local storiesDecember 2, 2022

Community Highlights: Meet Emily Rogath Steckler of Bisous For Léo

Local Stories

Today we’d like to introduce you to Emily Rogath Steckler.

Hi Emily, thanks for sharing your story with us. To start, maybe you can tell our readers some of your backstory.
In 2018, I co-founded the organization Bisous For Léo with Deborah Vauclare, my dear friend of nearly 30 years. Her son Léo was diagnosed with a rare life-threatening neurodegenerative disorder called Infantile Neuroaxonal Dystrophy (INAD). Never heard of it? We hadn’t either. Out of the gate, that in itself was (and continues to be) a huge challenge.

We all face challenges, but looking back would you describe it as a relatively smooth road?
Getting people to stay focused long enough to learn about these children and why they are so important has proven to be half the battle. The other half? Identifying and funding a path forward to best medically support them. The children with INAD share the exact same gene mutation found in some forms of Parkinson’s (PLA2G6) and share a pathogenesis with Alzheimer’s and Lewy Body Dementia. There are 50 million people worldwide that are genetically linked to them. It behooves everyone trying to eradicate the larger neurodegenerative diseases to put greater focus on these children. I view them as a missing puzzle piece in a much larger neurodegenerative puzzle. They are rare but oh so important.

We’ve been impressed with Bisous For Léo, but for folks who might not be as familiar, what can you share with them about what you do and what sets you apart from others?
I can’t take full credit for anything the collective “we” are doing to treat and cure INAD. Together with the INADcure Foundation, of which Bisous For Léo is an ancillary arm, we have invested in discovering the underlying mechanisms of the disease, supported a 5-year natural history study, funded a repository of patient cells in collaboration with the New York Stem Cell Foundation as well as successfully edited a patient cell line using base editing technology. We have also funded and supported the necessary pre-clinical studies for our gene therapy program with a roadmap of less than 24 months to the clinic.

When the INADcure Foundation was formed in 2016, there was nothing available that would stop or slow the progression of INAD and there was very little was known about it. I’m proud that no one at the foundation or Bisous For Léo is taking “no” for an answer. It is with this determined mindset that we have been able to persevere.

How do you define success?
Success to me is whenever someone listens and takes the time to help, whatever that may look like. It’s cliche, but it’s true that it takes a village.

Thanks to our scientific advisory board, we have the best team in place to potentially move forward with a gene therapy clinical trial. Aldevron has recently completed the production of our plasmids and Andelyn Biosciences is on board for our GMP and GLP manufacturing.

Our biggest obstacle is obtaining larger funding to keep our current work in motion. Every tax-deductible dollar donated to the cause ensures that we can keep pushing forward. Without funding coming in? Everything comes to an abrupt halt. I remain hopeful that with end-of-the-year giving, we can keep our mission afloat ultimately resulting in the success that the collective “we” so desires.

Contact Info:

Image Credits
The wall with me and Dylan + Chloe (my kids): Andrea Amaral Me and Deborah: Antoine Vauclare Leo: Deborah Vauclare Me and Chloe: Jeremy Steckler Headshot: Erin Scabuzzo

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