Treatment of Rett Syndrome with Copaxone

Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine, Children’s Hospital at Montefiore

$487,000 AWARDED

There is a multitude of data suggesting that mice models of Rett have low levels of a neurotrophic factor called BDNF (brain derived neurotrophic factor). BDNF is a very important and complex protein that is implicated in a variety of disorders. Increasing BDNF in the Rett mice models, either genetically or pharmacologically is beneficial. An FDA approved drug for multiple sclerosis called copaxone (or Glatiramer Acetate) is known for increasing BDNF and therefore of interest in treating Rett. It’s important to note that copaxone will not be a cure for Rett. The goal is symptom(s) improvement.

RSRT has funded an open label study of copaxone in two centers, the Tri-State Rett Syndrome Center at Children’s Hospital at Montefiore in the Bronx, under the supervision of Dr. Aleksandra Djukic, and at Sheba Medical Center in Ramat Gan in Israel under the supervision of Dr. Bruria Ben Zeev (budget of $200,000).

The trial in NY has finished and the results are encouraging. A paper has been submitted. A larger placebo controlled trial will need to be planned. The trial in Israel encountered some difficulties with several girls having allergic reactions and trial was stopped.

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