One of several letters written by LSD-affected children to the PM
What would you do if your child were struck by a strange illness and began to wilt before your eyes. An illness that few doctors understood, even fewer could treat, that most likely did not have a cure, and even if it did, you couldn’t afford it?
Faced by a parent’s worse nightmare come true would you cry your heart out? Curse your fate? Pray, pray, pray?
Prasanna Shirol and 14 other parents started what’s becoming a movement. Continue reading
The last couple of days has seen some speculation about sanofi aventis’ play for Genzyme. Big Pharma’s acquisitiveness for biotech’s not new – this is just the latest example.
In the last decade (maybe a little more), biotech seems to have been having better luck than Big Pharma putting new drugs into the pipeline especially for really difficult-to-treat diseases such as cancer as also for really rare diseases that were not worth the while of large pharmacos. In the case of Genzyme, I have quite literally come face to face with what that means.
I have met children with these rare diseases in India and their families and I know what it means when they have a cure and when they don’t. Here are two stories :
-Suyog is in his late teens and looking at him you wouldn’t say he was sick. He’s got Gaucher’s Disease which means his body lacks a vital enzyme that breaks down fat. This fat deposits on vital organs and muscles and leads to their steady deterioration until one day it proves fatal. When he was less than five (if I recall the age correctly) his spleen had ballooned until it pushed up his rib cage. It had to be surgically removed. The only solution was enzyme replacement therapy and the only company (then) in the world that made it was Genzyme. Luckily it had a compassionate access programme and he eventually got it. He’s been on free Cerezyme for years. His father couldn’t have afforded it – it costs lakhs and is taken for life. His father is now trying to raise awareness about rare diseases along with parents of other children with such diseases. Gaucher’s is just one type of a group of enzyme disorders known as LSD.
-Then there’s Rahul Chand, the son of a coconut vendor. Chand is about eight years old but apart from his face the rest of his body suggests he stopped growing at three. When he sits on a chair, his feet stick out before him clear off the floor. Even in repose he seems to be struggling to breathe, and when he speaks his name he’s barely audible. Chand has lost his ability to walk – the result of a weakness in the muscles caused by the deposition of unmetabolised fat. His features are distinctive – a prominent forehead, a flattish nose. Chand has MPS 2, a type of LSD, that a good number of other children in this group appear to be suffering from.By the time Chand’s parents found out he had MPS 2 and that it was a genetic condition, they had already produced Rohit. He also has MPS 2. I met Rahul and his father on International Rare Diseases Day in February this year. MPS 2 has a treatment – it’s made by Shire but they don’t make it available in India from what I was given to understand by a doctor at a reputed govt hospital in the capital.
There are plenty more such stories in India – for even a “rare” disease in a populous country such as ours is a helluva lot of people. The stories that I heard from patients and their families suggest an acute lack of knowledge from doctors about these diseases, the absence of diagnostic facilities to correctly diagnose the problem, and the waste of precious time while your child wastes away before your eyes.
Of course, the price of these therapies is mind-boggling – lakhs of rupees for life. What’s heartening is that at least some of these kids are getting it free. Hopefully, even under a new owner Genzyme will continue to honour its commitments.