Mount Sinai Researchers Receive NIH Grant to Study Promising Treatment for Autism Subtype
Mount Sinai scientists have received a grant from the NIH to study IGF-1, a promising treatment for a subtype of autism.
Scientists at the Seaver Autism Center at the Icahn School of Medicine at Mount Sinai have received a grant from the National Institutes of Health (NIH) to study Insulin-Like Growth Factor-1 (IGF-1), a promising treatment for a subtype of autism called Phelan McDermid Syndrome (PMS). The grant will allow researchers to expand upon an ongoing study assessing the clinical benefit of IGF-1 in children with this severe type of autism.
IGF-1 is a commercially-available compound for growth deficiency that is known to promote nerve cell survival as well as synaptic maturation and plasticity. The primary aim of the study is to target core features of PMS, including social withdrawal and language impairment, which will be measured using both behavioral and objective assessments. So far, nine patients have participated in a pilot study to assess safety and feasibility of IGF-1. The Seaver Autism Center team hopes to enroll 18 more participants with support from the NIH grant, in order to establish statistically significant clinical benefit of IGF-1. The NIH will provide more than $750,000 over three years to study IGF-1.
With the grant, Alex Kolevzon, MD, Clinical Director the Seaver Autism Center, will continue to enroll children ages 5 to 12 years old who have PMS in this double-blind, placebo-controlled crossover study. Patients will first receive three months of either active medication or three months of placebo. After a four-week break, patients who received active medication first will then receive three months of placebo, and patients who were first randomized to placebo will receive three months of active medication. Future trials are planned to explore the utility of IGF-1 in ASD without SHANK3 deficiency, the hallmark genetic mutation in PMS.
Dr. Kolevzon has also received a grant for $25,000 from the Autism Science Foundation to study IGF-1 as a treatment for idiopathic autism.
“IGF-1 has the potential to be effective in treating Phelan-McDermid Syndrome and other types of autism spectrum disorder,” said Dr. Kolevzon. “We are very pleased that the NIH and the Autism Science Foundation have recognized this by providing us funding to continue our work in bringing this medication to our patients.”
The clinical studies with IGF-1 are supported by studies in a genetically modified mouse with a mutation in SHANK3. These studies carefully examined brain function in the mice when SHANK3 was mutated, and provided preclinical evidence for a beneficial effect of IGF-1. Deficits in nerve cell communication were reversed and deficiencies in adaptation of nerve cells to stimulation, a key part of learning and memory, were restored. These studies were reported the April 27th issue of Molecular Autism.
Side effects of IGF-1 administration include low blood sugar, liver function abnormalities, and increased cholesterol and triglyceride levels. Study subjects will undergo rigorous safety screening before they are enrolled in the trial, and will be carefully monitored every two to four weeks with safety and efficacy assessments.
To learn more about the Seaver Autism Center, visit http://icahn.mssm.edu/research/centers/seaver-autism-center.
About The Mount Sinai Medical Center
The Mount Sinai Medical Center encompasses both The Mount Sinai Hospital and Icahn School of Medicine at Mount Sinai. Established in 1968, the Icahn School of Medicine is one of the leading medical schools in the United States, with more than 3,400 faculty in 32 departments and 14 research institutes. It ranks among the top 20 medical schools both in National Institutes of Health (NIH) funding and by U.S. News & World Report. The Mount Sinai Hospital, founded in 1852, is a 1,171-bed tertiary- and quaternary-care teaching facility and one of the nation’s oldest, largest and most-respected voluntary hospitals. In 2012, U.S. News & World Report ranked The Mount Sinai Hospital 14th on its elite Honor Roll of the nation’s top hospitals based on reputation, safety, and other patient-care factors.
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About the Mount Sinai Health System
The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care.
The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals."