Reasons to be thankful this Thanksgiving: creative nerds

We at the California Institute for Regenerative Medicine have a lot to be thankful for this Thanksgiving. We get to work with some extraordinary colleagues, we get to know some remarkable patient advocates who are pioneers in volunteering for stem cell and gene therapies, and we have a front row seat in a movement that is changing the face of medicine.

We also get to work with some brilliant scientists and help support their research. As if we needed any reminders of how important that funding is, we thought we would share this video with you. It’s from the talented post docs and researchers at the University of California San Diego. It’s a delightful parody of the Cyndi Lauper classic “Girls Just Wanna Have Fun”. Only in this case it’s “Nerds Just Wanna Have Funds.”

Enjoy, and Happy Thanksgiving.

From our house to the White House. Kinda

Jackie Ward, PhD. Photo courtesy National Institute of Neurological Diseases and Stroke

It’s always fun to meet someone early in their career and see how they grow and evolve and take on new challenges.

I first met Jackie Ward when she received a training grant from CIRM while she studied for her PhD at the University of California, San Diego. Jackie offered to write blogs for us about her experience and they were always fun, informative, elegantly written and very engaging. Fast forward a few years and Jackie became a part of Americans For Cures, then she became Chief of Staff at the National Institute of Neurological Disorders and Stroke (NINDS), and finally – at least so far – she took on the role of Assistant Director at the White House Office of Science and Technology Policy (OSTP).

Not too shabby eh.

So, I reached out to Jackie and asked her some questions about her work and career. She generously put aside keeping the nation healthy to answer them. Enjoy.

  1. What made you decide to move from research into government.

I think if you asked my high school government teacher (shout out to Mr. Bell!), he would be the least surprised person that I have ended up where I am currently. I was always interested in topics and activities beyond science, but at a certain point you have to choose a path. When it came time to deciding my undergraduate major, I figured that if I pursued my interest in biology it would still keep my options open to do something different in my career, but if I chose to be a French major, or Political Science major, or English major – I might close the door in my ability to pursue scientific research. When I got to graduate school, I saw the impact of government (both state and federal) decisions on work in the lab. This takes the form of where funding goes, but also in the rules you have to follow while doing research. Though I liked the pursuit of new knowledge and being the one designing and performing experiments, I was interested in understanding more about how those government decisions are made upstream of the lab bench.

  1. What’s the most surprising thing you have learned in your time at the White House Office of Science and Technology Policy (OSTP).

Maybe not “surprising” but the thing that may not be obvious to outsiders: OSTP’s budget is tiny compared to other Executive Branch agencies (like where I came from previously at NIH). The work we accomplish in this office is solely by forming partnerships and collaborations with others across the government. We are not typically the rowers of the boat, but we can be the steerer or navigator. (Is the term coxswain? I have never been on a crew team obviously.)

  1. Was it hard making the transition from research to advocacy and now policy?

Honestly I feel like my training in research set me up well for the jobs I’ve had in policy. There is often not someone telling you exactly how to do something – you have to do the work yourself to search the literature, talk to other people, find collaborators, and keep at it. And the skills that you hone in research – from keeping an organized lab notebook the whole way through to writing scientific papers – are some of the same skills you need in government. 

  1. At a time when so many people seem so skeptical of science how do you get your message out.

We have to meet people where they are. As a government official, I have great respect for messages that come from experts within the government – but that is not the only way the message should be getting out. Scientists and other experts within communities should also be spokespeople for science. I would urge scientists at every level – whether you are a citizen scientist, a medical doctor, a PhD student, or some other kind of expert – to engage with their communities and put the work in to understand how to effectively communicate at levels beyond just speaking to your colleagues.

  1. One of the issues that so many of us, including here at CIRM, are working on is improving our performance in diversity, equity and inclusion. How big an issue is that for you and your colleagues at OSTP and what are you doing to try and address it.

The mission of our office is to “maximize the benefits of science and technology to advance health, prosperity, security, environmental quality, and justice for all Americans.” Those final two words are key: “all Americans.” It is the policy of this Office and our Administration that it is not okay for the benefits of science & technology to only reach a select few – who can afford it or who live in a certain zip code or who know the right people. 

This takes different forms depending on what kind of S&T work we are talking about, but I will give you an example from my own work. I have been leading an effort that aims to explore and act upon how digital health care delivery technologies can be used to increase access to healthcare in community-based health settings. We know that these cutting edge technologies are most likely to get to people who, for example, get their care at academic medical centers, or who have primo health insurance plans, or who are already tech savvy. We feel that as these technologies continue to grow within the healthcare system, that it is an imperative to ensure that they are accessible to practitioners and patients at community health centers, or to people who may not be tech geeks, or that they can be interoperable with the systems used by community health workers.

  1. During a time of Covid and now Monkeypox, what’s it like to have a front row seat and watch how government responds to public health emergencies.

My colleagues who work on outbreaks and pandemic responses are some of the most dedicated public servants I know. They will be the first to admit that we are continually learning and integrating new tools and technologies into our toolbox, and that is a constant effort. Emergent issues like outbreaks force decisions when there may not be a lot of information – that is a hard job.  

  1. I’ve always felt that DC would be a fun place to live and work (except during the height of summer!) what do you most like about it.

DC is a city full of people who care deeply (almost to a pathological extent) about the work they do and how to make the world a better place. There’s also incredible diversity here – which means a variety of viewpoints, languages, and food! I love that.

Jackie is not just a good writer. She’s also a great speaker. Here’s a clip of her responding to our Elevator Challenge many years ago, when she was still a fledgling researcher. Her explanation of what she does, is a master class in turning a complex subject into something easy to understand.

Creating a ‘bespoke’ approach to rare diseases

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Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a bespoke anything.

I was wrong. Because CIRM is now part of a bespoke arrangement. It has nothing to do with suits, it’s far more important than that. This bespoke group is aiming to create tailor-made gene therapies for rare diseases.

It’s called the Bespoke Gene Therapy Consortium (BGTC). Before we go any further I should warn you there’s a lot of acronyms heading your way. The BGTC is part of the Accelerating Medicines Partnership® (AMP®) program. This is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations, such as CIRM.

The program is managed by the Foundation for the NIH (FNIH) and it aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases.

Why is it necessary? Well, it’s estimated that there are around 7,000 rare diseases and these affect between 25-30 million Americans. Some of these diseases affect only a few hundred, or even a few dozen people. With so few people they almost always struggle to raise the funds needed to do research to find an effective therapy. However, many of these rare diseases are linked to a mutation or defect in a single gene, which means they could potentially be treated by highly customizable, “bespoke” gene therapy approaches.

Right now, individual disease programs tend to try individual approaches to developing a treatment. That’s time consuming and expensive. The newly formed BGTC believes that if we create a standardized approach, we could develop a template that can be widely used to develop bespoke gene therapies quickly, more efficiently and less expensively for a wide array of rare diseases.

“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as severe combined immunodeficiency,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much. This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases.”

With gene therapy the goal is to identify the genetic defect that is causing the disease and then deliver a normal copy of the gene to the right tissues and organs in the body, replacing or correcting the mutation that caused the problem. But what is the best way to deliver that gene? 

The BGTC’s is focusing on using an adeno-associated virus (AAV) as a delivery vehicle. This approach has already proven effective in Leber congenital amaurosis (LCA), retinitis pigmentosa (RP), and spinal muscular atrophy. The consortium will test several different approaches using AAV gene therapies starting with basic research and supporting those all the way to clinical trials. The knowledge gained from this collaborative approach, including developing ways to manufacture these AAVs and creating a standard regulatory approach, will help build a template that can then be used for other rare diseases to copy.

As part of the consortium CIRM will identify specific rare disease gene therapy research programs in California that are eligible to be part of the AMP BGTC. CIRM funding can then support the IND-enabling research, manufacturing and clinical trial activities of these programs.

“This knowledge network/consortium model fits in perfectly with our mission of accelerating transformative regenerative medicine treatments to a diverse California and world,” says Dr. Millan. “It is impossible for small, often isolated, groups of patients around the world to fund research that will help them. But pooling our resources, our skills and knowledge with the consortium means the work we support here may ultimately benefit people everywhere.”

CIRM Board gives thumbs up to training and treatment programs

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CIRM Bridges student discusses her poster presentation

At CIRM, the bread and butter of what we do is funding research and hopefully advancing therapies to patients. But the jam, that’s our education programs. Helping train the next generation of stem cell and gene therapy scientists is really inspiring. Watching these young students – and some are just high school juniors – come in and grasp the science and quickly become fluent in talking about it and creating their own experiments shows the future is in good hands.

Right now we fund several programs, such as our SPARK and Bridges internships, but they can’t cover everything, so last week the CIRM Board approved a new training program called COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem Cell Science). The program will fill a critical need for skilled research practitioners who understand and contribute at all levels in the translation of science to medicine, from bench to bedside.

The objective of the COMPASS Training Program is to prepare a diverse group of undergraduate students for careers in regenerative medicine through the creation of novel recruitment and support mechanisms that identify and foster untapped talent within populations that are historically under-represented in the biomedical sciences. It will combine hands-on research with mentorship experiences to enhance transition of students to successful careers. A parallel objective is to foster greater awareness and appreciation of diversity, equity and inclusion in trainees, mentors, and other program participants

The CIRM Board approved investing $58.22 million for up to 20 applications for a five-year duration.

“This new program highlights our growing commitment to creating a diverse workforce, one that taps into communities that have been historically under-represented in the biomedical sciences,” says Dr. Maria T. Millan, President and CEO of CIRM. “The COVID19 pandemic made it clear that the benefits of scientific discovery are not always accessible to communities that most need them. CIRM is committed to tackling these challenges by creating a diverse and dedicated workforce that can meet the technical demands of taking novel treatment ideas and making them a reality.”

The Board also approved a new $80 million concept plan to expand the CIRM Alpha Stem Cell Clinic Network. The Network clinics are all in top California medical centers that have the experience and the expertise to deliver high-quality FDA-authorized stem cell clinical trials to patients.

There are currently five Alpha Clinics – UC San Diego; UCLA/UC Irvine; City of Hope; UCSF; UC Davis – and since 2015 they have hosted more than 105 clinical trials, enrolled more than 750 patients in these trials, and generated more than $95 million in industry contracts. 

Each award will provide up to $8 million in funding over a five-year period. The clinics will have to include:

  • A demonstrated ability to offer stem cell and gene therapies to patients as part of a clinical trial.
  • Programs to help support the career development of doctors, nurses, researchers or other medical professionals essential for regenerative medicine clinical trials.
  • A commitment to data sharing and meeting CIRM’s requirements addressing issues of diversity, equity and inclusion and meeting the needs of California’s diverse patient population.

Wit, wisdom and a glimpse into the future

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As of this moment, there are over two million podcasts and over 48 million episodes to listen to on your favorite listening device. If you’re a true crime enthusiast like me, you’ve surely heard of Casefile or one of the other 94 podcasts on the topic. But what if you’re looking for something a little less ghastly and a little more uplifting?

Dr. Daylon James, co-host of The Stem Cell Podcast

The Stem Cell Podcast is an informative and entertaining resource for scientists and science enthusiasts (or really, anyone) interested in learning about the latest developments in stem cell research.

Dr. Arun Sharma, co-host of The Stem Cell Podcast

On their latest episode, dynamic co-hosts and research scientists Dr. Daylon James and Dr. Arun Sharma sit down with our President & CEO, Dr. Maria Millan, to discuss the impact of California’s culture of innovation on CIRM, the challenge of balancing hope vs. hype in the context of stem cell research/therapies, and the evolution of the agency over the past 15 years.

Listen on as Dr. Millan highlights some of CIRM’s greatest victories and shares our mission for the future.

Building a better brain (model) in the lab

Leica Picture of a brain organoid: courtesy National Institute of Allergy and Infectious Diseases, NIH

One of the biggest problems with trying to understand what is happening in a disease that affects the brain is that it’s really difficult to see what is going on inside someone’s head. People tend to object to you trying to open their noggin while they are still using it.

New technologies can help, devices such as MRI’s – which chart activity and function by measuring blood flow – or brain scans using electroencephalograms (EEGs), which measure activity by tracking electrical signaling and brain waves. But these are still limited in what they can tell us.

Enter brain organoids. These are three dimensional models made from clusters of human stem cells grown in the lab. They aren’t “brains in a dish” – they can’t function or think independently – but they can help us develop a deeper understanding of how the brain works and even why it doesn’t always work as well as we’d like.

Now researchers at UCLA’s Broad Center of Regenerative Medicine have created brain organoids that demonstrate brain wave activity similar to that found in humans, and even brain waves found in particular neurological disease.

The team – with CIRM funding – took skin tissue from healthy individuals and, using the iPSC method – which enables you to turn these cells into any other kind of cell in the body – they created brain organoids. They then studied both the physical structure of the organoids by examining them under a microscope, and how they were functioning by using a probe to measure brain wave activity.

In a news release Dr. Ranmal Samarasinghe, the first author of the study in the journal Nature Neuroscience, says they wanted to do this double test for a very good reason: “With many neurological diseases, you can have terrible symptoms but the brain physically looks fine. So, to be able to seek answers to questions about these diseases, it’s very important that with organoids we can model not just the structure of the brain but the function as well.”

Next, they took skin cells from people with a condition called Rhett syndrome. This is a rare genetic disorder that affects mostly girls and strikes in the first 18 months of life, having a severe impact on the individual’s ability to speak, walk, eat or even breathe easily. When the researchers created brain organoids with these cells the structure of the organoids looked similar to the non-Rhett syndrome ones, but the brain wave activity was very different. The Rhett syndrome organoids showed very erratic, disorganized brain waves.

When the team tested an experimental medication called Pifithrin-alpha on the Rhett organoids, the brain waves became less erratic and more like the brain waves from the normal organoids.

“This is one of the first tangible examples of drug testing in action in a brain organoid,” said Samarasinghe. “We hope it serves as a stepping stone toward a better understanding of human brain biology and brain disease.”

A conversation with Bob Klein about the past, present and future of CIRM

Bob Klein

Anyone who knows anything about CIRM knows about Bob Klein. He’s the main author and driving force behind both Proposition 71 and Proposition 14, the voter-approved ballot initiatives that first created and then refunded CIRM. It’s safe to say that without Bob there’d be no CIRM.

Recently we had the great good fortune to sit down with Bob to chat about the challenges of getting a proposition on the ballot in a time of pandemic and electoral pandemonium, what he thinks CIRM’s biggest achievements are (so far) and what his future plans are.

You can hear that conversation in the latest episode of our podcast, “Talking ’bout (re) Generation”.

Enjoy.

Board Funds Fifteen Bridges to Stem Cell Research and Therapy Programs Across California and New Sickle Cell Disease Trial

Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to the University of California, San Francisco (UCSF) to fund a clinical trial for sickle cell disease (SCD).  An additional $51.08 million was awarded to fifteen community colleges and universities across California to fund undergraduate and master’s level programs that will help train the next generation of stem cell researchers. 

SCD is an inherited blood disorder caused by a single gene mutation that changes a single base in the B globin gene leading to the production of defective hemoglobin that polymerizes and damages red blood cells thus the “sickle” shaped red blood cells.  The damaged cells cause blood vessels to occlude/close up and that can lead to multiple organ damage as well as reduced quality of life and life expectancy. 

Mark Walters, M.D., and his team at UCSF Benioff Children’s Hospital Oakland will be conducting a clinical trial that uses CRISPR-Cas9 gene editing technology to correct the genetic mutation in the blood stem cells of patients with severe SCD.  The corrected blood stem cells will then be reintroduced back into patients with the goal of correcting the defective hemoglobin and thus producing functional, normal shaped red blood cells.

This clinical trial will be eligible for co-funding under the landmark agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI) of the NIH.  The CIRM-NHLBI agreement is intended to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” initiative.  The goal is to markedly accelerate the development of cell and gene therapies for SCD. CIRM has previously funded the preclinical development of this therapy through a Translational award as well as its IND-enabling studies through a Late Stage Preclinical award in partnership with NHLBI.

The CIRM Bridges to Stem Cell Research and Therapy program provides undergraduate and master’s students with the opportunity to take stem cell related courses and receive hands on experience and training in a stem cell research related laboratory at a university or biotechnology company.  Fifteen institutions received a total of $51.08 million to carry out these programs to train the next generation of scientists.

The awards are summarized in the table below.

ApplicationTitleInstitutionAward Amount
  EDUC2-12607Bridges to Stem Cell Research and Therapy at Pasadena City College  Pasadena City College$3,605,500
  EDUC2-12611CIRM Bridges to Stem Cell Research and Therapy Training Grant  CSU San Marcos$3,606,500
  EDUC2-12617Bridges to Stem Cell Research Internship Program  San Diego State University$3,606,500
EDUC2-12620CIRM Bridges 3.0  Humboldt State$3,605,495
  EDUC2-12638CIRM Regenerative Medicine and Stem Cell Research Biotechnology Training Program  CSU Long Beach$3,276,500
    EDUC2-12677Stem Cell Internships in Laboratory-based Learning (SCILL) continue to expand the scientific workforce for stem cells research and therapies.  San Jose State University$3,606,500
  EDUC2-12691Strengthening the Pipeline of Master’s-level Scientific and Laboratory Personnel in Stem Cell Research  CSU Sacramento$2,946,500
EDUC2-12693CIRM Bridges Science Master’s Program  San Francisco State University$3,606,500
      EDUC2-12695CIRM Graduate Student Training in Stem Cell Sciences in the Stem Cell Technology and Lab Management Emphasis of the MS Biotechnology Program  CSU Channel Islands$3,606,500
  EDUC2-12718CSUN CIRM Bridges 3.0 Stem Cell Research & Therapy Training Program  CSU Northridge$3,606,500
      EDUC2-12720Stem Cell Scholars: a workforce development pipeline, educating, training and engaging students from basic research to clinical translation.  CSU San Bernardino$3,606,500
  EDUC2-12726Training Master’s Students to Advance the Regenerative Medicine Field  Cal Poly San Luis Obispo$3,276,500
  EDUC2-12730Building Career Pathways into Stem Cell Research and Therapy Development  City College of San Francisco$2,706,200
      EDUC2-12734Bridges to Stem Cell Research and Therapy: A Talent Development Program for Training Diverse Undergraduates for Careers in Regenerative Medicine  CSU Fullerton$3,606,500
  EDUC2-12738CIRM Bridges to Stem Cell Research and Therapy  Berkeley City College  $2,806,896

“We are pleased to fund a promising trial for sickle cell disease that uses the Nobel Prize winning gene editing technology CRISPR-Cas9,” says Maria T. Millan, M.D., President and CEO of CIRM.  “This clinical trial is a testament to how the CIRM model supports promising early-stage research, accelerates it through translational development, and advances it into the clinics. As the field advances, we must also meet the demand for promising young scientists.  The CIRM Bridges programs across the state of California will provide students with the tools and resources to begin their careers in regenerative medicine.”

Welcoming back old friends and some new ones

When Proposition 14 was approved by voters in November we were given a chance to carry on the work we have been doing for more than 16 years. What we hadn’t anticipated was that we would also get a chance to do that with some of the team that helped us make CIRM what it is, but who had since moved on to other jobs.

We are delighted to say that as we build up our team again we are welcoming back a couple of dear friends, and welcoming in some new ones too. They’re a talented bunch and, if they don’t mind me saying so, a darned good looking group too.

Rosa Canet-Aviles, PhD., has been named as the new Vice President Scientific Programs. Rosa is a familiar face at the agency, serving as a Science Officer with CIRM from 2008 to 2014. During that time she helped oversee the development of our Translational program, managed a broad portfolio of projects and organized workshops on Parkinson’s and autism.

After leaving CIRM she joined the Foundation for the National Institutes of Health (FINH) where she served as the Director of Neuroscience Research Partnerships. In that role she led the successful development and management of 5 new large partnerships including the Biomarkers Consortium Neuroscience Steering Committee, the Accelerating Medicines Partnership (AMP) for Alzheimer’s disease 1.0 and 2.0, AMP Parkinson’s disease and AMP Schizophrenia.

Rosa has more than 15 years of experience working in industry, academia and government and her experience in developing and managing neuroscience programs will be invaluable as CIRM looks to invest some $1.5 billion in neuroscience under Proposition 14.

“I am very excited to be back,” says Rosa. ”It is a dream come true being able to translate all the skills, learning and networks gathered over the past 7 years towards the development and implementation of CIRM’s new phase and accelerate stem cell therapies for patients in need.” 

“We are thrilled to announce the timely return of Rosa to CIRM as we build our new strategic plan under Prop 14,” says Dr. Maria T. Millan, CIRM’s President & CEO. “Rosa has demonstrated time and again the unique ability to bring together often seemingly disparate stakeholders to successfully drive toward a common goal of advancing the science on behalf of patients with diseases of the brain and neuropsychiatric disorders. At CIRM, she assembled key international leaders who went on to form an international Parkinson’s Disease consortium. At the Foundation for NIH (FNIH), she directed the development of five prominent public-private partnerships. A neuroscientist by training, she is held in high regard and has been called a “quick study” in her ability to lead in new areas such as in genomics and data science, key components of her role at FNIH and at Eisai’s Center for Genetics Guided Dementia Discovery.“

In addition, CIRM is pleased to announce the following new team members:

Uta Grieshammer, PhD. is also returning to CIRM as the Senior Science Officer for our Discovery program. Uta was at CIRM from 2007 to 2015 and led the programs that created both our Genomics Initiative and our iPSC bank. She also organized several scientific conferences and workshops involving hundreds of CIRM-funded researchers.

After leaving CIRM she became the Scientific Director of the California Initiative to Advance Precision Medicine at the University of California San Francisco where she created and managed the application and peer review process. Most recently she was the Program Officer at the University of California Office of the President’s (UCOP) Tobacco Related Disease Research Program where she focused on the neuroscience of nicotine addiction. She also helped develop a scholarship program to attract students from diverse backgrounds to pursue a career in science. 

Michael Bunch joins CIRM as a Business Service Officer. Michael is a decorated veteran who has been working as the Chief Business Officer at the Veterans Home in Yountville, California. In that role he implemented new contract and reviewing processes and oversaw the income and insurance tracking for some 1,000 residents. With his extensive background in acquisition management, contingency contracting, and his deep knowledge of state regulations and guidelines Michael was able to increase funding, streamline processes and assist Veterans and their families to obtain the benefits and services that they qualified for.

Michael spent 25 years in the US Army including serving as part of the NATO peacekeeping force in Kosovo. During that deployment he was awarded the Joint Service Commendation Medal (JSCM) for managing the fuel needs of 4 Multinational Task Forces and 33 Nations, an essential element in helping the mission succeed.

A Senior Drill Sergeant, Infantry Instructor and Financial and Resource Manager Michael has been awarded the Army Commendation Medal with 4 Oak Leaves, Army Achievement Medal with 4 Oak Leaves, Global War on Terrorism Service Medal, KOSOVO Campaign Medal, Military Outstanding Volunteer Service Medal, NATO Medal, Expert Infantryman Badge, Honorary Kentucky Colonel and Honorary Kentucky Admiral.

Nellie Almazan joins CIRM as a Grants Management Specialist. Nellie comes to us from the California Department of Transportation (Caltrans) where she has worked for 16 years, most recently as the Associate Transportation Planner with the Low Carbon Transit Operations program. Nellie managed more than 150 projects, reviewing grants to help reduce greenhouse gas emissions in the state and overseeing programs that had an emphasis on serving Disadvantaged Communities.

She is currently enrolled at Sacramento City College where her focus is on Sociology and Deaf Culture.

Alexandra Caraballo joins CIRM as a Grants Management Specialist. Alex has more than 15 years of grant administration experience with a focus on incorporating equity, diversity, and inclusion into grantmaking practices and decision-making. She comes to CIRM from the Kaiser Foundation Health Plan where she was the National Manager of Philanthropy. There she was responsible for the administration of approximately 200 grants in the national community health portfolio. Before Kaiser she was the Program Assistant and Associate Program Officer at the East Bay Community Foundation, where she partnered with donors and community-based organizations to advance racial equity and transform political, social and economic outcomes for East Bay Communities.

Alex currently serves on the Board of Directors for the Lindsay Wildlife Experience and was a former Advisory Board member for Oakland Head Start.

Call for a worldwide approach to regulating predatory stem cell clinics

You can’t fix a global problem at the local level. That’s the gist of a new perspective piece in the journal Stem Cell Reports that calls for a global approach to rogue stem cell clinics that offer bogus therapies.

The authors of the article are calling on the World Health Organization (WHO) to set up an advisory committee to draw up rules and regulations to help guide countries trying to shut these clinics down.

In a news release, senior author Mohamed Abou-el-Enein, the executive director of the joint University of Southern California/Children’s Hospital of Los Angeles Cell Therapy Program, says these clinics are trying to cash in on the promise of regenerative medicine.

“Starting in the early 2000s… unregulated stem cell clinics offering untested and poorly characterized treatments with insufficient information on their safety and efficacy began emerging all over the world, taking advantage of the media hype around stem cells and patients’ hope and desperation.”

Dr. Larry Goldstein

The authors include Lawrence Goldstein, PhD, a CIRM Board member and a Science Policy Fellows for the International Society for Stem Cell Research (ISSCR).

Zubin Master, an associate professor of biomedical ethics at the Mayo Clinic, says the clinics prey on vulnerable people who have serious medical conditions and who have often tried conventional medical approaches without success.

“We should aim to develop pathways to provide patients with evidenced-based experimental regenerative intervention as possible options where there is oversight, especially in circumstances where there is no suitable alternative left.”

The report says: “The unproven SCI (stem cell intervention) industry threatens the advancement of regenerative medicine. Reports of adverse events from unproven SCIs has the potential to affect funding and clinical trial recruitment, as well as increasing burdens among regulatory agencies to oversee the industry.

Permitting unregulated SCIs to flourish demonstrates a lack of concern over patient welfare and undermines the need for scientific evidence for medicinal product R&D. While some regulatory agencies have limited oversight or enforcement powers, or choose not to use them, unproven SCI clinics still serve to undermine authority given to regulatory agencies and may reduce public trust impacting the development of safe and effective therapies. Addressing the continued proliferation of clinics offering unproven SCIs is a problem worth addressing now.”

The authors say the WHO is uniquely positioned to help create a framework for the field that can help address these issues. They recommend setting up an advisory committee to develop global standards for regulations governing these clinics that could be applied in all countries. They also say we need more educational materials to let physicians as well as patients understand the health risks posed by bogus clinics.

This article comes out in the same week that reports by the Pew Charitable Trust and the FDA also called for greater regulation of these predatory clinics (we blogged about that here). Clearly there is growing recognition both in the US and worldwide that these clinics pose a threat not just to the health and safety of patients, but also to the reputation of the field of regenerative medicine as a whole.

“I believe that the global spread of unproven stem cell therapies reflects critical gaps in the international system for responding to health crises, which could put the life of thousands of patients in danger,” Abou-el-Enein says. “Urgent measures are needed to enhance the global regulatory capacity to detect and respond to this eminent crisis rapidly.”