Navigation Links
Researchers from Mount Sinai receive NIH grant to study promising treatment for Autism subtype
Date:8/25/2013

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.


'/>"/>

Contact: Press Office
newsmedia@mssm.edu
212-241-9200
The Mount Sinai Hospital / Mount Sinai School of Medicine
Source:Eurekalert

Related medicine news :

1. NIH awards $20 million over 5 years to train next generation of global health researchers
2. Researchers develop a new cell and animal model of inflammatory breast cancer
3. Researchers uncover a viable way for colorectal cancer patients to overcome drug resistance
4. Researchers Find Gene Mutations That May Be a Key to Autism
5. Researchers find evidence of banned antibiotics in poultry products
6. NJ stroke researchers report advances in spatial neglect research at AAN Conference
7. Autism by the numbers: Yale researchers examine impact of new diagnostic criteria
8. Researchers Map Brain Regions Linked to Intelligence
9. Researchers ID Genes That May Determine Mental Illness
10. Researchers Develop Blood Test for Depression
11. University of Cincinnati researchers win $3.7M grant from US Department of Defense
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/18/2017)... ... January 18, 2017 , ... From a health perspective, 2017 will clearly be ... health to chronic disease, mental health and general physical well-being. The New York Times ... to consider. , For one Charlottesville restaurant, good gut health is clearly on the ...
(Date:1/18/2017)... ... January 18, 2017 , ... ... of Bioengineering are collaborating on a research project focused on multiple sclerosis (MS). ... seeks to use nanotechnology to control the disease without compromising normal immune function ...
(Date:1/18/2017)... ... January 18, 2017 , ... ... leading journal and most-read publication among specialty pharmacists and pharmacy professionals, has ... its Strategic Alliance Partnership (SAP) program, announced Brian Haug, president of Pharmacy ...
(Date:1/18/2017)... Palm Beach, FL (PRWEB) , ... January 18, 2017 , ... ... as a fund raiser for Smile Train, an international charity that provides free surgery ... a hobby of mine and in the past I have run to support the ...
(Date:1/18/2017)... (PRWEB) , ... January 18, 2017 , ... The Portee ... to families and business owners in central Maryland and the DC region, is inaugurating ... disease kills 787,000 people nationally every year, making it the #1 killer in America. ...
Breaking Medicine News(10 mins):
(Date:1/18/2017)... India , January 18, 2017 ... Imaging Technologies Market by Type: Global Opportunity Analysis and Industry ... size was valued at $2,544 million in 2015 and is ... CAGR of 8.4% from 2016 to 2022. North ... accounted for over three-fourths market share in 2015. Ionizing breast ...
(Date:1/18/2017)... N.J. , Jan. 18, 2017   Regenicin, ... a biotechnology company specializing in the development and commercialization ... damaged tissues and organs, recently reported the Company,s operating ... for 2017. As the Company described in ... been a year of substantial accomplishments. The Company,s contract ...
(Date:1/18/2017)... , Suiza, 18 de enero de 2017 /PRNewswire/ ... líderes lanzaron Access Accelerated, una iniciativa global para avanzar ... (NCD) y atención en países de renta baja y ... han alcanzado un punto de crisis, particularmente en países ... el 80 por ciento de las muertes relacionadas con ...
Breaking Medicine Technology: