Biotechs show keen interest in new drugs for Down Syndrome

The New York Times tackles the story of Alberto Costa, a neuroscientist inspired by the birth of a daughter with Down syndrome to seek a treatment for the genetic disorder. After 15 years of investigation Costa is engaged in a randomized human trial for a potential new therapy, reports Dan Hurley in an exhaustive article. And other researchers in the field have reported significant progress as well.

Working with a mouse model of the disease, Costa studied the positive effects of memantine, an Alzheimer's drug, on the affliction. Because Down syndrome patients have three copies of the hundreds of genes found on Chromosome 21, Costa theorized that the drug worked because it quieted down an over-stimulated neurotransmitter. The NMDA transmitters in the mice model had become hyperactive, he theorized, and malfunctioned.

Other drug development strategies for Down syndrome which have shown promise include clearing excess amounts of beta amyloid, a protein which builds up in the brains of Alzheimer's patients. And researchers' work has captured the attention of biotech companies interested in pushing a drug through the clinical trial process.

"There's been a sea change in our ability to understand and treat Down syndrome," UC San Diego's Dr. William C. Mobley, one of the most prominent scientists in the field, tells The Times. "There's just been an explosion of information. As recently as the year 2000, no drug company would possibly have thought about developing therapies for Down syndrome. I am now in contact with no less than four companies that are pursuing treatments."

But some, including Costa, believe that new tests that will make it quite simple to spot Down syndrome in fetuses will make future cases rare.

- here's the article from The New York Times