Stem Cells Publications








1st Published article on AUTISM

    • Intrathecal Autologous Bone Marrow Mononuclear Cell Transplantation in a Case of Adult Autism


    Autism is a complex neurodevelopmental disorder with a worldwide prevalence of 1 in 88. With greater understanding of mechanism of action of cellular therapy it is now possible to address the pathogenesis of autism. Recent findings of cellular therapy offering immunomodulatory, angigogenetic and paracrine effects make it a lucrative option for treatment of autism. We administered a 33 year old adult patient of autism intrathecally, with autologous bone marrow mononuclear cells (BMMNCs), twice with an interval of six months. On follow up at 3, 6 and 9 months post first intervention, he was re-evaluated clinically and no major or minor side effects were observed. At 6 and 9 months objective outcome measures of Indian Scale for Assessment of Autism (ISAA) and Clinical Global Impression (CGI) were used and they showed significant improvement. At the end of 9 months, on ISAA, the score improved from 94 to 64. The CGI showed improvement by change in severity of illness from 3 (Mildly ill) to 1 (Borderline mentally ill). Global improvement on CGI was scored 2 (much improved) with an efficacy index of 5 (moderate therapeutic effect). PET CT scan was repeated at 6 months which showed a balancing effect in the metabolism of affected areas. The changes observed on the PET CT scan correlated with clinical improvements. MRI remained same at 6 months thereby, indicating that PET CT scan may serve as a better monitoring tool for effects of cellular therapy. In this case study, we hypothesize that cellular therapy has repaired the neural connections and achieved balance in the excitatory and inhibitory neuronal cells by various mechanisms of neuroprotection, neuromodulation and neurorestoration. Cell therapy holds great potential and randomized controlled trials may be conducted to study their long term effects in treating autism. Read more...

    • Amelioration of Autism by Autologous Bone Marrow Mononuclear Cells and Neurorehabilitation: A Case Report


     Autism is a clinically and etiologically heterogeneous disorder characterized by deficits in social interaction, communication, behavior and cognitive skills. The etiological basis of autism still remains poorly understood despite several attempts to decipher its neuropathology from different perspectives. Presently available treatment modalities address only limited autism-associated symptoms and are at best palliative. In this report, we present the case of a 7 year old boy with autism treated with intrathecal administration of autologous bone marrow derived mononuclear cells (BMMNCs). On regular follow ups conducted at 3 and 6 months post-treatment, clinically significant behavioral, social, communication and cognitive improvements were reported. These findings were well supported by objective improvements on the Indian Scale of Assessment of Autism (ISAA), Childhood Autism Rating Scale (CARS), Clinical Global Impression (CGI) and Pediatric Functional Independence Measure (WeeFIM). The ISAA score improved from 131 to 112, CARS improved significantly from 40.5 (severely autistic) to 32 (mild to moderate autism), along with an improved WeeFIM score from 31 to 36. Severity of illness on CGI (CGI I) changed from 4 (moderately ill) to 3 (mildly ill). Global improvement on CGI (CGI II) was measured at a score of 2 (much improved), along with an efficacy index (CGI III) of 5 showing moderate therapeutic effect. No adverse events were reported throughout the course of the treatment. Through this case report, we demonstrate that treatment with autologous BMMNCs is safe, feasible and has the potential to ameliorate autism. Read more...

    • An Improved Case of Autism as Revealed by PET CT Scan in Patient Transplanted with Autologous Bone Marrow Derived Mononuclear Cells


    Autism, the most severe form of autism spectrum disorder (ASD), is a complex neurodevelopmental disorder, characterized by language developmental delay, social skills impairment, communication problems, and restricted, repetitive, and stereotyped patterns of behavior. There is no cure for Autism; hence therapies and behavioral interventions are designed to remedy specific symptoms. We used autologous bone marrow derived mononuclear cells intrathecally in a 14 yr old boy with severe autism to improve the quality of life. At six months, follow up after therapy the general impression on clinical assessment showed mild autism. It is exciting to see symptomatic improvement with shift on Childhood Autism Rating Scale (CARS) from 42.5 (Severely Autistic) to 23.5 (Non Autistic), which was also visualized as enhanced PET scan brain function. All these improvements have led to improved quality of life of the patient as well as the family. Several incurable neurological disorders have shown benefits with cellular therapy thus, autism should be explored as an indication and nuclear imaging can be used to study its effects.


    • Autologous Bone Marrow Mononuclear Cell Therapy for Autism: An Open Label Proof of Concept Study


    Cellular therapy is an emerging therapeutic modality with a great potential for the treatment of autism. Recent findings show that the major underlying pathogenetic mechanisms of autism are hypoperfusion and immune alterations in the brain. So conceptually, cellular therapy which facilitates counteractive processes of improving perfusion by angiogenesis and balancing inflammation by immune regulation would exhibit beneficial clinical effects in patients with autism. This is an open label proof of concept study of autologous bone marrow mononuclear cells (BMMNCs) intrathecal transplantation in 32 patients with autism followed by multidisciplinary therapies. All patients were followed up for 26 months (mean 12.7). Outcome measures used were ISAA, CGI, and FIM/Wee-FIM scales. Positron Emission Tomography-Computed Tomography (PET-CT) scan recorded objective changes. Out of 32 patients, a total of 29 (91%) patients improved on total ISAA scores and 20 patients (62%) showed decreased severity on CGI-I. The difference between pre- and postscores was statistically significant (𝑃<0.001) on Wilcoxonmatched-pairssignedranktest. On CGI-II 96% of patients showed global improvement. The efficacy was measured on CGI-III efficacy index. Few adverse events including seizures in three patients were controlled with medications. The encouraging results of this leading clinical study provide future directions for application of cellular therapy in autism. Read more...

    • Autologous Bone Marrow Mononuclear Cells may be Explored as a Novel Potential Therapeutic Option for Autism


    Cellular therapy has been viewed as a novel therapeutic modality for many neurological disorders. Autologous Bone Marrow Mononuclear Cells (BMMNCs) used in many studies, have a safe and ethical profile. These cells have been studied in great depth and have shown angiogenetic and immunomodulatory properties in addition to other neuroprotective effects. These peculiar mechanisms may serve to be beneficial in autism. Recently, hypoperfusion and immune alteration are identified as major underlying pathogenetic mechanisms in autism. We present a case of autism with comorbid mental retardation; treated with intrathecal administration of autologous BMMNCs. Results were documented objectively on Indian Scale for Assessment of Autism (ISAA) and Positron Emission Tomography Computed Tomography (PET CT) scan. On regular follow up assessment of the patient over 18 months, there was significant clinical improvement in social relationship, communication and behavior. On outcome measure, ISAA score improved from 111 (Moderate autism) to 73 (Mild Autism). PET CT scans comparison of pre and post therapy showed balancing effect on brain metabolism. This case provides a great insight into the clinical effects of autologous BMMNCs in autism. Though a case study, the improvements guide us to plan future studies to explore different options of cellular therapy in autism. Read more...

    • Neuropsychiatric Disorder Tackled by Innovative Cell Therapy: A Case Report in Autism


    Autism is a pervasive developmental disorder affecting socialization, communication and behavior. Neuropathology of autism spectrum disorders is poorly understood and may involve impaired connectivity in the brain, selectively affecting parts of the brain forming circuits supporting social behavior. The currently available treatment options do not address the core neuropathology of autism. Hence, it is important to develop a treatment modality for autism at the earliest. Cell therapy is recently emerging as a potential treatment option for autism. We hypothesize that it may assist in the repair of the disrupted neuronal circuit. The neural repair may take place not only by cellular restoration but also by paracrine and immunomodulatory effects. Cellular therapy has shown promising results for various other neurological disorders. We administered autologous bone marrow mononuclear cells, intrathecally in an 11 year old boy diagnosed with autism. He was assessed for a follow up period of eight months wherein his autistic features had reduced. This was objectively supported by improvement in scores of CARS (31 to 25), ISAA (130 to 98), CGI-I (6 to 5) and FIM (104 to 110). This case study may hint towards exploring cell therapy as a potential treatment alternative for autism, in addition to standard approach. A longer period of follow up along with functional imaging may further help us understand the repair of the impaired neuronal circuit at cellular level. Read more...