Monday, July 30, 2012

Translations bring stem cell information to the world

How do you say “what are stem cells ” in Portuguese? How about Turkish or even Hindi and Vietnamese? I had no idea either, at least not until I turned to Prof. Paul Knoepfler’s stem cell blog.

As regular readers of this blog know Prof. Knoepfler is a stem cell researcher at the UC Davis Institute for Regenerative Cures. He’s also an inveterate blogger and now, in his spare time, he’s trying to translate a fact sheet on stem cells into as many languages as possible. It’s for a project he calls the Stem Cell Outreach Program for Education or SCOPE.

Why is he doing this you might ask? Well, it’s simple. Stem cell research is of global importance. The therapies it produces stand to benefit people around the world so it just makes sense that we have materials for people around the world to read now, giving them a basic understanding of what this work is and why it’s so important. That reasoning is what led us to translate our seven pages of stem cell basics into Spanish (you can see those pages here).

So far SCOPE has translated the “What is Stem Cell” fact sheet into 12 different languages. But they want a lot more, and they’re asking for your help in getting them. They need volunteer translators, people who have a good grasp of a language and a good grasp of science too, to translate the fact sheet into many more languages.

Here are the ones they’re most interested in right now: Arabic, German, Italian, French, Hebrew, Bengali, Korean, Turkish, Urdu, and Tagalog.

So, if you are fluent in any of those languages and are interested in being part of this program drop Prof. Knoepfler a line at

You could help spread the word about stem cells, or as they say in Portugal, celulas tronco.


Friday, July 27, 2012

Patient advocates speak in support of disease teams, new therapies

Huntington's disease advocates speaking to the Governing Board
Yesterday’s governing board meeting should have been a happy one. The agency awarded $150 million dollars to fantastic teams of researchers whose work could result in therapies for some of the worst diseases – Huntington’s disease, Lou Gehrig’s disease, melanoma and heart disease, to name a few.

But that happiness was bittersweet. The reason we need to fund research is because there are currently no cures for those diseases. And for the people living with the diseases, the results of the research we funded might come too late.

For me, the hardest part of the day by far was hearing from those families whose children—almost exclusively boys—have muscular dystrophy. These are parents who saw their happy, healthy, active boys start to weaken at around age five. These parents will inevitably lose their children to the disease unless there is a cure.

My own happy, healthy, active boys are five and seven, exactly the ages many of those parents described. As those parents were before the diagnosis, I’m looking forward to seeing my kids grow up, have fun, find careers and start families. Instead, those families bought their kids wheelchairs and have watched them grow weaker and more dependent.

Later in the meeting a woman in a wheelchair spoke about raising her three children on her own and being diagnosed with Lou Gehrig’s disease (properly known as ALS). That disease is also inevitably fatal.

One woman who is going blind with retinitis pigmentosa spoke about wanting to see her husband’s face.

Things most of us take for granted—seeing our loved ones, hugging our kids—are the source of dreams for those who spoke at yesterday’s meeting.

Perhaps the most visible group of patient advocates at the meeting were the Huntington’s disease families who showed up en masse to support an application from Vicki Wheelock of the University of California, Davis. That community is so involved in driving new research that many have become old friends. They threw what might be the first confetti at a public state meeting when our governing board voted to approve funding for that award.

Their excitement wasn’t for a cure—even if Wheelock’s work is successful it will be many years before a treatment is widely available. It was for hope.

Speaking before the board, Katie Jackson, whose husband has Huntington’s disease said:
"When Mike was diagnosed, I couldn't believe there was nothing they could do for him. We need hope more than we can explain to you."
CIRM can’t know in advance which research projects are going to be successful. Some will inevitably not result in the hoped-for clinical trials. That’s the process of science—trying different approaches until you find one that works. All we can do is try to pick the best science and then do what we can help those teams succeed in turning the hope into a reality.

Here's our press release about the awards


Wednesday, July 25, 2012

CIRM grantees at the Gladstone Institutes launch powerful new videos

I’m always impressed by good science communication, especially in the field of stem cell research. That’s why I was excited to see the new video program unveiled this week by the Gladstone Institutes.

Gladstone, which is affiliated with UCSF, launched a new website featuring five videos produced by Wondros—an award winning company founded by Jesse Dylan, son of Bob Dylan. The videos each tell a short, compelling story about one area of research focus at the Institute, including stem cell research. (They’ve received more than $34 million in grants from CIRM for this work.)

A press release from Gladstone talks about the role of these videos:
“Gladstone scientists combine concern for patients with curiosity about the fundamentals of biology in a distinctive—and perhaps even unique—blend of motivation and drive,” said cardiologist R. Sanders “Sandy” Williams, MD, who became Gladstone’s president in 2010. “We expect that our new films and website will increase public awareness about the ongoing devastation caused by these diseases—and how Gladstone is making a difference.”

The movies and website are part of a Gladstone campaign to increase recognition of three global health crises—at least one of which impacts virtually every family worldwide.
The video about the program’s stem cell initiatives features stem cell legend Shinya Yamanaka, who first reprogrammed adult cells to an embryonic state, along with some of Gladstone’s top stem cell scientists. Together, they make a compelling case for the promise of their work. You can watch the stem cell video below. All the videos are available on the newly launched rebranded website.


Tuesday, July 24, 2012

Ask the stem cell expert: your questions answered on Alzheimer’s disease

A few weeks ago we asked people to submit questions for the first video in our Ask the Expert series. Well, you submitted questions and we asked stem cell expert Lawrence Goldstein, PhD, at the University of California, San Diego to answer them.

I’ve spoken with Dr. Goldstein in the past about his ALS research and about his involvement in preventing stem cell tourism. This was the first time we’d discussed his Alzheimer’s research. What I hadn’t known is that Goldstein’s mother had the disease, so he understands first-hand the devastating emotional and financial burden on the family.
It was just a terrible thing to go through. It was hard on my brothers, it was hard on me. It was very hard on her. And to be honest, I just got angry about it, you know, personally. And so, I've sort of resolved to do the best I can to hopefully develop something that will be meaningful in the lives of people who develop this terrible disorder.
When we started talking about the disease and why it’s so important to find a cure, one thing that struck me was the disproportionate amount of money that the disease costs compared to the amount being spent on finding a cure. In the video Goldstein says,
There’s a direct economic cost of about $200 billion per year in the United States and there’s probably an indirect economic cost of $200 or $300 billion would be a reasonable estimate so somewhere in the range of $400 to $500 billion a year in cost to the nation—all of us—and we only spend $500 million a year on research into finding what’s going wrong and finding a way to treat it. We’re outnumbered 1,000 to one.
In answer to a question about stem cell therapies for Alzheimer’s disease, Dr. Goldstein admitted that there are still a lot of unknowns in the field. His lab works on studying stem cells in a lab dish to better understand how the disease unfolds (we blogged about his work here). He’s hoping that research will result in a drug that can treat the disease. However, he says that the best way to find a cure is to have scientists trying many different approaches.
If somebody can figure out some way of reversing the course of this disease using stem cells themselves, great, I’m all for it. We have to do the work to find out—there’s no substitute for doing the research work.
Here is an exerpt of our conversation. We’ll be posting more answers as well as a full length version of the conversation on our YouTube channel.

Dr. Goldstein has several awards from CIRM, including a Comprehensive Award and an Early Translational III award focused on Alzheimer's disease, a Tools & Technology II Award to develop disease-specific stem cell lines and Disease Team Award working toward a therapy for ALS (Lou Gehrig's disease).


Monday, July 23, 2012

CIRM HIV/AIDS disease team making news

Paula Cannon, University of Southern California
Today brings more news from the AIDS conference being held this week in Washington D.C. The local San Francisco NPR affiliate did a story for their Quest science program about one approach to a possible disease cure. They interviewed Timothy Ray Brown, who is the first person to have the HIV virus eliminated from his body. He had a type of bone marrow transplant that can’t be applied to all people with the disease. In the interview he said:
I’m hoping that when and if there is a cure it can be administered to the entire world. I think that’s going to happen. I’m hoping for it.
They also spoke with Paula Cannon from the University of Southern California, who is part of a CIRM-funded disease team that’s developing a way of making Brown’s experience more widely available. She said:
What we’re talking about are new kinds of medicines. We certainly don’t have the processes in place to make this cheap and easy yet, but I happen to believe that that’s just an engineering problem. If this works there’s going to be such excitement about this – such a big effort into figuring out how to do it at scale, to do it cheaply, that we’re just going to invent whole different ways of doing medicine.
Cannon’s team expects to start testing their approach in clinical trials within the next few years. You can learn more about Cannon’s approach to treating HIV/AIDS in this video we produced with the team leader John Zaia at The City of Hope:

Here’s a summary of their project on the CIRM website.


Friday, July 20, 2012

2012 AIDS conference focuses on a cure

Next week begins the international AIDS conference, being held this year in Washington DC. CIRM’s board member Jeff Sheehy is there, in his role as long-time HIV/AIDS advocate.

For the first time, the news coming out in advance of the meeting is about cures, not long-term drugs. As the Washington Post reports, this is a sea change in how people think about the disease, driven in part by the experience of the man known as the Berlin patient.

Timothy Ray Brown received a bone marrow transplant in Berlin to treat his leukemia. But that transplant came from a person who had an unusual mutation that made his blood cells resistant to HIV. Once those cells repopulated Brown’s blood system, he too resisted the HIV virus that had once infected his cells.

That experience has driven HIV/AIDS researchers to mimic Brown’s experience. There aren’t enough donors who carry the appropriate mutations to cure all people. But through genetic manipulation and some stem cell know-how, two groups of CIRM grantees are working toward the goal of a cure.

The Washington Post story quotes Michel Sidibe, executive director of UNAIDS, the Joint United Nations Program on HIV and AIDS:
“The previous generation fought for treatment. Our generation must fight for a cure.”
There’s more information about the two CIRM teams on our website. I’ll be watching for news from the meeting about progress toward the ultimate goal of a world without HIV/AIDS.


Thursday, July 19, 2012

Confused about the many types of stem cells? Read on...

Induced pluripotent stem (iPS) cells reprogrammed from human skin
When I read about stem cells in the news it is often frustrating to see all stem cells lumped together as if they are the same. It gives the impression that what one stem cell can do, all can do. In fact, different types of stem cells have unique properties that make them especially suited to treating or understanding particular diseases.

Some, like embryonic stem cells, can make all the different tissues in the body. Others, like the stem cells of the blood, brain, skin and other organs, can only make a subset of cells. For example, blood-forming stem cells can become any type of cell in the blood, but they can’t make neurons or skin. Each of the different types of stem cell has potential to treat diseases, which is why CIRM funds them all. has posted a summary of the different types of stem cells that’s well worth a read. They conclude with this excellent explanation for why we need to pursue research with all types of stem cells:
Although there's been a lot of discussion about developing therapies with stem cells over the last several years, the science is still really just in its infancy. It is only in the last 10-20 years that we have had the genomic research tools to really start uncovering the mechanisms behind the process of differentiation. While it is clear this technology will have a major impact on health care in the years to come, at this point, there is no way to predict which lines of research will lead to the most promising treatments. Right now, it is important to push for more progress on all fronts.
CIRM has some addition background information about the different kinds of stem cells and information on why we fund all types of stem cells. We also have charts showing the distribution of funding to different stem cell types.


Wednesday, July 18, 2012

Bridges students start stem cell research careers

Ebony Flowers, a Bridges student at California State University, Long Beach, talks about her stem cell research with other students
“I had never done any work or research in a lab before this, but I loved it from the very first moment. It gives me hope that this is something I can do for the rest of my life.”
For California State University, Long Beach student Ebony Flowers, an internship with the stem cell agency’s Bridges program was a life-changing experience. It introduced her to research in a world-class laboratory, and gave her a glimpse into a career she had never imagined before. Now, she’s getting ready to go straight from a biology degree into a PhD course at UT Southwestern in Dallas.

Michael Silva, a student at Cal State Channel Islands, had friends who were caught up in gangs and his life could have taken any number of bad turns before he discovered science and stem cells. Now he’s planning on heading to medical school and start a career in research.

There were many other stories like that at the Bridges Training Meeting held this week in San Francisco. The CIRM Bridges to Stem Cell Research Program (to give the program its full title) is designed to give student trainees the basic training, skills and inspiration they need to pursue careers in stem cell research.

Some of the top stem cell researchers in California were on hand to talk about their work, but also about the path that led them where they are, the uncertainty and challenges they encountered along the way. For the students it was reassuring to hear that the doubts and questions they have are the same as those once faced by leaders in their field.

But while the researchers were there to help inspire the students, many said they were inspired by the students, by their excitement and enthusiasm for their work. Students like Kashfia Neherin at Cal State San Bernardino. She came to the US three years ago from Bangladesh with a limited science background but an endless curiosity. Kashfia saw a poster about the Bridges program and found out she had to take a tissue culture class to apply. “I had never done anything like this before,” she says. “It was completely new to me, but I really enjoyed it and it got me hooked on research.”

Now Kashfia is a pre-med student but also has her sights set on a PhD. All because of that one class.

For the staff at the stem cell agency, it was also a wonderfully inspiring couple of days. The enthusiasm of the students is infectious, and it reminded us all why we do this work, why it’s important, and what our ultimate goal is. And it introduced us to the next generation of stem cell researchers who are going to help us get there.

Here's more information about the Bridges program and a map of the programs.


Monday, July 16, 2012

Bridge to the future – creating the next generation of researchers

We might be a little biased here but at the stem cell agency we consider stem cell research an important part of the future of medicine. So it’s no surprise that we consider our Bridges students an important part of the future of that research.

The CIRM Bridges to Stem Cell Research Awards fund courses and research internships for undergraduates and masters-level students. The goal is to train the next generation of stem cell scientists and laboratory technicians.

Over the next two days we’ll be holding a conference to bring together more than 150 of these students with some of the leading stem cell researchers from around California. It’s a chance for some of these students, who are already part of the program, to present results from the work they have done. For others it’s a chance to meet some of the people they hope to work for.

For those of us who work at the stem cell agency, it’s a chance to recharge our batteries, to get inspiration from the enthusiasm and excitement of these students for the work they are doing or hope to do.

Over the past few years we have invested more than $50 million in this program, helping more than 350 students (352 to be precise) to do great research at 57 different academic institutions and biotech companies around California.

It’s an investment in the future. We think it’s money well spent.

We’ll bring you some highlights of the meeting in a future blog. In the meantime, watch the video at the top of this blog for highlights from last year's meeting.


Wednesday, July 11, 2012

New funding initiative will speed disease research

A colony of iPS cells, courtesy of Kathrin Plath at the University of California, Los Angeles.
Yesterday was an exciting day at CIRM headquarters, especially for some of our science officers who have been hard at work putting together a new three-part funding initiative. We posted those requests for applications yesterday and expect to start receiving applications in September. (You can read those requests here.)

Admittedly, the initiative doesn’t have an exciting sounding goal: we’re planning to fund groups to create, store and distribute stem cell lines with disease characteristics.

I know, storerooms full of frozen cells doesn’t have the cache of a novel disease research. But wait: those frozen cells are more exciting than they might sound.

We’ve blogged about so-called disease-in-a-dish research, most recently in Parkinson’s disease and Huntington’s disease. The idea is that in order to develop therapies for complex diseases we need to know more about what goes awry in people who get the disease. Since it’s often difficult to directly study the brain cells of someone with, say, Alzheimer’s disease or autism, scientists have turned instead to reprogrammed stem cells.

The scientists take skin cells from people with the disease they want to understand, reprogram the cells to an embryonic-like state, then mature those cells into the ones they want to study. In the case of both Parkinson’s disease and Huntington’s disease, the resulting neurons show signs of the disease. Scientists can then study those cells to understand what goes wrong, and also expose those cells to different drugs in an attempt to find a drug that eliminates symptoms.

It’s important work and is already pointing to potential drugs for several diseases. CIRM’s goal is to help speed that critical work along by funding people to collect tissue from patients, create stem cell lines and store the cell lines. That way, if a scientist wants to study a particular disease, he or she can turn to CIRM’s repository of frozen cells and get to work.

We wrote about this initiative in our annual report. You can read that story here.


Tuesday, July 10, 2012

New approach to healing hearts gets approved for clinical trial

One of the goals of the stem cell agency is to bring promising therapies into clinical trials, to see if what works in the laboratory can work in people. It’s exciting but challenging work and, like all good science, it takes time. That’s why the news this week from Dr. Eduardo Marban at Cedars-Sinai Heart Institute was so encouraging.

Dr. Marban and his company, Capricor, have been given approval by the Food and Drug Administration for an Investigational New Drug (IND). CIRM provided Cedars-Sinai with a $5.5 million Disease Team Award that contributed to this research and helped move it from the lab to clinical trials.

The therapy uses Cardiosphere Derived Cells (CDCs) to reduce scarring and repair the damage caused to heart muscle by a heart attack. The cells are found in heart tissue and have the potential to change into a variety of different heart cell types.

As Ellen Feigal, MD, CIRM’s Senior Vice President for Research and Development, said in a news release that we issued, “This is the first time that research by a CIRM-funded Disease Team has resulted in an Investigational New Drug (IND) approval from the FDA, a critical step in testing promising therapies in patients.”

This may be the first time one of the Disease Teams we have funded has reached this point. But we’re confident that others will be following along soon.


Monday, July 9, 2012

On stem cell clinics, clinical trials, and the pace of progress

We recently blogged about a Houston Chronicle story revealing a troublesome Food and Drug Administration review of the Texas company Celltex Therapeutics. This is the company that treated Texas Gov. Rick Perry last year. And in previous posts we’ve written about concerns surrounding stem cell tourism – in which unregulated companies overseas promote unproven stem cell therapies. One of these companies in Korea was recently sued for fraud (you can read details of that case here).

These concerns are balanced by the very real hope on the part of patients that stem cell therapies will one day work for them and frustration over the pace of the field.

Harold DeMonaco recently wrote an interesting column in Heath News Review about regulation of stem cell therapies, the pace of progress and his advice to people who are seeking treatment. He writes:
For the moment, if a member of my family asked about seeking this treatment at a local clinic, I would advise them to either enter into a clinical trial or explore other options. I would want to make certain that the cells were handled properly, that I received my own stem cells and not someone else’s and that the effects of the treatment were being reviewed, so that a body of knowledge could be developed as to whether or not the treatment did more good than harm. Call me cautious. I think that the phrase, “Caveat Emptor,” is appropriate here.
This advice is similar to that of the International Society for Stem Cell Research, which advocates that people research any clinic advertising stem cell therapies before signing up. Their website has a list of what to look for when evaluating possible therapies.

CIRM and ISSCR co-hosted a public seminar about stem cell tourism. You can watch that symposium here.


Friday, July 6, 2012

Parkinson's neurons susceptible to toxins, preventible with some drugs stem cell studies show

A colony of iPS cells, courtesy of Kathrin Plath at the University of California, Los Angeles.
There’s a lot of talk these days about studying diseases and finding new drugs using stem cells. The idea is so compelling that CIRM has entered into a partnership with the NIH to create the kinds of cell banks that would be needed to carry out this work, specifically in Parkinson’s disease, Huntington’s disease, and ALS (Lou Gehrig’s disease).

Now, results of that work are starting to be published. The most recent paper in the July 4 issue of Science Translational Medicine and shows that of the many different mutations that can lead to Parkinson’s disease some also cause neurons to be more susceptible to certain kinds of toxins. Drugs that normally protect cells from damage effectively protected the neurons created from people who had one Parkinson’s disease mutation, but not another.

What this means is that people who get Parkinson’s disease for different genetic reasons might also respond differently to drugs. In a press release, the NIH suggests that these findings could be used to make sure people get the right drugs for their form of the disease.

The idea behind this and other studies hinges on the fact that there is no way to directly study the neurons of people with neurological diseases like Parkinson’s, Alzheimer’s or Huntington’s disease and many others. So instead, scientists take skin cells from people with the disease. Those skin cells contain the same mutations that led to the disease in the people’s neurons. They then convert those skin cells into embryonic-like iPS cells, and mature the iPS cells into neurons. Those neurons mimic the neurons of people who have the disease, and in many cases behave very differently in a lab dish compared to neurons generated from the skin of healthy people.

With these diseased neurons to study, scientists are for the first time starting to understand the origin of many previously mysterious neurological diseases. They can also start testing drugs to see which ones eliminate symptoms of the disease in the lab.

This study came out of a collaboration between the NIH’s National Institute of Neurological Disorders and Stroke and several other funding agencies including CIRM. A similar consortium of funders sponsored related work in Huntington’s disease that we wrote about last week.

CIRM considers this work so important that we’ll soon be releasing a new round of funding to support creating and banking iPS cells from people with a wide variety of diseases beyond the three in our NIH agreement. This CIRM press release describes that program. You can also read about our banking initiative in our annual report.


ResearchBlogging.orgCooper O, Seo H, Andrabi S, Guardia-Laguarta C, Graziotto J, Sundberg M, McLean JR, Carrillo-Reid L, Xie Z, Osborn T, Hargus G, Deleidi M, Lawson T, Bogetofte H, Perez-Torres E, Clark L, Moskowitz C, Mazzulli J, Chen L, Volpicelli-Daley L, Romero N, Jiang H, Uitti RJ, Huang Z, Opala G, Scarffe LA, Dawson VL, Klein C, Feng J, Ross OA, Trojanowski JQ, Lee VM, Marder K, Surmeier DJ, Wszolek ZK, Przedborski S, Krainc D, Dawson TM, & Isacson O (2012). Pharmacological Rescue of Mitochondrial Deficits in iPSC-Derived Neural Cells from Patients with Familial Parkinson's Disease. Science translational medicine, 4 (141) PMID: 22764206

Thursday, July 5, 2012

Alan Trounson discusses nanomedicine and progress toward stem cell therapies

CIRM president Alan Trouson has been in Sydney, Australia at the International Nanomedicine Conference learning about how nanotechnology might benefit stem cell researchers trying to develop new disease therapies.

Nanotechnology is, essentially, the study of really small materials. In nanomedicine, those really small materials are put to use to treat patients. Tiny particles can ferry drugs or proteins to the place in the body where they are needed, for example.

Trounson was interviewed by ABC in Australia about the intersection of nanomedicine and stem cell biology. He said scientists could attach proteins to the tiny nanoparticles, and then use those to direct stem cells to mature into the type of cell that’s needed to treat the disease. He said:
“You take these very, very tiny particles. You can label them with proteins. You can actually direct cells with these nanoparticles. You can get them to move around and change their very nature."
You can listen to the whole interview here.

Trounson also discussed his role in developing in vitro fertilization technology, which is now responsible for more than five million births.

Australia is one of the 20 countries, states and foundations that have signed collaborative funding agreements with CIRM. You can read more about our collaborative funding agreement program and see a list of CIRM grants that have leveraged partner funding on our website's collaborative funding page.


Tuesday, July 3, 2012

Stem cell essays pose questions about self and point to new uses for stem cells

CIRM grantee Paul Knoepfler of UC Davis has been blogging about stem cell science. As part of his interest in drumming up public engagement in science he recently held a stem cell essay contest and has posted the two winners to his blog.

In the 18 and under category he received an interesting and thoughtful piece from 14 year old Claire August of Winchester, MA. You can read her full essay here. She poses some questions about stem cell research, including this interesting take on what it means to have cells replacing those we’ve lost to disease or injury:
If someone were to acquire a body part that would change their abilities or the way the world saw them, say becoming more athletic, is that person really the same?

Are we the sum of our organs or would changing things about ourselves also change how we treat the world or how the world treats us?
In the end she concludes that the questions are worth tackling as they arise:
I believe that we should tackle these issues as they come and advance forward into exploring regenerative properties of stem cells. We deserve the choices that they someday might offer.
The older than 19 winner was Agnes Oshiro of Davis, CA, who wrote about the potential role of reprogrammed iPS cells in the field of artificial intelligence. Her full essay is here. In her piece Oshiro suggests that divisions between university departments stand in the way of innovative collaborations, such as those between stem cell scientists and artificial intelligence. She writes:
However, the desire for innovation that runs in the scientific community as a whole will undoubtedly bridge any and all differences, in the search for a better, smarter, more powerful artificial intelligence.