After the Lights Go Out at CIRM

What happens when the California stem cell party is over? That's one of the questions posed to the agency as it maps a strategy to give away $3 billion. The question is not insignificant. CIRM has only about eight more years left on its clock. The measure that created CIRM gave it a 10-year life, nearly 20 percent of which has expired.

Martin McGlynn, president of StemCells, Inc., of Palo Alto, gave this response:

"I would ask CIRM to clearly define success. Publish a road map to achieve those successes. I would offer two thoughts as to what you might deem success. I look to the notion of what happens when the party is over....:

"There would be Centers of Excellence in the state with a critical mass of world-class talent and a proven track record or diversity at each center in its chosen field of endeavor.

"You would have created a vibrant, sustainable for-profit sector that focuses on applied research and the translation of discoveries into use for the benefit of mankind. Those entities by then should be fundable by more traditional sources of funds.

"The cycle time for the work that needs to be done is twice what VCs wants to see, so get out of blocks quickly, leverage your dollars, and make sure you are the first money to accomplish your mission."

McGlynn was among a number of private sector folks who discussed strategic planning issues last month with CIRM. Their comments are summarized in a 40-page document now available on the Web. The summary adds considerable meat to the skimpy bones of their Power Point presentations.

Here are a few more excerpts.

Bruce Cohen, president and chief executive officer, Cellerant Therapeutics of San Carlos, Ca., on CIRM's goals:

"The taxpayer needs to see a return on this investment. You need to find a way for them to see company and job creation, which will come sooner than cures for some of the debilitating disease we are trying to address. So we need to encourage capital to come in to accelerate new businesses and let existing businesses get bigger.

"You also need to get therapies into the clinic so voters will see a potential change in their lives. You need to accelerate the process by which adult and embryonic stem cells therapies find their way to people. Even if it's an inconclusive Phase I trial, you'll be happy someone with state funding is trying to accelerate the process.

"It's those two things that will make people who voted for this appreciate what’s been done on their behalf."

Sumit K. Chanda, group leader,, Division of Cellular Genomics, Genomics Institute of the Novartis Research Foundation, on core facilities:

"We have started to get into a little bit of stem cell screening. These are the major challenges and possibilities that we have seen using high-throughput screens using stem cells:

"One of the possible solutions that I am proposing is a core facility for screening where everyone in California who is interested in running high-throughput stem cell screens can go. This would be parallel to the NIH roadmap project where they had the MLSCN [Molecular Libraries Screening Centers Network] centers located in different areas in the States.

"The only exception I would make to this is that the NIH had the centers run by academic groups. It is really the private sector that has been making advances in screening in the last 20 years or so, so it might make more sense to have the private sector spearhead a screening facility."

Chanda continued on the subject of cultural roadblocks.

"We have found that there are culture roadblocks between the academic and biotech sectors, even though we have pretty good relationships with various academic groups. We find that the collaborations are very fruitful, but most of our knowledge of what is going on from academic groups comes from publications, which usually do not give you enough information and often occur a couple of years later than the initial discovery."

Ann F. Hanham, managing director, Burrill & Company of San Francisco on "concerns:"

"How much more basic research is going to need to be done? Many of the companies that pitch to our firm are not ready for VC funding yet. They have not worked up their business metrics or their technology or their idea or how much time/resources they need to get to a final product.

"Patents are a huge issue for VCs. We need to protect our investment. If we are going to put capital in, we need to ensure protection of that asset. The University of Wisconsin patents have raised issues about whether we can invest in this field.

"We also need to see a commercialization strategy. Right now, stem cell science is much more on the research side than the development side of the R&D process.
"There are issues to be addressed around manufacturing and the scalability and reproducibility of manufacturing.

"There are questions to be answered such as: Can you pool stem cells? What is the right commercialization strategy? Wow do you become a company and earn back that money that was put in?"

Thomas B. Okarma, president and chief executive officer, Geron Corp. of Menlo Park, Ca.

"My message is going to be straight forward. Because of the depths of your pockets and lack of competition at the state and federal level, we have an opportunity to shape the way the field is developed.

"My take home message to you today is the 'D' word - development. My advice to you is to subordinate research to support development. Don't eliminate the research in R&D, but do the research as necessary to support the 'D.'"