Can stem cells help people recovering from a stroke? You asked, and the experts answered

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We recently held our first ever Facebook Live event. It was focused on the use of stem cells and recovery from a stroke and featured three great guests: Dr. Gary Steinberg, chief of Neurosurgery at Stanford, Sonia Coontz, a patient of Dr. Steinberg’s, and CIRM’s own Science Officer Dr. Lila Collins.

We had an amazing response from people during the event and in the days since then with some 6,750 people watching the video and almost 1,000 people reacting by posting a comment or sharing it with friends. It was one of the most successful things we have ever done on Facebook so it’s not surprising that we plan on doing many more Facebook Live ‘Ask the Expert’ events in the future. We will post more details of that as we finalize them.

We tried to cover as many topics as possible during the hour but there were simply too many questions for us to get to all of them. So here is a recap of the key issues we covered, and a few we didn’t have a chance to answer.

Let’s start with Dr. Steinberg’s explanation of the research that led to his current clinical trial:

Dr. Steinberg: “I got interested in this about 18 years ago when I took human cells and transplanted them into rodent models of stroke. What we found was that when we transplanted those cells into the stroke region, the core of the stroke, they didn’t survive very well but when we moved them a few millimeters away from the stroke they not only survived but they migrated to the stroke.

The reason they migrate is that the stem cells have receptors on them that interact with chemicals given off by the stroke environment and that’s why they migrate to the stroke site. And when they get to the site they can turn into different kinds of cells. Very importantly we found these mice and rats that had behavioral problems – walking, moving – as a result of the stroke, we found we could improve their neurological outcomes with the stem cells.

With the help of CIRM, which has been very generous, we were fortunate enough to receive about $24 million in funding over the last 8 years, from 2010, to move this therapy into the clinic to understand the basic mechanisms of the recovery and to start clinical trials

One of the surprising things was that our initial notion was that the cells we transplanted into the brains would initially turn into the cells in the brain affected by the stroke and reconstitute those circuits. We were shocked to find that that was not what was happening, that only a few of the transplanted cells turned into neurons. The way they were recovering function was by secreting very powerful growth factors and molecules and proteins that enhanced native recovery or the ability of the normal brain to recover itself. Some of these processes included outgrowth of neurons, new connections, new synapses, not from the stem cells but from the native cells already in the brain.

This is not cell replacement but enhancing native recovery and, in a simple sense, what the cells are doing, we believe, is to change the adult brain, which has a hard time recovering from a stroke, into an infant brain and infants recover very well after a stroke.”

All this work was focused on ischemic strokes, where a blockage cuts off blood flow to the brain. But people like Cheryl Ward wanted to know: “Will this work for hemorrhagic stroke?” That’s where a blood vessel in the brain leaks or ruptures.

Dr. Steinberg: “I suspect we will be generalizing this therapy into hemorrhagic patients very, very soon and there’s no reason why it shouldn’t work there. The reason we didn’t start there is that 85% of strokes are ischemic and only 15% are hemorrhagic so it’s a smaller population but a very, very important population because when patients have a hemorrhage from a stroke they are often more seriously disabled than from ischemic.”

Dr. Lila Collins: “I would like to highlight one trial for hemorrhagic stroke with the Mayo Clinic and that’s using mesenchymal stem cells (normally found in bone marrow or blood). It’s an early stage, Phase 1 safety study in patients with recent cerebral hemorrhage.  They are looking at improvements in neurological function and patients have to be treated within 72 hours after the stroke.”

Dr. Steinberg explained that because it’s more difficult to enroll patients within 72 hours of a stroke that we may end up offering a combination of therapies spread out over months or even years.

Dr. Steinberg: “It may be that and we may figure this out in the next 5 to 10 years, that you might want to treat patients acutely (right away) with an intravenous therapy in the first 72 hours and then you might want to come in again sub-acutely within a few months, injecting the cells into the brain near the stroke, and then maybe come in chronically a few years later if there are still problems and place the cells directly in the brain. So, lots of ways to think about how to use this in the future.”

James Russell suffered a stroke in 2014 and wrote:

“My left side was affected. My vision was also impacted. Are any stroke patients being given stem cells seeing possible improvement in visual neglect?”

Dr. Steinberg: “We don’t know the answer to that yet, it’s quite possible. It’s true these vision circuits are not dead and could be resurrected. We have not targeted visual pathways in our work, we have targeted motor functions, but I would also be optimistic that we could target patients who have vision problems from stroke. It’s a very important area.

A number of people wondered if stem cells can help people recovering from a stroke can they also help people with other neurological conditions.

Hanifa Gaphoor asked “What about Parkinson’s disease?” and Ginnievive Patch wondered “Do you feel hopeful for neurological illnesses like Huntington’s disease and ALS? Dr. Steinberg was cautiously optimistic.

Dr. Steinberg: “We’ve extended this kind of treatment not just for ischemic stroke but into traumatic brain injury (TBI) and we just completed a trial for patients with chronic TBI or who have suffered a trauma to the brain. Many other indications may be possible. In fact, now that we know these circuits are not dead or irreversibly injured, we believe we could even extend this to neurodegenerative diseases like ALS, Parkinson’s, maybe even to Alzheimer’s disease in the future. So, lots of hope but we don’t want to oversell this, and we want to make sure this is done in a rigorous fashion.”

Several people had questions about using their own adipose, or fat stem cells, in therapies being offered at clinics around the US and in other countries. Cheri Hicks asked: “I’m curious if adipose stem cell being used at clinics at various places is helpful or beneficial?”

Dr. Steinberg: “I get emails or calls from patients every week saying should I go to Russia, India or Mexico and get stem cell transplants which are done not as part of a rigorous trial and I discourage patients from getting stem cells that are not being given in a controlled fashion. For one thing, patients have been getting hurt by these treatments in these clinics; they have developed tumors and infections and other problems. In many cases we don’t even know what the cells are, there’s not published information and the patients pay cash for this, of course.”

At CIRM we also worry about people going to clinics, in the US and in other countries, where they are getting therapies that have not been approved by the US Food and Drug Administration (FDA) or other appropriate regulatory bodies. That’s why we have created this page on our website to help people who want a stem cell therapy but don’t know what to look for in a clinical trial or what questions to ask to make sure it’s a legitimate trial, one that’s been given the go-ahead by the FDA.

Bret Ryan asked: “What becomes of the implanted cells?”

Dr. Steinberg: We found after transplanting the cells, one week after the transplant, we see a new abnormality in the premotor cortex, the area of the brain that controls motor function. We saw a new abnormality there or a new signal that disappears after a month and never comes back. But the size of that temporary abnormality after one week correlates very closely with the degree of recovery after six months, one year and two years.

One of the interesting things is that it doesn’t seem to be necessary for the cells to survive long term to have beneficial effects. The cells we used in the SanBio trial don’t survive more than a month and yet they seem to aid recovery function in our pilot studies which is sustained for years.”

And of course, many people, such as Karen Smart, wanted to know how they could get the therapy. Right now, the clinical trial is fully enrolled but Stanford is putting together a waiting list for future trials. If you are interested and would like more information, please email: stemcellstudy@stanford.edu.

Sonia Coontz, the patient who was also a key part of the Facebook Live event, has an amazing story to tell. She was left devastated, physically and emotionally, after having a stroke. But then she heard about Dr. Steinberg’s clinical trial and it changed her life. Here’s her story.

We were thrilled to receive all of your comments and questions during our first Facebook Live event. It’s this kind of dialogue between scientists, patients and the public that will be critical for the continued support of our mission to accelerate stem cell treatments to patients with unmet medical needs.

Due to the response, we plan to regularly schedule these “Ask the Expert” events. What disease area would you like us to focus on next time? Leave us a comment or email info@cirm.ca.gov

 

Stem Cell Roundup: Jake Javier’s amazing spirit; TV report highlights clinic offering unproven stem cell therapies

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Jake Javier: Photo Michael Clemens, Sees the Day

In the Roundup we usually focus on studies that highlight advances in stem cell research but today we’re going to do something a little different. Instead of relying on print for our stories, we’re turning to video.

We begin with a piece about Jake Javier. Regular readers of our blog will remember that Jake is the young man who broke his neck the day before he graduated high school, leaving him paralyzed from the upper chest down.

After enrolling in the CIRM-funded Asterias clinical trial, and receiving a transplant of 10 million stem cells, Jake regained enough use of his arms and hands to be able to go to Cal Poly and start his life over.

This video highlights the struggles and challenges he faced in his first year, and his extraordinary spirit in overcoming them.

(thanks to Matt Yoon and his Creative Services team at Cal Poly for this video)

Going Undercover

The second video is from the NBC7 TV station in San Diego and highlights one of the big problems in regenerative medicine today, clinics offering unproven therapies. The investigative team at NBC7 went undercover at a stem cell clinic seminar where presenters talked about “the most significant breakthrough in natural medicine” for improving mobility and reducing pain. As the reporter discovered, the reality didn’t live up to the promise.

NBC7 Investigative Report

 

Using biological “codes” to generate neurons in a dish

BrainWavesInvestigators at the Scripps Research Institute are making brain waves in the field of neuroscience. Until now, neuroscience research has largely relied on a variety of animal models to understand the complexities of various brain or neuronal diseases. While beneficial for many reasons, animal models do not always allow scientists to understand the precise mechanism of neuronal dysfunction, and studies done in animals can often be difficult to translate to humans. The work done by Kristin Baldwin’s group, however, is revolutionizing this field by trying to re-create this complexity in a dish.

One of the primary hurdles that scientists have had to overcome in studying neuronal diseases, is the impressive diversity of neuronal cell types that exist. The exact number of neuronal subtypes is unknown, but scientists estimate the number to be in the hundreds.

While neurons have many similarities, such as the ability to receive and send information via chemical cues, they are also distinctly specialized. For example, some neurons are involved in sensing the external environment, whereas others may be involved in helping our muscles move. Effective medical treatment for neuronal diseases is contingent on scientists being able to understand how and why specific neuronal subtypes do not function properly.

In a study in the journal Nature, partially funded by CIRM, the scientists used pairs of transcription factors (proteins that affect gene expression and cell identity), to turn skin stem cells into neurons. These cells both physically looked like neurons and exhibited characteristic neuronal properties, such as action potential generation (the ability to conduct electrical impulses). Surprisingly, the team also found that they were able to generate neurons that had unique and specialized features based on the transcription factors pairs used.

The ability to create neuronal diversity using this method indicates that this protocol could be used to recapitulate neuronal diversity outside of the body. In a press release, Dr. Baldwin states:

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Kristin Baldwin, PhD

“Now we can be better genome detectives. Building up a database of these codes [transcription factors] and the types of neurons they produce can help us directly link genomic studies of human brain disease to a molecular understanding of what goes wrong with neurons, which is the key to finding and targeting treatments.”

These findings provide an exciting and promising tool to more effectively study the complexities of neuronal disease. The investigators of this study have made their results available on a free platform called BioGPS in the hopes that multiple labs will delve into the wealth of information they have opened up. Hopefully, this system will lead to more rapid drug discovery for disease like autism and Alzheimer’s

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.

 

 

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

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Manufacturing stem cells: Photo courtesy of Pluristem

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

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

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

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

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

WHAT

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

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

The agenda of the conference will cover four main topics:

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

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

WHO

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

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

WHY

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

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

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

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

When and Where

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

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

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Mark Noble. Photo by Todd Dubnicoff

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

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

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

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

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

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

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

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

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

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

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

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

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

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

 

How Tom Howing turned to stem cells to battle back against a deadly cancer

As we enter the new year, CIRM’s 2017 Annual Report will be posted in less than two weeks!  Here’s one of the people we are profiling in the report, a patient who took part in a CIRM-funded clinical trial.

Tom Howing

In March of 2015, Tom Howing was diagnosed with stage 4 cancer. Over the next 18 months, he underwent two rounds of surgery and chemotherapy. Each time the treatments held the cancer at bay for a while. But each time the cancer returned. Tom was running out of options and hope when he heard about a CIRM-funded clinical trial using a new approach.

The clinical trial uses a therapy that blocks a protein called CD47 that is found on the surface of cancer cells, including cancer stem cells which can evade traditional therapies. CD47 acts as a ‘don’t eat me’ signal that tells immune cells not to kill off the cancer cells. When this ‘don’t eat me’ signal is blocked by the antibody, the patient’s immune system is able to identify, target and kill the cancer stem cells.

“When I was diagnosed with cancer I knew I had battle ahead of me. After the cancer came back again they recommended I try this CD47 clinical trial. I said absolutely, let’s give it a spin.

“I guess one is always a bit concerned whenever you put the adjective “experimental” in front of anything. But I’ve always been a very optimistic and positive person and have great trust and faith in my caregivers.

“Whenever you are dealing with a Phase 1 clinical trial (the earliest stage where the goal is first to make sure it is safe), there are lots of unknowns.  Scans and blood tests came back showing that the cancer appears to be held in check. My energy level is fantastic. The treatment that I had is so much less aggressive than chemo, my quality of life is just outstanding.”

Tom says he feels fortunate to be part of the clinical trial because it is helping advance research, and could ultimately help many others like him.

“The most important thing I would say is, I want people to know there is always hope and to stay positive.”

He says he feels grateful to the people of California who created CIRM and the funding behind this project: “I say a very heartfelt thank you, that this was a good investment and a good use of public funds.”

He also wants the researchers, who spent many years developing this approach, to know that they are making a difference.

“To all those people who are putting in all the hours at the bench and microscope, it’s important for them to know that they are making a huge impact on the lives of real people and they should celebrate it and revel in it and take great pride in it.”

Budgeting for the future of the stem cell agency

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The CIRM Board discusses the future of the Stem Cell Agency

Budgets are very rarely exciting things; but they are important. For example, it’s useful for a family to know when they go shopping exactly how much money they have so they know how much they can afford to spend. Stem cell agencies face the same constraints; you can’t spend more than you have. Last week the CIRM Board looked at what we have in the bank, and set us on a course to be able to do as many of the things we want to, with the money we have left.

First some context. Last year CIRM spent a shade over $306 million on a wide range of research from Discovery, the earliest stage, through Translational and into Clinical trials. We estimate that is going to leave us with approximately $335 million to spend in the coming years.

A couple of years ago our Board approved a 5 year Strategic Plan that laid out some pretty ambitious goals for us to achieve – such as funding 50 new clinical trials. At the time, that many clinical trials definitely felt like a stretch and we questioned if it would be possible. We’re proving that it is. In just two years we have funded 26 new clinical trials, so we are halfway to our goal, which is terrific. But it also means we are in danger of using up all our money faster than anticipated, and not having the time to meet all our goals.

Doing the math

So, for the last couple of months our Leadership Team has been crunching the numbers and looking for ways to use the money in the most effective and efficient way. Last week they presented their plan to the Board.

It boiled down to a few options.

  • Keep funding at the current rate and run out of money by 2019
  • Limit funding just to clinical trials, which would mean we could hit our 50 clinical trial goal by 2020 but would not have enough to fund Discovery and Translational level research
  • Place caps on how much we fund each clinical trial, enabling us to fund more clinical trials while having enough left over for Discovery and Translational awards

The Board went for the third option for some good reasons. The plan is consistent with the goals laid out in our Strategic Plan and it supports Discovery and Translational research, which are important elements in our drive to develop new therapies for patients.

Finding the right size cap

Here’s a look at the size of the caps on clinical trial funding. You’ll see that in the case of late stage pre-clinical work and Phase 1 clinical trials, the caps are still larger than the average amount we funded those stages last year. For Phase 2 the cap is almost the same as the average. For Phase 3 the cap is half the amount from last year, but we think at this stage Phase 3 trials should be better able to attract funding from other sources, such as industry or private investors.

cap awards

Another important reason why the Board chose option three – and here you’ll have to forgive me for being rather selfish – is that it means the Administration Budget (which pays the salaries of the CIRM team, including yours truly) will be enough to cover the cost of running this research plan until 2020.

The bottom line is that for 2018 we’ll be able to spend $130 million on clinical stage research, $30 million for Translational stage, and $10 million for Discovery. The impact the new funding caps will have on clinical stage projects is likely to be small (you can see the whole presentation and details of our plan here) but the freedom it gives us to support the broad range of our work is huge.

And here is where to go if you are interested in seeing the different funding opportunities at CIRM.

A funny thing happened on my way to a PhD: one scientists change of mind and change of direction

Laurel Barchas is an old and dear friend of the communications team here at CIRM. As a student at U.C. Berkeley she helped us draft our education portal – putting together a comprehensive curriculum to help high schools teach students about stem cells in a way that met all state and federal standards. But a funny thing happened on her way to her Ph.D., she realized she had changed her mind about research, and so she changed her career direction.  

Laurel recently wrote this blog about that experience for the new and improved website of the Student Society for Stem Cell Research (SSSCR) –

Laurel #1

Laurel Barchas at the World Stem Cell Summit 2013

Stem cell parental advice—you can grow up to be anything!

I was one of those students who, since high school, knew I was destined for the lab. Throughout some of high school, and all of college and graduate school, I had internships or positions in amazing labs that warmly took me in and trained me how to be a scientist. I loved designing and carrying out experiments on my stem cells, presenting at lab meetings, writing theses, and teaching others about my work through undergraduate lectures and high school presentations. My participation in the Student Society for Stem Cell Research hugely supported all of my efforts; it even enabled me to get one of my first jobs as a contract curriculum writer (a project manager role) with the California Institute for Regenerative Medicine, which launched my writing career.

Four years into my biology PhD program, things became clear that I didn’t want to do research anymore. I couldn’t handle the failure inherent in doing research. I wasn’t able to put in the time and focus necessary to do big experiments—then repeat them over and over. Although I loved science, I wasn’t meant to be a career scientist like many of my colleagues. I was a science communicator. Realizing this, and taking into account my personal struggles, my advisers and I decided the best thing was to get a terminal master’s degree.**

Differentiation—finding the right path

I struggled for a while finding a job that suited me. I worked as an education consultant, writing materials directed at teachers and students. I worked as a marketing, communications and operations assistant for a real estate group. I looked for jobs as a teacher, curriculum developer, and science education program coordinator, but none felt quite right for me. Although I had extensive experience in school developing materials for teachers and giving presentations to students, and I knew education could be a rewarding career path, I wasn’t sure I wanted to be in the academic world anymore.

Finally, I found some listings looking for technical writers. I didn’t even know what that was at the time. Various biotech companies had their feelers out for entry level writers with advanced degrees in biology or STEM fields—and a master’s degree was just fine. It turns out I was a perfect fit. Surprisingly, many people in the “tech com” (technical communications) and “mar com” (marketing communications) departments at my company had a similar experience; they didn’t want careers in research or the medical professions, so they chose communications.

Laurel #2

Life as a technical writer—feeling like a glial cell

As a technical writer at my company, I have many responsibilities beyond writing and editing user manuals, application notes, and diagrams. Tech writers are much like the oft-forgotten glial cells that “glue the brain together.” I manage each project from start to finish, and I get to work on all types of technical documentation and marketing collateral with a team of company scientists (R&D), graphic designers, marketing specialists, coders, product managers, and other writers. Often, I have major creative input on the content, design, and development of marketing campaigns. I enjoy starting with ideas—maybe a few bullet points or a rough draft—and building colorful, captivating content. It feels like solving a complex puzzle.

I’ve gotten the chance to write articles on human induced pluripotent stem cell-derived beta cells for a drug discovery publication and to create portals for our website. I’ve helped make booth panels and printed resources for conferences like the International Society for Stem Cell Research. Most importantly (to me), I’ve managed to stay within the field of stem cell research/regenerative medicine. I am the main writer for that product and service line, so I can use my expertise and experience (plus, knowledge of my audience) to present products that advance my audience’s basic, translational and clinical research.

I love my job. It pays well, has regular hours, and gives me a sense of belonging to a team. It’s fast paced, I’m working on a new thing every day, and I get to learn and write about the latest advancements from our R&D teams around the world. I could go on and on, but suffice it to say that the job fits like a glove, and I can see myself doing this long term. Also…I get to live in Silicon Valley! (Pros: great food, culture, people. Cons: cost of living, traffic.)

I hope you can get encouragement from the retelling of my experience that there is a space for you in this field. This is the first post in a series of articles about careers in regenerative medicine. I aim to take you through a tour of the vocational landscape—its ups, its downs—and am looking forward to hearing from you with any jobs/roles/scenarios you are curious about. Please comment on what you’d like to learn about next!

Remember: there are plenty of options and ways for you to apply your talent and experience to pushing our field forward. SSSCR is here to help!

*I want to thank everyone who serves in the research and medical areas. Without you our field would stop in its tracks. However, not everyone is cut out for such positions. Luckily, there are other options.

**Some reading this might say “awwwww, too bad, she was so close to that PhD” and some might say “that’s a major accomplishment and you can do a lot with that degree!” Both are right, but I choose to believe the latter, as I am so much happier now that I released myself from the allure of lab research and went into science communications. We tend to hold science and medicine up on pedestals; however, science communication facilitates almost all interactions between academic and industry scientists, clinicians, and the public. An understanding of and engagement with new science is critical to promoting a healthy democracy with citizens who can make informed decisions about their society’s future.

Laurel is a co-founder of SSSCR, the current Associate Director, and a member of the SSSCR International executive committee. She has been involved in SSSCR since 2004, when she helped start UC Berkeley’s chapter. Her main contributions are educating various communities about stem cell research and building career development opportunities for students. Along with a team of SSSCR members, Laurel created the California Institute for Regenerative Medicine’s stem cell education portal to provide teachers with the materials they need to engage students with the field. Currently, Laurel is a Senior Technical Writer focused on stem cell products and services.

FDA creates a forum for patients to guide its decision making

FDA

It’s not hard to find people who don’t like the US Food and Drug Administration (FDA), the government agency that, among other things, regulates medical therapies. In fact, if you type “do people like the FDA?” into an internet search engine you’ll quickly find out that for a lot of people the answer is “no”.

But the Agency is trying to change and deserves credit for taking seriously many of the criticisms that have been levelled at it over the years and trying to address them.

The latest example is the news that the FDA has set a date for the first-ever meeting of its first-ever Patient Engagement Advisory Committee (PEAC). On its website, the FDA says the PEAC will be focused on patient-related issues:

“The PEAC is a forum for the voice of patients. It will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into our oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.”

In the past, the FDA has created forums to allow patients to talk about the impact of a disease on their daily life and their views on treatment options. But those were considered by many to be little more than window dressing, providing a sounding boards for patients but not actually producing any tangible benefits or changes.

The FDA also has patient representatives who take part in FDA advisory committee meetings, but the PEAC is the first time it has ever had a committee that was solely focused on patients and their needs. The nine core members of the PEAC all have experience either as patients or patient advocates and care-givers for patients. A really encouraging sign.

We tip our CAP to the FDA

At CIRM we support anything that ensures that patients not only have a seat at the table, but also that their voices are heard and taken seriously. That’s why for every clinical trial we fund (and even some pre-clinical projects too) we create what we call a Clinical Advisory Panel or CAP (we do love our acronyms).

Each CAP consists of three to five members, with a minimum of one Patient Representative, one External Advisor and one CIRM Science Officer. The purpose of the CAP is to make recommendations and provide guidance and advice to the Project Team running the trial.

Having a Patient Representative on a CAP ensures the patient’s perspective is included in shaping the design of the clinical trial, making sure that the trial is being carried out in a way that has the patient at the center. Patients can ask questions or raise issues that researchers might not think about, and can help the researchers not only do a better job of recruiting the patients they need for the trial, but also keeping those patients involved. We believe a trial designed around the patient, and with the patient in mind, is much more likely to be successful.

In announcing the formation of the PEAC the FDA said:

“Patients are at the heart of what we do. It makes sense to establish an advisory committee built just for them.”

I completely agree.

My only regret is that they didn’t call it the Patient Engagement Advisory Committee for Health, because then the acronym would have been PEACH. And this is certainly a peach of an idea, one worthy of support.

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