|Huntington's disease advocates speaking to the Governing Board|
But that happiness was bittersweet. The reason we need to fund research is because there are currently no cures for those diseases. And for the people living with the diseases, the results of the research we funded might come too late.
For me, the hardest part of the day by far was hearing from those families whose children—almost exclusively boys—have muscular dystrophy. These are parents who saw their happy, healthy, active boys start to weaken at around age five. These parents will inevitably lose their children to the disease unless there is a cure.
My own happy, healthy, active boys are five and seven, exactly the ages many of those parents described. As those parents were before the diagnosis, I’m looking forward to seeing my kids grow up, have fun, find careers and start families. Instead, those families bought their kids wheelchairs and have watched them grow weaker and more dependent.
Later in the meeting a woman in a wheelchair spoke about raising her three children on her own and being diagnosed with Lou Gehrig’s disease (properly known as ALS). That disease is also inevitably fatal.
One woman who is going blind with retinitis pigmentosa spoke about wanting to see her husband’s face.
Things most of us take for granted—seeing our loved ones, hugging our kids—are the source of dreams for those who spoke at yesterday’s meeting.
Perhaps the most visible group of patient advocates at the meeting were the Huntington’s disease families who showed up en masse to support an application from Vicki Wheelock of the University of California, Davis. That community is so involved in driving new research that many have become old friends. They threw what might be the first confetti at a public state meeting when our governing board voted to approve funding for that award.
Their excitement wasn’t for a cure—even if Wheelock’s work is successful it will be many years before a treatment is widely available. It was for hope.
Speaking before the board, Katie Jackson, whose husband has Huntington’s disease said:
"When Mike was diagnosed, I couldn't believe there was nothing they could do for him. We need hope more than we can explain to you."CIRM can’t know in advance which research projects are going to be successful. Some will inevitably not result in the hoped-for clinical trials. That’s the process of science—trying different approaches until you find one that works. All we can do is try to pick the best science and then do what we can help those teams succeed in turning the hope into a reality.
Here's our press release about the awards.