Cure ALD empowers families and engages researchers to make meaningful progress toward preventative treatments, and ultimately, a cure for ALD. Find out more about our story.

Project Details

Purpose

Alex is exploring a new way to treat Adrenoleukodystrophy (ALD) using gene therapy. The goal is to safely deliver a working copy of the ABCD1 gene to the brain and nervous system—the areas most affected by ALD.

The challenge with treating ALD has always been getting therapeutic treatments into the brain, which is protected by something called the blood-brain barrier. He’s testing specially designed delivery vehicles that may be able to cross this barrier more safely and effectively.

Aligned with Cure ALD’s Mission

This research directly supports Cure ALD's top priority: developing new treatments and preventative therapies for Cerebral ALD and AMN.

We're not just working toward better treatments—we're working toward prevention. One of the biggest gaps in ALD care today is the ability to intervene early, before symptoms appear. Alex’s research aims to validate a gene therapy approach that's safe enough to use preventatively, potentially protecting boys and men with ALD before the disease progresses.

This is the vision the Wiener family identified as critical, and it's what drives Alex’s work forward.

Approach

Step 1: Finding the Best Combination

First, we're testing different genetic "switches" (called promoters) to find the one that works best in the brain while minimizing effects in other organs like the liver. Think of this as finding the right key that opens the door where we need it most.

Step 2: Determining Safety and Optimal Dosing

Next, we'll compare different delivery methods at various doses and test them at different ages. Our priority is finding an approach that's both safe and effective, using the lowest dose possible.

Step 3: Measuring Real-World Benefits

Finally, we'll test our leading candidate to see if it actually improves motor function and other outcomes over time. This is where we find out if our approach makes a meaningful difference.

Why this Matters for ALD Families

Families currently face a tragic choice between a fatal disease and treatments that require toxic chemotherapy or carry cancer risks. We are developing a safer, "chemo-free" gene therapy that uses a new delivery vehicle to slip through the blood-brain barrier from a simple IV infusion. If it works, it will replace a grueling, high-risk transplant procedure with a safe, off-the-shelf treatment that can be given immediately upon diagnosis. If it works, it could offer a cure that doesn't come with the threat of leukemia or lifelong side effects.

"Since my son was diagnosed through newborn screening, we have lived with the constant fear of what might appear on his MRIs and blood tests, knowing we had no way to prevent the disease itself. This research represents a fundamentally new approach rooted in prevention, with the potential to spare children from invasive monitoring and preventing the disease from ever developing at all." -- Alison Adler, ALD Parent

About Alex LeNail

Alex LeNail is a postdoctoral researcher in George Church’s lab at the Wyss Institute at Harvard University, where he develops gene therapies for neurodegenerative diseases. His work focuses on creating safer, more effective treatments for conditions like Adrenoleukodystrophy (ALD)

Alex earned his PhD in Computational and Systems Biology from MIT, where he studied ways to reverse age-related changes in neurons. During his doctoral work, he was awarded the PhRMA Foundation Graduate Research Fellowship in Drug Discovery and received recognition for his research at international conferences.

Before his PhD, Alex worked as a Research Associate in MIT's Biological Engineering department, contributing to major collaborative projects studying ALS and other neurodegenerative conditions. His research combines computational biology, machine learning, and experimental approaches to tackle complex neurological diseases.

Alex is passionate about advancing gene therapy technologies that can safely reach the brain and nervous system—work that directly benefits families affected by rare genetic diseases like ALD. Through his research, he aims to bridge the gap between cutting-edge science and real-world treatments that can improve lives.

Visit our website to learn more about Cure ALD

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ALD Research Project: Safer Gene Therapy for ALD

By Cure ALD

ALD Research Project: Safer Gene Therapy for ALD

driven by Alex LeNail, Wyss Institute at Harvard University

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