Medicine, and will also partner with scientists from within the University of Washington and other local health systems,Untzer said.

The goal is not only to develop brain solutions but to get them quickly put into practice, he said in an interview Wednesday.

In addition to clinical research on treatments for brain disorders, the money will also fund training efforts for scientists and researchers from different disciplines at the institute, as well as a place for them to work together.

These new programs will change the future of mental health and brain health in our region and beyond, Untzer said in a news release announcing the donation.

In its first five years, the Garvey Institute will focus on three main areas:cognitive aging and brain wellness, the effects of physical and emotional trauma on the brain, and addiction.

At some point, almost every family is affected by a brain-health problem such as depression, Alzheimers disease or addiction, Lynn Garvey said in the news release. These diseases are so common and so devastating, and we wanted to do something to help.

The Garveys, who declined to be interviewed, have previously donated money to UW Medicines Institute of Stem Cell and Regenerative Medicine, the psychiatry and behavioral sciences department, Harborview Medical Center, and the heart regeneration and gastroenterology programs.

Mike Garvey is the primary founder of Saltchuk, a Seattle-based family of transportation and distribution companies that reports a consolidated annual revenue of nearly $2.75 billion.

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UW Medicine receives $50 million donation to start brain institute that will study addiction, Alzheimers and other brain disorders - Seattle Times

Giving a Speed Boost to Nerve Regrowth – Technology Networks

Twenty million Americans suffer from peripheral nerve injuries, which can be caused by traumas such as combat wounds and motorcycle crashes as well as medical disorders including diabetes. These injuries can have a devastating impact on quality of life, resulting in loss of sensation, motor function and long-lasting nerve pain. The body is capable of regenerating damaged nerves, but this process is slow and incomplete.

Now, researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have discovered a molecular process that controls the rate at which nerves grow both during embryonic development and recovery from injury throughout life.

The study, led by senior author Samantha Butler and published in the Journal of Neuroscience, used experiments with mice to show that it is possible to accelerate peripheral nerve growth by manipulating this molecular process. The finding could inform the development of therapies that reduce the time it takes for people to recover from nerve injuries.

The human bodys nervous system is comprised of two components: the central nervous system, which includes the brain and spinal cord; and the peripheral nervous system, which encompasses all other nerves in the body. Peripheral nerves extend over long distances to connect limbs, glands and organs to the brain and spinal cord, sending signals that control movement via motor neurons, and relaying information such as pain, touch and temperature via sensory neurons.

Unlike the nerves in the brain and spinal cord, which are protected by the skull and vertebrae, the nerves of the peripheral nervous system have no such protection, leaving them vulnerable to injury. While the body has a mechanism to help peripheral nerves reestablish connections after injury, this process is slow; damaged nerves regrow at an average rate of just one millimeter per day.

The glacial pace of this recovery can take a tremendous toll on peoples lives, as they may have to live with impaired movement and sensation for many months or years.

People with severe peripheral nerve injuries often lose sensation, which makes them susceptible to further injury, and they lose mobility, which can lead to muscle atrophy, said Butler, who holds the Eleanor I. Leslie Chair in Pioneering Brain Research in the neurobiology department at the David Geffen School of Medicine at UCLA. The process of nerve regrowth can be extremely painful and if muscles have atrophied it requires a lot of physical therapy to regain function. My lab seeks methods to accelerate this healing process.

In a 2010 study in mice, Butler and her colleagues discovered they could control the rate at which nerves grow in the spinal cord during embryonic development by manipulating the activity of a gene called LIM domain kinase 1, or Limk1. Limk1 controls the rate of nerve growth by regulating the activity of a protein called cofilin. Cofilin plays a key role in a process known as actin polymerization, or treadmilling, which enables nerves to extend thread-like projections over long distances to form neural networks.

Butlers new paper builds on these findings by showing that Limk1 and cofilin also control the rate of growth of peripheral nerves during both development and regeneration.

We discovered that one of the first things a nerve does after injury is switch on all these early developmental molecules that controlled how it grew in the first place, said Butler, who is a member of the UCLA Broad Stem Cell Research Center. Its somewhat similar to how an adult in crisis might reach out to their childhood friends to renew themselves.

In preclinical tests using mouse models with peripheral nerve injuries, Butlers lab showed that this molecular process can be manipulated to make nerves grow faster. Specifically, they found that mice that were genetically engineered so that the Limk1 gene was removed exhibited a 15% increase in the speed of nerve regrowth following injury.

This is a modest improvement for a mouse but one that could translate into a major improvement for a human because our nerves have so much farther to grow, said Butler, who noted that nerves regrow at the same rate in both mice and humans.

This increased rate of nerve regrowth resulted in faster recovery of both motor and sensory functions as measured by how fast the injured mice regained the ability to walk and the sensation in their paws. This is significant because sensory function can take longer than motor function to recover after a traumatic injury, yet sensory function is critical to quality of life.

As a next step, Butler and her lab are using human stem cell-derived motor neurons to screen for drug candidates that could modify this molecular process and speed nerve regeneration in humans. They are also expanding the scope of their study by examining if adding more cofilin rather than inhibiting Limk1 could be even more effective in speeding up recovery from peripheral nerve injuries.

The experimental treatment model described above was used in preclinical tests only and has not been tested in humans or approved by the Food and Drug Administration as safe and effective for use in humans.

Reference

Frendo, M.E. et al. (2019) The cofilin/Limk1 pathway controls the growth rate of both developing and regenerating motor axons. The Journal of Neuroscience. DOI: https://doi.org/10.1523/JNEUROSCI.0648-19.2019

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.

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Giving a Speed Boost to Nerve Regrowth - Technology Networks

Opinion: Primate research is cruel, expensive, unnecessary and happening here – OregonLive.com

David Gomberg

Gomberg, D-Central Coast, represents House District 10 in the Oregon Legislature.

Behind tall fences and security gates, on a pristine wooded campus at the edge of Hillsboro lies the Oregon National Primate Research Center. School groups are invited to visit where they will see thousands of non-human primates in large airy cages or walled corrals happily socializing, playing and eating. What they will not see, a few steps away, are rows of buildings where these magnificent creatures macaques, baboons and monkeys are the subject of medical research, simply because they share 93 percent of the same DNA as we human primates.

Last summer I asked to see the research rooms with my legislative staff. Inside we found stacks of small, 3-by-3-foot cages. Residents, we were told, stayed in those cages, inside those windowless rooms, for up to three years. As we entered, one baby primate cowered behind her mother. Dont worry, the technician reassured her. Im not here to take your baby. Not today, I thought.

The primate research center is now being sued by the People for the Ethical Treatment of Animals to release videos of maternal nutrition research. According to PETA, in these experiments, pregnant monkeys are fed special diets. Their babies are later separated and deliberately frightened to test their response to stress. In a 2010 project, $750,000 was spent on similar research where technicians either stared at the infants to intimidate them or used a Mr. Potato Head doll to frighten them, PETA contends.

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U.S. Department of Agriculture inspection reports document ongoing problems: stress-based hair loss, burns from electric heating pads, incorrect injections, cages too small to sit up in, accidental strangulation by chains in the enclosure, death from being trapped by PVC pipes, deaths from stress induced riots", death from anesthesia mistakes, death from dehydration, death from being given a toxic substance and death from inattention during childbirth. Some of these mistakes resulted in fines exceeding $10,000, but curiously, these penalties are not listed among reports posted to the centers web page.

Since 2017, the research center has been cited at least 10 times for Animal Welfare Act violations, the inspection reports show. That is substantially more than any other such center in the United States during this same period, according to the animal rights group Stop Animal Exploitation Now.

Center administrators told me that, even during normal research, about 500 primates are killed annually in terminal protocols examining aging, AIDS, depression, infectious diseases, substance abuse and obesity. The sad fact is that one in 10 primates in Hillsboro will not survive the year.

What I saw inside the primate research center were young animals trained to extend their arms from cages for easy examination.

The scientists I met at the center are proud of their work. But not so proud it seems that they want to tell people what they do. Instead, they requested legislation to keep their names from the public. These protections are so broad that even the identity of the company delivering monkey chow to the center cannot be released.

These same scientists will argue they are doing important work that will improve or save lives. Perhaps so. But who measures the results? And why dont we Oregonians know more?

An analysis from the Legislative Fiscal Office details that the center, a unit of Oregon Health & Science University, receives no state money but over $50 million annually in federal grants and funds. We should all be asking if the money is well spent.

Is research on non-human primates relevant to human primates? Is research done here in Oregon duplicative of work done at one of the other six national primate centers? Are non-primate research technologies like computer modeling less expensive and more accurate? We wont learn the answer to these questions because the primate research industry is a multi-billion-dollar enterprise funded by and fueled by people invested in doing things tomorrow the same way they did yesterday.

Ethologists, geneticists, and other scientists have taught us that primates share many physiological and emotional characteristics with human beings. They have feelings like us, they suffer like us, and they have social relationships that are important to them just as our relationships are to us. As a nation, weve ended the use of chimpanzees in invasive medical experiments. Its time we extend that same policy of no-testing to all primates.

For those of us who believe primate research is unnecessary and unproductive, the answer is not passionate protests at the center gates. We can only end these cruel practices by pulling the federal financial plug. The time has come for fundamental changes in our research practices and the treatment of our primate cousins.

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Opinion: Primate research is cruel, expensive, unnecessary and happening here - OregonLive.com

Into the Future With CAR-T Cell Therapy – Curetoday.com

An early clinical trial participant likens his journey into the unknown to a space mission.

For my wife and me, part of that involved the decision to discuss starting a family. After all, my father, my uncle and I were the only men in our small family it was left to me to keep our name going.

Of course, it wasnt ideal timing for starting a family. It was eight years after losing my father, and I was facing liver failure, kidney failure, a feeding tube strapped to my face and significant weight loss 55 pounds, to be exact. I was in immense pain, both physical and emotional, and felt anything but myself.

Just before I received my first treatment of chemotherapy R-CHOP, short for Rituxan (rituximab), cyclophosphamide, Adriamycin (doxorubicin), Oncovin (vincristine) and prednisone my doctors asked if my wife and I planned to ever have a family of our own. If so, we would need to bank some sperm immediately. Considering my physical condition at the time, I was in no mood to fulfill this obligation to secure a future with children in it. Dont misunderstand me I wanted nothing more than to have a family with my wife but in that moment, I didnt think it would be possible. I thought I was dying.

With just minutes to spare, due to the distanceof the fertility clinic from where I was in the city, we took a chance on having a future. I couldnt have done any of this without the unconditional support of my wife, Rachel, who carried us both when I was unable to stand on my own.

Later that night, I received my first chemotherapy treatment. Three hours into it we got the call from our fertility doctor, telling us that she could definitely get us pregnant. I never cry, but Idid then. The cancer and the looming possibility of death didnt exist in the moment that we learned we still might have a chance to become parents. It was exactly what we needed to hear to get through that tough first night of chemo.

The journey wasnt easy. I endured two years of treatments that included chemotherapy, immunotherapy, autologous stem cell transplant, more chemotherapy and various medical trials that I kept failing. Through it all, I adopted the mindset of an astronaut. Like an astronaut, I had an obligation to my fellow man to discover the unknown in hopes of finding something that would save not just my life but also the lives of the patients after me. Like an astronaut, I didnt know if being launched would makeme a spectacle of fireworks or perhaps get me to my destination with no way to return. Or would I have the rare opportunity to complete my mission and get home safely, delivering something that would drive us all forward to a better life?

Thats the mission I set for myself during my treatment. It was a job, one I took passionately and wanted nothing more than to do well so that, as a result, all who followed would have a better chance at life. This was especially true becauseI was the fifth person in the world to be treated with chimeric antigen receptor-T cell therapy, more commonly referred to as CAR-T cell therapy. I was part of the JCAR017 clinical trial.

In the months leading up to the trial, not knowing what my fate would be, my wife and I decided to live for today and take the next steps toward becoming parents. I didnt truly think I would ever see the birth of my child. That was a very sad thought, but this was bigger than me; I was leaving a piece of me behind for my wife.Thankfully, the CAR-T cell therapy was a success and got me into remission. Shortly after that, my wife and I welcomed our daughter, Julia, into the world.

People always wonder, How should I interact with someone I care about who has cancer?

I think the answers easy: Give them a future. Give them something to look forward to. Plan things with them, because we all need a future to survive.

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Into the Future With CAR-T Cell Therapy - Curetoday.com

Walking in His Shoes – Curetoday.com

A man who received chimeric antigen receptor-T cell therapy shares his firsthand account.

Backer received a diagnosis of diffuse large B-cell lymphoma (DLBCL). It is the most common type of non-Hodgkin lymphoma, which affects nearly 75,000 people mostly men in the United States each year.

Despite multiple rounds of chemotherapy and a stem cell transplant, Backers disease kept relapsing. Then he participated in a clinical trial involving chimeric antigen receptor (CAR)-T cell therapy, which has left him cancer-free for almost three years. With this type of immunotherapy, a patients T cells are removed, altered in a lab and then reinfused in the hope that they will attack cancer cells.

Dr. Frederick L. Locke, a medical oncologist at Moffitt Cancer Center in Tampa, Florida, isco-lead investigator of the pivotal ZUMA-1 trial, which led to the Food and Drug Administration (FDA) approval of the second available CAR-T cell therapy, Yescarta (axicabtagene ciloleucel). During the National Comprehensive Cancer Network 2019 Annual Conference, Locke, Backer and Alix Beaupierre, a transplant nurse coordinator, took a 360-degree look at CAR-T cell therapy.

LIMITED OPTIONSOutcomes in refractory aggressive non-Hodgkin lymphoma are poor, Locke explained. Patientsare often treated upfront with combination chemotherapy, as Backer was. He initially wenton a chemotherapy regimen commonly known as R-CHOP Rituxan (rituximab), cyclophospha- mide, Adriamycin (doxorubicin), Oncovin (vincris- tine) and prednisone. He achieved a complete remission and went back to work.

We can cure up to about 60% of patients with initial chemotherapy, and thats pretty remarkable, Locke said. Unfortunately, 40% of patients either dont respond to chemotherapy or progress.

Backer was among that 40%. He relapsed about a year later and started on a new chemotherapy regimen with a planned autologous stem cell transplant, which would involve removing his own stem cells and later putting them back into his body to help fight the cancer. This treatment plan cures only about 5% of patients, Locke said. Prior to CAR-T cell therapy, more chemotherapy would have been next.

In the United States, two CAR-T therapies are available to patients with certain types of cancer. The first, Kymriah (tisagenlecleucel), was approved in August 2017 for patients up to 25 years old who have acute lymphoblastic leukemia that relapsed or did not go into remission with other treatments. Two months later, the FDA approved Yescarta to treat adult patients with certain types of large B-cell lymphoma who have not responded to or relapsed after at least two other kinds of treatment.In patients with DLBCL, durable responses the length of time that a partial or complete response is observed because of treatment have been seen in 40% of patients who received CAR-T cell therapy. We think we can cure about 15% of people, Locke said. We need these patients referred and referred early.

A NUCLEAR BOMBBacker first read about CAR-T cell therapy on clinicaltrials.gov, an online registry of all clinical trials that anyone can access to see what might be enrolling participants. Im the typeof person who needs a plan A and a plan B, Backer said.I received a plan A, but the plan B in the case of the transplant failing was not encouraging.

He reached out to Moffitt Cancer Center Tampa is not far from where he lived in Orlando, Florida to see if he was eligible. He wasnt. In December 2015, Backer went ahead with the stem cell transplant. A few months later,he again relapsed with growths all over his body butthis made his participation in the ZUMA-1 clinical trial possible. Being a participant in a clinical trial is scary and daunting at the same time, he said. I remember sitting there with the transplant coordinator and they handed me a 27-page consent form, and I could barely read page one. Ijust wanted to sign. I was ready to sign anything right then and there.

Although potentially curative, CAR-T cell therapy is not without risk. Patients can develop two serious side effects. Cytokine release syndrome, caused by a large, rapid release of cytokines (small proteins importantin cell signaling) into the blood from immune cells affected by the immunotherapy, can be life-threatening. Neurological events, such as confusion, tremor and seizures, can also occur.

Despite lengthy discussions with his medical teamat Moffitt and reading the consent form, Backer said,he wasnt fully prepared for the coming side effects and recovery when he went forward with CAR-T therapy in June 2016. Thats when the nuclear bomb set off, he said. Within 12 hours of receiving the infusion, he experienced severe chills, violent shaking and a high fever, and also felt certain he was experiencing side effects that were affecting his brain.

About two days after Backer received the reinfusedT cells, the infectious disease team rushed him in for a CT scan. They found no infection and no signs of the cancer.

For me, it was a miracle treatment, Backer said.

Locke has been following patients enrolled in ZUMA-1 for more than two years and said that half are still alive.

BEING PREPAREDBacker admitted that going in with a full understanding of CAR-T cell therapy would have made the experience dramatically different.

After seeing patients and their loved ones in distress, care providers at Moffitt Cancer Center learned to smooth the process, Beaupierre said. For instance, the 27-page document has been broken down into one-page educational handouts and shorter consent forms. The team also created a flow sheet and that grew to a detailed patient calendar. All CAR-T therapy recipients also now have a dedicated nurse and social worker.

In addition to those resources, Backer said, peer-to-peer support would be helpful.

As CAR-T cell therapy continues to evolve and be explored in the treatment of other cancer types, experts are learning more about how it works and how to improve the process.

For Backer, quality of life has changed a bit. Initially, he received blood and platelet transfusions every two weeks for several months following CAR-T cell therapy. Although he is back to work full time, he runs the risk of being exposed to other diseases and viruses. Its always at the back of my mind, he said. I wear a mask and goggles at work but still get sick.

He spends his free time hiking and fishing and feels blessed to still be alive. It worked out for me, and here we are 33 months into this thing, Backer said.

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American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND – GuruFocus.com

The Company and the Principal Investigator announce the addition of sub-investigators to the clinical trial team with extensive experience assessing and treating athletes and military personnel suffering from concussion injury, traumatic brain injury and Post Concussion Syndrome

EATONTOWN, NJ / ACCESSWIRE / October 3, 2019 / American CryoStem Corporation (OTC PINK:CRYO) a leading strategic developer, marketer and global licensor of patented adipose tissue-based cellular products and technologies for the Regenerative and Personalized Medicine industries, today announced the selection of three new Investigators to assist the Principal Investigator with CRYO's Phase I clinical study of ATcell("Investigational Drug") as part of a single center study under a protocol entitled: ATcell Expanded Autologous, Adipose-Derived Mesenchymal Stem Cells Deployed via Intravenous Infusion for the Treatment of Post Concussion Syndrome (PCS) in Retired Military and Athletes.

Dr. Tal David, a Sport Medicine specialist and former NFL Head Team Physician for the San Diego Chargers, is going to be the Co-Principal investigator for the study. Sub-Investigators, Dr. Jason Bailie, PhD, Senior Clinical Research Director at the Defense and Brain Injury Center (DVBIC) at the Naval Hospital Camp Pendleton, CA and Dr. Ettenhofer, Director of Research Operations, Defense and Veterans Brain Injury Center (DVBIC) Naval medical Center, San Diego, CA were the key designers of the evaluation aspect of the study. This included advising on the pre-treatment evaluations to rule out complex psychological co-pathology as well as the outcome measures to follow the efficacy of the treatments, since the chronic injuries seen in athletes mirrors those issues found in the military with multiple concussions and blast injuries.

Under the direction of Dr. Hanson and Dr. David, the sub-investigators will be responsible for assessing and screening all applicants for participation and completing the ongoing assessment of each participant. These evaluations include patient physical and neuropsychological assessment, testing and screening, preparation of source documentation and collection of assessment results, and assistance with completion of the final study reports and publications. The surgical tissue collection, ATcell treatments and follow up clinical visits will be conducted at BioSolutions Clinical Research Center facility in Le Masa, CA. The Company is pleased to present a world class team with significant experience in chronic concussion syndromes.

PRINCIPAL INVESTIGATOR: Dr. Peter Hanson, MD, as previously announced on September 11, 2019, has been appointed as the Principal Investigator, for the Company's study. Dr. Hanson is also Medical Director of BioSolutions Clinical Research Center the clinical research facility engaged by the Company to conduct the Study. During Dr. Hanson's career he has participated in approximately 41 clinical studies of which he led 24 as principal investigator. His clinical studies have been sponsored by many of the biggest names in the pharmaceutical and biotech industry such as Pfizer, Sanofi, Bristol Meyers Squibb, Regeneron, Cytori and InGeneron.

CO-PRINCIPAL INVESTIGATOR: Dr. Tal David, M.D. is a board-certified orthopedic surgeon specializing in arthroscopic and sports medicine surgery, is certified in both Orthopedic Surgery and Orthopedic Sports Medicine. He practices in San Diego, CA as part of the Synergy Specialists Medical Group and the San Diego Stem Cell Treatment Center. He is a clinical faculty member of Orthopedic Surgery at UC San Diego and a faculty member of the San Diego Arthroscopy and Sports Medicine Fellowship. He has cared for injured athletes for 15 years and has served on the medical staff of various professional sports teams including more than 11 years as an NFL team physician. In addition to his private practice, he served as the former Head Team Physician for the San Diego Chargers NFL football team and is Medical Director for the San Diego Gulls, AHL hockey team.

SUB-INVESTIGATOR: Jason M. Bailie, Ph.D, is a neuropsychologist who serves as Senior Clinical Research Director of the Defense and Veterans Brain Injury Center (DVBIC), Naval Hospital in Camp Pendleton, CA. Dr. Baille currently directs research, clinical and educational activities and supervises research staff. He is responsible for clinical research program development, experimental design, human-subject recruitment, experimental procedural implementation, data acquisition, and statistical analysis for local and multi-site research studies. Additional duties include oversight of research compliance with Institutional Review Board for the Department of Navy, Department of Defense clinical research and human subject research policies; and facilitating grant acquisitions and management. He supervises a clinical staff of social workers and psychologists, educational outreach personnel and provides educational presentations and grand rounds to providers and service members in collaboration with DVBIC Regional Education Coordinator.

SUB-INVESTIGATOR Mark L. Ettenhofer, Ph.D is a neuropsychologist and Director of Research Operations American Hospital Services Group (AHSG), Defense and Veterans Brain Injury Center (DVBIC), Naval Medical Center, San Diego (NMCSD): San Diego, CA.Dr. Ettenhofer's primary research focuses on the development and evaluation of novel approaches for neurocognitive assessment and rehabilitation of traumatic brain injury (TBI) for the US Navy. Dr. Ettenhofer currently directs DVBIC supported traumatic brain injury (TBI) research at NMCSD, including 8 approved protocols evaluating novel technologies for TBI assessment, methods for remediation of TBI-related impairment, natural history of TBI, and guidelines for progressive return to activity after injury. Additionally he provides programmatic review for DVBIC national research portfolio, supervises and mentors eight full-time staff members in research activities and is well published.

Overall, it is estimated that the cost of traumatic brain injuries (TBI) in the United States weighs in at $48.3 billion annually of which $31.7 billion is spent on hospitalization costs; an additional $16.6 billion is associated with costs attributed to fatalities. According to the Center for Disease Control, acute care and rehabilitation of brain injury patients in the United States costs about $9 billion to $10 billion per year. This does not include indirect costs to society and family caretakers due to lost productivity, work time and earnings, as well as costs linked to providing social services.

Brain Injury Cost

The Company stated that "The successful assembly of this expert team of investigators who are currently treating athletes and military personnel suffering from Concussive and Traumatic Brain Injury is a major step towards properly implementing, monitoring and effectively evaluating the treatment of the participants with ATcell. Working with this expert team will expand the Company's knowledge for treating concussion injury, traumatic brain injury and Post Concussion Syndrome and accelerate its ability to complete this Phase 1 study and provide the necessary input to continue our work toward final FDA approval of ATcell for Post Concussion Syndrome.

For further detailed Corporate or Regenerative Medicine information please visit:

http://www.americancryostem.com, request by email at [emailprotected] or gathering phone 732-747-1007

This press release may contain forward-looking statements, including information about management's view of American CryoStem Corporation's ("the Company") future expectations, plans and prospects. In particular, when used in the preceding discussion, the words "believes," "expects," "intends," "plans," "anticipates," or "may," and similar conditional expressions are intended to identify forward-looking statements. Any statements made in this press release other than those of historical fact, about an action, event or development, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other factors, which may cause the results of the Company, its subsidiaries and concepts to be materially different than those expressed or implied in such statements. Unknown or unpredictable factors also could have material adverse effects on the Company's future results. The forward-looking statements included in this press release are made only as of the date hereof. The Company cannot guarantee future results, levels of activity, performance or achievements. Accordingly, you should not place undue reliance on these forward-looking statements. Finally, the Company undertakes no obligation to update these statements after the date of this release, except as required by law, and also takes no obligation to update or correct information prepared by third parties that are not paid for by American CryoStem Corporation.

SOURCE: American CryoStem Corporation

View source version on accesswire.com: https://www.accesswire.com/561800/American-CryoStem-Expands-Investigator-Team-with-Sub-Investigators-for-Post-Concussive-Syndrome-IND

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American CryoStem Expands Investigator Team with Sub-Investigators for Post Concussive Syndrome IND - GuruFocus.com

Oncternal Therapeutics Announces Opening of Phase 1b Expansion Cohort of Clinical Trial of Cirmtuzumab in Combination with Ibrutinib in Patients with…

SAN DIEGO--(BUSINESS WIRE)--Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that it has opened for enrollment a Phase 1b expansion cohort of its Phase 1/2 clinical trial of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib, in patients with mantle cell lymphoma (MCL). The decision to open an expansion cohort in MCL of the ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was based on favorable interim results from the dose-finding cohort of the trial, including that the combination was well-tolerated and that complete responses were observed in two heavily pre-treated patients who had received and failed multiple chemotherapy regimens and an autologous transplant, as well as either an allotransplant or CAR-T therapy, prior to participating in this clinical trial.

In June, the Company presented interim data at the American Society of Clinical Oncology (ASCO) annual meeting, including the preliminary results from the first six patients with MCL treated in the CIRLL clinical trial. One patient with MCL, who had relapsed following an allogeneic stem cell transplant, experienced a confirmed complete response (CR) after three months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be sustained and has been confirmed to be ongoing after completing 12 months of cirmtuzumab plus ibrutinib treatment. Following ASCO, a second confirmed CR occurred in a patient who had progressive disease after failing several different chemotherapy regimens, autologous transplant and CAR-T therapy. Additional data from this clinical trial will be presented at a future medical conference.

It is encouraging to see that the drug has been well tolerated as well as the early signal of efficacy of cirmtuzumab with ibrutinib in MCL, particularly the rapid and durable complete responses of the heavily pre-treated patients after three months of therapy, which is an unusually fast response in this patient population, said Hun Lee, M.D., Assistant Professor of Medicine in the Department of Lymphoma & Myeloma at the University of Texas MD Anderson Cancer Center, who is an investigator on the CIRLL clinical trial.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California San Diego (UC San Diego).

We are pleased to be opening the expansion cohort portion of the CIRLL clinical trial for patients with MCL, and continue to be encouraged by the interim results from this study for both patients with MCL and patients with chronic lymphocytic leukemia, for whom a randomized Phase 2 portion of the trial was opened in August, said James Breitmeyer, M.D., Ph.D., Oncternals President and CEO.

About the CIRLL Clinical Trial

The CIRLL clinical trial (CIRM-0001) is a Phase 1/2 trial evaluating cirmtuzumab in combination with ibrutinib in separate groups of patients with chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). Part 1 of the clinical trial was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 is a Phase 1b expansion cohort to confirm the RDR. Additional information about the CIRM-0001 clinical trial and other clinical trials of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

ROR1 is a potentially attractive target for cancer therapy because it is an oncofetal antigen a protein that confers a survival and fitness advantage when reactivated and expressed by tumor cells. When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

About Oncternal Therapeutics

Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on developing product candidates for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 receptor, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), and TK216, an investigational small-molecule compound that is designed to inhibit E26 transformation specific (ETS) family oncoproteins, that is being evaluated in a Phase 1 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. In addition, Oncternal has a program to develop a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. More information is available at http://www.oncternal.com.

Forward-Looking Information

Oncternal cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as may, will, should, expect, plan, anticipate, could, intend, target, project, contemplates, believes, estimates, predicts, potential or continue or the negatives of these terms or other similar expressions. These statements are based on the Companys current beliefs and expectations. Forward looking statements include statements regarding: Oncternals plans to present additional data from its ongoing Phase 1/2 clinical trial of cirmtuzumab; the expectation that Oncternal will be able to enroll patients into the Phase 1b expansion cohort; and Oncternals belief that favorable outcomes from the ongoing Phase 1 portion of the clinical trial support opening the Phase 1b portion. The inclusion of forward-looking statements should not be regarded as a representation by Oncternal that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in Oncternals business, including, without limitation: uncertainties associated with the clinical development and process for obtaining regulatory approval of cirmtuzumab and Oncternals other product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the Companys dependence on the success of cirmtuzumab and its other product development programs; the risk that interim results of a clinical trial do not necessarily predict final results and that one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data become available; the risk that unforeseen adverse reactions or side effects may occur in the course of developing and testing product candidates such as cirmtuzumab and Oncternals other product candidates; the Companys limited operating history and that fact that it has incurred significant losses, and expects to continue to incur significant losses for the foreseeable future; risks related to the inability of Oncternal to obtain sufficient additional capital to continue to advance the development of cirmtuzumab and its other product candidates; and other risks described in the Companys prior press releases as well as in public periodic filings with the U.S. Securities & Exchange Commission. All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Oncternal undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

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Oncternal Therapeutics Announces Opening of Phase 1b Expansion Cohort of Clinical Trial of Cirmtuzumab in Combination with Ibrutinib in Patients with...

AIVITA Biomedical to Present at Meeting on the Mesa and Other Investor, Oncology and Regenerative Medicine Conferences in October – PRNewswire

IRVINE, Calif., Oct. 1, 2019 /PRNewswire/ -- AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, today announced that it will be presenting at the following investor, oncology and regenerative medicine conferences in October:

Cell & Gene Meeting on the Mesa Company Presentation Time: October 2, 11:00 AM PST Location: Park Hyatt Aviara Resort Golf Club & Spa, Cognate in the Bioservices Ballroom, Carlsbad, CA

World Immunotherapy Congress Keynote Title: Enhancing patient responses with autologous cancer stem cell vaccine in combination with checkpoint inhibitors Time: October 16, 9:25 AM CET Location: Congress Centre Basel, Basel, Switzerland

BIO Investor Forum Company Presentation Time: October 23, 10:15 AM PST Location: Elizabethan A, The Westin St. Francis,San Francisco, CA

World Vaccine Congress Talk Title:Autologous Cancer Stem Cell Vaccines for Solid Tumor Cancers When: October 29, 3:45 PM CET Location: Palau de Congressos de Catalunya, Barcelona, Spain

About AIVITA Biomedical AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products. All proceeds from the sale of AIVITA's skin care products support the treatment of women with ovarian cancer.

SOURCE AIVITA Biomedical, Inc.

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AIVITA Biomedical to Present at Meeting on the Mesa and Other Investor, Oncology and Regenerative Medicine Conferences in October - PRNewswire