Friday, December 13, 2013

New Disease Team Awards drive therapies for cancers, vision loss, and sickle cell disease

Most people think of Board meetings as rather dry affairs. That’s certainly not the case with our governing Board, the Independent Citizens Oversight Committee, particularly when they are awarding more than $61 million in funding for diseases that have a devastating impact on people, and when some of those people are in the room to tell their story.

This latest meeting focused on funding for Disease Teams. These are multidisciplinary teams who get up to $20 million with a goal of moving the most promising therapies out of the lab and into clinical trials. Voting to approve tens of millions of dollars for research into diseases like acute myeloid leukemia and other cancers, for sickle cell disease and vision loss is always tremendously rewarding - in every meaning of the word - but it’s doubly so when you get a chance to meet some of the patients and patient advocates who stand to benefit from that research.

Adrienne Shapiro is the mother of a woman who has lived with sickle cell disease for decades. Watching her daughter suffer from the crippling pain of the disease and the huge impact it has on her quality of life has been tough on Adrienne. So when our Board voted to approve almost $14 million in funding to move UCLA researcher Donald Kohn’s work on sickle cell into clinical trials (hopefully as early as 2014). Adrienne was delighted.

As she told us at the meeting, and we quoted in our news release:
“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. 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.”
This round of funding could also help move the work of Stanford researcher Irv Weissman M.D. into clinical trials. Weissman is working on a stem cell approach to boost the immune system of cancer patients. His therapy uses stem cells to counteract a protein called CD47 which creates what Weissman calls a “don’t eat me” protective cloak around the cancer. Once that cloak is removed the patient’s own immune system recognizes the cancer and does in fact eat it. This approach will be first tested in acute myeloid leukemia but it has also shown great promise in treating solid tumor cancers such as breast, prostate, brain, colon, ovarian, bladder and liver.

What is particularly gratifying about this Disease Team round is that five of the six winners have had previous funding from us. It’s proof of the importance of continuity of funding, enabling researchers to keep their teams together as they advance their work from the basic science level, through initial tests and to the point where it’s ready to be tried in people.

Those awards include one to Mark Humayun M.D., Ph.D. at UCLA for his work in macular degeneration, the leading cause of vision loss and blindness in the elderly and Dennis Slamon, M.D., Ph.D., at UCLA who is developing a treatment for solid tumors.

As our Chairman Jonathan Thomas, Ph.D., J.D., said:
“I think this round of funding speaks volumes for the quality of the work we support. 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.”
Kevin McCormack

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