The November 25, 2008 issue of the journal Neurology has two interesting pieces about the search for a treatment for spinal muscular atrophy (SMA): one is an article and the other is an editorial about the merits of the article’s subject.

In their editorial “Linking SMN to SMA: An assay for the rescuer,” Dr. Carsten Bonnemann and Dr. Richard Finkel give an overview of the current understanding of the genetic cause of SMA and how this is being applied to the search for therapies that lessen the severity of the disease. They then point out that there needs to be a reliable way to measure the results of these therapies to verify their effectiveness. The article entitled “A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy” introduces a new way to do just that. According to the editorial’s authors, while the two-site ELISA needs additional validation, it “advances the prospects of drug discovery efforts and clinical trials” and “holds promise of moving the field forward toward a therapy.”

FightSMA and Dr. Finkel have worked together in the past, when Dr. Finkel was a panelist for the “Thriving with SMA” panel at the 2007 FightSMA Annual Conference and when FightSMA assisted Dr. Finkel in distributing his 2006 survey of parents of SMA children. Two of the article’s authors, Dr. Alex MacKenzie and Dr. Glenn Morris, are also connected to FightSMA. Dr. MacKenzie is co-chair of FightSMA’s Scientific Advisory Committee and Dr. Morris has received funding from FightSMA for his research into an ELISA assay for SMN.