Title:Recent Advances in the Treatment Strategies of Friedreich’s Ataxia: A Review
of Potential Drug Candidates and their Underlying Mechanisms
Volume: 30
Issue: 19
Author(s): Aman Kumar Saini, Neha Anil, Ardra N. Vijay, Bharti Mangla*, Shamama Javed*, Pankaj Kumar and Waquar Ahsan
Affiliation:
- Department of Pharmaceutics, School of Pharmaceutical Sciences, Delhi Pharmaceutical Sciences and Research University (DPSRU),
New Delhi 110017, India
- Department of Pharmaceutics, College of Pharmacy, Jazan University, Jazan, P. Box No. 114,
Saudi Arabia
Keywords:
Friedreich ataxia, molecular pathway, neurodegenerative disorder, cardiomyopathy, diabetes, mitochondrial protein.
Abstract:
Background: Friedreich's Ataxia (FRDA) is a rare hereditary neurodegenerative disorder characterized
by progressive ataxia, cardiomyopathy, and diabetes. The disease is caused by a deficiency of frataxin, a
mitochondrial protein involved in iron-sulfur cluster synthesis and iron metabolism.
Objective: This review aims to summarize recent advances in the development of treatment strategies for FRDA,
with a focus on potential drug candidates and their mechanisms of action.
Methods: A comprehensive literature search was conducted using various authentic scientific databases to
identify studies published in the last decade that investigated potential treatment strategies for FRDA. The
search terms used included “Friedreich's ataxia”, “treatment”, “drug candidates”, and “mechanisms of action”.
Results: To date, only one drug got approval from US-FDA in the year 2023; however, significant developments
were achieved in FRDA-related research focusing on diverse therapeutic interventions that could potentially
alleviate the symptoms of this disease. Several promising drug candidates have been identified for the
treatment of FRDA, which target various aspects of frataxin deficiency and aim to restore frataxin levels, reduce
oxidative stress, and improve mitochondrial function. Clinical trials have shown varying degrees of success,
with some drugs demonstrating significant improvements in neurological function and quality of life in
FRDA patients.
Conclusion: While there has been significant progress in the development of treatment strategies for FRDA,
further research is needed to optimize these approaches and identify the most effective and safe treatment options
for patients. The integration of multiple therapeutic strategies may be necessary to achieve the best outcomes
in FRDA management.
