I guess you can say – “we are lucky.’ After 5 days of putting Alexander through extensive testing (a 3 hour MRI, extensive bloodwork, ECG, Pulmonary, 2 days of physical therapy assessment, fecal testing (ek!) and a physical exam), we received official word last night that he was accepted into a clinical trial for a DMD treatment drug. As I am sitting in a hospital room at UCLA with my baby hooked up to an IV – I am pondering whether to feel relieved and hopeful OR fearful and sad. Lucky – to be the chosen few (8 children so far around the world) to receive a treatment drug that possibly could help the disease and make his muscles larger and stronger. Fearful and sad – that he is a guinea pig and will be poked, prodded and examined for over 2 years while receiving a “substance” through an IV once a month that’s benefits are completely unknown. Oh – I forgot to mention that this is a double-blind trial, and he could in essence be receiving saline – or a “Placebo” – for the first 42 weeks. (!) I should also add that he is the FIRST patient so far at UCLA to receive the drug. Another child recently screened at UCLA did not make it in, as well as 2 other boys of parents I know from Jersey and New Hampshire were turned down, due to the quite unreasonable exclusion criteria that the scientists at Pfizer designed. How are these trials designed? by scientists (labcoats) and the FDA (suits), who come up with overly stringent guidelines that can be downright unreasonable, to decide who’s child can receive an experimental treatment drug – that may or may not work. The juxtaposition, is other countries like India, Cuba, Lebanon, etc. that are performing radical treatments like stem cell transplants, without any guidance, rigor and data collecting. Really crazy. Can’t there be a middle ground?
So the question is – do we celebrate? or are we fearful and nervous? Last night it hit me again of the situation we are in with Duchenne. That are only option is to potentially be putting an unknown substance voluntarily into our child through an IV that may help, may do nothing or could possibly have an adverse effect. And we are committing to doing that for TWO YEARS. It’s not easy.
It’s July. You know what other kids are doing right now? they are at camp or at the beach. I will not be bitter. I know that this is the life and journey we are supposed to be on. We will face it – strong and know that this is all in GOD’s hands.