CIRM invests in stem cell clinical trial targeting lung cancer and promising research into osteoporosis and incontinence

Lung cancer

Lung cancer: Photo courtesy Verywell

The five-year survival rate for people diagnosed with the most advanced stage of non-small cell lung cancer (NSCLC) is pretty grim, only between one and 10 percent. To address this devastating condition, the Board of the California Institute for Regenerative Medicine (CIRM) today voted to invest almost $12 million in a team from UCLA that is pioneering a combination therapy for NSCLC.

The team is using the patient’s own immune system where their dendritic cells – key cells in our immune system – are genetically modified to boost their ability to stimulate their native T cells – a type of white blood cell – to destroy cancer cells.  The investigators will combine this cell therapy with the FDA-approved therapy pembrolizumab (better known as Keytruda) a therapeutic that renders cancer cells more susceptible to clearance by the immune system.

“Lung cancer is a leading cause of cancer death for men and women, leading to 150,000 deaths each year and there is clearly a need for new and more effective treatments,” says Maria T. Millan, M.D., the President and CEO of CIRM. “We are pleased to support this program that is exploring a combination immunotherapy with gene modified cell and antibody for one of the most extreme forms of lung cancer.”

Translation Awards

The CIRM Board also approved investing $14.15 million in four projects under its Translation Research Program. The goal of these awards is to support promising stem cell research and help it move out of the laboratory and into clinical trials in people.

Researchers at Stanford were awarded almost $6 million to help develop a treatment for urinary incontinence (UI). Despite being one of the most common indications for surgery in women, one third of elderly women continue to suffer from debilitating urinary incontinence because they are not candidates for surgery or because surgery fails to address their condition.

The Stanford team is developing an approach using the patient’s own cells to create smooth muscle cells that can replace those lost in UI. If this approach is successful, it provides a proof of concept for replacement of smooth muscle cells that could potentially address other conditions in the urinary tract and in the digestive tract.

Max BioPharma Inc. was awarded almost $1.7 million to test a therapy that targets stem cells in the skeleton, creating new bone forming cells and blocking the destruction of bone cells caused by osteoporosis.

In its application the company stressed the benefit this could have for California’s diverse population stating: “Our program has the potential to have a significant positive impact on the lives of patients with osteoporosis, especially in California where its unique demographics make it particularly vulnerable. Latinos are 31% more likely to have osteoporosis than Caucasians, and California has the largest Latino population in the US, accounting for 39% of its population.”

Application Title Institution CIRM funding
TRAN1-10958 Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence

 

 

Stanford University

 

$5,977,155

 

TRAN2-10990 Development of a noninvasive prenatal test for beta-hemoglobinopathies for earlier stem cell therapeutic interventions

 

 

Children’s Hospital Oakland Research Institute

 

$1,721,606

 

TRAN1-10937 Therapeutic development of an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis  

MAX BioPharma Inc.

 

$1,689,855

 

TRAN1-10995 Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models

 

 

UC Irvine

 

$4,769,039

 

CIRM applauds FDA crackdown on stem cell clinics that “peddle unapproved treatments.”

FDA

CIRM is commending the US Food and Drug Administration (FDA) for its action against two stem cell clinics offering unapproved therapies.

On Wednesday, the FDA filed two complaints in federal court seeking a permanent injunction against California Stem Cell Treatment Center Inc. and US Stem Cell Clinic LLC. of Sunrise, Florida. The FDA says the clinics are marketing stem cell products without FDA approval and are not complying with current good manufacturing practice requirements.

“We strongly support the FDA’s strong stance to seek judicial action to stop these  clinics from marketing unproven therapies that pose a threat to the safety of patients” says Maria T. Millan, M.D., CIRM’s President and CEO. “We agree with FDA Commissioner Dr. Scott Gottlieb’s statement that these ‘bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk.’”

In his statement yesterday, Dr. Gottlieb denounced the clinics saying they are exploiting patients and causing some of them “serious and permanent harm.”

“In the two cases filed today, the clinics and their leadership have continued to disregard the law and more importantly, patient safety. We cannot allow unproven products that exploit the hope of patients and their loved ones. We support sound, scientific research and regulation of cell-based regenerative medicine, and the FDA has advanced a comprehensive policy framework to promote the approval of regenerative medicine products. But at the same time, the FDA will continue to take enforcement actions against clinics that abuse the trust of patients and endanger their health.”

At CIRM, we believe it is critically important for participants in stem cell treatments to be fully informed about the nature of the therapy they are receiving, including whether it is approved by the FDA. Last year we partnered with California State Senator Ed Hernandez to pass Senate Bill No. 512, which required all clinics offering unproven stem cell therapies to post notices warning patients they were getting a therapy that was not approved by the FDA.

The Stem Cell Agency has taken several other actions to protect people seeking legitimate stem cell therapies.

  • All the clinical trials we consider for funding must already have an active Investigational New Drug (IND) status with the FDA and go through a rigorous scientific review by leading experts.
  • All CIRM-funded trials must adhere to strict regulatory standards and safety monitoring.
  • We have created the CIRM Alpha Stem Cell Clinics, a network of six top California medical centers that specialize in delivering patient-centered stem cell clinical trials that meet the highest standards of care and research.
  • CIRM provides access to information on all the clinical trials it supports.

“Through its funding mechanism, active partnership and infrastructure programs, CIRM has shepherded 48 FDA regulated, scientifically sound, rigorously reviewed promising stem cell and regenerative medicine projects into clinical trials,” says Dr. Millan. “Some of these treatment protocols have already started to show preliminary signs of benefit for debilitating and life-threatening disorders. We are committed to doing all we can, in partnership with patients, the research community and with the FDA, to develop transformative treatments for patients with unmet medical needs while adhering to the highest standards to protect the health and safety of patients and the public.”

To help people make informed decisions we have created an infographic and video that detail the information people need to know, and the questions they should ask, before they agree to participate in a clinical trial or get a stem cell therapy.

 

 

CIRM President/CEO Presenting at Vatican Conference Targeting Cures for Deadly Diseases

It’s not often you get invited to a meeting of some of the leading scientists, ethicists, philosophers and faith leaders in the world so when the call comes in it’s an easy one to answer. Particularly when the call is from the Vatican.

View of Basilica di San Pietro in Vatican, Rome, Italy

Maria T. Millan, MD, President and CEO of the California Institute for Regenerative Medicine (CIRM), will be part of a panel discussion at the Fourth International Vatican Conference at Vatican City, Rome. The conference, titled: Unite To Cure: How Science, Technology and 21st Century Medicine Will Impact Culture and Society, runs from April 26-28 in Rome.

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Maria T. Millan, MD

“It is a tremendous honor to be part of this historic event,” says Dr. Millan. “CIRM funds the science and development of transformative cell and gene therapies for patients with unmet medical needs so it will be important to be part of the global conversation during such a propitious time in the history of medicine.”

“We’re thrilled to bring together the world’s best scientists, doctors, ethicists and leaders of faith, business, government and philanthropy to this extraordinary global event at The Vatican,” says Dr. Robin Smith, President of The Cura Foundation, the event organizer in partnership with the Vatican. “It’s a Davos for health care, and over the course of three days we will rally the world around a very simple idea — that tomorrow’s cures are just around the corner, and by uniting together and understanding the challenges that lie ahead, we can speed the delivery of cures and foster great hope for patients all over the world suffering from deadly diseases and dangerous medical conditions.”

Dr. Millan will be part of a panel discussion titled, Public Private Partnerships to Accelerate Discoveries. The panel will be moderated by award-winning medical journalist Max Gomez, PhD., and will include David Mazzo, PhD, CEO of Caladrius Biosciences, and David Pearce, PhD, the Executive VP for Research at Sanford Health.

The topic for this panel is particularly well suited for CIRM, an agency that is devoted to accelerating stem cell treatments to patients with unmet medical needs. CIRM has funded over 800 projects and over 45 novel stem cell and regenerative medicine clinical trials. It delivers a predictable and expedited funding mechanism, an active partnership and advisory role, strategic infrastructure, involvement of key opinion leaders and patient representatives and an industry alliance program, all to increase the chances of success for its programs and for the patients who would benefit.

To learn more about Unite To Cure: The Fourth International Vatican Conference, please visit: http://vaticanconference2018.com. Or, you can follow the event on Twitter @CuraFdn and on Facebook at facebook.com/TheCuraFoundation, and join the conversation with #UnitetoCure.

A road trip to the Inland Empire highlights a hot bed of stem cell research

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Gillian Wilson, Interim Vice Chancellor, Research, UC Riverside welcomes people to the combined Research Roadshow and Patient Advocate event

It took us longer than it should have to pay a visit to California’s Inland Empire, but it was definitely worth the wait. Yesterday CIRM’s Roadshow went to the University of California at Riverside (UCR) to talk to the community there – both scientific and public – about the work we are funding and the progress being made, and to hear from them about their hopes and plans for the future.

As always when we go on the road, we learn so much and are so impressed by everyone’s passion and commitment to stem cell research and their belief that it’s changing the face of medicine as we know it.

Dr. Deborah Deas, the Dean of the UC Riverside School of Medicine and a CIRM Board member, kicked off the proceedings by saying:

“Since CIRM was created in 2004 the agency has been committed to providing the technology and research to meet the unmet needs of the people of California.

On the Board I have been impressed by the sheer range and number of diseases targeted by the research CIRM is funding. We in the Inland Empire are playing our part. With CIRM’s help we have developed a strong program that is doing some exciting work in discovery, education and translational research.”

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CIRM’s Dr. Maria Millan at the Roadshow Patient Advocate event

CIRM’s President and CEO, Dr. Maria T. Millan, and our Board Chair, Jonathan Thomas then gave a quick potted history of CIRM and the projects we are funding. They highlighted how we are creating a pipeline of products from the Discovery, or basic level of research, through to the 45 clinical trials we are funding.

They also talked about the Alpha Clinic Network, based at six highly specialized medical centers around California, that are delivering stem cell therapies and sharing the experiences and knowledge learned from these trials to improve their ability to help patients and advance the field.

Researchers from both UCR then gave a series of brief snapshots of the innovative work they are doing:

  • Looking at new, more efficient and effective ways of expanding the number of human embryonic stem cells in the laboratory to create the high volume of cells needed for therapies.
  • Using biodegradable materials to help repair and regenerate tissue for things as varied as bone and cartilage repair or nerve restoration.
  • Exploring the use of epigenetic factors, things that switch genes on and off, to try and find ways to make repairs inside the body, rather than taking the cells outside the body, re-engineering them and returning them to the body. In essence, using the body as its own lab to manufacture replacement.

Another CIRM Board member, Linda Malkas, talked about the research being done at City of Hope (COH), where she is the associate chair of the Department of Molecular and Cellular Biology, calling it an “engine for discovery that has created the infrastructure and attracted people with an  amazing set of skills to bring forward new therapeutics for patients.”

She talked about how COH is home to one of the first Alpha Clinics that CIRM funded, and that it now has 27 active clinical trials, with seven more pending and 11 more in the pipeline.

“In my opinion this is one of the crown jewels of the CIRM program. CIRM is leading the nation in showing how to put together a network of specialized clinics to deliver these therapies. The National Institutes of Health (NIH) came to CIRM to learn from them and to talk about how to better move the most promising ideas and trials through the system faster and more efficiently.”

Dr. Malkas also celebrated the partnership between COH and UCR, where they are collaborating on 19 different projects, pooling their experience and expertise to advance this research.

Finally, Christine Brown, PhD, talked about her work using chimeric antigen receptor (CAR) T cells to fight cancer stem cells. In this CIRM-funded clinical trial, Dr. Brown hopes to re-engineer a patient’s T cells – a key cell of the immune system – to recognize a target protein on the surface of brain cancer stem cells and kill the tumors.

It was a packed event, with an overflow group watching on monitors outside the auditorium. The questions asked afterwards didn’t just focus on the research being done, but on research that still needs to be done.

One patient advocate couple asked about clinics offering stem cell therapies for Parkinson’s disease, wondering if the therapies were worth spending more than $10,000 on.

Dr. Millan cautioned against getting any therapy that wasn’t either approved by the Food and Drug Administration (FDA) or wasn’t part of a clinical trial sanctioned by the FDA. She said that in the past, these clinics were mostly outside the US (hence the term “stem cell tourism”) but increasingly they are opening up centers here in the US offering unproven and unapproved therapies.

She said there are lots of questions people need to ask before signing up for a clinical trial. You can find those questions here.

The visit was a strong reminder that there is exciting stem cell research taking place all over California and that the Inland Empire is a key player in that research, working on projects that could one day have a huge impact in changing people’s lives, even saving people’s lives.

 

California gets first royalty check from Stem Cell Agency investments

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.”

 

Stem Cell Awareness Day: Past, Present, Future

In 2008, the then California Governor Arnold Schwarzenegger  declared Sept. 25 to be Stem Cell Awareness Day. In the proclamation he said, ”The discoveries being made today in our Golden State will have a great impact on many around the world for generations to come.”

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Bob Klein (Left), Arnold Schwarzenegger (Middle), Don Reed (Right) in 2008.

In the years since, we have moved steadily towards turning those words into reality and using Stem Cell Awareness Day, now celebrated on the second Wednesday in October, as a symbol of the progress being made, not just in California but around the world.

Yesterday, for example, at a public event at UC Davis in Sacramento, Dr. Jan Nolta told an audience of patients, patient advocates, researchers and stem cell supporters that “we are part of a new era in medicine, one where it will one day be routine for prescriptions to be written for stem cell treatments for many different diseases.”

Those sentiments were echoed by Jonathan Thomas, Chair of the CIRM Board, who said:

“This is a time of truly extraodinary medical science.  We are lucky because, in our lifetime, we are going to see many of the biggest maladies plaguing people cured, in part because of developments in regenerative medicine. Every week you read about extraordinary developments in medicine and often those are here in California.”

In the early years Stem Cell Awareness Day was very much a creation of CIRM. We worked closely with our partners in academia and industry to host or stage events around the state. In 2009 for example, more than 40 CIRM grantees went to high schools in California, talking about stem cell research to more than 3,000 students. We also coordinated with researchers in Canada and Australia to create a global community of supporters.

We even hosted a poetry competition. No, really, we did. So, clearly not every idea we had back then was a winner.

These days CIRM doesn’t play as prominent a role in organizing these events for a very simple reason. We don’t have to. They have become such a popular part of the scientific calendar that individual institutions and schools organize their own events, without any pushing or prodding from us (though we are always happy to help when asked).

At UC Irvine this afternoon there is an Open House where you can take a self-guided tour of the facility, meet some of the scientists and watch lab demonstrations.

This weekend the UC  Berkeley’s Student Society of Stem Cell Research (SSSCR) is hosting its 5th annual Stem Cell Conference: Culturing a Stem Cell Community. This conference aims to bring together different aspects of stem cell research, from science to advocacy, to demonstrate the growth and success of the field. You can RSVP on Eventbrite (tickets cost a small fee of $7 or $12 including lunch to support the cost of the SSSCR conference)

The Gladstone Institutes in San Francisco just posted two new videos to its YouTube site:

In the early days of CIRM, Stem Cell Awareness Day was a valuable way for us to talk directly to the people of California – the ones who created CIRM. We felt it was important to let them know how their money was being spend and about the progress being made in stem cell research. And in the early years that progress was slower than all of us would have liked. Today, it’s a very different situation with CIRM now having funded 40 projects in clinical trials (and a goal of funding dozens more in the coming years) and with advances being made every day. We still reach out to our supporters and the patient advocate community but now we do it year round through our blog, social media and public events like the one yesterday at UC Davis.

While we are not as “hands on” as we were in the past we are still more than happy to provide tools for groups or organizations who want to hold their own stem cell awareness event – and it doesn’t have to be on October 11th, it can be any day of the year. Visit our Education Portal, Patient Resources page and video archive for various teaching tools.

CIRM Board Appoints Dr. Maria Millan as President and CEO

Dr. Maria Millan, President and CEO of CIRM, at the September Board meeting. (Todd Dubnicoff, CIRM)

Yesterday was a big day for CIRM. Our governing Board convened for its September ICOC meeting and appointed Dr. Maria Millan as our new President and CEO. Dr. Millan has been serving as the Interim President/CEO since July, replacing former President Dr. Randal Mills.

Dr. Millan has been at CIRM since 2012 and was instrumental in the development of CIRM’s infrastructure programs including the Alpha Stem Cell Clinics Network and the agency’s Strategic Plan, a five-year plan that lays out our agency’s goals through 2020. Previously, Dr. Millan was the Vice President of Therapeutics at CIRM, helping the agency fund 23 new clinical trials since the beginning of 2016.

The Board vote to appoint Dr. Millan as President and CEO was unanimous and enthusiastic. Chairman of the Board, Jonathan Thomas, shared the Board’s sentiments when he said,

“Dr. Millan is absolutely the right person for this position. Having seen Dr. Millan as the Interim CEO of CIRM for three months and how she has operated in that position, I am even more enthusiastic than I was before. I am grateful that we have someone of Maria’s caliber to lead our Agency.”

Dr. Millan has pursued a career devoted to helping patients. Before working at CIRM, she was an organ transplant surgeon and researcher and served as an Associate Professor of Surgery and Director of the Pediatric Organ Transplant Program at Stanford University. Dr. Millan was also the Vice President and Chief Medical Officer at StemCells, Inc.

In her permanent role as President, Dr. Millan is determined to keep CIRM on track to achieve the goals outlined in our strategic plan and to achieve its mission to accelerate treatments to patients with unmet needs. She commented in a CIRM press release,

“I joined the CIRM team because I wanted to make a difference in the lives of patients. They are the reason why CIRM exists and why we fund stem cell research. I am humbled and very honored to be CIRM’s President and look forward to further implementing our agency’s Strategic Plan in the coming years.”

The Board also voted to fund two new Alpha Stem Cell Clinics at UC Davis and UC San Francisco and five new clinical trials. Three of the clinical awards went to projects targeting cancer.

The City of Hope received $12.8 million to fund a Phase 1 trial targeting malignant gliomas (an aggressive brain cancer) using CAR-T cell therapy. Forty Seven Inc. received $5 million for a Phase 1b clinical trial treating acute myeloid leukemia. And Nohla Therapeutics received $6.9 million for a Phase 2 trial testing a hematopoietic stem cell and progenitor cell therapy to help patients suffering from neutropenia, a condition that leaves people susceptible to deadly infections, after receiving chemotherapy for acute myeloid leukemia.

The other two trials target diabetes and end stage kidney failure. ViaCyte, Inc. was awarded $20 million to fund a Phase 1/2 clinical trial to test its PEC-Direct islet cell replacement therapy for high-risk type 1 diabetes. Humacyte Inc. received $14.1 million to fund a Phase 3 trial that is comparing the performance of its acellular bioengineered vessel with the current standard of dialysis treatment for kidney disease patients.

The Board also awarded $5.2 million to Stanford Medicine for a late stage preclinical project that will use CRISPR gene editing technology to correct the sickle cell disease mutation in blood-forming stem cells to treat patients with sickle cell disease. This award was particularly well timed as September is Sickle Cell Awareness month.

The Stanford team, led by Dr. Matthew Porteus, hopes to complete the final experiments required for them to file an Investigational New Drug (IND) application with the FDA so they can be approved to start a clinical trial hopefully sometime in 2018. You can read more about Dr. Porteus’ work here and you can read our past blogs featuring Sickle Cell Awareness here and here.

With the Board’s vote yesterday, CIRM’s clinical trial count rises to 40 funded trials since its inception. 23 of these trials were funded after the launch of our Strategic Plan bringing us close to the half way point of funding 50 new clinical trials by 2020. With more “shots-on-goal” CIRM hopes to increase the chances that one of these trials will lead to an FDA-approved therapy for patients.


Related Links:

Confusing cancer to kill it

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Thomas Kipps, MD, PhD: Photo courtesy UC San Diego

Confusion is not a state of mind that we usually seek out. Being bewildered is bad enough when it happens naturally, so why would anyone actively pursue it? But now some researchers are doing just that, using confusion to not just block a deadly blood cancer, but to kill it.

Today the CIRM Board approved an investment of $18.29 million to Dr. Thomas Kipps and his team at UC San Diego to use a one-two combination approach that we hope will kill Chronic Lymphocytic Leukemia (CLL).

This approach combines two therapies, cirmtuzumab (a monoclonal antibody developed with CIRM funding, hence the name) and Ibrutinib, a drug that has already been approved by the US Food and Drug Administration (FDA) for patients with CLL.

As Dr. Maria Millan, our interim President and CEO, said in a news release, the need for a new treatment is great.

“Every year around 20,000 Americans are diagnosed with CLL. For those who have run out of treatment options, the only alternative is a bone marrow transplant. Since CLL afflicts individuals in their 70’s who often have additional medical problems, bone marrow transplantation carries a higher risk of life threatening complications. The combination approach of  cirmtuzumab and Ibrutinib seeks to offer a less invasive and more effective alternative for these patients.”

Ibrutinib blocks signaling pathways that leukemia cells need to survive. Disrupting these pathways confuses the leukemia cell, leading to its death. But even with this approach there are cancer stem cells that are able to evade Ibrutinib. These lie dormant during the therapy but come to life later, creating more leukemia cells and causing the cancer to spread and the patient to relapse. That’s where cirmtuzumab comes in. It works by blocking a protein on the surface of the cancer stem cells that the cancer needs to spread.

It’s hoped this one-two punch combination will kill all the cancer cells, increasing the number of patients who go into complete remission and improve their long-term cancer control.

In an interview with OncLive, a website focused on cancer professionals, Tom Kipps said Ibrutinib has another advantage for patients:

“The patients are responding well to treatment. It doesn’t seem like you have to worry about stopping therapy, because you’re not accumulating a lot of toxicity as you would with chemotherapy. If you administered chemotherapy on and on for months and months and years and years, chances are the patient wouldn’t tolerate that very well.”

The CIRM Board also approved $5 million for Angiocrine Bioscience Inc. to carry out a Phase 1 clinical trial testing a new way of using cord blood to help people battling deadly blood disorders.

The standard approach for this kind of problem is a bone marrow transplant from a matched donor, usually a family member. But many patients don’t have a potential donor and so they often have to rely on a cord blood transplant as an alternative, to help rebuild and repair their blood and immune systems. However, too often a single cord blood donation does not have enough cells to treat an adult patient.

Angiocrine has developed a product that could help get around that problem. AB-110 is made up of cord blood-derived hematopoietic stem cells (these give rise to all the other types of blood cell) and genetically engineered endothelial cells – the kind of cell that lines the insides of blood vessels.

This combination enables the researchers to take cord blood cells and greatly expand them in number. Expanding the number of cells could also expand the number of patients who could get these potentially life-saving cord blood transplants.

These two new projects now bring the number of clinical trials funded by CIRM to 35. You can read about the other 33 here.

 

 

 

Stem cell agency funds Phase 3 clinical trial for Lou Gehrig’s disease

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At CIRM we don’t have a disease hierarchy list that we use to guide where our funding goes. We don’t rank a disease by how many people suffer from it, if it affects children or adults, or how painful it is. But if we did have that kind of hierarchy you can be sure that Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, would be high on that list.

ALS is a truly nasty disease. It attacks the neurons, the cells in our brain and spinal cord that tell our muscles what to do. As those cells are destroyed we lose our ability to walk, to swallow, to talk, and ultimately to breathe.

As Dr. Maria Millan, CIRM’s interim President and CEO, said in a news release, it’s a fast-moving disease:

“ALS is a devastating disease with an average life expectancy of less than five years, and individuals afflicted with this condition suffer an extreme loss in quality of life. CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurological condition for which there is currently no cure.”

Having given several talks to ALS support groups around the state, I have had the privilege of meeting many people with ALS and their families. I have seen how quickly the disease works and the devastation it brings. I’m always left in awe by the courage and dignity with which people bear it.

BrainStorm

I thought of those people, those families, today, when our governing Board voted to invest $15.9 million in a Phase 3 clinical trial for ALS run by BrainStorm Cell Therapeutics. BrainStorm is using mesenchymal stem cells (MSCs) that are taken from the patient’s own bone marrow. This reduces the risk of the patient’s immune system fighting the therapy.

After being removed, the MSCs are then modified in the laboratory to  boost their production of neurotrophic factors, proteins which are known to help support and protect the cells destroyed by ALS. The therapy, called NurOwn, is then re-infused back into the patient.

In an earlier Phase 2 clinical trial, NurOwn showed that it was safe and well tolerated by patients. It also showed evidence that it can help stop, or even reverse  the progression of the disease over a six month period, compared to a placebo.

CIRM is already funding one clinical trial program focused on treating ALS – that’s the work of Dr. Clive Svendsen and his team at Cedars Sinai, you can read about that here. Being able to add a second project, one that is in a Phase 3 clinical trial – the last stage before, hopefully, getting approval from the Food and Drug Administration (FDA) for wider use – means we are one step closer to being able to offer people with ALS a treatment that can help them.

Diane Winokur, the CIRM Board Patient Advocate member for ALS, says this is something that has been a long time coming:

CIRM Board member and ALS Patient Advocate Diane Winokur

“I lost two sons to ALS.  When my youngest son was diagnosed, he was confident that I would find something to save him.  There was very little research being done for ALS and most of that was very limited in scope.  There was one drug that had been developed.  It was being released for compassionate use and was scheduled to be reviewed by the FDA in the near future.  I was able to get the drug for Douglas.  It didn’t really help him and it was ultimately not approved by the FDA.

When my older son was diagnosed five years later, he too was convinced I would find a therapy.  Again, I talked to everyone in the field, searched every related study, but could find nothing promising.

I am tenacious by nature, and after Hugh’s death, though tempted to give up, I renewed my search.  There were more people, labs, companies looking at neurodegenerative diseases.

These two trials that CIRM is now funding represent breakthrough moments for me and for everyone touched by ALS.  I feel that they are a promising beginning.  I wish it had happened sooner.  In a way, though, they have validated Douglas and Hugh’s faith in me.”

These therapies are not a cure for ALS. At least not yet. But what they will do is hopefully help buy people time, and give them a sense of hope. For a disease that leaves people desperately short of both time and hope, that would be a precious gift. And for people like Diane Winokur, who have fought so hard to find something to help their loved ones, it’s a vindication that those efforts have not been in vain.

CIRM & NIH: a dynamic duo to advance stem cell therapies

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National Institutes of Health

There’s nothing more flattering than to get an invitation, out of the blue, from someone you respect, and be told that they are interested in learning about the way you work, to see if it can help them improve the way they work.

That’s what happened to CIRM recently. I will let Randy Mills, who was our President & CEO at the time, pick up the story:

“Several weeks ago I got a call from the head of the National Heart. Lung and Blood Institute (NHLBI) asking would we be willing to come out to the National Institutes of Health (NIH) and talk about what we have been doing, the changes we have made and the impact they are having.”

Apparently people at the NIH had been reading our Strategic Plan and our Annual Report and had been hearing good things about us from many different individuals and organizations. We also heard that they had been motivated to engage more fully with the regenerative medicine community following the passage of the 21st Century Cures Act.

We were expecting a sit down chat with them but we got a lot more than that. They blocked out one and a half days for us so that we had the time to engage in some in-depth, thoughtful conversations about how to advance the field.

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Dr. Francis Collins, NIH Director

The meeting was kicked off by both Francis Collins, the NIH Director, and Gary Gibbons, the NHLBI Director. Then the CIRM team – Dr. Mills, Dr. Maria Millan, Gabe Thompson and James Harrison – gave a series of presentations providing an overview of how CIRM operates, including our vision and strategic priorities, our current portfolio, the lessons learned so far, our plans for the future and the challenges we face.

The audience included the various heads and representatives from the various NIH Institutes who posed a series of questions for us to answer, such as:

  • What criteria do we use to determine if a project is ready for a clinical trial?
  • How do we measure success?
  • How have our strategies and priorities changed under CIRM 2.0?
  • How well are those strategies working?

The conversation went so well that the one day of planned meetings were expanded to two. Maria Millan, now our interim President & CEO, gave an enthusiastic summary of the talks

“The meetings were extremely productive!  After meeting with Dr. Collins’ group and the broader institute, we had additional sit down meetings.   The NIH representatives reported that they received such enthusiastic responses from Institute heads that they extended the meeting into a second day. We met with with the National Institutes of Dental and Craniofacial Research, Heart, Lung and Blood, Eye Institute, Institute on Aging, Biomedical Imaging and Bioengineering, Diabetes, and Digestive and Kidney Diseases, and the National Center for Advancing Translational Sciences.  We covered strategic and operational considerations for funding the best science in the stem cell and regenerative medicine space.  We explored potential avenues to join forces and leverage the assets and programs of both organizations, to accelerate the development of regenerative medicine and stem cell treatments.”

This was just a first meeting but it laid the groundwork for what we hope will be a truly productive partnership. In fact, shortly after returning from Washington, D.C., CIRM was immediately invited to follow-up NIH workgroups and meetings.

As this budding partnership progresses we’ll let you know how it’s working out.