Alpha Stem Cell Clinic network

71 for Proposition 71

/ Geoffrey Lomax Dr. PH / 3 Comments

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.

 

 

New CIRM Alpha Stem Cell Clinic offers HOPE for boys with deadly disease

/ Kevin McCormack / 3 Comments

UC Davis Institute for Regenerative Cures

For people battling Duchenne Muscular Dystrophy (DMD), a rare and fatal genetic disorder that slowly destroys muscles, hope has often been in short supply. There is no cure and treatments are limited. But now a new clinical trial at the site of one of the newest CIRM Alpha Stem Cell Clinic Network members could change that.

The HOPE-2 clinical trial has treated its first patient at UC Davis Medical Center, inaugurating the institution’s Alpha Stem Cell Clinic. The clinic is part of a CIRM-created network of top California medical centers that specialize in delivering stem cell clinical trials to patients. The key to the Network’s success is the ability to accelerate the delivery of treatments to patients through partnerships with patients, medical providers and clinical trial sponsors.

UC Davis is one of five medical centers that now make up the network (the others are UC San Francisco, UCLA/UC Irvine, UC San Diego and City of Hope).

Jan NoltaIn a news release, Jan Nolta, the director of the UC Davis Institute for Regenerative Cures, says the UC Davis Alpha Clinic is well equipped to move promising therapies out of the lab and into clinical trials and people.

“We have the full range of resource experts in regenerative medicine, from the cellular to the clinical trials level. We’re also excited about the prospect of being able to link with other Alpha Stem Cell Clinics around the state to help speed the process of testing and refining treatments so we can get therapies to patients in need.”

The news of this first patient is a cause for double celebration at CIRM. The trial is run by Capricor and CIRM funded the first phase of this work. You can read the story of Caleb Sizemore, who took part in that trial or watch this video of him talking about his fight.

When the CIRM Board approved funding for the UC Davis Alpha Clinic in October of 2017, Abla Creasey, CIRM’s Vice President for Therapeutics and Strategic Infrastructure, said:

“The Alpha Clinics are a one-of-a-kind network that gives patients access to the highest quality stem cell trials for a breadth of diseases including cancer, diabetes, heart disease and spinal cord injury. Expanding our network will allow more patients to participate in stem cell trials and will advance the development of stem cell treatments that could help or possibly cure patients.”

The UC Davis Alpha Clinic provides a one-stop shop for delivering stem cell therapies, gene therapies and immunotherapies, as well as conducting follow-up visits. It’s this type of CIRM-funded infrastructure that helps steer potential clinical trial participants away from illegitimate, unproven and potentially harmful fee-for-service stem cell treatments.

The DMD trial is the first of what we are confident will be many high-quality trials at the Clinic, bringing promising stem cell therapies to patients with unmet medical needs.

 

Inspiring Video: UC Irvine Stem Cell Trial Gives Orange County Woman Hope in Her Fight Against ALS

/ Karen Ring / Leave a comment

Stephen Hawking

Last week, we lost one of our greatest, most influential scientific minds. Stephen Hawking, a famous British theoretical physicist and author of “A Brief History of Time: From the Big Bang to Black Holes”, passed away at the age of 76.

Hawking lived most of his adult life in a wheelchair because he suffered from amyotrophic lateral sclerosis (ALS). Also known as Lou Gehrig’s disease, ALS causes the degeneration of the nerve cells that control muscle movement.

When Hawking was diagnosed with ALS at the age of 21, he was told he only had three years to live. But Hawking defied the odds and went on to live a life that not only revolutionized our understanding of the cosmos, but also gave hope to other patients suffering from this devastating degenerative disease.

A Story of Hope

Speaking of hope, I’d like to share another story of an Orange County woman name Lisa Wittenberg who was recently diagnosed with ALS. Her story was featured this week on KTLA5 news and is also available on the UC Irvine Health website.

VIDEO: UCI Health stem cell trial helps Orange County woman fight neurodegenerative disease ALS. Click on image to view video in new window.

In this video, Lisa describes how quickly ALS changed her life. She was with her family sledding in the snow last winter, and only a year later, she is in a wheelchair unable to walk. Lisa got emotional when she talked about how painful it is for her to see her 13-year-old son watch her battle with this disease.

But there is hope for Lisa in the form of a stem cell clinical trial at the UC Irvine CIRM Alpha Stem Cell Clinic. Lisa enrolled in the Brainstorm study, a CIRM-funded phase 3 trial that’s testing a mesenchymal stem cell therapy called NurOwn. BrainStorm Cell Therapeutics, the company sponsoring this trial, is isolating mesenchymal stem cells from the patient’s own bone marrow. The stem cells are then cultured in the lab under conditions that convert them into biological factories secreting a variety of neurotrophic factors that help protect the nerve cells damaged by ALS. The modified stem cells are then transplanted back into the patient where they will hopefully slow the progression of the disease.

Dr. Namita Goyal, a neurologist at UC Irvine Health involved in the trial, explained in the KTLA5 video that they are hopeful this treatment will give patients more time, and optimistic that in some cases, it could improve some of their symptoms.

Don’t Give Up the Fight

The most powerful part of Lisa’s story to me was the end when she says,

“I think it’s amazing that I get to fight, but I want everybody to get to fight. Everybody with ALS should get to fight and should have hope.”

Not only is Lisa fighting by being in this ground-breaking trial, she is also participated in the Los Angeles marathon this past weekend, raising money for ALS research.

More patients like Lisa will get the chance to fight as more potential stem cell treatments and drugs enter clinical trials. Videos like the one in this blog are important for raising awareness about available clinical trials like the Brainstorm study, which, by the way, is still looking for more patients to enroll (contact information for this trial can be found on the clinicaltrials.gov website here). CIRM is also funding another stem cell trial for ALS at the Cedars-Sinai Medical Center. You can read more about this trial on our website.

Lisa’s powerful message of fighting ALS and having hope reminds me of one of Stephen Hawking’s most famous quotes, which I’ll leave you with:

“Remember to look up at the stars and not down at your feet. Try to make sense of what you see and wonder about what makes the Universe exist. Be curious. And however difficult life may seem, there is always something you can do and succeed at. It matters that you don’t just give up.”

Related Links:

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

/ Geoffrey Lomax Dr. PH / Leave a comment

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)

Related Links:

Using the courts to protect patients from unapproved stem cell therapies

/ Geoffrey Lomax Dr. PH / 2 Comments

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.

A Noble pursuit; finding the best science to help the most people

/ Kevin McCormack / Leave a comment
MarkNoble-46

Mark Noble. Photo by Todd Dubnicoff

Mark Noble, Ph.D., is a pioneer in stem cell research and the Director of the University of Rochester Stem Cell and Regenerative Medicine Institute in New York. He is also a member of CIRM’s Grants Working Group (GWG), the panel of independent scientific experts we use to review research applications for funding and decide which are the most promising.

Mark has been a part of the GWG since 2011. When asked how he came to join the GWG he joked: “I saw an ad on Craigslist and thought it sounded fun.”  But he is not joking when he says it is a labor of love.

“My view is that CIRM is one of the greatest experiments in how to develop a new branch of science and medicine. If you look at ventures, like the establishment of the National Institutes of Health, what you see is that when there is a concentrated effort to achieve an enormous goal, amazing things can happen. And if your goal is to create a new field of medicine you have to take a truly expansive view.”

Mark has been on many other review panels but says they don’t compare to CIRM’s.

“These are the most exciting review panels in which I take part. I don’t know of any comparable panels that bring together experts working across such a wide range of disciplines and diseases.   It’s particularly interesting to be involved in reviews at this stage because we get to look at the fruits of CIRM’s long investment, and at projects that are now in, or well on the way towards, clinical trials.

It’s a wonderful scientific education because you come to these meetings and someone is submitting an application on diabetes and someone else has submitted an application on repairing the damage to the heart or spinal cord injury or they have a device that will allow you to transplant cells better. There are people in the room that are able to talk knowledgeably about each of these areas and understand how the proposed project might work in terms of actual financial development, and how it might work in the corporate sphere and how it fits in to unmet medical needs.  I don’t know of any comparable review panels like this that have such a broad remit and bring together such a breadth of expertise. Every review panel you come to you are getting a scientific education on all these different areas, which is great.”

Another aspect of CIRM’s work that Mark admires is its ability to look past the financial aspects of research, to focus on the bigger goal:

“I like that CIRM recognizes the larger problem, that a therapy that is curative but costs a million dollars a patient is not going to be implemented worldwide. Well, CIRM is not here to make money. CIRM is here to find cures for unmet medical needs, which means that if someone comes in with a great application on a drug that is going to cure some awful disease and it’s not going to be worth a fortune, that is not the main concern. The main concern is that you might be able to cure this disease and yeah, we’ll put up money to help you so that you might be able to get into clinical trials, to get enough information to find out if it works. And to have the vision to go all the way from, ‘ok, you guys, we want you to enter this field, we want you to be interested in therapeutic development, we are going to help you structure the clinical trials, we are going to provide all the Alpha Stem Cell Clinics that can talk to each other to make the clinical trials happen.

The goal of CIRM is to change medicine and these are the approaches that have worked really well in doing this. The CIRM view clearly is:

‘There are 100 horses in this race and every single one that crosses the finish line is a success story.’ That’s what is necessary, because there are so many diseases and injuries for which new approaches are needed.”

Mark says working with CIRM has helped him spread the word back home in New York state:

“I have been very involved in working with the New York state legislature over the years to promote funding for stem cell biology and spinal cord injury research so having the CIRM experience has really helped me to understand what it is that another place can try and accomplish. A lot of the ideas that have been worked out at CIRM have been extremely helpful for statewide scientific enterprises in New York, where we have had people involved in different areas of the state effort talk to people at CIRM to find out what best practice is.”

Mark says he feels as if he has a front row seat to history.

“Seeing the stem cell field grow to its present stage and enhancing the opportunity to address multiple unmet medical needs, is a thrilling adventure. Working with CIRM to help create a better future is a privilege.”

 

California gets first royalty check from Stem Cell Agency investments

/ Kevin McCormack / Leave a comment

COH image

CIRM recently shared in a little piece of history. The first royalty check, based on CIRM’s investment in stem cell research, was sent to the California State Treasurer’s office from City of Hope. It’s the first of what we hope will be many such checks, helping repay, not just the investment the state made in the field, but also the trust the voters of California showed when they created CIRM.

The check, for $190,345.87, was for a grant we gave City of Hope back in 2012 to develop a therapy for glioblastoma, one of the deadliest forms of brain cancer. That has led to two clinical trials and a number of offshoot inventions that were subsequently licensed to a company called Mustang Bio.

Christine Brown, who is now the principal investigator on the project, is quoted in a front page article in the San Francisco Chronicle, on the significance of the check for California:

“This is an initial payment for the recognition of the potential of this therapy. If it’s ultimately approved by the FDA as a commercial product, this could be a continued revenue source.”

In the same article, John Zaia, Director of the City of Hope Alpha Stem Cell Clinic, says this also reflects the unique nature of CIRM:

“I think this illustrates that a state agency can actually fund research in the private community and get a return on its investment. It’s something that’s not done in general by other funding agencies such as the National Institutes of Health, and this is a proof of concept that it can work.”

Maria Millan, CIRM’s President & CEO, says the amount of the payment is not the most significant part of this milestone – after all CIRM has invested more than $2.5 billion in stem cell research since 2004. She says the fact that we are starting to see a return on the investment is important and reflects some of the many benefits CIRM brings to the state.

“It’s a part of the entire picture of the return to California. In terms of what it means to the health of Californians, and access to these transformative treatments, as well as the fact that we are growing an industry.”

 

Alpha clinics and a new framework for accelerating stem cell treatments

/ Kevin McCormack / 2 Comments

IMG_1215

Last week, at the World Stem Cell Summit in Miami, CIRM took part in a panel discussion about the role and importance of Alpha Clinics in not just delivering stem cell therapies, but in helping create a new, more collaborative approach to medicine. The Alpha Clinic concept is to create  a network of top medical centers that specialize in delivering stem cell clinical trials to patients.

The panel was moderated by Dr. Tony Atala, Director of the Wake Forest Institute for Regenerative Medicine. He said the term Alpha Clinic came from CIRM and the Alpha Stem Cell Clinic Network that we helped create. That network now has five specialist health care centers that deliver stem cell therapies to patients: UC San Diego, UCLA/UC Irvine, City of Hope, UC Davis, and  UCSF/Children’s Hospital Oakland.

This is a snapshot of that conversation.

Alpha Clinics Advancing Stem Cell Trials

Dr. Maria Millan, CIRM’s President & CEO:

“The idea behind the Alpha Stem Cell Clinic Network is that CIRM is in the business of accelerating treatments to patients with unmet medical needs. We fund research from the earliest discovery stage to clinical trials. What was anticipated is that, if the goal is to get these discoveries into the clinics then we’ll need a specific set of expertise and talents to deliver those treatments safely and effectively, to gather data from those trials and move the field forward. So, we set out to create a learning network, a sharing network and a network that is more than the sum of its parts.”

Dr. Joshua Hare,  Interdisciplinary Stem Cell Institute, University of Miami, said that idea of collaboration is critical to advancing the field:

 

“What we learned is that having the Alpha Stem Cell Clinic concept helps investigators in other areas learn from what earlier researchers have done, helping accelerate their work.

For example, we have had a lot of experience in working with rare diseases and we can use the experience we have in treating one disease area in working in others. This shared experience can help us develop deeper understanding in terms of delivering therapies and dosing.”

Susan Solomon, CEO New York Stem Cell Foundation Research Institute. NYSCF has several clinical trials underway. She says in the beginning it was hard finding reputable clinics that could deliver these potentially ground breaking but still experimental therapies:

 

“My motivation was born out of my own frustration at the poor choices we had in dealing with some devastating diseases, so in order to move things ahead we had to have an alpha clinic that is not just doing clinical trials but is working to overcome obstacles in the field.”

Greg Simon represented the, Biden Cancer Initiative, whose  mission is to develop and drive implementation of solutions to accelerate progress in cancer prevention, detection, diagnosis, research, and care, and to reduce disparities in cancer outcomes. He says part of the problem is that people think there are systems already in place that promote collaboration and cooperation, but that’s not really the case.  

 

“In the Cancer Moonshot and the Biden Cancer Initiative we are trying to create the cancer research initiative that people think we already have. People think doctors share knowledge. They don’t. People think they can just sign up for clinical trials. They can’t. People think there are standards for describing a cancer. There aren’t. So, all the things you think you know about the science behind cancer are wrong. We don’t have the system people think is in place. But we want to create that.

If we are going to have a unified system we need common standards through cancer research, shared knowledge, and clinical trial reforms. All my professional career it was considered unethical to refer to a clinical trial as a treatment, it was research. That’s no longer the case. Many people are now told this is your last best hope for treatment and it’s changed the way people think about clinical trials.”

The Process

Maria Millan says we are seeing these kinds of change – more collaboration, more transparency –  taking place across the board:

“We see the research in academic institutions that then moved into small companies that are now being approved by the FDA. Academic centers, in conjunction with industry partners, are helping create networks and connections that advance therapies.

This gives us the opportunity to have clinical programs and dialogues about how we can get better, how we can create a more uniform, standard approach that helps us learn from each trial and develop common standards that investigators know have to be in place.

Within the CIRM Alpha Stem Cell Clinic Network the teams coming in can access what we have pulled together already – a database of 20 million patients, a single IRB approval, so that if a cliinical trial is approved for one Alpha Clinic it can also be offered at another.”

Greg Simon says to see the changes really take hold we need to ensure this idea of collaboration starts at the very beginning of the chain:

“If we don’t have a system of basic research where people share data, where people are rewarded for sharing data, journals that don’t lock up the data behind a paywall. If we don’t have that system, we don’t have the ability to move therapies along as quickly as we could.

“Nobody wants to be the last person to die from a cancer that someone figured out a treatment for a year earlier. It’s not that the science is so hard, or the diseases are so hard, it the way we approach them that’s so hard. How do we create the right system?”

More may not necessarily be better

Susan Solomon:

“There are tremendous number of advances moving to the clinic, but I am concerned about the need for more sharing and the sheer number of clinical trials. We have to be smart about how we do our work. There is some low hanging fruit for some clinical trials in the cancer area, but you have to be really careful.”

Greg Simon

“We have too many bad trials, we don’t need more, we need better quality trials.

We have made a lot of progress in cancer. I’m a CLL survivor and had zero problems with the treatment and everything went well.

We have pediatric cancer therapies that turned survival from 10 % to 80%. But the question is why doesn’t more progress happen. We tend to get stuck in a way of thinking and don’t question why it has to be that way. We think of funding because that’s the way funding cycles work, the NIH issues grants every year, so we think about research on a yearly basis. We need to change the cycle.”

Maria Millan says CIRM takes a two pronged approach to improving things, renovating and creating:

“We renovate when we know there are things already in place that can be improved and made better; and we create if there’s nothing there and it needs to be created. We want to be as efficient as we can and not waste time and resources.”

She ended by saying one of the most exciting things today is that the discussion now has moved to how we are going to cover this for patients. Greg Simon couldn’t agree more.

“The biggest predictor of survivability of cancer is health insurance. We need to do more than just develop treatments. We need to have a system that enables people to get access to these therapies.”

Taking a new approach to fighting a deadly brain cancer

/ Kevin McCormack / Leave a comment
Christine Brown DSC_3794

Christine Brown, Ph.D., City of Hope researcher

CIRM’s 2017 Annual Report will be going live online very soon. In anticipation of that we are highlighting some of the key elements from the report here on the Stem Cellar.

One of the most exciting new approaches in targeting deadly cancers is chimeric antigen receptor (CAR) T-cell therapy, using the patient’s own immune system cells that have been re-engineered to help them fight back against the tumor.

Today we are profiling City of Hope’s Christine Brown, Ph.D., who is using CAR-T cells in a CIRM-funded Phase 1 clinical trial for an aggressive brain cancer called malignant glioma.

“Brain tumors are the hardest to treat solid tumors. This is a project that CIRM has supported from an early, pre-clinical stage. What was exciting was we finished our first milestone in record time and were able to translate that research out of the lab and into the clinic. That really allowed us to accelerate treatment to glioblastoma patients.

I think there are glimmers of hope that immune based therapies and CAR-T based therapies will revolutionize therapy for patients with brain tumors. We’ve seen evidence that these cells can travel to the central nervous system and eliminate tumors in the brain.

We now have evidence that this approach produces a powerful, therapeutic response in one group of patients. We are looking at why other patients don’t respond as well and the CIRM funding enables us to ask the questions that will, we hope, provide the answers.

Because our clinical trial is a being carried out at the CIRM-supported City of Hope Alpha Stem Cell Clinic this is a great example of how CIRM supports all the different ways of advancing therapy from early stage research through translation and into clinical trials in the CIRM Alpha Clinic network.

There are lots of ways the tumor tries to evade the immune system and we are looking at different approaches to combine this therapy with different approaches to see which combination will be best.

It’s a challenging problem and it’s not going to be solved with one approach. If it were easy we’d have solved it by now. That’s why I love science, it’s one big puzzle about how do we understand this and how do we make this work.

I don’t think we would be where we are at without CIRM’s support, it really gave the funding to bring this to the next level.”

Dr. Brown’s work is also creating interest among investors. She recently partnered with Mustang Bio in a $94.5 million agreement to help advance this therapy.

Turning the corner with the FDA and NIH; CIRM creates new collaborations to advance stem cell research

/ Kevin McCormack / Leave a comment

FDAThis blog is part of the Month of CIRM series on the Stem Cellar

A lot can change in a couple of years. Just take our relationship with the US Food and Drug Administration (FDA).

When we were putting together our Strategic Plan in 2015 we did a survey of key players and stakeholders at CIRM – Board members, researchers, patient advocates etc. – and a whopping 70 percent of them listed the FDA as the biggest impediment for the development of stem cell treatments.

As one stakeholder told us at the time:

“Is perfect becoming the enemy of better? One recent treatment touted by the FDA as a regulatory success had such a high clinical development hurdle placed on it that by the time it was finally approved the standard of care had evolved. When it was finally approved, five years later, its market potential had significantly eroded and the product failed commercially.”

Changing the conversation

To overcome these hurdles we set a goal of changing the regulatory landscape, finding a way to make the system faster and more efficient, but without reducing the emphasis on the safety of patients. One of the ways we did this was by launching our “Stem Cell Champions” campaign to engage patients, patient advocates, the public and everyone else who supports stem cell research to press for change at the FDA. We also worked with other organizations to help get the 21st Century Cures Act passed.

21 century cures

Today the regulatory landscape looks quite different than it did just a few years ago. Thanks to the 21st Century Cures Act the FDA has created expedited pathways for stem cell therapies that show promise. One of those is called the Regenerative Medicine Advanced Therapy (RMAT) designation, which gives projects that show they are both safe and effective in early-stage clinical trials the possibility of an accelerated review by the FDA. Of the first projects given RMAT designation, three were CIRM-funded projects (Humacyte, jCyte and Asterias)

Partnering with the NIH

Our work has also paved the way for a closer relationship with the National Institutes of Health (NIH), which is looking at CIRM as a model for advancing the field of regenerative medicine.

In recent years we have created a number of innovations including introducing CIRM 2.0, which dramatically improved our ability to fund the most promising research, making it faster, easier and more predictable for researchers to apply. We also created the Stem Cell Center  to make it easier to move the most promising research out of the lab and into clinical trials, and to give researchers the support they need to help make those trials successful. To address the need for high-quality stem cell clinical trials we created the CIRM Alpha Stem Cell Clinic Network. This is a network of leading medical centers around the state that specialize in delivering stem cell therapies, sharing best practices and creating new ways of making it as easy as possible for patients to get the care they need.

The NIH looked at these innovations and liked them. So much so they invited CIRM to come to Washington DC and talk about them. It was a great opportunity so, of course, we said yes. We expected them to carve out a few hours for us to chat. Instead they blocked out a day and a half and brought in the heads of their different divisions to hear what we had to say.

A model for the future

We hope the meeting is, to paraphrase Humphrey Bogart at the end of Casablanca, “the start of a beautiful friendship.” We are already seeing signs that it’s not just a passing whim. In July the NIH held a workshop that focused on what will it take to make genome editing technologies, like CRISPR, a clinical reality. Francis Collins, NIH Director, invited CIRM to be part of the workshop that included thought leaders from academia, industry and patients advocates. The workshop ended with a recommendation that the NIH should consider building a center of excellence in gene editing and transplantation, based on the CIRM model (my emphasis).  This would bring together a multidisciplinary disease team including, process development, cGMP manufacturing, regulatory and clinical development for Investigational New Drug (IND) filing and conducting clinical trials, all under one roof.

dr_collins

Dr. Francis Collins, Director of the NIH

In preparation, the NIH visited the CIRM-funded Stem Cell Center at the City of Hope to explore ways to develop this collaboration. And the NIH has already begun implementing these suggestions starting with a treatment targeting sickle cell disease.

There are no guarantees in science. But we know that if you spend all your time banging your head against a door all you get is a headache. Today it feels like the FDA has opened the door and that, together with the NIH, they are more open to collaborating with organizations like CIRM. We have removed the headache, and created the possibility that by working together we truly can accelerate stem cell research and deliver the therapies that so many patients desperately need.