PRECISION MEDICINE FOR ALS

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.

Precision Medicine for ALS

ALS GENE DISCOVERY

ALS Progression

Brown et al., 2017. N Engl J Med 2017; 377:162-72. DOI: 10.1056 / NEJMra1603471

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.

JOIN US IN THE FIGHT AGAINST ALS

Whether you or a loved one is battling ALS and you want to be a part of our clinical trials, or you want to invest in the fight against this debilitating disease, we want to hear from you. Fill out the form below today and we will get back to you as quickly as possible.

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