How partnering with someone half way around the world could help develop new treatments here in California

Much as we love California, and we really do, even we have to admit that genius knows no boundaries and that great scientific research is taking place all over the world. As our goal as an agency is to accelerate the development of successful therapies for people in need it only makes sense that we would try and tap into that genius, wherever it is, in whatever way we can. That’s where our Collaborative Funding Partnership (CFP) program comes in.

Michel Hivert, Executive Director at MATIMOP (L) and ICOC Chairman Jonathan Thomas

Michel Hivert, Executive Director at MATIMOP (L) and ICOC Chairman Jonathan Thomas

Under Proposition 71, the voter-approved initiative that created the stem cell agency, all the research we fund has to be in California. But that doesn’t mean we can’t help create collaborations between researchers here – that we fund – and researchers in other parts of the world who get funding from other sources. And we do just that. In fact we now have 24 CFPs stretching from New York state to Brazil, Japan, the UK and Australia.

And now we have added two more. One with Poland two weeks ago  and today, with Israel. As the Chair of our governing Board, Jonathan Thomas said in a news release , the goal of these agreements is simple, to advance stem cell research around the world:

“Israel has long had a robust stem cell research community. Through this newly announced collaboration, we hope to generate partnerships between Israeli and California scientists that build on our complementary strengths and generate joint research projects that will benefit patients everywhere.”

Dr. Andy David, Consul General of Israel to the Pacific North West, echoed those sentiments:

“It represents a practical expression of shared interests that is unusual for its depth and range. Israel and California are on opposite corners of the globe geographically, but they are practically coming closer every day. The reason for this thriving relationship is the understanding that we are strong mutual assets.”

But nice as these partnerships are the only questions that really matter are do these collaborations really make a difference; do they really help increase the likelihood of a successful therapy? The answer from our experience is yes. For example, a team we are funding at Stanford is collaborating with a team from the Medical Research Council in the UK, focused on solid tumor cancers. The Stanford team has been given approval by the Food and Drug Administration (FDA) to run a clinical trial testing this approach on solid tumors, while the UK team is using the same approach to tackling acute myeloid leukemia (AML) an often-fatal cancer of the blood and bone marrow. Knowledge gained from one trial may well benefit the other and could ultimately lead to approaches to treating other solid tumor cancers such as breast, ovarian, bladder and colon.

Disease does not stop at the border and we see no reason for our engagement with the best science, and the best scientists, to stop there either. Our goal is to find cures, and we’ll go wherever we have to and work with whoever we can to meet that goal.

 

 

 

 

Tune into Famelab: “American Idol” for scientists and engineers

I sometimes joke that I consider myself and my communications colleagues the “official translators” at the stem cell agency, trying to turn complex science into everyday English. After all, the public is paying for the research that we fund and they have a right to know about the progress being made, in language they can understand.

famelab

That’s why events like Famelab are so important. Famelab is like American Idol for scientists. It’s a competition to find scientists and engineers with a flair for public communication, and to help them talk about their work to everyone, not just to their colleagues and peers. Famelab gives these scientists and engineers support, encouragement and training them to find their voices, and to put those voices to use wherever and whenever they can; in the media, in public presentations, even just in everyday conversations.

Kathy Culpin works with the British Council to promote Famelab here in the US. She says it’s vitally important for scientists to be able to talk about their work:

“At the British Council we have worked with people who are doing amazing things but they can’t communicate to a broader audience. If scientists, particularly younger scientists, are unable to communicate effectively and clearly in a way that people want to listen to, in a way that people can understand, how are they going to have public support for their work, how are they even going to be able to raise funds for their work?”

The premise behind Famelab is simple: young up-and-coming scientists have just three minutes to present their research to a panel of three judges. They can’t use any slides or charts. Nothing. All they have is the power of their voice and whatever prop they can hold in their hands. For many scientists, taking away their PowerPoint presentation is like asking them to walk a tight rope without a safety net. It’s uncomfortable territory. And yet many respond magnificently.

Here’s Lyl Tomlinson, the winner of the most recent U.S. event, competing in the international finals. Appropriately enough Lyl’s presentation was on the role of running and stem cells in improving memory.

Famelab began in England but has now spread to 19 other countries. The competition starts at the regional level before progressing on to the national finals (April 2016) and then the international competition (June 2016, at the Cheltenham Science Festival in the UK).

In the U.S. there are a number of regional heats (you can find out by going here)

NASA helps run Famelab in the U.S. Daniella Scalice, the Education and Public Outreach Lead for the Astrobiology program at the agency, says Famelab is fun, but it has a serious side to it as well:

“We feel strongly that good communications skills are essential to a scientist’s training, especially for a Federal agency like NASA where we have a responsibility to the taxpayers to ensure they understand what their hard-earned dollars are paying for.  With FameLab, we hope to make learning best practices in communications easy and fun, and provide a safe environment for young scientists to get some experience communicating and meet other like-minded scientists.”

The next event in the U.S. is here in San Francisco on Monday, December 15 at the Rickshaw Stop at 155 Fell Street. Doors open at 6.30pm, competition starts at 7:30 P.

What is most fun about Famelab is that you never really know what to expect. One person will talk about the lifespan of the wood frog, the next will discuss the latest trends on social media. One thing is certain. It is always entertaining. And informative. And engaging. And isn’t that what science is supposed to be!

If you want to see how my colleagues and I at the stem cell agency tried to get stem cell scientists to develop sharper communication skills check out our Elevator Pitch Challenge.

Using stem cells paves new approach to treating a blistering skin disease

Imagine a child not being able to run or jump or just roll around, for fear that any movement could strip away their skin and leave them with open, painful wounds. That’s what life is like for children with a nasty genetic disease called epidermolysis bullosa or EB. The slightest touch can cause their skin to peel off. People with the disease often die in their late teens or early 20’s from skin cancer, caused by repeated cycles of skin wounding and healing.

Now Stanford researchers, funded by the stem cell agency, have found a way to correct the faulty gene and grow healthy skin, a technique that could completely change the lives of children with EB. This new approach, which the researchers call “therapeutic reprogramming”, is reported in the journal Science Translational Medicine

In the study the researchers took skin cells from patients with EB and reprogrammed them to become induced pluripotent stem (iPS) cells that have the ability to become any of the other cells in the body. They then replaced the faulty gene that caused that particular form of EB and then turned the cells into keratinocytes, the cells that make up most of our outer layer of skin. When they grafted these cells onto the back of laboratory mice they grew into normal human skin.

In a news release about the work, Dr. Anthony Oro, one of the senior authors of the paper, says the work represents a completely different approach to treating EB.

“Normally, treatment has been confined to surgical approaches to repair damaged skin, or medical approaches to prevent and repair damage. But by replacing the faulty gene with a correct version in stem cells, and then converting those corrected stem cells to keratinocytes, we have the possibility of achieving a permanent fix — replacing damaged areas with healthy, perfectly matched skin grafts.”

One of the key words in that quote is “healthy”. Because the skin cells that they got from the patient probably already included some that had a skin cancer-causing mutation, the researchers carefully screened the cells to make sure they removed any that looked suspicious.

Oro says tests showed the resulting skin from these iPS cells was very similar to human skin made from normal keratinocytes.

“The most difficult part of this procedure is to show not just that you can make keratinocytes from the corrected stem cells, but that you can then use them to make graftable skin. What we’d love to do is to be able to give patients healthy skin grafts on the areas that they bang a lot, such as hands and feet and elbows — those places that don’t heal well. That alone would significantly improve our patients’ lives. We don’t know how long these grafts might last in humans; we may need some improvements. But I think we’re getting very close.”

Having seen that this works in mice the team are now eager to see if they can replicate their results in people. With CIRM support they have already been working with the Food and Drug Administration (FDA) to pave the way for that to happen. Dr. Marius Wernig, one of the senior authors of the paper, says that focus on patients is driving their work:

“CIRM made sure that we were always keeping in mind the need to translate our results to the clinic. Now we’ve shown that this approach that we call ‘therapeutic reprogramming’ works well with human cells. We can indeed take skin cells from people with epidermolysis bullosa, convert them to iPS cells, replace the faulty collagen 7 gene with a new copy, and then finally convert these cells to keratinocytes to generate human skin. It is almost like a fountain of youth that, in principle, produces an endless supply of new, healthy skin from a patient’s own cells.”

Taking stock: ten years of the stem cell agency, progress and promise for the future

Under some circumstances ten years can seem like a lifetime. But when lives are at stake, ten years can fly by in a flash.

Ten years ago the people of California created the stem cell agency when they overwhelmingly approved Proposition 71, giving us $3 billion to fund and support stem cell research in the state.

In 2004 stem cell science held enormous potential but the field was still quite young. Back then the biology of the cells was not well understood, and our ability to convert stem cells into other cell types for potential therapies was limited. Today, less than 8 years after we actually started funding research, we have ten projects that are expected to be approved for clinical trials by the end of the year, including work in heart disease and cancer, HIV/AIDS and diabetes. So clearly great progress has been made.

Dean Carmen Puliafito and the panel at the Tenth Anniversary event at USC

Dean Carmen Puliafito and the panel at the Tenth Anniversary event at USC

Yesterday we held an event at the University of Southern California (USC) to mark those ten years, to chart where we have come from, and to look to where we are going. It was a gathering of all those who have, as they say, skin in the game: researchers, patients and patient advocates.

The event was held at the Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research. As Dr. Carmen Puliafito, Dean of USC’s Keck School of Medicine noted, without CIRM the building would not even exist.

“With this funding, our researchers, and researchers in 11 other facilities throughout the state, gained a dedicated space to hunt for cures for some of the most pernicious diseases in the world, including heart disease, stroke, cancer, diabetes, Alzheimer’s and Parkinson’s disease.”

Dr. Dhruv Sareen from Cedars-Sinai praised CIRM for creating a whole new industry in the state:

“What Silicon Valley has done for technology, CIRM is doing for stem cell research in California.”

One of the beneficiaries of that new industry has been ViaCyte, a San Diego-based company that is now in clinical trials with a small implantable device containing stem cell-derived cells to treat type 1 diabetes. ViaCyte’s Dr. Eugene Brandon said without CIRM none of that would have been possible.

“In 2008 it was extremely hard for a small biotech company to get funding for the kind of work we were doing. Without that support, without that funding from CIRM, I don’t know where this work would be today.”

As with everything we do, at the heart of it are the patients. Fred Lesikar says when he had a massive heart attack and woke up in the hospital his nurse told him about a measure they use to determine the scale of the attack. When he asked how big his attack had been, she replied, “I’ve never seen numbers that large before. Ever.”

Fred told of leaving the hospital a diminished person, unable to do most basic things because his heart had been so badly damaged. But after getting a stem cell-based therapy using his own heart cells he is now as active as ever, something he says doesn’t just affect him.

“It’s not just patients who benefit from these treatments, families do too. It changes the life of the patient, and the lives of all those around them. I feel like I’m back to normal and I’m so grateful for CIRM and Cedars-Sinai for helping me get here.”

The team behind that approach, based at Cedars-Sinai, is now in a much larger clinical trial and we are funding it.

The last word in the event was left to Bob Klein, who led the drive to get Proposition 71 passed and who was the agency’s first Chair. He said looking at what has happened in the last ten years: “it is beyond what I could have imagined.”

Bob noted that the field has not been without its challenges and problems to overcome, and that more challenges and problems almost certainly lie in the future:

“But the genius of the people of this state is reflected in their commitment to this cause, and we should all be eternally grateful for their vision in supporting research that will save and transform people’s lives.”

Ten at ten at the stem cell agency: sharing the good news about progress from the bench to the bedside

Ten years ago this month the voters of California overwhelmingly approved Proposition 71, creating the state’s stem cell agency, the California Institute for Regenerative Medicine, and providing $3 billion to fund stem cell research in California.

That money has helped make California a global leader in stem cell research and led to ten clinical trials that the stem cell agency is funding this year alone. Those include trials in heart disease, cancer, leukemia, diabetes, blindness, HIV/AIDS and sickle cell disease.

To hear how that work has had an impact on the lives of patients we are holding a media briefing to look at the tremendous progress that has been made, and to hear what the future holds.

When: Thursday, November 20th at 11am

Where: Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at the University of Southern California, 1425 San Pablo Street, Los Angeles, CA 90033

Who: Hear from patients who have benefited from stem cell therapies, the researchers who have done the work, and the key figures in the drive to make California the global leader in stem cell research

To listen in to the event by phone:

Call in: 866.528.2256  Participant code: 1594399

For more information contact: Kevin McCormack, Communications Director, CIRM kmccormack@cirm.ca.gov

Cell: 415-361-2903

How venture capital became a capital adventure for stem cell agency’s newest Board member

Kathy LaPorte, the newest member of the CIRM Board

Kathy LaPorte, the newest member of the CIRM Board

There’s something fascinating about looking at the arc of a person’s career. So often we start out thinking we are going to be one thing, and over the years we move in a different direction and end up doing something else entirely.

That’s certainly the case with Kathy LaPorte, the newest addition to our governing Board, the Independent Citizens Oversight Committee (ICOC).

Ms. Laporte started out with dreams of being a doctor and, after getting a biology degree at Yale University, she applied to go to medical school at both Stanford and Harvard (she was accepted at both, which tells you something about her ability). But somewhere along the way she realized that being a doctor was not for her and so she started thinking about other directions. The one she ultimately chose was business.

And she went about it in style. After gaining experience with a number of firms she teamed up with some colleagues to start New Leaf Venture Partners, a venture capital firm based in Silicon Valley.

A profile of her in the Silicon Valley Business Journal described her as “smart, thorough and solution-oriented, Ms. LaPorte has spent nearly her entire professional life in venture capital — something of a rarity — and is considered a quick study by those who have worked with her.”

But it’s not just her business acumen that earned her the respect of colleagues and an appointment to our Board by State Treasurer Bill Lockyer. It’s also her experience working in the biotech and healthcare field, evaluating and mentoring later stage biotech companies and early stage medical device and diagnostic companies.

“I’m honored to be joining the Board, and excited about CIRM’s mission to bring new regenerative medicine therapies to patients with chronic diseases,” says Ms. LaPorte. “I hope my experience from 28 years of helping to finance and guide the work of passionate scientists and entrepreneurs, enabling their ideas to get to the people who really need them, will be helpful to the CIRM team.”

In a news release announcing the news, Jonathan Thomas, the Chair of our Board, said:

“We are thrilled to have Kathy join us on the ICOC. As a representative of a life science commercial entity she brings with her a wealth of knowledge and expertise in biotech and business development for healthcare companies and products. Her keen intellect and analytical skills are going to be terrific assets for the Board.”

Ms. LaPorte’s career took a few twists and turns before it led to us, but we’re delighted it brought her here, and we welcome her to the Board.

Hands-on science turns kids heads

Making science fun. That was the goal of the Discovery Days event on Saturday in San Francisco, part of the Bay Area Science Festival. If numbers alone are any measure of success they certainly met their goal. The place was packed. But it was more than just the size of the crowd that demonstrated how successful the event was; it was also the makeup and enthusiasm of those there.

Using Play-Doh to explain the wonders of stem cells

Using Play-Doh to explain the wonders of stem cells

For five hours on a beautiful, sunny Saturday – when they could have gone anywhere and done anything – tens of thousands of people, parents and children, chose to come to Discovery Days and immerse themselves in science. And they clearly loved it.

There were more than 150 exhibits to choose from with a wide variety of topics to learn about – everything from climate change and exploring outer space to life in the ocean and everything in between.

In just the small section where the stem cell agency had its booth there were exhibits on DNA and genetics, the power of imagination, and a program designed to encourage more young women to pursue careers in engineering and orthopedics.

Each one chose a different way to engage the crowd, some used fancy high tech tools, others chose more basic approaches. At our booth we used Play-Doh to draw children to us where they could learn about cellular development. It’s always fun to see their eyes widen in amazement when you show them how we all began: as a single, solitary cell. And how that single cell quickly divides into many, eventually making up all the different types of cells that make us human.

The stem cell agency booth at Discovery Days at AT&T Park

The stem cell agency booth at Discovery Days at AT&T Park

The enthusiasm by kids and parents alike was infectious—children racing from one booth to the next, eager to see what each one had in store. Of course the fact that some booths wowed the parents as well as the kids didn’t hurt—but the bottom line was the science and the scientists, showing that it could be fun and fascinating and engaging. While not many parents got into the Play-Doh themselves, they spent considerable time talking with us about the progress in stem cell science.

When you look around and see so many children wearing big goggles, pretending to be scientists, it’s not hard to think of them years later, wearing those same goggles and no longer pretending but actually working as researchers—truly making the world a better place.

And ultimately that was the goal of the event, helping the kids find “something that will unleash their inner scientist.”

Discovery Days; bringing new life to the life sciences

Here are three words you don’t often see strung together: free, science, extravaganza. Yet that’s how Saturday’s Discovery Days at AT&T Park in San Francisco (home of the newly crowned baseball world champion Giants) is being described.

Robots on the rampage at last year's Discovery Days science fair

Robots on the rampage at last year’s Discovery Days science fair

The event truly is a celebration of science. It features more than 150 exhibits on everything from stem cells (that’s us) to rockets and robots and learning how your body and your brain work. It lets you learn about the world through interactive displays, games and experiments that engage and entertain.

Discovery Days is part of the Bay Area Science Festival. The Festival hopes that by making this a fun event it will encourage kids – and that’s the main audience here – to think about pursuing a career in science.

Parents and teachers are an important part of it too. The event gives them both ideas and tools on how to make learning about and teaching science more enjoyable, to help them get young people thinking about science outside the classroom, and to get them to understand that everything they see and do – from throwing a baseball to building a house – involves science.

Engaging the public in science is more than just an academic exercise. In recent years we have seen some fairly sizable cuts in funding for health, medical and scientific research in the US. These cuts are already slowing down our ability to do the research that can lead to new treatments for deadly diseases. Public support for scientific research is essential if we are to stop the cuts and increase funding. Events like Discovery Days can not only educate the public on how fascinating science is, but also how essential public funding for it is.

Bay Area Science Fair logo

So come along tomorrow (November 1) to Discovery Days. The event runs from 11am to 4pm and it’s FREE. It’s at AT&T Park (did I mention that’s the home of the newly crowned champions of baseball, the San Francisco Giants).

Here’s how you can get there

Blood Test Reveals Alzheimer’s Disease Risk, CIRM-Funded Study Finds

Could a simple blood test predict your risk for one day developing Alzheimer's disease?

Could a simple blood test predict your risk for developing one day developing Alzheimer’s disease?

By the time someone begins to experience the clinical symptoms of Alzheimer’s disease, the damage has already been done. An accumulation of toxic proteins is causing brain cells to whither and die, taking with them a lifetime of precious memories.

But what if we had a definitive test that could predict one’s risk of developing Alzheimer’s, even before the onset of symptoms? Could we use it to develop an early-detection method and—even more importantly—a way to slow or halt the disease before it is too late?

While this may seem closer to fiction than reality, scientists from the Western University of Health Sciences are reporting that they’ve done just that: a simple blood test that can accurately predict one’s Alzheimer’s risk—up to ten years before symptoms begin to develop.

Reporting in the latest issue of Translational Psychiatry, senior author Dr. Doug Ethell and his research team describe their ingenious method of tracking the earliest stages of Alzheimer’s via a simple blood test.

Their test, called the CD4see assay, tracks the body’s early immune response to toxic proteins—called amyloid beta proteins—that accumulate and form harmful plaques in the brains of Alzheimer’s patients.

Ethell has long been studying how a class of immune cells, called T cells, responds to the buildup of amyloid beta. Previously, he showed that these so-called amyloid beta-specific T cells could actually counter the cognitive decline seen in Alzheimer’s. So, lower amyloid T cell levels should correlate with symptoms. As he explained in an interview:

“If our mouse studies were correct, then there should be fewer of those cells in Alzheimer’s patients. Translating those studies from mouse to man was going to take a big effort—characterizing the small proportion of T cells that respond to amyloid-beta from the millions of other kinds of T cell would require technology that didn’t exist yet.”

So Ethell turned to stem cells. With support from CIRM, Ethell and his team took human embryonic stem cells (hESCs) and developed a type of immune system cell called dendritic cells. These cells stimulated the growth of amyloid-beta T cells—effectively bringing them out of hiding and allowing the researchers to locate and count them.

“Everyone showed a decrease in these T cells as they aged, but the decline occurred earliest in women with the apoE4 gene (the single greatest genetic risk factor for Alzheimer’s), often right around the same time as menopause,” explained Ethell. “When our raw data was pasted on foam boards all over my office it seemed to us that older women had lower responses than men, and when the data was finally plotted the dramatic decline around menopause was clear.”

Interestingly, this observation seems to correlate with the fact that Alzheimer’s is more prevalent in women than in men.

Ethell and his team propose that the CD4see assay could soon be used to measure amyloid-beta-specific T cells against one’s age, sex and whether they carry apoE4. This could then be used to calculate the individual’s risk for developing Alzheimer’s symptoms in the future.

This assay could also prove helpful when looking to test new therapeutic strategies that treat early-stage Alzheimer’s—something that has proven difficult without a reliable early detection method.

“Alzheimer’s disease is a puzzle and every bit of knowledge adds a new piece,” added Ethell. “We now view Alzheimer’s disease very differently than we did even just a few years ago.”

Spiderman Sets the Tone for Stem Cell Agency Board Meeting

I don’t often think about Spiderman at meetings of our governing Board – no, really I don’t – but yesterday was an exception. Not that I was daydreaming, rather I was listening to our new President & CEO C. Randal Mills, Ph.D., talk about his determination to set a very specific tone in leading the agency.

shutterstock_109433912

Randy had just explained to the Board that he had asked the agency’s General Counsel to draw up an agreement stating he – Randy, not the lawyer – will not accept a job with any company funded by CIRM for at least one year following his departure from the agency. In addition he will also refuse to accept gifts or travel payments from any company, institution or individual who receives agency funding.

In a news release we issued following the Board meeting he explained his reasons for making this commitment:

“I want the people of California to know that my sole interest in being at CIRM is to help advance stem cell treatments to patients who are in need. I will do so with a full commitment to transparency and by never compromising the integrity of our mission nor our trust to the taxpayers of California.”

And that’s where Spiderman comes in. As any fan of the movie or comic books can tell you one of the things Spiderman says a lot is “With great power comes great responsibility.”

In making his commitment Randy wanted to send a very clear and very strong message that he understands what his role as the President involves, and that it’s important for him to demonstrate that through his actions.

Board member and patient advocate, Sherry Lansing, echoed that saying:

“We take even the possibility of a perception of a conflict of interest very seriously and are determined to do whatever is necessary to ensure that we protect the reputation of the agency and the work that we do. We fully support Dr. Mills in the way he is handling this issue.”

Randy decided to make that commitment after his predecessor, Dr. Alan Trounson joined the Board of Stem Cells Inc., a company that we awarded more than $19 million to develop a therapy for Alzheimer’s disease. While there is nothing illegal about Dr. Trounson’s actions the news did cause a bit of a stir with a few commentators saying this was a dark mark against the agency – even though there is nothing we could have done to stop it because we did not know it was happening.

Randy is not asking anyone else to make the same commitment he has made, but he says it was important for him to do so. His role as President & CEO carries great responsibility and he says he wants to show that he takes it very seriously and will lead by example.

I rather think Spiderman would approve.

Kevin McCormack