Only 11 Of 19 Children In India Diagnosed With Cystinosis Are Alive Researchers in IIT Madras have developed a life-saving drug that can keep children suffering from a rare genetic disorder alive. There are only 11 children in the country who suffer from the condition called cystinosis. The drug, if made and marketed in the country , will bring relief to the parents of these children who at present import them from Europe at a huge price as it is not approved in India.

Children with cystinosis suffer from growth retardation, kidney failure, bone deformities like rickets and blindness. In India, only 19 children have so far been diagnosed with the disorder, and nine of them are no more.

Cystinosis Foundation, India has collaborated with IIT-M to make the drug Cysteamine Bitartrate to treat the disorder, which is now in the final stages of development. Professor in the Chemistry department S Sankaraman said they are now in the process of purifying the compound. "We began developing the drug a year back.It will be ready in another one or two months' time," he said. The drug is now being manufactured by Orphan Europe, a firm headquartered in Paris. Parents in India import the drug once in every three months from there.

Nephrologist Dr Rajan Ravichandran said the drug removes the amino acid cysteine that accumulates in cells. The patients have to be given the drug once every six hours. Though it is not a cure, it can contain the disorder. However, the drug is very expensive. "For a three-month course, it costs about `2 lakh. There is also the customs duty , which is 10% to 15%," he said.

According to the Drugs and Cosmetics Rules, 1945, an individual can import lifesaving drugs in small quantities upon obtaining a permit from the Drugs Controller General . "Every time we have to obtain permission. It is a very tedious procedure to import these drugs," said Sujatha Sivarasu, mother of two boys who were diagnosed with the disorder.

Dr Ravichandran said pharmaceutical companies do not manufacture or market the drug in India as it has no commercial value since there are only 11 known consumers. Further, to get the controller's approval to commercialise the drug in India, extensive clinical trials are needed . "But we cannot have a trial as we need large sample size," the Dr Ravichandran said."Fortunately , the Madras high court has given a direction asking the controller to approve the drug without clinical trials as it is a life-saving drug. Also, the patent for the drug made in Europe has expired as it is more than 25 years old," he said.

A pharmaceutical company has volunteered to make and market the drug. "We have to scale it up and formulate it into a capsule or tablet. It would be made available at a nominal price," Dr Ravichandran said.