Couple hopes to crowdfund Rs 16 crore to treat 2-year-old son’s genetic disorder

Inspired by a Mumbai couple, a Hyderabad-based couple has begun a painstaking campaign to crowdfund Rs 16 crore for a gene therapy treatment to treat their two-year-old son. The Novartis manufactured Zolgensma gene therapy to treat spinal muscular atrophy (SMA) is the most expensive medicine globally; it costs 2.1 million US dollars, about Rs 15.24 crore in Indian currency. One injection is enough to correct the genetic defect.

Last week Mumbai-based Priyanka and Mihir Kamat managed to gather Rs 14.92 crore through crowdfunding for the couple’s six-month-old daughter Teera, who suffers from Type I Spinal muscular atrophy, requires ventilator at home and a feeding tube. Her parents began a campaign last year and managed to gather Rs 14.92 crore this month. The injection is slated to reach Mumbai next week, and will be immediately administered to Teera in PD Hinduja hospital.

SMA is a genetic disorder in which one defective gene causes muscles weakness and degenerates its normal functions. This rare disorder is found in one of every 10,000 babies with most unable to survive beyond a few years.

Dr Neelu Desai, consultant paediatric neurosurgeon in PD Hinduja where Teera is undergoing treatment, sees four-five such children a year. “Until three-four years ago there was no treatment. We provided supportive care to children. But three treatment options have come up in last three years. One is gene therapy which is considered revolutionary, other two are oral medication called Risdiplam and injection Spinraza. All treatment options are extremely expensive,” Desai said.

The Zolgensma gene therapy is a one-time injection that replaces the defective gene with normal gene and rectifies the disorder. In 2019, US FDA approved this therapy for children aged less than two years. Desai said just one injection is enough to replace the defective gene.

Desai said every year Novartis conducts a lottery and provides the injection free to 100 children. From India 10 children were fortunate to have got it free of cost in this way. Teera was two months old when she was diagnosed with SMA. Four months ago after they could not make it through the lottery, her parents started collecting funds on ImpactGuru portal where over 87,000 people donated money. Currently she suffers from muscle and limb weakness.

“We didn’t believe we could ever gather such huge funds. But after seeing their case, we began a crowdfunding campaign this month,” said Hyderabad-based Yogesh Gupta, whose son Ayaansh, aged two, also suffers from SMA.

Gupta said he noticed his son’s head movement and growth was unusual when he was six months old. He was diagnosed late after he turned one in 2019. “By now his respiratory muscles are weak. His limb movement is affected. Initially doctors told us even if we sell our property we won’t be able to raise such a sum. So they asked us to focus on occupational therapy and physiotherapy,” Gupta said.

Dr Ramesh Solanki, paediatric neurosurgeon in Rainbow Children hospital in Hyderabad, said he sees four-five children a year with SMA and has to advise occupational therapy and physiotherapy as supportive treatment as many cannot afford treatment. Solanki has been actively campaigning for Ayaansh. Till now the family has gathered Rs 2 crore through visits to NGOs, philanthropists, and online platforms.

In Teera’s case, PM Narendra Modi waived off Rs 6.5 crore for GST and import duty to import the medicine from US on request of Maharashtra leader of opposition Devendra Fadnavis. Gupta is hopeful of a similar concession by the government.