Wednesday, August 19, 2015

California Spending More than $19 Million on Search for Stem Cell Arthritis Therapy

Stem cell agency summary of Peter Schultz proposal
First in class therapy
Avoids risks, costs
Uses drug-like kartogenin
The California stem cell agency tomorrow is set to boost its spending on arthritis to $19.5 million with a new grant to a San Diego researcher who is preparing a clinical trial for his possible therapy.

The agency’s governing board is all but certain to approve a $1.7 million award to Peter Schultz of the Scripps Research Institute in La Jolla on top of the $6 million he has already received.

Meeting behind closed doors, the agency’s blue-ribbon scientific reviewers earlier this summer approved the application on a 12-0 vote. The agency board almost never overturns a positive decision by its reviewers.

The review summary said the proposed therapy would be “first in class regenerative medicine” for osteoarthritis as well as cartilage injury. Currently no disease-modifying drugs are approved for clinical treatment of arthritis, which afflicts 27 million people in the United States. The award is to assist in preparation for a federally approved clinical trial. 

Peter Schultz -- Scripps photo
Schultz, who has close ties to Merck and who is also director of the California Institute for Biomedical Research, laid out some details of his approach in an earlier progress report to the stem cell agency.

He said,
“Because limited joints are affected in most (osteoarthritis) patients, intra-articular drug injection is an attractive treatment approach that allows high local drug concentration with limited systemic exposure. Targeting resident stem cells pharmacologically also avoids the risks and costs associated with cell-based approaches.”
 Schultz continued,
“Cartilage contains resident mesenchymal stem cells (MSCs) that can be differentiated in vitro to form chondrocytes. This observation suggests that intra-articular injection of a small molecule that promotes chondrogenesis in vivo will preserve and regenerate cartilage in OA-affected joints. Using an image-based screen, we identified a drug-like small molecule, kartogenin (KGN), that promotes efficient and selective chondrocyte differentiation from MSCs in vitro. Intra-articular injection of KGN also shows beneficial effects in surgery-induced acute and enzyme-induced chronic cartilage injury models in rodents, as well as positive effects in incapacitance pain models.”
The agency has already pumped in around $17 million for arthritis research, including funds given earlier to Schultz.

As usual, the award tomorrow involves an institution that has a representative on the agency board. About 88 percent of the money that the agency has handed out since 2005 -- $1.9 billion -- has gone to institutions that have ties to agency directors, according to calculations by the California Stem Cell Report.

The stem cell agency does not identify the winning applicants for awards until after the board acts. However, based on information on the agency's Web site, the California Stem Cell Report was able to verify that Schultz was the applicant up for approval tomorrow.

Tomorrow’s teleconference meeting is scheduled for only one hour beginning at 9 a.m.. The main session will be in San Francisco, which has another location there as well. Other locations where the public can participate include Irvine, San Diego, Napa, Redwood City, two in Sacramento, Los Gatos, San Francisco, Elk Grove, Beverly Hills, Fresno, Los Angeles, Cambridge, Ma., and Ballard, Ca., in Santa Barbara County. Specific addresses can be found on the agenda.

The public can also make comments  on any issue during the board session.

(Editor's note: The figures on CIRM's total spending for arthritis have been increased from an earlier version of this story. The previous figures were based on erroneous numbers on the agency's "arthritis fact sheet," which did not include a $2.3 million award to Schultz in March.)
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