Thursday, November 20, 2014

Birthday Party for California's 10-year-old Stem Cell Research Program

Fred Lesikar, a heart attack victim aided by
a CIRM-backed cell treatment
CIRM photo
The California stem cell agency today celebrated its 10th birthday with a media event in Los Angeles where its supporters declared that the effort marked “one of the seminal events in the history of medical research.”

Attending the party in Los Angeles were a number of scientists and university officials whose institutions and research have received hundreds of millions of dollars from the program, which is funded by money that the state borrows(bonds).

California voters created the program in 2004 when they approved a ballot initiative that established the California Institute for Regenerative Medicine(CIRM), as the San Francisco-based agency is formally known.

CIRM has awarded $1.8 billion so far out of its $3 billion allotment. Because the money is borrowed, the effort will cost taxpayers about $6 billion, including interest. The agency is controlled by a 29-member board of directors. Eighty-eight percent of the $1.8 billion in awards has gone to institutions linked to current or past directors, according to calculations by the California Stem Cell Report. (For a recent assessment of the agency, see here.)

CIRM Chairman Jonathan Thomas told the gathering at USC, which has received $88.5 million in funding, that the state research effort amounted to a “seminal event.” California began its program when stem cell research was at a low ebb, Thomas said. He said it helped to keep stem cell research alive and  “galvanized” efforts globally.

He and others celebrated the 10 early stage clinical trials that CIRM now expects to be part of this year. None of the speakers mentioned studies that show that only one out 10 potential conventional treatments that enter clinical trials emerges as a therapy that is widely available to the public. The odds for stem cell therapies are unknown because they are so new.

Thomas and others pointed to news this week from UCLA about a genetic treatment that they said saved the lives of 18 children who had an immune deficiency affliction called the “bubble boy syndrome.” The cure involved insertion of a missing gene into the child’s blood stem cells. The work by researcher Donald Kohn received national attention. Oddly, however, the Los Angeles Times has not written about the research, according to a Google search this afternoon. CIRM's Todd Dubnicoff wrote a very nice piece about the research earlier this week.

The stem cell agency did not directly fund the “bubble boy” experiment, although CIRM awards, ranging from training programs to lab construction, did help to make the effort a reality.  The research is expected to be used in devising a sickle cell treatment in a $13 million CIRM program involving Kohn. 

The man sometimes referred to as the father of the California stem cell effort, Robert Klein, was also on hand today.  Klein led the $35 million ballot campaign in 2004 to win approval for creation of CIRM, which functions outside of the normal controls on nearly all state departments. Klein, a Palo Alto real estate investment banker, was also the first chairman of the agency. 

Klein said that a “revolution is underway” in medicine and that CIRM is leading the way. The dream of patients has become a reality, Klein said.

Only one patient appeared along with researchers, Fred Lesikar, a heart attack patient who was treated with his own heart cells at Cedars-Sinai in Los Angeles a few years ago.

Lesikar told the audience that “little by little over the next six months to a year, they (the cells) went in and started replacing the dead tissue.” He said he is now feeling great.

The researcher involved in that treatment, Eduardo Marban, has moved forward with a trial that CIRM is helping to fund through Capricor, Inc., a publicly traded  Beverly Hills firm.

On the podium today were researchers or officials from USC, UCLA, Cedars-Sinai and the City of Hope. All have representatives on the agency’s board. A researcher from ViaCyte, Inc., of San Diego, another CIRM award recipient, also spoke.

Today’s media event, which was available by a telephonic link, represents a major push to generate favorable coverage of the California stem cell program. It was held early enough in the day to meet deadlines for the early TV news shows along with allowing enough time for preparation of fulsome stories. However, Los Angeles is a tough news market. We will be watching later today and tomorrow for coverage of the event and bring you additional information as warranted.

(An earlier version of this item incorrectly said that Anne Holden wrote the CIRM blog item on Kohn's work.)
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