Denying Treatment

Denying Treatment

By Michael Leshinski

The NY Times reports on Jan. 17^th about a dying, teenage boy who is being denied treatment for a serious illness and a lawsuit with the company who manufactures the drug that he needs to survive. The disease is Duchenne muscular dystrophy and the afflicted is merely 16 years old. The National Institutes of Health describe Duchenne muscular dystrophy as a “rapidly-worsening form of muscular dystrophy” that usually affects males. The disease is inherited and relatively rare. According to the family, a bio-tech company called PTC Therapeutics initially made a commitment to treat his rare disease, but had recently denied ever making these statements.

The mother of the sick child had previously been an advocate for this same bio-tech company, but has presumably stopped since suit had been filed. Representatives from PTC Therapeutics have strongly stated that “We haven’t promised the drug to anyone.” Imagine that you had a horrific but curable disease, and the treatment was kept just out of your reach. How disheartening that must be. In regards to the drug treatment, the family’s major setback is the fact that the wheelchair bound teen no longer qualifies to be included in the drug’s clinical trial, because he does not have the use of his legs. You can’t get treatment for the disease, because you can’t walk and you can’t walk because you have the disease. What can a person do?

The next logical step is to apply for a compassionate use waiver, since the drug is experimental and the company would not be held responsible for any unfortunate consequences. The company, however, is reluctant to give the drug to anyone. Also, even if they did, is it fair to give the drug to just one person?