Prerna Badhe

By Alok Sharma, Hemangi Sane, Nandini Gokulchandran, Prerna Badhe, Pooja Kulkarni, Suhasini Pai, Ritu Varghese and Amruta Paranjape

Pediatric neurological disorders represent a major part of the disabilities worldwide. In over 10 decades of research to find a cure for these disorders, medical science has not been able to repair the underlying brain injury. This chapter focuses on recent advances in the application of stem cells as a therapeutic tool for some of the common neurodevelopmental disorders (cerebral palsy, autism, intellectual disability and muscular dystrophy). The mechanism of action of stem cells in each disorder has been explained. A review of clinical data has been described giving a clear understanding of current status of stem cell therapy in these disorders. Various factors influencing the outcome of stem cell therapy such as different types of cells, different routes of administration and dosage and frequency of transplantation have also been discussed. Our experience of treating these disorders is exhibited in the form of our published data. Use of novel monitoring tools such as MRI MSK and PET‐CT scan brain to track the changes occurring at cellular level after stem cell therapy are described. We also highlight the importance of a multidisciplinary approach of combining rehabilitation with stem cell therapy.

Part of the book: Physical Disabilities

By Alok Sharma, Hemangi Sane, Nandini Gokulchandran, Prerna Badhe, Amruta Paranjape, Radhika Pradhan, Rohit Das and Hema Biju

Motor neuron disease (MND) is an insidious, fatal disorder that progresses with the selective loss of anterior horn cells of the spinal column. Over 150 years since it was first described, various therapeutic approaches have been tested in the quest of a cure but with little success. Current standard therapy only improves lifespan by a few months; palliative care is the only option available for patients. Stem cell therapy is a potent approach for the treatment of this devastating disease. A multitude of vitalizing effects, both paracrine and somatic, a robust safety profile, as well as ease of availability make a strong case for using these cells for therapeutic purposes. Coupled with rigorous rehabilitation, this powerful treatment modality has been shown to slow disease progression, improve quality of life, and increase survival, along with being well tolerated by amyotrophic lateral sclerosis (ALS)/MND patients. Compelling preclinical as well as clinical evidence abounds that stem cells hold great potential as a therapy for ALS/MND. Although not a definitive solution yet, stem cells have been verified to have slowed and/or halted disease progression in a subset of ALS/MND patients.

Part of the book: Novel Aspects on Motor Neuron Disease

By Alok Sharma, Hemangi Sane, Nandini Gokulchandran, Prerna Badhe, Amruta Paranjape, Pooja Kulkarni and Vivek Nair

Spinal cord injury is one of the leading causes of disability worldwide. Current mainstay treatment strategies consist of surgical and medical management in acute and subacute stage. Rehabilitative management in the chronic stage. None of the existing strategies can repair the damage to the spinal cord and recover neurological functioning. Stem cells have promising results in pre-clinical and clinical studies. Various pre-clinical studies have evidenced neuro-regenerative capabilities of stem cells and shown neural recovery. Clinical studies have also shown improvements in neurological functions and quality of life. This chapter discusses about different types of cells available, routes of administration available to transplant these cells, dosages of cell and optimum time after injury at which cells should be transplanted based on world-wide literature. We have also discussed results following our protocol of intrathecal transplantation of autologous bone marrow mononuclear cells. Although, not a cure, stem cell therapy further improves quality of life, functional independence and reduces secondary complications when combined with existing treatment strategies; neuroregenerative rehabilitative therapy.

Part of the book: Spinal Cord Injury Therapy

By Alok Sharma, Hemangi Sane, Nandini Gokulchandran, Amruta Paranjape, Zubiya Shaikh, Arjun KM and Prerna Badhe

Muscular dystrophy is a major unmet medical need associated with an inevitable progressive muscle damage and loss of function. Currently, treatment is only symptomatic and supportive. This chapter focuses on cell therapy as a potential treatment approach for muscular dystrophy. Mechanism of action of cell therapy and its ability to alter disease pathology have been discussed. A review of preclinical and clinical studies has been presented with the advantages and shortcomings of various cell types. Rationale for our treatment protocol and experience of treating muscular dystrophy patients has been discussed. Our published results have shown the efficacy of the intrathecal and intramuscular administration of autologous bone marrow mononuclear cells in different types of muscular dystrophy patients. The scores on outcome measures such as 6-minute walk distance, North star ambulatory assessment, Brooke and Vignose scale, Functional independence measure, and manual muscle testing either improved or were maintained suggestive of slowing down disease progression. Efficacy and safety of the treatment was also studied using comparative MRI-MSK and EMG showing decreased fatty infiltration in various muscles post-cellular therapy. Thus, it was found that autologous BMMNC transplantation is a safe and effective treatment option and improves the quality of life of MD patients.

Part of the book: Potential Therapeutic Strategies for Muscular Dystrophy