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Preliminary Pages.P65 i Stem Cell Therapy & Other Recent Advances in Muscular Dystrophy (100 Case Reports) Dr. Alok Sharma Co-Authors : Dr. Prerna Badhe, Dr. Nandini Gokulchandran, Dr. Guneet Chopra, Dr. Mamta Lohia, Ms. Pooja Kulkarni NeuroGen Brain & Spine Institute, Chembur, Mumbai India. www.neurogen.in i ii Published by Neurogen Brain and Spine Institute Surana Sethia Hospital, Suman Nagar, Sion-Trombay Road, Chembur, Mumbai - 400071. Contact details : Tel. : (+91) 22 25281610 (+91) 22 65220066 (+91) 9920 200400 Email : [email protected] Website: www.neurogen.in Author Details : Dr. Alok Sharma Mobile : (+91) 9820046663 Tel. : (+91) 22 25283706 E-mail : [email protected] ii iii Stem Cell Therapy & Other Recent Advances in Muscular Dystrophy Dr. Alok Sharma, M.S., M.Ch. Professor of Neurosurgery and Head of Department LTMG Hopsital and LTM Medical College, Sion, Mumbai Director, NeuroGen Brain & Spine Institute, Chembur, Mumbai Consultant Neurosurgeon Fortis Hospital, Mulund, Mumbai, India Dr. Prerna Badhe, MD Neuropathologist NeuroGen Brain & Spine Institute, Chembur, Mumbai, India Dr. Nandini Gokulchandran, MD Head- Medical Services & Clinical Research NeuroGen Brain & Spine Institute. Chembur, Mumbai, India Dr. Guneet Chopra, MBBS, PGDM (Medical Genetics) Deputy Head- Medical Services & Clinical Research NeuroGen Brain & Spine Institute, Chembur, Mumbai, India Dr. Mamta Lohia, B.P.Th., F.N.R, M.I.A.P Physiotherapist and Clinical Research Associate NeuroGen Brain & Spine Institute, Chembur, Mumbai, India Ms. Pooja Kulkarni, M.Sc (Biotechnology) Clinical Research Scientist NeuroGen Brain & Spine Institute, Chembur, Mumbai, India iii iv Stem Cell Therapy & Other Recent Advances in Muscular Dystrophy © 2011 by NeuroGen Brain and Spine Institute ISBN 81-86876-07-3 All rights reserved. This book is protected by copyright. No part of this book may be reproduced in any form by any means, including photocopying, or utilized by any information storage and retrieval system without written permission from the copyright owner, except for the brief quotations embodied in critical articles and reviews. This book has been published in good faith that the material presented is original. Every effort is made to ensure accuracy of material, but the publisher, printer and author will not be held responsible for any inadvertent error(s). Cover Page by Leena A. Damle Published by NeuroGen Brain and Spine Institute Pvt. Ltd. Printed by SUREKHA PRESS A-20, Shalimar Industrial Estate, Matunga Labour Camp, Matunga, Mumbai 400 019 Price : ` 2000 iv v Scientific and Editorial Co-ordinators Ms. Pooja Kulkarni, M.Sc (Biotechnology) Dr. Mamta Lohia, B.P.Th., F.N.R., M.I.A.P Dr. Sanjay Kukreja, M.B.B.S. Special Contribution Dr. Jaykumar Nair (Consultant Radiologist) Dr. Bhavna Doshi (Consultant Neuro-electrophysiologist) Dr. Hema Sriram (Consultant Anesthetist) Mrs. Priya Karkera (Nutritionist) Dr. Sanjay Kukreja (Clinical Associate, Department of Neurosurgery, LTMG Hospital) Acknowledgement Dr. V. C. Jacob, B.Sc., DPT, M.I.A.P (Physiotherapist) Dr. Hema Biju, M.O.Th. (Occupational Therapist) Dr. Joji Joseph (Physiotherapist) Dr. Nancy Thomas, M.P.Th. (Neuro), M.I.A.P. (Physiotherapist) Dr. Priti Mishra, B.O.Th. (Occupational Therapist) Dr. Manasi Jani (Speech therapist) Ms. Akshata Shetty, M.A. (Clinical Psychologist) v vi 8th International Symposium on Experimental Spinal Cord Repair and Regeneration in Brescia, Italy, May 2011 Aaron Ciechanover Nobel Prize winner in Chemistry in 2004 "In basic science versus clinical medicine, there is a problem of time scale. In science, unrevealing secrets takes a lot of time but meanwhile, patients are suffering and even dying and therefore we need to find intermediate solutions for their problems." vi vii From President Obama’s Speech on Stem Cell Policy Change, March 9, 2009 President Barack Obama "There is no finish line in the work of science. The race is always with us - the urgent work of giving substance to hope and answering those many bedside prayers, of seeking a day when words like "terminal" and "incurable" are finally retired from our vocabulary" vii viii This Book is Dedicated to all our Patients and their Families and Caretakers viii ix Preface Over the last 150 years or so, the evolution of modern medicine, has had several success stories. From the wiping killer diseases like small pox off the face of the planet to reducing former killer diseases such as tuberculosis and the plague to treatable conditions, from advanced intervention and surgical techniques in cardiac conditions that have prevented many deaths to microsurgery for the brain, spine, eyes, ears and other organs that have given relief from suffering of many debilitating conditions, all of us are able to live better lives today due to the availability of many drugs and surgical procedures. There are however still some conditions that modern medicine had not been able to touch. Curing them was a distant dream even giving symptomatic relief or palliation was not possible. Many of these diseases were referred to as incurable neurological conditions. One such disease is Muscular Dystrophy. The tragedy of the muscular dystrophies was that it is a slowly progressive condition that over several years results in limb weakness reducing a normal human being to a wheelchair and later a bedridden existence. Limb contractures and scoliosis that occurred often result in a painful day to day existence. In the most common form of the muscular dystrophy , Duchene Muscular Dystrophy, death followed at the age of 20-25. The mental and emotional agony of parents watching their child slowly progress painfully to a certain death is indescribable. Many mothers had watched their brothers go through this and then having to watch their sons also go through the same agony over 15-20 years is something none of us in the medical profession can understand. DMD children are most often very cute, remarkably intelligent and have a very non complaining and positive attitude to life. Watching these courageous kids wither away slowly over several years was bad enough. Being told my multiple doctors that absolutely nothing could be done for them made things so much worse. But all this is now about to change. The development of regenerative medicine with the availability of cellular transplantation in the form of Stem Cells has given us the first major breakthrough in the management of Muscular Dystrophy. No longer do we have to turn away these patients with the advice that "nothing can be done". Despite the availability of stem cell therapy the majority of treating physicians are still not recommending these patients for this therapy. Their argument is that "there is not enough evidence available". In today's world of evidence based medicine this argument is justified. However for once we should look at this from the patients and their families point of view. It will take several years before class 1 evidence will be available in the form of prospectively randomized controlled muiltcentric studies. What do the patients do till then ? What if instead of looking at evidence based medicine we looked at practice based evidence. What is there was enough practice based evidence to show that this was a ix x completely safe therapy and had a reasonable degree of effectiveness. This book was written for this purpose. Although at the time of publishing this book, we have treated over 200 patients of muscular dystrophies, we are presenting in this book a summary of 100 patients detailing their clinical presentation, improvements after stem cell therapy both in symptoms, signs as well in their investigations. We believe that with this large body of evidence there is no need to wait till multicentric trials are done and class 1 evidence collected. Ethically this is in complete accordance with the World Medical Associations " WMA Declaration of Helsinki" which states that " In the treatment of a patient , where proven interventions do not exist or have been ineffective, the physician, may use an unproven intervention if in the physicians judgment if offers hope of saving life, re-establishing health or alleviating suffering. " In this book we have attempted to put together and summarize all the basic science and clinical research that has been done all over the world as well as share our own clinical results. Based on this we can now make a reasonable conclusion that the treatment of Muscular Dystrophy with adult stem cells combined with an aggressive rehabilitation program is a safe and effective form of therapy in slowing down/halting the progression of the disease and in producing functional and neurological improvements in the patients. To all the patients, their families, caretakers, therapists and doctors who have this question in their mind " Can Muscular dystrophy be treated", the answer all these years may have been no, but now, based on the evidence we have, we can say with a profound confidence :- "Yes we can". Dr. Alok Sharma x xi The Impossible Dream To dream the impossible dream, to fight the unbeatable foe, to bear with unbearable sorrow, to run where the brave dare not go. To right the unrightable wrong, to love pure and chaste from afar, to try when your arms are too weary, to reach the unreachable star. This is my quest, to follow that star -- no matter how hopeless, no matter how far. To fight for the right without question or pause, to be willing to march into hell for a heavenly cause. And I know if I'll only be true to this glorious quest that my heart will be peaceful and calm when I'm laid to my rest. And the world will be better for this, that one man scorned and covered with scars still strove with his last ounce of courage. To reach the unreachable stars. xi xii xii xiii Contents SECTION A: Basics and Management of Muscular Dystrophy 1. Introduction and Clinical Features ......................................................
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