Efficacy of Fetal Stem Cell Transplantation in Autism Spectrum Disorders: An Open-Labeled Pilot Study

Abstract

Autism spectrum disorders (ASDs) are heterogeneous complex neurodevelopmental pathologies defined by behavioral symptoms, but which have well-characterized genetic, immunological, and physiological comorbidities. Despite extensive research efforts, there are presently no agreed upon therapeutic approaches for either the core behaviors or the associated comorbidities. In particular, the known autoimmune disorders associated with autism are appealing targets for potential stem cell therapeutics. Of the various stem cell populations, fetal stem cells (FSCs) offer the potent immunoregulatory functions found in primordial mesenchymal stem cells, while exhibiting rapid expansion capacity and recognized plasticity. These properties enhance their potential for clinical use. Furthermore, FSCs are potent and implantable "biopharmacies" capable of delivering trophic signals to the host, which could influence brain development. This study investigated the safety and efficacy of FSC transplantations in treating children diagnosed with ASDs. Subjects were monitored at pre, and then 6 and 12 months following the transplantations, which consisted of two doses of intravenously and subcutaneously administered FSCs. The Autism Treatment Evaluation Checklist (ATEC) test and Aberrant Behavior Checklist (ABC) scores were performed. Laboratory examinations and clinical assessment of adverse effects were performed in order to evaluate treatment safety. No adverse events of significance were observed in ASD children treated with FSCs, including no transmitted infections or immunological complications. Statistically significant differences (p < 0.05) were shown on ATEC/ABC scores for the domains of speech, sociability, sensory, and overall health, as well as reductions in the total scores when compared to pretreatment values. We recognize that the use of FSCs remains controversial for the present. The results of this study, however, warrant additional investigations into the mechanisms of cell therapies for ASDs, while prompting the exploration of FSCs as "biopharmacies" capable of manufacturing the full array of cell-signaling chemistry. This manuscript is published as part of the International Association of Neurorestoratology (IANR) special issue of Cell Transplantation.

Mesenchymal Stem Cell Transplantation Promotes Neurogenesis and Ameliorates Autism Related Behaviors in BTBR Mice

“…Importantly, clinical studies evaluating MSC efficacy are currently underway for a variety of brain disorders (Tanna & Sachan, ). In the context of ASD, several studies have provided encouraging data on the potential of fetal stem cells as well as umbilical cord‐ and bone marrow‐ derived mononuclear cells, which include MSC, in children suffering from ASD (Bradstreet et al, ; Lv et al, ; Sharma et al, ).…”

Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism

“…Clinical trials of cell therapy in patients with ASD are still in the early phases, with a handful of exploratory studies underway in several different countries. Cell sources for these studies include autologous (69) or allogeneic umbilical CB, autologous bone marrow (70), fetal stem cells (71), and mesenchymal stromal cells derived from adipose tissue or umbilical cord tissue. To date, results of four clinical trials have been published.…”

Immunological Dysfunction in Autism Spectrum Disorder: A Potential Target for Therapy

“…A significant lowering of ATEC overall score was seen 12 months after therapy, and in total ABC scores after 6 and 12 months. No side effects were observed [111].…”