BENGALURU: A lot goes on behind the doors of India’s only paediatric neuromuscular centre, where the world’s most expensive one-time drug Zolgensma (worth Rs 16 crore) and Spinraza, which costs Rs 78 lakh per vial and is needed for a lifetime, are administered to rare disease patients suffering from Spinal Muscular Atrophy (SMA).
While the company that manufactures Zolgensma offers it for free to some patients in the world on a lottery system, NGO Direct Relief sponsors Spinraza through their compassionate access programme to select patients. The application process is traumatic to go through for a patient’s family, said Dr Ann Agnes Mathew, a neuromuscular specialist and visiting consultant at Bangalore Baptist Hospital. To even know if the patient is eligible for Zolgensma, a test needs to be done to check for any antibodies against the drug, as it is a live virus being injected.
“Only one lab in the world, located in the Netherlands, does this test, costing Rs 72,000. The company that offers Zolgensma on a lottery, pays for this. If the patient has no immunity to the drug, then he or she is gene therapy ready,” Dr Ann said. As both these drugs are not approved in India, the hospital has to get case-wise permission from the government on compassionate grounds, stating that the child will not live without this medicine.
“Both the medicines have to be imported in -60 degrees of dry ice, and kept at 2 to 8 degrees. Zolgensma must be used within 14 days while Spinraza can be kept for longer,” said Dr Madhuri Maganthi, Consultant (Paediatrician & Specialisation in Paediatric Genetics) and HoD at Baptist. Many children die waiting for this medicine. Even after the drugs are administered, the woes of these children do not end.
As their chest muscles are weak, they cannot breathe. Some undergo a tracheostomy and require artificial support with a BiPAP machine to help them breathe. As they are unable to swallow easily, they don’t get adequate nutrition, and food can often go into the lungs, causing pneumonia. Owing to this, a gastrostomy is done to feed directly into the stomach. Zolgensma gene therapy can damage the liver for which steroids have to be given too.
“Two weeks after a baby from Coimbatore got Zolgensma, he stopped breathing. We were able to intervene through surgery and save the child. Hence, children cannot go home immediately after being administered these drugs,” Dr Ann explained. Supportive care is often beyond the means of poor patients, for which fundraising is done, and the hospital too absorbs the cost for deserving patients.