Giving Thanks by Looking Forward


The CIRM Team gather to give Thanks

Thanksgiving is traditionally a time of reflection, a time to look back and express gratitude for all the good things that have happened in the past year. At CIRM we have a lot to be thankful for but this Thanksgiving we are looking forward, not backward. We’re unveiling our new Strategic Plan, our blueprint for the future, and we would love to hear what you think about it.

Randy Mills, our President and CEO, calls the Strategic Plan “a bold, new vision” for what we hope to achieve over the next five years. After reading it we hope you’ll agree.

Taking it on the road

We actually began this process several months ago with a talking tour of California. Randy Mills went around the state talking to researchers, academics, company officials, patients and patient advocates – anyone who has a stake in what we do. He posed a few simple questions such as: “what’s impeding progress?” and “how do you think we could do better?” and asked them what they thought we should focus on in the next five years.

As you can imagine we got a wide range of answers, but there was also surprising agreement on some key issues – such as the need to push for regulatory reform to help remove some of the obstacles slowing down the ability of researchers to move their therapies into clinical trials.

Bold ideas

The plan is an ambitious one, but then as Sherry Lansing, the cancer Patient Advocate member of our Board, says in a news release, why aim low:

“As we enter what could be CIRM’s last phase, we want to show the people of California that we are doing everything we can to fulfill the hopes of all those who voted to create the agency when they supported Proposition 71 in 2004.  That’s what this Strategic Plan demonstrates. It’s an ambitious plan, but you never achieve anything worthwhile by playing it safe. Too many lives are at stake for us to do anything less than work as hard as we can, as long as we can, to achieve as much as we can.”

Over the course of the next five years we hope to:

  • Launch 50 new clinical trials covering at least 20 unique diseases or conditions, and including at least 10 rare and 5 pediatric indications
  • Increase the number of projects advancing to the next stage of development by 50%
  • Work with patient advocates, the FDA and researchers to develop a new, more efficient regulatory process for cell therapies
  • Reduce the time it takes a stem cell therapy to move from discovery into a clinical trial by 50%

But wait, there’s more

And that’s just a taste of what we are planning. For the full picture you need to check out the Strategic Plan. But as Randy Mills says, we don’t want you to just read it. This process began with us asking you for your thoughts. Now we want to end it the same way.

“Your input was invaluable in helping us chart an ambitious course and giving us the inspiration to be bold and think outside of the box. Now, as we get ready to put this new vision for the agency into action, we want to share it with the public, with patients and patient advocates, scientists and researchers, and give them a chance to let us know what they think.”

Here’s where you can find the Strategic Plan.

What do you think?

If you have any thoughts or comments send them to me by 5pm, Thursday, December 3rd at

The Strategic Plan is due to go before the CIRM Science Subcommittee on Monday, November 30th and the full Board for its approval on Thursday, December 17th.


Stem cells could offer hope for deadly childhood muscle wasting disease

Duchenne muscular dystrophy (DMD) is a particularly nasty rare and fatal disease. It predominantly affects boys, slowly robbing them of their ability to control their muscles. By 10 years of age, boys with DMD start to lose the ability to walk; by 12, most need a wheelchair to get around. Eventually they become paralyzed, and need round-the-clock care.

There are no effective long-term treatments and the average life expectancy is just 25.

Crucial discovery

Duchenne MD team

DMD Research team: Photo courtesy Ottawa Hospital Research Inst.

But now researchers in Canada have made a discovery that could pave the way to new approaches to treating DMD. In a study published in the journal Nature Medicine, they show that DMD is caused by defective muscle stem cells.

In a news release Dr. Michael Rudnicki, senior author of the study, says this discovery is completely changing the way they think about the condition:

“For nearly 20 years, we’ve thought that the muscle weakness observed in patients with Duchenne muscular dystrophy is primarily due to problems in their muscle fibers, but our research shows that it is also due to intrinsic defects in the function of their muscle stem cells. This completely changes our understanding of Duchenne muscular dystrophy and could eventually lead to far more effective treatments.”

Loss and confused

DMD is caused by a genetic mutation that results in the loss of a protein called dystrophin. Rudnicki and his team found that without dystrophin muscle stem cells – which are responsible for repairing damage after injury – produce far fewer functional muscle fibers. The cells are also confused about where they are:

“Muscle stem cells that lack dystrophin cannot tell which way is up and which way is down. This is crucial because muscle stem cells need to sense their environment to decide whether to produce more stem cells or to form new muscle fibers. Without this information, muscle stem cells cannot divide properly and cannot properly repair damaged muscle.”

While the work was done in mice the researchers are confident it will also apply to humans, as the missing protein is almost identical in all animals.

Next steps

The researchers are already looking for ways they can use this discovery to develop new treatments for DMD, hopefully one day turning it from a fatal condition, to a chronic one.

Dr. Ronald Worton, the co-discoverer of the DMD gene in 1987, says this discovery has been a long-time coming but is both welcome and exciting:

“When we discovered the gene for Duchenne muscular dystrophy, there was great hope that we would be able to develop a new treatment fairly quickly. This has been much more difficult than we initially thought, but Dr. Rudnicki’s research is a major breakthrough that should renew hope for researchers, patients and families.”

In this video CIRM grantee, Dr. Helen Blau from Stanford University, talks about a new mouse model created by her lab that more accurately mimics the Duchenne symptoms observed in people. This opens up opportunities to better understand the disease and to develop new therapies.






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.

How do you know if they really know what they’re saying “yes” to?

How can you not love something titled “Money, Mischief and Science.” It just smacks of intrigue and high stakes.

And when the rest of the title is “What Have We Learned About Doing Stem Cell Research?” you have an altogether intriguing topic for a panel discussion.

Sue and Bill Gross Hall: Photo by Hoang Xuan Pham/ UC Irvine

Sue and Bill Gross Hall: Photo by Hoang Xuan Pham/ UC Irvine

That panel – featuring CIRM’s own Dr. Geoff Lomax, a regular contributor to The Stem Cellar – is just one element in a day-long event at the University of California, Irvine this Friday, November 13.

Super Symposium

The 2015 Stem Cell Symposium: “The Challenge of Informed Consent in Times of Controversy” looks at some of the problems researchers, companies, institutions and organizations face when trying to put together a clinical trial.

In many cases the individuals who want to sign up for a clinical trial involving the use of stem cells are facing life-threatening diseases or problems. Often they have tried every other option available and this trial may be their last hope. So how can you ensure that they fully understand the risks involved in signing up for a trial?

Equally important is that many of the trials now underway now are Phase 1 trials. The main goal of this kind of trial is to show that the therapy is safe and so the number of cells they use is often too small to have any obvious benefit to the patient. So how can you explain that to a patient who may chose to ignore your caveats and focus instead on the hope, distant as it may be, that this could help them?

Challenging questions

The symposium will feature experts in the fields of science, law, technology and ethics as they consider:

  • Does informed consent convey different meanings depending on who invokes the term?
  • When do we know that consent is informed?
  • What are human research subjects entitled to know before, during and after agreeing to participate in clinical trials?
  • How might the pushback on fetal tissue research impact the scientific development of vaccines, research on Alzheimer’s disease or other medical advancements?

So if you are looking for something thought provoking and engaging to do this Friday, here you are:

“The Challenge of Informed Consent in Times of Controversy,” Friday, Nov. 13, 9am – 4:30pm, at the Sue & Bill Gross Stem Cell Research Center on the University of California, Irvine campus.

The symposium will be livestreamed, and a video recording will be available on following the event.

REGISTER: The symposium is free to UCI student, staff and faculty. There is a $20 registration fee for non-UCI attendees. Visit the event page to register.

CRISPR cluster: How the media spotlight is focusing on gene editing tool

Illustration by Ashley Mackenzie: from New York Times Sunday Magazine

Illustration by Ashley Mackenzie: from New York Times Sunday Magazine

Getting in-depth stories about science in general, and regenerative medicine in particular, into the mainstream media is becoming increasingly hard these days. So when you get one major media outlet doing a really long, thoughtful piece about a potential game-changing gene-editing technology it’s good news. But when you get three major media outlets, all reporting on the same technology, all in the space of less than one week, and all devoting lots of words to the pieces, then it’s really a cause for celebration.

That’s what happened in the last few days with features on the gene editing technology CRISPR in the New York Times Sunday Magazine,  the New Yorker Magazine,  and STAT, a new online health and life-sciences publication produced by the Boston Globe.

Making the story personal

Feng Zhang: photo courtesy of the Broad Institute

Feng Zhang: photo courtesy of the Broad Institute

Each takes a similar approach, focusing on the individuals behind the new approach – Feng Zhang at Harvard/MIT and Jennifer Doudna at the University of California, Berkeley. The fact that the two are involved in a fight over patent rights for the process adds an extra element of friction to a story that already has more than its share of drama.

In the New Yorker, Michael Specter neatly summarizes why so many people are excited about this technology:

“With CRISPR, scientists can change, delete, and replace genes in any animal, including us. Working mostly with mice, researchers have already deployed the tool to correct the genetic errors responsible for sickle-cell anemia, muscular dystrophy, and the fundamental defect associated with cystic fibrosis. One group has replaced a mutation that causes cataracts; another has destroyed receptors that H.I.V. uses to infiltrate our immune system.”

Jennifer Doudna: Photo courtesy of

Jennifer Doudna: Photo courtesy of

Sharon Begley in STAT, writes that this discovery could bring cures to some of the deadliest health problems we face, from cancer to Alzheimer’s, but that it also comes with big ethical questions hanging over it:

“He (Zhang) has touched off a global furor over the possibility that a genetics tool he developed could usher in a dystopian age of designer babies.”

Jennifer Kahn in the New York Times Sunday Magazine follows up on that thought, writing about Doudna:

“But she also notes that the prospect of editing embryos so that they don’t carry disease-causing genes goes to the heart of CRISPR’s potential. She has received email from young women with the BRCA breast-cancer mutation, asking whether CRISPR could keep them from passing that mutation on to their children — not by selecting embryos in vitro, but by removing the mutation from the child’s genetic code altogether. ‘‘So at some point, you have to ask: What if we could rid a person’s germ line, and all their future generations, of that risk?’’ Doudna observed. ‘‘When does one risk outweigh another?’’

Each article makes for fascinating reading. Collectively they highlight why CRISPR is such a hot topic, on so many different levels, in science right now.

The topic is going to be the focus of a conference, featuring scientists from the US, Europe and China, being held at the National Academy of Sciences in Washington DC the first week of December.

CIRM is also getting involved in the debate and is holding a science-policy workshop on February 4th, 2016 in Los Angeles to consider the future use of genome editing technologies in studies sponsored by CIRM.

Glimpse the future at a fun-filled Festival of Science

Hands-on science and fun

Hands-on science and fun

Imagine a giant circus but instead of performing animals you have a Robot Zoo; instead of scary clowns you have colorful chemicals in glass beakers. That’s what AT&T Park will look like this Saturday when the 5th Annual Discovery Day opens its doors.  It’s a hands-on, eye-opening, brain-engaging celebration of science for everyone.

It’s a lot of fun

You’ll get a chance to learn about the science of sports – an appropriate subject as you’ll be doing it at the home of the 3-time World Champions of baseball, the San Francisco Giants. You’ll also be able to experience some of the training it takes to become an astronaut, without any of that pesky going-into-space business.

All in all you’ll be able to visit more than 150 hands-on exhibits and activities spread throughout the park, put together by the top science organizations, institutions and companies from all over the Bay Area. We’re talking Stanford University, UCSF, The Tech Museum, the Exploratorium, KQED, US Geological Society and the list goes on and on.

Meet the future right now

Today's scientists inspiring tomorrow's

Today’s scientists inspiring tomorrow’s

You’ll get to meet the scientists who are exploring outer space and the depths of the ocean, who are doing cutting edge research into health and who are pushing the boundaries of scientific knowledge.

And you will get a chance to meet us, the CIRM Team. We’re going to be there all day talking about the exciting progress being made in the field of stem cell research, and about the 15 clinical trials we are currently funding in heart disease, diabetes, cancer, HIV/AIDS and blindness (to name just a few).

You can find us on the Promenade level at booth P50. We’re easy to spot. We’re the coolest ones around. And if you have kids who enjoy PlayDoh, we will give them a chance to use the fun stuff to make stem cells.

But best of all Discovery Day is a chance for kids to learn how amazing science can be, to meet the scientists who are helping shape their future, and to consider a future as scientists themselves. And for the rest of us, it’s a chance to remind ourselves why we fell in love with science to start with.

And as if that wasn’t enough, the whole shebang is FREE.

The event is this Saturday, November 7 from 10am – 4pm. For details on where it is and how to get there – go to Discovery Day

Fun on the field at AT&T Park

Fun on the field at AT&T Park

The key to unlocking stem cell’s potential and blocking a deadly threat

A small slice of who you are - brain cells made from embryonic stem cells.

A small slice of who you are – brain cells made from embryonic stem cells.

Our bodies are amazingly complex systems. By some estimates there are more than 37 trillion cells in our bodies.  That’s trillion with a “t”. Each of those cells engages in some form of communication and signaling with other cells which makes our bodies one heck of a busy place to be.

Yet all this activity may owe much of its splendor and complexity to a relatively small number of starting materials. Key among those may be one protein which seems to act like a “master switch” and can determine if a cell changes and multiplies, or just stays the same.

Starting out

But let’s begin at the beginning. We all start out as a single fertilized egg that develops into embryonic stem cells, which in turn become adult stem cells, which then give rise to all the different cells and tissues and structures in our body – such as our bones and brains and blood.

But how do those cells know when to change, what to change into, and when to stop? Change too little and something is undeveloped. Change too much and you risk the kind of explosive uncontrolled multiplication of cells that you see in cancer.

So, clearly, knowing what controls those changes in stem cells, and learning how to use it, could have an enormous impact on our ability to use stem cells to treat a wide range of diseases.

What’s in a name, or a number

Now researchers at Mount Sinai have identified a single protein that appears to play a major role in this control process. The protein is called zinc finger protein 217 (ZFP217) and it controls the actions of genes that in turn control whether a cell changes into another kind of cell and how often it keeps dividing and multiplying.

The study is published in Cell Stem Cell  and there is some pretty complex science involved but ultimately what it boils down to is that ZFP217 has an impact on m6A (scientists really need to start coming up with more imaginative names) which is a protein that helps determine if a gene is turned on or off. If turned on the gene performs one function. If turned off it doesn’t.

By, in effect, blocking the action of m6A, ZFP217 is able to stop the process that would allow stem cells to differentiate, or change, into other cells and also ends their ability to keep renewing themselves.

But wait, there’s more!

One other important role that ZFP217 plays is in helping spur the growth of cancerous tumors. Too much of the protein allows these cells to multiply in an unlimited and uncontrolled fashion, typical of the kind of growth we see in tumors.

The study was done in mice but in a news release  the lead study author, Martin Walsh, PhD, talked about the possible significance of the findings for people:

“The hope is that ZFP217 could be used to maintain supplies of therapeutic stem cells. At the same time, as the human ZPF217 is associated with poor survival in a variety of cancers, understanding how this protein operates in physiological conditions may help to predict cancer risk, achieve earlier diagnosis and provide novel therapeutic approaches.”

Having a deeper understanding of what makes some stem cells multiply and change into other cells could enable researchers to better use stem cells to develop new approaches to treating some of the most intractable diseases of our time.

If that happens then ZFP217 might be a name to remember after all.

Embryonic and man-made stem cells are almost identical

iPS cell

iPS cell

Embryonic stem cells

Embryonic stem cells

For years it has been the stem cell equivalent of the feud between the Hatfields and McCoys. The dispute centered on the question of which is better for advancing scientific research and developing new therapies, embryonic stem (ES) cells or induced pluripotent stem (iPS) cells?

The somewhat surprising answer may be that they are both pretty much the same, at least according to a new study in the journal Nature Biotechnology.

For years many researchers felt that ES cells were better because they knew more about them and had a deeper understanding of how they worked. However, because ES cells are derived from human embryos they also carry ethical baggage for some people.

In contrast iPS cells are made from skin cells, or other adult cells, that have been reprogrammed to behave in an embryonic-like way and, like an ES cell, have the ability to turn into any other kind of cell in the body. However, some earlier studies had raised questions about iPS cells, after genetic variations were found in some batches, suggesting that the very act of creating the cells was causing potentially dangerous mutations.

This new study seems to calm those fears, confirming that these variations are due to differences that existed in the original skin cells that the iPS cells were made from, and did not arise during the reprogramming process.

The researchers, led by Konrad Hochedlinger of Massachusetts General Hospital in Boston, took two lines of ES cells and allowed them to mature into adult cells before reprogramming them into iPS cells. They were then able to analyze the iPS cells and measure gene activity in them to see how alike or different they were compared to the original ES cells.

They found that even though they were made from ES cells the iPS cells differed in some of their gene activity from their “parents”. However, when they examined if these variations changed the cells’ abilities to function they found no differences.

Equally important was the finding that ES and iPS cells were both equally good at being able to change into a variety of nervous system cells.

“The two cell types appear functionally indistinguishable based on the assays we used,” Hochedlinger said.

The magazine Science quotes two CIRM-funded scientists, neither of who were involved in the study, weighing in on its findings. Joseph Wu of Stanford said:

“I think it was a very well done study, and it will ease some of the concerns about ES cells versus iPS cells.”

William Lowry of the University of California, Los Angeles agreed:

“It’s probably going to be a lot easier to go forward with iPS cells because of studies like this.”

Science being science it is unlikely this one study will completely end the debate, and there are still many researchers who will prefer to use ES cells for their work because they are much more familiar with them. Nonetheless this may at least take some of the heat out of the debate and create an easier path for approval from the Food and Drug Administration (FDA) for researchers who hope to use iPS cell-based treatments in people.

Seeing is believing: using video to explain stem cell science

People are visual creatures. So it’s no surprise that many of us learn best through visual means. In fact a study by the Social Science Research Network found that 65 percent of us are visual learners.

That’s why videos are such useful tools in teaching and learning, and that’s why when we came across a new video series called “Reaping the rewards of stem cell research” we were pretty excited. And to be honest there’s an element of self-interest here. The series focuses on letting people know all about the research funded by CIRM.

We didn’t make the videos, a group called Youreka Science is behind them. Nor did we pay for them. That was done by a group called Americans for Cures (the group is headed by Bob Klein who was the driving force behind Proposition 71, the voter-approved initiative that created the stem cell agency). Nonetheless we are happy to help spread the word about them.

The videos are wonderfully simple, involving just an engaging voice, a smart script and some creative artwork on a white board. In this first video they focus on our work in helping fund stem cell therapies for type 1 diabetes.

What is so impressive about the video is its ability to take complex ideas and make them easily understandable. On their website Youreka Science says they have a number of hopes for the videos they produce:

“How empowering would it be for patients to better understand the underlying biology of their disease and learn how new treatments work to fight their illness?

How enlightening would it be for citizens to be part of the discovery process and see their tax dollars at work from the beginning?

How rewarding would it be for scientists to see their research understood and appreciated by the very people that support their work?”

What I love about Youreka Science is that it began almost by chance. A PhD student at the University of California San Francisco was teaching some 5th graders about science and thought it would be really cool to have a way of bringing the textbook to life. So she did. And now we all get to benefit from this delightful approach.

One man’s story points to hope against a deadly skin cancer

At our May Board meeting a gentleman presented his story, which exemplifies being a patient and patient advocate. His name is Norm Beegun. And this is his story.

Norm Beegun was treated in an early phase of the Caladrius trial.

Norm Beegun was treated in an early phase of the Caladrius trial.

Norm lives in Los Angeles. In 2002 he went to see his regular doctor, an old high school friend, who suggested that since it had been almost ten years since he’d had a chest x-ray it might be a good idea to get one. At first Norm was reluctant. He felt fine, was having no health problems and didn’t see the need. But his friend persisted and so Norm agreed. It was a decision that changed, and ultimately saved, his life.

The x-ray showed a spot on his lung. More tests were done. They confirmed it was cancer; stage IV melanoma. They did a range of other examinations to see if they could spot any signs of the cancer on his skin, any potential warnings signs that they had missed. They found nothing.

Norm underwent surgery to remove the tumor. He also tried several other approaches to destroy the cancer. None of them worked; each time the cancer returned; each time to a different location.

Decided to try a new approach

Then a nurse who was working with him on these treatments suggested he see someone named Dr. Robert Dillman, who was working on a new approach to treating metastatic melanoma, one involving cancer stem cells.

Norm got in touch with Dr. Dillman and learned what the treatment involved; he was intrigued and signed up. They took some cells from Norm’s tumor and processed them, turning them into a vaccine, a kind of personalized therapy that would hopefully work with Norm’s own immune system to destroy the cancer.

That was in 2004. Once a month for the next six months he was given injections of the vaccine. Unlike the other therapies he had tried this one had no side effects, no discomfort, no pain or problems. All it did was get rid of the cancer. Regular scans since then have shown no sign that the melanoma has returned. Theoretically that could be because the new therapy destroyed the standard tumor cells as well as the cancer stem cells that lead to recurrence.

Didn’t miss one of son’s football games

Norm says when you are diagnosed with an incurable life-threatening disease, one with a 5-year survival rate of only around 15%, you will try anything; so he said it wasn’t a hard decision to take part in the clinical trial, he felt he had nothing to lose.

“I didn’t know if it would help me. I didn’t think I’d be cured. But I wanted to be a guinea pig and perhaps help others.”

When he was diagnosed his son had just won a scholarship to play football at the University of California, Berkeley. Norm says he feared he would never be able to see his son play. But thanks to cleverly scheduling surgery during the off-season and having a stem cell therapy that worked he not only saw his son play, he never missed a game.

Norm returned to Berkeley on May 21st, 2015. He came to address the CIRM Board in support of an application by a company called NeoStem (which has just changed its name to Caladrius Biosciences). This was the company that had developed the cell therapy for metastatic melanoma that Norm took.

“Talking about this is still very emotional. When I got up to talk to the CIRM Board about this therapy, and ask them to support it, I wanted to let them know my story, the story of someone who had their life saved by this treatment. Because of this I am here today. Because of this I was able to see my son play. But just talking about it left me close to tears.”

It left many others in the room close to tears as well. The CIRM Board voted to fund the Caladrius application, investing $17.7 million to help the company carry out a Phase 3 clinical trial, the last hurdle it needs to clear to prove to the Food and Drug Administration that this should be approved for use in metastatic melanoma.

Norm says he is so grateful for the extra years he has had, and he is always willing to try and support others going through what he did:

“I counsel other people diagnosed with metastatic melanoma. I feel that I want to help others, to give them a sense of hope. It is such a wonderful feeling, being able to show other people that you can survive this disease.”