The New York TimesSaturday January 5, 2013 7:31 AM
A closely watched experimental drug to treat Lou Gehrig’s disease failed to work in a late-stage clinical trial, the drug’s developer, Biogen Idec, said, disappointing medical experts who had hoped that it might be a useful tool against the poorly understood and fatal illness.
The company said Thursday that it would discontinue work on the drug.
Biogen said the drug was not effective in either slowing the loss of muscular function or prolonging the lives of people with the disease, formally known as amyotrophic lateral sclerosis, or ALS.
There also were no signs that the drug, dexpramipexole, worked in any subgroup of patients, Biogen said.
“As a physician who has treated people with ALS, I hoped with all my heart for a different outcome,” Dr. Douglas Kerr, director of neurodegeneration clinical research at Biogen, said in a statement.
ALS, which attacks the nerves that control muscles, causes gradual paralysis and typically results in death within a few years of diagnosis.
Only one drug has been approved to treat it, Rilutek, made by Sanofi, which doctors say is only moderately effective. Many other drugs have failed in clinical trials, in part because scientists do not understand the cause of ALS.
While expectations were not high that dexpramipexole would succeed in its Phase 3 trial, there was more optimism than for many previous ALS drugs, given what some doctors viewed as strong results in a smaller, Phase 2 trial.
The only hope for ALS patients now is in stem cells trials and research. As I followed Iplex years ago, now I follow Neuralstem (Nyse: CUR) which the next 8th of January will give un update on 18 ALS patients. At least one of them (Ted Harada) has recieved a strong improvement from stem cells injections. Why not hoping to combine Iplex with stem cells treatement to arrive finally to defeat this horrible desease?