Tay-Sachs Drug Trial Yields ‘Neutral’ Results

By Marc Tracy

Published August 25, 2006, issue of August 25, 2006.
  • Print
  • Share Share

Two clinical studies for the treatment of late-onset Tay-Sachs with Zavesca, a drug used to treat Gaucher disease, produced neutral results — “a scientific way of saying that it didn’t work,” said Kim Crawford, director of member services for the National Tay-Sachs & Allied Diseases Association.

According to Crawford, Actelion, which produces Zavesca, will not seek FDA approval for the drug’s use on late-onset Tay-Sachs. Tay-Sachs is a lysosomal storage disorder that involves insufficient activity of the hexosaminidase A enzyme. Both Gaucher and late-onset Tay-Sachs disproportionately afflict Ashkenazic Jews.

Dr. Edwin Kolodny of New York University, who conducted one of the studies (the other was at Case Western Reserve University), was quick to emphasize that anecdotal evidence was far more promising. “Most of the patients who began the trial, and almost all the patients who remained at the end of the trial, want to continue taking the drug,” he said. “That means to me that they appreciate some positive benefits.”

Such benefits include improvement in speech patterns and articulation, reduction in psychotic episodes and general mental stabilization.

However, the hope was that Zavesca would stop or at least slow the decrease in muscle strength characteristic of such progressive neurodegenerative diseases as Tay-Sachs. “The data did not confirm that,” Kolodny said.

According to Kolodny, there are two other potential routes to treatment of late-onset Tay-Sachs. At least one biotechnology company, Genzyme, is developing a substrate synthesis inhibitor — the same type of drug as Zavesca — that would block production of the lipid that builds up in nerve cells of Tay-Sachs patients. It may soon be used in human trials.

The second option, known as the “chaperone molecule approach,” is currently in development. Here, doctors produce a small molecule in Tay-Sachs patients that can help correct the faulty hexosaminidase A enzyme.

Meanwhile, the jury is still out on trials currently being conducted for the use of Zavesca to treat juvenile and infantile (early-onset) Tay-Sachs. “The hope is that maybe in children and infants [Zavesca] is metabolized differently, and has an impact on the disease,” Crawford said.






Find us on Facebook!
  • When is a legume not necessarily a legume? Philologos has the answer.
  • "Sometime in my childhood, I realized that the Exodus wasn’t as remote or as faceless as I thought it was, because I knew a former slave. His name was Hersh Nemes, and he was my grandfather." Share this moving Passover essay!
  • Getting ready for Seder? Chag Sameach! http://jd.fo/q3LO2
  • "We are not so far removed from the tragedies of the past, and as Jews sit down to the Seder meal, this event is a teachable moment of how the hatred of Jews-as-Other is still alive and well. It is not realistic to be complacent."
  • Aperitif Cocktail, Tequila Shot, Tom Collins or Vodka Soda — Which son do you relate to?
  • Elvis craved bacon on tour. Michael Jackson craved matzo ball soup. We've got the recipe.
  • This is the face of hatred.
  • What could be wrong with a bunch of guys kicking back with a steak and a couple of beers and talking about the Seder? Try everything. #ManSeder
  • BREAKING: Smirking killer singled out Jews for death in suburban Kansas City rampage. 3 die in bloody rampage at JCC and retirement home.
  • Real exodus? For Mimi Minsky, it's screaming kids and demanding hubby on way down to Miami, not matzo in the desert.
  • The real heroines of Passover prep aren't even Jewish. But the holiday couldn't happen without them.
  • Is Handel’s ‘Messiah’ an anti-Semitic screed?
  • Meet the Master of the Matzo Ball.
  • Pierre Dulaine wants to do in his hometown of Jaffa what he did for kids in Manhattan: teach them to dance.
  • "The first time I met Mick Jagger, I said, 'Those are the tackiest shoes I’ve ever seen.'” Jewish music journalist Lisa Robinson remembers the glory days of rock in her new book, "There Goes Gravity."
  • from-cache

Would you like to receive updates about new stories?




















We will not share your e-mail address or other personal information.

Already subscribed? Manage your subscription.