Spinraza is the first drug for the treatment of Spinal Muscular Atrophy (SMA), the medical condition I have.
When it was released to the market and approved by the United States Food and Drug Administration (FDA) at the end of 2016, there was much fanfare and parents of SMA kids had hope that their children would be able to lead a better life with the help of the new medication.
Recently, Spinraza was also cleared for use in the European Union.
We haven't got Spinraza here in Singapore yet, but some parents I know are actively lobbying for the Ministry of Health and local hospitals to quickly bring it in.
My parents and I are not part of the movement. And here's why.
The most serious drawback of Spinraza is its prohibitively exorbitant price tag. It is really really really really really expensive. The cost of a single dose can buy three luxury cars with Certificate of Entitlement (COE). It can buy a small flat. It can buy enough chicken rice to feed three people for their entire lives if they eat chicken rice for breakfast, lunch and dinner every day.
One shot of Spinraza is estimated to cost up to $750,000. Three quarters of a million dollars. Oh, by the way, that's in US currency.
Parents in the US have managed to pressure the insurance companies to foot a portion of the bill, but I doubt our insurers in this part of the world will agree to do the same. They can be very annoying.
When I fell ill with swine flu (also known as H1N1) during the 2009 global outbreak, my father tried to claim insurance to cover some of the hospital fees, but they said they won't approve a payout because I have a "pre-existing condition" (referring to SMA). Hello? I got swine flu because I was unlucky! It has nothing to do with SMA! You mean other people without SMA didn't get swine flu? How then did it turn into an epidemic?
Okay, back to the topic. The hefty cost may be less of a problem if not for the fact that Spinraza needs regular booster shots in order to sustain the benefits. It's just like when normal people have a fever and take Panadol. After a while, the effect wears off and you have to pop another pill. Same here.
Imagine that. Every year, hundreds of thousands of dollars essentially disappearing into thin air. If everyone were a Bill Gates or Warren Buffett that wouldn't be an issue. But in reality, how are ordinary people supposed to afford it?
Another problem I have with Spinraza is that it needs to be delivered by injection directly into the spinal cord. That gives me the creeps! No way I'm going to allow anyone to stick foreign objects into my spinal cord!
Spinraza is also an unproven drug. Yes, it went through clinical trials and showed promising results. It significantly reduced impairment and improved motor function in the test subjects without killing any of them. But it hasn't been in general circulation for that long. Who knows? Maybe after a while some heinous side effects will rear up. Or perhaps those undergoing the treatment will develop resistance to the drug and all their SMA symptoms will come rushing back.
Wouldn't that be terrible? To have finally had the chance to experience a relatively normal life, only to have it snatched away and being consigned back to the wretched existence from before? What a massive psychological blow that would be.
I'm not wishing bad things on anybody but I'm just pointing out that negative consequences could still occur. That's the trouble with new treatments like this. Nobody knows what's going to happen.
A few years ago, a random two-bit small-time physician in China claimed to have developed a cure for SMA. The parents of an SMA kid I knew got very excited and shelled out a huge sum of money relocating to China so their child could receive the treatment. Just like Spinraza, it was injected in multiple doses at intervals.
The family eventually moved back to Singapore. Their child wasn't cured. A little bit later, the poor kid died. I don't have any solid scientific evidence that the so-called cure hastened the kid's untimely demise, but my gut tells me it played a role. The kid was actually healthier and stronger than me, but passed away at a much earlier age. Coincidence or something more sinister? Whatever it is, it serves as a cautionary tale against chasing after pipe dreams.
I'm glad my parents don't go haring off at every whiff of a possible hypothetical cure. I've lived all my life not knowing anything different. It doesn't mean I don't mind being disabled. Sure, it sucks and I'd gladly get rid of it if I had a genie in a bottle. But over the years, I've come to accept it as something I can't change. I just have to deal with it and live one day at a time. When it comes to a cure, it's too late for me anyway. No point getting all excited about something which may not work properly, or at worst might even rob me of the most precious gift of all: my life.
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