The discovery of a drug that may treat the fatal disease known as Huntington’s is being hailed as “historic” by Louise Vetter, president and CEO of the Huntington’s Disease Society of America.

The results have been hailed as “enormously significant” because it is the first time any drug has been shown to suppress the effects of the Huntington’s mutation that causes irreversible damage to the brain. 

Current treatments only help with symptoms, rather than slowing the disease’s progression.

The results have also caused ripples of excitement across the scientific world because the drug, which is a synthetic strand

of DNA, could potentially be adapted to target other incurable brain disorders such as Alzheimer’s and Parkinson’s. 

The research, supported by Ionis Pharmaceuticals in Carlsbad, Calif., was conducted by scientists at more than half a dozen locations in Europe and Canada. This international team collaborated to create IONIS-HTTRx, the first drug to directly target the cause of Huntington’s.

The Huntington’s gene sends instructions via RNA for cells to create a protein. In people with the mutation, the protein is a toxic form that kills nerve cells and damages the brain. The new drug works by delivering a small piece of genetic material that sticks to the RNA and prevents it from building proteins.

The early phase clinical trial involved three dozen subjects with the disease and was conducted to establish the safety and tolerability of IONIS-HTTRx. There were no reported side effects.

Ionis has been awarded a $45 million license fee from its commercial partner, Roche, which will now manage all future studies.

Those diseases are more complicated, and involve multiple genes, but the idea of “silencing” mutant genes is an approach they think will be widely explored.

The good news for Huntington’s patients is the hope that the drug might even reverse the progression of the disease.

“What’s really interesting in animal studies, if you stop the production of the mutant protein, not only does progression stop, but the brain starts to heal itself,” said Christopher Ross, director of the Huntington’s Disease Center at Johns Hopkins Medicine, who was not involved in the research. “Which means there might be improvement.”

Read futher: The Guardian