Friday, February 02, 2018

Millions, Billions and Babies: The 2017 Stem Cell Story from California

Cover of CIRM annual report
California's 13-year-old stem cell research program has lured in nearly $390 million in private investment this past year, an accomplishment touted in its just released annual report for 2017.

The 23-page document is chock-a-block with facts and figures about the agency along with upbeat stories about its impact on a handful of patients, young and old.

All told, one could consider the report a key marketing tool for the agency's efforts to stave off its own financial demise, now slated for just two years down the road unless a rescue effort is successful.

The $3 billion stem cell agency, formally known as the California Institute for Regenerative(CIRM), expects to run out of cash for new awards in 2019. Its survival depends on a $200 million-plus, private fund-raising effort now underway and voter approval of a yet-to-be-written, $5 billion bond measure in 2020. The agency has been funded nearly entirely by state bonds, an unusual approach for state agencies, which generally survive on year-to-year appropriations.

The 2017 annual report, which cost $34,000, lays out the case for the agency's work, from clinical trials to patient advocacy. Included is less well-known information about the private investment that

has been drawn in to match awards and to support spin-off companies.

Private investment is critical to translating basic research into therapies and cures. So far, the agency has not delivered on expectations of voters in 2004 that it would produce a treatment that can be used by the general public. But it now has invested in 44 clinical trials and hopes that a therapy will emerge that will resonate with voters.

The agency cited its impact in the private sector with a comment from Deepak Srivastava, president of the Gladstone Institutes in San Francisco.
"CIRM has funded the full pipeline of our work on cardiac regeneration—from basic discoveries, all the way to preclinical studies. As a result of their support, we established Tenaya Therapeutics, a local start-up company that launched with $50 million in Series A investment and aims to tackle heart failure."
Maria Millan, CEO of CIRM since July, said,
"If not for CIRM, many programs currently in clinical trials to address debilitating and fatal medical conditions might have stalled or have been discontinued due to lack of funding. As a result, we are seeing more partnerships and follow-on industry investment—almost $390 million this year—to  advance CIRM-funded programs.
"By investing when others are not yet ready to do so, CIRM’s partnership enables researchers to develop a value proposition that attracts follow-on investors and industry partnerships."
The annual report will undoubtedly be a key document for CIRM Chairman Jonathan Thomas this year as he seeks to raise more than $200 million in private donations to tide the agency over until a bond election in November of 2020.

But the agency has a significant, additional challenge in reaching the general public, a point noted by Robert Klein,  the real estate investment banker who led the 2004 campaign that created the agency.  He cited the "lack of communication" in the mainstream media about CIRM.

Klein told CIRM directors last fall that 90 percent of the science reporters in the media have vanished in the last 20 years. He said that science writers were once the key to telling the story of scientific research as well as illuminating the progress of the quest for stem cell cures. Today is different, he said, and a more intense effort will be needed to win support for more billions.

"We've lost our communication link," Klein said.

(CIRM's summary of its impact graphically displayed below.)

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