CIRM Sponsoring Online Session with FDA on Thursday

One of the lesser known activities of the California stem cell agency is webinars that put researchers together with the folks who make the federal decisions about whether stem cell research will be turned into therapies.

One of those sessions is coming up on Thursday, and it is not too late for scientists and other interested parties to get on board.

Writing on the stem cell agency's blog, Cynthia Schaffer, CIRM's contract administrator and compliance officer had this to say today about the webinars.

“The FDA very graciously donates their time to speak on these webinars because they too have pledged to maintain an active dialogue with the industry and provide education on their regulatory expectations for product development in the regenerative medicine field. CIRM science officer Kevin Whittlesey recently wrote a paper with Celia Witten of the FDA about the role of the FDA in reaching out to regenerative medicine community, including webinars such as these. 

“In that paper they point out that the communication goes both ways:

“'Appropriate regulation requires a strong understanding of the latest scientific developments to meet current and future regulatory needs and challenges.'

“So the FDA benefits by learning from the other speakers in the webinar – what is the current state of the technology, what are investigator’s current thoughts on best practices and the latest research findings, etc. They also learn what the industry is facing by listening to the questions asked and the discussion of the challenges during the Q&A sessions. A group of FDA employees attend each of these CIRM sponsored webinars, and the wide variety of other workshops and meetings that CIRM hosts throughout the year.”  

(Editor's note: An earlier version of this item incorrectly identified Cynthia Schaffer as Cynthia Adams.)

New Collaborative Agreement Announced Between California Stem Cell Agency and NIH

The California stem cell agency and the NIH today announced a collaborative agreement aimed at accelerating the transformation of basic research into cures.

The agreement came up several times during this morning's discussion of changes in CIRM's strategic plan in the context of leveraging the agency's efforts. Some directors indicated that the agreement could mean that CIRM could spend less on costly translational grant rounds. Here are some excerpts from the agency's press release.

"The memorandum of understanding signed by CIRM President Alan Trounson and Michael Gottesman, Deputy Director for Intramural Research at NIH, is a pilot project of these partnerships in clinical and translational research. This MOU establishes a framework to advance the complementary and synergistic goals of CIRM and the NIH to 'help NIH and CIRM researchers work together to bring their special talents in stem cell and regenerative medicine research to bear upon prevention, diagnosis, and treatment of various diseases.'"

"The NIH, through its trans-NIH Center for Regenerative Medicine (NIH CRM), the NIH Clinical Center, and the newly established NIH Center for Translational Therapeutics (NCTT), will foster these interactions. The NIH Clinical Center is the world’s largest hospital dedicated entirely to clinical research. The Center houses basic, translational and clinical research efforts of the NIH intramural research community, including the recently established NIH CRM funded through the NIH Common Fund."

The press release also mentioned possible opportunities for collaboration. It said,

"One relates to the current round of applications for CIRM’s Disease Team Therapy Development Research awards. During the planning phase of these awards, California teams could develop collaborations with researchers at the NIH Clinical Center on various aspects of a preclinical, Phase I, Phase I/II or Phase II clinical trial. Another option is to enable California researchers with the opportunity to take part in NIH Clinical Center visiting fellowship or clinical investigator training programs. A third is to access unique resources they could apply to use that may not be available at their home institution."

Andy Grove: More on Speeding Cures

Recently we carried an item on Andy Grove's views on translational medicine, a subject that now deeply engages the California stem cell community. Today, the San Jose Mercury News ran an interview with Grove, a Silicon Valley pioneer, former CEO of Intel and currently a patient advocate. The piece dealt with his campaign to generate therapies more quickly as well providing a glimpse into his current life.

The article by Lisa Krieger is well worth reading. Here are some excerpts.

"He handles (Parkinson's) disease the way he handles everything else: open and direct. He adopts none of the common tricks employed by sufferers -- clutching a pen, or stuffing hands into pockets -- often used to minimize its visibility. His torso keels with each step, a distorted but determined gait. His arms bob; his fingers fidget.

"Underneath is keen intellect and a growing sense of frustration that medical science has offered him and other patients so little after decades of experimentation."

Krieger continued,

"'He's turned his own encounters into problems to be solved,' said Robert A. Burgelman, professor of management at the Stanford University Graduate School of Business. "He takes a strategic approach.

"'He's an extraordinary man, one of the most focused persons I have every come across. Once he comes to an insight, he follows through on it, turning it into action,' he said. 'And he has an ability to reflect on his own experience and draw insights from that, which is an unusual and powerful thing for leaders to have.'"

Montana Lad Speaks Out on Scientists, Stem Cells and Obama

Nature Reports Stem Cells this week carried an interview with the son of some fur traders and junk dealers on the subject of stem cells.

Among other things, he said most scientists don't know what medicine is and then took on President Obama concerning the new NIH rules on stem cell research.

The comments came from Irv Weissman of Stanford in a Q&A with Monya Baker, editor of Nature Reports Stem Cells. The interview keyed on his role as the new president of the International Society for Stem Cell Research.

Here is the section on scientists and medicine.

Baker: "Why is it so hard to move science into medicine?"

Weissman: "First, most scientists don't know what medicine is. They don't know what whole-body physiology or pathology [is]. So they tend to be, at least at the beginning, unrealistic in their expectations. Second, there are not many people trained to do clinical trials.

"And you have to realize that things don't make it into clinical, commercial therapies without a lot of money."

He spoke about overcoming nontechnical barriers in the stem cell field.

“(G)going from labs to commercial and clinical products, we need to understand how it could be a commercially viable business. Stem cells, unlike drugs or proteins, self-renew and differentiate in a fashion regulated by the body; they regenerate systems for life from a single therapy. If you're going to deliver stem cells for lifetime therapy, the cost of goods and reasonable profit has to be priced with the knowledge it replaces daily therapies like insulin or blood transfusions.

“One of the greatest experiments I know of is being done in California. The California Institute of Regenerative Medicine [CIRM] sent out last year an RFA [request for applications] for disease teams to take stem cells and/or therapies derived from them through preclinical right up to the filing of the first IND [investigational new drug application] with the FDA. That's an area that was formerly funded only by companies. Now universities and other nonprofit institutions need to learn what companies do, including rigorous manufacturing practices [GMP] and FDA-compliant regulatory documents.”

Weissman also answered a question about how he came to be a scientist:

“I was born and brought up in Great Falls, Montana. My father and grandfather were fur traders and junk dealers. I was about to be the first in those generations to get an education. When I was ten years old I read a book called Microbe Hunters, which was about the lives of people like Robert Koch and Louis Pasteur. It was incredibly exciting to me.

“I really looked for a way to do research after that. I should say I was never a straight A student. When I was about 16, I met Ernst Eichwald at the local hospital. Ernst was German, his father was Jewish.

“During the beginnings of WWII Ernst belonged to a student group against the Nazis; somebody in his group was arrested, and without going home he crossed the border. He came to Harvard, he was on faculty there; he came to Utah, was on faculty there for awhile. He got sick of academic politics and decided to run the pathology service at the Montana Deaconess Hospital. Very early he handed me a paper [George Snell's paper on histocompatibility genes] as if I could read it, and I told him that I didn't understand the first word! He spent a whole Saturday with me, and we covered just that one paper.

“I realized that the language of science substitutes Greek and Latin terms for plain English, that it was a way to keep people out of the field. He didn't need to say histocompatibility. He could say "tissue-transplantation compatibility", but I also realized I could understand it. Thanks to the freedom Dr. Eichwald gave me, I began designing my own experiments at 16.”

CIRM Approves $68 Million in Grants; Wrestles with Executive Evaluation

LOS ANGELES -- The California stem cell agency today approved $68 million in grants for early translational research and left open the possibility that more might be awarded in June.

CIRM said in a news release,

"The 15 early translational grants approved by the board will go to 13 not-for-profit and two for-profit organizations. These grants are intended to either lead to a drug candidate for an unmet medical need or address a bottleneck in the development of new therapies."

CIRM President Alan Trounson was quoted as saying,

“With these early translational grants CIRM has taken the first step in funding translational research that will be critical for the development of future therapies.”

CIRM has talked about the importance of making grants to business. In this round, Novocell of San Diego, received $5.4 million and BioTime of Alameda, Ca., received $4.7 million. You can see a complete list of the approved applicants here.

Twelve additional grants had been recommended for funding by reviewers – if funds were available. But the board decided to put off a decision on those until June in hopes that CIRM will have an improved financial situation.

The board took no action on four letters from applicants seeking to reverse negative decisions by reviewers, but it rejected all applications ranked in the bottom tier. You can find the text of the petitions via the meeting agenda.

The board additionally approved $200,000 to co-sponsor the annual convention of the International Society for Stem Cell Research in San Francisco during the summer of 2010. John M. Simpson, stem cell project director for Consumer Watchdog of Santa Monica, Ca., supported the move although he was critical when the subject was first broached last December with a $400,000 price tag. Simpson praised as thorough the CIRM staff justification for the expenditure.

In other business, CIRM directors approved an evaluation process for the CIRM chair, vice chairs and president – the first such in the four-year history of the organization. The plan stirred debate concerning the composition and chairmanship of a new Evaluation Subcommittee of directors.

Some directors objected to permitting the Evaluation Subcommittee, which includes three of the four persons to be evaluated, decide who chairs the panel. Board member Jeff Sheehy, director for communications at the AIDS Research Institute at UC San Francisco, moved to designate the chair and vice chair of the Governance Subcommittee to fill the same positions on the Evaluation Subcommittee. Sherry Lansing, a UC regent and former movie studio CEO, and Claire Pomeroy, dean of the UC Davis School of Medicine, are chair and vice chair of the governance panel. They were key to working out the evaluation process.

Sheehy said it was not appropriate for the people being evaluated to have a voice in picking the chair of the Evaluation Subcommittee.

Klein, who will sit on the Evaluation Subcommittee, opposed Sheehy's motion although he indicated he was willing to permit the full board to select the heads of the evaluation panel. Sheehy's motion failed on a vote of 5-15 with four self-recusals and two abstentions.

Directors Robert Azziz, chairman of the department of obstetrics and gynecology at Cedars of Sinai in Los Angeles, and Carmen Puliafito, dean of the USC School of Medicine, expressed concern about the composition of the committee. Azziz said he wanted to avoid the perception of a conflict of interest. Puliafito said that the membership could give the appearance of "self-dealing."

The process was finally approved on an 18-5 vote with one self-recusal and two abstentions.

The board will make a decision on the chairs at a later date.

Here is the structure of the subcommittee as presented during today's board meeting. It will include:

• The chair and vice chairs of the Governance(Sherry Lansing), Finance (Michael Goldberg) and Legislative(CIRM Chairman Robert Klein) directors subcommittees.
• The chairs of the IP Task Force(Ed Penhoet) and the Biotech Loan Task Force (Duane Roth, who is also vice chair of the CIRM board of directors).
• The vice chairs or acting vice chair or co-chair where applicable of the Grants(Joan Samuelson or Sheehy), Standards (Lansing), Facilities (David Serrano Sewell) working groups.
• Two members appointed by the board but not yet named.
• The chair (Klein) and vice chairs (Roth and Art Torres)of the board except when they are the subject of an evaluation.

Potholes in Freeways vs. Therapies in the Clinic

"Where are the cures?" is the headline on the piece in the Nov. 10 issue of Newsweek.

The article by science columnist Sharon Begley virtually cried out for a sidebar on California.

Begley wrote about the "valley of death," translational research and the need for industrial skills that can make the production of stem cell therapies economic.

She quoted Hans Kierstead of UC Irvine, mentioned Geron of Menlo Park, Ca., as well as a proposal to create a "center for cures" at the NIH. Along the way, she noted that scientists involved in basic research are wary of the "center" proposal – a feeling that has surfaced indirectly at the California stem cell agency.

But Begley said:

"The existence of such a center would free scientists to go back to making important discoveries, not figuring out large-scale pipetting, for goodness' sake."

All of what Begley wrote about is on the $3 billion plate at the California stem cell agency. And some of the CIRM actions are coming quite soon. The "valley of death," for example, is scheduled to be dealt with next month through a $500 million lending program. Waiting for action from the new presidential administration is not good enough for CIRM Chairman Robert Klein and company.

As Begley concluded:

"There is lots of talk these days about increasing the nation's spending on infrastructure, such as roads and bridges, to lift the economy out of its doldrums. Me, I'd be willing to put up with potholes in exchange for a new administration spending serious money to take the discoveries taxpayers have paid for and turn them into cures."

Amy Daly Statement on Bridge to Cures

We queried Amy Daly, executive director of the Americans for Stem Cell Therapies and Cures, concerning the proposal for a land development project and stem cell research center near Sacramento. Here is her response verbatim.

Bob and Angelo had been spending time talking about this project to fund therapy development around the same time that Americans for Stem Cell Therapies & Cures was holding a fundraising dinner at the home of Robin and Marsha Williams to retire the outstanding campaign debt. The suggested donation for a couple to attend this dinner was $125,000. Angelo is very supportive of stem cell research (as you can see by his incredible generosity with this project) and so he and his wife attended this dinner.

As far as the project referred to in the Sacramento Bee today, there are
many details that were discussed but not included in the article.

"No one tried to strangle Herceptin, but it came near to starving in the
cradle."

As you may be aware, there is a gap in funding in the life of therapy
development where many good ideas for therapies and cures die for lack of
funding. It is this funding gap that we hope to address with this project.
California Institute for Regenerative Medicine has focused (and we believe
will continue to focus) much of its funding to fill the funding gap in early
research. We hope to take the ideas that come from early research and safely
see them to the point in time where they are picked up by biotech and VCs.
An example of this funding gap is found in the history of the development of
Herceptin, as noted here:
http://www.nytimes.com/books/98/09/20/reviews/980920.20henigt.html?_r=1&oref
=slogin.

Without philanthropic financial support for Herceptin during that funding
gap, it would not have been developed to the point where Genentech picked it
up. It is now the number one choice for the treatment of certain types of
breast cancer and saves countless lives.

This project will create a non-profit to bridge that funding gap and we
believe it will be called Bridge to Cures. The family donating the land and
endowment funds, the Tsakopoulos family, have asked Bob to chair the board
of Bridge to Cures. The structure of the board will be similar to the
Independent Citizens Oversight Committee with the addition of Angelo and
Kyriakos Tsakopoulos and Eleni Tsakopoulos-Kounalakis. We expect that the
board will choose to have working groups make recommendations to them
similar to the working groups of California Institute for Regenerative
Medicine. We hope to inspire international collaboration by having board
members from world-class institutions from around the world. These
institutions, as well as UC Davis and other California institutions, will
also have the opportunity to have satellite lab space near the incubator
space that we are building for the therapy development.

This is an incredible opportunity to help further stem cell research and
mitigate human suffering and I am proud that our non-profit has chosen to
support this effort.