Mumbai: Lack of government intervention and apprehension of starting clinical trials in India is probably delaying rare treatment options available for children suffering from genetic obesity.
In March, two-and-a-half-year morbidly obese Srijit Hingankar died at a Pune hospital due to chest infection triggered by his excess weight. Two months later, his doctor received an approval from a German hospital about his enrolment for a drug trial for leptin hormone deficiency, which was diagnosed as the reason for his excess weight. At the time of his death, Srijit weighed 31.5 kg, equivalent to the average weight of a 10-year-old.
“I plan to utilise the approval to enrol another baby so that someone else doesn’t meet Srijit’s fate,” said bariatric surgeon Dr. Shashank Shah, who practises in Mumbai and Pune.
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Srijit, a resident of Akola, was referred to Dr. Shah when he was three months old and weighed 11 kg. Examinations carried out at the National Institute for Research in Reproductive Health in Mumbai revealed that he had leptin hormone deficiency. Known as satiety hormone, leptin helps regulate hunger. But the leptin replacement drug made by Aegerion Pharmaceuticals is under clinical trial and is available only in the U.S., U.K. and Germany.
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“We received a green signal from the U.K. centre after which we made a passport for the baby and the parents. But at the last moment, the pharma company refused permission to give the drug. After that, we began correspondence with the Germany centre, which finally sent an approval on May 9. But by then Srijit was gone,” said Dr. Shah, adding that it is time that the government stepped in. “We have several children with genetic obesity. If our government tries, we can have these rare drug trials here as well.”
Srijit’s distraught parents haven’t been able to get over his loss. “He was the most loved baby in our locality. Every child from the neighbourhood only wanted to play with him,” said his father Amol, who works as a helper in a private firm in Akola. “His mother has not eaten properly since he has gone.” Srijit’s elder brother does not suffer from such deficiency. His father said his diet is as much as a full grown adult — six chapatis, vegetable, daal and rice — and he still cries with hunger throughout the day.
Leptin receptor
Bariatric surgeon Dr. Mufazzal Lakdawala, known for operating on the world’s heaviest woman, is also in the process of getting approvals to enrol seven morbidly obese children, including three siblings from Gujarat, for a clinical trial on a drug to tackle a mutation of the LEPR gene, also known as the leptin receptor.
While leptin deficiency means shortfall of the hormone, leptin receptor deficiency indicates that the patient has the hormone but there is lack of signalling to the brain. The drug for this particular condition is made by Rhythm Pharmaceuticals and so far only three patients are said to be under trial in the U.K. and the U.S. “Childhood obesity is a growing problem. We definitely don’t want our kids go down in this way and therefore our government has to intervene,” said Dr. Lakdawala, adding that corporates should offer help through CSR projects.
Dr. Lakdawala said he has sent the samples of his patients to the U.K. for further tests and is coordinating for the trial. “It takes time because the pharma companies obviously want to ensure a watertight trial.”