Sheba News Page New 2019 25

Sheba’s Angelman Syndrome Clinic Joins International Network of ASF Accredited Clinics

In December 2018, the Angelman Clinic at Sheba Medical Center became the first clinic outside North America to join the Angelman Syndrome Foundation’s (ASF) global network of certified clinics. Sheba’s clinic is located in the Edmond and Lily Safra Children’s Hospital, a comprehensive pediatric facility on the vast Tel HaShomer medical campus, and the ASF is based in Chicago, Illinois.

The highly respected Israeli clinic was established only seven years ago by Dr. Gali Heimer, a pediatric neurologist. Since then, it has evolved into the fourth largest clinic worldwide dedicated to Angelman syndrome. Presently, there are 100 known cases of Angelman syndrome in Israel, and Dr. Heimer and her team treat 85 of them. Patients travel from across the country to visit all the experts they need in one day, all under one roof.

Angelman syndrome is a complex neurological disorder; it is genetic and occurs in approximately one in 15,000 births. Most people with this illness suffer from many physical and mental disabilities, and they cannot speak. Angelman afflicts all population groups, and it has not been associated with any ethnic, racial, geographic, or gender component.

Statistically, Israel (with a population of 9 million) should have at least 400 Angelman patients. But as Dr. Heimer explains, “The reason we don’t have 400 is that when many of them were born, there was no good testing for Angelman. A lot of relatively old undiagnosed patients are probably being taken care of in group homes, where they’re considered ‘intellectually disabled.’”

Safra Childrens hospital

The Edmond and Lily Safra Children’s Hospital

The Angelman clinic at Sheba generally receives patients once a month, and Dr. Heimer sits with each person for at least an hour. Along with Heimer, the staff includes five multidisciplinary medical professionals: an endocrinologist, orthopedist, dietitian, psychiatrist, and adolescent specialist.

“Our holistic approach saves families the need to come back many times, which is very difficult — especially for people who don’t live nearby,” said Heimer. “Another advantage is that over time, our physicians see a lot of Angelman patients and become experts on the subtle nuances of treating the disease.”

At the Sheba clinic, patients are helped with many serious issues caused by Angelman, such as seizures and digestive problems, as well with more “trivial” problems – such as sleep disorders, dysfunctional behavior, constipation, and drooling.

Not only does the Israeli Angelman clinic provide patients with the most progressive care possible, it also enables Sheba to attract international clinical trials and the development of new therapies. One such trial is the New York-based Ovid study on treating Angelman with OV101 (gaboxadol), a pill that can be swallowed whole or dissolved into powder.

The Phase 2 Ovid study, also called the STARS trial, demonstrated that OV101 improved behavioral abilities, mobility, and sleep in adolescents and adults with Angelman. 78 patients participated in STARS, ten of whom were from Israel. Ovid’s seminal Phase 3 trial is currently underway, and Ovid plans to submit a New Drug Application to the US FDA for approval as a treatment for people with Angelman.

The Angelman Syndrome Foundation’s annual conference will be held at Sheba Medical Center on December 5, where Ovid CEO Jeremy Levin will discuss the drug in detail.

“It’s my hope that OV101 will be covered by the Israeli health basket, because it really improved many of the symptoms, and increased quality of life for both the patient and their family,” said Heimer. “There’s a lot of promise for Angelman patients these days. It’s a very good time to be treating these patients. We didn’t have this hope five years ago.”