It is a genuine dilemma in this age of new-fangled “molecular” drugs, that some seem to work very well during their first human trials, but we have to wait years to get them to the public.
Of course there is massive cost to consider in some cases, but I don’t know about eteplirsen. Public health advocates can have a terrible time deciding if a few people can have an expensive drug vs. a lot of people getting a cheap one. For instance, how many doses of polio vaccine would you trade for one of eteplirsen? These are the realities of government budgets. I wouldn’t like to do it, would you?
Originally posted on Health & Family:
Austin and Max Leclaire are brothers. Austin is older, Max is younger. Like most siblings, they have many things in common and just as many that set them apart. For now, though, their strongest bond is over something they share — Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children. Duchenne’s hallmark is increasing muscle weakness that eventually makes it hard to breathe and confines kids to wheelchairs, which is where Austin finds himself these days. Now 14, he lost the ability to walk four years ago, a development that helps explain why the disease looks so different in the two brothers today.
Because Max, 11, was still able to put one foot in front of another in the summer of 2011, he was eligible to enroll in a drug trial for Eteplirsen, which is designed for children like the Leclaire brothers who have trouble producing a protein called dystrophin. It’s likely that you’ve never heard of dystrophin, but it’s responsible for reinforcing and strengthening muscle fibers to gird them against wear and tear. Without it, normal activity strains the muscles, including the heart and lungs. Without it, kids with DMD die.
Eteplirsen helps affected children make small amounts of the protein, which moderates the breakdown of muscles so when the fibers contract, they’re not as damaged or inflamed. Eteplirsen doesn’t restore muscle so it can’t help a child like Austin walk again, but it does seem to slow, even stop, the progression of disease. That’s important since most kids with DMD don’t make it past their 25th birthday. It could be the reason that Max is still walking and Austin is not.