…When you look at the big picture of Parkinson’s, Alzheimer’s and Lewy Body Dementia, these are giant problems that fall under the even larger category of Neurological Diseases. Saying you want to cure these diseases is a goal. To achieve the goal requires lateral thinking as well as Socratic logic. The INAD gene mutation — PLA2G6 might be the key that unlocks the solution to diseases that affect tens of millions and their families.
As a part of my series about “Big Ideas That Might Change The World In The Next Few Years” I had the pleasure of interviewing Emily Rogath Steckler
Emily has extensive experience in Public Relations and Marketing and has applied that knowledge to Bisous For Léo. With a Master of Arts from New York University in Visual Arts Administration, she worked in PR for such companies as I|D Public Relations and Warner Brothers Pictures Group. She also worked in Marketing for Terry Hines & Associates and Creative Riff. Bisous For Léo is Emily’s first foray into the nonprofit world.
Thank you so much for doing this with us! Before we dig in, our readers would like to get to know you a bit. Can you please tell us a story about what brought you to this specific career path?
Thank you. Prior to co-founding Bisous For Léo with Deborah Vauclare, there was nothing to suggest that this is where I would focus my attention. Yet, this is exactly where I was destined to be. Deborah and I met when we were 14 years old in New York City and quickly became friends. When it was time to apply to college, we decided to go together to The University of Wisconsin in Madison. After college, we returned to New York City and furthered our education — Pratt Institute for Deborah, NYU for me. Our lives were in sync until Deborah won a lottery ticket of an internship to work for a famed interior designer in Paris. This seismic life change led to the love of a co-worker — Antoine, who would eventually become Deborah’s husband and the father of their two children, Léo and Éva.
Léo, their eldest, was born in April 2016. Coincidentally, I was in Paris and was able to visit them in the hospital the day he was born. I’ve seen Deborah become many things over the years, but watching her become a mother was a new privilege. On top of that, Léo was the cutest! Dark hair and deep hazel eyes. As he grew, the playful personality and the omnipresent smile would follow. It wasn’t until around 14 months when Deborah and Antoine started to become concerned. The boy who had learned to walk, talk and hit his developmental milestones like a champ was slowly and then more rapidly starting to lose all his acquired skills. Genetic testing resulted in something none of us had ever heard of: Infantile Neuroaxonal Dystrophy (INAD), a neurodegenerative disease with no treatment or cure with a projected life expectancy of 5–10 years. As fate would have it, I was slated to be in Paris the day after Deborah and Antoine received this news. When I got there, we processed the news, sat in silence trying to digest the inconceivable and then we initiated our research. We learned the children with INAD share a gene mutation with some forms of Parkinson’s (PLA2G6) and have the same pathogenesis as those affected with Alzheimer’s and Lewy Body Dementia. Not similar; the same! Babies and children affected by a gene defect that I, and most people, had previously believed could only impact adults for early onset and more commonly, the elderly. I had watched my own grandmother change and eventually succumb to Alzheimer’s with Lewy Body Dementia and was horrified to realize that Léo and other children could also suffer the same fate. As a result of a personal shock and our research into broader social impact, Deborah and I created Bisous For Léo (Kisses for Léo), an ancillary arm of the INADcure Foundation, the only US-based nonprofit working to eradicate INAD. Through awareness, education and funding, we are striving to support treatment and cure development to remediate INAD.
Can you please share with us the most interesting story that happened to you since you began your career?
It’s not so much a story but a recognition of not being in the right place. I quit a job once because it was too much of a good thing in that I truly enjoyed the work, but was constantly overwhelmed by the amount of it. I felt I couldn’t be effective in that construct. My focus on Bisous For Léo is intense and unwavering.
Which principles or philosophies have guided your life? Your career?
Fate has been an overriding theme. If something so unbelievable, even inexplicable, presents itself, rather than question why, I’m inclined to embrace it. I grew up listening to a story my father told me about his own father, who had survived the Holocaust. When my grandfather was a young boy, his father took him to a neighboring town to see the Rabbi. Not just any Rabbi, but the Rabbi. There was a long line that ran from morning to night. When my grandfather and his father were finally ushered into the great man’s inner sanctum, the Rabbi refused a donation and instead gave the pair a gold coin, explained that this coin must remain with my grandfather at all times and stated it would save his life. His father agreed and upon returning home, his mother sowed an inner pocket inside each pair of my grandfather’s pants. This coin was present in various custom pockets for years. After capture by the Nazis, he worked in a concentration camp and at the point of starvation, he was able to bribe a guard with the coin to exchange it for food which saved his life. My grandfather’s name? Leo.
Ok thank you for that. Let’s now move to the main focus of our interview. Can you tell us about your “Big Idea That Might Change The World”?
Think small. Perhaps not a novel idea, but arguably the most important. I recently listened to the true-crime podcast In The Dark about the kidnapping and murder of an 11-year-old-boy named Jacob Wetterling. The set-up is gut wrenching enough, but where it becomes completely implausible is that it took 27 years to solve the case that rocked a small town in St. Joseph, Minnesota. Jacob was kidnapped on a dead-end road in a town of 3,000 people. Lead reporter Madeleine Baran and the APM Reports team initially investigated what was taking so long to solve the case given that there were witnesses as well as a clear suspect. They investigated the Sterns County Sheriff’s Office and discovered systemic failures in this case as well as numerous others. Some of the failures included not interviewing neighbors, not following up on witness leads, not connecting obvious dots to eventually convict, and ultimately casting too wide a net to solve the case rather than starting with a small geographical circle. For two plus decades, the crime was described as “the perfect crime” and it was widely accepted that law enforcement had done everything they could when they launched a massive, thorough investigation and that simply wasn’t true. Law enforcement failed because they went too big too quickly and failed to act on what was right in front of them all along.
Another example that is seared into memory is the movie Children of Men. The world is in chaos and women are infertile. Miraculously there is one pregnant woman; just one. Humanity and the fight for the world’s survival rests with this one improbable baby. I was overwhelmed by the thematic construct that one child could be deemed so vital. So it may be regarding Léo and INAD. There are mysteries that lie within each of us and the children who have INAD are far and few between. There are about 200 of them worldwide. No race, sex or religion ties them together, but each hold this Parkinsonian mutation and the genetic ties to Alzheimer’s and Lewy Body Dementia. Today, 50,000,000 people can potentially benefit from INAD research and remediation. Helping 200 children may lead to treatments and possibly a cure for these neurodegenerative diseases.
How do you think this will change the world?
When you look at the big picture of Parkinson’s, Alzheimer’s and Lewy Body Dementia, these are giant problems that fall under the even larger category of Neurological Diseases. Saying you want to cure these diseases is a goal. To achieve the goal requires lateral thinking as well as Socratic logic. The INAD gene mutation — PLA2G6 might be the key that unlocks the solution to diseases that affect tens of millions and their families.
Let’s revisit the Jacob Wetterling case for a minute. The takeaway is that narrowing the focus solved a big problem. Rather than looking at the INAD afflicted children and saying there are “only” a couple of hundred impacted, I believe their gene mutation may unlock answers that will help millions of afflicted adults.
Recently Bisous For Léo and the INADcure Foundation expanded its partnership with The New York Stem Cell Foundation to explore the possibility of developing a cellular therapy for INAD patients using gene-corrected neurons. In collaboration with CRISPR pioneer Dr. Feng Zhang of MIT, NYSCF has successfully gene-edited a patient iPSC line to correct their PLA2G6 mutation. The next step is differentiating this line into neurons, to verify that the gene edit successfully reverses disease phenotypes. If a proof of concept is established, it will open doors to pursue a cell replacement therapy. In other words, we’re thinking small — on a cellular level — but the impact could be big.
Keeping “Black Mirror” and the “Law of Unintended Consequences” in mind, can you see any potential drawbacks about this idea that people should think more deeply about?
The only potential drawback is inaction. Consider Covid-19 and its global impact. Initially a few infections in a city in China hardly made the news. Most didn’t notice. Today, it has impacted the entire world. It’s unclear if the INAD gene mutation might increase over time, but until it’s eradicated we simply can’t control or stop it.
Was there a “tipping point” that led you to this idea? Can you tell us that story?
When I was in Paris the day after Léo had been diagnosed, my four year old daughter Chloe was with me. She had been listening to me speak with Deborah over the phone prior to arrival and though neither of us had any idea what exactly was wrong, she instinctively picked up that something was amiss. When we ultimately walked into their apartment, the first thing Chloe did was walk over and kiss Léo. Deborah and I watched the exchange and thought, how wonderful it would be if only we could kiss Léo and make him better. The basis of the campaign was inspired by that image.
What do you need to lead this idea to widespread adoption?
Focus and funding. We have the smartest medical minds working on treatment and cure options for the children who are impacted by INAD. We need money to actualize initiatives with urgency. Watching “Operation Warp Speed” plow through trials and testing has been awe inspiring because it shows what is possible with necessary funding and focus.
What are your “5 Things I Wish Someone Told Me Before I Started” and why. (Please share a story or example for each.)
- PEOPLE WILL SURPRISE YOU: There are people who I thought would jump when I asked for help and many didn’t even hop. Then there are people who I’ve met along the way, with whom I had no prior history with, who have gone above and beyond. I’m surprised and disappointed by the former and awed and grateful for the latter.
- YOU WILL GET MORE NOs THAN YESSES: For every successful collaboration or funding initiative that has come to fruition, there are many more examples of “Nos” or passes behind the scenes. Similar to how the medical world views “failures,” we would be absolutely nowhere if we just gave up upon hearing the word “No.”
- STAY ON YOUR OWN YOGA MAT: When I was in grad school one of my yoga teachers at Juvamukti Yoga advised the class to stay on their own yoga mat. She didn’t mean literally, she meant that if you spent your time looking around the room at what others were doing, then you would never reach your own full potential. This notion has stayed with me ever since.
- DO UNTO OTHERS…EVEN IF THEY DON’T DO UNTO YOU: If you treat others the way you want to be treated, good will eventually find its way back to you. If you don’t, something negative will likely follow.
- GET MOVING NOW: Don’t wait to do something…anything! Just create and push forward. The ball doesn’t roll itself.
Can you share with our readers what you think are the most important “success habits” or “success mindsets”?
Above all else, stay focused. That doesn’t mean sit in front of the computer all day waiting for inspiration to strike, it means focus your mind to identify the goal and then work backwards and pinpoint the steps needed to actualize it. I walk around my neighborhood daily and don’t listen to music when doing so. This allows me to process my thoughts and ideas, so that when I do get back in front of the computer I can actualize them.
Some very well-known VCs read this column. If you had 60 seconds to make a pitch to a VC, what would you say? He or she might just see this if we tag them 🙂
We have three pieces of a four piece puzzle in place…1) Children who are affected with INAD 2) Medical experts who have identified clear paths to treating and hopefully curing those children and 3) The connection between the INAD gene and diseases that kill tens of millions each year. What we are missing is funding to much more quickly address the problem.
If any VC is interested in exploring biotech, it feels like an obvious next step. Look at BioNTech in Germany, a startup that is the first drug maker to show successful results from clinical trials of the coronavirus vaccine. Their value has ballooned. Now think about the neurodegenerative space…it would be highly remunerative to solve the Alzheimer’s, Lewy Body Dementia and Parkinson’s puzzle. Further medical development surrounding the shared gene mutation and pathogenesis might provide the key.
How can our readers follow you on social media?
We’re @BisousForLeo across the board. Or people can check out BisousForLeo.org for additional information, or to make a tax-deductible donation.
Thank you so much for joining us. This was very inspirational.
I appreciate the opportunity.
Emily Rogath Steckler’s Big Idea That Might Change The World was originally published in Authority Magazine on Medium, where people are continuing the conversation by highlighting and responding to this story.