Most parents would do anything to keep their children safe. But what can parents do when their child is sick with a deadly disease? And to what length will they go?
As I reflect on the past 5 & 1/2 years of Jack’s life I could write a very long list of sacrifices I made. And I could also write a list of all the things I have done to get to where we are now. But this blog is not about whoa is me or please feel sorry for me. For those who know me closely, I have an amazing team who provide support on those days :-). And thank you.
Back to the matter at hand, how far would I go to save Jack?
Personally I find myself extremely willing to do whatever it takes. In the world of rare disease the “whatever it takes” usually means fighting with physicians, pharmaceutical companies, investigational reviews boards, schools, insurance companies, the FDA and TIME. Usually, or realistically all of the above. And sometimes it means moving your family cross the states which we recently did.
So here’s my current situation. I am aware of a drug, lets call it Elmiron, well because that’s its name. Elmiron was FDA approved in 1995 for patients suffering from intestinal cystitis, a bladder condition. Then later on a scientist decides to study how the drug might effect forms of human tissue inflammation, specifically for the inflammation that occurs in MPS patients. And through study and observation there are some profound effects to the MPS rat models used, primarily improvement to the airway and bones, something ERT hasn’t shown to help. Even reversing damage!
So a drug exists, it has significant potential, but it also sits in the hands of a large pharmaceutical company whose not willing to support a human MPS trial. For many reasons, but to make it simple $$$. Do I accept NO for an answer? Do I curse and be mad and move on?
I have to share a personal and funny story. When Jack was diagnosed and I sat in darkness wondering how I would ever function or even survive this devastating news. After days of being overwhelmed, shocked, saddened beyond belief, shattered and such I did experience “that moment” that stuck. The day I was able to get out of bed, I was able to get dressed and I stepped outside my front door for the first time since D Day (diagnosis day) and the earth grounded me. And as I connected to the earth something happened that day, something profound…I became a fighter, a mother bear, a dragon mom, and Wonder Woman. I wanted to take on the world, well maybe not the world, but I was determined to get Jack the help he needed at any cost. And soon after the moment I sat with a colleague of mine who held my hand witnessing this change, presented an important gift in a box- Wonder Woman underoos. I giggled and said to her,”yeah I wish I had her strength”. And soon after I received from my best friend a Wonder Woman tee shirt she stumbled upon in a Cali store, I laughed again. And yet another tee came soon. My new identity evolved, with WW wardrobe to assist. Did you know that Wonder Woman was created by a psychologist? Very fitting! Her abilities – endurance, agility, empathy, a healer and mostly a fighter!
Basically, Wonder Woman is who I needed to be to live in this God chosen life. It’s who I had to be to be able to walk out that door that day. It’s how I became a fighter for my son.
So, with joined forces (Andrew, Ellen, Deb, & Melissa) we hit the battlefield fully armed, armed with children we love and other children suffering from this horrific disease. We are and we will take on a large pharmaceutical company. We will not back down. We will not accept less then best. We will use plan A, plan B, C, D…. We will not sleep and work around the clock. We will forget about ourselves and make this a priority.
I ask the MPS community, family, friends, neighbors, and even strangers please join the crusade to help the children who don’t have the voice and powers to fight. And help the families that are exhausted from caring for their children -constantly advocating for needs, sacrificing their own needs, searching for answers, hoping for the best and praying for yet another day. Lets convince Johnson & Johnson to help the MPS community. And in the end lets all be superheroes! I will even send JnJ some underoos for those extremely hard days.
Please take a moment and visit http://www.shameonjnj.com and please click on Leave JnJ message. Let them know how you feel.
And if you would like to do more please contact me today at firstname.lastname@example.org.