Peering inside the brain: how stem cells could help turn skin into therapies for dementia

To truly understand a disease you need to be able to see how it works, how it causes our body to act in ways that it shouldn’t. In cancer, for example, you can take cells from a tumor and observe them under a microscope to see what is going on. But with diseases of the brain it’s much harder. You can’t just open someone’s skull to grab some cells to study. However, now we have new tools that enable us to skip the skull-opening bit, and examine brain cells in people with diseases like dementia, to see what’s going wrong, and maybe even to get some ideas on how to make it right.

AF_neuronTHMito(2)_webThe latest example of this comes from researchers in Belgium who have developed a new strategy for treating patients with an inherited form of dementia. They used the induced pluripotent stem cell (iPSC) method, taking take skin cells from patients with frontotemporal dementia, and turning them into neurons, the kind of brain cell damaged by the disease. They were then able to study those neurons for clues as to what was happening inside the brain.

The study is reported in the journal Stem Cell Reports, and in an accompanying news release the senior author, Catherine Verfaillie, says this approach allows them to study problems in the brain in ways that weren’t possible before.

“iPSC models can now be used to better understand dementia, and in particular frontotemporal dementia, and might lead to the development of drugs that can curtail or slow down the degeneration of cortical neurons.”

The researchers identified problems with a particular signaling pathway in the brain, Wnt, which plays an important role in the development of neurons. In patients with frontotemporal dementia, the neurons weren’t able to mature into cortical neurons, which play a key role in enabling thought, perception and voluntary movement. However, by genetically correcting that problem they were able to restore the ability of the neurons to turn into cortical neurons.

Philip Van Damme, a lead researcher on the project, says this may open up possible ways to treat the problem.

“Our findings suggest that signaling events required for neurodevelopment may also play major roles in neurodegeneration. Targeting such pathways, as for instance the Wnt pathway presented in this study, may result in the creation of novel therapeutic approaches for frontotemporal dementia.”

A look at 2014: some of the lowlights of stem cell research this past year

It’s been quite a year in stem cell research. Here at the stem cell agency eight projects that we are funding have been approved for clinical trials and several more hope to get approval in early 2015. And Dr. Don Kohn and his team at UCLA announced that they have effectively cured Severe Combined Immunodeficiency or SCID  a fatal disease that leaves infants with no immune system.

But the news hasn’t been all good. A number of high profile retractions of studies published in prestigious journals have drawn attention to some of the less lovely aspects of science. There are many reasons why a researcher or scientific journal decides to retract a study – falsified data, inability of others to reproduce the findings etc. – but the end result is always the same, a stain on the reputation of science in general.

Of course the only thing worse than a retraction is bad science that is not retracted. That’s why websites such as Retraction Watch are so important. They keep an eye on the field and help draw attention to questionable papers (in all areas of science, not just stem cell research).

Ivan Oransky of Retraction Watch

Ivan Oransky of Retraction Watch

The two founders of the site, Evan Marcus and Ivan Oransky, do a remarkable job of highlighting work that doesn’t stand up to closer scrutiny. This year they worked with the magazine Science to highlight The Top 10 Retractions of 2014.  Sadly, two of the top 10 – including the number one story of the year – concern stem cell research.

The list is a reminder, as we look forward to 2015 for more progress in the field, that we need to always check the credibility of studies or sources we are using. Sometimes something that seems too good to be true, is too good to be true.

Tomorrow, we’ll take a look at the flip side of this discussion, the “Biggest Scientific Breakthroughs of 2014”. It’s always good to end the year on a positive note.

Maintaining the momentum: a good start but CIRM 2.0 is just the first step

Sir Isaac Newton

Sir Isaac Newton

Newton’s First Law of Physics states that an object either remains at rest or continues to move at a constant velocity unless acted upon by an external force. Well, for the stem cell agency the external force was an exercise in thinking differently about how we do business. That resulted in our governing Board approving CIRM 2.0 yesterday. And we intend to keep that momentum going for as long as we can.

CIRM 2.0 is a streamlined process that will make it easier and faster to apply for funding from the stem cell agency, and is designed to attract high quality clinical stage projects that are ready to start within 45 days of being approved for funding.

As our President and CEO Dr. C. Randal Mills said in a news release:

“Our mission is to accelerate the development of stem cell treatments for patients with unmet medical needs. With many of these diseases, time lost waiting for a treatment means lives lost. We must continue to find new and innovative ways to speed up our process and make it easier to get promising therapies into clinical trials, and to give them all the support they need to be successful. That’s why we undertook this radical overhaul of the way we do business.”

In the past it could take up to two years for a researcher or company to move from applying for funding to getting the money as part of an approved contract. CIRM 2.0 simplifies and accelerates the process, cutting that two years down to just four months. And instead of just one single round of funding with an application deadline every 12-to-18 months, CIRM 2.0 will have an open application process for clinical stage programs with deadlines every month. That means companies and researchers can apply when they are ready and won’t have to try and rush an application in prematurely, for fear it could be another year or more before the chance comes around again.

It’s a big change in the way we work and as Dr. Mills told the Board at yesterday’s meeting, there are bound to be problems:

“There will be bumps in the road, you can’t make radical changes of this nature and scope without running into problems. I know that, my team knows that and we are ready to handle whatever unforeseen consequences come up.”

We plan on monitoring 2.0 as we unveil it, constantly checking to see what’s working and to fix what isn’t. In the short term we will use several measures of how well it’s working such as how many high quality applications we get, how quickly we can move these applications through the approvals process and how long it takes to get successful applicants their money. In the long term the best indication of success will be the quality of the programs we fund and how well they do in completing clinical trials.

This first phase of CIRM 2.0 will cover funding for clinical work but it will later be expanded to include discovery (also known as basic research) and translational research (moving promising discovery research to the clinic). But as Dr. Mills says, even while we are implementing CIRM 2.0 we are already thinking about the next step.

“Soon as this is done we have to start working on how we can improve CIRM 2.0 and keep that sense of urgency and innovation in front of us so that we always look to build a better product and fulfill our mission in a better way. Because there are many sick people out there looking to us for help and until that changes we need to be always looking to improve. Which is why as soon as CIRM 2.0 is done, we’re looking to create CIRM 3.0”

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.”

CIRM 2.0; saving time, saves lives

It’s been almost ten years since the voters of California approved Proposition 71, creating the Stem Cell Agency and giving us $3 billion to fund stem cell research. So this is an appropriate time to look back and see what we have done with the money so far, the progress that’s been made, and where we are heading in the next ten years.

Over the next few weeks we’ll be taking a more detailed look at all these elements – it’s too much to cover in one blog – but let’s start with where we’re heading. At yesterday’s meeting of our governing Board, the Independent Citizens Oversight Committee, President & CEO, C. Randal Mills, Ph.D. charted a course for future funding.

Randy Mills, Stem Cell Agency President & CEO

Randy Mills, Stem Cell Agency President & CEO

Right now it can take up to two years for a project going into clinical trials to apply for and get funding from us. As Randy said in a news release we issued after the meeting, “That’s just unacceptable”:

Under what he is calling CIRM 2.0, Randy is proposing to trim that down dramatically:

“We are going to shorten that to just 120 days. But we’re not just making it faster, we’re also making it easier for companies or institutions with a therapy that is ready to go into clinical trials to be able to get funding for their project when they need it. Under this new system they will be able to apply anytime, and not have to try and shoehorn their needs into our application process.”

The goal is not just to make it easier to apply for funding, but also to get more, high quality applications. Right now there is pressure on companies to apply before they are really ready because they know if they miss a current application deadline it could be another year to 18 months before another award cycle comes around. Under CIRM 2.0 application will be accepted on a monthly basis, so applicants won’t have to worry about missing one deadline – they can just apply the following month. Applying when they are ready will increase the likelihood that the projects will be and of high quality.

And as Randy points out, if it works at the clinical stage of funding, it can work at every stage:

“Speeding up the process, at all stages of research, just makes sense. The faster that researchers can get access to the funds they need to do their work, the faster they are likely to be able to produce something that helps patients.”

The speeding up of the process doesn’t just involve companies and researchers being able to apply anytime, it also means that when they do apply they’ll have to have all the supporting documentation and studies on hand to show they are ready to go as soon as the Board approves funding.

In the past there was often a delay of six months or even more after an application had been approved for funding while research milestones were negotiated and agreements signed. Because CIRM 2.0 will involve identifying milestones much earlier in the application process that delay will disappear.

This new approach involves a complete overhaul of the way we currently work but we think it’s worth it. We plan to start by introducing these changes for the projects that are furthest along, those ready to go into clinical trials, but in time we intend extending this to cover everything we fund.

Making these changes will help us trim a two-year process down to just three months. That means any therapy that proves successful is getting to the patients who need it much sooner than it otherwise would. And with many of the diseases we are targeting, saving time means saving lives.

Stem cell stories that caught our eye: Some good news got a little overplayed on blindness and Alzheimer’s

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.

Stories on blindness show too much wide-eyed wonder. While our field got some very good news this week when Advanced Cell Technologies (ACT) published data on its first 18 patients treated for two blinding diseases, many of the news stories were a little too positive. The San Diego Union Tribune ran the story from Associated Press writer Maria Cheng who produced an appropriately measured piece. She led with the main point of this early-phase study—the cells implanted seem to be safe—and discussed “improved vision” in half the patients. She did not imply their sight came back to normal. Her third paragraph had a quote from a leading voice in the field Chris Mason of University College London:

“It’s a wonderful first step but it doesn’t prove that (stem cells) work.”

The ACT team implanted a type of cell called RPE cells made from embryonic stem cells. Those cells are damaged in the two forms of blindness tested in this trial, Stargardt’s macular dystrophy and age-related macular degeneration, the leading cause of blindness in the elderly. Some of the patients have been followed for three years after the cell transplants, which provides the best evidence to date that cells derived from embryonic stem cells can be safe. And some of the patients regained useful levels of vision, which with this small study you still have to consider other possible reasons for the improvement, but it is certainly a positive sign.

CIRM funds a team using a different approach to replacing the RPE cells in these patients and they expect to begin a clinical trial late this year

Stem cells create stronger bone with nanoparticles.   Getting a person’s own stem cells to repair bad breaks in their bones certainly seems more humane than hacking out a piece of healthy bone from some place else on their body and moving it to the damaged area. But our own stem cells often can’t mend anything more than minor breaks. So, a team from Keele University and the University of Nottingham in the U.K. laced magnetic nanoparticles with growth factors that stimulate stem cell growth and used external magnets to hold the particles at the site of injury after they were injected.

It worked nicely in laboratory models as reported in the journal Stem Cells Translational Medicine, and reported on the web site benzinga. Now comes the hard step of proving it is safe to test in humans

Stem cells might end chronic shortage of blood platelets. Blood platelets—a staple of cancer therapy because they get depleted by chemotherapy and radiation—too often are in short supply. They can only set on the shelf for five days after a donation. If we could generate them from stem cells, they could be made on demand, but you’d have to make many different versions to match various peoples’ blood type. The latter has been a bit of a moot point since no one has been able to make clinical grade platelets from stem cells.

plateletsA paper published today by Advanced Cell Technologies may have solved the platelet production hurdle and the immune matching all at once. (ACT is having a good week.) They produced platelets in large quantities from reprogrammed iPS type stem cells without using any of the ingredients that make many iPS cells unusable for human therapy. And before they made the platelets, they deleted the gene in the stem cells responsible for the bulk of immune rejection. So, they may have created a so-called “universal” donor.

They published their method in Stem Cell Reports and Reuters picked up their press release. Let’s see if the claims hold up.

Alzheimer’s in a dish—for the second time. My old colleagues at Harvard got a little more credit than they deserved this week. Numerous outlets, including the Boston Globe, picked up a piece by The New York Times’ Gina Kolata crediting them with creating a model of Alzheimer’s in a lab dish for the first time. This was actually done by CIRM-grantee Lawrence Goldstein at the University of California, San Diego, a couple years ago.

But there were some significant differences in what the teams did do. Goldstein’s lab created iPS type stem cells from skin samples of patients who had a genetic form of the disease. They matured those into nerve cells and did see increased secretion of the two proteins, tau and amyloid-beta, found in the nerves of Alzheimer’s patients. But they did not see those proteins turn into the plaques and tangles thought to wreak havoc in the disease. The Harvard team did, which they attributed, in part, to growing the cells in a 3-dimensional gel that let the nerves grow more like they would normally.

The Harvard team, however, started with embryonic stem cells, matured them into nerves, and then artificially introduced the Alzheimer’s-associated gene. They have already begun using the model system to screen existing drugs for candidates that might be able to clear or prevent the plaques and tangles. But they introduced the gene in such a way the nerve cells over express the disease gene, so it is not certain the model will accurately predict successful therapies in patients.

Don Gibbons

Stem cell stories that caught our eye: fast track marketing in Japan, a 3D cell tour and autism

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.

Event showed great progress, but Japan nipping at our heals. The San Diego Union Tribune’s Brad Fikes seemed to be enjoying covering the Stem Cell Meeting on the Mesa in his own backyard in La Jolla. He stayed for the full two days of the industry Partnering Forum and when we chatted he said he had more good material than he could use. I was certainly willing to second the sentiment of the opening paragraph of the story he wrote:

“More than ever before, stem cell therapies appear poised to transform medicine — potentially curing heart disease, diabetes and paralyzing injuries, among other ailments.”

But the last portion of his piece was a little unsettling. He noted the frequent discussion at the meeting of Japan’s new fast track path for marketing stem cell therapies. The CEO of Athersys, one of the leading companies in the field, announced at the meeting that his company would be taking their lead product to Japan first for marketing, not the U.S.

Turning cell biology into a 3-D game. The ability to track cells has become one of the most important limiting factors in stem cell biology. We need to know where cells go when they are transplanted in the body, but even before that, we have found that the interaction of cells with their environment often determines if stem cell offspring do their jobs and we need to track cells to understand this.

Now a team at Drexel University that includes an expert in computer software and hardware used in gaming has

Researchers at Drexel touring a group of cells using 3D glasses.

Researchers at Drexel touring a group of cells using 3D glasses.

provided the field with an invaluable tool. They can label various cells with distinctive markers and follow their movements. More important they can use an elaborate software program to integrate individual slices of a tissue into a 3D sample that researchers can “tour” while wearing 3D glasses.

Red Orbit quoted Andrew Cohen the leader of the computer development team:

“It’s like Photoshop for cell biologists. The software outlines cells and blood vessels, keeping track of them as they’re dividing and moving around one another. This provides a wealth of information on the patterns of cell shape, motion and division. Visualization of the 3-D microscopy data together with the analysis results is a key step to measure and ultimately understand what drives these cells.”

Cally Templea, a leading expert from the Neural Stem Cell Institute in Rensselaer, NY, was also quoted about the power of this new tool to help stem cell biologists understand how stem cells interact with their environment:

“LEVER 3-D is amazing, it opens new vistas for understanding the stem cell niche.”

Autism linked to stem cell burst (in mice). The accelerated brain growth seen right after birth in many people with autism spectrum disorder has been linked to a burst of nerve stem cell division triggered by inflammation. The study at the University of California, Los Angeles, could explain why inflammation during pregnancy, either due to an autoimmune reaction or an infection, has been shown to be a risk factor for the disorder.

Health Canal posted the press release from the university that quoted the senior author of a paper in the journal Stem Cell Reports, Harley Kornblum:

“We have now shown that one way maternal inflammation could result in larger brains and, ultimately, autistic behavior, is through the activation of the neural stem cells that reside in the brain of all developing and adult mammals.”

The researchers gave pregnant mice a toxin found in bacteria and discovered that it triggered an excess production of nerve stem cells in their pups. This resulted in enlarged brains and behavior associated with autism, such as a reduced interest in interacting with other mice.

Little guy regrowing his head could help us. While a few species have the ability to regrow a severed body part, the tiny Hydractinia—commonly called snail fur—out does the rest in that it can regrow its head. BBC did a nice job of describing work at the University of Galway trying to explain how it accomplishes the feat and putting the work into perspective with other recent research findings.

After harvesting the creatures off the backs of hermit crabs the Galway team isolated embryonic stem cells from them, to which they attributed the ability to regrow something as complex as a head. The snail fur may be unique in that no other adult animal is believed to harbor embryonic stem cells. The researchers hope to use the tiny creature to learn how we might be able to turn on some ancestral regenerative capacity in humans.

Don Gibbons

Stem Cell Awareness Day and the Stem Cell Person of the Year

Today is Stem Cell Awareness Day, an event that seeks to bring together individuals and organizations around the world working to celebrate and raise awareness about the tremendous progress being made in stem cell research, and to ensure we remain focused on keeping that progress going until we have cures or treatments for people in need.

Paul Knoepfler

Paul Knoepfler

Today is also the day that our colleague, U.C. Davis stem cell researcher and avid blogger, Paul Knoepfler reveals the nominations for his annual Stem Cell Person of the Year award. It’s perfect timing, because many of those nominated have been pivotal in helping move the research forward, and in helping raise awareness about the field in general.

The rules for the competition are pretty simple. In Paul’s own words: “Who has been the single most important, influential person in the world of stem cells this year? Who has made the biggest positive contribution in 2014?”

This is the third year of the competition and Paul is not just a tireless champion of stem cell research, he’s also someone who puts his money where his mouth is. The prize for this year’s winner is $2,000, double that of the previous two years, and all that cash comes out of Paul’s own pocket. It’s a generous gesture from a tireless advocate. In fact those two qualities alone would suggest Paul qualifies to win his own competition. But he has too much integrity to ever even consider that.

So without further ado here is the list of nominations with quotes from some of the people who nominated them in green.

*Spoiler alert: a couple of those nominated are colleagues of mine here at the stem cell agency. I didn’t nominate them, but I can certainly testify to the fact that they deserve it.

Bernie Siegel – a long-time stem cell advocate who runs the yearly World Stem Cell Summit. “Has any one single person done more for the stem cell field?”

Chris Fasano: A principal investigator at the Neural Stem Cell Institute where he uses stem cells to study early nervous system development. Chris stands out for his energy, enthusiasm, dedication to the field, creativity and accomplishments.”

Diana DeGette, the Democratic Congresswoman from Colorado: a politician who has been working to pass important stem cell legislation. “Long time supporter of legislation to support stem cell research and regenerative medicine.”

Don Reed: long-time stem cell research advocate who played a key role in the success of Prop 71 and the creation of CIRM. “A tireless stem cell advocate always there to make a positive difference.”

Emmanuel (Ed) Baetge: Head of Nestlé Institute of Health Sciences at Nestlé Health Science and formerly CSO of Novocell/ViaCyte. “Ed was the driving force behind the development of Novocell (Viacyte’s) diabetes program using hESCs to develop a cell therapy for T1/2 patients…the phase 1 trial is enrolling this year. It has huge potential and Ed deserves the credit for his leadership and development of the technology as well as the company.”

Ellen Feigal; Vice President, Research and Development at the California Institute for Regenerative Medicine (CIRM). “At CIRM Dr. Feigal makes the Development Program happen.”

Ian mcNiece: Professor, Department of Stem Cell Transplantation,
Division of Cancer Medicine, The University of Texas MD Anderson
Cancer Center.

Janet Rossant: Senior Scientist in the Developmental & Stem Cell Biology Program and Chief of Research at The Hospital for Sick Children, Toronto

Jeanne Loring: Stem cell researcher and scholar, leading iPS cell clinical work in the pipeline for Parkinson’s Disease, and patient advocate. “Supportive to the advocacy community. Courage in supporting the challenge to WARF patents. Excellent scientist engaged in policy discussions.”

John Sinden: The co-founder of ReNeuron, a biotech company based in UK. “Thanks to his determination, and drive ReNeuron has a clinical approved stem cell product currently tested in two clinical trials.”

Judy Roberson: Long-time Huntington’s Disease patient advocate. “She makes concrete positive developments happen such as millions of dollars in research funding for HD.”

Leigh Turner: Outspoken advocate of evidence-based medicine in the stem cell field. “Gave an inspiringly frank talk about for-profit stem cell clinics flouting FDA regulations at ISSCR 2014.”

Malin Parmer: Associate Professor, Developmental and Regenerative Neurobiology, Lund University. Top neural regeneration scientist. “Young, hard worker who is doing very well”.

Masayo Takahashi: Stem cell researcher leading the team that is doing the first ever clinical study based on human iPS cells. “Creative and courageous clinical stem cell researcher.”

Mike West: He founded Geron and was CSO of Advanced Cell Technology before his current position as head of BioTime. “He has been a leader in our regen field for many years but he made a bold decision to resurrect Geron’s cell therapy for spinal cord injury this year and hopefully confirm the promise of hESC technology clinically.”

Patricia Olson: Executive Director of Scientific Activities at CIRM and active in CIRM scientific leadership from day 1. “A driving force in the stem cell field.”

Peter Zandstra: Professor at University of Toronto. Stem cell researcher. Canada Research Chair of Stem Cell Bioengineering. “Peter is a recognized pioneer and respected world leader at bringing concepts of scale-up, regulation, and industrialization to stem cell-derived cell therapy technologies.”

Pope Francis: Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.

Richard Cohen: An MS patient in a clinical trial who has chronicled his experiences. An author and Meredith Vieira’s husband, father of three children. “Mr. Cohen is an inspiration to me and others because he’s very honest and doesn’t sugar-coat his struggles, losses, frustrations, anger, embarrassments, and he also shares his gains.”

Richard Garr: CEO of Neuralstem, conducting work on using stem cells for ALS and advocate of Right To Try laws. “Richard does two excellent things at once: making a treatment for ALS and connecting with ALS patients.”

Robert Lanza: CEO of Advanced Cell Technology, which has multiple ES cell-based clinical trials ongoing. “Visionary and practical so makes the impossible possible”.

Shoukhrat Mitalipov: Stem cell researcher who first successfully made nuclear transfer human ES cells by therapeutic cloning and developing oocyte transfer-based therapies for mitochondrial disorders. “Gutsy pioneer of new, game changing technologies.”

Susan Solomon: Co-Founder and CEO of The New York Stem Cell Foundation (NYSCF). Remarkably effective advocate for stem cell research. “not many leaders have created their own research laboratories and raised $100 million plus. Seriously, what an accomplishment!”

Takaho Endo: Senior Researcher at the Riken Center for Integrative Medical Sciences in Yokohama who published key genetic work on the possible origin of STAP cells. “Courage to publish this being from RIKEN.”

Ted Harada: Leading stem cell research advocate and very effective ALS patient advocate. “An Energizer Bunny for the ALS community and stem cell advocate”

Tory Williams: Stem cell advocate and author of the 2014 book, Inevitable Collision. Co-Founder and Executive Director of the Alabama Institute of Medicine (AIM). “A true hero who inspires and makes concrete things happen like AIM”.