What role will genetic therapy play in ALS in the future?

Around 15-20% of all people with ALS are thought to have genetic changes that cause ALS or at least increase the risk of developing the disease. Gene therapy refers to various procedures to reduce or even correct the harmful effects of genetic changes (mutations). The two most important ALS genes are the SOD1 and C9orf72 genes.

In 2024, the first genetic therapy, the drug Tofersen (Qalsody) against SOD1 mutations, was approved in Germany. Further genetic drugs against mutations in the FUS or C9orf72 gene are expected to be developed in the next five years. Gene therapy is only effective if corresponding genetic changes (mutations) are also present. Genetic testing to determine whether mutations are present in ALS genes will become increasingly relevant in the future.