In Your Face! ...It's Not Just Pie In The Sky
When speaking with people about cancer research over the years I often heard that a cure, or even advanced targeted treatments (especially for lung cancer) are just "pie in the sky" thinking. Of course, in my response now, I point to precision medicine approaches to treatment now available due to advances in genomic understanding about specific mutations in cancer. I heard the exact same thing a couple years ago when speaking with a parent of a child with Fragile X syndrome who asked me if using CRISPR to treat Fragile X was just "pie in the sky" - as in, is there a chance it could work, or is it just impossible? As an optimistic person, my answer was that given how rapidly technologies such as gene therapy and next generation sequencing are evolving, many things that seemed impossible are definitely not pie in the sky, even for complex disorders like Fragile X or diseases such as lung cancer.
The context of that conversation was an article she had read about how for the first time, researchers at the Whitehead Institute at MIT had restored activity to the Fragile X syndrome gene in affected neurons using a modified CRISPR/Cas9 system that removes the methylation, suggesting that this method may be useful for targeting brain disorders like Fragile X. Was this just pie in the sky? Well, I decided to go directly to the source - I would go to MIT and meet with the researchers, Rudolf Jaenisch and Shawn Liu and ask them both that exact question - "Is CRISPR for Fragile X just pie in the sky?" And the answer from both of them was an emphatic no - it is definitely not pie in the sky. I went back to that parent and shared my conversation with her, with the caveat that I am not a scientist but I do know some really smart ones! LOL. Dr Jaenisch added that even though there are many serious challenges with using gene therapy in neurodevelopmental disorders, he thought Fragile X was actually one of the best test cases for the use of CRISPR.
Fast forward to 2020 and in a completely different context, I was talking to my friend Nicole Eisdorfer, who also has a son with Fragile X and she said she had this idea. She said she wanted to start an awareness campaign for Fragile X called the Pie In The Sky Challenge. Of course my ears perked up! She went on to describe how she is an optimistic person and believes that with research and the relentless pursuit of a cure for Fragile X it may not be pie in the sky to think her son could receive a breakthrough treatment someday. Unbeknownst to her at the time, she was totally speaking my language! She described her plan to get a viral campaign she would start by challenging her family and friends to do a video tossing a pie in their face and put it on social media... and challenge 3 other people to do the same, or make a donation to support Fragile X research (or both). I told her it was brilliant and I would do anything I could to help her because it really is the perfect message for someone like me, who is a relentless research evangelist. As my friend Bob Coughlin, CEO of Massachusetts Biotechnology Council (MassBio) recently said at a Swissnex patient advocacy event where we were both speaking, "if you're not persistent, you don't belong here."
Well here is her video. When I first watched it I was blown away. And then I saw all the videos many of her family and friends did - with the hashtag #PieInTheSkyChallenge. She said she had 45 or so lined up to help from the get go.
And then I saw the video that her cousin, Eliza Dushku did. Holy moly. It was awesome. Huge thanks to Eliza and her husband Peter Palandjian, and the many friends and family members who took part in the challenge. And it's still going! You may get challenged one of these days...I love supporting research.
And then I saw the video that her cousin, Eliza Dushku did. Holy moly. It was awesome. Huge thanks to Eliza and her husband Peter Palandjian, and the many friends and family members who took part in the challenge. And it's still going! You may get challenged one of these days...I love supporting research.
My experience with Nicole and her pie in the sky challenge has been super rewarding. Her original video was beautiful. So genuine and heartfelt you can just feel her sincerity and love for her kids, who are both in the video. She wasn't sure how Elijah, her son with Fragile X would do in a video. Turns out he was amazing. And every time he turned to kiss his mom on the cheek it was a beautiful connection. And the way that Nicole expresses her authentic personality, it is clear this is 100% her idea. I was never going to get in her way with my ideas because it's best to let people be themselves. I just want to help amplify her creation. I will forever remember that Nicole is the one who brought Pie in the Sky to life, and I will think of her every time someone asks me if a cure for cancer, or Fragile X is "pie in the sky." This experience also reinforces my heartfelt belief that if you surround yourself with good people, good things happen. I'm a believer in Simon Sinek's philosophy that everyone has a 'WHY' - The WHY is the purpose, cause or belief that drives every one of us. My WHY is "because I believe that good things happen when you bring together and connect good people who care." I add Nicole and her family and friends to the list of good people who care.
"And don't let anyone tell you your dreams are pie in the sky" - Nicole Eisdorfer
#gratitude #trust #ElizaDushku
