On September 13th, the UCSF Memory and Aging Center hosted the 11th Annual Huntington Disease Research Symposium. The symposium featured scientists discussing their work in basic science and clinical HD research. It was a great opportunity for HD patients and families to meet local researchers, become more informed about progress in HD research, and learn about ways get involved.
Stem cells figured heavily in the basic science portion of the symposium. Steve Finkbeiner from the Gladstone Institute of UCSF kicked off the conference by discussing among other things, the use of skin or blood samples from HD patients to make stem cells, which then can then be differentiated into brain cells. This process preserves the patient’s genetics and may help more directly test the efficacy of prospective therapies. These stem cells are called induced pluripotent stem cells and induced neuronal stem cells, respectively. Scientist Vikki Wheelock discussed a different kind of stem cell, mesenchymal stem cells, and the potential ability to engineer non-HD mesenchymal stem cells to deliver drug treatments. These stem cells come from adult bone marrow.
Many of the researchers rely on bioinformatics and the use of new research methods like optogenetics to make advancements in understanding HD. For example, Alexandra Nelson discussed her efforts to understand HD’s effects on the brain using optogenetics, a technique that a lab at Stanford pioneered. Optogenetics takes advantage of light sensitive proteins in algae. The genes for these proteins can be packaged inside of a virus and injected into an animal’s brain to make nearby neurons light-sensitive. Through this technique, Alexandra Nelson hopes to understand how the living cells that survive HD can be harnessed to deal with the symptoms.
Three researchers from the Memory and Aging Center at UCSF, Michael Geschwind, Natasha Boissier, and Erica Pitsch, discussed the clinical side of HD research. Overall, despite the acknowledgement of the scientists that many of these treatments and advancements are in preliminary stages, and that clinical research may be halted if it is determined to be ineffective or unsafe, hope was in the air at the symposium.