₹17-Crore For a Hope - Fighting Spinal Muscular Atrophy (SMA) Type 1

Imagine a child born in a family with a rare genetic disease that could showly take away the child's ability to move, breathe or even cry and whose treatment would cost more than 17 crore for a single dose. For a parent it might be a mixture of fear, helplessness and hope as well. The disease is Spinal Muscular Atrophy (SMA), a genetic disorder. It is estimated that about 1 in 10,000 people are affected by this disease globally. When it comes to India, we don't have a proper number due to limited reporting but experts suspect that hundreds of cases are occurring. So let's examine the disease SMA and see what challenges it brings to the affected one.

Spinal Muscular Atrophy (SMA) is a genetic disease also known as infantile onset becasue it appears to an infant within the first six months of life. When a child is affected with SMA he/ she show low muscle tone, difficulty breathing and swallowing and when they try to cry, their cry is weak.

In this disease, 'motor neurons' which are essential for muscle control get affected. Most of the time the infant dies even before their 2nd birthday. Mostly the cause of death is respiratory failure.

Zolgensma & Zolgensma are both medical treatments for Spinal Muscular Atrophy (SMA). But both of these medicines are too expensive. Zolgensma costs about RS 16-17 crore for a single dose in India which is around 2.1 million USD. Spinraza is approximately RS 87 lakh just for one injection and that often does not cure the disease and requires further doses making one expend their life saving in crores.

There is a cheaper oral drug, Risdiplam, a bottle of which can cost around RS 6.2 lakh depending on age or weight as treatment is ongoing.

Now there are a few regulatory guidelines applied here in India, though globally all these three drugs (Zolgensma, Spinraza, Risdiplam) are approved but DCGI only officially allows use of Risdiplam in India. If one wants Zolgensma and Spinraza they have to apply through humanitarian access programs as a compassionate means and it will be imported case by case with regulatory permission.

• Asmika Das: a girl from Ranaghat, West Bengal, became the first person in eastern India for whom the SMA type 1 drug, Zolgensma, came from California. All these happened due to crowdfunding. Many celebrities, including Rupam Islam, Subhamita Banerjee and Rupsha Dasgupta, participate in the donation campaign. Even the drug manufacturer agreed to lower the price out of compassion from RS 16 crore to RS 9 crore.

• Nirvaan Menon: a 15-month-old boy from Kerala was diagnosed with SMA Type 2 in 2023. His parents opened an account on crowdfunding platforms and successfully raised about RS 5.42 crore for Zolgensma. Then, miracles happened; a donor anonymously donated about 11 crore. His father posted on Facebook, "It is heartwarming to know that there are still angels in the world."

• Kanav Jangra: an 18-month-old boy diagnosed with SMA Type 1 from Delhi in 2023. Central government, Delhi government and Bollywood celebrities came forward to back the cause, such as Sonu Sood, Rajpal Yadav, Kapil Sharma, Vidya Balan and Shakti Kapoor utilised their social media to support the cause. Even the manufacturer of Zolgensma agreed to reduce the price in a compassion gesture to RS 10.5 crore.

Though there are NGOs, hospitals and advocacy groups that are trying their best to help more and more people but in the arena of awareness there requited still a lot of effort. If you come across or know someone who is facing the trails of SMA, know that all they require is your compassion. Support them with any potential you have that may be donation or guidance. As several gene therapies and their clinical trials are underway, so the fight against SMA is entering a new era. Maybe days are arriving soon, when one could receive cheap SMA treatment providing every affected child a chance to fight.