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.




Friday, December 4, 2015

PWS and Russian Roulette



If you are a long time reader of our blog, you may recall last year when Nicholas was invited to participate in a new drug trial for a medication called beloranib. The drug and clinical trial was offered by a Boston-based company named Zafgen.

The medication, originally developed to treat cancer, showed more promise in the area of fat reduction and was promoted instead as a treatment for obesity in individuals diagnosed with Prader Willi Syndrome.

The drug provided much hope to families who struggle raising children who cannot control their appetites and thus are at risk of eating themselves to death.

Since we live locally here in Boston, Nick's endocrinologist explained that because Nicholas was diagnosed with PWS and struggled with weight issues, he qualified for the study. She asked if we wanted to participate.

I felt a mixture of fear and hope as I weighed the pros and cons of this difficult decision.

But like the dangerous game of Russian roulette, this reward carried some very big risks.

I wrote a post about the anxiety I felt having to make this kind of difficult decision. You may read about it here.

After much angst, I decided that Nicholas would not participate.

Today, I learned that a patient diagnosed with PWS who was enrolled in this drug trial has died of a pulmonary embolism.

In an effort to learn more, I performed some research and discovered that this is the second PWS participant in just two months who participated in this study and died from a blood clot.

Further still, there has been a law suit filed in Boston against this company.

If you're thinking the suit was filed for wrongful death....you would be wrong!

The suit, has been filed by investors of the company who claim they were swindled out of their money by the company and CEO. The Federal Security Exchange is investigating their claims that the company under-reported the number of adverse events (including clotting) to its investors.

In the world of civil suit is the loss of money more important than the loss of human life?

I would like to extend my deepest condolences to both of these families.

Our hearts, thoughts and prayers are with you. We share in your sadness.

I feel a mixture of emotion

A deep sadness and uncontrollable anger mixed with an overwhelming sense of relief and guilt.

Like a bullet to the brain, I am reminded once again,

like all families of children diagnosed with PWS,

no matter what we do,

or what decision we make,

we live our lives in fear.

We fear the Grim Reaper's icy breath upon our necks,

as he waits anxiously to rob us of our children.