Parents with kids that have serious diseases like mine have to organize. We have to push for a system that makes more sense. In the case of Reed (my son), he can’t get treatment because he lives in the US and the US FDA didn’t approve a Phase I/II trial for Sanfilippo Syndrome. I am told that this is due to the fact that the FDA asked for extra safety data on animals. My son, who has a disease that is 100% fatal, is being protected against a potentially effective therapy. Meanwhile, the biotech company Shire HGT, simply took the drug trial overseas and, because of how small the trial is, they won’t let people move.
It is difficult for parents that haven’t experienced a similar situation to relate, I know that. The potential therapy for my son’s disease would involve placing a catheter directly into the spine. Through a port just underneath their skin, treated children would have the missing enzyme injected directly into their spinal fluid every other week for the rest of their life.
Let me just stop for a moment and say there was a moment about two months ago when I realized there were no easy path going forward. Whatever happened next, life was going to be challenging and the illusion of a cushy life was gone. Nevertheless, I would absolutely accept the risk of treatment if there is a good chance that Reed could live a near normal life.
I would choose difficulty and risk over a slow degeneration any day. The idea that my boy may some day lose the ability to talk or walk haunts me. Sure, it is a slippery slope and, of course, the FDA serves a useful purpose. They are there to protect us from people who would sell us “snake oil” or – worse – products that are worse than the disease. However, it is just common sense to say that they should adapt their rules to the situation. Don’t apply a cookie cutter approach to approving new treatments. Kids like Reed fall through the cracks.
The system should weigh the risk of the “do nothing” option as it looks to approve trials and new treatments.
