To Boldly Go Where No Child Has Gone Before…

by David Bernstein

Friends of Zachary,

Today is a day to remember because Zachary is the first pediatric patient to ever receive under compassionate use, Novartis’s BKM120 drug. This is the 3rd drug in combination that was identified by Dr. Oren Becher that will hopefully target the remaining strand in Zachary’s tumor. We have suspended his 2 current drugs for a week and will reintroduce them back into his therapy soon. A bit of scientific background…

PI3K inhibitor BKM120 An orally bioavailable specific oral inhibitor of the pan-class I phosphatidylinositol 3-kinase (PI3K) family of lipid kinases with potential antineoplastic activity. PI3K inhibitor BKM120 specifically inhibits class I PIK3 in the PI3K/AKT kinase (or protein kinase B) signaling pathway in an ATP-competitive manner, thereby inhibiting the production of the secondary messenger phosphatidylinositol-3,4,5-trisphosphate and activation of the PI3K signaling pathway. This may result in inhibition of tumor cell growth and survival in susceptible tumor cell populations. Activation of the PI3K signaling pathway is frequently associated with tumorigenesis. Dysregulated PI3K signaling may contribute to tumor resistance to a variety of antineoplastic agents… HUHHHHHHHH

Exactly…Your guess is as good as mine. In its’ simplest terms…This is the 3rd drug in a series of targeted drugs that has been approved by the FDA for Zachary’s personal use. I wanted to share an open letter to all the researchers, lawyers, assistants, medical providers & FDA Officials who all in a collaborative effort reached beyond the normal protocols to push the approval of this drug in 6 days vs 6 weeks. Here’s the note…
Team Zachary,
I recently had a conversation with my friend, who’s daughter was diagnosed at the same exact time and is suffering from the same disease as Zachary. As my friends daughter continues to fight the fight, I pray what we have all accomplished with a successful biopsy, targeted treatments and now compassionate use of BKM120 will give my son the life and hope for a brighter future. I honor and praise everyone listed on this note for their countless hours of amazingly hard work and tremendous commitment to go above and beyond traditional protocols and organization procedures. 
You have given my family renewed hope when at times it seems to have dissipated. More importantly, you have moved the practice of medicine way beyond the standard with constant collaboration, realt-time discussions and complete transparency. Whatever the future holds for my son…I am proud to say my family has worked alongside the most amazing individuals. I assume you all entered medicine to make a difference..I hope this past week of collaboration, is confirmation of your commitment to your calling. I personally want to acknowledge Dr. Sharon Gardner, Dr. Oren Becher and Dr. Mark Souweidane. They have been by our sides 24/7.
On behalf of my entire family, we thank you for your courage to go further than ever before and continued commitment to my family and my son Zachary. 
Thank you from the bottom of our hearts.
Warmest Regards,
The Bernstein and Korfman Family

I share this note at a point in time where I think all of our hard work has provided more options and opportunities for all pediatric patients and families who are struggling with similar diseases and are uncertain about the direction in which to navigate treatment. There is new hope that the medical community is moving faster and further than ever before. I take great pride in the work we all have done to get to this place in time. Evidence of our progress is measured by the various media outlets that have contact us to hear about Zachary’s story and progress of moving medicine forward. I was also asked to be on a Children’s Brain Tumor Project committee, supported by New York Presbyterian Hospital / Weill Cornell Medical Center. The purpose of this committee seeks to share vision, commitment and willingness to change the playing field of medicine for pediatrics brain tumor patients. The true measure of success is the way Zachary continues to improve each and every day

My family and I  take great pride in what we have accomplished and eagerly anticipate where we are going. It is my families purpose to maintain our vision for Zachary’s well-being and continue to “To Boldly Go Where No Child Has Gone Before…”

Our Sincerest Appreciation,

Deena, David, Matthew, Zachary & Molly

“It Takes A Village”