CIRM 2.0: How to Build a Better Stem Cell Agency and Speed up Treatments to Patients

Change is never easy. We all get used to doing things in a certain way and it can sometimes be difficult to realize that the way we have chosen, while it may have worked well at one time is perhaps not the best way to achieve our goals at this time. Well, change is coming to the stem cell agency.

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It’s not surprising that our new President & CEO, C. Randal Mills, Ph.D., would want to introduce some of his own ideas about how best to run the agency in the current moment of stem cell science. After all, it’s those ideas that landed him the job in the first place. Now Randy wants us to develop a clearer focus, one that is more aligned with his 4-point criteria for assessing everything we do.

  1. Will it speed up treatments to patients
  2. Will it increase the likelihood of successful treatments for patients
  3. Does it target an unmet medical need
  4. Is it efficient.

That new focus begins with re-imagining how we can be most effective in the way we fund research. Right now we put out what’s called an RFA or Request for Application, telling people who have promising projects in a particular area of stem cell research to submit an application and if they are successful they’ll get up to $20 million, depending on the kind of project.

The problem is, we often have long gaps between each round of funding and so a company or institution with a promising therapy will sometimes have to wait as much as a couple of years before they can apply again. If they do wait and are successful in their application it could still be another year or two before they are able to gain actual funding and begin a clinical trial. But when lives are at stake, you can’t afford to wait that long. So we’re looking at ways of speeding things up, making it easier for the best science to get the funds needed when they are needed.

At our Board meeting yesterday Randy outlined some broad concepts about what he wants to do and how it can be done. It’s part of his vision for the agency, a new focus that he is calling CIRM 2.0 (with acknowledgments to Dr. Paul Knoepfler who coined the term earlier this year)

As with any simple idea it’s really complicated. We need to achieve greater speed, to streamline the way we do things, without sacrificing the quality of the review process because we need to ensure that we only fund the best science.

In the months to come, as the precise details about these proposed changes are fine tuned, the Board will hear in greater detail how this will work and, as always, it will be up to them to decide if they think it’s a good idea.

Either way it will start a conversation about how we can become more efficient and more effective at living up to our mission, of accelerating therapies that target patients with unmet medical needs. And that always has to be a good thing.

For more details about the other big events at yesterday’s Board meeting, including awarding $16 million to ViaCyte to help it advance its promising therapy for type 1 diabetes, you can read the news release posted on our website.

September ICOC Boarding Meeting Begins Soon

The September ICOC Boarding Meeting begins this morning in Berkeley, CA.

The complete agenda can be found here, including a special Spotlight on Disease focusing on Inflammatory Bowel Disease.

For those not able to attend, feel free to dial in:

Dial in Infomation:

United States: (800) 230-1093
Access Code: 334835

WEB MEETING ACCESS INFORMATION:
——————————-
* https://www.webmeeting.att.com
* Meeting Number(s): 5114686455
* PARTICIPANT CODE: 313650

WEBEX LINK:
1. Go to https://cirm.webex.com/cirm/onstage/g.php?MTID=ef6aa60e45eb581e0e24ea4d2…
2. Click “Join Now”.

We will be providing a summary of the meeting’s highlights after the meeting—so stay tuned!

CIRM at Business of Personalized Medicine Summit

Exciting new technologies such as regenerative medicine, tissue engineering and gene therapy are already at the forefront of a new era of medicine. And today, CIRM’s own Business Development Officer, Neil Littman, moderated a panel titled The Impact of Next Generation Personalized Medicine Technologies: How Disruptive Tech Continues to Advance the Industry, at the annual Business of Personalized Medicine Summit.

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The panel discussed the innovative technologies we have at our disposal today, and provided a glimpse into the future—highlighting promising therapies already in the clinic as well as technologies that may be available in 5 to 10 years. For example, Curt Herberts, Senior Director of Corporate Development & Strategy from Sangamo BioSciences, discussed Sangamo’s grant under CIRM’s Strategic Partnership II Award, which uses genome-editing technology for a one-time treatment for the blood disorder Beta-thalassemia.

Importantly, the panel delved into potential paradigm shifts in medical care that may arise as a result of these new technologies, and discussed how to translate these cutting-edge technologies into human clinical trials. Carlos Olguin, Head of Bio/nano/Programmable Matter Group, Autodesk and Dr. Kumar Sharma, who directs the Center for Renal Translational Medicine University of California, San Diego La Jolla, rounded out the panel.

Finally, Neil asked panel members to discuss the issues surrounding market adoption and the potential resistance to paradigm-shifting technologies, the final hurdle in the delivery of much-needed therapies to patients.

CIRM Creativity Student Cindy Nguyen Goes “Beyond the Classroom”

This summer we’re sponsoring high school interns in stem cell labs throughout California as part of our annual Creativity Program. We asked those students to share their experiences through blog posts and videos.

Today in our final installment, we hear from Cindy Nguyen, who has been busy at Stanford University’s Beckman Center for Molecular and Genetic Medicine.

Beyond the Classroom

Cindy Nguyen

“And these are human induced pluripotent stem cells.”

I stood in awe. It was my first day in the lab, and I could not believe what I was seeing for the first time. I remembered reading about these “inner healers” in AP Biology class just a year ago and thinking about the endless possibilities of research that these induced pluripotent stem cells (iPSCs) could lead to. In a small classroom miles away from Stanford University, the existence of iPSCs seemed surreal and inaccessible. However, here I was standing before these cells, as one of the post-doctoral fellows of my lab was culturing them while describing their purpose.

Picking colonies at the bench.

Picking colonies at the bench.

One of the projects of my lab involves differentiating iPSCs into beating cardiomyocytes. It is almost unbelievable that fibroblasts could have their “biological clocks” rewounded and then be differentiated into pulsing heart cells so easily. I was reminded yet again of the incredible power of scientific research and all the open questions left to answer about iPSCs.

Spending the summer at a research laboratory at Stanford has given me the opportunity to become involved in life-changing research with access to everything I could ever need to conduct an investigation. Ranging from the thermal cycler to pipettes, all these commodities would be considered rare specialties in a high school biology classroom. I feel especially grateful to have the opportunity not only to conduct cutting-edge research in a lab on one of the most prestigious campuses in the country but also to learn about the world of research at my age.

Performing my first immunohistochemistry stain!

Performing my first immunohistochemistry stain!

Just a few months before, I had felt unsure about my future prospects. I did not have the chance to explore what having a career in science really meant. My family had a very little idea of what research was like and was not sure if this would be a rewarding career. However, after this summer’s incredible internship, I am confident in diving into biological sciences in the future. This position has given me the opportunity to show my family the great work that scientific researchers do every day and how rewarding it can be. The ambiguity of lab research has dissolved, and my future choices seems that much clearer.

A life long battle with Parkinson’s disease earns Dr. David Higgins a place on our Board

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson's disease

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson’s disease

As a child David Higgins imagined that the reason his grandmother wouldn’t talk to him was because of something he did, some fault of his. In time he came to realize that the reason was because his grandmother had Parkinson’s disease and she had trouble communicating with, not just him, but with everyone. It was the start of his life-long relationship with the disease, one that led to his own diagnosis with Parkinson’s a few years ago, and that has now led to him becoming the newest member of our governing Board, the Independent Citizens Oversight Committee.

David, now Dr. Higgins, was appointed by State Controller John Chiang. In his official letter announcing the news – which you can also find in our news release – he said:

“Diagnosed with Parkinson’s in 2011, his experience with the disease and its impact on a personal level brings a distinct perspective to the ICOC. Dr. Higgins also brings a broad range of experience from the biotechnology field. As a trained molecular biologist, his involvement in drug development and business operations places him in a unique position, understanding both science and process.”

David says he learned many important lessons from his grandmother, including the power of medical research:

“She was in one of the first L-dopa trials (this is a drug that converts into dopamine in the brain, to help counter the disease). I saw how that therapy helped change her life and it gave me my first lesson in how science and scientific research could change someone’s life.”

David’s mother was also diagnosed with, and later died from, a Parkinson’s-like form of dementia. His own diagnosis only served to further reinforce his commitment to doing something to fight the disease.

“I probably had symptoms that I was suspicious of for about two years before I was diagnosed. It was a mixed bag because as a scientist I finally had a label for what was going on and I thought at least now I can start fighting it. But the other side of me said “Oh my God I know exactly where this is going.”

As a research scientist he has held positions at several biotech companies, most recently as Head of US Operations for BioMedica, Inc., a UK-based gene therapy company. He also became a highly active Patient Advocate, and is now the California Assistant State Director with the Parkinson’s Action Network, as well as the new President of the Board of Directors of the Parkinson’s Association in San Diego.

David says he was completely stunned when he was asked if he would like to be on the CIRM Board. But the Chair of our Board, Jonathan Thomas, Ph.D., J.D., says it’s clear that he will be a terrific addition to the team:

“He is a powerful and passionate advocate not just for people with Parkinson’s disease but for science in general. He understands on a personal level how important it is for the public to support research trying to find new treatments and cures for currently incurable diseases.”

David says his personal experience with the disease has clearly influenced his life but does not mean he will focus on that alone:

“One thing I feel strongly about is that, yes I’m the Parkinson’s Patient Advocate on the Board, but first and foremost I’m an Advocate for everyone and I want to make sure that we spend our money wisely, and that we use our resources to identify and nurture the most promising stem cell projects across all target diseases.”

He says he takes up his new role with mixed emotions:

“I am thrilled to be a part of the CIRM governing Board, but I am also humbled to follow in the footsteps of Joan Samuelson, who has been CIRM’s Parkinson’s Patient Advocate from the beginning. She is a hard act to follow.”

Dr. Thomas echoed those sentiments saying:

“Joan set the gold standard for patient advocacy. She was a part of the agency from the very first and her passion, dedication and commitment to helping others was an inspiration to all of us. Joan was always willing to ask tough questions and push us all to do more to speed up the development of new therapies. We are all better for her service, and she will be missed.”

We are delighted to have David join us and we’re looking forward to working with him in the years to come.

kevin mccormack

Stem Cell Stories that Caught our Eye: “Let it Grow” Goes Viral, Stroke Pilot Study, The Bowels of Human Stem Cells, Tumor ‘Safety Lock.’

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

“Let it Grow” Goes Viral (and National!): Last week on The Stem Cellar we shared one of our favorite student videos from our annual Creativity Program. The video, a parody of the hit song from the movie Frozen, highlighted the outstanding creativity of a group of high school students from City of Hope in Los Angeles. And now, the song has made a splash nationwide—with coverage from ABC 7 Bay Area and even NBC New York!

Students from the City of Hope practice their routine for the group video

Students from the City of Hope practice their routine for the group video

Watch the full video on our YouTube page.

Stroke Pilot Study Shows Promise. Researchers at Imperial College London are currently testing whether stem cells extracted from a patient’s bone marrow can reverse the after effects of a stroke.

Reporting in this week’s Stem Cells Translational Medicine the team, lead by Dr. Soma Banjeree, describe their pilot study in which they collect a type of bone marrow stem cells called CD34+ cells. These cells can give rise to cells that make up the blood and the blood vessel lining. Earlier research suggested that treating stroke victims with these cells can improve recovery after a stroke—not because they replace the brain cells lost during a stroke, but because they release a chemical that triggers brain cells to grow. So the team decided to take the next step with a pilot study of five individuals.

As reported in a recent news release, this initial pilot study was only designed to test the safety of the procedure. But in a surprising twist, all patients in the study also showed significant improvement over a period of six months post-treatment. Even more astonishing, three of the patients (who had suffered one of the most severe forms of stroke) were living assistance-free. But since the first six months after injury is a time when many patients see improved function, these results need to be tested in a controlled trial where not all patients receive the cells

Immediate next steps include using advancing imaging techniques to more closely monitor what exactly happens in the brain after the patients are treated.

Want to learn more about using stem cells to treat stroke? Check out our Stroke Fact Sheet.

Deep in the Bowels of Stem Cell Behavior. Another research advance from UK scientists—this time at Queen Mary University of London researchers—announces important new insight into the behavior of adult stem cells that reside in the human gastro-intestinal tract (which includes the stomach and intestines). As described in a news release, this study, which examined the stem cells in the bowels of healthy individuals, as well as cells from early-stage tumors, points to key differences in their behaviors. The results, published this week in the journal Cell Reports, point to a potential link between stem cell behavior and the development of some forms of cancer.

By measuring the timing and frequency of mutations as they occur over time in aging stem cells, the research team, led by senior author Dr. Trevor Graham, found a key difference in stem cell behaviors between healthy individuals, and those with tumors.

In the healthy bowel, there is a relative stasis in the number of stem cells at any given time. But in cancer, that delicate balance—called a ‘stem cell niche’—appears to get thrown out of whack. There appears to be an increased number of cells, paired with more intense competition. And while these results are preliminary, they mark the first time this complex stem cell behavior has been studied in humans. According to Graham:

“Unearthing how stem cells behave within the human bowel is a big step forward for stem cell research. We now want to use the methods developed in this study to understand how stem cells behave inside bowel cancer, so we can increase our understanding of how bowel cancer grows. This will hopefully shed more light on how we can prevent bowel cancer—the fourth most common cancer in the UK.”

Finding the ‘Safety Lock’ Against Tumor Growth. It’s one of the greatest risks when transplanting stem cells: the possibility that the transplanted cells will grow out of control and form tumors.

But now, scientists from Keio University School of Medicine in Japan have devised an ingenious method that could negate this risk.

Reporting in the latest issue of Cell Transplantation and summarized in a news release, Dr. Masaya Nakamura and his team describe how they transplanted stem cells into the spinal columns of laboratory mice.

And here’s where they switched things up. During the transplantation itself, all mice were receiving immunosuppressant drugs. But then they halted the immunosuppressants in half the mice post-transplantation.

Withdrawing the drugs post-transplantation, according to the team’s findings, had the interesting effect of eliminating the tumor risk, as compared to the group who remained on the drugs. Confirmed with bioluminescent imaging that tracked the implanted cells in both sets of mice, these findings suggest that it in fact may be possible to finely tweak the body’s immune response after stem-cell transplantation.

Want to learn more about stem cells and tumor risk? Check out this recent video from CIRM Grantee Dr. Paul Knoepfler: Paul Knoepfler Talks About the Tendency of Embryonic Stem Cells to Form Tumors.

CIRM Creativity Student Hanan Sinada’s ‘Extraordinary’ Journey as a Budding Scientist

This summer we’re sponsoring high school interns in stem cell labs throughout California as part of our annual Creativity Program. We asked those students to share their experiences through blog posts and videos.

Today, we hear from Hanan Sinada, who has been busy at the Gladstone Institutes in San Francisco.

Extraordinary. That is the word I would use to describe my time here at Gladstone. This summer I have been an intern at the Gladstone Institute of Neurology, studying microglia. The brain has two main types of cells. Those cells are neurons and glial cells. Glia makes ninety percent of the cells in your brain. Although the word “glia” is derived from the Greek word meaning “glue”, glia cells are more like the support system that surround the neurons in the brain. Many people have not heard of glial cells because they are the dark matter of the brain and not involved in synaptic transition. However, glial cells have many significant functions in the central nervous system (CNS). Their main functions are to supply oxygen and nutrients to the neurons, hold neurons in place, destroy infectious agents, eliminate dead cells, and provide insulation (myelin) to neurons.

Hanan Sinada with her mentor, Gladstone Postdoctoral Researcher Dr. Grietje Krabbe

Hanan Sinada with her mentor, Gladstone Postdoctoral Researcher Dr. Grietje Krabbe

There are three main types of glial cells: microglia, astrocytes, and oligodendrocytes. In my research we focus specifically on microglial cells. Microglia only make up 10-15 percent of the total glia population. Microglia serve as the central nervous system’s macrophages. One function of microglia is to act as antigen presenting cells. Two other roles of the microglia are phagocytosis and cytotoxicity. In cytotoxicity, microglia release cytotoxic substances such as Nitric Oxide (NO) or hydrogen peroxide (H2O2), to damage neurons that have been infected. This leads to cell death. Microglia’s main function is to maintain homeostasis. As a result, microglia are constantly scavenging for apoptotic cells, infectious agents, or any foreign material. Microglia are the main orchestrators of the inflammatory response in the central nervous system (CNS). When an injury occurs in the spinal cord or the brain, microglia release cytokines that cause inflammation in that given area.

In my research we look closely at microglia because they are related to many neurodegenerative diseases, such as Alzheimer’s disease and Parkinson’s disease. My lab started to question about what would happen if we annihilated all the microglia in the brain. Would it decrease the possibility of avoiding the development of those diseases? So we gave wild type mice a drug that depleted all the microglia in the brain, and surprisingly enough the microglia repopulated the brain rapidly after a couple of days. By doing immunohistochemistry and using certain markers, I was able to find where this new microglia-like cell was coming from. From previous studies we already know that this new microglia is not from the periphery. Monocytes cannot cross the blood brain barrier to replace the microglia. We believe that this new microglia is coming from progenitor cell (a type of stem cell). However, we do not know which cell type is giving rise to this new microglia population.

Before starting my internship I did not know that it was going to be the most amazing and interesting learning experience I have ever had in my life. Although every now and then I would have a science crisis, such as having to change antibody because a certain staining would not work, I am so happy and lucky to be doing this cutting edge research. Not only did I learn so much but I am proud to say that I have contributed to the future of science.

Creativity Program Students Reach New Heights with Stem Cell-Themed Rendition of “Let it Go”

This summer we’re sponsoring high school interns in stem cell labs throughout California as part of our annual Creativity Program. We asked those students to share their experiences through blog posts, photos and videos.

Today, we bring you an outstanding group video from CIRM Interns at City of Hope in Los Angeles, with their own special version of the popular song, “Let it Go” from the movie Frozen.

These students have without a doubt showcased their extensive scientific knowledge in one of the most creative ways we at CIRM have ever seen!

Without further ado, we present “Let it Grow.”

CIRM Creativity Program: Interns Document their Experiences, One Photo at a Time

This summer we’re sponsoring high school interns in stem cell labs throughout California as part of our annual Creativity Program. We asked those students to share their experiences through blog posts, videos and on Instagram.

Today, we take a look at some of the top Instagram photos from our students. Want to take a peak at the rest? Search for the #CIRMCreativityLab hashtag on your Instagram app!

Megan Handley, a Creativity student in the Denise Montell lab at UCSB, snapped this image of a Drosophila ovariole(egg string) taken in fluorescence microscopy. The blue is DAPI(stains nucleus, and the green is anti-HTs(stains membranes).

Megan Handley, a Creativity student in the Denise Montell lab at UCSB, snapped this image of a Drosophila ovariole(egg string) taken in fluorescence microscopy. The blue is DAPI(stains nucleus, and the green is anti-HTs(stains membranes). [Credit: Megan Handley]

Students from the City of Hope practice their routine for the group video

Students from the City of Hope practice their routine for the group video[Credit: Grace Lo]

Emma Cruisenberry, an intern in the Rothman Lab at UCSB, snapped these two photos C. elegans—the top under normal conditions, versus C. elegans expressing the GFP marker under UV light in the intestinal cells. [Credit: Emma Cruisenberry]

Emma Cruisenberry, an intern in the Rothman Lab at UCSB, snapped these two photos C. elegans—the top under normal conditions, versus C. elegans expressing the GFP marker under UV light in the intestinal cells. [Credit: Emma Cruisenberry]

Bridging the gap: helping create a new generation of stem cell scientists

Inspiration comes in many different shapes and sizes, but when you see it there is no mistaking it. And when you meet and talk to the students in our Bridges program you find inspiration in each and every one of them.

The program is designed to train the next generation of stem cell scientists, bridging (hence the name) the gap between undergraduate and Master’s level training in research. But it’s so much more than just a recruiting and training program because one of the goals of Bridges is to find students who are often overlooked for opportunities like this: students who may be the first in their family to go to college, who don’t come from a wealthy family or fancy school. These students seize the opportunity with both hands and their sense of delight at being given a chance, and enthusiasm for the work is exciting and infectious.

We held our annual Bridges Trainee Meeting in Burlingame this week, a chance for all the students in the program to come together, listen to lectures from world-class stem cell researchers, and show their posters describing the work they have done over the past year.

At first many of them seem a little shy but once you ask them about their experiences their enthusiasm simply bubbles over. Shayda Kianfar graduated from Berkeley City College and is now studying at the University of California, Berkeley. She says she was accepted into the program even though she had no prior lab experience:

“This has given me an amazing experience. To be surrounded by so many incredible people, to have great mentors is life changing. You learn so many new skills and it opens your eyes. I hadn’t thought about stem cell work before but now I would love to do this. It’s so exciting.”

Kevin Martinez talks to fellow Bridges student David James

Kevin Martinez talks to fellow Bridges student David James

Kevin Martinez graduated from San Francisco State University and says getting a chance to work with extraordinary researchers like Thea Tlsty, Ph.D., at the University of California, San Francisco, was incredible. Kevin got to work with Tlsty and her team on their discovery that certain rare cells extracted from adult breast tissue can be instructed to become different types of cells – a discovery that could have important potential for regenerative medicine.

He says what surprised him most of all was how much independence they gave him, he wasn’t treated like a student but like a colleague:

“They trained me and gave me the experience and opportunity to do amazing work. This is great training for a career either in academia or industry because they teach you how to do research independently, but to also work as part of a team.”

Eleanor Kim, spent her year at City of Hope near Los Angeles. She focused on leukemia stem cells (LCS), testing different medications to see if they could be effective at preventing recurrence of the leukemia or the speed with which it spreads.

Bridges student Eleanor Kim

Bridges student Eleanor Kim

Eleanor was a pre-med student who hadn’t really thought about research until she found out about the Bridges program. Now she’s set her sights on becoming an MD/PhD:

“This got me much more interested in the biology of cancers, what is driving them, what controls them. I want to be able to talk to my patients about what is happening to them but also to be able to do research that might be able to help them.”

Eleanor says she also learned a valuable lesson about the need for a good night’s sleep:

“I learned that you have to work hard but that you also can’t work to the point where you are sleep deprived. This is such detail-oriented work that being sleepy can lead to mistakes and one mistake can set you back days.”

Each Bridges student has their own story; each brings their own unique perspective to their work and to the field. You can hear some of our students talk about how important this opportunity was for them, and how it has changed them in so many ways.

kevin mccormack