PHILADELPHIA, Oct. 29, 2016 /PRNewswire/ -- Medgenics, Inc. (NASDAQ: MDGN) – New data presented at AACAP's 63rd Annual Meeting (Oct. 24-29, 2016 in New York, NY) confirmed the presence of specific genetic mutations in many children with Attention Deficit Hyperactivity Disorder (ADHD). The study team, led by Dr. Josephine Elia, M.D., Neuroscience Center, Department of Child and Adolescent Psychiatry, Nemours/Alfred I. DuPont Hospital for Children, studied a US population of children suffering from ADHD. The data confirm the observations previously made by Dr. Elia and colleagues at The Children's Hospital of Philadelphia (CHOP) in 2010 and underscore the importance of the role of glutamate receptors (mGluR) in diseases like ADHD. The study was sponsored by Medgenics, Inc.
"There is increasing interest in the critical role of glutamate neurotransmission in ADHD and other neuropsychiatric disorders," said Josephine Elia, M.D., Neuroscience Center, Department of Child and Adolescent Psychiatry, Nemours/Alfred I. DuPont Hospital for Children and principal study investigator. "This study suggests that mutations that can disable genes in this critical network can be causally associated with ADHD. Such mutations are present in up to 25 percent of children with ADHD, and suggest new genomics targeting strategies to better treat the disease."
A total of 23 investigators in centers across the USA enrolled 1,013 children, aged 6-17 years, with established ADHD. Phenotype data was collected and saliva samples were submitted to The Center for Applied Genomics (CAG) at CHOP for genotyping. Overall, the mutation frequency was 22%, with a higher prevalence of 25% observed in patients aged 6-12. Interestingly, when compared to mutation negative ADHD patients, the patients with the mGluR mutations were more likely to have concerns about anger control and disruptive behaviors. Medgenics is currently conducting additional research to better understand the relative severity of ADHD in patients with a network mutation and the contribution of individual genes.
"The results of this study support our ongoing Phase 2/3 interventional adolescent ADHD trial, known as SAGA" said Liza Squires, M.D., Vice President, Research & Development, Medgenics, Inc. "Our ultimate aim is to develop a genomically targeted, safe and superior product for this subpopulation of ADHD patients. This would be the first such targeted therapy in any CNS disease and highlights the emergence of precision medicine in this therapeutic area."
ADHD is the most common neurodevelopmental disorder of childhood and is generally characterized as a persistent pattern of inattention and/or hyperactivity-impulsivity that interferes with functioning or development. Approximately 11% – or 6.4 million – of U.S. children (4-17 years of age) have been diagnosed with ADHD, according to a recent study conducted by the Centers for Disease Control and Prevention (CDC) and the Health Resources and Services Administration.
Poster 6.67: Glutamatergic Network Gene Mutations in Adolescents and Children with Attention Deficit Hyperactivity Disorder (ADHD)
Saturday, October 29, 2016 | 10:00am-12:30pm ET
Josephine Elia, M.D., Neuroscience Center, Department of Child and Adolescent Psychiatry, Nemours/Alfred I. DuPont Hospital for Children, presented findings estimating the prevalence of rare, recurring copy number variants (CNVs) of specific metabotropic glutamate receptor (GRM, mGluR) and related network genes in a pediatric population with ADHD.
An abstract of the accepted presentation can be accessed on AACAP's 63rd Annual Meeting website.
About the SAGA Trial
The purpose of this multicenter, dose-optimized trial in adolescents with ADHD is to confirm the results from the Phase 1b GREAT study. The trial is designed as a randomized, double-blind, placebo-controlled, parallel-group Phase 2/3 study of NFC-1 versus placebo in adolescent patients with ADHD who have genetic disorders impacting the mGluR network. The trial will enroll 90 patients between the ages of 12 to 17 years old. The primary and key secondary endpoints in the trial will be the change from baseline in the ADHD-rating scale Total Score (ADHD-RS-5) and change from baseline in Clinical Global Impression - Global Improvement Scale (CGI-I). Patients will be randomized 1:1 to receive either a six-week course of NFC-1 or placebo, with a one-week follow-up. Patients will be enrolled from the same 25 sites that were used in the recent phenotype/genotype study. More information on the SAGA trial is available at www.ClinicalTrials.gov (Identifier: NCT02777931).
About Medgenics, Inc.
Medgenics, Inc. is dedicated to unlocking the potential of genomic medicine to identify and treat patients with life-altering conditions. Its efforts, including its internal research and development and ongoing sponsored research and licensing agreements with a well-respected pediatric academic medical center, give Medgenics the ability to focus on the underlying genetic pathway of pediatric diseases with the goal of finding therapeutic solutions for subpopulations of both children and adults living with rare and other difficult-to-treat diseases. Medgenics is also the developer of TARGT™ (Transduced Autologous Restorative Gene Therapy), a proprietary gene therapy platform. For more information, visit the Company's website at www.medgenics.com.
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 Centers for Disease Control and Prevention. Attention Deficit Hyperactivity Disorder. Available at http://www.cdc.gov/ncbddd/adhd/index.html. Accessed September 9, 2016.
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