Friday, March 30, 2012

Guest blogger Alan Trounson — March stem cell research highlights

Each month CIRM President Alan Trounson gives his perspective on recently published papers he thinks will be valuable in moving the field of stem cell research forward. This month’s report, along with an archive of past reports, is available on the CIRM website.

The lead article in this month’s report focuses on CIRM-funded work in Irv Weissmann’s lab at Stanford. It reports on a protein on the surface of cancer cells that in essence says “don’t eat me” to the immune system cells that are supposed to seek out, engulf and destroy tumor cells. This cell surface protein is called CD47. More important, an antibody that blocks CD47 seems pretty effective in reactivating the immune system’s attack on the tumors—at least in mice. My colleague Amy Adams blogged about this research here.

There are two quite different aspects of this research that excite me. First, it is a finding that could be very important clinically that clearly derived from basic research. Irv’s team originally found the cell surface protein when they were trying to understand the fundamental relationship between the immune system and cancer cells. It turns out CD47 is expressed on the leukemia cells they were studying, but highly expressed on leukemia stem cells, which are hypothesized to be responsible for the tendency of leukemia to relapse after treatment.

The other reason to be excited about this work is that it goes counter to much of the direction of cancer research today. Many of the exciting new therapies are coming from an increased understanding of how individual most cancers are from one another. The newest therapies tend to be most effective for a narrow set of tumors, often with specific genetic compositions. This latest work showed the single antibody to be able to shrink and sometimes eliminate many different solid tumors. The Stanford team reported results for cancer cells from seven different human tumors transplanted into mice. Such a broad-spectrum cancer treatment, even if it has to be paired with other agents, has the potential to be less costly because of the market size.

My full report is available online, along with links to my reports from previous months.


Thursday, March 29, 2012

David Serrano Sewell reflects on MS Awareness Month, the need for therapies

David Serrano Sewell is a patient advocate member of CIRM’s governing board. His bio is available on our website.

When I began serving on the CIRM governing board in 2005 it was with the hope that I could be part of guiding the agency’s decisions in a way that would benefit the many people in California who are looking to CIRM for new therapies.

I have been active with the Northern California Chapter of the National Multiple Sclerosis Society since my own diagnosis with MS. Right now, there are no cures for the disease, just drugs that can help slow it’s progression or mitigate the symptoms, though those also come at a price. The most common drug causes flu-like symptoms and the effectiveness diminishes over time. It’s a disease in need of more effective therapies.

March is National Multiple Sclerosis Awareness Month, and it seems like a good time to look back on what we’ve accomplished at CIRM to find a stem cell-based therapy for the disease.

We’ve funded two awards to researchers who are directly studying MS. One is to Thomas Lane at the University of California, Irvine (here is a summary of that award). He is looking at ways of using human embryonic stem cells to replace the insulation surrounding nerves that is lost in MS. The other is to Samuel Pleasure at the University of California, San Francisco (here is a summary of that award). He is attempting to start with nerve stem cells and mature them into precursors of the types of cells that form that insulation. He then proposes transplanting those precursors into people with MS to treat the disease.

Both of these projects are in their early stages and it’s too soon to know whether they will blossom into new therapies. What I do know is that without these kinds of early stage projects we will never see more effective therapies for MS reaching patients.

As part of the governing board I am part of the group that makes decisions not only on which individual awards to fund, but also on big picture issues. We make decisions on whether new stem cell research facilities would speed new therapies, and how to ensure that California remains a leader in stem cell research by funding training and career development. Although those awards don’t have a single-minded focus on MS, they will help bring new therapies for all the patients who I was elected to represent.

I hope in future MS Awareness Months to be able to point to new research projects picking up where these early stage ones leave off. Getting from early research to promising therapies is a long and often difficult process, but it’s one that I’m proud to play a role in directing.

You can read more about MS on the CIRM website.

David Serrano Sewell

Wednesday, March 28, 2012

Single antibody developed by CIRM grantee fights many tumors

Stanford researcher Irv Weissman made the news yesterday when he published a paper showing that  a single antibody-based therapy can effectively slow the growth and in some cases eliminate many different kinds of human tumors, at least in mice.

Weissman leads a $20 million CIRM disease team that is developing an antibody-based therapy to treat leukemia. In previous work, Weissman had found that leukemia cells evade the immune system by placing a protein on their surface that tells the immune system “don’t eat me.” Blood-forming stem cells in the bone marrow also use that protein on occasion to avoid immune detection, as do red blood cells.

Weissman and his group are developing an antibody that latches onto that protein, called CD47, and makes the protein invisible to the immune system, particularly immune cells called macrophages. Without their “don’t eat me” signal, the macrophages ingest the leukemia cells.

Now, it looks as if that antibody therapy might be effective for cancers in addition to leukemia. Weissman and his group published a paper March 26 in the Proceedings of the National Academy of Sciences showing that CD47 is present at high levels on the surface of tumor samples taken from people with ovarian, breast, colon, bladder, brain, liver and prostate cancers.

What’s more, their antibody therapy prevented the growth of those tumor cells that had been implanted in mice, and in some instances eradicated them. A story in the Los Angeles Times describes the work:
Placing the cells in lab dishes, the team administered an antibody: a protein that binds to CD47 and blocks it from warding off immune system cells. Macrophages ate the cells.

The researchers then implanted human tumor cells in mice for further study. They allowed the cancers to grow, and administered the antibody against CD47.

Antibody treatment inhibited the growth of almost all of the solid tumors and was able to wipe out some smaller cancers altogether, according to the report, which was published Monday in the journal Proceedings of the National Academy of Sciences.
A press release from Stanford quotes Weissman:
“Blocking this ‘don’t-eat-me’ signal inhibits the growth in mice of nearly every human cancer we tested, with minimal toxicity. This shows conclusively that this protein, CD47, is a legitimate and promising target for human cancer therapy.”
A story in Science quotes Weissman talking about testing the drug in human trials:
Weissman's team has received a $20 million grant from the California Institute for Regenerative Medicine to move the findings from mouse studies to human safety tests. "We have enough data already," says Weissman, "that I can say I'm confident that this will move to phase I human trials."

Tuesday, March 27, 2012

Stem cell therapy for heart attack

We are rolling out our 2011 Annual Report stories throughout March. The full report will be posted online and available for download later this month.

Each year in our Annual Report we highlight some of the patients and scientists who came to speak to our governing board about the search for cures. This year one of our stories focuses on research by Eduardo Marbán, who is leading a CIRM disease team developing a therapy to help repair damage after a heart attack.

Marbán spoke to our governing board about a clinical trial using the patient’s own heart cells to repair the damaged heart. This trial is the precursor to the next generation research being funded by CIRM, which uses heart cells from a donor heart. (We blogged about his Dec. 8 talk here.)

Marbán brought with him Frank Lesikar, who had participated in Marban’s clinical trial after a heart attack had damaged his heart. From the annual report story:
I'm in better shape than I've been in in years," he says. Some months after his attack, he enrolled in a clinical trial, in which researchers harvested a bit of tissue from his heart, coaxed stem cells from the tissue to grow, and put the cells back into his heart again. The results? Lesikar’s heart is functioning better and the scar left from his heart attack appears to be reduced.

The study that helped Lesikar is one that led up to a $5 million disease team award from CIRM to fund the next generation therapy.
Read more about Lesikar’s story and about Marbán’s research in the annual report story posted online. This is the seventh annual report story we’ve posted. Here are the others:
The full report will be available for download later this month.


Monday, March 26, 2012

Progress report from the CIRM disease teams

At last week's meeting in Sacramento, our governing board heard an update on the progress being made by the 14 CIRM disease teams that they had approved in October 2009. A list of those teams is available on our website.

Funding teams of researchers with scientific and regulatory expertise to work together was an innovative step—one that CIRM anticipates will help those groups get their therapies to clinical trials faster than they would without this team approach.

Before issuing the awards, CIRM’s science staff worked with the teams to come up with milestones and success criteria. Periodically, CIRM meets with the teams along with a group of experts who have experience taking therapies into clinical trials and through to approved therapies.

Taking the advice of these experts into consideration, CIRM decides whether the projects are achieving the agreed upon milestones. These assessments help protect the state’s financial investment by making sure the teams are focused and have the resources and expertise they need to achieve the goals of their research. It also identifies projects that need additional support, or ones in which the original idea is not ready to continue.

Ellen Feigal, CIRM’s senior VP of R&D, summarized the progress being made by each of the teams. You can read the background material she gave to the board members, which has detailed information about the achievements by each group.

The first CIRM and expert assessments of the disease teams met in 2011, at the 12 to 18-month milestones. The group found that thirteen of the projects are on a trajectory toward meeting their milestones. Of those, one is undergoing some revisions to focus the work. One of the projects is not continuing. CIRM will save roughly $13 million on this award through early termination.

The team that is not continuing, led by researchers at the University of California, San Francisco, produced data that did not meet the agreed upon success criteria. That criteria had to do with selecting the best stem cell type to use in their proposed therapy for a type of brain tumor called recurrent glioma.

During the course of their work, the team had generated some new methods that may be useful for other researchers and published two papers. These lessons learned are part of the iterative process of trying to create new therapies.

As with all potential therapies, none of the therapies developed by the CIRM-funded teams can enter clinical trials until they provide evidence to the U.S. Food and Drug Administration (FDA) that the therapy is safe to test in humans. Carrying out these experiments is costly and brings with it the possibility that the original idea won’t work. The researchers then needs to go back a step and take what they learned from the first attempt and use those lessons to guide other approaches.

CIRM created the disease teams as a way of funding this critical stage of research. We instituted the milestones and the input of product development experts to better position the teams for success and protect the state’s financial investment. This way, we are trying to ensure the teams have the resources and expertise they need to bring these much needed therapies into clinical trials in humans.

CIRM and our panel of experts will be meeting with the thirteen teams at their 24- to 30-month milestones in 2012 to assess progress. Regardless of the outcome of those assessments, each of the teams is producing data and gaining experiences that will help guide the next generation of stem cell-based therapies.


Thursday, March 22, 2012

California's diverse high school students get additional stem cell lab experience

Yesterday’s governing board meeting in Sacramento included an agenda packed with meaty discussion items. We’ll be blogging about those items over the next few days. You can read the agenda here to see what was up for vote and discussion. We always audiocast our board meetings and put that information in the agenda for those who want to hear the conversation in real time. Transcripts are also posted a few weeks later to the page listing all public meetings.

We encourage people to attend our board meetings in person. For those who can’t make it, here’s one scientist’s impression of what a meeting is like. Paul Knoepfler is a CIRM grantee at the University of California, Davis, who also has a blog about stem cell science. He attended yesterday’s meeting and introduced himself to the board as both a scientist and a patient advocate—he is a cancer survivor.

One notable item from yesterday was the decision by our governing board to support $1.7 million for a high school summer internship program called the Creativity Awards. We’ve blogged quite a bit about this program including this post previewing their meeting, this post about the work the students presented at their meeting, this post about one of the recipients who went on to be a semifinalist in two national science competitions, and this post about an award going to one of the Creativity Award hosts.

The reason for all these posts is that we’re really excited about this program. It brings in high school students from all socioeconomic backgrounds and gives them experience in California’s stem cell labs.

Here’s what CIRM president Alan Trounson had to say about the awards in the press release from yesterday’s meeting:
“This program exposes high school students to cutting edge medical research and encourages the kind of creative thinking that leads to groundbreaking discoveries,” said Alan Trounson, CIRM President. “The pilot program last summer demonstrated the demand for stem cell research opportunities among California’s young people. Expanding the program and extending it for three years will inspire more students from all socioeconomic backgrounds to pursue careers in stem cell science and nurtures the creative thinking that will help them be successful.”
That release also lists the nine recipients of the awards. This video gives a flavor of the kinds of research carried out by the diverse students who participated in last year’s pilot program of the Creativity Awards:


Tuesday, March 20, 2012

Stories of Hope: finding a therapy for neuromyelitis optica

We are rolling out our 2011 Annual Report stories throughout March. The full report will be posted online and available for download later this month.

Each year in our Annual Report we highlight some of the patients and scientists who came to speak to our governing board about the search for cures. This year one of our stories focuses on a rare disease called neuromyelitis optica, also known as NMO or Devic’s disease. You can read that story here.

Neuromyelitis optica is so rare that when Ali Guthy was diagnosed her mother Victoria Jackson could only find one expert in the U.S. to treat her daughter. Jackson, who was the creator of Victoria Jackson Cosmetics, quickly started the Guthy-Jackson Charitable Foundation and began funding researchers to work together to better understand and hopefully find a therapy for the disease. CIRM chose to highlight Jackson and NMO because it provides such a vivid example of what dedicated patient advocacy can accomplish.

Last year CIRM produced a video about Jackson’s efforts to start the foundation and turn her life’s focus, as she says, from “mascara to medicine.”

Like multiple sclerosis, NMO is a disease in which the body’s immune system attacks the insulation that surrounds nerves. Michael Yeaman, who studies the disease at UCLA, told the governing board that until recently the only way of slowing the disease was through sterioids, which he said leaves patients vulnerable to other infections and can increase the risk of cancer. He said:
“Ultimately, reversing the damage caused by NMO or other autoimmune diseases will require regenerative medicines, and stem cells will play a key role in that respect. To restore the central nervous system, we're going to need regenerative help.”
Read more about Jackson’s story and about progress toward therapies in the annual report story posted online. This is the fifth annual report story we’ve posted. Here are the others:
The full report will be available for download later this month.


Friday, March 16, 2012

Stem cell image of the week: Pancreatic cells for a diabetes therapy

Pancreatic cells derived from human embryonic stem cells. ViaCyte, Inc.

This colorful river of cells is really a side shot of pancreatic cells within a thin device. The cells, which were matured from embryonic stem cells, are being developed as a therapy for diabetes by a CIRM-funded disease team led by ViaCyte in La Jolla.

The group is maturing embryonic stem cells into the type of pancreatic cells that produce insulin in response to blood sugar. These are the cells that are destroyed in people with diabetes. The group puts those cells within a thin device to protect them from the body’s immune system. When they implant the device in animals, the cells appear to function properly and release appropriate amounts of insulin to control blood sugar.

The group is now working toward clinical trials to test the device in humans. We wrote more about the ViaCyte-led team last week. You can read that story and watch a video about the work here.

We have more diabetes images on our Flickr site, where you can also learn more about what the different colors represent in today's image.


Thursday, March 15, 2012

Stem Cell Therapy for the Economy

We are rolling out our 2011 Annual Report stories throughout March. The full report will be posted online and available for download later this month. Today we are introducing a story about the economic benefits of funding stem cell research in the state: Stem Cell Therapy for the Economy.

CIRM’s primary mission is to develop new stem cell-based therapies for disease. But the process of driving those new therapies has other benefits to the state—economic ones.

Scientists and industry have moved to California due in part to hopes of receiving funding from CIRM, and also to support the growing stem cell research industry. With these companies and new labs come jobs, tax revenue and additional non-CIRM grant money.

A few years ago CIRM commissioned a report to find out how many jobs and associated tax revenues CIRM had created for the state. As we say in our annual report story:
Counting only the first $1.1 billion in funding, by 2014 CIRM’s initiatives are expected to have brought in $200 million in tax revenue to the state and created 25,000 job years—that’s economist speak for employing 25,000 people for a year, or 5,000 people for five years. Those jobs each have ripple effects for the state when employed people pay taxes and buy groceries, electronics and homes.
Our story specifically describes research by Robert Wechsler-Reya and Peter Coffey, who set up labs in California to study childhood brain tumors and blindness, respectively.

Wechsler-Reya, who is now director of the Tumor Development program at the Sanford-Burnham Medical Research Institute in La Jolla, moved into the new Sanford Consortium building partially funded by CIRM (read more about the CIRM-funded facilities here). In our story he said that facility and the collaborations it encourages was one reason to move to California from Duke University:
“One of the amazing things about this place is that there are a lot of interactions not only among academic institutions, but also between academia and industry. It is the kind of thing that I could not have done back east.”
Read our story online for more about Wechsler-Reya, Coffey, and The Jackson Laboratory West, which greatly scaled up after receiving a CIRM award in 2009. The most important aspect of these grantees’ work is their progress toward therapies. But it’s good to know that the state’s growing stem cell industry is good for the economy as well as eventually being good for patients.

This is the fifth annual report story we’ve posted. Here are the others:
The full report will be available for download later this month.


Wednesday, March 14, 2012

CIRM Bridges students at Parkinson's Institute get job training, experience

The Parkinson’s Institute in Sunnyvale not only carries out CIRM-funded research into therapies for Parkinson’s disease (you can see a list of their awards here), but they are also training the next generation of researchers to continue that work in the future. They’ve partnered with the San Jose State University CIRM-funded Bridges program to provide stem cell research experience to undergraduate and masters students. The Parkinson’s Institute recently featured their two Bridges interns Orlando Macaranas and Leo Rodriguez in their newsletter, writing:
“Though eventually Parkinson’s will be a disease of the past, research will continue, and we are proud to help foster the next generation of researchers.”
One goal of the Bridges program is to provide students with the kinds of hands-on experience they need to work in California’s growing stem cell industry. The newsletter quotes Macaranas:
“Being able to work in a lab and gain hands-on experience has been invaluable. I am more prepared for a job after graduation than my peers who have not completed this program.”
Orlando Macaranas, CIRM Bridges student

Rodriguez said:
“The Parkinson’s Institute is a perfect place to gain lots of experience. The stem cell researchers here are eager to teach and I have been given more responsibility and increased my skills more than I would have at a much larger facility.”
Leo Rodriguez, CIRM Bridges student
This page on our website describes the Bridges program here you can see the locations of Bridges programs and their mentor institutions [pdf]. You can also see a list of the Bridges award recipients in our list of all funded CIRM awards.

Here is a video shot at last year’s Bridges meeting where the students discussed their research progress.


Tuesday, March 13, 2012

2011 Annual Report: Finding a cure for sickle cell disease

We are rolling out our 2011 Annual Report stories throughout March. The full report will be posted online and available for download later this month.

Each year in our Annual Report we highlight some of the patients and scientists who represent diseases that are the focus of CIRM awards. This year one of our stories focuses on sickle cell disease. You can read that story here.

Nancy Rene spoke to our governing board about her grandson Joseph, whose sickle cell disease was diagnosed through California’s newborn screening program. Joseph had a stroke at nine months old and receives regular blood transfusions to prevent additional strokes and the severe pain that accompanies the disease.

Despite the leg brace he still wears due to the stroke damage, Rene told our governing board, “Being a little boy trumps having a stroke.” As she said this she showed a picture of Joseph, smile on his face, jumping on a bed.

But not all of her stories were so positive. She works with families of kids with sickle cell and spoke of discrimination in the schools and medical system. The genetic disease is most common in people of African descent.

CIRM board member Bertram Lubin is president of Children’s Hospital and Research Center of Oakland and has a 40 year interest in treating sickle cell disease. In his introduction he said CIRM’s $9 million disease team award to find a therapy for sickle cell disease could significantly help children and families with the disease:
"This demonstrates CIRM’s commitment to minority health, health disparities and social justice issues and I think when you look at the people in the state of California who voted for proposition 71 there is a substantial number of minorities who are committed to improving the health of people within their communities."
Lubin went on to say that each year 150 children in California are diagnosed with sickle cell disease through the newborn screening program that identified Joseph’s disease. Altogether, he said there are 9,000 people in California with the disease. For each person, the yearly medical costs are between $10,000 to $15,000. Lubin said:
"This is going to create a major burden. Any approach that has the potential to change that not only has major economic benefit but has phenomenal quality of life benefits for the children and for the family."
You can read about the CIRM disease team approach led by Donald Kohn, who is part of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and about Joseph in our annual report story online.

This is the fourth annual report story we’ve posted. Here are the others:
 The full report will be available for download later this month.


Friday, March 9, 2012

2011 Annual Report: Toward a stem cell therapy for #diabetes

We are rolling out our 2011 Annual Report stories throughout March. Many of these stories feature multimedia material such as a video that we are releasing today alongside a story about diabetes research. The full report will be posted online and available for download later this month.

Todd Dubnicoff is CIRM’s videographer and video editor.

One thing I’ve learned producing videos at CIRM is that you don’t truly know a disease until you hear first-hand from the people who live with it. Take, for example, the new diabetes video that we’re rolling today out along with the 2011 Annual Report story on the same subject.

For the video, my colleague Amy Adams and I filmed interviews with Chris Stiehl and Sarah Young, both living with type 1 diabetes, as well as Kevin D’Amour, the chief scientific officer at Viacyte, Inc., which has a $20 million CIRM Disease Team award to bring an embryonic stem cell-based diabetes therapy to clinical trials. JDRF recently co-funded the project. Our annual report story has more details about Viacyte’s therapeutic strategy.

Before the interviews I brushed up on my knowledge of diabetes. I felt I had a solid grasp of the metabolic basis of the disease from my biology classes in school. But it wasn’t until I pressed the start button on my video camera and watched Chris and Sarah tell their stories that I got a glimpse of what diabetes was all about: it’s about having the courage and positive outlook to live with a daily and life-long burden. Chris who was diagnosed at the age of 10 and is now 62 described diabetes this way:
It’s a 24-hour a day job, 7 days a week you never get a day off. I would give anything for a day off. Just to not have to think about it. Besides all the things you have to do for your work and your family and everything, you have to be constantly thinking: “What’s my blood sugar? What have I eaten? Have I exercised too much or too little? How much insulin should I take based on the exercise I just did? Gee by the way is my insulin pump running out of insulin? How’s the battery in the pump? How’s the battery in the blood tester?” There’s a whole entourage of things to worry about and it occupies your mind a lot.
Sarah, who was also diagnosed at 10 and is now just 14 spoke about the meal-by-meal vigilance of insulin dosing that she must think through to keep her blood sugar levels safely in check:
Every time I eat a meal or a snack or if someone [at school] brings something in for their birthday, I have to sit down and think, “what do I want to eat?” And I have to really be sure that I’m right because if I don’t end up eating everything I dosed myself for, I’ll have a low blood sugar which is bad. And if I want to eat more I’ll have to dose again which is just complicated and annoying. So it was really a pretty big change for me after I got diagnosed to have to make a commitment about the amount of food I was going to eat and stick to that and not to be able to change based on, “oh, I feel full now” or “you know, I think I’ll have another cookie.”
While rewatching those interviews to create the video, I felt like I had gotten to know Chris and Sarah and I became impressed with how they and their families have bravely confronted diabetes. Personally, it makes the prospect of Viacyte’s stem cell-based therapy for diabetes even more exciting. Kevin D’Amour echoed that thought during his interview:
I’ve been here [at Viacyte] nine years and in the early days we didn’t have much. But now to see the program evolve and blossom to the point where it represents a clinical reality for helping patients has been very motivating and very satisfying.
This is the third annual report story we’ve posted. Here are the others:
The full report will be available for download later this month.


Wednesday, March 7, 2012

2011 Annual Report: Spotlight on Autism

We are rolling out our 2011 Annual Report stories throughout March. The full report will be posted online and available for download later this month.

Each year in our Annual Report we highlight some of the patients and scientists who represent diseases that are the focus of CIRM awards. This year one of our stories focuses on autism.

Laureen Forman got her first hint that her son Brandon had autism when he was 18 months and wouldn’t look at her husband. Her experience with Brandon, who is now 9, has led her to believe that the generally held view of autism needs to change.
"People need to realize this isn’t a psychological disorder. These kids aren't mentally ill. These children are physically sick."
Among those people working to understand what goes wrong within the brains of kids like Brandon is CIRM grantee Ricardo Dolmetsch of Stanford University, who also has a son on the autism spectrum.

Dolmetsch created brain cells from the skin cells of people with a form of autism. He found that those brain cells had some unusual qualities. As we write in the annual report:
When he compared the neurons generated from people without autism to those with the disease, he noticed three basic differences: the electrical signaling disruption, the lack of long-distance connections, and elevated levels of two of the chemicals that brain cells use to communicate. Norepinephrine, which plays a role in anxiety and stress, was four times to five times higher than normal, and dopamine, which is involved in attention and social behavior, was two times to three times higher.
You can read more about Forman’s story and Dolmetsch’s research in the annual report online. We also blogged about Dolmetsch’s research in November.

Previously posted annual report stories:


Tuesday, March 6, 2012

Stem Cell Research and Aging Symposium Highlights

Last week the Buck Institute for Age Research in Novato, CA held a symposium on stem cell research and aging, which CIRM helped sponsor. The Buck Institute has received more than $36 million in grants from CIRM, including awards to researchers working toward stem cell therapies for Parkinson’s disease.

Benjamin Blackwell, a research associate at Buck recently wrote about some of the highlights of the meeting for the Buck Institute web site.
“The 2012 Buck Symposium on Stem Cell Research and Aging brought together some of the world’s cutting-edge researchers, but more importantly, it provided a space for the formal convergence of stem cell-based regenerative medicine and aging research.”
He goes on to describe talks covering Huntington’s disease, basic stem cell biology, neural development, Parkinson’s disease and emerging targets for new therapies.

In another report, Kevin Perrott, a PhD candidate wrote:
“It is tremendous to see regenerative medicine targeted directly at the general phenomenon of aging and the underlying biological mechanisms which lead to many degenerative conditions.”
Several people from CIRM and our grantees from other institutions attended the meeting and participated in talks about how stem cells could generate new therapies for diseases of aging.

Our website has a list of all CIRM-funded grants, which you can sort by disease area. These include a wide variety of grants attempting to develop new therapies for age-related diseases.


Monday, March 5, 2012

CIRM 2011 Annual Report: A Functional Cure for HIV

Two teams of CIRM-funded researchers are working toward what they hope will be a functional cure for HIV infection.

These groups are the focus of our initial annual report story, which you can read here. That story quotes CIRM governing board member Jeff Sheehy, who is a long-time HIV/AIDS advocate:
"I'm incredibly optimistic. I don't think we're going to get there tomorrow, but I think the train has left the station to get there. The whole dialog has been completely upended, and CIRM has been at the cutting edge."
Throughout the next weeks we will be previewing our online annual report stories here. They cover promising science, patient stories, and CIRM initiatives that will help accelerate the development of the therapies those patients are waiting on.

We’re doing the annual report a little differently this year. In the past, we printed and mailed annual report books. (You can download PDFs of those books here.) This year we’re saving money and instead printing smaller brochures with some financial data and excerpts of the full news and patient stories that can be found online. Those brochures will be available for download, and in hard copy at our meetings, in the next few weeks.

Taken together, the stories in this year’s annual report show the many ways in which California is leading in the development of new stem cell-based therapies, in initiatives to speed those therapies, and in the involvement of patient advocates to help us achieve our mission.

As Paula Cannon, who is one of the HIV/AIDS researchers who appears in today’s featured story, says:
"This could only happen in California."
I hope you find the stories interesting, informative and inspiring.


Friday, March 2, 2012

Guest blogger Alan Trounson — February’s stem cell research highlights

Each month CIRM President Alan Trounson gives his perspective on recently published papers he thinks will be valuable in moving the field of stem cell research forward. This month’s report, along with an archive of past reports, is available on the CIRM website.

I am very proud that two of the articles this month’s report discusses are from the new journal CIRM supports financially, Stem Cells Translational Medicine. Just a little more than a year ago CIRM solicited proposals from publishers to launch a journal that would give a home to and consolidate articles on translational research—those papers focused on pushing our field into clinical trials and beyond to routine care. Too often, these sorts of very practical papers cannot get accepted at routine research journals. Yet, it is critical this information spread to other teams in the field quickly. Our solution, provide the seed money to jumpstart a journal that would publish in a free online format with rapid peer review turnaround. Our ultimate publishing partner, AlphaMed Press, also publishes Stem Cells, the oldest journal in the field.

The two articles I cite both deal with the critical issue of scaling up growth of cells to produce sufficient quantities to supply large clinical trials and to do this using methods that limit as many cumbersome and expensive steps as possible. Especially important is avoiding anything that would reduce the chances of satisfying the FDA’s requirements for Good Manufacturing Practice (GMP), which is needed for cells being transplanted into people. (This short video has more information about GMP.)

The first paper details a method to get around the tendency of pluripotent stem cells, either embryonic or reprogrammed iPS cells, when grown in culture to want to mature into adult cell types. Early methods for keeping cells in their embryonic, or pluripotent, state had the double problem of being difficult to scale up and requiring animal feeder cells, which would greatly complicate creating GMP grade cells. Numerous reports have shown various ways to eliminate the animal cells, but those systems are still quite complex involving several human-derived proteins. The CIRM-funded team in this study was able to eliminate two of those human proteins with a single small molecule, greatly simplifying the process.

The second paper looks at the exploding use of Mesenchymal stem cells (MSC), now deployed in several hundred early stage clinical trials. The problem is that the usual source of these cells, bone marrow and fat tissue, have miniscule numbers of MSCs. This could make scaling up those small early-stage clinical trials into large definitive clinical trials difficult and costly. So, the Australian team in this study tried using iPSCs as the source, which has been done before but with slow and cumbersome techniques. They succeeded in shaving off about half the time to go from iPS pluripotent cells to MSCs and reduced the number of steps in the process.

My full report goes on to discuss one of my favorite topics, using iPSCs for modeling disease, but for the first time an infectious disease, hepatitis C. My full report is available online [pdf], along with links to my reports from previous months.