Our Journey Raising Two Children with Special Needs

This blog chronicles our life raising two children, Nicholas 14, diagnosed with Prader-Willi Syndrome and Weston 17, diagnosed with Autism/Asperger's/ADHD. It's the ups, the downs, the joys, the sorrows and most importantly, the beauty of living a life less perfect, a life more meaningful.




Saturday, September 20, 2014

A Very BIG Decision


OK, so remember what I told you about special needs parenting and BIG decisions?

It's been what....maybe 24 hours? And already I am faced with a decision that may alter the course of my son's ongoing battle with the ferocious hunger and obesity symptoms associated with Prader Willi Syndrome.

Yesterday, Nick and I met with Dr. Stafford, a Children's Hospital Endocrinologist and good friend. Yes, "good friend". Nicholas has seen her once, every four months, since the day he was born, which is more than we see some of our own family members. You get to know a lot about a person when you see them this often.

Anyway, she has been asked to participate in a Clinical Trial for a potential new drug designed to target hyperphagia (uncontrollable eating) and obesity, both severe issues that plague individuals diagnosed with Prader Willi Syndrome.

The new drug is named Beloranib from a company named Zafgen. Initial results are promising and have been reported in the New York Times, click here to read.

Dr. Stafford explained that all of her patients diagnosed with PWS would need to be evaluated and questioned regarding their suitability for participation in this study. Based on our response to these questions, and Nick's height/weight profile, there may be a chance that he would qualify.

Say what?

Tears begin to roll uncontrollably down my cheeks.

I am overwhelmed with emotion, knowing that we are getting close to finding a cure for the uncontrollable hunger that plagues my son every single second of every single day.

I try to squelch my overwhelming elation for as every parent of every child diagnosed with special needs will tell you, when you are emotional, it is impossible to think clearly. And thinking clearly about something as important as this......is crucial.

My mind whirled and questions began popping up in my brain. How often would we need to come to Boston? How much blood work would need to be done?  How long is the trial? Is it an injection or a pill? When will this happen? How much school would he miss?

And perhaps most importantly.......what are the risks?

It is a lot to digest.

Participating in something like this will require a lot of our time and effort. Precarious drives into Boston every two weeks, lots of missed school, blood draws.....which, according to Nick, are worse than pulling fingernails. Nurses coming to our house to give injections. Nasty and potentially serious  side effects. And to top it off......there is a chance we could be one of the lucky ones who receive the placebo........!

Is this just more false promises or could we be a part of something very, very big?

I am hopeful, awed, concerned, scared, intrigued....and without a doubt emotionally overwhelmed.

I will need some time to make a very BIG decision.