CIRM boosts industry outreach efforts in 2011, more to come

Cynthia Schaffer is Contract Administrator and Compliance Officer at CIRM

As CIRM-funded teams move closer to clinical trials, we’re ramping up our engagement with the industry partners who are integral to the success of most future stem cell therapies. In my role supporting many of these endeavors I saw a significant uptick in 2011 in events and activities specifically focusing on these crucial industry groups.

For the second year in a row, CIRM organized webinars and roundtables with the FDA, industry and investigators from academia. These events focused on the challenges and/or roadblocks on the regulatory pathway to develop stem cell based therapies. Topics of these webinars included Imaging in May, Scaffolding in September and Immune Response in October.

Through our active engagement with the Alliance for Regenerative Medicine (ARM), CIRM sponsored the first ever regenerative medicine investor partnering event in November in conjunction with CONNECT and the Stem Cell Meeting on the Mesa. My colleague Elona Baum, CIRM General Counsel and Vice President of Business Development, was one of the primary organizers of this event, which was attended by more than 240 regenerative medicine professionals and featured 40 company presentations and a full day of panel discussions addressing key business topics. Participants came from across the regenerative medicine sector including big pharma, investors, leaders from Europe and Asia, therapeutic and platform companies, investigators and representatives from several prominent CIRM disease teams.

As part of the update to CIRM’s Strategic Plan, CIRM held four public meetings in October, two of which specifically targeted input from the for-profit sector of the Regenerative Medicine industry. The Strategic Plan update will be presented to the board at the upcoming governing board meeting January 17 in San Diego (location and audiocast details are available here).

At CIRM’s Grantee Meeting in September, 12 vendors from various for-profit companies who serve the regenerative medicine industry were able to exhibit their wares. This program was well received and we plan to incorporate this element again at our next CIRM Grantee Meeting in 2013.

In 2011 CIRM hosted two webinars on its new Disease Team Therapy RFA to help potential applicants understand CIRM’s expectations for their proposals and budgeting materials. These webinars append the 2010 Grant Writing webinar (archived on CIRM’s website) and similar curriculum is planned for 2012.

Also this year, CIRM allocated a significant $57 million to be invested in initiatives that will make the organization more flexible in seizing funding opportunities and partnering with industry (we blogged about those initiatives here). Baum said:

“We were very pleased with the Board’s approval in December of all three components of the Opportunity Fund: Bridge Funding ($12 million), External Innovation ($15 million) and the Strategic Partnership program ($30 million). We believe the Opportunity Fund and its components will give us increased flexibility in responding to opportunities that arise in the Regenerative Medicine industry.”

In addition to resources I link to here, I would specifically like to call your attention to the materials on Resources for Researchers page.

If you want to be alerted about upcoming industry events, you can sign up to receive notifications for all upcoming meetings here.

C.S.

CIRM-funded building at UC Irvine receives LEED Platinum certification

Michelle Kim / University Communications

In May 2010 University of California Irvine opened their new stem cell building funded in part by CIRM. In addition to its state-of-the art research facilities designed to speed the development of stem cell therapies, the Sue & Bill Gross Hall also recently received LEED Platinum certification from the U.S. Green Building Council.

According to a press release from UCI:

Gross Hall scored points for such features as dimmable and occupancy-controlled lighting, use of energy-saving building materials, and mechanical systems that are 50 percent more efficient than required by California’s Title 24 energy code. The operable windows are tied into the heating and air-conditioning controls so that when a window is opened, mechanical ventilation of that room shuts off.

An $80 million, 100,000-square-foot structure, Gross Hall was designed to facilitate contact between patients in the first-floor clinic and rehabilitation center and stem cell researchers on the first, second and third floors. Labs are equipped with Aircuity technology that monitors indoor air quality and adjusts air-change rates based on contaminant levels.

UCI received $27 million from CIRM for the building and a $10 million from Sue & Bill Gross. The remaining funds came from donations and institutional commitments leveraged by CIRM’s investment. This is one of the 12 buildings supported through CIRM’s Major Facilities programs. The complete list of those buildings is available here. The 12 major facilities received $272 million from CIRM, with private donations and institutional investments bringing the project totals to more than $1 billion. An independent review of the impact of this investment for the state economy suggested that the projects would create 13,000 job years of employment and $100 million in tax revenue.

A.A.

60 Minutes highlights concerns about clinics marketing unverified stem cell treatments

Last night, 60 minutes aired a report describing the dangers of what has become known as stem cell tourism – people traveling to other countries to receive unverified “treatments”. You can watch the episode here. In the story, they spoke with Gary Susser, whose son Adam has cerebral palsy. Susser and his wife took Adam to a doctor in Mexico, who injected Adam with stem cells but provided no evidence that the injections would be effective. They were not. In the story Susser said:

“I see how people are preyed upon by hucksters and charlatans. And people who have a special child don’t need any more expense, don’t need any more heartache, and don’t need any more false promises. They need the truth and they need hope.”

Susser and his wife worked with 60 minutes to investigate a clinic in Ecuador that advertises therapies for 70 incurable diseases including cerebral palsy.

This trend of overseas clinics offering “modern day miracles,” as the Ecuadorian clinic’s website promises, is one that has concerned both CIRM and the International Society for Stem Cell Research (ISSCR). In 2010, when the ISSCR held its annual meeting in San Francisco, CIRM and ISSCR co-hosted a symposium about these clinics and the concerns they raise. A video of that session is available on the CIRM website.

At that same meeting, then-head of the ISSCR Irv Weissman used his opening address to both tout a new ISSCR website designed to help people identify clinics offering true therapies (A Closer Look at Stem Cell Treatments), and also discussed what he saw as the dangers of stem cell tourism. In countries without strong oversight bodies such as the Food and Drug Administration in the U.S., clinics can make claims about potential therapies without backing those claims up with any proof that the procedure will work or even be safe. An investigational treatment offered for sale, which is what these clinics described on 60 minutes are offering, is not the same as a clinical trial, which would show whether the new treatments are safe and effective.

In the 60 minutes piece, the clinic under investigation had proposed injecting cells that, when scrutinized by Dr. Joanne Kurtzberg of Duke University, turned out to be disintegrating. She said:

“There are huge dangers if you injected that into someone’s blood or spinal fluid because all these little fragments and debris would get trapped somewhere in the blood stream and could cause a stroke, or in the brain could cause an inflammatory reaction.”

Weissman spoke with Stanford’s Krista Conger, who wrote a story on the topic for the magazine Stanford Medicine. In it Weissman said:

“This is a new field. We are learning what kinds of stem cells can regenerate which kinds of failing organs. But we always remember that our first goal is to do no harm. That’s why we begin our tests in animals before moving into early phase clinical trials in humans, and at every stage we verify the solidity and reproducibility of the science and that the investigators have no potential commercial or personal conflicts of interest that could influence the outcome of the trials. Any attempt to commercialize unproven treatments in the absence of independent or regulatory oversight endangers not only the lives of those who receive the treatments, but also the entire field of regenerative medicine.”

Conger also quoted CIRM grantee Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute in La Jolla, Calif.:

“When we report something good about stem cells, it gets picked up in the media, or in a blog that patients read. It gives them more ammunition to say that the FDA is stupid for denying access to treatments that seem like they should work.”

At CIRM, we know how frustrating the timeline of developing new therapies can be. We also have friends and family members with diseases desperately in need of new therapies. That sense of urgency is constantly reiterated by our board members, ten of whom serve on the board as patient advocates. (Our board member Jeff Sheehy has blogged about the role of advocates in accelerating the pace of new therapies.)

That’s why we’re working with industry, pushing teams to collaborate, and engaging the FDA to try to speed that timeline and get new therapies to the patients who need them. Today we have 43 projects in various stages of working their way to the clinic, with several we hope to see enter clinical trials in the next few years. You can read more about our projects in development and the steps along the path to a new therapy on our website.

If people are considering clinics outside the U.S., please do read the ISSCR web page. They have a good list of qualifications to look for in identifying clinics that are being truthful about what they offer rather than simply peddling hope. Included in what they suggest people look for is oversight of investigational treatments to be sure the physicians are qualified, the investigational treatment is prepared appropriately, and that the risks and potential benefits are accurately and clearly explained. People should also look for published records showing results from clinical trials. CIRM also has a page about stem cell tourism and what we are doing to try to speed the timeline to new therapies.

A.A.

CIRM’s 1,000th published paper targets Huntington’s disease

CIRM recently realized a noteworthy milestone with the publication of the 1,000th CIRM-funded paper in early December (here is a link to that paper). This is exciting to me personally because one of my first assignments when I joined CIRM three years ago was to develop (in collaboration with my Science Office colleague Rahul Thakar) a system and database for tracking CIRM-funded publications. At that point, there were only around 100 CIRM-funded papers, mostly from training grants, which were the first grants CIRM awarded in the spring of 2006. Watching the database grow and the publication rate accelerate has been exciting because these papers describe real progress that CIRM grantees are making in understanding stem cell biology, mechanisms of human disease and advancing stem cell-based therapies to the clinic.

The 1,000th paper itself is a great example of this progress. It was published in the journal Molecular & Cellular Neuroscience by Drs. Scott Olson, Jan Nolta and colleagues at UC Davis with the title “Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington’s disease affected neuronal cells for reduction of huntingtin”. The fact that the thousandth journal article comes from Jan Nolta’s lab has added significance given that she was just yesterday named editor of one of the leading specialty journals in the field Stem Cells.

The promising research described in this paper was funded in large part by a CIRM Early Translational grant to Nolta, which has the goal of developing a combination cell and gene therapy candidate to treat Huntington’s Disease, a devastating neurological disease for which there is no effective treatment. Among those who have suffered and died from Huntington’s is one of my favorite songwriters, Woody Guthrie, whose 100th birthday will be celebrated in July. There’s more information about Huntington’s disease and awards we’ve funded available on our website.

In Huntington’s Disease, a mutated form of a protein called huntingtin causes certain neurons in the brain to die. The goal of Nolta’s award is to use stem cells to disable the ability of those neurons to make the disease-causing protein. In their paper, Nolta and colleagues show that a type of stem cell found in the bone marrow called mesenchymal stem cells (MSCs) can be engineered to produce a molecule that inhibits the production of the mutated huntingtin protein. They show, in a petri dish, that this molecule is secreted by the stem cells, is taken up by nearby neuronal cells, and reduces the amount of the disease-causing protein produced by those cells. The next important step will be testing these engineered stem cells in an animal model of Huntington’s.

With over a thousand CIRM-funded publications come a lot of data and statistics that allow us to assess our programs and progress. Of the 1,009 CIRM-funded publications, 770 describe original research, 175 are literature reviews and 64 describe novel scientific methods. The top four RFAs by publication number are, not surprisingly, the four earliest in CIRM’s funding history: Training grants with 574 publications, New Faculty with 178, Comprehensive with 169 and SEED with 160. CIRM funding has resulted in an impressive number of papers in elite journals: 55 in Nature, 47 in Cell Stem Cell, 24 in Cell and 13 in Science. In total,298 CIRM-funded papers have been published in journals with impact factors greater than 10. There were 10 CIRM-funded publications in 2006, 68 in 2007, 151 in 2008, 224 in 2009, 261 in 2010, and 295 in 2011, so I predict that I’ll be back to blog about the 2,000th paper before the end of 2014!

Zachary Scheiner is a Science Officer at CIRM

ResearchBlogging.orgOlson SD, Kambal A, Pollock K, Mitchell GM, Stewart H, Kalomoiris S, Cary W, Nacey C, Pepper K, & Nolta JA (2011). Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington’s disease affected neuronal cells for reduction of huntingtin. Molecular and cellular neurosciences PMID: 22198539

Looking ahead to 2012

Yesterday CIRM president Alan Trounson took a look back over his science picks from 2011. Today, we’re looking ahead to what’s in store in 2012.

First, anyone interested in participating in the 2012 board meetings should take a look at the governing board meeting schedule. That’s available here. We’ll be having seven meetings at locations throughout California. As always, you can see any scheduled subcommittee or working group meetings on this page and you can also sign up to receive email notifications whenever we post new agendas. That sign up is here.

Those meetings will be action-packed in 2012, with $370 million in new awards slated for review by the governing board and a new strategic plan that will help guide the agency’s priorities going forward. The awards scheduled for review this year include the next round of Creativity Awards worth $2.2 million, which fund high school students to carry out research in California stem cell labs. Last year’s students who participated in our pilot program were outstanding, as you can see in this video:

The governing board will also be considering research awards that cover the complete therapy development pipeline (you can read more about that development pipeline on our website). The fourth round of Basic Biology awards, worth up to a total of $35 million, will continue CIRM’s commitment to funding the basic discoveries that fuel new therapy ideas and also help resolve scientific issues that arise further down the pipeline.

The Early Translational III awards, worth up to a total of $95 million, will fund researchers who are in the early stages of translating a basic discovery into a future therapy. Some of our past recipients of Early Translational Awards have had such success that they are now applying for our second round of Disease Team Awards, worth up to $240 million. As with the past round of these awards, the disease teams include people with clinical, research and regulatory expertise who work together to bring their therapy candidate to clinical trials.

If you don’t already receive CIRM press releases or monthly newsletters you can sign up here. They’ll include information about awards we’ve funded and other decisions by the governing board.

A.A.

Guest blogger Alan Trounson — December’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 full report, along with an archive of past reports, is available on the CIRM website.

My review this month starts with a couple papers that further cement the high value of reprogrammed iPS cells as “disease-in-a-dish” models. This time they found the underlying mechanisms of the autism seen in Timothy Syndrome (blogged about here) and the bone and cartilage abnormalities seen in Marfan Syndrome.

But I want to take this space to note a different type of paper, one discussing a single patient, but a patient that would probably not be alive today without stem cells and a very creative international team of scientists. The team headed by a group at the Karolinska Institute in Sweden also had inputs from researchers in England, Germany and Iceland.

The patient had recurrent cancer in his trachea and little hope with traditional therapy, so the team built him a new airway. They first imaged his diseased trachea with a CT scan and then used that picture to build an artificial polymer scaffold of the same size and shape. They seeded this replica with stem cells from the patient’s own bone marrow and grew it in a bioreactor for 36 hours.

The new trachea was functional at the time of transplant, but the team gave the patient two growth factors to enhance the maturation of the airway. It seems like the patient’s own repair systems also kicked in because the team was able to detect other stem cells summoned to the site and other growth factors released by neighboring cells.

The patient is alive and cancer-free five months after the surgery. He is living “proof of concept” for an entire branch of regenerative medicine that seeks to build replacement tissues to order. We blogged about this advance here.

I began blogging about my science picks just a few months ago. You can read my 2011 science highlights in review in the following blog entries:

A.T.