|Neurologist Valerie Cwik, senior executive vice president-research and medical director at MDA, and neurologist Stan Appel, a longtime MDA research grantee and director of the MDA/ALS Center at Methodist Neurological Institute in Houston, at the Oct. 24 meeting
An all-day "think tank" to consider priorities for amyotrophic lateral sclerosis (ALS) research based on the latest information was conducted with ten of the leading scientists and clinicians in the field and senior MDA staff in Tucson, Ariz., on Saturday, Oct. 24, 2009.
Each specialist introduced his or her area of expertise and led an approximately 20-minute discussion, gathering input and answering questions from the other participants.
The topics included explorations of the state of current knowledge about several aspects of ALS, the contributions of each aspect, and some ideas about therapeutic targets.
Discussions covered the cutting-edge of ALS research:
- ALS-related defects in the mitochondria, the parts of cells where energy is produced;
- oxidative stress, a type of cellular damage that results from accumulation of metabolic byproducts;
- dysfunction of a protein known as TDP43;
- ER stress, a type of cell damage that results from effects on the endoplasmic reticulum, a microscopic network inside cells;
- injury to oligodendrocytes, cells in the central nervous system that make an insulating coating for nerve fibers;
- positive and negative contributions of astroglia, cells that normally support the primary nerve cells (neurons);
- positive and negative contributions of microglia, immune-system cells of the nervous system;
- cell replacement therapies in ALS;
- blocking of harmful genetic instructions (such as instructions that lead to the manufacturing of toxic SOD1 protein molecules in one form of familial ALS) with compounds known as antisense oligonucleotides;
- the potential role of growth factors, such as vascular endothelial growth factor, in the treatment of ALS;
- what has been and can be learned from clinical trials of lithium; and
- what we know about the emerging field of lipid (fat) metabolism in this disease.
As a result of this session and follow-up communications among the attendees, a prioritized list of research directions will be created.
Participants in the meeting were Jinsy Andrews, Stanley Appel, Robert Baloh, Merit Cudkowicz, Jonathan Glass, Michio Hirano, Robert Miller, Timothy Miller, Hiroshi Mitsumoto and Jeffrey Rothstein.