A new compound which blocks the most common genetic cause of familial amyotrophic lateral sclerosis (ALS), a disease which disconnects muscles from nerves and leads to death, has been announced by Scripps Research chemist Matthew Disney PhD. The compound works differently to most drugs on the market, binding with the materials involved in making the toxic protein behind the disease instead of with the protein itself, like most market drugs.

The DNA damage responsible for ALS sits in a non-coding section of the ninth chromosome. A repeat of letters there causes the cell to begin producing a toxic protein, C9RAN, which is thought to disrupt the nerve cell’s normal metabolism. The material creator of the protein is a particular form of RNA folded over like a hairpin.

The molecule created by the scientists, referred to as “4” in the Cell Chemical Biology journal where it was published, interferes with the production of the C9RAN protein. Interestingly, the researchers noted that they believe the form of RNA being targeted by most drugs today is not the one which is actually driving neuron death.

The scientists must now prove that the molecule is both effective and safe. The research is ongoing.