Funding Of First Clinical Trial With Disease-Modifying Therapy For Rett Syndrome Announced By IRSF

Main Category: Neurology / Neuroscience
Also Included In: Clinical Trials / Drug Trials;  Pediatrics / Children's Health;  Genetics
Article Date: 22 Sep 2009 - 2:00 PDT

email to a friend   printer friendly   opinions  

Today, the International Rett Syndrome Foundation announced that it will provide $200,000 in 2009 to support a newly proposed clinical trial with a growth-factor based treatment for Rett Syndrome. The study will be carried out by a team of clinical trials specialists at the Boston Children's Hospital, led by Dr. Omar Khwaja MD, PhD and will be the first potentially disease modifying therapy ever to be tested in Rett syndrome patients. Investigators will test a drug called Increlex® . This is an engineered form of the human protein Insulin-like Growth Factor-1, an FDA approved drug for children with short stature due to severe Primary IGF-1 deficiency. Preliminary evidence in animal models of Rett syndrome has recently suggested that targeting the IGF-1 signaling axis may provide a potential avenue for therapy.

"We are pleased to announce the funding of this groundbreaking new clinical study which presents a novel therapeutic strategy for the treatment of Rett syndrome" said Dr. Antony Horton Chief Scientific Officer at the International Rett Syndrome Foundation. "This study will be carried out by a collaborative network of leading clinicians and neuroscientists in some of the World's most prestigious research institutes at the cutting edge of biomedical research. Through our funding of this study we demonstrate our commitment to moving forward the most advanced treatments to prevent, treat and ultimately reverse Rett syndrome".

Dr. Omar Khwaja, principal investigator on the study commented further "Since the 2007 studies which demonstrated potential reversal of Rett syndrome in animal models, there has been a paradigm shift in our thinking about ways to treat this devastating condition. Our pilot study will utilize an existing FDA approved medication, seeking to boost growth of synapses in the brain of patients with Rett syndrome".

Increlex® will be provided gratis by Tercica Inc., A Subsidiary of the IPSEN Group, who have reviewed and approved the trial through their internal scientific review board.

Dr. Khwaja and his team have also secured additional funding for this clinical trial through a grant provided by Harvard University's Catalyst Pilot Awards for Clinical Translational Research

IRSF provides careful due diligence on all clinical studies involving patients with Rett syndrome since safety is of paramount importance. We accomplish this through a rigorous peer-review process, drawing on the external advice and expertise of leading clinical trials specialists in the fields of Rett syndrome, Autism, Pediatric Neurology and other relevant disciplines as appropriate.

Source:
Mary Joyce Griffin
International Rett Syndrome Foundation

• Additional
• References
• Citations