The 8th ACARM was held on December 12th in Sacramento from 9:30-4:00. Seven leading Researchers (all PhD’s) spoke to the audience of Ataxians and their families. Three of the speakers addressed ‘on-going’ studies at their Universities using stem cells. Much of the research is being directed toward Huntington’s Disease because funding is actively available for that research. Dr. Perlman and other researchers commented that progress with regenerative cures or delaying the progression of HD will also be a breakthrough for Ataxia as well.
Much of the funding for stem cell research in California is coming through California Institute for Regenerative Medicine. CIRM. Check out their website and all the institutions they have funded.
We heard about research being done with adult stem cells, embryonic stem cells, umbilical blood cord stem cells, mesenchymal stem cells, pluripotent stem cells.
The three speakers represented UC Davis, (Dr Jan A. Nolta), UC Berkeley,(Dr. David Schaffer)and University of CA, Irvine,(Dr.Leslie Thompson. These universities all have Stem Cell Research programs.
Dr. Jan Nolta has published over 100 manuscripts in the stem cell field and is editor of the book, Genetic Engineering of Mesenchymal Stem Cells. Dr. Nolta has more than 20 years experience with human stem cells.
Dr. David Shaffer is Co-Director of the UC Berkeley Stem Cell Center. Dr. Shaffer applies engineering principles to enhance stem cells and gene therapy approaches for regeneration.
Leslie Thompson helped identify the gene that causes Huntington’s Disease. She stated that stem cells have offered us a new way to study the disease by allowing us to examine human neurons carrying the mutation.
Leslie left our meeting to fly to Washington to meet with National Institute of Health and some Pharma Companies who would like to partner with stem cell research programs. How exciting is that!!! She has reported back that the meetings went exceptionally well. NIH and Pharma are very interested in stem cell research.
Also from UC Davis, Dr. Heike Wulff shared how her research is focused on potassium channel activators as potential therapeutics for the treatment of Ataxia and other Neurological Diseases characterized by Hyperexcitability.
The ever popular and respected, Dr. Susan Perlman gave an informative talk about Research and treatments for non-genetic Ataxia. 40% of the Ataxia population are genetic, 60% are non-genetic.
Dr. Lisa Ellerby is actively doing research at the distinguished Buck Institute for Age Research in Novato, CA. Her team is researching the mechanism of neuro-degeneration and therapeutic treatments for Huntington’s Disease and other polyglutamine expansion diseases.
Dr. Theresa Zeseiwicz, was our special out of state speaker from the University of Southern Florida. USF spearheaded the clinical trials using the FDA approved drug, Chantix. Her report was, Chantix up until now. Chantix has shown to help gait and balance in some individuals with Ataxia. It seems to be the most helpful to those who have Spinocerebellar Ataxia. Paul Konanz shared that his daughter, who has Friedreichs Ataxia, has clearer speech and better coordination when she takes Chantix. Theresa did share about negative side effects.
A big thank you to Mike Fernandez who lined up these very accomplished Researchers.
Also, thank you for Dr. Gail Kang, from UCSF, who MC’d the event. Dr. Kang will be speaking at our next Support Group meeting. She is a Neurologist and Professor at UCSF Memory and Aging Center. She trained under Dr. Perlman and has lot of interest in supporting patients with Ataxia.