Clinical trials networks are nothing new. But a clinical trials network focused around a broad therapeutic platform, rather than a particular disease, disease area or specific type of treatment, would be.

On 25 July, the governing board of the California Institute for Regenerative Medicine (CIRM) will decide whether to fund a $70 million network of so-called 'Alpha' stem cell clinics that, as currently envisioned, would bring together the necessary scientific, technical and medical expertise to host and advise clinical trials for stem cell–based therapies for a range of illnesses. “It would make sense to start building a brain trust of individuals who have that expertise,” says CIRM's Natalie DeWitt, special projects officer at the state stem cell agency, which is headquartered in San Francisco.

Few stem cell therapies, with the exception of hematopoietic stem cells derived from bone marrow or cord blood, are currently used in routine medical practice. The proponents of the Alpha clinics hope to lay the groundwork for more. “We're kind of doing this in anticipation of this field really growing,” DeWitt says.

The Alpha clinic investment represents about 10% of the total money left in CIRM's coffers. Under the proposal, the funding would be spread over five years and would go toward supporting a handful of clinical sites at academic medical centers and a central coordinating and information management center, all based in California. An as-yet-unspecified proportion of the money would additionally be spent on developing education and outreach resources for the general public. The participating sites, which would be decided by a peer-reviewed application process, would receive training in the unique scientific and regulatory challenges of working with cell-based therapies—storage, shipping, administration, manufacturing, cell tracking and more.

It's precisely issues like these that often make it difficult for stem cell biologists with promising translational data to negotiate an investigational new drug application (IND) with the US Food and Drug Administration (FDA), says CIRM medical officer Maria Millan. “There's not a lot of expertise regarding navigating the proper IND-enabling activities—being able to have meaningful discussions with the FDA and crafting the right pathway to bring some of these product candidates to the clinic—and as a consequence there's maybe promising projects that stay in the lab and get held up there.”

What happens in California...

Mahendra Rao, director of the Center for Regenerative Medicine at the US National Institutes of Health (NIH) in Bethesda, Maryland, applauds the Alpha clinic model. But he doesn't see the same approach being tried nationally anytime soon, noting that the NIH already supports many networks designed to translate cell therapies into the clinic. For example, the agency's National Heart, Lung and Blood Institute already spends around $12 million per year on the Production Assistance for Cellular Therapies (PACT) program, which goes toward supporting a coordinating center and five cell-processing facilities that provide clinical-grade cell products for investigators across the country. Additional training in bringing therapies forward is available through initiatives such as the Clinical and Translational Science Awards (CTSA) program.

Rao regularly asks researchers hoping to advance promising stem cell therapies whether they require additional clinical infrastructure. “So far, what they've told us is they'll let us know if they need anything more than CTSA and the PACT programs that we have already established,” he says.

Clinical catalyst: CIRM expects requests to start human testing with investigational cell therapies to rise dramatically. Credit: Sci. Transl. Med. 4, 149fs31 (2012).