Stem Cell Agency Board Funds Cancer, Sickle Cell Disease and Vision Loss

This round of awards targets diseases like leukemia and other solid tumor cancers – such as breast and prostate – that have not responded to conventional treatment. Other diseases targeted include sickle cell disease and macular degeneration, one of the leading causes of blindness in the elderly.

“Funding this research to find a cure for sickle cell disease would mean that I am the last generation of mothers in my family to spend her adult life keeping her child alive,” says Adrienne Shapiro, a patient advocate whose daughter has the potentially deadly blood disorder. “It means I will no longer have to watch my bright, beautiful, brave girl as this horrible painful disease ravages her body and the pain medicines impair her mind.  It means I will no longer have to keep fighting for her while the weight of the psychological and social issues surrounding the treatment of Sickle Cell wears away at her spirit.”

“The goal of the Disease Team award is to help accelerate the development of new therapies,” says Alan Trounson, Ph.D., President of the agency. “I think this is the sharp end of the CIRM program – we need to get therapies into clinical trials. The scientists are working together as teams to demonstrate the safety and efficacy of their products that have evolved from discoveries in the laboratory. What’s impressive about this series of awards is that five of the six successful applications are for the continuation of work we had previously funded. It’s a reflection of the importance of continuity of funding, enabling scientists to keep their teams together and move their work forward as quickly as possible.”01

The Disease Team awards are designed to encourage multidisciplinary teams of researchers from academic institutions, medical centers and industry to work together and to develop new treatments for a broad range of therapies. The recipients were selected from 14 applications, all of which were reviewed by an independent group of internationally renowned scientists.

“Three of these successful studies arose as collaborations between California scientists and those in other countries 4 years ago,” observed Trounson. “Funding agencies in Canada and the United Kingdom supported work in those countries, and CIRM funded the teams in California. This international teamwork has been crucial to the projects’ success.”

The funding was approved by the stem cell agency’s governing Board, the Independent Citizen’s Oversight Committee (ICOC), at a two-day meeting in Los Angeles.

“I think this round of funding speaks volumes for the quality of the work we support,” says Jonathan Thomas, Ph.D., J.D., Chairman of the Board. “Many of these projects are ones we have previously funded so to have our outside expert reviewers look at them and recommend continued investment in this research shows we are on the right path.”

The Board also discussed the recommendations of the President’s Scientific Advisory Board (SAB) that were presented at the last ICOC meeting in October.  The SAB was composed of experts in the scientific, clinical, ethical, industry and regulatory aspects of stem cell biology to give the President advice and suggestions on strategic priorities for future research and how best to allocate existing funds.

Based on the report the Board decided to create an Accelerated Development Pathway, putting aside $200 million in a strategic reserve, to be used in helping select projects already funded by the agency speed up their development and move them through the clinical trial approval process as quickly and safely as possible. The money would be used for research that is already in or close to a clinical trial but needs additional funds to get through a Phase II trial to prove the therapy is both safe and effective.

In other recommendations by the SAB the Board voted to discontinue further funding for the Shared Laboratories award, to continue funding for the Early Translation program, and to continue funding, but at a reduced level, for the Basic Research program. The Board also asked staff to provide them with a detailed analysis of the effectiveness of the Training Grants, Bridges and Creativity programs, to determine if they wanted to continue funding them.

“We deeply appreciate the thought and consideration the Board put into making these decisions as they will have long-term implications for the way the agency works,” said Trounson. “Setting priorities is never easy but this gives us a much clearer sense of where we want to go and where we want to focus our resources and energies.”

The Board also took the opportunity to honor two former ICOC members, Leeza Gibbons, the patient representative for Alzheimer’s disease, and Jon Shestack, the patient representative for mental health.

“You never really say goodbye to people who are as passionate and committed to this work as Leeza and Jon are,” says Thomas. “We know they will both remain engaged in this work, but we shall miss their presence on the Board. They enriched our work with their compassion, their intellect and their unfailing focus on the agency’s goal of finding cures and treatments for deadly diseases.”