New drug shows promise in treating Fragile X
By ANISaturday, January 8, 2011
WASHINGTON - A new drug to treat the underlying disorder instead of the symptoms of Fragile X has shown some promise in a study conducted by researchers from Rush University Medical Center.
Fragile X is the most common cause of inherited intellectual disability. It is the most common known single gene cause of autism or “autistic-like” behaviours.
Symptoms also can include characteristic physical and behavioural features and delays in speech and language development. The impairment can range from learning disabilities to more severe cognitive and intellectual disabilities.
The drug, called AFQ056, made by Novartis Pharmaceuticals, helped improve symptoms in some Fragile X patients.
Patients who had the best response have a kind of “fingerprint” in their DNA that could act as a marker to determine who should get treatment.
“This drug could be a model for treatment of other disorders such as autism,” said paediatric neurologist Dr. Elizabeth Berry-Kravis,
“The treatment period in this pilot study was very short and longer treatment might have been needed to see improvement in the whole group of patients. Importantly, the drug was well-tolerated and there were no safety problems,” said Berry-Kravis.
Further study is required to test the effects of a longer period of treatment.
The study is published in the January issue of Science Translational Medicine. (ANI)
