Sail for Epilepsy, Part 7: A Challenge for Our Industry

Emily Walsh Martin, PhD - June 29, 2022

Her own journey across the Atlantic is now complete, but Dr. Emily Walsh Martin says the industry is still in the earliest stages of its journey developing novel treatments to improve the lives of people with epilepsy.

I’ll be honest; this blog series was not an easy one for me to embark upon. As I’m sure you’ve concluded, it is exceedingly difficult to navigate a path to develop novel gene therapies. The concept is straightforward: gene is broken, add back normal gene. And yet, for every new therapy, development teams must labor to develop the evidence that they have designed a safe and effective vector and selected appropriate doses that will give every patient a reasonable hope for benefit in the clinic. As someone who has seen teams face these challenges, it is hard to describe how paralyzing it can feel to face unknowns and still find a path forward to the clinic.  And yet, as an industry, we try to do just that.   

Still, in our development teams, we can feel very isolated as we face these obstacles. We sometimes lose sight of the fact that we are all part of a larger team – team gene therapy. And for that team to be successful, we must maintain our sense of community and transparency. It is understandable in this very crowded field for scientists to feel a sense of “us versus them” competition with other companies or institutes who are pursuing similar indications and sometimes even the same indication as they are. However, for the past 20 years, the best part of the gene therapy industry has been our willingness for transparent, in-depth, open sharing of experiences regardless of institute or company. This sharing is not just for our successes, but also our challenges and failures from discovery to manufacturing to clinical application.   

We cannot lose our dedication to the knowledge sharing at the very core of our success as our field expands. And frankly, as companies grow and new, larger players enter the field, we will need them all to be leaders in this approach, even though it may be counter to their quieter corporate traditions for more traditional therapies. This is necessary because the stakes are simply too high. To continue to ask patients and their caregivers to volunteer for therapies that may be their “one shot on goal,” we need to be able to adequately explain the risks and uncertainties for these therapies. And the greatest resource we have to help explain that context is our shared community experience with dosing therapeutic vectors across many different types of diseases and genes and patients. Therefore, we must continue to build and publish that knowledge. 

Thank you all so much for following the blog as we voyage through the complexities of developing novel therapies for monogenic epilepsies. While the journey across the Atlantic is now complete, the industry is still in the earliest stages of its journey developing novel treatments to improve the lives of people with epilepsy.

Read the Series

Part 6: Key Considerations of Gene Therapy Development: A Caregiver's Perspective

Part 5: Realities of Selecting Clinical Dose Levels

Part 4: Key Elements for Selecting a Proper Clinical Dose

Part 3: How Would Gene Therapy Work to Address Monogenic Epilepsies?

Part 2: An Overview of Syndromic and Monogenic Non-syndromic Epilepsies

Part 1: A Worthy Excuse for Missing the ASGCT Annual Meeting

Emily Walsh Martin is a volunteer crew member for Sail For Epilepsy’s Atlantic crossing on the vessel Ingwe. When she’s not sailing, she is a consultant for gene and cell therapy companies and investors who are seeking to advance novel therapies in the clinic.