Foundation for Angelman Syndrome Therapeutics (FAST) Funds Human Clinical Trial

Could an acne treatment transform lives for kids with a rare disease

Australian families dealing with the rare disease, Angelman Syndrome (AS), have a glimmer of hope on the horizon as a small clinical trial starts in the US this month.

The FDA approved drug, Minocycline, has been used to treat adult acne and other bacterial infections in several organ systems. In addition to being an antibiotic, Minocycline has also been found to have neuroprotective properties. Researchers found that Minocycline restored motor function and cognition in the mouse model for the syndrome.

Angelman Syndrome is a genetic neurocognitive disorder that significantly impacts children neurologically from birth rendering few able to speak more than a few words, some unable to ever walk and most suffer from severe, and at times uncontrollable seizures. AS affects all races and both genders equally and occurs in approximately one in 10,000 to 15,000 live births.

The US trial, sponsored by the Foundation for Angelman Syndrome Therapeutics (FAST) and conducted by the University of South Florida, will enroll 24 children with Angelman Syndrome between 4 and 12 years of age in order to determine if Minocycline is a viable treatment for AS. In the meantime, families of children with Angelman Syndrome around the world will hold their breath and wait.

President of FAST (Australia) and current FAST (US) Board Member, Meagan Cross said, This is a really exciting time for everyone in the AS community. The short amount of time it has taken to get this drug to testing and then to trial is something we as a foundation are all really proud of, needless to say its pretty exciting to be at the forefront of making things happen.

Work has already begun with specialists from the Mater Medical Research Institute in Queensland on preparing to expand the clinical trial to Australia if the initial findings are positive and could start by the end of 2012. continued Cross.

Well-known actor and FAST supporter Colin Farrell summed it up well when speaking of the upcoming trial and his son James who has AS, It doesnt mean that I think theres anything wrong with James. It doesnt mean I have any doubts about the foundation of his spirit. The foundation of his heart and the foundation of his mind are better and purer than mine will ever be. But there are aspects to his physical self that dont do him any favors he said.

The US based trial has been made possible through unprecedented support for the Foundation in a Facebook competition last year reaping the first prize pool of $250,000; these monies do not extend to Australia, where significant funds are still required to support a clinical trial.

Information about the clinical trial is the at FAST website