Monday, December 12, 2016

California's Stem Cell Year: 70 New Projects, 10 New Clinical Trials Plus a 'Stem Cell Pitching Machine'

The California stem cell agency weighed in today with an upbeat prelude to a review of its performance in 2016, declaring it a "very productive year" that is counting 10 new clinical trials.

And Gov. Jerry Brown, in a short note to the agency, said that he looked "forward to its continuing success."

Brown, who is famously careful with words, made his very brief comment in a letter renominating Jonathan Thomas as chairman of the $3 billion agency, formally known as the California Institute for Regenerative Medicine or CIRM.

Karen Ring, website and social media manager for CIRM, had a little more to say in a piece today on the agency's blog, The Stem Cellar. Her comments came on the eve of the CIRM board meeting in Oakland tomorrow, which includes a self-assessment of the state of the 12-year-old research effort. 

Ring noted that the agency is on target to meet its ambitious goal of helping to fund 50 new clinical trials through 2020. The board has backed eight trials so far this year and is virtually certain to add two more tomorrow at a cost of $14.9 million. In all, the agency is currently supporting 22 clinical trials, which are the last stage of research prior to bringing a therapy to market. Trials, however, can take years.

Declaring 2016 a "very productive year," Ring wrote that this year CIRM funded "70 promising stem cell projects ranging from education to discovery, translational and clinical projects."  She said that the eight board-approved trials include potential therapies for muscular dystrophy, kidney disease, primary immune diseases, and multiple types of cancer and blood disorders.

She also singled out a $30 million effort this year that Randy Mills, president of the agency, believes could be its most significant achievement. Mills sometimes calls it a stem cell "pitching machine."

Ring said,
"Collectively called The Stem Cell Center, the goal of this new infrastructure is to increase efficiency and shorten the time it takes to get human stem cell trials up and running."
No other state has mounted such an effort. The agency predicts that it could make California the world leader in the stem cell field, which is predicted to generate hundreds of billions of dollars globally by 2020 by some, perhaps optimistic accounts.

Regarding the two new clinical trials, already approved by the agency's reviewers, Ring said,
"The first trial is testing a stem cell treatment that could improve the outcome of kidney transplants. For normal kidney transplants, the recipient is required to take immunosuppressive drugs to prevent their body from rejecting the donated organ. This clinical trial aims to bypass the need for these drugs, which carry an increased risk of cancer, infection and heart disease, by injecting blood stem cells and other immune cells from the kidney donor into the patient receiving the kidney. You can read more about this proposed trial here.
"The second clinical trial is a stem cell derived therapy to improve vision in patients with a degenerative eye disease called retinitis pigmentosa. This disease destroys the light sensing cells at the back of the eye and has no cure. The trial hopes that by transplanting stem cell derived retinal progenitor cells into the back of the eye, these injected cells will secrete factors that will keep the cells in the eye healthy and possibly improve a patient’s vision. You can read more about this proposed trial here."
Names of the recipient researchers have been withheld by the agency until the board tomorrow ratifies the decisions by its reviewers.

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