Article preview from “The Pink Sheet”- February 25 , 2013
New understanding of the spectrum of CNS disorders and rising interest from biotechs have revealed new targets, new biomarkers and new opportunities to treat the fast-acting and deadly neurodegenerative disease. Add to that an open stakeholder forum hosted by FDA Feb. 25 that could clarify regulatory and clinical expectations, and the tide may be turning on amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis has never attracted the same interest from the pharmaceutical industry as fellow neurodegenerative disease multiple sclerosis, which unlike the aggressive and fatal ALS offers a chronic treatment opportunity – but the possibility that ALS lies among a spectrum of related diseases that could form a network of niche indications could be a major draw for sponsors.
ALS occurs in the U.S. at about the same rate as multiple sclerosis, but it is often fatal within two to five years after diagnosis. An orphan indication also referred to as motor neuron or Lou Gehrig’s disease, ALS is not a market with time to progress through a series of pricy therapies the way multiple sclerosis patients do.
But the flow of genomic data informing every aspect of medical research is infusing neurology with new energy and insights into the mysteries of central nervous system disorders. Through that molecular-level looking glass, ALS is being exposed as a heterogeneous disease that may well turn out to be a spectrum of disorders that is part of a larger spectrum of spectrums, in which many neurodegenerative diseases are interrelated.