Investigators at the Vinmec Stem Cell and Gene Technology Research Institute in Hanoi, Vietnam are studying the safety and efficacy of autologous bone marrow stem cell treatment for autism spectrum disorder in combination with rehabilitation and psychotherapy.
Autism Spectrum Disorder
The CDC reports that autism is present in 1 in 68 children in America. Autism is not a single medical condition but rather a spectrum of conditions characterized by repetitive behaviors, problems with verbal and nonverbal communication, and challenges with social skills. Autism spectrum disorder is believed to be caused by a combination of genetic and environmental factors. People with autism can have a wide variation in their strengths and challenges. People with autism may have associated mental health and medical issues such as sleep disturbances, phobias, anxiety, and gastrointestinal problems. Most often, autism is diagnosed in children between the ages of 2 and 3 and is typically associated with some developmental lag and intellectual disability. Early evaluation and intervention have been found to improve outcomes.
New Autism Treatment
Researchers are studying whether stem cell therapy in combination with rehabilitation and psychological therapy is safe and effective for children with autism. The phase 2 interventional study is an open-label, non-randomized, controlled clinical trial that will assess change in autism using tools such as CARS, DENVER II, autism diagnostic observation schedule 2nd edition, and the Indian scale for assessment of Autism. Study subjects will be evaluated 6 and 12 months following stem cell transplantation. The number of adverse events during the study period will also be recorded to assess the safety of the stem cell therapy.
The study has enrolled 60 patients aged between 3 and 7 years of both sexes. Healthy children are accepted into the study as volunteers. The results are expected in December 2018. Children with confirmed autism by DSM-IV criteria are eligible for the study. Children with certain medical conditions, including severe psychiatric issues, active infections, epilepsy, are excluded from the study.
Autologous Bone Marrow Stem Cell Therapy for Autism
The study participants will receive two intrathecal injections of autologous bone marrow mononuclear stem cells – one at the time of entry to the study and a second 6 months later. Additional interventions will include 3 months of psychotherapy and rehabilitation.