Child with killer disease gets new lease on life
14-month-old Fatima faced a bleak future afflicted with a killer muscular disorder, but a Rs. 16 crore "revolutionary" gene therapy she underwent after winning a lottery has given her a new lease on life. Fatima, daughter of Mohammed Basil and Khadija from Bhatkal, coastal Uttara Kannada, Karnataka, is recovering after she was given "Zolgensma," the gene therapy at Bangalore Baptist Hospital late January.
A lottery helped Fatima's parents get her the costly treatment
She emerged "a lucky winner of a lottery" through a compassionate access program by drug major Novartis that helped her get the costly treatment, affordable only by multi-millionaires, the hospital said. "The cost of this medicine is about $2.1 million, which is roughly about Rs. 16 crore," hospital Director (CEO) Naveen Thomas said.
The couple lost their earlier child to the same disease
"There is gradual improvement. She is now able to move her leg. It will take time to become like a normal child," her father Basil told PTI. The toddler was diagnosed with Spinal Muscular Atrophy or SMA, a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. Incidentally, the couple lost their earlier child to SMA.
What is SMA, the fatal disease Fatima was diagnosed with?
"The protein needed for this signaling is coded by a gene for which everyone has two copies, one from the mother and the other from the father," informed Thomas. A child develops this disorder only if both the copies are faulty, and without treatment, this disease ultimately proves fatal. But the problem is that the treatment is out of reach of most people.
Zolgensma gene therapy replaces the faulty gene
"Only multi-millionaires can afford it! Current treatment options range from medicines, which increase these proteins, to replacing the faulty gene. Zolgensma gene therapy is a revolutionary treatment, which aims at curing the disease by replacing the faulty gene," he said. This was the first instance in Karnataka where Zolgensma was given at Bangalore Baptist hospital to a child who was a lucky lottery winner.
Baptist Hospital is treating about 200 children for genetic diseases
"On the 21st day of the 21st year of the 21st century, the baby was given the injection, which is a one-shot cure for this rare disease," said Dr. Ann Agnes Mathew, Consultant Paediatric Neurologist, and Neuromuscular Specialist. "There are about 200 children getting treatment in the Baptist Hospital which specializes in treating genetic diseases, more specifically SMA and Duchenne Muscular Dystrophy," she added.
Last year, 38 children at the hospital died of SMA
She added that in the previous year alone, 38 children who were getting treatment in the hospital breathed their last in the absence of this expensive treatment. In Fatima's case, Thomas said, "It is a dream come true for doctors in this field. We hope more children receive this treatment and many such treatments will become affordable in the future."
