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:2/23/2017)... ... 23, 2017 , ... Dr. David Mahon leads Siena Dental, a comprehensive dental ... named a 2017 Top Patient Rated Henderson Dentist by Find Local Doctors ... local physicians and dentists who have earned high ratings and superior patient reviews from ...
(Date:2/23/2017)... ... February 23, 2017 , ... ... clinical trials, today announced that Premier Research, a leading clinical development service provider, ... trials are becoming increasingly complex, due in part to an array of circumstances ...
(Date:2/23/2017)... , ... February 23, 2017 , ... ... publication and community for those in the fight against cancer, has produced a ... their success. HRA will release top-line findings in a webinar, Defining Compassionate ...
(Date:2/23/2017)... VA – , ... ... coming. It’s the perfect opportunity for the nation to come ... a college basketball bracket – with its favorite fruit – ... is encouraging apple lovers to join the “Apple Madness” bracket ...
(Date:2/23/2017)... Los Angeles, CA (PRWEB) , ... February 23, 2017 , ... ... of her Smiling Patriot program today with a new Indiegogo campaign ... dental care to homeless veterans in the Los Angeles area, either as a participating ...
Breaking Medicine News(10 mins):
(Date:2/23/2017)... Group, Inc. (NASDAQ: MDXG), the leading regenerative medicine company ... and market advanced products and therapies for the Wound ... sectors of healthcare, today announced its record results for ... 2016. Full Year 2016 Highlights are: ... full year 2015 revenue ...
(Date:2/23/2017)...   SeraCare Life Sciences , a manufacturer ... manufacturers and clinical laboratories, is announcing an upcoming ... Tests" to be hosted by GenomeWeb ... Standard Time (US). The goal of ... improved performance and global standardization in the implementation ...
(Date:2/23/2017)... , February 23, 2017 The U.S. ... legalized cannabis for recreational use in 2016. According to a ... market was worth an estimated $7.2 billion in 2016 and ... of 17%. While adult recreational sales are projected to jump ... with a 25% CAGR. The market may grow faster as ...
Breaking Medicine Technology: