Her son, Affan Farooquie, suffers from spinal muscular atrophy (SMA), a crippling neuromuscular disease that affects one in 8,000 children.
Over the past four years, the hitherto incurable disease has had three distinct options. While the cost of Farooquie (Spinraza) spinal injection treatment will become Rs 3.2 crore from the second year, a single gene therapy shot (Zolgensma) costs Rs 16 crore. A daily syrup (Evrysdi) has been available in the country since July at a price of Rs 72 lakh the first year and Rs 56 lakh thereafter. In contrast, a lung or liver transplant would cost between Rs 25 lakh and Rs 30 lakh or advanced cancer immunotherapy around Rs 2 lakh per month.
Gufran, whose husband works in Egypt, says she cannot finance her son’s treatment. This was made possible through a compassionate use program started at Nair Hospital run by BMC, Mumbai Central, with manufacturer Biogen. “My youngest son, who is 3, has been taking the syrup daily for 10 months as part of Roche’s compassionate use program,” she said.
While the Indian government recently announced that it will help raise funds for the treatment of certain rare diseases through crowdfunding on its portal, parents want a more sustainable solution.
“When it comes to SMA, we are all BPL families,” said Alpana Sharma, resident of Kharghar, who founded the Cure SMA Foundation in 2014, and mother of 9-year-old Aarav with SMA. She wants the government, whether in the center or in the state, to start helping soon. “We have lost 48 children in the past six months alone,” said Sharma, who has around 650 children, including 70 from Maharashtra, on the NGO registry.
Pharmaceutical companies have been running compassionate use programs in the country for about a year now. Professor Dr Shefali Gulati, who heads AIIMS Neuromuscular Disorders Clinic in Delhi, was the first in the country to use the drugs and says the children have improved. She used Spinraza for 14 children, Zolgensma for six and Evrysdi for seven children.
“Although the children cannot be expected to be cured and start running, it can be said that most of the children have had significant improvement in some motor functions,” she said. A 3 year old, for example, who could not sit up on his own can now hold his head and sit for a brief moment. “The improvement in this child occurred within eight months of taking the daily medicine,” added Dr Gulati.
At Hinduja Hospital in Mumbai, pediatric neurologist Dr Neelu Desai obtained approval for daily compassionate use therapy for three children, including Gufran’s youngest son. The parents of one of his SMA patients, Teera Kamat, were the first to raise Rs 16 crore for gene therapy.
“Children show subtle changes. One of the children who falls while sitting down may be able to sit up for a while, ”said Dr Desai. Change is slow. “These are kids who have never walked or sat before. With the drugs they are better, but the changes could take months or even years, ”she said.
She expects the cost of treatment to decrease over time, but parents are concerned. Zeba Gufran said: “The drugs are so expensive that we cannot pay out of pocket and have been at the mercy of the pharmaceutical companies. So either you wait for your child to get help or you die. ”
The NGO Cure SMA Foundation has proactively met with health ministers and bureaucrats to design a sustainable program. Its representatives met with Maharashtra’s Health Secretary P Vyas on Friday, who said the government did not have the funds to support SMA programs.
“The point is that such a program is not sustainable without government funding,” said founder Sharma. Even if the government were to hold talks with the pharmaceutical companies, it would help the costing mechanism. “The government has several unclaimed accounts or cash rich programs such as ESIS. Even if interest from those funds were diverted to SMA processing, it would help a lot, ”Sharma said.
The biggest problem, according to parents and doctors, is the lack of awareness of rare diseases. By the time the parents contact the doctors, there has already been a delay; in SMA, gene therapy cannot be offered to children over two years of age. “In addition, parents must believe that these drugs offer a magic cure, children will need supportive therapies such as physiotherapy for a long time,” said Dr Gulati of AIIMS.