The country’s first-ever human clinical trial for a drug to overcome a genetic disorder named Duchenne muscular dystrophy has been approved by the Drugs Controller General of India. This disorder affects around five lakh male children in India. Nine hospitals will conduct trials across the nation in 2021.
The drug is based on personalized genetic intervention and will be the first of it’s kind to treat this disease in the country.
As per the order issued by Dr. V G Somani (Drugs Controller General of India), Dystrophy Annihilation Research Trust (DART) will carry out multi-center clinical trials for the new drug/investigational new drug in nine selected hospitals situated in Maharashtra, Delhi, Chandigarh, Karnataka, Madhya Pradesh, Gujarat, Jaipur, and Telangana. Due to the lack of a protein named Dystrophin, loss of muscle mass and progressive weakness is observed in DMD affected children. This genetic disorder affects only males and those who suffer through this condition eventually require a wheelchair as the patients stop walking by the age of 8-12 due to lack of muscle mass.
Due to complications such as cardiac or respiratory failure, the average life expectancy for the patients is around 18 to 24 years. DMD has observed in around 3,500 boys in India annually.
Patients have to rely on medication and physiotherapy as no treatment is available yet for this rare genetic disorder. Patients having DMD have very low bone density hence, prone to fractures. DMD patients have moderate levels of static intellectual impairment and disabilities in learning, according to the National Organisation of Rare Disorders. Dystrophy Annihilation Research Trust has produced one of it’s a kind personalized medication for DMD which uses antisense oligonucleotides (AO) based on exon-skipping technology.
Laying down guidelines for undertaking the trials and identifying the clinical trial sites, the Drugs Controller in his order said that the trial should be registered with the Clinical Trial Registry of India maintained by the ICMR before enrolling the first subject for the trial.
The Drugs Controller General of India has laid down guidelines for conducting trials and identifying the critical sites for the clinical trials. The DCGI also mentioned that the trials must be registered with the Clinical Trial Registry of India which is maintained by the ICMR.