A $40 million grant awarded last week — the largest ever by California’s stem cell funding agency — is aimed at putting the state in a world leadership role in stem cell genomics, an emerging field with potential for creating genetically specific medical treatments for individual patients.
In a controversial vote after a hotly-contested competition, California Institute for Regenerative Medicine directors awarded the funding to a consortium of seven institutions led by Stanford University and the Salk Institute for Biological Studies. The new entity, to be called Center of Excellence in Stem Cell Genomics, also includes four San Diego institutions — UC-San Diego, the Scripps Research Institute, the J. Craig Venter Institute and Illumina. UC-Santa Cruz will oversee data collection and coordination.
“With advances in genomics, there is great synergy between genomic analysis and stem cell therapy that can be used to develop individually personalized medical treatments,” said Jon Thomas, chair of CIRM’s Independent Citizens Oversight Committee.
“As one of the leaders in stem cell funding in the world, we wanted to make sure we have a world-class genomics effort to take advantage of that. We think we picked a world-class team,” Thomas said.
It didn’t happen easily. As reported in the Sacramento Bee and San Francisco Chronicle, the vote represented less than a third of the 29-member governing board and came amid accusations of unfair and preferential treatment. In most grant-making decisions, 16 members of the board’s Application Review Subcommittee are eligible to vote. In this instance only seven voted (6-1). Many members have ties to institutions vying for funding and were not eligible to vote.
“It was a complicated situation with a number of entities and individuals that applied in different ways with different affiliations,” Thomas said. “It was a complicated final vote that allowed for projects in institutions that weren’t included in the main grant to participate.”
The CIRM board added about $7 million to the winning group’s $33 million proposal to fund collaborators outside the consortium.
In total, the award includes $19 million earmarked for independent and collaborative projects including investigating disease mechanisms and the development of new technologies.
Changes Coming ‘at Rapid-Fire Pace’
Genomics — the study of genetic processes in organisms — can help researchers better understand the way stem cells mutate into other kinds of cells, which, in turn, may lead to revolutionary new therapies for diseases including cancer, diabetes, heart disease and some kinds of mental illnesses.
“Genomic advances are coming at rapid-fire pace, and it’s important we have the capabilities to complement the changes,” Thomas said.
“I think in the near term you’re going to start seeing stem cell clinical trials that are suggestive of the kind of potential there is in this research,” Thomas said.
One of CIRM’s grantees is about to go into human clinical trials this year for a treatment of Type 1 diabetes, Thomas said. “Cells are inserted under the skin that will develop into two beta cells that will develop insulin. I think projects like this will begin to show early signs of proof of concept, which will in turn lead to real advances in actual treatment options. The research in genomics will clearly inform those advances,” Thomas said.
New Technologies Will Beget New Policy, Financing
With the increase in new treatment options, government entities, insurers and providers are going to have to keep pace with changes in policy and financing, Thomas said.
“There are policy issues involved at many stages in this process,” Thomas said. “There are the policies involved in getting these efforts through the FDA and then there are going to be a whole new set of significant policy issues regarding reimbursement and coverage for some of these treatments.”
“It’s going to be a societal shift as well as a shift in medical science,” Thomas said, adding, “It’s going to take a mindset with insurers that things developed from these technologies should be covered.”
He said, “You’re going to see an explosion of technological development that will require policies to adapt relatively quickly. This technology’s not going away.”
In addition to the $40 million genomics grant, CIRM board members approved $27 million in grants for additional projects at 11 California institutions, including UC-Berkeley, UC-San Francisco, Stanford University and Gladstone Institutes.
Stem Cell Agency’s Funding Days Are Numbered
Established in 2004 when California voters approved a ballot measure to put the state’s weight behind stem cell research, CIRM is approaching the end of its tenure, as well as the end of its funding.
CIRM is authorized to generate and distribute $3 billion of funding from state bonds by the end of 2017.
Thomas and other CIRM officials are beating the bushes to find new funding streams to keep the effort afloat past 2017.
“Sustaining research and retaining the program is my highest priority,” Thomas said. Thomas said he’s exploring several promising possibilities but isn’t prepared yet to discuss particulars. Pharmaceutical and biotechnology companies are likely candidates, according to some industry experts.
“Wherever it comes from, it’s going to be a vital necessity to find alternative funds to keep the ball rolling,” Thomas said. “There will be a lot of scientists out there who will have projects at mid-stream and it just wouldn’t make any sense to cut them off.”
Thomas is confident CIRM officials will find a way to keep funds flowing.
“Especially in light of this new genomics research. There are definitely other major genomics programs elsewhere in the world but nobody is tying it to stem cell research in the same way and I think that has great, global significance,” Thomas said.