Author: Geoffrey Lomax Dr. PH

Expanding CIRM’s Alpha Clinics Network to deliver transformative regenerative medicine treatments 

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Almost every day, we hear new reports from the thousands of regenerative medicine clinical trials globally sponsored by hundreds of companies and academic researchers. The California Institute for Regenerative Medicine (CIRM) is a leader in this space supporting some of the most advanced cell and gene therapy clinic trials for a variety of unmet medical needs. With all this current activity, it’s easy to forget that there were only a handful of clinical trials going on just seven years ago. 

A New System for Delivering Treatments 

In 2015, CIRM’s leadership recognized that we were on the cusp of introducing an array of new regenerative medicine clinical trials. However, there was one big concern—the existing clinical delivery systems had limited experience and capacity for managing these new and comparatively complex clinical trials. Cell and gene therapy regenerative medicine treatments require new systems for manufacturing, processing, and delivering treatments to patients.  

In anticipation of the need for clinical bandwidth to support clinical trials, CIRM funded a network of California medical centers to develop teams dedicated to supporting regenerative medicine clinical trials. This network was called the Alpha Clinics Network. 

Since 2015, the Alpha Clinics Network has grown to include six academic medical centers in California. The Network has treated over a thousand patients in more than 100 clinical trials. CIRM frequently encounters companies and academic researchers that are specifically interested in bringing their research to California to be performed in the Alpha Clinics Network. These research sponsors cite expertise in manufacturing, process, delivery and regulatory compliance as the Networks value proposition. One sponsor summed it up by indicating there are “fewer protocol deviations (errors)” in the Alpha Clinics. 

Expanding the Alpha Clinics Network 

As we enter 2022 with CIRM’s new five year strategic plan, a major aim is to create a broad network of medical centers capable of supporting diverse patient participation in clinical trials.  

As a first step in this effort, CIRM recently announced $80 million in funding to expand the Alpha Clinics Network. This funding is intended to expand both the scale and scope of the Network. This funding will allow the scale to grow from six medical center to up to ten. Scale is important because as the number of clinical trials grow, there needs to be increased coordination and sharing of the workload. Alpha Clinic sites already collaborate to conduct individual clinical trials, and an expanded network will enable a greater number of trials to occur simultaneously. 

In addition, the Expansion Awards will enable the Network to expand the scope of its activities to address current needs of the field. These needs include new research platforms for conducting clinical trials. For example, sites are looking at integrating new types of genomic (DNA sequencing) tools to support improved diagnosis and treatment of patients.  

Also, CIRM is committed to funding research to treat neurological diseases. We anticipate network sites will develop advanced systems for delivering treatments to patients and evaluating the effectiveness of these treatments. In addition, sites will be developing training programs to address the growing workforce needs of the field of regenerative medicine. 

In 2015, CIRM invested in the Alpha Clinics Network which positioned California as a leader in supporting regenerative medicine clinical trials. In 2022, we will be expanding the Network with the aim of delivering transformative treatments to a diverse California and the world. The Network will fulfill this aim by expanding its reach in the state, developing advanced research planforms and technologies, and by training the next generations of researchers with the skills to deliver patient treatments. 

Watch a recording of our recent Alpha Clinics concept plan webinar: 

Regulated, Reputable and Reliable: FDA’s Taking Additional Steps to Advance Safe and Effective Regenerative Medicine Products

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Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research

In February 2020, CIRM presented a series of benchmarks for the responsible delivery of stem cell and regenerative medicine products. These benchmarks are outlined in the publication Regulated, reliable and reputable: Protect patients with uniform standards for stem cell treatments. In a nutshell, CIRM advocates for the delivery of regenerative medicine products in a context where:

  • The product is authorized by the Food and Drug Administration (FDA) and is overseen by an IRB or ethics board,
  • The treatment is delivered by qualified doctors, nurses, and technicians,
  • Treatment occurs at a clinical treatment center with expertise in regenerative medicine, and
  • There is ongoing monitoring and follow-up of patients.

On April 21 of 2021, Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research, indicated the FDA’s intent to ensure new regenerative medicine products are FDA-authorized. Specifically, the FDA will require product developers to obtain an Investigational New Drug or IND authorization. In his news release Dr. Marks says the agency is willing to exercise more enforcement of these rules should clinics or therapy producers fail to follow these guidelines.

“These regenerative medicine products are not without risk and are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they haven’t been adequately studied in clinical trials. We’ve said previously and want to reiterate here – there is no room for manufacturers, clinics, or health care practitioners to place patients at risk through products that violate the law, including by not having an IND in effect or an approved biologics license. We will continue to take action regarding unlawfully marketed products.”

IND authorization is particularly important as the agency pays close attention to how the product is produced and whether there is a scientific rationale and potential clinical evidence that it may be effective against the specific disease condition. All CIRM-funded clinical trials and all trials conducted in the CIRM Alpha Stem Cell Clinics Network must have IND authorization.

Regenerative medicine products are generally created from human cells or tissues. These products are frequently referred to as “living medicines.” The “living” nature of these products is what contributes to their remarkable potential to relieve, stop or reverse disease in a durable or sustainable manner.

The risk with unregulated products is that there is no assurance that they have been  produced in a quality controlled process or manner  where all components of the  injected material have been well characterized and studied for safety and efficacy for a given disease as well as a specific site in the body. In addition, there is no way to ensure that unregulated products meet standards or quality specifications such as ensuring that they have the active and beneficial component while making sure that they do not include harmful contaminants..  There have been documented examples of patients being severely injured by unregulated and inadequately characterized products. For example, in 2017 three Florida women were blinded by an unauthorized product.  Dr. George Daley, a stem cell expert and the Dean of Harvard Medical School, described the clinic operators as “charlatans peddling the modern equivalent of snake oil.”

To receive FDA authorization, detailed scientific data and well controlled clinical data are required to ensure safety and a demonstration that  the product is safe has the potential to improve or resolve the patient’s disease condition.

While it seems both important and self-evident that stem cell products be safe and effective and supported by evidence they can impact the patient’s disease condition, that doesn’t always happen. Unfortunately, too many patients have experienced unnecessary medical risks and financial harm from unauthorized treatments. CIRM applauds the FDA for taking additional steps to advance regenerative medicine products where the clinical benefits of such therapies outweigh any potential harms.

Enabling the Best Choice for Patients: The Need for Effective Patient Navigation

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Making sure patients get the treatment they need and not a “snake oil” substitute

We are at a turning point in regenerative medicine as the first wave of treatments have obtained FDA approval. But at the same time as we see the advance of scientifically rigorous research and regulated products we are also witnessing the continued proliferation of “unproven treatments.” This dueling environment can be overwhelming and distracting to individuals and families trying to manage life-threatening diseases.

How does a patient navigate this environment and get trusted and reliable information to help sort through their options?

CIRM teamed up with the CURA Foundation to organize a roundtable discussion intended to answer this question. The conversation included thought leaders involved in patient advocacy, therapy research and development, public policy and research funding. The roundtable was divided into three segments designed to discuss:

  1. Examples of state-of-the-art patient navigation systems,
  2. Policy, research and infrastructure needs required to expand navigation systems, and
  3. Communication needs for engaging patients and the broader community.

Examples of Navigation Systems:

This session was framed around the observation that patients often do not get the best medicines or treatments available for their condition. For example, in the area of cancer care there is evidence that the top 25% of cancers are not being treated optimally. Historic barriers to optimal treatment include cost pressures that may block access to treatments, lack of knowledge about the available treatments or the absence of experts in the location where the patient is being treated.  Much of the session focused on how these barriers are being overcome by partnerships between health care provides, employers and patients.

For example, new technologies such as DNA sequencing and other cell-based markers enable better diagnosis of a patient’s underlying disease. This information can be collected by a community hospital and shared with experts who work with the treating doctor to consider the best options for the patient. If patients need to access a specialty center for treatment, there are new models for the delivery of such care. Emphasis is placed on building a relationship with the patient and their family by surrounding them with a team that can address any questions that arise. The model of patient-centered care is being embraced by employers who are purchasing suites of services for their employees.

Patient advocacy groups have also supported efforts to get the best information about the patients’ underlying disease. Advocacy organizations have been building tools to connect patients with researchers with the aim of allowing secure and responsible sharing of medical information to drive the patient-centered development of new treatments. In a related initiative, the American Society of Hematology is creating a data hub for clinical trials for sickle cell disease. Collectively, these efforts are designed to accelerate new treatments by allowing critical data to be shared among researchers.

Essential Policy Infrastructure for Regenerative Medicine:

Session two dovetailed nicely with first discussion. There was continued emphasis on the need for additional evidence (data) to demonstrate that regenerative medicine treatments are having a significant effect on the patient’s disease. Various speakers echoed the need for patients in clinical trials to work with researchers to determine the benefits of treatments. Success stories with gene therapies in blood diseases were cited as proof of concept where treatments being evaluated in clinical trials are demonstrating a significant and sustained impact on diseases. Evidence of benefit is needed by both regulatory bodies that approve the treatments, such as the FDA, and by public and private payers / insurers that pay for treatments and patients that need to know the best option for their particular disease.

In addition, various speakers cited the continued proliferation of “unproven treatments” being marketed by for-profit centers. There was broad concern that the promotion of treatment where there is no evidence of effectiveness will mislead some patients and potentially harm the scientifically rigorous development of new treatments. Particularly for “stem cell” treatments, there was a desire to develop evaluation criteria that are clear and transparent to allow legitimate treatments to be distinguished from those with no evidence of effectiveness. One participant suggested there be a scorecard approach where specific treatments could be rated against specific indicators of safety, medical benefit and value in relation to alternative treatments. The idea would be to make this information widely available to patients, medical providers and the public to inform everything from medical decision making to advertising.

Communicating the Vision

The final session considered communication needs for the field of regenerative medicine. Patients and patient advocacy organizations described how they are using social media and other networking tools to share information and experiences in navigating their treatment options. Patient advocacy groups also described the challenges from providers of unproven treatments. In one case, a for profit “pop up” clinic had used the group’s videos in an attempt to legitimize their unproven treatment.

There was general consensus among the panelists that the field of regenerative medicine needs “trusted intermediaries” who can evaluate claims and help patients distinguish between high quality research and “snake oil”. These intermediaries should have the capacity to compile the most reliable evidence and utilize it to determine what options are available to patients. In addition, there needs to be shared decision making model where patients have the opportunity to explore options in an unbiased environment so they may make the best decision based on their specific needs and values.

Creating this kind of Navigation System will not be easy but the alternative is unacceptable. Too many vulnerable patients are being taken advantage of by the growing number of “predatory clinics” hawking expensive therapies that are both unproven and unapproved. We owe it to these patients to create a simple way for them to identify what are the most promising therapies, ones that have the highest chance of being both safe and effective. The roundtable discussion marked a starting point, bringing together many of the key players in the field, highlighting the key issues and beginning to identify possible solutions.

71 for Proposition 71

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Proposition 71 is the state ballot initiative that created California’s Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at leading California Medical Centers.

StateClinics_Image_CMYK

These teams work with academic and industry partners to support patient-centered for over 40 distinct diseases including:

  • Amyotrophic Lateral Sclerosis (ALS)
  • Brain Injury & Stroke
  • Cancer at Multiple Sites
  • Diabetes Type 1
  • Eye Disease / Blindness Heart Failure
  • HIV / AIDS
  • Kidney Failure
  • Severe Combined Immunodeficiency (SCID)
  • Sickle Cell Anemia
  • Spinal Cord Injury

These clinical trials have treated over 400 patients and counting. The Alpha Stem Cell Clinics are part of CIRM’s Strategic Infrastructure. The Strategic Infrastructure program which was developed to support the growth of stem cell / regenerative medicine in California. A comprehensive update of CIRM’s Infrastructure Program was provided to our Board, the ICOC.

CIRM’s infrastructure catalyzes stem cell / regenerative medicine by providing resources to all qualified researchers and organizations requiring specialized expertise. For example, the Alpha Clinics Network is supporting clinical trials from around the world.

Many of these trials are sponsored by commercial companies that have no CIRM funding. To date, the ASCC Network has over $27 million in contracts with outside sponsors. These contracts serve to leverage CIRMs investment and provide the Network’s medical centers with a diverse portfolio of clinical trials to address patients’’ unmet medical needs.

Alpha Clinics – Key Performance Metrics

  • 70+ Clinical Trials
  • 400+ Patients Treated
  • 40+ Disease Indications
  • Over $27 million in contracts with commercial sponsors

The CIRM Alpha Stem Cell Clinics and broader Infrastructure Programs are supporting stem cell research and regenerative medicine at every level, from laboratory research to product manufacturing to delivery to patients. This infrastructure has emerged to make California the world leader in regenerative medicine. It all started because California’s residents supported a ballot measure and today we have 71 clinical trials for 71.

 

 

Lessons Learned & Knowledge Shared: 3rd Annual Alpha Clinics Symposium Celebrates the Delivery of Stem Cell Treatments to Patients

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The CIRM Alpha Stem Cell Clinics (ASCC) Network was launched in 2015 to address a compelling unmet medical need for rigorous, FDA regulated, stem cell-related clinical trials for patients with challenging, incurable diseases. Since its inception, the Network has treated more than 200 patients in over 40 clinical trials at six leading California medical centers: UC San Diego, City of Hope, UCLA and UC Irvine, UCSF and UC Davis. That has enabled the Network to accumulate a wealth of experience and insight into how best to deliver treatments to patients, and each year it celebrates and showcases this knowledge at the CIRM Alpha Clinics Annual Symposium.

The Network is celebrating the 3rd anniversary of the ASCC Symposium on April 19th on the campus of the University of California at Los Angeles. This year’s theme is the Delivery of Stem Cell Therapeutics to Patients. Clinical investigators, scientists, patients, patient advocates, and the public will engage in thoughtful discussions on how novel stem cell treatments are now a reality. The symposium will address advancements and accomplishments of the ASCC Network in addition to developments and applications in the field of stem cell-based therapeutics. Treatments for cancer, HIV/AIDS, spinal cord injury and stroke will be featured. In addition, this year’s featured keynote speaker is David Mitchell President and Founder of Patients for Affordable Drugs.

The symposium is open to the public and is free. You can find the full agenda for the symposium here and registration can be found on the UCLA ASCC Eventbrite page. The event is highly interactive allowing participants opportunities to ask questions, network and learn about the latest developments in stem cell treatments.

Researcher and patient advocate panel at a past CIRM Alpha Clinic symposium: L to R: David Higgins, CIRM Board; David Parry, GSK; Catriona Jamieson, UCSD: John Zaia, City of Hope; John Adams, UCLA

Patient advocates speak up at the City of Hope 2nd Annual ASCC Network Symposium. (Image courtesy of the City of Hope)

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If you’re into stem cell manufacturing, this is the conference for you!

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GMP cells

Manufacturing stem cells: Photo courtesy of Pluristem

Fulfilling CIRM’s mission doesn’t just mean accelerating promising stem cell treatments to patients. It also involves accelerating the whole field of regenerative medicine, which involves not just research, but developing candidate treatments, manufacturing cell therapies, and testing these therapies in clinical trials.

Manufacturing and the pre-clinical safety evaluation of cell therapies are topics that don’t always receive a lot of attention, but they are essential and crucial steps in bringing cell therapies to market. Manufacturing cells that meet the strict standards for use in human trials is often a bottleneck where different methods of making pluripotent stem cells (PSCs) are used and standardization is not readily possible.

Abla-8Abla Creasey, Vice President of Therapeutics and Strategic Infrastructure at CIRM, notes:

“The field of stem cell research and regenerative medicine has matured to the point where there are over 900 clinical trials worldwide. It is critical to develop a system of effective regulation of how these stem cell treatments are developed and manufactured so patients can benefit from future treatments.”

To address this challenge, CIRM has teamed up the International Alliance for Biological Standardization to host the 4th Cell Therapy Conference on Manufacturing and Testing of Pluripotent Stem Cells on June 5-6th in Los Angeles, California.

WHAT

The aim of this conference is twofold. Speakers will discuss how product development programs can be moved forward in a way that will meet regulatory requirements, so treatments can be approved.

The conference will also focus on key unresolved issues that need to be addressed for the manufacturing and safety testing of pluripotent stem cell-based therapies and then make recommendations to inform the future national and international policies. The overall aim is to provide participants with a road map so new treatments can achieve the highest regulatory standards and be made available to patients around the world.

The agenda of the conference will cover four main topics:

  1. Learning from the current pluripotent space and the development of international standards
  2. Bioanalytics and comparability of therapeutic stem cells
  3. Tumorigenicity testing for therapeutic safety
  4. Pluripotent stem cell manufacturing, storage, and shipment Issues

Using this “big tent” approach, speakers will exchange knowledge, experience and expertise to develop consensus recommendations around stem cell manufacturing and testing.  New data in this area will be introduced at the conference for the first time, such as a multi-center study to identify and optimize manufacturing-compatible methods for cell therapy safety.

WHO

The conference will bring together leading experts from industry, academia, health services and therapeutic regulatory bodies around the world, including the US Food and Drug Administration, European Medicines Agency, Japan Pharmaceuticals and Medical Devices Agency, and World Health Organization.

CIRM and IABS encourage individuals and organizations actively pursuing the development of stem cell therapies to attend.

WHY

robert deansIf you’re interested, but not quite sold on this conference, take the word of these experts:
Robert Deans, Chief Technology Officer at BlueRock Therapeutics:

“I believe standardization will be an increasingly crucial element in securing commercial success for regenerative cell therapies.  This applies to all facets of development, from cell characterization and patent protection through safety testing of final product.  Most important is the adherence of players in this sector to harmonized standards and creation of a scientifically credible market to the capital community.”

martin-pera-profileProfessor Martin Pera of the Jackson Laboratory, who directs the International  Stem Cell Initiative Genetics and Epigenetics Study Group:

“Participants at this meeting will survey and discuss the state of the art in the development of definitive assays for assessing the safety of pluripotent stem cell based therapies, a critical issue for the future of the field.  Anyone active in cell therapy should attend this meeting to contribute to a dialogue that will impact on research directions and ultimately help to define best practice in this sector.”

When and Where

The conference will be held in Los Angeles Airport Marriott on June 5-6th, 2018. Registration is now open on the IABS website and you can take advantage of discounted early bird registration before April 24th.

Using the courts to protect patients from unapproved stem cell therapies

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A recent article in Nature looked at using lawsuits to help rein in the activities of clinics offering “unapproved” therapies. CIRM’s Geoff Lomax explains.

Stem-Cell-Clinics-to-Trust

When public health officials wanted to raise awareness about the dangers of smoking they filed lawsuits against the tobacco companies. They accused Big Tobacco of deceptive marketing and hiding the negative health effects of smoking. Ultimately, they won. Now a new study says a similar tactic could prove effective in combating clinics that offer unproven stem cell therapies.

CIRM works tirelessly to accelerate the delivery of stem cell treatments to patients with unmet medical needs. But, that doesn’t mean we support any treatment that claims to help people. CIRM only partners with projects that have been given the go-ahead by the US Food and Drug Administration (FDA) to be tested in people in a clinical trial.  That’s because FDA approval means the clinical trial will be monitored and evaluated under high scientific and ethical standards.

In contrast, there are numerous examples where “stem-cell treatments” not sanctioned by the FDA are being marketed directly to patients. For years the FDA, CIRM and others have been warning consumers about the risks involved with these untested treatments. For example, just last  November the FDA issued a warning and advice for people considering stem cell treatments.

Legal steps

Last year CIRM also helped author a new California law designed to protect consumers. The law requires health care providers to disclose to patients when using a treatment that is not FDA approved or part of an FDA-sanctioned clinical trial.

At CIRM, we frequently direct patients seeking treatments to our Alpha Stem Cell Clinics Network. The Alpha Clinics only perform clinical trials that have been given the green light by the FDA, and they provide expert consultation and informed consent to patients to help ensure they make the best choice for themselves. Further, the Alpha Clinics follow up with patients after their treatments to evaluate safety and the effectiveness of the treatments.

These are steps that clinics offering unproven and unapproved therapies typically don’t follow. So, the question is how do you let people know about the risks involved in going to one of these clinics and how do you stop clinics offering “therapies” that might endanger the health of patients?

Using the law to hit clinics where it hurts

In a recently published perspective in the journal Nature an international team of policy experts considered whether civil lawsuits may play a role in stemming the tide of unproven treatments. In the article the authors say:

“The threat of financial liability for wrongdoing is the primary means by which civil law governs behavior in the private sector. Despite calls for stepping up enforcement efforts, the US Food and Drug Administration (FDA) is currently restricted in its ability to identify and target clinics operating in apparent violation of regulations. The fear of tort liability {lawsuits} may provide sufficient incentive for compliance and minimize the occurrence of unethical practices.”

The authors identified nine individual and class action lawsuits involving clinics offering what they called “unproven stem cell interventions.” A few of those were dismissed or decided in favor of the clinics, with judges saying the claims lacked merit. Most, however, were settled by the clinics with no ruling on the merits of the issue raised. Even without definitive judgements against the clinics the authors of the article conclude:

“Stem cell lawsuits could intensify publicity and raise awareness of the harms of unproven treatments, set legal precedent, reshape the media narrative from one focused on the right to try or practice to one highlighting the need for adequate safety and efficacy standards, and encourage authorities to turn their attention to policy reform and enforcement.”

The authors suggest the courts may provide a forum where medical experts can inform patients, the legal community and the public about good versus harmful clinical practices. In short, the authors believe the legal process can be an effective forum for to provide education and outreach to those with disease and the public at large.

The better option of course would be for the clinics themselves to reform their practices and engage with the FDA to test their therapies in a clinical trial. Until that happens the courts may offer an alternative approach to curbing the marketing of these unproven and unapproved therapies.

The Alpha Stem Cell Clinics: Innovation for Breakthrough Stem Cell Treatments

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During this third week of the Month of CIRM, we are focusing on CIRM’s Infrastructure programs which are all focused on helping to accelerate stem cell treatments to patients with unmet medical needs.

So here is the question of the day: What is the world’s largest network of medical centers dedicated to providing stem cell treatments to patients?

The answer is the CIRM Alpha Stem Cell Clinics Network.

The CIRM Alpha Stem Cell Clinics Network consists of leading medical institutions throughout California.

The ASCC Network consists of six leading medical centers throughout California. In 2015, the Network was launched in southern California at the City of Hope, UC Irvine, UC Los Angeles, and UC San Diego. In September 2017, CIRM awarded funding to UC Davis and UC San Francisco to enable the Network to better serve patients throughout the state. Forty stem cell clinical trials have been conducted within the Network with hundreds of patients being treat for a variety of conditions, including:

  • Cancers of the blood, brain, lung and other sites
  • Organ diseases of the heart and kidney
  • Pediatric diseases
  • Traumatic injury to the brain and spine

A complete list of clinical trials may be found on our website.

The Alpha Clinics at UC Los Angeles and San Francisco are working collaboratively on breakthrough treatments for serious childhood diseases. This video highlights a CIRM-funded clinical trial at the UCLA Alpha Clinic that is designed to restore the immune system of patients with life-threatening immune deficiencies. A similar breakthrough treatment is also being used at the UCLA Alpha Clinic to treat sickle cell disease. A video describing this treatment is below.

Why do we need a specialized Network for stem cell clinical trials?

Stem cell treatments are unique in many ways. First, they consist of cells or cell products that frequently require specialized processing. For example, the breakthrough treatments for children, described above, requires the bone marrow to be genetically modified to correct defects. This “gene therapy” is performed in the Alpha Clinic laboratories, which are specifically designed to implement cutting edge gene therapy techniques on the patient’s stem cells.

Many of the cancer clinical trials also take the patient’s own cells and then process them in a laboratory. This processing is designed to enhance the patient’s ability to fight cancer using their own immune cells. Each Alpha Clinic has specialized laboratories to process cells, and the sites at City of Hope and UC Davis have world-class facilities for stem cell manufacturing. The City of Hope and Davis facilities produce high quality therapeutic products for commercial and academic clinical trial sponsors. Because of this ability, the Network has become a prime location internationally for clinical trials requiring processing and manufacturing services.

Another unique feature of the Network is its partnership with CIRM, whose mission is to accelerate stem cell treatments for patients with unmet medical needs. Often, this means developing treatments for rare diseases in which the patient population is comparatively small. For example, there about 40-100 immune deficient children born each year in the United States. We are funding clinical trials to help treat those children. The Network is also treating rare brain and blood cancers.

To find patients that may benefit from these treatments, the Network has developed the capacity to confidentially query over 20 million California patient records. If a good match is found, there is a procedure in place, that is reviewed by an ethics committee, where the patient’s doctor can be notified of the trial and pass that information to the patient. For patients that are interested in learning more, each Alpha Clinic has a Patient Care Coordinator with the job of coordinating the process of educating patients about the trial and assisting them if they choose to participate.

How Can I Learn More?

If you are a patient or a family member and would like to learn more about the CIRM Alpha Clinics, click here. There is contact information for each clinic so you can learn more about specific trials, or you can visit our Alpha Clinics Trials page for a complete list of trials ongoing in the Network.

If you are a patient or a trial sponsor interested in learning more about the services offered through our Alpha Clinics Network, visit our website.

A Clinical Trial Network Focused on Stem Cell Treatments is Expanding

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Geoff Lomax is a Senior Officer of CIRM’s Strategic Initiatives.

California is one of the world-leaders in advancing stem cell research towards treatments and cures for patients with unmet medical needs. California has scientists at top universities and companies conducting cutting edge research in regenerative medicine. It also has CIRM, California’s Stem Cell Agency, which funds promising stem cell research and is advancing stem cell therapies into clinical trials. But the real clincher is that California has something that no one else has: a network of medical centers dedicated to stem cell-based clinical trials for patients. This first-of-its-kind system is called the CIRM Alpha Stem Cell Clinics Network.

Get to Know Our Alpha Clinics

In 2014, CIRM launched its Alpha Stem Cell Clinics Network to accelerate the development and delivery of stem cell treatments to patients. The network consists of three Alpha Clinic sites at UC San Diego, City of Hope in Duarte, and a joint clinic between UC Los Angeles and UC Irvine. Less than three years since its inception, the Alpha Clinics are conducting 34 stem cell clinical trials for a diverse range of diseases such as cancer, heart disease and sickle cell anemia. You can find a complete list of these clinical trials on our Alpha Clinics website. Below is an informational video about our Alpha Clinics Network.

So far, hundreds of patients have been treated at our Alpha Clinics. These top-notch medical centers use CIRM-funding to build teams specialized in overseeing stem cell trials. These teams include patient navigators who provided in-depth information about clinical trials to prospective patients and support them during their treatment. They also include pharmacists who work with patients’ cells or manufactured stem cell-products before the therapies are given to patients. And lastly, let’s not forget the doctors and nurses that are specially trained in the delivery of stem cell therapies to patients.

The Alpha Clinics Network also offers resources and tools for clinical trial sponsors, the people responsible for conducting the trials. These include patient education and recruitment tools and access to over 20 million patients in California to support successful recruitment. And because the different clinical trial sites are in the same network, sponsors can benefit from sharing the same approval measures for a single trial at multiple sites.

Looking at the big picture, our Alpha Clinics Network provides a platform where patients can access the latest stem cell treatments, and sponsors can access expert teams at multiple medical centers to increase the likelihood that their trial succeeds.

The Alpha Clinics Network is expanding

This collective expertise has resulted in a 3-fold (from 12 to 36 – two trials are being conducted at two sites) increase in the number of stem cell clinical trials at the Alpha Clinic sites since the Network’s inception. And the number continues to rise every quarter. Given this impressive track record, CIRM’s Board voted in February to expand our Alpha Clinics Network. The Board approved up to $16 million to be awarded to two additional medical centers ($8 million each) to create new Alpha Clinic sites and work with the current Network to accelerate patient access to stem cell treatments.

CIRM’s Chairman Jonathan Thomas explained,

Jonathan Thomas

“We laid down the foundation for conducting high quality stem cell trials when we started this network in 2014. The success of these clinics in less than three years has prompted the CIRM Board to expand the Network to include two new trial sites. With this expansion, CIRM is building on the current network’s momentum to establish new and better ways of treating patients with stem cell-based therapies.”

The Alpha Clinics Network plays a vital role in CIRM’s five-year strategic plan to fund 50 new clinical trials by 2020. In fact, the Alpha Clinic Network supports clinical trials funded by CIRM, industry sponsors and other sources. Thus, the Network is on track to becoming a sustainable resource to deliver stem cell treatments indefinitely.

In addition to expanding CIRM’s Network, the new sites will develop specialized programs to train doctors in the design and conduct of stem cell clinical trials. This training will help drive the development of new stem cell therapies at California medical centers.

Apply to be one our new Alpha Clinics!

For the medical centers interested in joining the CIRM Alpha Stem Cell Clinics Network, the deadline for applications is May 15th, 2017. Details on this funding opportunity can be found on our funding page.

The CIRM Team looks forward to working with prospective applicants to address any questions. The Alpha Stem Cell Clinics Network will also be showcasing it achievement at its Second Annual Symposium, details may be found on the City of Hope Alpha Clinics website.

City of Hope Medical Center and Alpha Stem Cell Clinic

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Helping patient’s fight back against deadliest form of skin cancer

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Caladrius Biosciences has been funded by CIRM to conduct a Phase 3 clinical trial to treat the most severe form of skin cancer: metastatic melanoma. Metastatic melanoma is a disease with no effective treatment, only around 15 percent of people with it survive five years, and every year it claims an estimated 10,000 lives in the U.S.

The CIRM/Caladrius Clinical Advisory Panel meets to chart future of clinical trial

The CIRM/Caladrius Clinical Advisory Panel meets to chart future of clinical trial

The Caladrius team has developed an innovative cancer treatment that is designed to target the cells responsible for tumor growth and spread. These are called cancer stem cells or tumor-initiating cells. Cancer stem cells can spread in the body because they have the ability to evade the body’s immune defense and survive standard anti-cancer treatments such as chemotherapy. The aim of the Caladrius treatment is to train the body’s immune system to recognize the cancer stem cells and attack them.

Attacking the cancer

The treatment process involves taking a sample of a patient’s own tumor and, in a laboratory, isolating specific cells responsible for tumor growth . Cells from the patient’s blood, called “peripheral blood monocytes,” are also collected. The mononucleocytes are responsible for helping the body’s immune system fight disease. The tumor and blood cells (after maturation into dendritic cells) are then combined and incubated so that the patient’s immune cells become trained to recognize the cancer cells.

After the incubation period, the patient’s immune cells are injected back into their body where they generate an immune response to the cancer cells. The treatment is like a vaccine because it trains the body’s immune system to recognize and rapidly attack the source of disease.

Recruiting the patients

Caladrius has already dosed the first patient in the trial (which is double blinded so no one knows if the patient got the therapy or a placebo) and hopes to recruit 250 patients altogether.

This is the first Phase 3 trial that CIRM has funded so we’re obviously excited about its potential to help people battling this deadly disease.  In a recent news release David J. Mazzo, the CEO of Caladrius echoed this excitement, with a sense of cautious optimism:

“The dosing of the first patient in this Phase 3 trial is an important milestone for our Company and the timing underscores our focus on this program and our commitment to impeccable trial execution. We are delighted by the enthusiasm and productivity of the team at Jefferson University (where the patient was dosed) and other trial sites around the country and look forward to translating that into optimized patient enrollment and a rapid completion of the Phase 3 trial.”

And that’s the key now. They have the science. They have the funding. Now they need the patients. That’s why we are all working together to help Caladrius recruit patients as quickly as possible. Because their work perfectly reflects our mission of accelerating the development of stem cell therapies for patients with unmet medical needs.

You can learn more about what the study involves and who is eligible by clicking here.