The Foundation for Angelman Syndrome Therapeutics (FAST) recently entered a partnership with Rush University in order to establish a clinical trial and translational research center for rare neurodevelopmental disorders. It will be called the Rush F.A.S.T. Center for Translational Research and will be directed by Dr. Elizabeth Berry-Kravis. The center was established with a $5 million donation from FAST. The center is anticipated to be a global headquarters that will help train personnel in the delivery of novel treatments and the operation and execution of clinical trials for genetic disorders.
Recently, Patient Worthy spoke with Alana Newhouse, President of FAST, to learn more.
“We now have four clinical trials under way, with more in the pipeline. Most of trials are quite complicated, and not everyone that works in this space has the experience to run these complicated clinical trials and administer these types of innovative therapies.”
About Angelman Syndrome
Angelman syndrome is a type of genetic disorder that impacts the central nervous system. This disorder is usually not inherited from one’s parents, but instead occurs as a spontaneous genetic alteration, typically resulting from the deletion of a segment of chromosome 15 involving a gene called UBE3A. A mutation of this gene can also cause Angelman syndrome. Symptoms of this disorder can include seizures, developmental delays, an excited and happy demeanor, trouble with balance and movement, sleep challenges, and a near universal lack of verbal speech. Patients may share certain facial characteristics. Symptoms generally become noticeable at about one year of age. Currently, treatment is only supportive and symptomatic, and there is currently no cure. Patients have a strong desire for social interaction, and despite limited language are able to understand much of what is happening around them. Life expectancy is generally close to that of an unaffected person. To learn more about Angelman syndrome, click here.
Preparing for the Future
FAST began to realize that as more of these complex trials were reaching fruition, there was a major need for specially trained clinicians and other professionals to conduct them.
“Dr. Berry-Kravis is a dynamo in the universe of clinical trials. She runs them very efficiently and we wanted other people to benefit from her expertise and arborize her efficiencies and skillset to other clinicians globally.”
This goal was the genesis of the new translational research center that will soon be operational at Rush University. The center aims to establish a fellowship program for candidates from around the world. Once a FAST fellow completes training at the center, which will last one year, they will be able to share their knowledge at other centers around the world, improving the effectiveness of future trials for neurogenetic conditions like Angelman syndrome, of which there are hundreds if not thousands.
To learn more about this initiative, click here.
