FAST Creating Training Center for Clinical Trials at Rush University

$5 million gift aims to bring promising Angelman therapies into testing and use

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by Steve Bryson, PhD |

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The Foundation for Angelman Syndrome Therapeutics (FAST) has given a gift of $5 million to establish the Rush F.A.S.T. Center for Translational Research, intending to train physicians to run clinical trials and deliver therapies requiring specialized delivery and care.

Reported to be the first-of-its-kind, the center will be directed by Elizabeth Berry-Kravis, MD, PhD, affiliated with Rush University Medical Center in Illinois, and work to establish new clinical trial and translational research efforts for rare neurodevelopmental disorders, such as Angelman syndrome.

While many physicians successfully run care facilities for people with neurological conditions, few have direct training in setting up, running, and reporting on clinical trials, which can limit trial enrollment and slow therapeutic development.

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Promoting trials as therapy development reaches ‘a turning point’

“This is the dawn of a new era,” Allyson Berent, FAST’s chief science officer, said in a press release. “Today, there are over 25 therapeutic programs in the development pipeline for Angelman syndrome, with a majority robustly funded by FAST.

“We are at a turning point, where we are ready for many of these programs to reach human patients for early-stage first-in-human clinical trials, but most hospital centers don’t have the bandwidth to keep up with this exploding need,” Berent added.

The new center will establish a fellowship program and provide organized training for individuals focusing on clinical trial implementation and novel delivery systems for rare neurological genetic disorders. Both national and international physicians will be trained in various areas, including understanding the steps to set up a clinical trial, mastering regulatory hurdles, providing infrastructure for patient testing, and putting together specialized clinical teams.

FAST fellows will have hands-on training for one year, focusing on running clinical trials for neurodevelopmental disorders.

In addition, the programs will support the creation of a state-of-the-art facility at Rush, with clinical trial capacity to meet patient needs. When FAST fellows complete their training and return to institutes worldwide, FAST aims to financially support the upgrading of clinical trial centers and establish new trial sites.

Angelman syndrome is a genetic disorder marked by developmental delays, neurological problems, and seizures. So far, available medications can only control seizures, and physical, communication, and behavioral therapy can help manage symptoms. Research into new potential Angelman treatments, including gene therapy, is ongoing.

“We are on the cusp of so many vital breakthroughs, which is why it is an honor to be named director of the Rush F.A.S.T. Center at such a critical time,” Berry-Kravis said. “This is going to help so many people, and I am looking forward to working with the brightest minds in the field to generate the medical breakthroughs of tomorrow.”