BENGALURU: Three girls suffering from the rare disease, Spinal Muscular Atrophy, were administered the expensive gene therapy, Zolgensma, for free at Bangalore Baptist Hospital on Thursday. Each vial of the one-time injection costs Rs 16 crore and is capable of changing the genetic code in the body.
In all, the initiative amounts to Rs 48 crore for the three children, and is being given for free by the manufacturing company, through a lottery system (CSR). Three other girls with the same disease are being administered Spinraza, a drug costing Rs 70 lakh each (Rs 2.10 crore for three kids) in the same hospital. This is also being given for free by the Compassionate Access Programme of the NGO Direct Relief. The total cost is Rs 50.1 crore.
Spinal Muscular Atrophy (SMA) is a rare disease affecting one in 6,000 children, caused by the loss of nerve cells, which carry electrical signals from the brain to muscles. The protein needed for the body to carry the electrical signals is coded by a gene which has two copies -- one from the mother and the other from the father. A child develops the disorder only if both copies are faulty. In SMA, the muscles get weak and shrink, the child cannot breathe or swallow, has trouble controlling head movement, walking, and gradually dies before reaching the age of two.
“Two of the children who received Zolgensma are 16 months old, and one is 14 months old. This is a one-time drug that essentially changes the spelling mistake in the gene permanently. The three other girls are being administered Spinraza on a continuous basis since 2019. This medicine encourages the body to produce protein, keep the spinal cord alive and give strength to the body. Signals are carried from the brain, spinal cord to motor nerve cells, which need protein. Spinraza works with the faulty genetic code to make protein,” said Dr Ann Agnes Mathew, head of neuromuscular services at Bangalore Baptist Hospital, who is treating the children.
Of the three children who were administered Zolgensma, two had SMA type 2 and one had SMA type 1.
“While those with type 1 do not survive beyond 2 years, patients with type 2 have a risk of dying but with extra care, can survive for long, though they remain weak. The hospital has sent in three more applications to the company for Zolgensma. Apart from these six girls, two boys and one girl are receiving free gene therapy treatment this month,” Dr Ann said.
The cost for treatment for rare diseases is high as it takes 8-10 years to develop a drug, explained Prasanna Shirol, founder of Bengaluru-based Organization for Rare Diseases India. “Companies invest huge amounts of money on research and development, while not being sure of its success,” Shirol explained.