GHP September 2015

ghp September 2015 | 7 New Study Will Attempt to Confirm Biometric Identifier of ASD Stemina Biomarker Discovery Inc. has announced the launch of the largest clinical study of the me- tabolism of children with autism spectrum disorder (ASD) ever conducted. The Children’s Autism Metabolome Project or “CAMP” study will enroll 1,500 subjects from six sites across the country. CAMP will attempt to confirm that sets of metabolic biomarkers can detect subtypes of ASD. This new study is an expanded version of Stemina’s three pi- lot studies of more than 500 children. CAMP is also designed to contribute to identification of new bio- markers with the goal of advancing a panel of tests for earlier diagnosis and more precise treatment of ASD based on the metabolism of patients. Metabolomics is the study of differences in a person’s metabolism as genomics is the study of differences in a person’s genes. Metabolom- ics focuses on identifying normal and abnormal patterns of small molecules which indicate the presence of illness. Altered metabolism of patients offers more insight into the individual patient and potential treatments that may be effective based on the patient’s own metabolism. Stemina’s proprietary metabolomics technology is capable of identifying not only biomarkers associated with disease, but also metabolite patterns associated with toxicology and cellular response to drugs or chemicals this may be used in future studies to try to identify envi- ronmental factors associated with autism and other neurological disorders. Stemina has received a $2.7mn grant from the National Institute of Mental Health (NIMH) to support CAMP. The study is also supported by a $2.3mn investment from the Nancy Lurie Marks Family Foundation (NLMFF). Children are being enrolled at the MIND Institute at the University of California – Davis, Arkansas Children’s Hospital Research Institute in Little Rock, Vanderbilt Uni- versity in Nashville, Cincinnati Children’s Hospital, Nationwide Children’s Hospital in Columbus, and the Melmed Center in Phoenix. CAMP requires the enrollment of 500 children with autism, 500 with other neurodevelopmental disorders without autism and 500 typically developing children in the 18 to 48 month age range. The research has the potential to enable earlier diagnosis and individualized treatment of children with ASD from a small blood sample. Stemina has conducted three independent proof-of-concept studies with more than 500 patients with ASD from the MIND Institute and Arkansas Children’s Hospital Research Institute. The first study was published in PLOS One in November with collabo- rators from the MIND Institute. “We are excited about participating in CAMP and continuing our work with Stemina. Confirmation of findings from our previous two pilot studies would be an important step towards developing an early diagnostic marker of ASD. Metabolomics may be the key to detecting clinically meaningful subtypes of autism,” said Dr. David Amaral, Distinguished Professor of Psychiatry and Research Director of Research of the MIND Institute at UC Davis. “Autism is a very complex disorder, really a series of disorders. CAMP has the potential to deliver some important diagnostic tools as well as to increase our understanding of these subtypes of autism from a metabolic perspective. Earlier diagnosis can lead to earlier intervention resulting in the most effective reduction of symptom severity.” Stemina Biomarker Discovery launches largest ever clinical study of metabolism of children with autism. Diagnosis of ASD at an early age is important for initiating the most effective intervention. Today, behavioral therapy is the standard of care for children with ASD. It is important that intervention begin as early as possible to achieve optimal out- come. Patients can be reliably diagnosed through behavioral testing at age 2 at health care facilities with expertise in diagnosing autism and yet the average age of diagnosis according to the Centers for Disease Control is 4½ years. Stemina’s goal is to significantly reduce the average age of diagnosis through a diagnostic panel of metabolic biomarkers being refined through the CAMP study. This will offer both improved and earlier diagnosis as well as the potential for individualized therapy and better outcomes for patients and their families. “Stemina’s proprietary metabolomics platform tech- nology will revolutionize the way ASD is diagnosed and treated,” said Elizabeth Donley, Chief Executive Officer of Stemina. “Autism is a spectrum disor- der from a cognitive and behavioral perspective. Stemina’s work is demonstrating that it is also a spectrum disorder from a metabolism perspective. By diagnosing ASD based on the patient’s metabo- lism, we hope to understand what is different about the metabolism of children with ASD and each subtype compared to typically developing children. This approach will open up a whole new frontier for understanding the disorder and how to treat it.”