The California Institute for Regenerative Medicine today awarded $230 million in disease team awards, intended to move stem cell therapies into the clinic within four years.
Fourteen teams, including twelve academic institutions and two companies as principal or co-principal investigators, received the awards. Canada’s Cancer Stem Cell Consortium will pay an additional $35 million for two of the grants that aim to target cancer stem cells, and the United Kingdom’s Medical Research Council will award $8 million for two grants that aim to treat macular degeneration and target leukemia stem cells.
CIRM regards the disease team awards as a crown jewel of its portfolio. The grants include specific milestones that will try to greatly condense the time it takes to move experimental therapies from the lab to an investigational new drug filing with the U.S. Food and Drug Administration. One estimate pegs the cost of that development timeline at $800 million over 7 years, with biologics costing even more at $1.2 billion over 8 years. The CIRM grants award between $5 and $20 million, or up to $40 million for some of the grants that received additional funding from the international partners, for therapies that, in some cases, are unlike any ever before approved by the FDA. But CIRM says that by coordinating basic and clinical researchers from the start, and requiring the teams to abide by development milestones, it can chop years and dollars off the typical drug development timeline.
The grants target a range of diseases including brain cancer, a rare skin disorder, stroke, heart attack, amyotrophic lateral sclerosis, macular degeneration, and sickle cell anemia. The controversial cancer stem cell hypothesis won big, with three approved grants targeting cancer stem cells. Two grants support work in HIV, and one in diabetes. Only a handful will employ human embryonic stem cells, despite the fact that most of the fanfare surrounding the passage of Proposition 71, the ballot measure that created CIRM, concerned the fact that CIRM would fill the gap left by a lack of federal funding for work on these cells. But Bob Klein, architect of Proposition 71 and chair of CIRM’s governing board, said, “Our commitment to the voters was that we would pursue the very best cell type for each disease based on the scientific and clinical evidence.”
The other approaches involve some that have already yielded treatments for other diseases, such as modifying blood-forming stem cells taken from a patient’s own bone marrow. One grant will use induced pluripotent cells, which can be tailored to match individual patients, but are still relatively new to science.
Three of the grants had not been recommended for funding after scientific review, but were moved up into the funding category by CIRM’s governing committee at its meeting last night. Read the review reports on all the grants here.
CIRM will make additional disease team awards in an 18-to-24 month cycle.