Happy social media day, stem cell fans

Mashable, the online go-to source for information on digital and social media, has declared today, June 30, to be social media day. We're celebrating with a who's who of those promoting stem cell science through social media and a comment on why social media is such a valuable tool for science education.

First, why social media? Many times I've had disbelievers tell me that there's no way to educate people about complex topics in 140 characters or less (Twitter) or through a sentence or two on Facebook. This is true. However, there's nothing like Twitter or Facebook for propagating interesting news articles, exciting discoveries or fascinating blog entries (ahem).

In today's media landscape there's a shortage of science reporters writing accurate, in-depth news stories. If, for example, Keith Darce at the San Diego Union Tribune writes something excellent about stem cell science I want all stem cell fans in Los Angeles, San Francisco, D.C., and internationally to see it. If I post a link to that article it goes to our ~700 Twitter followers and ~1,300 Facebook fans, but if those people "like" or retweet the link, it can easily be seen by tens of thousands of people. Not a bad return on 140 characters and a few minutes of my time, I'd say.

Here's the other thing. When I post to Facebook or Twitter the first people I'm talking to are those who are already following CIRM. It's important to keep our existing supporters informed — but it's also important to be reaching new people. Social media helps with that. I can't assume that everyone out there on Facebook, Twitter or reading blogs knows or cares about stem cell research. But many of those people care about diabetes, cancer, Alzheimer's disease, spinal cord injury or other disorders. If I write a blog entry about CIRM's diabetes awards and post it to Twitter, thousands of people who follow the word "diabetes" on Twitter will see it. (On Twitter you can follow words or topics, not just individuals.) And maybe because a person follows diabetes, they'll click my link and read my post and learn how CIRM-funded stem cell scientists are working toward a therapy for that disease. Education! In 140 characters or less.

Are you inspired? I'd love to see a few more stem cell advocates following CIRM and being a part of the online stem cell conversation. Here's a link to our Facebook page and Twitter account.

CIRM isn't alone in valuing social media. Many other California state agencies are keeping taxpayers in the loop through social media (here's a list), and other state or national stem cell organizations are quite active, most notably Missouri Cures, Texans for Stem Cell Research, New York Stem Cell Foundation, Irish Stem Cell Foundation, Scottish Stem Cell Network, Australian Stem Cell Centre, and the Canadian Stem Cell Network (which recently had an excellent blog post about the value of scientists communicating on the web). Patient advocates like Roman Reed also promote stem cell research to a wide fan-base online.

But the social media stem cell conversation isn't all backslapping among like-minded organizations. As Paul Knoepfler, a CIRM grantee at University of California, Davis, often points out on his excellent blog, those opposed to stem cell research are also active, and very organized in how they promote their views.

If you want to see research with all forms of stem cells and good national stem cell research policies, then celebrate Social Media Day by following your favorite stem cell or science organization and joining the conversation. Invite a few friends to join with you. (Have I mentioned our Facebook page and Twitter accounts?)

You can generally find Facebook and Twitter links on an organization's home page. Comment on posts, retweet articles you want people to read. It only takes a minute and can help spread the word about progress being made in stem cell science.

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Majority of Americans support using stem cells to treat serious disease

Amidst the political and media back and forth on the ethics and value of human embryonic stem cell research there's been little information available about what people actually think — For what purposes they find the use of embryonic stem cells appropriate and how they form those opinions.

This month a group of researchers from the University of Nevada published a Nature Biotechnology paper filling in some of those blanks.

Among the findings I found most interesting reported in a Nevada Today story was this quote from lead author Mariah Evans:

“The vast majority, over two-thirds, said that in deciding whether it is right to allow these treatments, they would follow their own judgment,” she said. “Only 4 percent gave greater moral weight to the Catholic Church than to themselves, and even among committed church-going Catholics, only about one in five defer to the church on these matters.”

The story sums up the report's key findings:

- More than two-thirds of respondents approved of using therapeutic cloning (nuclear transfer of the patient’s own genes) and stem cells from in vitro fertilized embryos to cure cancer or treat heart attacks, while only about one in six respondents did not approve. About one in six respondents had mixed feelings or was undecided.

- Over two-thirds of respondents also approved of a newer, less-researched method – using modified adult cells as an alternative to using cells from in vitro fertilized embryos – if the use could cure cancer or treat heart attacks. Less than 15 percent did not approve. About one in five had mixed feelings or was undecided.

- Almost half (43 to 47 percent) of respondents also approve of use of therapeutic cloning, stem cells from in vitro fertilized embryos and stem cells from an adult to treat allergies, but slightly over one in four do not. And, 28 to 29 percent have mixed feelings or undecided in this regard. These findings indicate that while more respondents approve of the use of these methods for treatment of less-serious conditions than disapprove of it, the approval is not as strong as it is for using these methods to treat more serious conditions and diseases, such as cancer or heart attacks.

- Respondents were not as approving of use of these methods for cosmetic purposes, such as creating new skin to restore someone’s youthful appearance. Almost one-half (45 to 50 percent) disapproved of this use, while only slightly more than one-quarter (25 to 29 percent) approved of this use. About one-quarter had mixed feelings or were undecided.

- Respondents did not support human reproductive cloning, neither of themselves nor of a child who died, with almost three-quarters (71 to 73 percent) disapproving and only about one in 10 approving. About one in five had mixed feelings or was undecided.

- Respondents were quite evenly divided in their thoughts on animal cloning with slightly over a third approving, slightly over a third disapproving, and about one-quarter having mixed feelings or being undecided.

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In vitro fertilization technique receives patent

Last December CIRM grantee Renee Reijo Pera spoke to the CIRM governing board about her work identifying which in vitro fertilization embryos were most likely to result in a successful pregnancy (you can watch that video here). That work has resulted in a patent to Stanford University, with an exclusive license to Menlo Park-based Auxogyn, which was founded by Pera and her colleagues at Stanford.

In the video of her talk, Pera shows several IVF embryos formed in the lab by fusing human sperm and eggs. By videotaping those embryos and watching them develop, she can tell by day two which are going to be ready to implant in a woman's uterus, a step that normally happens on day five.

Pera has a long-standing interest in the earliest stages of human development, where she says many common diseases may originate. During her talk, Pera, who is director of Stanford's Center for Human Embryonic Stem Cell Research and Education, said:

"I can’t believe the progress we’ve made in the past years with human embryonic stem cells and embryology. We have unprecedented tools to understand human development and we can begin to understand basic questions like where does sporadic disease come from in the population.”

This new technology has the potential to help infertile couples successfully conceive children through in vitro fertilization. Having a patent on the technology also creates jobs and tax revenue in California -- one of the great benefits of having a thriving biotechnology community in the state.

Pera has a CIRM Comprehensive Award and a New Cell Lines Award.

Origin of lung mucus glands found, insights for cystic fibrosis, asthma

Last week's big news at CIRM was the election of Jonathan Thomas as the new governing board chair, as we announced late Wednesday night. He will be replacing Robert Klein, who has served the agency since its inception in 2004. Not that anyone can replace Klein, exactly, but Thomas seems eager to step in and start leading the agency.

While many of us at CIRM were distracted by our board meeting and subsequent leadership change, CIRM grantees kept on doing science, as evidenced by a paper in Stem Cells which came out today.

Scientists with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have fund the stem cell that makes all the cells of the mucus glands in the airways of the lungs. By and large, scientists assume that most tissues of the body arise from a pool of tissue-specific stem cells. These stem cells have been identified in the blood system, brain, muscle, skin and a variety of other tissues. Once found, scientists can begin developing ways of harnessing those cells to treat disease.

Until assistant professor Brigitte Gomperts and postdoctoral scholar Ahmed Hegab published this work, nobody knew the origin of the mucus cells in the airway. These cells play a critical role in protecting the body from infectious agents or toxins in the environment. A UCLA press release quotes Gomperts:

“We’re very excited that we found this population of cells because it will allow us to study mechanisms of diseases of the upper airway. For example, there currently are no treatments for excess mucus production, which we see in cystic fibrosis, asthma and chronic obstructive pulmonary disease (COPD). But if we can understand the mechanisms of how these stem cells repair the mucus glands, then we may be able to find a way to put the brakes on the system and prevent mucus over production.”

I often read about people who claim that adult stem cells are as effective at treating disease as embryonic stem cells. What people seem not to understand is that there is no one adult stem cell. Stem cells of the blood system are fantastic, but they don't repair muscle, skin, brain, or, in this case, mucus glands. Finding these tissue-specific stem cells is the necessary first step to to developing new therapies based on these cells.

Stem Cells, June 27, 2011
CIRM Funding: Brigitte Gomperts (RN2-00904-1)

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On stem cells, aging and hopes for spryer golden years

Last week my three year old scraped up the entire left side of his face. Today, there's barely a trace of the injury. That's the glory of three year old skin, or more precisely, the glory of three year old stem cells.

Erin Allday at the San Francisco Chronicle had a story last week about the issue of aging stem cells featuring several CIRM grantees who are, like me, curious about why stem cells heal damage more slowly as we age. Her story includes Thomas Rando of Stanford University, whose work I wrote about several years ago. What I found fascinating then, and what still isn't understood, is why a stem cell grows less able to repair damage over time. Rando and his former postdoctoral fellow Irina Conboy (now at University of California, Berkeley) have found that in older muscle, the stem cells are still able to respond, but the signals themselves may not be as strong. The stem cells are there, they just don't hear damaged muscle's cry for help.

Allday quotes Rando, who is director of the Glenn Laboratories for the Biology of Aging at Stanford:

“I don’t necessarily see it as a way of reversing Alzheimer’s or making people live to 200 years old, but there’s this dormant potential that can be unleashed that can profoundly affect the way stem cells repair tissues.”

Allday also quotes Irina Conboy, who spoke at last week's annual meeting of the International Society for Stem Cell Research in Toronto:

Like physicists trying to find the unified theory of everything, we’re trying to find the unified theory of all these bad things that happen with aging. I think they all stem from a lack of stem cell responses.

Conboy has a New Faculty Award from CIRM to learn more about how stem cells age.

Nobody is arguing that studying stem cells will uncover the fountain of youth (at least, CIRM scientists aren't). Instead, CIRM President Alan Trounson said that by understanding how and why our body's stem cells age scientists could learn how to keep those stem cells more lively during a person's golden years. We wouldn't live longer, maybe, but as long as we're alive it would be nice to heal more effectively or resist disease. Just having bones heal more quickly could significantly reduce health care costs for the elderly.

“With aging, there are a lot of systems that start to become less efficient or break down or be more inclined to diseases. We may work out ways to provide stem cells that would enable people to remain vigorous.”

Remaining vigorous sounds pretty good to me, even if I don't ever again heal with the speed of a three year old.

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Stories of hope and scientific progress in the CIRM Annual Report

This past year, CIRM scientists made significant progress toward new therapies. Several disease teams showed that their approach is likely to be effective and they are likely to be reaching clinical trials on target in the next few years. Other groups made progress in understanding how embryonic stem cells form adult cells and tissues and mimicking disease in a laboratory dish. CIRM formed new global partnerships to leverage world-wide stem cell expertise and seven of our major facilities opened their doors to stem cell scientists.

I know all this because it's in the just-published annual report. You can read the stories here, along with letters from Governing Board chair Robert Klein and President Alan Trounson.

My favorite part of every annual report is the focus on patient advocates and their stories. This year, we feature people living with (or caring for) those with amyotrophic lateral sclerosis (Lou Gehrig's disease), Huntington's disease, HIV/AIDS, epidermolysis bullosa and stroke, as well as a follow-up story on a woman living with a form of pre-leukemia who we featured in last year's report. She's doing well on a new therapy that came out of CIRM-funded research.

The patients and their stories keep CIRM scientists' focus where it belongs -- on developing new therapies for chronic disease and injury.

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CIRM grantee Robert Blelloch wins ISSCR Outstanding Young Investigator Award

CIRM grantee Robert Blelloch of the University of California, San Francisco won the 2011 Outstanding Young Investigator Award from the International Society for Stem Cell Research. The society's annual meeting is taking place now in Toronto.

Blelloch presented his research June 15 at 6pm and will participate in a press briefing at noon June 16. His work focuses on the role of small molecules called microRNAs and their role in stem cell biology and cancer.

Jennifer O'Brien described Blelloch's work in a press release from UCSF:

During the last few years, Blelloch’s team has reported several key findings. In 2008, they reported that microRNAs promote self renewal of embryonic stem cells in mice (Nature Genetics, 2008). In 2009, they showed that when those same microRNAs were inserted into adult cells the cells de-differentiated back into embryonic stem cells (Nature Biotechnology, 2009). In 2010, they inserted a microRNA into embryonic stem cells and promoted differentiation, but determined that the microRNA had to compete with microRNAs that promote embryonic stem cell self-renewal (Nature, 2010). This year, his laboratory has been looking at microRNAs as a potential tool to systematically dissect the molecular pathways that regulate cell fate transitions, including dedifferentiation of adult cells to create induced pluripotent stem cells (Nature Biotechnology, 2011).

“People have come to realize microRNAs are remarkably powerful,” said Blelloch, associate professor in the Departments of Urology, Obstetrics, Gynecology and Reproductive Sciences and Pathology and a member of the Helen Diller Family Comprehensive Cancer Center.

Using microRNAs for therapeutic purposes has great potential , he said. “They could be used either to induce adult cells to de-differentiate to embryonic stem cells, which could be expanded, manipulated and returned to a patient, or to promote differentiation of embryonic stem cells to produce tissues that would remain integrated in the body once re-introduced.” They also could be used to target cancers, and they attract interest from biotechnology companies.

Blelloch has a SEED Award and a New Faculty II Award, both looking at the role of microRNAs in embryonic stem cell biology. Not to blow our own horn, but CIRM does know how to pick high quality research. Last year Stanford's Joanna Wysocka won the same award. She has a SEED Award and a New Faculty I Award from CIRM.

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ISSCR public symposium: Hope, passion and restraint for stem cell clinical trials

The annual meeting of the International Society for Stem Cell Research is Mecca for the hardcore stem cell research community. The next four days will be filled with intense and detailed discussions of all of the molecular details of how stem cells work--details we need to understand to move stem cells into the clinic effectively and safely.

But last night, as always, the meeting officially started with a symposium designed specifically for the public. For the third year I have had the pleasure of being on the planning committee for this event and this year we decided the field was ready to discuss clinical trials. We chose the title “The Stem Cell Promise: Moving to the Clinic” and decided to alternate brief talks by patient advocates and scientists/clinicians. Here is a list of speakers from the event.

What a rapt full house of attendees heard was an impassioned evening of hope and excitement balanced nicely with restraint and patient persistence. Early on California’s spinal cord injury advocate Roman Reed reminded the audience that the Geron trial, the first using cells derived from embryonic stem cells, is really about safety, not someone getting up out of a wheel chair. But at the same time he urged everyone in the audience to become an active advocate; to write to their legislatures to make sure this work gets funded: “Take a stand for research; take a stand for stem cells, so that one day we all can stand.”

Michael Fehling, a neurosurgeon at Toronto West Hospital, acknowledged that if scientists are going to design the perfect trial there's more they need to know about how neuronal progenitor cells will behave at the site of an injury. But he said, “You don’t have to have all the answers,” adding that most scientists believe our knowledge level is ready to begin clinical work. He admonished those in the field to make sure they learn from clinical trials and any information they can glean from how the cells perform in patients to direct a new wave of basic research to learn more of the answers needed to design a better trail. “We need reverse translational research.”

D.G.

CIRM sends Patient advocates to international stem cell meeting

Today stem cell scientists and patient advocates are descending on Toronto for the annual meeting of the International Society for Stem Cell Research. Among those registering and preparing for three days of science is Don Reed, who is one of 20 patient advocates who received a CIRM stipend to attend. 

On his blog today, Reed writes about his trip to Toronto and the important role of patient advocates in promoting science:

Patient advocates like my son Roman Reed are the emotional muscle behind research for cure. It is not easy for a paralyzed person to travel, but he will be there, listening and learning, and speaking.

Because there will be funding fights ahead, both nationally, and in every state.

When a politician says, “We can’t afford to fund the research”—someone must be there to say: “We cannot afford NOT to fund the research—and here’s why.” That someone is us. If it is just the scientists talking, it is all too easy for politicians to ignore them.

Think of he numbers. There are only a handful of top research scientists. Politically, they are negligible. If they only talk to each other, they might as well pack up their test tubes and go home, because they will never get public funding.

But there are millions of patients and family– patient advocates. Working together, we cannot be ignored.

From its inception, CIRM has recognized the importance of patient advocate voices in scientific decision-making, including ten such advocates on our governing board (bios of all board members are available here). Jeff Sheehy, patient advocate board member for HIV/AIDS blogged about his role on our board:

I serve on the governing board as a patient advocate for HIV/AIDS, and in that role I along with the other patient advocate board members have been able to directly influence the direction of the agency. Our voice has helped shape decisions regarding CIRM policies and funding. As [vice chair Duane] Roth writes, patient advocates can grasp some of the most complex and thorny policy and scientific issues and “tip the scales” in the direction of sound public policy that seeks prudently to accelerate progress towards cures.

ISSCR is kicking off the meeting tonight with a public symposium consisting of patient advocates and scientists discussing progress being made in spinal cord injury (featuring Reed's son Roman Reed), multiple sclerosis and blood disorders. Hopefully those advocate voices will help drive home the importance of continued funding for stem cell research.

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Techniques for tracking stem cells necessary for possible therapies

Last week The Scientist carried a story addressing a topic near and dear to the heart of anyone trying to develop a therapy based on transplanting stem cells, whether they are embryonic, adult, or iPS cells: Where do the cells go once they are transplanted?

The problem is this — if you, as a scientist, transplant stem cells near some damage that you are hoping they will repair, you've got to hope those cells actually make it to the damaged tissue. If they make a run for the liver when you are trying to treat the heart, or simply sit in a lump where you implanted them, those cells aren't going to fulfill their mission.

The story quotes CIRM grantee Joseph Wu of Stanford University who has SEED and Basic Biology III Awards to detect stem cells implanted into the heart and to develop stem cell transplantation therapies for hypertrophic cardiomyopathy.

“If you want to understand what happens to these stem cells, it’s important to track the fate of these cells without having to kill the animal,” says Joseph Wu, a cardiologist at Stanford University School of Medicine in Palo Alto, California. Stem cell transplants may settle down, proliferate, and differentiate as desired; they may form dangerous tumors; or they may simply falter and die.

The issue is also one CIRM grantee Paul Knoepfler of the University of California, Davis, touched on in his blog last week, saying:

Once these cells, which have spent weeks in a lab environment, are injected into a person, what happens next?

This is arguably the most important question in the regenerative medicine field, but there are few answers. We are literally mostly in the dark about what cells do after transplant, but there are some things that can be predicted pretty confidently.

He goes on to discuss some of what's known about the issue using Geron's clinical trial as an example.

In their article, the Scientist discusses a few techniques scientists are using (including some nice images) to address the question of where the cells go. The story includes a technique being used by CIRM grantee Eduardo Marban at Cedars-Sinai Medical Institute, who has a Disease Team Award to develop a therapy for heart disease.

This is the type of research that comes to mind when people who don't follow the science comment on the lack of cures. CIRM is funding a broad range of science, some of which is primarily dedicated developing new therapies, and some of which is working to understand these kinds of basic questions that need to be addressed before those therapies can become widespread.

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Heart, heal theyself

A group of researchers from University College London made a splash this week with their work prodding heart muscle to repair itself. This is big news, given both the number of people who have heart attacks (more than 1 million per year in the US) and the number of stem cell scientists working to regenerate the damage (23 awards worth $46 million from CIRM).

The big problem has been this: The heart appears to have some stem-like cells, but in adults they don't do much. They certainly aren't able to repair damage after a heart attack. When the heart is developing, however, those cells are the major source of new heart muscle. So, what gives? Why can't those cells perform in adults the way they do during development?

A story by Mitch Leslie in ScienceNOW has this to say about the UCL work:

To recapture the cells' youthful vigor, the researchers injected mice with thymosin β4, a compound already undergoing clinical trials as a heart attack treatment because it helps cardiomyocytes survive and spurs the growth of new blood vessels. The researchers then mimicked a heart attack in the animals by tying off one of the arteries that deliver blood to the heart, injuring part of the muscle.

Unlike control mice that didn't appear to fashion any new cardiomyocytes, animals dosed with thymosin β4 made some of the cells, the team reports online today in Nature. The cells infiltrated the damaged zone left by the simulated heart attack and meshed with other cardiomyocytes physically and electrically, allowing them to beat. They also seemed to prevent some of the damage that can result from a heart attack. Magnetic resonance imaging scans showed that the hearts of mice that had received thymosin β4 had smaller scars and were able to pump more blood with each contraction than were the hearts of untreated rodents.

The news is good, but thymosin β4 wasn't all that efficient. The group is hoping to find other compounds that can more effectively prod progenitors into action.

This work is interesting, too, because it shows the interplay between stem cell science and traditional drug-based medicine. Transplantation therapies are what grab the stem cell headlines. But studying how stem cells normally function can also lead to the development of new drugs, such as ones that could help the heart heal itself.

The ScienceNOW story quotes CIRM grantee Deepak Srivastava, who made headlines last year when he was able to directly transform support cells in the heart into heart muscle — a trick he'd like to replicated not in a lab dish but in an actual heart.

The study "provides strong evidence that there is a population of cells from the epicardium that can turn into new muscle," Srivastava says. "The real question is how robust is the process [of cell transformation] and how can it be improved." He recommends that researchers also investigate whether the cells can rebuild cardiac muscle during heart failure, a condition that afflicts some 5 million U.S residents and causes the organ to progressively weaken.

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Hit embryonic stem cell research, hurt iPS research too

Those of you who follow this space have read our opinions on embryonic vs. adult vs. reprogrammed iPS cells. For those of you who don't watch this space, here's our opinion in a nutshell: There is no "vs." All types of stem cells could be therapeutically valuable, and what we learn in one cell type often directly translates to discoveries in another cell type.

A paper coming out tomorrow in Cell supports that opinion. Christopher Scott at Stanford University and colleagues at the Mayo Clinic and the University of Michigan analyzed more than 2,000 papers published since 2007, when human iPS cells were first reported. According to a Stanford press release:

[The team] found that the iPS field is dominated by well-established, senior hES cell researchers. Many of these researchers are publishing studies that directly compare hES cells with iPS cells, rather than focusing exclusively on one cell type.

However, stem cell scientists are not abandoning hES cells in favor of iPS cells. In 2008, only three of the 15 iPS cell papers (5 percent) published also reported hES cell results; in 2010, 98 of the 158 iPS cell papers (about 26 percent) did so.

The work is especially important given an unresolved lawsuit that temporarily suspended federal funding for embryonic stem cell research last fall.

Stanford writes:

“If federal funding stops for human embryonic stem cell research, it would have a serious negative impact on iPS cell research,” said Stanford bioethicist Christopher Scott, citing a “false dichotomy” between the cell types. “We may never be able to choose between iPS and ES cell research because we don’t know which type of cell will be best for eventual therapies.”

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Blood from stem cells?

Blood has been among the most sought after and hardest to achieve tissue that CIRM grantees are attempting to derive from embryonic stem cells. It's an obvious target. The medical system needs a constant influx of blood, which comes entirely from volunteer donors. Creating that blood in an unlimited supply from human embryonic stem cells would significantly ease concerns about blood shortages at hospitals. We blogged about a Los Angeles Times story last January that discussed the value of this type of work.

The National Blood Data Resource Center has this to say about how much blood was used in 2001:

U.S. hospitals transfused nearly 14 million units of whole blood and red blood cells to 4.9 million patients in 2001 - that's an average of 38,000 units of blood needed on any given day.

Given those needs, the findings in a Nature paper by CIRM grantee David Traver at the University of California, San Diego could prove helpful. He and his team have discovered a gene called Wnt16 that, in the lab animal zebrafish, is key to the animal eventually developing a pool of hematopoietic stem cells, which are the source of all blood in the body.

In a press release from UCSD Traver said:

“What we need is the ability to generate self-renewing [human embryonic stem cells] from patients for treatments. But accomplishing this goal means first understanding the mechanisms involved in creating HSCs during embryonic development.”

Traver's work follow that of another CIRM grantee Inder Verma of the Salk Institute, who last month published a protocol for creating blood-forming progenitor cells from human embryonic stem cells and reprogrammed iPS cells. Discussing this work in his monthly stem cell research update, CIRM President Alan Trounson wrote:

Many more cancer and blood disorder patients could benefit from stem cell transplants if large numbers of blood forming stem cells could be grown in the laboratory. Because mature hematopoietic stem cells (HSCs) don’t expand well in culture, researchers have been trying to grow these cells from pluripotent stem cells, both embryonic stem cells and reprogrammed iPS cells. Most of these attempts have generated very low numbers of bone marrow colonizing blood precursors, and none have shown robust generation of transplantable HSCs. Now, Verma’s team has shown that with five iPS cells lines and two embryonic lines that they can efficiently generate precursors and progenitors of HSCs.

This work brings up another point often made by CIRM grantee Paul Knoepfler at the University of California, Davis. In his blog and in the Sacramento Bee Knoepfler has argued that supporting stem cell research is a matter of national security. Soldiers wounded on the battlefield need a source of blood for transfusions. Knoepfler wrote in his Sacramento Bee Op-Ed:

I hope that in the future stem cell research can perhaps slightly lessen the burden on our servicepeople and their families through technologies to save the lives of wounded soldiers.

Nature, June 9, 2011
CIRM funding: David Traver (RN1-00575-1)

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30th Anniversary of HIV/AIDS, CIRM teams making progress

Thirty years ago the first reports of a mysterious illness began appearing in the media. This illness would eventually become known as AIDS.

CIRM board member Jeff Sheehy recently spoke as part of a KQED Forum radio show about the 30th anniversary of HIV/AIDS. As a long-time AIDS activist, Sheehy has been part of the fight for a cure. In his introduction, Sheehy talked about limitations of the current drug regimen for HIV/AIDS:

“We’re still losing people and I think that gets lost in a lot of this. People have a treatment optimism belief. HIV or medication side effects are shortening lifespans. Things are still tough for people with HIV and one of the things we need to talk about is a cure.”

That cure is looking more hopeful with the announcement of a man who has come to be known as the Berlin patient (we blogged about him here). He received a bone marrow transplant in Berlin from someone who was effectively resistant to HIV infection. That man, Timothy Brown, also became resistant to infection and now doctors are unable to detect HIV in his body.

In the Forum discussion, Steven Deeks, professor of medicine at UCSF and a leader in HIV/AIDS research, pointed out that although Brown’s HIV is now undetectable, his isn’t the treatment that will become a widespread cure. First, there aren’t enough bone marrow donors who are resistant to HIV. The bone marrow transplant itself is also an extremely risky procedure.

Deeks pointed to work being carried out by CIRM grantees who are attempting to engineer a person’s own bone marrow stem cells to carry the mutation that makes the cells resistant to HIV. Sheehy pointed out that CIRM has been alone in funding this type of work:

“It’s been lonely. Thank god for the voters in 2004 who voted for proposition 71[ the proposition that created CIRM]. What surprised me when I got appointed to this board I really didn’t think there was much in HIV that could be done.”

CIRM now funds more than $40 million in HIV/AIDS research (see a list of those awards here), including two disease teams that are both working toward beginning clinical trials in two to three years.

This video features Sheehy and John Zaia from the City of Hope who leads one of those disease teams.



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Marius Wernig on why we need many stem cell approaches to new therapies

Last week we blogged about work by Marius Wernig of Stanford University, who has successfully converted human skin into nerves, skipping the step of first converting the cells into embryonic-like iPS cells.

Wernig is quoted in a Nature news story talking about whether the work could replace induced pluripotent stem (iPS) cells or embryonic stem cells:

"I would say that both approaches should be actively pursued because you never know for which cases and specific applications one or the other may be more suitable."

I think the best example of why we need many approaches to treating disease came from patient advocate Rodney Paul, who spoke to an external review committee last year about CIRM. Here's what we wrote in our Best. Analogy. Ever. blog entry on October 13, 2010:

He pointed out that on this day the world saw awe-inspiring images of the first of 33 miners rising out of the Chilean mine where they’d been trapped — and that those miners were rescued through one of three shafts that had been dug as part of the rescue mission.

The shaft in question was dubbed “Plan B”. Drilling on plans A and C didn’t go as smoothly as hoped. That’s why on an important mission where time is limited and lives are at stake it’s important not to pin all hopes on one strategy.

With embryonic, adult, iPS and cancer stem cells plus the new direct conversion techniques CIRM is drilling a series of shafts all leading toward possible disease therapies.

We have a list of all our grants online. You can use the filters to see how many awards we're funding using different types of cells. Right now, the numbers are:

• Embryonic: 215
• iPS: 78
• Adult: 47
• Cancer: 10

Those numbers are updated whenever we fund new awards.

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Stem cell banking and the making of a patient "advocist"

In 2005 Chris Hempel gave birth to twin daughters Cassi and Addi. In 2007, she and her husband learned that their girls had a rare, fatal disease called Neiman Pick Type C.

Four years later, Hempel describes herself as a "advocist" for rare diseases. She's an advocate for scientific research but also an activist seeking to bridge the gap between patients and researchers. One of her primary messages is this: If patients donate tissue (skin, in her case) that contribute to science, then they should get to know the results and participate in the research.

That sounds easy, but has proven difficult. She spoke to CIRM's Standards Working Group in April to discuss her experiences and encourage CIRM to take a leadership role in creating policies that engage and inform the tissue donors.

During her talk, she said, "If the entire goal is really to use a patient's own cells to cure them, well, you really can't cure a patient if they are just a number."

CIRM funds several awards that have the intention of creating reprogrammed stem cell lines from skin samples to better understand genetic diseases, much like the research Hempel participated in. These scientists have creating disease-in-a-dish models of schizophrenia, Parkinson's disease and autism using this approach.

One of the issues the working group discussed is the types of standards that should be in place to protect the rights of the people who donate tissues for these and other CIRM studies. In addition there was discussion about ways of providing information to potential donors, research participants and the public.

The agenda from that meeting contains additional information about tissue donation and iPS banking: available here.

Here is Hempel's talk:



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