Stealing hope

A.L.S is a death sentence.

Amyotrophic lateral sclerosis is a fatal type of motor neuron disease. It is characterized by progressive degeneration of nerve cells in the spinal cord and brain. It’s often called Lou Gehrig’s disease, after a famous baseball player who died from it[1]Amyotrophic Lateral Sclerosis (ALS).

The muscles don’t get nutrition, and without nutrition, they die. There is no know cure, and the degeneration is a matter of months. The cause is also unkown.

It’s rare, and so often misdiagnosed at first.

Although they know that there is no chance that the trials that they volunteer for will save them, sufferers who sign up are determined that their efforts will save victims in the future. The anecdotes leave no doubt: they are heroes.

They are active on social media, and that heroism has drawn the attention of some big names: Elon Musk, Bill Gates, and Barack Obama. With that attention, money has flooded in to fund research. And this is where it starts to get tricky.

There is a drug, Relyvrio, which initial trials suggested slow the degeneration by five months. But the results are not conclusive, with statisticians and the FDA all saying that a more detailed trial needs to be undertaken.

There is just a little problem. Under extensive pressure from lobby groups, the FDA granted special approval for the drug to be prescribed, and to date, sales have already exceed $400 million. That at $158,000 per prescription.

There is little incentive to conduct a trial for a money-spinner that has a high probability will result in having the drug withdrawn[2]Hope For Sale. Get Your Hope Now..

The real life evidence does not support the original results that the drug extends life by five months, and the side effect is euphemistically termed as gastrointestinal problems. You pretty much have to be close to a loo at all times, otherwise you run the risk of needing a nappy. 

That is not quality of life, and already people who have been prescribed the drug are choosing not to take it[3]When Dying Patients Want Unproven Drugs.

Those heroes need to stand up again, and insist that the drug goes through a proper trial, that proves conclusively, one way or the other wether the drugs works. 

If they don’t, their heroism will be have been in vain.