Safety and Efficacy of The Stem Cell Transplantation in Friedreich’s Ataxia: A Report of Three Cases

Authors

  • Riza Azeri Istinye University, School of Medicine, Liv Hospital/Ulus, Department of Physical Therapy and Rehabilitation, Istanbul, Turkey.
  • Duygu Koyuncu Irmak Istinye University, School of Medicine, Department of Histology & Embryology, Istanbul, Turkey Topkapi Campus, Maltepe Mah., Teyyareci Sami Sk., No.3, Zeytinburnu, Istanbul, 34010, Turkey E-mail: Duygu.irmak@istinye.edu.tr
  • Eda Sun Istinye University, Stem Cell, and Tissue Engineering R&D Center, Istanbul, Turkey.
  • Erdal Karaöz Istinye University, School of Medicine, Department of Histology & Embryology, Istanbul, Turkey. Istinye University, Stem Cell, and Tissue Engineering R&D Center, Istanbul, Turkey. Istinye University, 3D Bioprinting Design & Prototyping R&D Center, Istanbul, Turkey. Liv Hospital/Ulus, Center for Stem Cell and Regenerative Therapies (LivMedCell), Istanbul, Turkey.

DOI:

https://doi.org/10.15621/ijphy/2021/v8i1/903

Keywords:

Friedreich’s ataxia, Hereditary Ataxia, Cellular Treatment, Regenerative Medicine, Stem Cell.

Abstract

Background: Friedreich’s ataxia is a progressive degenerative disorder caused by deficiency of the frataxin protein. Expanded GAA repeats in intron 1 of the FXN gene lead to its heterochromatinization and transcriptional silencing. Strategies being trialed to treat Friedreich’s ataxia include drugs that improve mitochondrial function and reduce oxidative injury. It has been shown that mesenchymal stem cell (MSC)-derived factors can restore cellular homeostasis and function to frataxin deficient cells.
Case Summary: Here, we report three FRDA cases treated with four consecutive allogeneic transplantations of umbilical cord-derived MSCs with 30 days interval, upon per patient regulatory approvals for advanced cellular therapy.
Outcome Measures: The cases were followed up after the treatment in means of the therapeutic effect of the cellular treatment by attenuating the neurological findings and gene expression parameters.
Conclusions: Closely followed promising safety and efficacy outcomes demonstrated that the MSC treatment for FRDA might positively affect the clinical results caused by the defect in this genetic-based disease.

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Published

2021-03-09

How to Cite

Riza Azeri, Duygu Koyuncu Irmak, Eda Sun, & Erdal Karaöz. (2021). Safety and Efficacy of The Stem Cell Transplantation in Friedreich’s Ataxia: A Report of Three Cases. International Journal of Physiotherapy, 8(1), 31–35. https://doi.org/10.15621/ijphy/2021/v8i1/903

Issue

Section

Original Articles