Lucky gene-therapy break for daily wage earner’s son
Times of India | 6 November 2024
123456 Kolkata: A 16-month-old child with spinal muscular atrophy (SMA) was chosen for gene therapy and was administered the drug at a private hospital off EM Bypass on Monday. The drug used for the therapy, Zolgensma, costs around Rs 17.5 crore but Deen Mohammad, son of a daily wage earner from a village in Mahishadal in East Midnapore, received it for free as part of a global trial.
SMA is a rare disorder that affects an infant's motor neurons and nerve cells that control voluntary muscles. It affects the movements of limbs of those affected. According to experts, it is incurable but can be managed with the help of therapy. After the medicine was administered on him, Deen was put in the isolation ward of the hospital on Tuesday and once he recovers, he will be discharged on Wednesday. The child would now have to follow a rigid physiotherapy regime and come for follow-up check-ups, said doctors, adding that they expected him to take around two years to gain movements in his limbs.
When Deen was six months old, his mother, Dilwara Sultana, a daily wage earner, noticed that he could not move his hands or legs. Her husband abandoned her after the child was found unwell. "I took my son to a local doctor who advised me to take him to a hospital in Kolkata. I could not come immediately as I did not have enough money for his treatment at that time. My brother, a farmer, was of great financial support to me. We took Deen to Peerless Hospital, where doctors diagnosed him with this disorder and said it required an expensive treatment," said Dilwara. "Those at the hospital enlisted my son for a programme, and by lottery, his name was chosen for the free treatment."
Deen would have to visit the hospital for follow-ups, said Sanjukta Dey, who received special training to administer the medicine. Dey said, "The SMA carrier rate is 1 in 35, according to recent data. Its is believed to be the second-most common genetic disorder after Thalassaemia." Ravindra Pai, MD of Peerless Hospital, said, "There needs to be private-public partnerships for early detection and treatment of the disorder, with more funding initiatives under the national policy for rare diseases."
Under this global free trial, Deen was the third and last child in eastern India to be registered for the free gene therapy, the registration for which ended on July 31. Of the two other children, one received treatment at the same private hospital and the other at NRS Hospital. Jasodhara Chaudhuri, assistant professor of neuromedicine at NRS, said, "A US-based pharma company made the trial available for free in 36 countries. The company has sought the Centre's permission to sell the drugs in India. Effort is underway to fund more SMA patients through crowdfunding initiatives. We urge the govt to quickly approve the medication so that children from ordinary families can access it and to focus more on research in gene therapy."