• Application Of Autologous Bone Marrow Stem Cells In Giant Axonal Neuropathy

Abstract

Giant axonal neuropathy is a rare disorder of autosomal recessive inheritance, morphologically characterized by accumulation of neurofilaments in enlargements of preterminal regions of central and peripheral axons. We present a 7-year-old girl with thick and tightly curled lackluster hair suffering from giant axonal neuropathy. The diagnosis was confirmed on the brain MRI which showed white matter abnormalities in the anterior and posterior periventricular regions as well as the cerebellar white matter. In view of the same, the patient was given intrathecal autologous bone marrow-derived stem cell therapy as part of the neuroregenerative rehabilitation therapy protocol. The patient showed functional improvements in her disability after receiving the therapy. A detailed case report is presented here with. Read more...

Administration of Autologous Bone Marrow Stem Cells Intrathecally in Multiple Sclerosis Patients is Safe and Improves their Quality-of-life

Abstract

Multiple sclerosis (MS) is widely believed to be an autoimmune disorder characterized by multifocal lesions of the central nervous system (CNS) myelin and accumulating clinical signs due to axonal damage. Persistent loss of myelin can make axons more vulnerable to repeated injury, induce axons to make compensatory changes in their properties that can result in further delayed insults to the axon. Autologous hematopoetic stem cell transplantation (HSCT) has been used as a treatment for MS. Stem cells have the ability to greatly assist in regenerating the damaged glial cells. We present herein a case of a patient who underwent transplant twice to improve his disability. Read more...

• Seizures as an Adverse Event of Cellular Therapy in Pediatric Neurological Disorders and its Prevention

Abstract

This study reports seizures as an adverse event (AE) in children with neurological diseases treated with autologous bone marrow mononuclear cells (BMMNCs) intrathecal transplantation. It also assesses the effectiveness of antiepileptic prophylactic regimen to reduce the incidence of seizures as an AE. Seizures were considered as an AE if there were increased pre-existing seizures or new onset seizures. In part I; seizures as an AE was analyzed in detail. Seizures occurred as an AE in 8/131 (6%) in part I. Out of these 8, one patient’s EEG was not available, but showed increased preexisting seizures and 7 had a pre-existing epileptogenic focus on EEG. Out of these 7, 3 developed new onset seizures and 4 had increased pre-existing seizures. Based on this analysis an antiepileptic prophylactic regimen was designed. In Part II effect of this regimen was studied and a significant decrease in the incidence of seizures as an AE was observed along with no new onset seizures. Though small proportions of patients show seizures as an AE of cellular therapy, it definitely requires attention and is preventable. Pre-existing epileptogenic focus is an independent predictor for seizures as an AE. Future clinical trials of cellular therapy in pediatric population may consider these findings.

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• Administration of Autologous Bone Marrow-Derived Mononuclear Cells in Children With Incurable Neurological Disorders and Injury Is Safe and Improves Their Quality of Life

Abstract

Neurological disorders such as muscular dystrophy, cerebral palsy, and injury to the brain and spine currently have no known definitive treatments or cures. A study was carried out on 71 children suffering from such incurable neurological disorders and injury. They were intrathecally and intramuscularly administered autologous bone marrow-derived mononuclear cells. Assessment after transplantation showed neurological improvements in muscle power and a shift on assessment scales such as FIM and Brooke and Vignos scale. Further, imaging and electrophysiological studies also showed significant changes in selective cases. On an average follow-up of 15 ± 1 months, overall 97% muscular dystrophy cases showed subjective and functional improvement, with 2 of them also showing changes on MRI and 3 on EMG. One hundred percent of the spinal cord injury cases showed improvement with respect to muscle strength, urine control, spasticity, etc. Eighty-five percent of cases of cerebral palsy cases showed improvements, out of which 75% reported improvement in muscle tone and 50% in speech among other symptoms. Eighty-eight percent of cases of other incurable neurological disorders such as autism, Retts Syndrome, giant axonal neuropathy, etc., also showed improvement. No significant adverse events were noted. The results show that this treatment is safe, efficacious, and also improves the quality of life of children with incurable neurological disorders and injury. Read more...

• Adult Stem Cells for Spinal Muscular Atrophy

Abstract

Spinal muscular atrophy (SMA) is a genetic disease that attacks the motor neurons of the spinal cord. This results in the progressive wasting of the voluntary muscles of the limbs and trunk. We present a 26 year old girl diagnosed with SMA since last ten years. The diagnosis was confirmed on the basis of muscle biopsy and electromyography (EMG) showing anterior horn cell involvement. In view of the same; the patient was given intrathecal autologous bone marrow derived stem cell therapy as part of the neuron regenerative rehabilitation therapy (NRRT) protocol. The patient showed functional improvements in her disability post therapy. A detailed case report is presented here with. Read more...