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A novel approach of human embryonic stem cells therapy in treatment of Friedreich's ataxia
Geeta Shroff
MBBS, Director, Nutech Mediworld, New Delhi, India.

doi:10.5348/ijcri-201503-CS-10054

Address correspondence to:
Dr. Geeta Shroff
Nutech Mediworld, H-8, Green Park Extension
New Delhi-110016
India
Phone: +91 11 26180039
Email: +91 11 26560089

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How to cite this article
Shroff G. A novel approach of human embryonic stem cells therapy in treatment of Friedreich's ataxia. Int J Case Rep Images 2015;6(5):261–266.


Abstract
Introduction: Friedreich's ataxia (FRDA) is an autosomal recessive inherited disease that damages nervous system and impairs muscle coordination. FRDA usually begins in childhood and is caused by expanded GAA triplet repeat within the first intron of the frataxin (FXN) gene leading to reduced level of mitochondrial protein frataxin. There is no effective treatment for FRDA. If stem cells are transplanted near the affected cells under oxidative stress in FRDA patients, they can produce tropic factors, thereby increasing the survival of the cells. In FRDA, the mechanism to remove the reactive oxygen species (ROS) is impaired leading to oxidative stress and cell death. Stem cells may have ability to protect cells susceptible to oxidative stress that occurs in FRDA. In our previous studies we have shown the improvement in the patients' condition who were suffering from cerebral palsy and cortical visual impairment after human embryonic stem cells (hESCs) therapy.
Case Series: Herein, I report three cases of FRDA patients who were treated with hESCs therapy. All the patients were suffering from problems like difficulty in walking, standing or climbing stairs and muscle weakness. After undergoing hESCS therapy, improvement in condition of all the patients was observed.
Conclusion: The hESCs therapy was effective in treating patients with FRDA. Further research is required to understand the mechanism of action of hESCs.

Keywords: Friedreich's ataxia, GAA triplet repeat human embryonic stem cells, Neurodegenerative diseases


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Author Contributions:
Geeta Shroff – Substantial contributions to conception and design, Acquisition of data, Analysis and interpretation of data, Drafting the article, Revising it critically for important intellectual content, Final approval of the version to be published
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The corresponding author is the guarantor of submission.
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Conflict of interest
Authors declare no conflict of interest.
Copyright
© 2015 Geeta Shroff. This article is distributed under the terms of Creative Commons Attribution License which permits unrestricted use, distribution and reproduction in any medium provided the original author(s) and original publisher are properly credited. Please see the copyright policy on the journal website for more information.



About The Author

Geeta Shroff Founder of Nutech Mediworld, New Delhi, India, has developed hESCs therapy. She is a graduate in medicine from the University of Delhi and her postgraduation is in Gynecology & Obstetrics. Since 2002, more than 1300 patients suffering from various incurable conditions have been treated using hESCs. She has presented her work at various national and international forums. In 2014, she has published two studies on the use of hESC therapy in the treatment of patients with cortico visual impairment and cerebral palsy. Dr. Shroff envisions making hESC therapy available globally for many of mankind's worst afflictions.