The year since May 2006 has witnessed several steps in the evolution of ALS research. Results aren’t yet available for several causative factor and treatment studies, but in many cases, they’re expected soon.
MDA’s “whole-genome association” project conducted at the Translational Genomics Institute (TGen) in Phoenix yielded some potentially important and previously unidentified differences between the DNA of ALS-affected and unaffected study subjects. These results will likely provide raw material for a number of future investigations.
To block post-DNA genetic instructions (RNA), “antisense” RNA can be used to target “sense” RNA. Researchers hope to block erroneous, ALS-causing instructions for the SOD1 protein.
This year, with funding made possible by MDA’s Augie’s Quest initiative, MDA also teamed up with the Cambridge, Mass., ALS Therapy Development Institute. This state-of-the-art laboratory specializes in efficient testing of the biochemical effects of compounds of interest in cells alone and in mice with ALS.
In another area, a technology known as “antisense,” which blocks genetic instructions, has matured to the point that an MDA-supported clinical trial of antisense in a familial form of ALS is contemplated for the near future. In this trial, an antisense compound will block incorrect and highly toxic instructions for the SOD1 protein, a known cause of ALS.
Also of note are recent findings that inflammation, a natural process that occurs when the body feels under siege, isn’t just a marker of ALS but is likely a contributor to it. In ALS, inflammation is in the nervous system and is carried out by cells known as microglia. They’re likely to become targets of research and treatment trials soon.
Here’s a summary of where we are on several fronts: