Major Points and Findings:
- Background: Cellular therapy is emerging as a potential treatment for autism. The primary pathogenetic mechanisms of autism are believed to be hypoperfusion (reduced blood flow) and immune alterations in the brain. Cellular therapy aims to counter these processes by improving blood flow through angiogenesis and regulating immune responses.
- Study Design: This is an open-label proof of concept study where 32 patients with autism underwent intrathecal transplantation of autologous bone marrow mononuclear cells (BMMNCs). Following the transplantation, patients also received multidisciplinary therapies. The follow-up period for all patients was 26 months, with an average of 12.7 months.
- Outcome Measures: The study utilized the ISAA (Indian Scale for Assessment of Autism), CGI (Clinical Global Impression), and FIM/Wee-FIM scales to measure outcomes. Additionally, Positron Emission Tomography-Computed Tomography (PET-CT) scans were used to record objective changes in the brain.
- Results:
- 29 out of 32 patients (91%) showed improvement in total ISAA scores.
- 20 patients (62%) exhibited decreased severity on the CGI-I scale.
- The difference between pre-and post-scores was statistically significant (P < 0.001) based on the Wilcoxon matched-pairs signed rank test.
- On the CGI-II scale, 96% of patients demonstrated global improvement.
- The efficacy of the treatment was measured using the CGI-III efficacy index.
- PET-CT Scan Findings:
- Before the intervention, PET-CT scans showed reduced FDG uptake in areas of the brain such as the frontal lobe, cerebellum, amygdala, hippocampus, parahippocampus, and mesial temporal lobe.
- Six months after the intervention, PET-CT scans revealed increased FDG uptake in the same areas, indicating improved brain activity and perfusion.
- Adverse Events: Some adverse events were noted, including seizures in three patients. However, these were controlled with medications.
- Conclusion: The results from this study are promising and suggest that cellular therapy might be a beneficial treatment for autism. The improvements observed in the majority of the patients and the PET-CT scan findings provide a direction for future research and application of cellular therapy in treating autism.