Friday, December 22, 2017

California Stem Cell Agency's 'Holiday' Awards: $25 Million for Research Ranging from Liver Failure to Dementia

OAKLAND, Ca. -- Directors of the California stem cell agency last week approved nearly $25 million for research but dashed the hopes of two scientists who were pushing extra hard for funding from the 13-year-old state enterprise.

Thirteen researchers ultimately received awards for tackling such afflictions as diabetes, dementia and liver failure. Thirty-two scientists who applied in the round were originally rejected by reviewers for the California Insitute for Regenerative Medicine, (CIRM), as the agency is formally known.

Reviewers make the de facto decisions on awards by the agency, although its governing board can override their actions. Four researchers whose proposals were nixed during an earlier, closed-door reviewer session sought to reverse those decisions.

Only two out of the four won over the board. One was Mark Tuszynski, director of the Translational Neuroscience Institute at UC San Diego, who sought $2.1 million to support his research to use neural stem cells to grow new connections through injured spinal cord. In a letter to the board prior to the meeting, he said ,
"As the present round of...funding winds down, this may be our last opportunity to develop this work to benefit the citizens of the state of California."
Tuszynski's application received a score of 80 out of 100, five points below the cutoff line of 85.

CIRM directors discussed the application briefly during which San Diego researcher addressed the board.

In addition to Tuszynski's application, the board reversed reviewers on an application for a cellular treatment for epilepsy. The $1.6 million proposal by Neurona Therapeutics of South San Francisco scored 80. Arnold Kriegstein, co-founder of the firm and head of the UC San Francisco stem cell program, spoke on behalf of the Neurona. 

The other appellants were Alice Tarantal of  UC Davis and Gregorio Chazenbalk of UCLA.

Tarantal filed a $1.1 million application (DISC2-10599) that she said involved "total-body positron emission tomography (PET) imaging technology, which currently is only available in California."
Her application was scored at 84 by reviewers, just one point below the cutoff line. Board members in the past have noted that such narrow differences are statistically meaningless.

Chazenbalk's $2.2 million application (DISC2-10473) involved a "new population of pluripotent stem cells" known as Muse cells, which he said can be used to treat acute myocardial infarction with a "high potential rate of success." His score was not disclosed but appears to be below 65.

Both Tarantal and Chazenbalk addressed the board but none of the directors discussed their applications. 

No discussion of the winning applications was conducted by the board members. A list of their names, institutions and projects is below.  

Here is the CIRM press release on the awards. It includes the application numbers for all proposals, which can be used to find the summaries of reviewer comments and scores. The specific review summaries can be found by scrolling more deeply into that document.
Preclinical development of an immune evasive islet cell replacement therapy for
type 1 diabetes


Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction
of Hemophilia A

Saswati Chatterjee

City of Hope

Towards hepatocyte cell replacement therapy: developing a renewable source of
human hepatocytes from pluripotent stem cells

Irv Weismann

Engineering Lifelong Cellular Immunity to HIV

Scott Kitchen

U.C. Los Angeles

iPS Glial Therapy for White Matter Stroke and Vascular Dementia

Stanley Thomas Carmichael

U.C. Los Angeles

Stimulating endogenous muscle stem cells to counter muscle atrophy

Helen Blau



Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy
for chronic cervical spinal cord injury.

Aileen Anderson

U.C. Irvine

Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1
Diabetes treatment

Shuvo Roy

U.C. San Francisco

Identification and Generation of Long Term Repopulating Human Muscle Stem
Cells from Human Pluripotent Stem Cells

April Pyle

U.C. Los Angeles

Targeting Cancer Stem Cells in Hematologic Malignancies

Tannishtha Reya

U.C. San Diego

Development of immune invisible beta cells as a cell therapy for type 1 diabetes
through genetic modification of hESCs

Yang Xu

U.C. San Diego

Development of a cellular therapeutic for treatment of epilepsy

Neurona Therapeutics

Neural Stem Cell Relays for Severe Spinal Cord Injury

Mark Tuszynski

U.C. San Diego

Chart by CIRM

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