Breakthrough to find a cure for muscular dystrophy

Those that suffer from muscular dystrophy lack the function to repair and regenerate injured muscles.

However, scientists from the Centre of Cellular and Molecular Biology (CCMB) have revealed a pathway which sets the stage for additional research which could help to formulate drugs for those who suffer from the disease.

Further research will be required by the CCMB scientists on dystrophic mice to know whether the infusion of chemicals in the pathway can work towards a cure.

As a result of an injury, wear and tear or traumatic muscle damage in the body – the sleeping muscle stems wake up. Although these stems are always present, it does not mean they are active. The muscle only becomes active after there is damage caused to the muscle.

Dr. Jyotsna Dhawan, chief scientist at CCMB, alongside her colleague Ajoy Aloysius, identified a pathway in normal mice where the stem cells were triggered awake during injury and as a result, this worked towards regeneration of the muscle.

Dr. Dhawan said, “Awakening of the sleeping cells shows that the body mechanism is in place to repair the damage. In those who are dystrophic, either the stem cells get exhausted due to the constant workload or they require outside intervention wherein they can work towards repair. This process is yet to be understood and needs further research on dystrophic mice.”

The ‘cross-talk’ between two signalling pathways works like a switch for the stem cells, said CCMB director, Dr. Rakesh Kumar Mishra.

Dr. Mishra explained, “This is an important phenomenon in biology as the switch brings in a new hope that there can be drug therapy possible in cases of disease of the muscle.”

There are two proteins that are the signalling mechanisms these are called ‘Lef1’ and ‘Smad3’, which are identified as switching the genes on and off.

“The finding will help to devise new therapeutic strategies to enhance the stock of stem cells in diseased muscles. Following this line of research can potentially improve muscle regeneration and reduce muscle deterioration,” he said.

In muscular dystrophy individuals, there is a deficiency of the X chromosome, which commonly occurs in males. It is a rare disease and a common effect is lack of mobility which initially starts with the inability to walk and then later leads to becoming confined to bed.