California Stem Cell Agency’s Road to Success for Next Five Years Will Have to Run Through the Clinic

On January 17, the governing board of the California Institute for Regenerative Medicine (CIRM) began discussing a draft strategic plan covering 2012–2016. Its call for the agency to push more of the science it funds into clinical trials is one of four objectives articulated in the plan. The other three are: accelerating understanding of stem cell science, promoting economic development for California, and positioning the state as a world leader in stem-cell science.
“We intend to fund work that will result in clinical proofs of concept while maintaining the full pipeline of discovery,” the agency declared in its strategic plan. That full pipeline will not neglect basic research, Don Gibbons, a spokesman for the stem cell agency, told GEN. “We plan to continue funding basic research because that work yields the breakthroughs that turn into completely new therapy options, and it is also necessary to develop ways around roadblocks that are discovered in early clinical work.”
One priority for members of CIRM’s governing board, the Independent Citizens Oversight Committee (ICOC), should be positioning the agency as a partner in overcoming the risks of stem cell R&D that have deterred most biopharma investors and businesses from the field. The agency was reminded of those risks last fall when Geron ended its development programs. The firm, which focused on human embryonic stem cells (hESCs), had the most advanced stem cell program as well as the most money in the stem cell field.
Going forward CIRM will need to reconcile the high cost of clinical development with the need to plant numerous proverbial seeds in the discovery patch to find the few that blossom into effective stem cell treatments. Discussion on the strategic plan is expected to take several months before it is approved by the ICOC.

R&D Initiatives

On the basic research side, CIRM plans to create one or two Stem Cell Genomics Centers of Excellence to carry out genomic studies. CIRM envisions spending up to $40 million over five years. At least one center will include infrastructure and expertise to analyze the torrent of data that the centers are expected to generate. “We expect these will be stem-cell-specific add-ons to existing centers,” Gibbons explained. “We very much want to leverage the expertise and equipment they already have.”
On the clinical development end, the agency said it will add product development, regulatory, and commercial viability experts to its peer-review groups, which decide on how disease teams are set up and future translational awards.
According to the draft plan CIRM also wants to attract more co-funding and follow-on financing as well as forge more collaborations. To fund clinical research, the agency said, it will work to draw investment from industry and venture capital through a recently approved $30 million program.

iPSC Bank

CIRM also plans to develop a bank for induced pluripotent stem cells (iPSCs) carrying disease mutations. It would make those cells available to researchers in California and worldwide. The first part of this initiative is already in place: a CIRM-NIH collaboration was set up last October to generate cell lines from people with Huntington disease, Parkinson disease, and amyotrophic lateral sclerosis.
In May CIRM expects to issue formal requests for applications from institutions capable of collecting samples from people with specific prevalent diseases. The aim is to derive iPSC lines from 1,200 people at a $4 million cost. The lines would be free to research groups that acquired the samples. CIRM will also be looking for organizations that can derive additional iPSC lines at a cost of $16 million.
Furthermore CIRM will call for applicants that can create a stem cell bank with the iPSC and hESC lines already created by California researchers at a cost of $10 million over three years. The bank would make disease-specific stem cell lines available to researchers worldwide.
The bank reflects how the science of regenerative medicine has changed from a decade ago, when CIRM was created in no small part to fill the federal funding gap left by the restrictions imposed on hESC research by President George W. Bush in 2001. President Barack Obama removed the restrictions, and since then the U.S. Department of Health and Human Services (HHS) has grown an hESC registry with 146 approved lines. But long-term federal funding of hESC research is still uncertain, as the D.C. Court of Appeals is set to review this March written arguments in Sherley et al. v. Sebelius et al., the court case concerning federal funding of research on hESCs.
CIRM says its iPSC bank isn’t considered a Plan B in case the appeals panel rules to stop federal funds being used toward hESC research. “This is all about the ability to make disease-specific cell lines with iPSCs,” Gibbons asserted. “This does not change our commitment to ESCs.”

Stem Cell Clinics

CIRM’s draft plan commits the agency to exploring and facilitating creation of new “Alpha Stem Cell Clinics” that would deliver stem cell based therapies to patients. It would then aim to replicate the model nationally and internationally with help from its “collaborative funding” partners, which committed about $60 million as of December 2011.
How many clinics will be created and whether they will be within existing medical centers or new facilities has not been determined. Writing in Stem Cells Translational Medicine, CIRM president Alan Trounson, Ph.D., elaborated that the clinics would be “modeled on a framework that has been used successfully by cancer clinics, bone marrow transplant units, and in vitro fertilization clinics.
“As a first goal, the Alpha Clinic model can immediately target therapies toward those patients who can be helped, based on proven clinical evidence of efficacy and safety, such as for limbal cell treatments for people suffering from corneal burn injuries,” according to Dr. Trounson.
While California has sophisticated clinical trial venues, CIRM has ruled them out for Alpha clinics: “Existing trial centers do not have the expertise and equipment needed for cell-based therapy.”

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