We now know that one therapy will not work for all ALS patients. The reason is that there are many different underlying causes of ALS. Researchers all over the world have now identified almost 30 different genes that can cause the development of ALS. Most of these genes have very different roles to play inside a cell. When these genes are not working well, we need to make a very specific medicine to help them work better. Looking back, it is not surprising that medicines that have been tested on ALS patients without understanding what causes their disease have not worked.
ALS GENE DISCOVERY
ALS has been discovered as a disease in 1862 when Jean-Martin Charcot recognized the unique degeneration of the motor system. It then took 131 years before Rosen and colleagues in 1993 found the first ALS gene, SOD1 which is responsible for the development of ALS in about 2% of patients. Since then, the success of the human genome project has accelerated the discovery of ALS genes by comparing the genome of healthy individuals to that of ALS patient. In 2011, a big breakthrough was made by the identification the ALS gene C9orf72 which accounts for about 10% of all ALS patients. New ALS genes, such as TBK1 are identified with increasing speed and researchers estimate that about 60% of the genes that cause familial ALS have now been found. We now also know that 50% of ALS genes can also cause Frontotemporal Dementia.