2 min read.Updated: 05 Oct 2020, 04:26 PM ISTSeethalakshmi S
The disease affects only boys, and those who have this condition eventually need a wheelchair because they stop walking by the age of 10 or 12
BENGALURU:The Drugs Controller General of India has approved the first human clinical trial for a drug to treat Duchenne muscular dystrophy, a genetic disorder that affects about five lakh boys in India. The trials will be conducted in nine hospitals across the country next year. This will be India’s first personalised genetic intervention to treat the disorder.
In the order, Drugs Controller General of India Dr V G Somani approved multi-centre clinical trials by the Dystrophy Annihilation Research Trust (DART) for a new drug or investigational new drug in nine hospitals located in Delhi, Karnataka, Telangana, Madhya Pradesh, Gujarat, Chandigarh, Jaipur, and Maharashtra.
DMD patients suffer from progressive weakness and loss of muscle mass due to lack of a protein called dystrophin. “The disease affects only boys, and those who have this condition eventually need a wheelchair because they stop walking by the age of 10 or 12. Their average life expectancy is 18-24 years due to the onset of either cardiac or respiratory failure. One in 3,500 boys are affected by this terrible disease," said Dr Arun Shastry, Chief Scientific Officer at DART.
So far there is no cure for this rare disease and parents rely on physiotherapy and medication. Children with DMD have reduced bone density and an increased risk of developing fractures. According to the National Organisation of Rare Disorders, children with DMD have mild to moderate degrees of non-progressive intellectual impairment and learning disabilities.
DART has developed the first personalised medicine for DMD using antisense oligonucleotides (AO) based exon skipping technology. It acts like a molecular patch so that the DMD gene can produce the dystrophin protein to help protect and maintain the strength of muscles.
It was DART’s founding president Ravedeep Singh Anand’s personal experience that led to this research. “My son Karanveer had trouble climbing stairs, getting up or running. His paediatrician told us he has DMD and would be wheelchair-bound before becoming bedridden. I looked for help across the country but did not find any. I closed my business and immersed myself in DMD, working to understand the pathology of the disorder, the extent of research investment and the mechanisms for optimal care," he said.
Karan’s mother Movin Anand said they collaborated with researchers abroad and with the support from Indian Council for Medical Research (ICMR) in 2018 Karanveer was the first DMD patient in the country to receive the treatment under compassionate grounds. “This treatment was shown to be safe and effective. After we submitted our results from pre-clinical studies which demonstrated safety, the Union health ministry has permitted human clinical trials from 2021," she said.
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.