The research is an international collaboration sponsored by an American pharmaceutical company.
Tofersen is a new form of gene therapy that inhibits the new production of so-called SOD1 protein. The protein, when it mutates, is thought to be one of the causes of ALS.
The researchers conducted so-called phase 1 and phase 2 studies to investigate the safety of using the drug in patients with ALS who have the SOD1 gene mutation.
Unexpected effects
However, the researchers found that the drug, in the patients who received the highest doses, also delayed the development of the disease and resulted in better muscle function and lung strength than in the patients who received placebo or low doses of the drug.
It was also seen that the degree of nerve damage decreased significantly in the patients who received the drug.
A larger phase-3 study in ALS patients with SOD1 gene mutation has now been initiated to investigate whether the results can be further confirmed.