Directors of the California stem cell
agency today deferred action on a $20 million proposal that was
rejected by its grant reviewers and sent it back for more consideration.
The move involved an application by
Stanley Nelson and M. Carrie Miceli of UCLA dealing with Duchenne
muscular dystrophy. They are parents of a child with the affliction,
which is usually fatal by age 25. The CIRM review summary said it is
“a devastating and incurable muscle-wasting disease caused by
genetic mutations in the gene that codes for dystrophin, a protein
that plays a key role in muscle cell health.”
Stanley Nelson and M. Carrie Miceli of UCLA dealing with Duchenne
muscular dystrophy. They are parents of a child with the affliction,
which is usually fatal by age 25. The CIRM review summary said it is
“a devastating and incurable muscle-wasting disease caused by
genetic mutations in the gene that codes for dystrophin, a protein
that plays a key role in muscle cell health.”
Nelson had filed an appeal seeking to
overturn reviewers' action. Five mothers and two fathers with
children suffering from the disease made emotional appeals to
CIRM directors on behalf of the application. Following their presentation, Art
Torres, co vice chairman of the CIRM board, responded equally
emotionally that the board is dedicated to finding therapies for such
afflictions as Duchenne muscular dystrophy
overturn reviewers' action. Five mothers and two fathers with
children suffering from the disease made emotional appeals to
CIRM directors on behalf of the application. Following their presentation, Art
Torres, co vice chairman of the CIRM board, responded equally
emotionally that the board is dedicated to finding therapies for such
afflictions as Duchenne muscular dystrophy
The board approved more review for the
application after it was disclosed that a company issued a press
release two days ago that showed that a drug involved in the
proposal was more effective than reviewers believed. Philip Pizzo, a
member of the board and dean of the Stanford medical school,
expressed caution about the press release, given its timing and
source. His comments came prior to the appearance of the parents.
application after it was disclosed that a company issued a press
release two days ago that showed that a drug involved in the
proposal was more effective than reviewers believed. Philip Pizzo, a
member of the board and dean of the Stanford medical school,
expressed caution about the press release, given its timing and
source. His comments came prior to the appearance of the parents.
The application is scheduled to be
brought back to the full board in September for further action.
brought back to the full board in September for further action.
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