Child with killer disease gets new lease of life after USD 2.1 mn treatment at Bengaluru hospital

Fourteen-month old Fatima faceda bleak future afflicted with a killer muscular disorder, buta Rs 16 crore 'revolutionary' gene therapy she underwent at acity hospital after winning a 'lottery' has given her a newlease of life.

Fatima, daughter of Mohammed Basil and Khadija fromBhatkal town in the coastal Uttara Kannada district inKarnataka, is recovering after she was given 'Zolgensma', thegene therapy at Bangalore Baptist Hospital late last month.

She emerged ''a lucky winner of a lottery'' through acompassionate access programme by drug major Novartis thathelped her get the costly treatment, affordable only bymulti-millionaires, the hospital said.

''The cost of this medicine is about 2.1 million USdollars, which is roughly about Rs. 16 crore,'' hospitalDirector (CEO) Naveen Thomas said.

''There is gradual improvement. She is now able to moveher leg. It will take time to become like a normal child,'' herfather Basil told PTI.

The toddler was diagnosed with Spinal Muscular Atrophy orSMA, a disease caused by loss of nerve cells, which carryelectrical signals from the brain to the muscles.

The protein needed for this signaling is coded by a genefor which everyone has two copies --- one from the mother andthe other from the father, according to Thomas.

He said a child develops this disorder only if both thecopies were faulty and without treatment, this disease wasultimately fatal.

But the problem is that the treatment is out of reach ofmost people.

''Only multi-millionaires can afford it! Current treatmentoptions range from medicines, which increase these proteins toreplacing the faulty gene. Zolgensma, a gene therapy is arevolutionary treatment, which aims at curing the disease byreplacing the faulty gene'', he said.

''For the first time in Karnataka, Zolgensma was given atBangalore Baptist hospital to a child who was the lucky winnerof a lottery through a compassionate access programme byNovartis'', Thomas said.

Incidentally, the couple had earlier lost a child, whowas also suffering from SMA.

''On the 21st day of the 21st year of the 21st century, thebaby was given the injection, which is a one-shot cure forthis rare disease, said Dr Ann Agnes Mathew, ConsultantPaediatric Neurologist and Neuromuscular Specialist.

At present there were about 200 children gettingtreatment in the Baptist Hospital which is specialised ingenetic diseases, more specifically SMA and Duchenne musculardystrophy (DMD), said the doctor.

She added that previous year alone, 38 children who weregetting treatment in the hospital breathed their last in theabsence of this expensive treatment.

In Fatima's case, Thomas said: It is a dream come truefor doctors in this field. We hope more children receive thistreatment and many such treatments will become affordable inthe future.'' PTI GMS RSVS VS