|The Lorry I. Lokey Stem Cell Research Building
We’re in the middle of a big week for CIRM-funded research facilities. UCLA opened the doors to their new CIRM-funded stem cell research space Monday (here’s a video about that building) and today Stanford cuts the ribbon on the largest of the CIRM buildings — a gleaming 200,000 square-foot state-of-the-art facility. According to the Stanford press release about their Lorry I. Lokey Stem Cell Research Building:
The building’s 200,000 square feet of floor space, serving about 550 occupants, makes it the largest dedicated stem cell research building in the country, if not the world. But just as important, it was financed without any federal funding — buffering its occupants to some degree from the vagaries of embryonic stem cell politics.
The buildings are part of CIRM’s Major Facilities RFA, given out in May 2008 when federal funding could only be spent for stem cell research involving a small number of approved cell lines. That included working with unapproved lines in buildings created or supported through federal funds, which ruled out most available research space for work involving unapproved human embryonic stem cell lines. The CIRM buildings provide space free and clear for working with whichever stem cell lines scientists think will move the field toward new cures.
CIRM awarded $271 million to 12 institutions, each of which was required to raise additional funds through private donations. Altogether, CIRM’s investment brought in an additional $800 million in financial commitments and created construction jobs throughout the state at a time when those jobs were very much needed. This week’s building openings came about thanks to a $20 million donation to UCLA and a $30 million gift to USC by The Eli and Edythe Broad Foundation and a $75 million gift from Lorry I. Lokey to Stanford University.
Following today’s ceremony at Stanford, the University of Southern California will hold their grand opening Friday. Altogether, those three buildings created 322,000 square feet of new research space, all of it free of federal funding and therefore available for research into all types of stem cells regardless of what happens with the current court cases. This space has the capacity to house nearly 1,000 members of research teams.
Stanford gave a nice description of how having stem cell researchers housed together in well equipped space will speed the research that takes place. They quote Theo Palmer, who works on stem cell therapies for neurodegenerative diseases:
“We used to have to plan our day around getting our samples where they needed to be when they needed to be there. Now some of the best resources in the world are immediately available — including extraordinary cell-sorting capabilities and some of the most advanced single-cell genetic-profiling equipment. At other places in the country these resources are essentially not available, or available only by special arrangement.”
That time saved means CIRM grantees can work faster than ever toward new disease therapies.
The Victoria, Australia, innovation Minister Gavin Jennings is talking up a Victoria-California research team working to develop a therapy for autoimmune diseases such as type 1 diabetes and multiple sclerosis.
“The project highlights the value of collaboration between world-leading centres like Victoria and California in biotechnology and stem cell research. It is yet another demonstration of our commitment to take action to improve the quality of life for millions of people around the world.”
The team is one of CIRM’s Transplantation Immunology Awards, this one led by Kenneth Weinberg at Stanford University (read a summary of their project). In that same round of funding a Victorian scientist teamed up with Scripps Research Institute’s Jeanne Loring.
CIRM has formed collaborative partnerships with ten organizations in U.S. States or internationally. These are intended to build partnerships between leading stem cell scientists, wherever they may be working. As a state agency, CIRM’s money only goes to the California researchers, with our partners supporting their scientists. So far it’s been a very successful program, with 14 awards including international research partners in Canada, Germany, Japan, the U.K., and Victoria, Australia. You can find those awards on our stem cell grants table.
In the project Jennings discussed, the researchers are looking for a way of regulating the immune system, which causes autoimmune diseases when it attacks the body’s own organs and tissues.
“This project is critical to us learning more about how to regulate the immune system in a bid to enhance the potential of stem cell derived tissue transplants as therapies to combat MS and other conditions.”
On January 16, 2009 CIRM began our glamorous TV career, launching the YouTube channel CIRMTV. Despite our fabulous onscreen talent the Emmys have yet to call (though they are welcome to comment below…). Still, we’re pretty pleased with the channel’s success. Today we reached 100,000 views to our videos, which include some educational pieces about stem cell research and a disease-focused series about individual conditions that CIRM-funded researchers are working to address.
Overall, our videos are most popular in the U.S., Canada, the U.K., Australia, the Philippines, and Sweden. The top ten views have gone to:
1) Parkinson’s Disease: Progress and Promise in Stem Cell Research
2) Hans Keirstead: Developing therapies based on embryonic stem cells
3) Jerome Zack: Creating iPS Cells
4) Macular Degeneration: Progress and Promise in Stem Cell Research
5) Irv Weissman: Differences between Adult and Embryonic Stem Cells
6) Bringing Stem Cell Cures to the Clinic: UC Davis GMP Facility
7) Catriona Jamieson: Therapies Based on Cancer Stem Cells
8) Paul Knoepfler: Tumor Formation in Embryonic Stem Cells
9) Huntington’s Disease: Progress and Promise in Stem Cell Research
10) CIRM Major Facilities Speed Stem Cell Science and Create Jobs
I’m going to give an editor’s choice award to a video we made about our Bridges to Stem Cell Research program. The passion these students have for stem cell research and for their future careers is exciting to see:
CIRM Bridges Award: Building California’s Stem Cell Research Workforce
A good report about bone marrow transplantation in progress comes from the National Hockey League, of all places. Mandi Schwartz, a Yale women’s hockey player, was diagnosed with acute myeloid leukemia and is being treated at the Seattle Cancer Care Alliance.
In the name of full disclosure, that’s the center that cured my mother’s lymphoma, so let’s just say I’m a fan.
NHL.com wrote a nice description of how bone marrow transplantation knocks out leukemia stem cells. Acute myeloid leukemia is a cancer that begins in the bone marrow stem cells — the cells that continuously produce new blood and immune cells throughout a person’s life. A bone marrow transplant essentially replaces the cancerous leukemia stem cells with new ones from a donor, like an organ transplant but with bone marrow. The NHL writes:
Engraftment, which was the next phase in her recovery, is needed in order for the transplanted stem cells to begin to grow in her bone marrow and manufacture new blood cells and immune cells… Complete recovery of a new immune system can take a year or longer depending on any complications as a result of the transplant.
Bone marrow transplants like this one are effective, but dangerous. The process of eliminating a person’s diseased bone marrow leaves the person extremely weak and prone to infections. This danger is why several CIRM grantees are working on a less toxic way of killing off the diseased bone marrow stem cells (here’s a list of our awards targeting blood cancers). Irving Weissman at Stanford University has found molecules on the surface of the stem cells underlying acute myelogenous leukemia. He has a CIRM disease team award to develop a chemotherapy that could destroy those cells in a way that’s far less toxic than bone marrow transplant.
If that research is successful, future people like Schwartz may recover from acute myelogenous leukemia with fewer side effects. As someone who has seen a family member battle cancer, fewer side effects for an effective therapy is a winning combination.
Two interesting reports today discuss the relationship between basic research and the kind of translational research that is the most visible sign of progress toward cures.
In his blog, the director of the bay area biotech collaborative QB3 Regis Kelly writes about public speakers at the Translational Medicine Alliance speaking out against basic research. He says:
Repeatedly mentioned with disdain was the amount of money that went into R01 grants, the single investigator grants that are the backbone of fundamental research in the US. Over 80% of the grants go to R01s, it was said, while only 2 to 5% go to translational research, the mantra of the meeting participants.
This is the same type of research funded by CIRM’s Basic Biology Awards and in some New Faculty Awards. The basic discoveries that come out of this kind of research are considered to be the fuel in the pipeline leading to new cures: No new ideas? No new therapies.
Kelly advocates rather than ignoring the complaints, “put our house in order and mount a major public education campaign to validate our position.” That is, do what can be done to make basic research as efficient and effective as possible then explain to members of the public just what they are getting for their money.
Or we can discount the criticisms as uninformed foolishness, and do nothing. That could be suicidal!
Kelly’s point is a good one. Most people have at least one friend or family member with a critical disease, and those people want to see new cures coming from publically funded research. Until the relationship between basic research and new cures is made clearer people will likely continue pushing for less basic research.
While Kelly defends basic research, the head of the National Cancer Institute Harold Varmus held a brainstorming session to figure out the basic questions in cancer research. According to Science magazine:
Once the list is finished, Varmus might hold a special competition to invite proposals for several questions and fund, say, 15 of the best ideas. Research funding may be tight, but “we’ve got over a $5 billion budget,” Varmus says. Nothing has been decided, though. Right now, he says, “we’re just trying to have a conversation that evolves into something useful.”
Stanford’s Irv Weissman, who was at Varmus’ session, gave an excellent talk to the CIRM governing board about the value of basic research. In his case, it was a basic discovery about stem cell biology that led to cancer research that’s now moving toward the clinic.
One amazing aspect of living in the era of social media is the incredible way information spreads. A butterfly batting its little orange wings in a monarch grove in Santa Cruz could influence a tweet of a blogger heard ‘round the world.
Or, in CIRM’s case, a few videos playing on YouTube could be used by a teacher heard ’round the world. In the past week a video about the difficulties of differentiating stem cells into therapeutically useful cell types has popped up in the curriculum of Harrison College, which offers a number of online and classroom courses. The video, which has been watched hundreds of times in the past week by those students, features Mark Mercola of Sanford-Burnham Medical Research Institute who is working to differentiate cardiac cells from human embryonic stem cells. Here’s that video:
In the past, a video about iPS cells featuring Jerome Zack from UCLA has made its way into college curricula, as has a video discussing the different types of stem cells with Stanford University’s Irv Weissman. These videos are all part of a stem cell basics CIRM put together to help educate people about stem cell research both in written form and in short videos.
Given the misperceptions of stem cell research in the public and in the media its nice to see these videos getting discovered and used for educational purposes.
|On left and right, Berkeley Stem Cell Center co-directors
David Schaffer and Randy Shekman, and center,
Mary West, manager of the new lab. (Photo by Jan Ambrosini)
Berkeley is the most recent institution to open new stem cell space funded by CIRM. Their CIRM-funded stem cell facility, which had its opening Oct. 5, is also a core facility for QB3, a bay area biotech incubator. David Shaffer, co-director of the Berkeley Stem Cell Center, said of the facility:
“The new facility will serve as a central resource to greatly enhance stem cell research amongst Berkeley and QB3 investigators, as well as collaborators at Lawrence Berkeley National Lab and Children’s Hospital Oakland Research Institute.”
To date, University of California campuses at Irvine and Davis have both opened their new stem call buildings amidst much fanfare. By the end of October, UCLA, University of Southern California, and Stanford will all have cut their respective ribbons.
These buildings are, to a one, beautiful, gleaming, well equipped centers for cutting edge research. But they are more than that. They are also a safe haven for stem cell research, protected from the ups and downs of federal funding. CIRM first dreamed up and approved funding for these stem cell buildings when President Bush was in office and most stem cell research had to occur in isolation from the microscopes, the pipettes, the refrigerators, the reagents, and the latex gloves most labs purchase with their NIH funds. The research had to take place in space and on lab benches supported only through private or state dollars.
That space was hard to come by, making the early days of stem cell research a considerable challenge. Take Susan Fisher at UCSF who lost her stem cell lines to a power outage while working in a converted dentist office in San Francisco in order to put distance between her cells and federal dollars. (Here’s a video about Fisher’s experience)
In the past year President Obama opened up federal funding for more stem cell research, but now recent events put that funding back in question. During this time of uncertainty, it’s reassuring to know that so many institutions in California have space where their work toward new therapies can continue uninterrupted by political turmoil.
Today CIRM began a three-day external review of its activities. It began with a public session which included what might be the best analogy ever for why we need to continue working with all types of stem cells: adult, cancer, iPS and embryonic.
Rodney Paul, who has type 1 diabetes, showed up to discuss his hopes for a future cure. 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.
Will one stem cell type turn out to be most effective? Maybe. Do we know which one that is? Not yet.
That’s why CIRM funds all types of stem cell research in the hopes of reaching new cures as quickly as possible. You can find out more about the different stem cell types used in CIRM research projects in our searchable table.
Today Geron announced that the first patient had been treated in their groundbreaking trial based on human embryonic stem cells.
At first blush: Woo hoo!! This is the first test of technology that holds hope for millions of people living with spinal cord injury and other diseases.
But it’s important to remember that this first trial is a very, very early step. Any potential therapy goes through three rounds of human trials. The first, phase I, trial is intended only to verify that the potential therapy is safe. This trial can establish dosing and begin to monitor side effects of the drug or treatment. In some cases a phase I trial will also begin to reveal whether the drug or therapy has signs of effectiveness, but that’s not the primary role of the trial.
After the small phase I trial comes a slightly larger phase II trial. It confirms safety and also begins to look directly at whether the drug or therapy is effective.
It isn’t until some years later that a large phase III trial confirms whether the drug or therapy is effective compared to standard therapy.
Many potential therapies fail after the phase I trial, even after years of positive results in animals. Humans aren’t mice (or other laboratory animals) and scientists can’t ever know what side effects will turn up in their human patients.
That’s why it is important to temper that initial “woo hoo” with a bit of patience. This could be one of those trials that fails phase I. Or phase II. Or phase III. But as many years of biomedical research have shown, for every failed trial there are also a number of successful trials. Whatever the results of this first phase I trial, it’s the beginning of a new era that will one day result in the first successful therapy based on human embryonic stem cells. That day will have scientists and patients alike whooping for joy.
Here’s patient advocate Roman Reed talking about what a stem cell therapy for spinal cord injury means to him:
Nature Medicine carried a piece Friday by CIRM governing board member Jeff Sheehy, writing about the importance of having a patient advocate voice in biomedical research. Sheehy, who is living with HIV, is a long-time advocate for HIV/AIDS research. He has been on the CIRM board since the beginning in November 2004, and is a vocal participant in CIRM working groups including the group that makes research funding recommendations to the full board (the Grants Working Group), for which he is vice-chair.
The presence of vocal, engaged patient advocates has added an indispensable dimension to the proceedings. In measuring research quality, advocates tend to focus on a project’s ability to benefit people—not just drive scientific curiosity—which keeps even basic biomedical research grounded in its ability to produce concrete health benefits.
CIRM’s governing board includes 12 patient advocates representing HIV/AIDS, MS, diabetes (type 1 and type 2), heart disease, spinal cord injury, cancer, Alzheimer’s disease, Parkinson’s disease and autism. Sheehy goes on to say:
… CIRM is made up of patient advocates from a wide spectrum of diseases and conditions who work together to advance therapies across the board. And contrary to critics’ assertions, these advocates have not narrowly focused on their own diseases, but have uniformly advocated for the best approaches for moving basic research towards the clinic. They support each other.
Patient advocates serve a powerful role even if they aren’t directly involved in funding decisions. Don Reed has been a vocal supporter of stem cell research since his son Roman Reed suffered a spinal cord injury. He is sponsor of the Roman Reed Spinal Cord Injury Research Act that funds spinal cord research in California, founder and co-chair of Californians for Cures and blogger on his own site www.stemcellbattles.org and for the Huffington Post.
In a recent Huffington Post blog entry about the World Stem Cell Summit he wrote about the importance of patient advocates staying involved in stem cell research at a political level, in order to maintain the U.S. leadership in stem cell research:
But unless we in the patient advocacy community can encourage Congress to pass a stem cell research protection act the dream will have been stolen.
CIRM came about in part because of the passion and support of patient advocates like Sheehy and Reed, and they continue to be a crucial part of the success of CIRM and of the progress made in stem cell research.
Here’s Sheehy advocating for a stem cell therapy for HIV/AIDS. CIRM has funded two disease teams (City of Hope and UCLA) focusing on developing therapies for the disease.