New Delhi, May 23
Treatment with an experimental drug has shown significant improvements in young patients with a rare form of Amyotrophic Lateral Sclerosis (ALS) -- a progressive neurodegenerative disorder, said a team of US researchers on Friday.
ALS, also known as Lou Gehrig's disease, is a rare disorder that affects nerve cells in the brain and spinal cord, leading to the loss of motor neurons causing difficulty with movement, balance, coordination, and potentially even breathing.
While experimental therapies have so far slowed down the disease or halted its progression, the new treatment using ulefnersen (previously known as jacifusen) -- showed that functional losses in young patients can be reversed.
"When testing new drugs for ALS, we do not expect to see clinical improvement," said neurologist and scientist Neil Shneider at Columbia University.
But, "what we've seen in one patient is really unprecedented functional recovery. It's surprising and deeply motivating for us, the ALS research community, but also the community of ALS patients," he added.
Data from 12 patients -- all treated with the novel therapy for a rare form of ALS caused by a genetic mutation in a gene called FUS -- were presented in a case series published by Shneider online in The Lancet.
Though these gene mutations are responsible for only 1-2 per cent of ALS cases, they cause some of the most aggressive forms of ALS that begin in adolescents and young adults.