When we produced our first Huntington’s disease (HD) video two years ago, I was really taken in by the close bonds between those living with the disease and the stem cell researchers looking for a treatment. The feelings of mutual respect, admiration, and friendship were very palpable. They spoke of each other as if they were family. I’ve come to learn that this bond has a lot to do with the fact that Huntington’s is a family disease.
A child of a parent with HD has a 50 percent risk of developing this neurodegenerative disorder which typically strikes in a person's thirties and leads to death about 10 to 15 years later. With no effective therapy for the disease, the individual families affected by HD have banded together as one big family for support and to advocate for a cure. And in the process they’ve welcomed the stem cell researchers as one of their own.
Several members of this Huntington’s community were in town two weeks ago with confetti, hugs, and bittersweet tears to celebrate the CIRM governing board’s approval of a $19 million UC Davis Huntington’s Disease Team II grant. The two minute video above captures the excitement of the moment (you can also view it here). Judy Roberson, a patient advocate whose husband died of HD, explained the importance of the award:
This disease team award for the first time in history - we have a chance to make a difference for a treatment for Huntington’s disease.The UC Davis team led by Vicki Wheelock and Jan Nolta, plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor to patients’ damaged and endangered nerves. The factor they have chosen, called BDNF, has been shown to be effective in laboratory studies in reducing nerve cell death and improving the function of nerves. During this project they will finalize laboratory tests and begin a phase 1 clinical trial in patients to test the safety of the approach. Although Dr. Wheelock’s team was the recipient of the grant, her focus at the meeting was on the entire HD community:
This gives hope to the families who have been suffering with this dreadful disease without any meaningful treatment to halt it or to slow it down.This grant was one of eight Disease Team II awards approved by the governing board which total $150 million in new research funding (Read our earlier blog here and the press release here). Claire Pomeroy, a CIRM board member and Dean of the UC Davis School of Medicine, reflected on the big picture of this new CIRM award:
I think it is the epitome of what Proposition 71 hoped which was scientists and patients coming together to find hope.TD