Super stem cell exhibit opens in San Diego

Stem cell exhibit

The best science museums are like playgrounds. They allow you to wander around, reading, watching and learning and being amazed as you go. It’s not just a feast for the mind; it’s also fun for the hands.  You get to interact with and experience science, pushing buttons, pulling levers, watching balls drop and electricity spark.

The best science museums bring out the kid in all of us.

This Saturday a really great science museum is going to be host to a really great exhibition. The Reuben H. Fleet Science Center in San Diego is the first stop on a California tour for “Super Cells: The Power of Stem Cells”. The exhibit is coming here fresh from a successful tour of Canada and the UK.

The exhibit is a “hands-on” educational display that demonstrates the importance and the power of stem cells, calling them “our body’s master cells.” It uses animations, touch-screen displays, videos and stunning images to engage the eyes and delight the brain.

stem cell exhibit 2Each of the four sections focuses on a different aspect of stem cell research, from basic explanations about what a stem cell is, to how they change and become all the different cells in our body. It has a mini laboratory so visitors can see how research is done; it even has a “treatment” game where you get to implant and grow cells in the eye, to see if you can restore sight to someone who is blind.

 

In a news release the Fleet Science Center celebrated the role that stem cells play in our lives:

“Stem cells are important because each of us is the result of only a handful of tiny stem cells that multiply to produce the 200 different types of specialized cells that exist in our body. Our stem cells continue to be active our whole lives to keep us healthy. Without them we couldn’t survive for more than three hours!”

It is, in short, really fun and really cool.

Of course we might be a tad biased here as we helped produce and develop the exhibit in collaboration with the Sherbrooke Museum of Science and Nature in Canada, the Canadian Stem Cell Network, the Centre for Commercialization of Regenerative Medicine in Canada; the Cell Therapy Catapult in the UK, and EuroStemCell.

stem cell exhibit 3

The exhibit is tri-lingual (English, Spanish and French) because our goal was to create a multi-lingual global public education program. San Diego was an obvious choice for the first stop on the California tour (with LA and the Bay Area to follow) because it is one of the leading stem cell research hubs in the U.S., and a region where CIRM has invested almost $380 million over the last ten years.

As our CIRM Board Chair, Jonathan Thomas, said:

“One of our goals at CIRM is to help spread awareness for the importance of stem cell research. San Diego is an epicenter of stem cell science and having this exhibition displayed at the Reuben H. Fleet Science Center is a wonderful opportunity to engage curious science learners of all ages.”

The Super Cells exhibit runs from January 23 to May 1, 2016, in the Main Gallery of the Reuben H. Fleet Science Center. The exhibition is included with the cost of Fleet admission.

For more information, visit the Reuben H. Fleet Science Center website.

Training the Next Generation of Stem Cell Scientists

Nobel prize winners don’t come out of thin air, they were all young, impressionable kids at one point in time.  If you ask any award-winning scientists how they got into science research, many of them would likely tell you about an inspiring teacher, an encouraging parent, or a hands-on research opportunity that inspired or helped them to pursue a scientific career.

Not every student is lucky enough to have one of these experiences, and many students, especially those from low income families, might never be exposed to good science or have the opportunity to pursue a career as a scientist.

CIRM is changing this for students in California by committing a significant portion of its funds to educating and training future stem cells scientists.

Yesterday, the Board approved over $42 million to fund two of CIRM’s educational programs, the Bridges to Stem Cell Research and Therapy Awards (Bridges) and the Summer Program to Accelerate Regenerative Medicine Knowledge (SPARK).

Bridging the Stem Cell Gap

The Bridges program supports undergraduate and master’s level students by providing paid research internships at California universities or colleges that don’t have a major stem cell research program. This program has evolved over the past seven years since it began, and now includes training and education courses in stem cell research, and direct patient engagement and outreach activities within California’s diverse communities.

CIRM’s president, Randy Mills explained in a press release:

Randy Mills, Stem Cell Agency President & CEO

Randy Mills, CIRM President & CEO

“The goal of the Bridges program is to prepare undergraduate and Master’s level students in California for a successful career in stem cell research. That’s not just a matter of giving them money, but also of giving them good mentors who can help train and guide them, of giving them meaningful engagement with patients and patient advocates, so they have a clear vision of the impact the work they are doing can have on people’s lives.”

Chairman of the CIRM Board, Jonathan Thomas, added:

Jonathan Thomas

Jonathan Thomas, Chairman of the CIRM Board

“The Bridges program has been incredibly effective in giving young people, often from disadvantaged backgrounds, a shot at a career in science. Of the 700 students who have completed the program, 95 percent are either working in a lab, enrolled in school or applying to graduate school. Without the Bridges program this kind of career might have been out of reach for many of these students.”

The CIRM Board voted to approve $40.13 million for the Bridges program, which will fund 14 programs at California state universities and city colleges. Each program will be able to support ten students for five years.

SPARKing Interest in Stem Cells

The SPARK program supports summer research internships for high school students that represent the diversity of the state’s population. It evolved from an earlier educational program called Creativity, and now emphasizes community outreach, direct patient engagement activities, and social media training along with training in stem cell research techniques.

“SPARK is all about helping cultivate high school students who are interested in science, and showing them it’s possible to have a career doing something they love,” said Randy Mills.

The Board approved $2.31 million for the SPARK program, which will provide California institutions funding support for five to ten students each year. Seven programs received funding including the Children’s Hospital Oakland Research Institute, UC San Francisco, UC Davis, Cedars-Sinai, City of Hope, USC and Stanford.

2015 Creativity Program students (now called SPARK).

2015 Creativity Program students (now called SPARK).

Training the Next Generation

For years, national leaders, including President Obama, have warned that without skilled, experienced researchers, the U.S. is in danger of losing its global competitiveness in science. But cuts in federal funding for research mean this is a particularly challenging time to begin a scientific career.

Our goal with the Bridges and SPARK programs is to address both these issues and support young scientists as they get the experience they need to launch their careers.


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The Critical Role of Patient Advocates in Accelerating Stem Cell Cures

At CIRM, our goal is to bring stem cell therapies to patients with unmet medical needs, and we do that by funding the most promising and innovative research in regenerative medicine. A critical component of this goal is to support our patient advocates and make sure that their voices are heard.

At this year’s World Stem Cell Summit, patient advocates from around the world, representing a breadth of diseases and disorders, came together to share their stories, goals, and needs with the larger scientific community.

One session that particularly stood out, was “Accelerating Cures: The Critical Role of Patient Advocates” on Day 3 of the conference. This panel featured key leaders in patient advocacy:

  • Don Reed, the “Grandfather of Stem Cell Research Advocacy”, Vice President of Public Policy at the Americans for Cures Foundation
  • Frances Saldaña, an advocate for Huntington’s disease (HD) and founder of HD-Care at UC Irvine, which is a support group to advance HD research and clinical care
  • Tory Williams, the Executive Director of the Alabama Institute of Medicine (AIM) which raises funds and awareness for stem cell treatments and cures of disease and injury and the author of “Inevitable Collision

The panel was moderated by our fearless leader and head of communications, Kevin McCormack. Each speaker shared their story about how they became a patient advocate and what they are currently doing to push the pace of stem cell research.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed described the heartbreaking story of his son Roman Reed, who suffered a severe spinal cord injury while playing football. Through Don and Roman’s relentless efforts, “Roman’s Law” was passed in 1999, which raised $17 million in California state funding for spinal cord injury research. Don was also a key instigator for the passage of Proposition 71, which gave $3 billion dollars to our agency to fund stem cell research. He continues to be a passionate advocate for stem cell research and spinal cord injury patients, and recently published a book called “Stem Cell Battles: Proposition 71 and Beyond” which you can read more about in our recent blog.

Next, Frances Saldana told a compelling story of raising a family of three beautiful children with a husband who had Huntington’s disease. Unaware of his condition when they were together, Frances’ world took a devastating turn when he died of HD, leaving her to question whether her children would face the same fate. Sadly, all three of Frances’s kids carried the HD mutation. Having to deal with the passing of her two daughters, and a son who is battling the end stages of this disease, Frances decided to share her experience with others and to create a support organization called HD-Care so that others wouldn’t have to face similar experiences alone. HD-Care is conducting an aggressive campaign to bring visibility to HD and supports cutting-edge research in the field including the work done by CIRM-grantee Dr. Leslie Thompson at UC Irvine.

Frances told the audience that her happiest moment since this all began was when her daughter Margie, already suffering from symptoms of HD, spoke at CIRM in 2007. She saw the Board and the scientists and thought, “somebody cares, and somebody will find a cure.” It was a new chapter for her, she explained, and she knew something good was going to happen.

Lastly, Tory Williams, introduced the Alabama Institute of Medicine, which is a non-profit organization that supports the stem cell community with education and public dialogue. She started the institute following both personal and family experiences with cancer and after TJ Atchinson, a close family friend, suffered a severe spinal cord injury. Along the way, she forged a close relationship with Roman Reed who helped her pass TJ’s law in 2013, which is an Alabama state law that promotes spinal cord injury research.

“The goal [of AIM],” said Williams, “is to make a difference in people’s lives affected by disease and injury by helping to advance medicine to eradicate these debilitating issues.”

Laurel Barchas, Student Society for Stem Cell Research

Laurel Barchas, Student Society for Stem Cell Research

When the session was opened up to questions, the atmosphere in the room turned electric. Patients and scientists stood up to tell their stories and asked hard questions. One question came from Laurel Barchas, one of the founders of the Student Society for Stem Cell Research, who asked how we as a society can advocate for mental illness and similar diseases where the symptoms are not visible and where patients are either embarrassed or hesitant to make their disease public. Another question was how emerging countries like Mexico who don’t have the same benefits and infrastructure as the US can promote and support patient advocacy.

The mood of the advocates was positive but measured. They know that new treatments and cures take time but they also pointed out that many people don’t have much time so we have to work as hard as we can to help them.

The panel ended with the consensus that the voices of patient advocates are invaluable, and that they will be the key to accelerating stem cell therapies into cures. Frances Saldaña urged other patient advocates that the key to progress is to be aggressive, and be unafraid to be out there. Don Reed concluded on a similar note with quote from Shakespeare’s Hamlet:

“Whether ’tis Nobler in the mind to suffer

The Slings and Arrows of outrageous Fortune,

Or to take Arms against a Sea of troubles,

And by opposing end them.”


Related links:

Why “Right to Try” laws are more feel good than do good

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L to R: Don Gibbons, CIRM; Jeanne Loring; Beth Roxland; Aaron Levine

In the last few years some 24 states have approved so-called “Right to Try” laws. These are intended to give terminally ill patients faster and easier access to experimental therapies. But a panel of experts at the World Stem Cell Summit in Atlanta today said they are more symbolic than anything and do little to actually help patients get much-needed therapies.

The Right to Try laws are modeled after a federal law that allows “compassionate use” of experimental medications and lets doctors prescribe investigational medicines being safely used in early stage clinical trials.

Beth Roxland, a bioethicist with Johnson & Johnson, says the name of the law is misleading:

“If you look at the actual text of these laws they only say you have the right to “ask” for these drugs. That right already exists in federal law but neither federal law nor these Right to Try laws say you have the right to access.”

Aaron Levine from Georgia Tech says it’s also misleading to assume that just because a state passes a Right to Try law that it has any legal impact. He says state laws don’t over rule the Food and Drug Administration’s (FDA) regulation of this area and so the federal government would still have the authority to stop this kind of access.

But Levine says these laws are interesting in that they are indicative of the growing determination of patients and patient advocates to work around obstacles to access and have a bigger say in their own care.

One of the audience members, William Decker from Baylor College of Medicine, says that in Texas a law was recently crafted saying that as long as a potential therapy had gone through a Phase 1 safety trial it should be offered to the public and the public should be able to pay for it.

“If you know how clinical trials work you know you can get almost any schlock through a Phase 1 trial and the kinds of things that you can get to the public without any idea if they work often turn out to not be very useful. We saw this as an avenue to promote fraud, and the last thing you should be doing to a dying patient is take their money or divert their attention away from something that might help them.”

Decker and his colleagues argued before the Texas Legislature that potential therapies should at least have to go through a Phase 2 trial to make sure they were not only safe but also showed some benefit for patients. In the end Texas lawmakers rejected the Phase 2 idea but did say patients could not be charged for the therapy, and there could be no compensation from insurers or anyone else for the manufacturer of the therapy.

He says removing the financial benefits and incentives pretty much ensured that no company would offer patients a therapy under this law.

Jeanne Loring, a CIRM grantee from the Scripps Research Institute, says that likely won’t stop other clinics in other states:

“Some stem cell clinics are using adipose (stem cells derived from fat) therapy as an option for every disease imaginable and I’m sure some will take advantage of these laws to say it gives them the right to offer these to patients and the patients will pay for them directly. “

Roxland says that may already be happening:

“I think there is some evidence on the stem cell side that companies have popped up in states that have these laws, to make it easier to offer their therapies to patients.”

The panel agreed that in most cases these laws don’t give patients any rights they don’t already have, but do give the appearance of making access easier. They said it’s feel-good legislation, allowing people to feel they are doing something without actually doing anything.

Aaron Levine said that while some companies may try to take advantage of these laws, the most serious ones won’t:

 “Almost any legitimate company that wants an FDA approved product wouldn’t want to take advantage of these laws. It could put their product at risk. Most companies that need to work with the FDA have no incentive to go this route.”

 

 

The bottom line: stem cell therapies will never be widely available if insurers won’t pay for them

The second session of the World Stem Cell Summit in Atlanta moved past all the promising science and right to the nitty-gritty of making cell-based therapies common. Four panelists reminded the audience that while they too are super excited by the potential for this field, unless folks developing therapies think about reimbursement early those therapies will not become a reality in routine clinical care.

reimbursement_shutterstock_326604206

“Stem cell therapies seem unstoppable with seemingly limitless possibilities, but success requires early planning for reimbursement,” said moderator Michael Levinson, a lawyer and physician with the law firm Hogan Lovells.

Elizabeth Powers of the IMS Consulting group suggested the audience pay close attention to the cancer market.  She said insurers and other payers of health care services are tired of paying for “statistically significant” improvements in survival that only translate to a few weeks on average. She said payers are moving away from just whether a new therapy is different from prior therapies and want to be shown true value.

A further reminder to start the reimbursement process early came from panelist Deborah Dean of MiMedx.  She said the process of just applying for a reimbursement code takes two years and after that it can take months or more to then present your case to insurers to turn that code into actual payments.

During the question period there was a bit of potential good news attached to an industry trend I did not expect. The consolidation of insurers, with two major mergers on deck, could actually extend the average length of time a customer is with an insurer from between two to five years to between five to ten years. This may make insurers more willing to pay for a one-time curative therapy that is expensive but eliminates chronic therapy costs.

Call to Action by FDA at World Stem Cell Summit

Califf

FDA Deputy Commissioner Dr. Robert Califf talking at the World Stem Cell Summit

The World Stem Cell Summit annual conference in Atlanta kicked off today with a clarion call from Dr. Robert Califf, the Deputy Commissioner for the Food and Drug Administration. He told the audience:

“We want you to accelerate translation to produce safe and effective therapies that can be delivered reliably”

It was a message that everyone in the room, scientists and patient advocates, would love to be able to comply with. The question of course is how do you do that in a way that puts the emphasis on both speed, to get the therapies to patients who need them, and safety, so you don’t put those patients at risk.

That’s quite a challenge considering that, as panel moderator Julie Allickson of Wake Forest Institute for Regenerative Medicine said:

“the estimate now is it costs $2.4 billion and up to ten years to take something to the clinic.”

Even if that dollar amount is higher than many think it would take to bring a stem cell therapy to a clinical trial it is an indication of the challenge the field faces.

Califf, who has only been at the FDA for 8 months, says that regenerative medicine is:

“not the only field exploding with scientific knowledge and seeing a future that’s very different from what we see today so it’s exciting but also an enormous challenge for the FDA. One of the real eye openers for me is to be at the FDA and hear about drugs that have been on the market for 45 years and we’re still learning about them.”

He says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. “That’s an issue we struggle with every day.”

Califf was optimistic that the balance can be struck and progress can be made, but said that this can only truly be done if the patient is at the table as an active participant.

“Our national clinical research system is well intention but flawed. We need to have a new system that shares information right across the system and where patients are at the center. Patients should be driving the national research infrastructure. They are an essential part of change. It’s happening in Congress because they are hearing from constituents that this is what they want, a voice in the research being done that affects them.”

For the patients and patient advocates in the audience it was a welcome message. For years they have been calling for a louder voice in the research that affects them and their loved ones. Knowing they have a sympathetic ear in the FDA could be an encouraging sign that their voices are finally being heard.

We will be writing more as the conference unfolds so stay tuned!

 

 

 

A scientific conference we can all enjoy

Scientific conferences are fascinating events. You get a chance to mingle with some of the leading researchers and thinkers in the field, and to learn about the latest advances. But, to be honest, for those of us who don’t have a scientific background, they can also be a little bit intimidating.

This is sometimes how I feel at them.

Courtesy The New Yorker

Courtesy The New Yorker

That’s where the World Stem Cell Summit comes in. It’s an annual event that brings together researchers, companies, scientists and patient advocates to talk about the progress being made in stem cell research and to explore ways to advance the field even further, and faster by working together.

Changing the tone

The patient advocate role is a critical one here. It makes the voice of the patient a key element in every discussion and changes the tone of the event from talking about what is being done to or for patients, to what is being done with patients. It’s a small but tremendously important difference.

Dr. Evan Snyder, Director of the Stem Cells and Regenerative Medicine program at Sanford – Burnham Medical Research Institute captures that feel when he says:

“We’re looking forward to the valuable information-sharing opportunities and discussions that only occur when stem cell researchers, patient advocates, and representatives of many other stakeholder groups converge at the World Stem Cell Summit. Occasions like these help us advance our research on the basic biology of stem cells and spur the development of new, and more personalized, medical applications for this science.”

Because more than ten percent of those attending are patient advocates the talks are given at a level that someone without a science background can generally understand. The presentations are no less fascinating; they are just a lot more accessible.

Stephen Rose, the Chief Research Officer with the Foundation Fighting Blindness says it brings different groups together in a way other conferences usually don’t:

“Policy experts learned about researchers’ needs. Advocates learn about policy and legislation. It also brought ethical issues to the table, which is critical if we’re going to resolve them and keep the research moving forward.”

Researchers have a lot of opportunities throughout the year to meet with other scientists but patient advocates don’t, so the World Stem Cell Summit is a great chance for them to meet with their colleagues and counterparts from all over the US. It gives them a chance to share ideas, offer support and explore ways they can collaborate.

More than just a meeting

For many advocates who are focused on diseases that affect relatively small numbers of people these events are a great way to recharge their batteries and to remind themselves they are not alone in this fight.

If you are thinking about going to one conference this year, this is a great one to chose. This year the World Stem Cell Summit is being held December 10 – 12 in Atlanta, Georgia.

We’ll be there and we’d love to see you there too.

New Video: Spinal Cord Injury and a CIRM-Funded Stem Cell-Based Trial

Just 31 years old, Richard Lajara thought he was going to die.

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Richard Lajara, the 4th participant in Geron’s stem cell-based clinical trial for spinal cord injury.

On September 9, 2011 he slipped on some rocks at a popular swimming hole and was swept down a waterfall headfirst into a shallow, rocky pool of water. Though he survived, the fall left him paralyzed from the waist down due to a severed spinal cord.

Patient Number Four
At that same time period, Geron Inc. had launched a clinical trial CIRM helped fund testing the safety of a stem cell-based therapy for spinal cord injury (SCI). It was the world’s first trial using cells derived from human embryonic stem cells and Lajara was an eligible candidate. Speaking to CIRM’s governing Board this past summer for a Spotlight on Disease seminar, he recalled his decision to participate:

“When I participated with the Geron study, I was honored to be a part of it. It was groundbreaking and the decision was pretty easy. I understood that it was very early on and I wasn’t looking for any improvement but laying the foundation [for future trials].”

A few months after his treatment, Geron discontinued the trial for business reasons. Lajara was devastated and felt let down. But this year the therapy got back on track with the announcement in June by Asterias Biotherapeutics that they had treated their first spinal cord injury patient after having purchased the stem cell assets of Geron.

Getting Hope Back on Track
Dr. Jane Lebkowski, Asterias’ President of R&D and Chief Scientific Officer, also spoke at the Spotlight on Disease seminar to provide an overview and update on the company’s clinical trial. A video recording of Lebkowski’s and Lajara’s presentations is now available on our web site and posted here:

As Dr. Lebkowski explains in the video, Asterias didn’t have to start from scratch. The Geron study data showed the therapy was well tolerated and didn’t cause any severe safety issues. In that trial, five people (including Richard Lajara) with injuries in their back received an injection of two million stem cell-derived oligodendrocyte progenitor cells into the site of spinal cord damage. The two million-cell dose was not expected to show any effect but was focused on ensuring the therapy was safe.

Oligodendrocyte Precursors: Spinal Cord Healers
As the former Chief Scientific Officer at Geron, Lebkowski spoke first hand about why the oligodendrocyte precursor was the cell of choice for the clinical trial. Previous animal studies showed that oligodendrocyte progenitors, a cell type normally found in the spinal cord, have several properties that make them ideal cells for treating SCI: first, they help stimulate the growth of damaged neurons, the cell type responsible for transmitting electrical signals from the brain to the limbs.

Second, the oligodendrocytes produce myelin, a protein that acts as an insulator of neurons, very much like the plastic covering on a wire. In many spinal cord injuries, the nerves are still intact but lose their myelin insulation and their ability to send signals. Third, the oligodendrocytes release other proteins that help reduce the size of cysts that often form at the injury site and damage neurons. In preclinical experiments, these properties of oligodendrocyte progenitors improved limb movement in spinal cord-severed rodents.

Together, the preclinical animal studies and the safety data from the Geron clinical trial helped Asterias win approval from the Food and Drug Administration (FDA) to start their current trial, also funded by CIRM, this time treating patients with neck injuries instead of back injuries.

The Asterias trial is a dose escalation study with the first group of three patients again receiving two million cells. The trial was designed such that if this dose shows a good safety profile in the neck, as it did in the Geron trial in the back, then the next cohort of five patients will receive 10 million cells. In fact, Asterias reported in August that the lower dose was not only safe but also showed some encouraging results in one of the patients. And just two days ago Asterias announced their data monitoring committee recommended to begin enrolling patients for the 10 million cell dose.  If all continues to go well with safety, the dose will be escalated to 20 million cells in the third cohort of five patients. While two million cells was a very low safety dose, Asterias anticipates seeing some benefit from the 10 and 20 million cell doses.

Changing Lives by Increasing Independence
Does Lebkowski’s team expect the patients to stand up out of their wheelchairs post-treatment? No, but they do hope to see a level of improvement that could dramatically increase quality of life and decrease the level of care needed. Specifically, they are looking to see a so-called “two motor level improvement.” In her talk Lebkowski explained this quantitative measure with the chart below:

“If a patient is a C4 [meaning their abilities are consistent with someone with a spinal cord injury at the fourth cervical, or neck, bone] they will need anywhere from 18 to 24 hours of attendant care for daily living. If we could improve their motor activity such that they become a C6, that is just two motor levels, what you can see is independence tremendously increases and we go from 18 to 24 hour attendant care to having attendant care for about four hours of housework.”

Slide13 cropped

Small improvements in movement abilities can be life changing for people with spinal cord injuries.

It’s so exciting the field is at a point in time that scientists like Dr. Lebkowski are discussing real stem cell-based clinical trials that are underway in real patients who could achieve real improvements in their lives that otherwise would not be possible.

And we have people like Richard Lajara to thank. I think Dr. Oswald Stewart, the Board’s spinal cord injury patient advocate, summed it up well when speaking to Lajara at the meeting:

“Science and discovery and translation [into therapies] doesn’t happen without people like you who are willing to put yourselves on the line to move things forward. Thank you for being in that first round of people testing this new therapy.”

Don Reed Reflects on the California Stem Cell Initiative

StemCellBattlesCoverYesterday was stem cell awareness day. In honor of this important event, Don Reed held a book reading at CIRM for his newly released book, STEM CELL BATTLES: Proposition 71 and Beyond: How Ordinary People Can Fight Back Against the Crushing Burden of Chronic Disease.

Don has worn many hats during his life. He’s been a power lifter, a diver at Sea World, and is one of California’s most tenacious stem cell research advocates. His stem cell journey began when his son, Roman Reed, was seriously injured in a football accident, leaving him mostly paralyzed from the neck down.

Both Don and Roman didn’t let this tragic event ruin their lives or steal their hope. In fact, both Don and his son were instrumental for getting proposition 71 to pass, leading to the birth of CIRM and new hope for patients with uncured diseases.

At yesterday’s book reading, Don chronicled the early battles to get human stem cell research off the ground in California, the progress that’s been made so far and the promise for future therapies. It was truly an inspiring event, bringing together patients, friends of Don and his wife Gloria, and CIRM scientists to celebrate the stem cell research accomplishments of the past ten years.

DonReedBook-reading2

Enjoy more pictures of the event below and a short video of Jonathan Thomas, Chair of the Governing Board of CIRM, who said a few words in praise of Don Reed’s efforts to fight for stem cell research in California.

DonReedBook-group

Don Reed and his wife Gloria share a smile with CIRM’s Pat Olson.

DonReedBook-signing

Jonathan Thomas and Don Reed.


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CIRM Scholar Spotlight: Matt Donne on Lung Stem Cells

CIRM has funded a number of educational and research training programs over the past ten years to give younger students and graduate/postdoc scholars the opportunity to explore stem cell science.

Two of the main programs we support are the Bridges and the CIRM Scholars Training Program. These programs fund future scientists from an undergraduate to postdoctoral level with a goal of creating “training programs that will significantly enhance the technical skills, knowledge, and experience of a diverse cohort of… trainees in the development of stem cell based therapies.”

The Stem Cellar team was interested to hear from Bridges and CIRM scholars themselves about their experience with these programs, how their careers have benefited from CIRM funding, and what research accomplishments they have under their belt. We were able to track some of these scholars down, and will be publishing a series of interview-style blogs featuring them over the next few months.

Matt Donne

Matt Donne

We start off with a Matt Donne, a PhD student at the University of California, San Francisco (UCSF) in the Developmental and Stem Cell Biology graduate program. Matt is a talented scientist and has a pretty cool story about his research training path. I sat down with Matt to ask him a few questions.


Q: Tell us how you got into a Stem Cell graduate program at UCSF.

MD: I was fortunate to have Dr. Carmen Domingo from San Francisco State support my application into the CIRM Bridges Program. I’d been working for Dr. Susan Fisher at UCSF for a couple of years and realized that I wanted to get a PhD and go to UCSF. I thought the best way to do that was improve my GPA and get a masters degree in stem cell biology. I applied to the CIRM program at SF State, and was accepted.

The Bridges Program has been a great feeder platform to get students more science experience exposure than they would have otherwise received, and prepares them well to move on to competitive graduate schools.

After receiving my Masters degree, I was admitted into the first year of the Developmental and Stem Cell Biology program at UCSF. When the opportunity to apply for a training grant from CIRM came about between my first and second year of at UCSF, I knew I had to give it a chance and apply. With the help of my mentor, Dr. Jason Rock, I wrote a solid proposal and was awarded the fellowship.

While at SF State, Carmen was extremely supportive and always available for her students. Since then, many of us still keep in touch and more have joined the UCSF graduate school community.

Q: Can you describe your graduate research?

MD: The field of regenerative medicine is searching for ways to allow us to repair injuries similar to how the Marvel Comic Wolverine can repair his wounds in the movies. One interesting fact which has been known for several decades, but has not been able to be investigated more deeply until now, is the innate ability for the adult lung to regrow lost lung tissue without any sort of intervention. My thesis focuses on defining the molecular mechanisms and stem cell niches that allow for this normal, healthy adult lung tissue growth. The working hypothesis is if we can understand what makes a cell undergo healthy tissue proliferation and differentiation, we could stimulate this response to cure individuals who suffer from diseases such as chronic obstructive pulmonary disease (COPD). Similarly, if we understand how a cell decides to respond in a diseased way, we could stop or revert the disease process from occurring.

One of the models we use in our lab is a “pneumosphere” culture. We essentially grow alveoli, which are the site of gas exchange in the lung, in a dish to attempt to understand how specific alveolar stem cells signal and interact with one another. This information will teach us how these cells behave so we can in turn either promote a healthy response to injury or, potentially, stop the progression of unhealthy cell responses. The technique of growing alveoli in a dish allows us to cut down on the “noise” and focus on major cellular pathways, which we can then more selectively apply to our mouse model systems.

Pneumospheres. (Photo by Matt Donne)

Pneumospheres or “lung cells in a dish”. (Photo by Matt Donne)

Lung cells.

Lung pneumospheres under a microscope. (Photo by Matt Donne)

We are now in the process of submitting a paper demonstrating some of the molecular players that are involved in this regenerative lung response. Hopefully the reviewers will think our paper is as awesome we as believe it to be.

Q: How has being a CIRM scholar benefited your graduate research career?

MD: Starting in my second year at UCSF, I was awarded the CIRM fellowship. I think it helped the lab to have the majority of my stipend covered through the CIRM fellowship, and personally I was very excited about the $5,000 discretionary budget. These monies allowed me to go to conferences every year for the past three years, and also have helped to support the costs of my experiments.

The first conference I attended was a Gordon Conference in Italy on Developmental Biology. There I was able to learn more about the field and also make friends with many professors, students, and postdocs from around the world. Last year, I went to my first lung-specific conference, and attended again this year. That has been one of the highlights of my PhD career. While there, one is able to speak and interact with professors whose names are seen in many textbooks and published papers. I never thought I would be able to so casually interact with them and develop relationships. Since then, I have been able to work on small collaborations with professors from across the US.

It was great that I could go to these conferences and establish important relationships with professors without being a major financial burden to my Professor. Plus, it has been hugely beneficial for my career as I now have professors whom I can reach out to as I look towards my future as a scientist.

Q: What other benefits did the CIRM scholars program provide you?

MD: Dr. Susan Fisher has been in charge of the CIRM program at UCSF. She organized lunch-time research talks that involved both academic as well as non-academic leaders in the field. I enjoyed the extra exposure to new fields of stem cell biology as well as the ability to learn more about the start-up and non-academic world. There are not many programs that offer this type of experience, and I felt fortunate to be a part of it. Also, the free lunches on occasion were a nice perk for a grad student living in San Francisco!

I attended the CIRM organized conferences whenever they happened. It’s always great presenting at or attending poster sessions at these events, seeing familiar faces and meeting new people. I took full advantage of the learning and networking that CIRM allowed me to do. The CIRM elevator pitch competition was really cool too. I didn’t win, came in third, but I enjoyed the challenge of trying to break down my thesis project into a digestible one-minute pitch.

Q: Where do you see the field of lung biology and regenerative medicine heading?

MD: My take away from the research conferences I have attended with the help of CIRM-funding is that we are in a very exciting time for lung stem cell research. The field overall is still young, but there are many labs across the world now working on a “lung mapping project” to better define stem cell populations in the lung. I see this research in the future translating in to regenerative therapies by which diseased cells/tissue will be targeted to actually stop the disease progression, and in turn possibly repair and regenerate healthy new tissue. This research has wide reaching implications as it has the potential to help everyone from a premature baby more quickly develop mature healthy lungs, to adults suffering from COPD brought on by environmental factors, such as air pollution. As many scientists are often quoted, “This is a very exciting time for our field.”

Q: What are your future plans?

MD: I expect to graduate in about a year’s time. In the future, I want to pursue a career focusing on the social impact of science. I aspire to be someone like UCSF’s former chancellor Dr. Susan Desmond-Hellmand. It’s really cool to go from someone who was the president of product development at Genentech, to chancellor at UCSF, to now president of the Bill and Melinda Gates Foundation. Bringing science to impact society in that way is what I hope to do with my future.


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