Thursday, June 29, 2017

Alpha Thalassemia to Strokes to Zika: A $64 Million Boost by California for Stem Cell Research

BURLINGAME, Ca. -- The California stem cell agency today awarded more than $64 million for research tackling cancer, stroke, leukemia, heart failure, brain injury, Zika and much, much more

The agency's directors allotted $44 million of the amount for two clinical trials and potentially two more trials, which are the last stages before approval of treatments for use by the general public. Prior to today's action, the agency -- known officially as the California Institute for Regenerative Medicine or CIRM -- was helping to finance 27 active clinical trials.

The agency has yet to develop therapies that would be available for widespread use that voters were led to expect in 2004 when they approved creation of the $3 billion research effort. CIRM expects to run out of cash for new awards in three years. 

The most advanced clinical research approved today involves a phase 2b trial for which CIRM ponied up $20 million. The effort is also backed with co-funding of $22 million by the recipient, SanBio, Inc., a Mountain View, Ca., subsidiary of a Japanese firm, SanBio Co., Ltd

The review summary of the application (CLIN2-10344) said the trial had already demonstrated safety and "a trend toward efficacy" involving ischemic strokes. The research is aimed at improving motor function of victims of strokes, which is the leading cause of adult disability.

Directors also approved $5.3 million for work aimed at starting a clinical trial for a treatment that would enhance the brain's ability to create new blood vessels to replace those damaged during a stroke. (The review summary for CLIN-09433 can be found here.)

The funds went to Gary Steinberg of Stanford University.

Maria Millan, interim president and CEO of the agency, said in a news release,
"Today the CIRM Board approved two very different methods, using different kinds of stem cells, to address this need. By funding 'multiple shots on goal' we believe that we have a better chance of finding a way to repair the damage caused by stroke and give people a better quality of life.”
The second phase 2 clinical trial backed by CIRM involves transplantation of maternal bone marrow stem cells into fetuses discovered to have alpha thalassemia major, an affliction that is almost always fatal in utero.

The $12.1 million award (CLIN2-09183) went to Tippi MacKenzie of UC San Francisco. (The review summary can be found here.)

The fourth application (CLIN1-09776) involves acute myeloid leukemia and preparation for a clinical trial application. CIRM awarded $6.9 million to Cellerant Therapeutics, Inc., of San Carlos. The agency reported that $1.7 million in co-funding would be provided by Cellerant. (The review summary can be found here.)

Also approved was $20 million for 13 awards in what the agency calls its Quest program, which is aimed at supporting promising, less developed research that is likely to move on to the next stage of development within two years. Twenty-six applications were rejected.

The review summaries and scores on the awards can be found in this document. The list of recipients can be found in the CIRM press release.

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