Stem Cell Agency Invests in New Immunotherapy Approach to HIV, Plus Promising Projects Targeting Blindness and Leukemia

HIV AIDS

While we have made great progress in developing therapies that control the AIDS virus, HIV/AIDS remains a chronic condition and HIV medicines themselves can give rise to a new set of medical issues. That’s why the Board of the California Institute for Regenerative Medicine (CIRM) has awarded $3.8 million to a team from City of Hope to develop an HIV immunotherapy.

The City of Hope team, led by Xiuli Wang, is developing a chimeric antigen receptor T cell or CAR-T that will enable them to target and kill HIV Infection. These CAR-T cells are designed to respond to a vaccine to expand on demand to battle residual HIV as required.

Jeff Sheehy

CIRM Board member Jeff Sheehy

Jeff Sheehy, a CIRM Board member and patient advocate for HIV/AIDS, says there is a real need for a new approach.

“With 37 million people worldwide living with HIV, including one million Americans, a single treatment that cures is desperately needed.  An exciting feature of this approach is the way it is combined with the cytomegalovirus (CMV) vaccine. Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.”

This is a late stage pre-clinical program with a goal of developing the cell therapy and getting the data needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial.

The Board also approved three projects under its Translation Research Program, this is promising research that is building on basic scientific studies to hopefully create new therapies.

  • $5.068 million to University of California at Los Angeles’ Steven Schwartz to use a patient’s own adult cells to develop a treatment for diseases of the retina that can lead to blindness
  • $4.17 million to Karin Gaensler at the University of California at San Francisco to use a leukemia patient’s own cells to develop a vaccine that will stimulate their immune system to attack and destroy leukemia stem cells
  • Almost $4.24 million to Stanford’s Ted Leng to develop an off-the-shelf treatment for age-related macular degeneration (AMD), the leading cause of vision loss in the elderly.

The Board also approved funding for seven projects in the Discovery Quest Program. The Quest program promotes the discovery of promising new stem cell-based technologies that will be ready to move to the next level, the translational category, within two years, with an ultimate goal of improving patient care.

Application Title Institution CIRM Committed Funding
DISC2-10979 Universal Pluripotent Liver Failure Therapy (UPLiFT)

 

Children’s Hospital of Los Angeles $1,297,512

 

DISC2-11105 Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer

 

Stanford $1,415,016
DISC2-10973 Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases

 

U.C. San Diego $1,160,648
DISC2-11070 Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs

 

Scripps $1,827,576
DISC2-11183 A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

 

University of Southern California $833,971
DISC2-11199 Modulation of the Wnt pathway to restore inner ear function

 

Stanford $1,394,870
DISC2-11109 Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome

 

Stanford $1,415,016

Finally, the Board approved the Agency’s 2019 research budget. Given CIRM’s new partnership with the National Heart, Lung, Blood Institute (NHLBI) to accelerate promising therapies that could help people with Sickle Cell Disease (SCD) the Agency is proposing to set aside $30 million in funding for this program.

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Congresswoman Barbara Lee (D-CA 13th District)

“I am deeply grateful for organizations like CIRM and NHLBI that do vital work every day to help people struggling with Sickle Cell Disease,” said Congresswoman Barbara Lee (D-CA 13th District). “As a member of the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, I know well the importance of this work. This innovative partnership between CIRM and NHLBI is an encouraging sign of progress, and I applaud both organizations for their tireless work to cure Sickle Cell Disease.”

Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund promising therapies targeting SCD.  Programs that are ready to start an IND-enabling or clinical trial project for sickle cell can apply to CIRM for funding from both agencies. CIRM will share application information with the NHLBI and CIRM’s Grants Working Group (GWG) – an independent panel of experts which reviews the scientific merits of applications – will review the applications and make recommendations. The NHLBI will then quickly decide if it wants to partner with CIRM on co-funding the project and if the CIRM governing Board approves the project for funding, the two organizations will agree on a cost-sharing partnership for the clinical trial. CIRM will then set the milestones and manage the single CIRM award and all monitoring of the project.

“This is an extraordinary opportunity to create a first-of-its-kind partnership with the NHLBI to accelerate the development of curative cell and gene treatments for patients suffering with Sickle Cell Disease” says Maria T. Millan, MD, President & CEO of CIRM. “This allows us to multiply the impact each dollar has to find relief for children and adults who battle with this life-threatening, disabling condition that results in a dramatically shortened lifespan.  We are pleased to be able to leverage CIRM’s acceleration model, expertise and infrastructure to partner with the NHLBI to find a cure for this condition that afflicts 100,000 Americans and millions around the globe.”

The budget for 2019 is:

Program type 2019
CLIN1 & 2

CLIN1& 2 Sickle Cell Disease

$93 million

$30 million

TRANSLATIONAL $20 million
DISCOVER $0
EDUCATION $600K

 

 

California’s Stem Cell Agency Accelerates Treatments to Patients

The following article is an Op Ed that appeared in today’s print version of the San Francisco Chronicle

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Biotechnology was born in California in the 1970s based on the discovery out of one of its universities and California is responsible for an industry that has impacted the lives of billions of people worldwide. In 2004, the voters of California approved Proposition 71, creating the California Institute for Regenerative Medicine and setting the state on the path to becoming a global leader in stem cell research. Today the therapies resulting from the institute’s work are not just changing lives, they are already saving lives.

Lives like Evie Vaccaro, who is alive today because of a treatment CIRM is funding. Vaccaro was born with SCID, also known as “bubble baby disease,” an immune disorder that often kills babies in their first two years. Vaccaro and dozens of other babies were given stem cell treatments thanks to the institute. All are showing improvement; some are now several years past treatment and considered cured.

An accident left Jake Javier from Danville paralyzed from the chest down on the eve of his high school graduation. Javier was treated in a CIRM-funded clinical trial. Today he has regained the use of his arms and hands, is driving a car and is a sophomore at Cal Poly San Luis Obispo. Five other patients treated at the same time as Javier have all experienced improvements meaning that instead of needing round-the-clock care, they can lead independent lives.

A study by the Tufts Center for the Study of Drug Development estimated it takes at least 10 years and $2.6 billion to develop one successful drug. In 14 years, and with just $3 billion, CIRM has funded 1,000 different projects, enrolled 900 patients, and supported 49 different clinical trials targeting diseases such as cancer, kidney failure and leukemia. Four of these programs have received an expedited designation by the U.S. Food and Drug Administration, meaning they could get faster approval to help more patients

We have created a network of world class medical clinics that have expertise in delivering treatments to patients. The CIRM Alpha Clinics offer treatments based on solid science, unlike the unlicensed clinics sprouting up around California that peddle unproven and potentially harmful therapies that cost patients thousands of dollars.

CIRM has:

  • Supported the creation of 12 stem-cell research facilities in California
  • Attracted hundreds of top-tier researchers to California
  • Trained a new generation of stem-cell scientists
  • Brought clinical trials to California — for example, one targeting ALS or Lou Gehrig’s disease
  • Deployed rigorous scientific standards and support so our programs have a “seal of approval” to attract $2.7 billion in additional investments from industry and other sources.

We recently have partnered with the National Institutes of Health to break down barriers and speed up the approval process to bring curative treatments to patients with Sickle Cell Disease.

Have we achieved all we wanted to? Of course not. The first decade of CIRM’s life was laying the groundwork, developing the knowledge and expertise and refining processes so that we can truly accelerate progress. As a leader in this burgeoning field of regenerative medicine, CIRM needs to continue its mission of accelerating stem-cell treatments to patients with unmet medical needs.

Dr. Maria T. Millan is President and CEO and Jonathan Thomas, JD, PhD, is the Board Chairman of the California Institute of Regenerative Medicine. 

 

 

How small talk led to a big break; a summer internship at CIRM

At CIRM, California’s Stem Cell Agency, we are fortunate to work with some amazing people. This summer we added another name that list when Melissa Cairos joined us for an internship. Melissa is now on to the next part of her adventure, as a policy wonk in Washington DC., but before she left we asked her to write about her experiences, and thoughts after her time at the Stem Cell Agency.

Melissa

Melissa Cairos

In January of 2018, I had a casual conversation with a woman, whom I had never met before, at a high school basketball game. Through small talk about my studies in school and my career interests for the future, the woman suggested I may be interested in her work because it seemed to be aligned with what I wanted to do. Her work happened to be at CIRM and she happened to be Maria Millan, the President and CEO.

Interestingly, I had never heard of CIRM (the California Institute for Regenerative Medicine) and had limited knowledge of regenerative medicine. But, I had dedicated a semester in spring of 2015 to analyzing and lobbying for the 21st Century Cures Act. I engaged in that work because I believe in the importance of investing in, and expediting the regulatory process for, lifesaving medical innovations, so that they can be accessed faster by patients and at a lower cost. The 21st Century Cures Act has since become law and has created incredible opportunities for both CIRM and the entire field of regenerative medicine.

Since joining CIRM, I have been able to continue with similar work by analyzing legislation, policies and regulations that affect patients’ abilities to access regenerative medicine therapies and our grantees’ abilities to receive reimbursement for their therapies. Because the stem cell and gene therapies CIRM’s grantees are coming up with are so new and innovative, I quickly realized that the legislative, policy and regulatory solutions for them needed to reflect that innovative spirit.

Working alongside Geoff Lomax, (the Senior Officer for CIRM Strategic Infrastructures)  my manager and mentor, we identified a number of potential barriers to access and reimbursement and tried to come up with policy solutions to address them.

For one project, we looked at the high cost of regenerative medicine therapies. Because high cost affects both patient access and potential reimbursement problems for the companies that develop those therapies we felt it was essential to try and come up with policy solutions to address these issues. To do this, we studied the traditional payment structure for drugs and medical devices and found it inappropriate for regenerative medicine in most cases.

This is because regenerative medicine requires a one-time high cost payment, but the regenerative medicine treatments/cures would eliminate long term costs including: previous treatment cost, complications from that treatment, progression of disease cost, hospitalizations, disability, quality of life, co-morbidities, disease effect on longevity etc. Thus, we proposed that payment models for regenerative medicine should consider their unique value benefits, such as the number of additional years of life the treatment added, and the overall cost-effectiveness of a one-time treatment compared to years of  treatment. With this in mind, we suggested innovative payment models that accounted for these factors and further proposed changes that need to be made so that different manufacturers and payors can engage in innovative financing agreements.

Through my work at CIRM, I found that what makes regenerative medicine unique is that it not only offers new ways of treating previously untreatable diseases, but it has additional benefits or value. Not only the economic value, but also the human value, as regenerative medicine offers patients with life threatening or painful chronic diseases a solution that will change their lives and the lives of their families for the better. Through this understanding, I grew an incredible appreciation for CIRM, for not only being a great place to work with incredibly talented and kind people, but also an incredibly unique government agency that reflected the value and innovative spirit of the research it supports.

I am so grateful that I met Maria at that basketball game and got the opportunity to support CIRM in adding value to California in my role this summer as a Policy Fellow. I plan to return to California in the future and work in the health policy field to further support programs, policies, and/or agencies, like CIRM, that bring so much value to this state.

 

 

A brief history of the Stem Cell Agency

On Wednesday, August 15 the California State Assembly Select Committee on Biotechnology held an informational hearing on CIRM as part of its mission of ensuring the legislature is up to date and informed about the biotech industry in California. The committee heard from CIRM’s President and CEO Dr. Maria T. Millan and the Vice Chair of our Board, Senator Art Torres (Ret.); two of CIRM’s Patient Advocates (Pawash Priyank and Don Reed) and Dr. Jan Nolta, the Director of the Institute for Regenerative Cures at UC Davis.

The final speaker was David Jensen, whose California Stem Cell Report blog has charted the history of CIRM since its inception. At CIRM we know that not everyone agrees with us all the time, or supports all the decisions we have made in the years since we were approved by voters in 2004, but we do pride ourselves on being open to a thoughtful, vigorous debate on all aspects of stem cell research. David’s presentation to the committee was nothing if not thoughtful, and we thought you might enjoy reading it and so we are presenting it here in its entirety.

For those who prefer to watch than read, here is a video of the entire hearing:

https://www.assembly.ca.gov/media/assembly-select-committee-biotechnology-20180815/video

California’s Stem Cell “Gold Rush:” A Brief Overview of the State’s $3 Billion Stem Cell Agency
Prepared testimony by David Jensen, publisher/editor of the California Stem Cell Report, before the Assembly Select Committee on Biotechnology, Aug. 15, 2018
I was in Mazatlan in Mexico in the fall of 2004 when I first heard about the creation of
California’s stem cell agency. I was reading the Wall Street Journal online and saw a headline that said a new Gold Rush was about to begin in California — this one involving stem cells instead of nuggets.

“Holy Argonauts,” I said to myself, using the term, of course, that refers to the tens of thousands of people who rushed to the California gold fields in 1849. I wanted to know more about what was likely to happen with this new stem cell gold rush.

Today, nearly 14 years later, I still want to know more about the California Institute for
Regenerative Medicine or CIRM, as the agency is formally known. But I can tell you that certain facts are clear.

Borrowing and Autonomy
The agency is unique in California history and among the states throughout the nation. It is the first state agency to fund scientific research with billions of dollars – all of it borrowed. At one point in its history, it is safe to say that the agency was the largest single source of funding in the world for human embryonic stem cell research.

The agency operates with financial and oversight autonomy that is rare in California government, courtesy of the ballot initiative that created it. But that measure also proved to be both a blessing and a curse. The agency’s financial autonomy has allowed it to provide a reasonably steady stream of cash over a number of years, something that is necessary to sustain the long-term research that is critical for development of widely available treatments.

At the same time, the ballot measure carried the agency’s death warrant — no more money after the $3 billion was gone. Cash for new awards is now expected to run out at the end of next year. Over its life, the agency has had a national and somewhat more modestly global impact, both as a source of funding and international cooperation, but also in staying the course on human embryonic stem cell research when the federal government was backing away from it.

Beyond that, the stem cell agency is the only state department whose primary objective is to produce a marketable commercial product. In this case, a cure or treatment for afflictions now nearly untreatable.

Finally, I am all but certain that CIRM is the only state agency that takes back money when a project winds up on the rocks. By the end of last month, that figure totalled in recent years more than $34 million in two big categories of awards. This sort of cash recovery is not a practice that occurs with federal research dollars. With CIRM the money goes back into the pot for more research aimed at treating horrible afflictions.

Evaluations of the Research Effort
Nonetheless the agency has hit some shoals from time to time. In 2010, the agency’s governing board commissioned a $700,000 study of its efforts by the prestigious Institute of Medicine. Two years later, the IOM reported to CIRM that it had some significant flaws.

The IOM study said that the agency had “achieved many notable results.” But it also
recommended sweeping changes to remove conflict of interest problems, clean up a troubling dual-executive arrangement and fundamentally change the nature of the governing board.

The report said,“Far too many board members represent organizations that receive CIRM funding or benefit from that funding. These competing personal and professional interests compromise the perceived independence of the ICOC (the CIRM governing board), introduce potential bias into the board’s decision making, and threaten to undermine confidence in the board.”

The conflict issues are built in by the ballot measure, which gave potential recipient institutions seats on the 29-member governing board. Indeed, in 2017, the last time I calculated the correlation between the board and awards, roughly 90 percent of the money given out by CIRM had gone to institutions with ties to members of the governing board.

About two months after the IOM presented its report, the CIRM board approved a new policy that bars 13 of its 29 members from voting on any grants whatsoever to help deal with questions concerning conflicts of interest on the board.

Other studies about the agency’s performance resulted from a 2010 law in which the legislature modified the initiative to require triennial performance audits that would be paid for by the agency itself. The requirement specifically excluded “scientific performance” from the audit.

The first audit results came in 2012 and contained 27 recommendations for improvement. The most recent performance audit came last spring. The audit firm, Moss Adams, recommended improvements in the areas of private fund-raising, retention of staff and better utilization of board members. The board was told that the agency had made “incredible progress” and that the auditors “usually see a lot of good things.”

The Story of CIRM 2.0
In recent years the agency has been on a self-improvement regime. The effort began in 2014 and was dubbed CIRM 2.0 — a term that was originally coined by a stem cell researcher at UC Davis.

The new direction and emphasis was described by the agency as “radical.” It was aimed at improving speed, efficiency and innovation. And it seems to have largely succeeded.
In 2014, it took almost two years for a good idea to go from application to the final funding stage. The goal was to shorten that to 120 days. Delays in funding are of particular concern to businesses, often for cash flow reasons, but they also mean delays in actually developing a treatment.

This week, the agency said the cash delivery figure now stands at less than 90 days for clinical awards and about 120 days for translational awards.

In 2014, the agency was participating in nine clinical trials, the last stage before a treatment is certified by the federal government for widespread use. Today the agency is involved in 49. In 2014, about 50 patients were involved in those trials. Today the figure is more than 800.

One of the more interesting aspects of CIRM 2.0 marked a departure from what might be called an academic pass-fail approach to the “final exam” for applications from scientists. Instead, CIRM is engaged in a more partner-oriented approach that can be found in some businesses.

Instead of flatly failing an application that is not quite ready for prime time, the idea is to coach applicants along to help bring them up to approval level. Today the agency can count 30 applications that won approval through that process. All of which is work could have slipped away in the more distant past.

CIRM and the Biotech Biz
CIRM is now much more engaged with industry than during its earlier years, when it drew bitter criticism from some business executives. Engagement with biotech firms is critical to bringing a treatment to the public. CIRM is not in the business of actually manufacturing, marketing and selling products. That is a matter left to the private sector.

One reason for closer business connections involves maturation of the work in the field, which has brought research closer to reality. But it is also due to a different focus within the agency as top management has changed.

One of the more difficult areas involving stem cell research and likely treatments is their cost. It is rare to hear researchers or companies talk forthrightly in public about specific dollar amounts. But the cost of drugs and treatment are high visibility matters for patients and elected officials. And estimates of stem cell treatments have run up to at least $900,000.

In 2010, the California legislature moved to help assure affordability by requiring grantees to submit affordable access plans with the caveat that the agency could waive that requirement. How that will ultimately play out as actual products come into the marketplace is yet to be determined.

The Public Policy Questions
A number of significant public policy questions surround the California’s stem cell program involving its creation and execution. They include:
● Is a ballot initiative the best way to approach research and create new state programs?
The initiative is very difficult to alter when changes are needed or priorities change. .
● Does the state have higher health priorities, such as prenatal health care, than supplying
researchers with cash that they could well secure from other sources?
● Is borrowing money to finance the research the best way to go about it? The interest
expense raise the total cost of a $20 million research award to $40 million.
● Should executives of potential recipient institutions serve on the board that awards their employers hundreds of millions of dollars?

This is just a short list of some of the policy matters. Other questions can and should be asked in the wake of the agency’s nearly 14 years of work.

Lives Saved but No Widespread Therapies
Returning to our earlier list of the clear facts about CIRM, another fact is that lives have been saved as the result of clinical trials that the agency it has helped to finance. The youngster from Folsom mentioned earlier in this hearing is one of a number of cases.

That said, these patients received treatment in clinical trials, which may or may not succeed in producing a commercial product that is available to the general public.

Little doubt exists that the agency has advanced the stem cell field and is building towards a critical mass in California. The burgeoning research program at UC Davis, with $138 million in CIRM funding, is one example. Another is the $50 million Alpha Clinic network aimed at creating powerful collaboration within institutions and throughout the state. In addition to Davis, UC San Francisco, UCLA, UC Irvine, UC San Diego and the City of Hope in the Los Angeles area are all part of the Alpha network.

Nonetheless, CIRM has not yet backed a stem cell treatment that is ready for widespread use and fulfilled the voter expectations from 2004 that stem cell cures were right around the corner.

The agency itself also has something of a deadline that is right around the corner in political and scientific terms. Backers of the agency are hoping for another ballot initiative in November 2020 that would pump $5 billion into the program and stave off its slow demise as research winds down. Development of a stem cell treatment that would resonate with voters would be an invaluable development to encourage voters to continue this unique experiment — even if California’s stem cell gold rush does not quite measure up to the dramatic events of 169 years ago.
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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

UCR#1

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

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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.