Stem Cell Stories that Caught Our Eye: New law to protect consumers; using skin to monitor blood sugar; and a win for the good guys

Hernendez

State Senator Ed Hernandez

New law targets stem cell clinics that offer therapies not approved by the FDA

For some time now CIRM and others around California have been warning consumers about the risks involved in going to clinics that offer stem cell therapies that have not been tested in a clinical trial or approved by the U.S. Food and Drug Administration (FDA) for use in patients.

Now a new California law, authored by State Senator Ed Hernandez (D-West Covina) attempts to address that issue. It will require medical clinics whose stem cell treatments are not FDA approved, to post notices and provide handouts to patients warning them about the potential risk.

In a news release Sen. Hernandez said he hopes the new law, SB 512, will protect consumers from early-stage, unproven experimental therapies:

“There are currently over 100 medical offices in California providing non-FDA approved stem cell treatments. Patients spend thousands of dollars on these treatments, but are totally unaware of potential risks and dangerous side effects.”

Sen. Hernandez’s staffer Bao-Ngoc Nguyen crafted the bill, with help from CIRM Board Vice Chair Sen. Art Torres, Geoff Lomax and UC Davis researcher Paul Knoepfler, to ensure it targeted only clinics offering non-FDA approved therapies and not those offering FDA-sanctioned clinical trials.

For example the bill would not affect CIRM’s Alpha Stem Cell Clinic Network because all the therapies offered there have been given the green light by the FDA to work with patients.

Blood_Glucose_Testing 

Using your own skin as a blood glucose monitor

One of the many things that people with diabetes hate is the constant need to monitor their blood sugar level. Usually that involves a finger prick to get a drop of blood. It’s simple but not much fun. Attempts to develop non-invasive monitors have been tried but with limited success.

Now researchers at the University of Chicago have come up with another alternative, using the person’s own skin to measure their blood glucose level.

Xiaoyang Wu and his team accomplished this feat in mice by first creating new skin from stem cells. Then, using the gene-editing tool CRISPR, they added in a protein that sticks to sugar molecules and another protein that acts as a fluorescent marker. The hope was that the when the protein sticks to sugar in the blood it would change shape and emit fluorescence which could indicate if blood glucose levels were too high, too low, or just right.

The team then grafted the skin cells back onto the mouse. When those mice were left hungry for a while then given a big dose of sugar, the skin “sensors” reacted within 30 seconds.

The researchers say they are now exploring ways that their findings, published on the website bioRxiv, could be duplicated in people.

While they are doing that, we are supporting ViaCytes attempt to develop a device that doesn’t just monitor blood sugar levels but also delivers insulin when needed. You can read about our recent award to ViaCyte here.

Deepak

Dr. Deepak Srivastava

Stem Cell Champion, CIRM grantee, and all-round-nice guy named President of Gladstone Institutes

I don’t think it would shock anyone to know that there are a few prima donnas in the world of stem cell research. Happily, Dr. Deepak Srivastava is not one of them, which makes it such a delight to hear that he has been appointed as the next President of the Gladstone Institutes in San Francisco.

Deepak is a gifted scientist – which is why we have funded his work – a terrific communicator and a really lovely fella; straight forward and down to earth.

In a news release announcing his appointment – his term starts January 1 next year – Deepak said he is honored to succeed the current President, Sandy Williams:

“I joined Gladstone in 2005 because of its unique ability to leverage diverse basic science approaches through teams of scientists focused on achieving scientific breakthroughs for mankind’s most devastating diseases. I look forward to continue shaping this innovative approach to overcome human disease.”

We wish him great success in his new role.

 

 

 

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CIRM Board Appoints Dr. Maria Millan as President and CEO

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

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

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

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

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

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

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

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

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

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

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

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

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

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


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A trip to the OR started CIRM’s latest Board member on a career in science

The CIRM Board is pretty big, 29 members, all of whom have very different backgrounds and experiences. That’s one of its strengths, the diversity of members and the sheer range of expertise they bring to this work.

David Martin

Our newest member, Dr. David Martin, is the Chair and CEO of AvidBiotics Corp., a biotech company in South San Francisco. He has a very impressive resume including leadership roles at Genentech, DuPont Merck and Chiron. You can read more about that in our news release.

But we wanted to go beyond the obvious reasons why he was appointed by California State Treasurer John Chiang (who celebrated Dr. Martin’s “very distinguished career in both academics and the biotech industry”) and find a little bit more about him as a person.

We began by asking him how he got interested in science:

“When I was in junior high school, my father, a pediatrician, managed for me to witness at close-hand several surgical procedures in the O.R. When I was in high school my biology teachers were disasters, but I really enjoyed math and physics so I went to an engineering school.  After a year I rejected carrying a 14-inch slide rule on my belt like the other geeks and switched my major to biology. The biology lab excited me, and I changed my courses to prepare for medical school.  There I took off a year for a research training program and a real research lab experience.  I was hooked.”

What have been some of the biggest influences in your career?

Jim Wyngaarden’s research training program (supported by the National Science Foundation – as a precursor to the National Institute of Health’s  Medical Scientist Training Program) and working in Jim’s lab at Duke.  I then had nearly a decade of direct exposure to Gordon Tomkins, first when I was as a post-doc at NIH and then as a faculty member at UCSF.  Third was my many years exposure to Bob Swanson at Genentech.  Each was a remarkable and quite unique mentor.”

You have been a part of some of the biggest players in drug research and development – Genentech, DuPont Merck, Chiron – what are the biggest advances you have seen over the years?

“The discovery, early development, and nearly explosive expansion of recombinant DNA technologies and of their broad applications in the life sciences. Today one can already see on the near horizon a similar, very rapid expansion of stem cell applications to regenerative medicine, and it will not be limited to regenerative medicine.”

Dr. Martin says he feels privileged and enthused to be joining the CIRM Board and hopes his experience will be valuable to the agency:

“Fortuitously, I’ve been in the right place at the right time more than once as a physician-scientist—in both academe and industry; hopefully those experiences and perspectives may be of benefit to CIRM.”

Like many people fortunate enough to live in the San Francisco Bay Area he likes to get out of the lab/office as much as possible to enjoy all that the region has to offer:

“I enjoy bicycling, hiking and fly fishing—when I can find the time.”

We are delighted to welcome Dr. Martin to the CIRM team.

Confusing cancer to kill it

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

 

 

 

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

ALS

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

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

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

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

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

BrainStorm

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

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

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

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

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

CIRM Board member and ALS Patient Advocate Diane Winokur

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

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

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

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

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

Emotions and gratitude at changing of the guard at Stem Cell Agency

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Randy Mills and his family

Randy, as regular readers of this blog know, is, or rather was, the President and CEO of CIRM. James Harrison is less well known to the outside world but his imprint on CIRM, as our General Counsel and one of the key figures behind Proposition 71, is even bigger than that of Randy’s.

Randy came to the stem cell agency a little over three years ago and in pretty quick order completely refashioned us. Under his guidance CIRM 2.0 became a sleek, streamlined funding machine, turning what had been an almost two-year process from application to funding into one that took just 120 days. He revamped the frequency with which we offered specific programs, making it more predictable and so easier for researchers to know when the next round was coming up. He helped usher in a new Strategic Plan that is a blueprint for us until 2020.

But the changes he implemented were not just about the way we worked, it was also about how we worked and particularly how we worked together. He turned the agency into a true team, one where everyone felt they not only had a role to play but that what they did was important in determining the success of the agency.

Not surprisingly there was no shortage of people ready to praise him. CIRM Board Chair Jonathan Thomas (JT) thanked Randy for turning the agency around, transforming it into an organization that even the National Institutes of Health (NIH) now looks to as a model (more on that in a subsequent blog). Vice Chair Art Torres thanked Randy for his leadership and for his compassion toward patients, always putting them first in everything that he and the agency did. Board member Sherry Lansing called Randy “a genius and visionary”.

But perhaps the most moving tributes came from patients advocates.

Don Reed said; “When I first met Randy I didn’t like him. I thought CIRM was one of the best, if not the best, organization out there and who was this person to say they were going to come in and make it better. Well, you did Randy and we are all so very grateful to you for that.”

Adrienne Shapiro from Axis Advocacy, an organization dedicated to finding a cure for sickle cell disease, presented Randy with the “Heart of a Mother” award, thanking him for his tireless support of patients and their families.

Jake Javier, a participant in the Asterias spinal cord injury trial, wrote a note saying: “You positively affect so many through your amazing funding efforts for life changing research, and should be very proud of that. But something I will always remember is how personal and genuine you were while doing it. I hope you got the chance to meet as many of the people you helped as possible because I know they would remember the same.”

Randy – who is leaving to become President/CEO of the National Marrow Donor/Be The Match program – was clearly deeply moved by the tributes, but reminded everyone that he was leaving us in good hands. The Board named Dr. Maria Millan as the interim President and CEO, pending a meeting of a search committee to determine the steps for appointing a permanent replacement.

Randy praised Maria for her intelligence, compassion and vision:

“Maria Millan has been a great partner in all that we have achieved at CIRM. She was a key part of developing the Strategic Plan; she  understands it inside out and has been responsible for administering it. She is a wonderful leader and is going to be absolutely phenomenal.”

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James Harrison (left) with CIRM Board members Jonathan Thomas and Bert Lubin

The tributes for James Harrison were ever bit as moving. James has been a part of CIRM since before there was a CIRM. He helped draft Proposition 71, the ballot initiative that created the stem cell agency, and has played a key role since as General Counsel.

JT: “James has been a part of literally every decision and move that CIRM has made in its entire history. He’s been integral in everything. When I first came to CIRM, I was told by Bob Klein (JT’s predecessor as Chair) ‘Don’t brush your teeth without checking with James first’ suggesting a level of knowledge and expertise that was admirable.”

Jeff Sheehy “We would not be here without James. He organized the defense when we were sued by our opponents in the early days, through the various leadership challenges we had, all of the legal difficulties we had James was there to guide us and it’s been nothing short of extraordinary. Your brilliance and steadiness is amazing. While we are screaming and pulling our hair out there was James. Just saying his name makes me feel more relaxed.”

Sherry Lansing: “One thing I never worried about was our ethics, because you protected us at all times. You have such strong ethical values, you are always calm and rational and no matter what was going on you were always the rock who could explain things to everyone and deal with it with integrity.”

James is leaving to take a more active role in the law firm Remcho, Johansen & Purcell, where he is partner. Succeeding him as General Counsel is Scott Tocher, who has been at CIRM almost as long as James.

Randy; “To have someone like Scott come in and replace someone who wrote Proposition 71 speaks for the bench strength of the agency and how we are in very good hands.”

Art Torres joked “Scott has been waiting as long as Prince Charles has to take over the reins and we’re delighted to be able to work with him.”

We wish Randy and James great good luck in their next adventures.

 

Bridging the divide: stem cell students helping families with rare diseases become partners in research

Bridges & Rare Science

CIRM’s Bridges students and Rare Science’s families with rare diseases

Sometimes it’s the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own.

Want proof? This year, students in CIRM’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) San Marcos teamed up with parents of children with rare diseases, and the partnerships had a profound impact on all of them, one we hope might produce some long-term benefits.

Christina Waters, who helped create the partnerships, calls it “science with love.”

“We wanted to change the conversation and have researchers and families communicate, making families equal stakeholders in the research. The students bonded with the families and I truly feel that we made a difference in the lives of future researchers, in knowing how much their work can make a life changing impact on the lives of patients’ families who now have hope.”

The CIRM Bridges program helps prepare California’s undergraduate and master’s graduate students for highly productive careers in stem cell research. Students get a paid internship where they get hands-on training and education in stem cell research. They also work with patients and take part in outreach activities so they get an understanding of research that extends beyond the lab.

That’s where Christina Waters comes in. Christina is the founder of Rare Science, a non-profit group focused on rare diseases in children – we blogged about her work here – and she teamed up with CSU San Marcos to partner their Bridges students with five patient families with different rare diseases.

Cutting edge science

One of those families was Aaron Harding’s. Aaron’s son Jaxon has SYNGAP, a genetic disorder that can cause seizures, mental retardation, speech problems and autistic-like behavior. Two of the Bridges students who were doing their internship at ThermoFisher Scientific, Uju Nwizu and Emily Asbury, were given the task of using the gene-editing tool CRISPR Cas9 to help develop a deeper understanding of SYNGAP.

The students say it was an amazing experience:

Uju: “It had a huge impact on me. Every time I thought about SYNGAP I saw Jaxon’s face. This motivated me a lot.”

Emily: “People who work in labs everyday are most often working out the minutiae of research. They don’t often get a chance to see how their research can change or save the lives of real people. Meeting patients is so motivating because afterwards you aren’t just studying a mechanism, you now have a friend with the disease, so you can’t help but be personally invested in the search for a treatment.”

Emily and Uju are working to create iPSCs (induced pluripotent stem cells) that have the SYNGAP mutation. They hope these can be used to study the disease in greater depth and, maybe one day, lead to treatments for some of the symptoms.

Aaron says for families like his, knowing there are scientists working on his child’s disorder is a source of comfort, and hope:

“Personalizing diseases by connecting scientists with those they seek to impact is so important. Emily and Uju took this opportunity and ran with it, and that says a lot about them, and the team at ThermoFisher, taking on an exploring the unknown. That attitude is the heart of a scientist.”

Hearing stories like this is very gratifying, not just for the students and families involved, but for everyone here at CIRM. When we created the Bridges program our goal was to help students get the skills and experience needed to pursue a career in science. Thanks to the people at CSU San Marcos and Rare Science these students got a whole lot more.

Christina Waters: “We learned, we shared hope, we celebrated the courage of our families and the commitment of the students. It takes a village, and it is all of us working together that will make great changes for kids with rare diseases.”

For Uju and Emily, their experience in the Bridges program has made them doubly certain they want to pursue a career in science.

Uju: “I love stem cells and the promise they hold. After this program I hope to be part of a team that is committed to accelerating new stem cell therapies for rare and chronic diseases.”

Emily: “I’ve learned that I love research. After I finish my bachelor’s degree at CSU San Marcos I plan to pursue a graduate degree in molecular or cellular biology.”

 

Baseball’s loss is CIRM’s gain as Stanford’s Linda Boxer is appointed to Stem Cell Agency Board

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Dr. Linda Boxer: Photo courtesy Stanford University

One of the things that fascinates me is finding out how people end up in the job they have, the job they love. It is rare that the direction they started out on is the one they end on. Usually, people take several different paths, some intended, some unintended, to get to where they want to be.

A case in point is Dr. Linda Boxer, a renowned and respected researcher and physician at the Stanford School of Medicine, and now the newest member of the CIRM Board (you can read all about that in our news release).

In Dr. Boxer’s case, her original career path was a million miles from working with California’s stem cell agency:

“The first career choice that I recall as a young child was professional baseball—growing up in Minnesota, I was a huge Twins fan—I did learn fairly quickly that this was not likely to be a career that was available for a girl, and it wasn’t clear what one did after that career ended at a relatively young age.”

Fortunately for us she became interested in science.

“I have always been curious about how things work—science classes in grade school were fascinating to me. I was given a chemistry kit as a birthday gift, and I was amazed at what happened when different chemicals were mixed together: color changes, precipitates forming, gas bubbles, explosions (small ones, of course).

Then when we studied biology in middle school, I was fascinated by what one could observe with a microscope and became very interested in trying to understand how living organisms work.

It was an easy decision to plan a career in science.  The tougher decision came in college when I had planned to apply to graduate school and earn a PhD, but I was also interested in human health and disease and thought that perhaps going to medical school made more sense.  Fortunately, one of my faculty advisors told me about combined MD/PhD programs, and that choice seemed perfect for me.”

Along the way she says she got a lot of help and support from her colleagues. Now she wants to do the same for others:

“Mentors are incredibly important at every career stage.  I have been fortunate to have been mentored by some dedicated scientists and physicians.  Interestingly, they have all been men.  There were really very few women available as mentors at the time—of course, that has changed for the better now.  It never occurred to me then that gender made a difference, and I just looked for mentors who had successful careers as scientists and physicians and who could provide advice to someone more junior.

One of the aspects of my role now that I enjoy the most is mentoring junior faculty and trainees.  I don’t think one can have too many mentors—different mentors can help with different aspects of one’s life and career.  I think it is very important for established scientists to give back and to help develop the next generation of physicians and scientists.”

Dr. Boxer is already well known to everyone at CIRM, having served as the “alternate” on the Board for Stanford’s Dr. Lloyd Minor. But her appointment by State Controller Betty Yee makes her the “official” Board member for Stanford. She brings a valuable perspective as both a scientist and a physician.

The Minnesota Twins lost out when she decided to pursue a career in science. We’re glad she did.

 

CIRM’s Randy Mills leaving stem cell agency to take on new challenge

Mills, Randy Union Tribune K.C. Alfred

Some news releases are fun to write. Some less so. The one that CIRM posted today definitely falls into that latter group. It announced that CIRM’s President and CEO, Randy Mills, is leaving us to take up the role of President and CEO at the National Marrow Donor Program – NMPD/Be The Match.

It’s a great opportunity for him but a big loss for us.

Be The Match is a non-profit organization that delivers cures to patients in need of a life-saving marrow or cord blood transplant. The organization operates the national Be The Match Registry®—the world’s largest listing of potential marrow donors and donated umbilical cord blood units—matches patients with their marrow donor, educates healthcare professionals and conducts research so more lives can be saved. The organization also recently created a subsidiary—Be The Match BioTherapiesSM—that supports organizations pursuing new life-saving treatments in cellular therapy.

Randy has been at CIRM since April 2014. In that time he has dramatically re-shaped the agency, and, more importantly, dramatically improved the speed with which we are able to fund research. It’s no exaggeration to say that Randy’s drive to create CIRM 2.0 was a radical overhaul of the way we work. It made it easier for researchers to apply to us for funding, made our funding cycles more consistent and the application process simpler – though no less rigorous.

As our CIRM Board Chair Jonathan Thomas said in the news release:

“CIRM has experienced a remarkable transformation since Randy’s arrival. He has taken the agency to a new level by developing and implementing a bold strategic plan, the results of which include an 82% reduction in approval time for clinical trial projects, a 3-fold increase in the number of clinical trials, and a 65% reduction in the time it takes to enroll those trials. The opportunity for Randy to lead a tremendously important organization such as the NMDP/Be The Match is consistent with the values he demonstrated at CIRM, which put the well-being of patients above all else. We shall miss him but know he will do great things at NMDP/Be The Match.”

From a personal perspective, what most impressed me about Randy was his willingness to involve every person in the agency in changing the way we work. He could easily have come in and simply issued orders and told people what to do. Instead he invited every person at CIRM to sit in on the meetings that were shaping the new direction we took. You didn’t have to go, but if you did you were expected to offer thoughts and ideas. No sitting idly by.

Those meetings not only changed the direction of the agency, they also re-energized the agency. When people feel their voice is being heard, that their opinion has value, they respond by working harder and smarter.

The CIRM of today has the same mission as always – accelerating stem cell treatments to patients with unmet medical needs – but the people working here seem to have a renewed commitment to making that mission a reality.

Randy brought to CIRM energy and a renewed sense of purpose, along with some truly terrible jokes and a strange conviction that he could have been a great rock and roll drummer (suffice to say he made the right career choice when he went into research).

He changed us as an agency, for the better. We shall miss him, but know he will do great things in his new role at NMDP/Be The Match and we wish him success in his new job, and his family great joy in their new home.

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Maria Millan

Randy will be with us till the end of June and starting July 1st Dr. Maria Millan will take on the role of interim President and CEO.

 

 

 

You Are Invited: CIRM Patient Advocate Event, San Diego April 20th

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The word “cured” is one of the loveliest words in the English language. Last year we got to use it twice when we talked about stem cell therapies we are funding. Two of our clinical trials are not just helping people, they are curing them (you can read about that in our Annual Report).

But this was just part of the good news about stem cell research. We are making progress on many different fronts, against many different diseases, and we want to tell you all about that.

That’s why we are holding a special Patient Advocate event at UC San Diego on Thursday, April 20th from 12 – 1pm to talk about the progress being made in stem cell research, the problems we still face and need help in overcoming, and the prospects for the future.

We will have four terrific speakers:

  • Catriona Jamieson, Director of the CIRM UC San Diego Alpha Stem Cell Clinic and an expert on cancers of the blood
  • Jonathan Thomas, PhD, JD, Chair of CIRM’s Board
  • Jennifer Briggs Braswell, Executive Director of the Sanford Stem Cell Clinical Center
  • David Higgins, Patient Advocate for Parkinson’s on the CIRM Board

We will give updates on the exciting work taking place at UCSD and the work that CIRM is funding. We have also set aside some time to get your thoughts on how we can improve the way we work and, of course, answer your questions.

So we would love for you to join us, and tell your friends about the event as well. Here are the basic details.

What: Stem Cell Therapies and You: A Special Patient Advocate Event

When: Thursday, April 20th 12-1pm

Where: The Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037

Why: Because the people of California have a right to know how their money is helping change the face of regenerative medicine

Who: This event is FREE and open to the public

We have set up an EventBrite page for people to RSVP and let us know if they are coming.

We hope to see you there.