$2.4m for a chance to live: 3-month-old baby with rare condition requires 'world's most expensive medicine'

It is known as the "world's most expensive medicine", priced at a jaw-dropping $2.4 million per pop. But it's also what three-month-old baby Lucas needs in order to have a chance at life.

Lucas was born with a rare genetic disorder known as Spinal Muscular Atrophy Type 1, reported Shin Min Daily News on Saturday (July 8).

The neuromuscular disorder progressively weakens and damages muscles over time, affecting the brain and spinal cord. Any damage done is irreversible.

In an interview with the Chinese evening daily, Lucas' parents — Andy Wang and Lavin Chong — shared how when Lucas was born, they'd notice that his head tended to hang from one side.

He also wasn't like other infants who enjoyed kicking their legs and could only feed for a short period of time. But being new parents, they didn't think that anything was out of the ordinary.

"Whenever we went to see the doctor for his vaccinations, they'd say that he was still too young," they shared.

When Lucas was admitted to hospital for a fever on June 10 and diagnosed with a Covid-19 infection, his parents once again requested for doctors to do a check.

After a litany of tests, it was confirmed that Lucas had spinal muscular atrophy.

"We were devastated at the time. The only good thing was that we'd discovered it early," said Wang. A drug known as Zolgensma is able to cure the condition completely, Wang added, but it costs an eye-watering $2.4 million.

Not only is it an astronomical sum, but "it has to be paid upfront", said Wang. "Even if it can be paid in installments, we can't afford it with both our salaries combined, even if we were to go without food."

The one-time-only gene therapy treatment was approved by the US Food and Drug Administration (FDA) in 2019. According to a report by the FDA, spinal muscular atrophy is the leading genetic cause of infant mortality. 

The drug targets the root cause of the disease and is effective upon intravenous injection, Shin Min Daily News reported. Zolgensma was registered locally in April this year, and according to Wang, two children have previously received the same treatment and are alive and well.

He pointed out that their doctor has shared that the earlier Lucas can receive the treatment, the better.

A crowdfunding campaign has been set up for Lucas at Ray of Hope on July 7. As of writing, the fund has received over $260,000 — a fraction of the targeted $2,409,000 that the family hope to raise.

On the page, it is noted that Zolgensma is known to be the "world's most expensive drug", but its treatment is not subsidised by the government nor covered by the Rare Disease Fund in Singapore.

Doctors have currently prescribed Lucas a drug which costs $9,000 as "temporary medication" but its efficacy lasts "only 64 days", according to the description.

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His parents also indicated that without the drug Zolgensma, Lucas might not live to past his second birthday. 

On receiving news of his diagnosis, they wrote: "By 26 June 2023, the results confirmed our greatest fear. Lucas was not only born with Spinal Muscular Atrophy (SMA), but was a Type 1-SMA in its most severe and cruel form, with a life expectancy of only two years.

"SMA will relentlessly ravage his tiny muscles, denying him the simple pleasures we often take for granted - sitting, crawling, standing, walking, swallowing, and even breathing.

"Our hearts broke. That's the reason why Lucas' legs were not kicking. And that's why his head hangs to the side when he is tired."

They added that Lucas now depends on a nose mask connected to a ventilator as well as a feeding tube in order to survive.

"As parents, it pains us to see him like this. But in our hearts, hope persists that one day, he will run and race with other little boys at the playground," they wrote.

ALSO READ: Woman finds out son has rare genetic disorder after getting pregnant though IVF

candicecai@asiaone.com

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