My story is not unique. Many parents who have children diagnosed with extremely rare diseases have confronted the same challenges I have faced. When your child is diagnosed with a fatal, devastating and progressive disease, and you’re told there is nothing you can do to help them, you have pretty limited options. You can find some level of acceptance and try to build a new life which includes the diagnosis and prognosis, or you can choose to ignore the doctors and try to change the course of the disease. No choice is better or worse than the other, and this is a very personal decision. Both roads may even end up leading you to the same place.
I could not find a way to accept the diagnosis, so I chose to follow the second path. I thought about nothing else. I was consumed with the idea that if I raised enough money for experimental medical interventions, my son would not only live but one day walk, talk and lead a healthy life. Helping Max became my life’s work, my mission, my passion and my way of life. In order to do this, I had to set out on a journey to cure Canavan disease. For me there was no other way.
As my efforts gained momentum and garnered attention from the media, I was urged by many people and organizations to align myself with other diseases in order to help more people and potentially raise even more money. I was urged to stray from my mission of curing one “tiny,” unknown disease and focus more on rare disease, genetic diseases, Jewish diseases, allied diseases, or leukodystrophies.
At that time, there were already many existing umbrella organizations focusing on groups of diseases, and not one of them had ever been able get a single clinical trial for Canavan disease. I felt a laser beam of focus on one tiny disease was my best and fastest option to move medicine ahead more quickly towards something to help my own son.
This is why I am a selfish parent. I would love to save every child living with every similar disease, but realistically, I have been able to make more of a difference for more children by choosing to focus solely on Canavan disease. We have developed a Canavan cocktail of medications and orchestrated two gene therapy trials for Canavan disease. These successes were only possible with an extremely narrow area of focus.
I know I am not alone. There are countless diseases that require the same type of single-minded mission to find a cure. Sometimes there is no one else willing to do this job except for the parents. That’s why I believe sometimes it’s OK to be selfish as a parent.