Earlier this week the CIRM ICOC Board awarded $14.5 million to fund three translational stage research projects (TRAN1), whose goal is to support early development activities necessary for advancement to a clinical study or broad end use of a potential therapy. Although all three projects have their distinct area of focus, they all utilize CAR-based cell therapy to treat a certain type of cancer. This approach involves obtaining T cells, which are an immune system cell that can destroy foreign or abnormal cells, and modifying them with a chimeric antigen receptor (CAR). This enables the newly created CAR-engineered cells to identify specific tumor signals and destroy the cancer. In the sections below we will take a deeper look at each one of these recently approved projects.
$2,663,144 was awarded to the University of California, San Francisco (UCSF) to develop specialized CAR-T cells that are able to recognize and destroy tumor cells in glioblastoma, an aggressive type of cancer that occurs in the brain and spinal cord. The specialized CAR-T cells have been created such that they are able to detect two specific signals expressed in glioblastoma. Hideho Okada, M.D., Ph.D. and his team at UCSF will test the therapy in mice with human glioblastoma grafts. They will be looking at preclinical safety and if the CAR-T cell therapy is able to produce a desired or intended result.
$5,949,651 was awarded to the University of California, Los Angeles (UCLA) to develop specialized CAR-engineered cells from human blood stem cells to treat multiple myeloma, a type of blood cancer. Lili Yang, Ph.D. and her team have developed a method using human blood stem cells to create invariant natural killer T (iNKT) cells, a special kind of T cell with unique features that can more effectively attack tumor cells using multiple mechanisms and migrate to and infiltrate tumor sites. After being modified with CAR, the newly created CAR-iNKT cells are able to target a specific signal present in multiple myeloma. The team will test the therapy in mice with human multiple myeloma. They will be looking at preclinical safety and if the CAR-iNKT cells are able to produce a desired or intended result.
Another $5,904,462 was awarded to UCLA to develop specialized CAR-T cells to treat melanoma, a form of skin cancer. Cristina Puig-Saus, Ph.D. and her team will use naïve/memory progenitor T cells (TNM), a subset of T cells enriched with stem cells and memory T cells, an immune cell that remains long after an infection has been eliminated. After modification with CAR, the newly created CAR-TNM cells will target a specific signal present in melanoma. The team will test the therapy in mice with human melanoma. They will be looking at preclinical safety and if the CAR-TNM cells are able to produce a desired or intended result.
Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) approved $8.5 million to continue funding of the Summer Program to Accelerate Regenerative Medicine Knowledge (SPARK) and Alpha Stem Cell Clinics (ASCC).
This past February, the Board approved continued funding for stem cell focused educational programs geared towards undergraduate, masters, pre/postdoctoral, and medical students. The SPARK program is an existing CIRM educational program that provides for a summer internship for high school students.
To continue support for SPARK, the Board has approved $5.1 million to be allocated to ten new awards ($509,000 each) with up to a five-year duration to support 500 trainees. The funds will enable high school students all across California to directly take part in summer research at various institutions with a stem cell, gene therapy, or regenerative medicine focus. The goal of these programs is to prepare and inspire the next generation of scientists and provide opportunities for California’s diverse population, including those who might not have the opportunity to take part in summer research internships due to socioeconomic constraints.
CIRM’s ASCC Network is a unique regenerative medicine-focused clinical trial network that currently consists of five medical centers across California who specialize in accelerating stem cell and gene-therapy clinical trials by leveraging of resources to promote efficiency, sharing expertise, and enhancing chances of success for the patients. To date, over 105 trials in various disease indications have been supported by the ASCC Network. While there are plans being developed for a significant ASCC Network expansion by some time next year, funding for all five sites has ended or are approaching the end of their current award period. To maintain the level of activity of the ASCC Network until expansion funding is available next year, the Board approved $3.4 million to be allocated to five supplemental awards (up to $680,000 each) in order to provide continued funding to all five sites; the host institutions will be required to match the CIRM award. These funds will support talent retention and program key activities such as the coordination of clinical research, management of patient and public inquiries, and other operational activities vital to the ASCC Network.
“Education and infrastructure are two funding pillars critical for creating the next generation of researchers and conducting stem cell based clinical trials” says Maria T. Millan, M.D., President and CEO of CIRM. “The importance of these programs was acknowledged in Proposition 14 and we expect that they will continue to be important components of CIRM’s programs and strategic direction in the years to come.”
The Board also awarded $14.5 million to fund three translational stage research projects (TRAN1), whose goal is to support early development activities necessary for advancement to a clinical study or broad end use of a potential therapy.
The awards are summarized in the table below:
Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma
CAR-Tnm cell therapy for melanoma targeting TYRP-1
HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma
It’s hard enough trying to follow the movements of individuals in a crowd of people but imagine how much harder it is to follow the movements of stem cells, crowded into a tiny petri dish. Well, researchers at the Gladstone Institutes in San Francisco have done just that.
In a CIRM-funded study ($5.85M) Dr. Todd McDevitt and his team created a super smart artificial intelligence way of tracking the movements of hundreds of stem cells growing together in a colony, and even identify “leaders” in the pack.
In our bodies groups of stem cells are able to move in specific ways to form different organs and tissues when exposed to the right environment. Unfortunately, we are still trying to learn what “the right environment” is for different organs.
In a news release, McDevitt, the senior author of the paper published in the journal Stem Cell Reports, says this method of observing cells may help us better understand that.
“If I wanted to make a new human heart right now, I know what types of cells are needed, and I know how to grow them independently in dishes. But we really don’t know how to get those cells to come together to form something as complex as a heart. To accomplish that, we need more insights into how cells work cooperatively to arrange themselves.”
Normally scientists watch cells by tagging them with a fluorescent marker so they can see them under a microscope. But this is slow, painstaking work and not particularly accurate. This new method used a series of what are called “neural networks”, which are artificial intelligence (AI) programs that can detect patterns in the movements of the cells. When combined together the networks proved to be able to track the movement of 95 percent of the cells. Humans by comparison can only manage up to 90 percent. But the nets were not only sharper, they were also faster, much faster, some 500 times faster.
This enhanced ability to watch the cells showed that instead of being static most of the time, as had previously been thought, they were actually on the move a lot of the time. They would move around for 15 minutes and then take a breather for ten minutes (time for the stem cell equivalent of a cup of tea perhaps).
Some cells moved around a lot in one direction, while others just seemed to shuffle around in the same area. Some cells even seemed to act as “leaders” while other cells appeared to be “followers” and shuffle along behind them.
None of this would have been visible without the power of the AI networks and McDevitt says being able to tap into this could help researchers better understand how to use these complex movements.
“This technique gives us a much more comprehensive view of how cells behave, how they work cooperatively, and how they come together in physical space to form complex organs.
Follow the Leader is not just a kids’ game anymore. Now it’s a scientific undertaking.
Following the passage of Proposition 14 CIRM has hired five new employees to help increase the team’s ability to respond to new challenges and responsibilities.
Prop 14, which was approved by voters in November 2020, gives CIRM $5.5 billion in new funding. Those funds mean CIRM can once again fund research from Discovery, through Translational and Clinical, as well as create new education and training programs. Prop 14 also adds new areas of focus for the Stem Cell Agency including creating an Accessibility and Affordability Working Group, expanding the Alpha Stem Cell Clinic network and creating new Centers of Excellence in underserved parts of California. To meet those new responsibilities the Agency has hired a highly talented group of individuals. Those include:
Kevin Marks is CIRM’s new General Counsel. Kevin studied Russian at college and originally wanted to be a diplomat, but when that didn’t work out he turned to the law. He became a highly accomplished, skilled advisor with global and domestic expertise and a history of delivering innovative legal and business results. He has served as Vice President and Head of Legal and Compliance at Roche Molecular Solutions, VP and General Counsel at Roche Molecular Diagnostics and VP and General Counsel at Roche Palo Alto, LLC.
“We are so delighted to have Kevin Marks join CIRM as a member of our executive Leadership Team,” says Maria T. Millan, MD, CIRM’s President and CEO. “He brings unique qualifications and critical skills during the formative phase and launch of our new strategic plan for California’s $5.5B investment in stem cell, genomics and regenerative medicine research and therapy development. As general counsel, he will oversee the legal department, human resources, grants management and operations at the Agency. Kevin has an established track record with global and domestic expertise and a history of delivering innovative legal and business solutions.”
“He is revered by his colleagues as an exceptional leader in his profession and in the community. Kevin is known for developing people as well as programs, and for promoting racial, ethnic and gender diversity.”
“I am incredibly honored to be joining CIRM at this stage of its journey,” says Marks. “I see the opportunity to contribute to positive patient outcomes–especially those patients with unmet medical needs–by working towards accelerating stem cell research in California as a member of the CIRM team as rewarding and perfectly aligned with my professional and personal goals.”
Pouneh Simpson as Director of Finance. Pouneh comes to CIRM from the Governor’s Office of Emergency Services in California, where she served as the Recovery Financial Administration Chief. At OES she worked with local, state, and federal government stakeholders on disaster recovery planning, exercises, and grant administration, specifically, overseeing the grant processing of all disaster recovery activity.
Prior to that Pouneh worked as the Chief Financial Officer of the Veterans Homes, where she managed finances at eight Veterans Homes with over 2,800 positions and $365 million in General Fund support. She also led the writing of legislation, regulations, policies and procedures for Cal Vet, overhauling the business and financial portions of eight Veterans Homes.
Mitra Hooshmand, PhD. as Senior Science Officer for Special Projects and Initiatives. Mitra joins CIRM after more than five years of leadership experience at Americans for Cures, a stem cell advocacy group. During this time, she mobilized hundreds of stakeholders, from scientists to national and local patient advocacy organizations, to generate support for CIRM’s mission.
Mitra has a PhD. in Anatomy and Biology from the University of California at Irvine. She also worked as a Project Scientist at the Sue and Bill Gross Stem Cell Research Center at UC Irvine, where she conducted and published academic and industry-partnered research in studies investigating the therapeutic benefit of human neural stem cell transplantation in preclinical models of spinal cord injury and aging.
Vanessa Singh, as Human Resources Manager. Vanessa has 15 years of experience working for the state of California, working at the Departments of General Services, Insurance and Human Services. In those roles she gained experience in performing, processing, developing, implementing, and advising on many personnel aspects such as compensation, benefits, classifying positions, recruitment and hiring, salary structure (exempt and civil service), organization structure, and retirement.
When COVID struck Vanessa stepped up to help. She worked as a Case Investigator for San Bernardino Local Health Jurisdiction, Department of Public Health, doing contact tracing. She talked to people diagnosed with coronavirus and collected information on people they may have had close contact with who may have been exposed to the virus.
Claudette Mandac as Project Manager Review. Claudette has more than seven years’ experience with UCSF’s Human Research Protection Program. In that role she prepared protocols for scientific, regulatory and ethical review, pre-screening submissions to ensure they were complete and consistent, and then routing them to the appropriate reviewers for administrative, expedited or Committee review. She also managed an Institutional Review Board Committee, preparing and distributing protocols for review by designated scientific and nonscientific reviewers, coordinating meetings, recruiting and training members, and maintaining records of conflicts of interest. At UCSF she annually helped process up to 3,000 IRB modifications, continuing reviews, and post-approval safety reports for domestic and international socio-behavioral or biomedical research.
Claudette has two degrees from U.C. Berkeley; one in Arts and History and another in Science, Conservation and Resource Studies.
All the cells in your body work together and each can have a different role. Their individual function not only depends on cell type, but can also depend on their specific location and surroundings.
A CIRM supported and collaborative study at the Gladstone Institutes, UC San Francisco (UCSF), and UC Berkeley has developed a more efficient method than ever before to simultaneously map the specialized diversity and spatial location of individual cells within a tissue or a tumor.
The technique is named XYZeq and involves segmenting a tissue into microscopic regions. Within each of these microscopic grids, each cell’s genetic information is analyzed in order to better understand how each particular cell functions relative to its spacial location.
For this study, the team obtained tissue from mice with liver and spleen tumors. A slice of tissue was then placed on a slide that divides the tissue into hundreds of “microwells” the size of a grain of salt. Each cell in the tissue gets tagged with a unique “molecular barcode” that represents the microwell it’s contained in, much like a zip code. The cells are then mixed up and assigned a second barcode to ensure that each cell within a given square can be individually identified, similar to a street address within a zip code. Finally, the genetic information in the form of RNA from each cell is analyzed. Once the results are obtained, both barcodes tell the researchers exactly where in the tissue it came from.
The team found that some cell types located near the liver tumor were not evenly spaced out. They also found immune cells and specific types of stem cells clustered in certain regions of the tumor. Additionally, certain stem cells had different levels of some RNA molecules depending on how far they resided from the tumor.
The researchers aren’t entirely sure what this pattern means, but they believe that it’s possible that signals generated by or near the tumor affect what nearby cells do.
In a press release, Alex Marson, M.D., Ph.D., a senior author of the study, elaborates on what the XYZeq technology could mean for disease modeling.
“I think we’re actually taking a step toward this being the way tissues are analyzed to diagnose, characterize, or study disease; this is the pathology of the future.”
The full results of the study were published in Science Advances.
Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #6 was Accelerate.
Ever wonder how long it takes for a drug or therapy to go from basic research to approval by the US Food and Drug Administration (FDA)? Around 12 years on average is the answer. That’s a long time. And it can take even longer for stem cell therapies to go that same distance.
There are a lot of reasons why it takes so long (safety being a hugely important element) but when we were sitting down in 2015 to put together our Strategic Plan we wanted to find a way to speed up that process, to go faster, without in any way reducing the focus on safety.
So, we set a goal of reducing the time it takes from identifying a stem cell therapy candidate to getting an Investigational New Drug (IND) approval from the FDA, which means it can be tested in a clinical trial. At the time it was taking us around eight years, so we decided to go big and try to reduce that time in half, to four years.
Then the question was how were we going to do that? Well, before we set the goal we did a tour of the major biomedical research institutions in California – you know, University of California Los Angeles (UCLA) UC San Francisco, Stanford etc. – and asked the researchers what would help them most. Almost without exception said “a clearing house”, a way to pair early stage investigators with later stage partners who possess the appropriate expertise and interest to advance the project to the next stage of development, e.g., helping a successful basic science investigator find a qualified partner for the project’s translational research phase.
So we set out to do that. But we didn’t stop there. We also created what we called Clinical Advisory Panels or CAPs. These consisted of a CIRM Science Officer with expertise on a particular area of research, an expert on the kind of research being done, and a Patient Representative. The idea was that CAPs would help guide and advise the research team, helping them overcome specific obstacles and get ready for a clinical trial. The Patient Representative could help the researchers understand what the needs of the patient community was, so that a trial could take those into account and be more likely to succeed. For us it wasn’t enough just to fund promising research, we were determined to do all we could to support the team behind the project to advance their work.
How did we do. Pretty good I would have to say. For our Translational stage projects, the average amount of time it took for them to move to the CLIN1 stage, the last stage before a clinical trial, was 4.18 years. For our CLIN1 programs, 73 percent of those achieved their IND within 2 years, meaning they were then ready to actually start an FDA-sanctioned clinical trial.
Of course moving fast doesn’t guarantee that the therapy will ultimately prove effective. But for an agency whose mission is “to accelerate stem cell therapies to patients with unmet medical needs”, going slow is not an option.
Elena Flowers, PhD, RN, an associate professor of physiological nursing at the University of California, San Francisco (UCSF) is joining the Board of the California Institute for Regenerative Medicine (CIRM), the state’s Stem Cell Agency.
Dr. Flowers was appointed to the Board by State Controller Betty T. Yee who said: “Ms. Flowers’ experience and express commitment to equitable health outcomes for California’s diverse communities will bring a valued perspective to the work ahead.”
Dr. Flowers is a member of the UCSF Institute for Human Genetics and the International Society of Nurses in Genetics. As a researcher her work focuses on genomics involving precision medicine and risk factors for cardiovascular health and type 2 diabetes. She is also a teacher and has lectured internationally on issues such as topics from racial disparities in Type 2 Diabetes to the implications of genomic technologies for the nursing workforce.
CIRM Board Chair, Jonathan Thomas, PhD, JD, welcomed the appointment: “Dr. Flowers brings a wealth of experience and expertise to our Board and, as a nurse, she will bring a different perspective to the work we do and help us in trying to better address the needs of underserved communities.”
“I am honored to have the opportunity to serve the citizens of California in this capacity,” says Dr. Flowers. “CIRM has ambitious goals, seeking to improve upon common limitations of public research agencies by its commitment to delivering meaningful findings and ultimately treatments for patients as rapidly as possible. I’m particularly committed to improving inclusion and access to these treatments across the entire diverse California population.”
Dr. Flowers got her undergraduate degree at UC Davis and then served as a research assistant at Zuckerberg San Francisco General Hospital. She then went on to get her MS and Doctor of Philosophy degrees at the UCSF School of Nursing.
In her spare time she has no spare time because she is the mother of two young daughters.
Over 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. The best available treatment for these patients is a kidney transplant from a genetically matched living donor. However, patients who receive a transplant must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer and diabetes. Despite these drugs, many patients still lose transplanted organs due to rejection.
To tackle this problem Medeor is developing a stem cell-based therapy called MDR-101. This is being tested in a Phase 3 clinical trial and it’s hoped it will eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.
The company takes blood-forming stem cells and immune cells from the organ donor and infuses them into the patient receiving the donor’s kidney. Introducing the donor’s immune cells into the patient creates a condition called “mixed chimerism” where immune cells from the patient and the donor are able to co-exist. In this way, the patient’s immune system is able to adapt to and tolerate the donor’s kidney, potentially eliminating the need for the immunosuppressive drugs that are normally necessary to prevent transplant rejection.
So how does getting RMAT designation help that? Well, the FDA created the RMAT program to help speed up the development and review of regenerative medicine therapies that can treat, modify, reverse, or cure a serious condition. If MDR-101shows it is both safe and effective RMAT could help it get faster approval for wider use.
In a news release Giovanni Ferrara, President and CEO of Medeor, welcomed the news.
“This important designation underscores the tremendous unmet medical need for alternatives to today’s immunosuppressive therapies for transplantation. We have the potential to help people live longer, healthier lives without the need for high dose and chronic immunosuppression and we thank the FDA for this designation that will assist us progressing as efficiently as possible toward a commercially available product.”
One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances in stem technology.
But there are equally exciting signs of progress that are not always so obvious to the untrained eye- those that we are privileged to witness behind the scenes at CIRM. These efforts don’t always lead to a splashy news article or even to a scientific publication, but they nonetheless drive the evolution of new ideas and can help steer the field away from futile lines of investigation. Dozens of such projects are navigating uncharted waters by filling knowledge gaps, breaking down technical barriers, and working closely with regulatory agencies to define novel and safe paths to the clinic.
These efforts can remain “hidden” because they are in the intermediate stages of the long, arduous and expensive journey from “bench to beside”. For the pioneering projects that CIRM funds, this journey is unique and untrod, and can be fraught with false starts. But CIRM has developed tools to track the momentum of these programs and provide continuous support for those with the most promise. In so doing, we have watched projects evolve as they wend their way to the clinic. We wanted to share a few examples of how we do this with our readers, but first… a little background for our friends who are unfamiliar with the nuts and bolts of inventing new medicines.
A common metaphor for bringing scientific discoveries to market is a pipeline, which begins in a laboratory where a discovery occurs, and ends with government approval to commercialize a new medicine, after it is proven to be safe and effective. In between discovery and approval is a stage called “Translation”, where investigators develop ways to transition their “research level” processes to “clinically compatible” ones, which only utilize substances that are of certified quality for human use.
Investigators must also work out novel ways to manufacture the product at larger scale and transition the methods used for testing in animal models to those that can be implemented in human subjects.
A key milestone in Translation is the “preIND” (pre Investigational New Drug (IND) meeting, where an investigator presents data and plans to the US Food and Drug Administration (FDA) for feedback before next stage of development begins, the pivotal testing needed to show it is both safe and effective.
These “IND enabling studies” are rigorous but necessary to support an application for an IND and the initiation of clinical trials, beginning with phase 1 to assess safety in a small number of individuals, and phase 2, where an expanded group is evaluated to see if the therapy has any benefits for the patient. Phase 3 trials are studies of very large numbers of individuals to gain definitive evidence of safety and therapeutic effect, generally the last step before applying to the FDA for market approval. An image of the pipeline and the stages described are provided in our diagram below.
The pipeline can be notoriously long and tricky, with plenty of twists, turns, and unexpected obstacles along the way. Many more projects enter than emerge from this gauntlet, but as we see from these examples of ‘works in progress”, there is a lot of momentum building.
Caption for Graphic:This graphic shows the number of CIRM-funded projects and the stages they have progressed through multiple rounds of CIRM funding. For example, the topmost arrow shows that are about 19 projects at the translational stage of the pipeline that received earlier support through one of CIRM’s Discovery stage programs. Many of these efforts came out of our pre-2016 funding initiatives such as Early Translation, Basic Biology and New Faculty Awards. In another example, you can see that about 15 awards that were first funded by CIRM at the IND enabling stage have since progressed into a phase 1 or phase 2 clinical trials. While most of these efforts also originated in some of CIRM’s pre-2016 initiatives such as the Disease Team Awards, others have already progressed from CIRM’s newer programs that were launched as part of the “2.0” overhaul in 2016 (CLIN1).
The number of CIRM projects that have evolved and made their way down the pipeline with CIRM support is impressive, but it is clearly an under-representation, as there are other projects that have progressed outside of CIRM’s purview, which can make things trickier to verify.
We also track projects that have spun off or been licensed to commercial organizations, another very exciting form of “progression”. Perhaps those will contribute to another blog for another day! In the meantime, here are a just a few examples of some of the progressors that are depicted on the graphic.
Project: stem cell therapy to enhance bone healing in theelderly
– Currently funded stage: IND enabling development, CLIN1-11256 (Dr. Zhu, Ankasa Regenerative Therapeutics)
Brain Neurotherapy Bio, Inc. (BNB) is pleased to announce the treatment of the first patient in its Parkinson’s gene therapy study. The CIRM-funded study, led by Dr. Krystof Bankiewicz, is one of the 64 clinical trials funded by the California state agency to date.
Parkinson’s is a neurodegenerative movement disorder that affects one million people in the U.S alone and leads to shaking, stiffness, and problems with walking, balance, and coordination. It is caused by the breakdown and death of dopaminergic neurons, special nerve cells in the brain responsible for the production of dopamine, a chemical messenger that is crucial for normal brain activity.
The patient was treated at The Ohio State University Wexner Medical Center with a gene therapy designed to promote the production of a protein called GDNF, which is best known for its ability to protect dopaminergic neurons, the kind of cell damaged by Parkinson’s. The treatment seeks to increase dopamine production in the brain, alleviating Parkinson’s symptoms and potentially slowing down the disease progress.
“We are pleased to support this multi-institution California collaboration with Ohio State to take a novel first-in-human gene therapy into a clinical trial for Parkinson’s Disease.” says Maria T. Millan, M.D., President and CEO of CIRM. “This is the culmination of years of scientific research by the Bankiewicz team to improve upon previous attempts to translate the potential therapeutic effect of GDNF to the neurons damaged in the disease. We join the Parkinson’s community in following the outcome of this vital research opportunity.”
CIRM Board Member and patient advocate David Higgins, Ph.D. is also excited about this latest development. For Dr. Higgins, advocating for Parkinson’s is a very personal journey since he, his grandmother, and his uncle were diagnosed with the disease.
“Our best chance for developing better treatments for Parkinson’s is to test as many logical approaches as possible. CIRM encourages out-of-the-box thinking by providing funding for novel approaches. The Parkinson’s community is a-buzz with excitement about the GDNF approach and looks to CIRM to identify, fund, and promote these kinds of programs.”
In a news release Dr. Sandra Kostyk, director of the Movement Disorders Division at Ohio State Wexner Medical Center said this approach involves infusing a gene therapy solution deep into a part of the brain affected by Parkinson’s: “This is a onetime treatment strategy that could have ongoing lifelong benefits. Though it’s hoped that this treatment will slow disease progression, we don’t expect this strategy to completely stop or cure all aspects of the disease. We’re cautiously optimistic as this research effort moves forward.”
Other trial sites located in California that are currently recruiting patients are the University of California, Irvine (UCI) and the University of California, San Francisco (UCSF). Specifically, the Irvine trial site is using the UCI Alpha Stem Cell Clinic, one of five leading medical centers throughout California that make up the CIRM Alpha Stem Cell Clinic (ASSC) Network. The ASSC Network specializes in the delivery of stem cell therapies by providing world-class, state of the art infrastructure to support clinical research.
For more information on the trial and enrollment eligibility, you can directly contact the study coordinators by email at the trial sites listed: