Affordable Treatment of Duchenne Muscular Dystrophy (Dmd)

Pioneering Efforts In India For Affordable Treatment Of Duchenne Muscular Dystrophy (Dmd)

Source: The Hindu

       Introduction:

• Duchenne Muscular Dystrophy (DMD) is a rare and incurable genetic disorder, predominantly affecting boys, which leads to progressive muscle degeneration, weakness, and premature death.
• In India, researchers from the Indian Institute of Technology (IIT), Jodhpur, have taken up the challenge to develop an affordable treatment for DMD, aiming to alleviate the financial burden on affected families and increase the accessibility of therapeutic options.

Understanding Duchenne Muscular Dystrophy (DMD):

• DMD is characterized by alterations in the dystrophin protein, leading to muscle weakness and degeneration. The disorder initially affects proximal muscles and gradually spreads to distal limb muscles, hindering basic motor functions like walking, running, and jumping.
• Over time, patients experience enlargement of calves, a waddling gait, and lumbar lordosis. DMD can also affect the heart and respiratory muscles, leading to pulmonary function impairment and respiratory failure, making it the most common and fatal type of muscular dystrophy.

Current Challenges in DMD Treatment:

• The existing therapeutic options for DMD are scarce and prohibitively expensive, with treatment costs reaching up to Rs 2-3 crore per child per year. The predominantly imported treatments create significant financial barriers, rendering them unattainable for the majority of affected families in India.

Promising Efforts in Developing Affordable Therapeutics:

[A] Antisense Oligonucleotide (AON)-Based Therapeutics:

• The researchers at IIT Jodhpur are at the forefront of developing an affordable treatment for DMD based on Antisense Oligonucleotide (AON) technology. AONs have the ability to mask specific exons in a gene sequence, potentially addressing the challenges faced by DMD patients.

[B] Personalized Medicine Approach:

• The variations in mutations among DMD patients necessitate a personalized medicine approach. The research team is actively working on optimizing AON-based therapeutics to cater to individual genetic variations, improving treatment effectiveness.

[C] Multi-Centric Clinical Trials and Molecular Tags:

• To advance their research, the IIT Jodhpur team has received approval from the Drugs Controller General of India (DCGI) to conduct multi-centric clinical trials on AON-based exon skipping in DMD patients. This will pave the way for a more extensive evaluation of the treatment’s efficacy and safety.
• Furthermore, the researchers are exploring innovative methods to reduce the therapeutic dose of AON by incorporating new molecular tags. This step can potentially minimize treatment costs while maintaining or enhancing the treatment’s benefits.

The Conclusion:

• The researchers at IIT Jodhpur are making remarkable strides in the development of affordable therapeutics for Duchenne Muscular Dystrophy.
• By leveraging the potential of AON-based treatments, personalized medicine, and multi-centric clinical trials, they are bringing hope to thousands of affected families in India.
• Their efforts exemplify the significance of collaborative research in combating rare genetic disorders and advancing accessible healthcare options for all.