Friends and supporters,

Late last year, we sent our first newsletter. We had just funded our first research projects, William had just received his first clear MRI, and we were cautiously optimistic.

Since then, we’ve experienced both the promise and the reality of this disease.

• William was diagnosed with adrenal insufficiency in the spring.

• We hosted our first donor dinner.

• Researchers have begun reporting results from the projects you made possible.

• One of those projects helped secure a $1 million NIH award.

And somewhere along the way, it became clear that Will to Cure ALD could play a meaningful role in shaping the future of ALD research.

We’re beginning to see that strategic philanthropy can do more than fund research. It can accelerate it.

Thank you for believing in what we’re building and for making it possible.

With gratitude,
The Hall Family
Will to Cure ALD

In December, we deployed our first research portfolio across six institutions. Six months later, the first reports are coming back from the labs.

The progress has been real. Some projects have evolved as we’ve learned more, and a few findings have surprised even the researchers, including one that may point to a treatment approach nobody in the field had previously identified.

Here's what we're seeing.

A New Approach to Gene Therapy

One of the most exciting updates came from Dr. Joshua Bonkowsky's lab at the University of Utah. His team is developing a new approach to gene therapy that targets RNA rather than making permanent changes to DNA. If successful, it could offer a safer and more flexible treatment option for patients.

The early results have exceeded expectations. The team is reporting correction rates roughly ten times better than comparable technologies. A scientific publication is planned later this year, with animal studies expected to follow.

While there is still significant work ahead, these are the kinds of results that give us reason for optimism.

The Power of a Coordinated Research Portfolio

When we funded Dr. Marc Engelen's team at Amsterdam UMC to sequence the genomes of 100 ALD patients, we discovered that another research team we support was doing the same thing independently. The two teams are now combining their datasets, and that matters. In rare disease research, the biggest obstacle is sample size.

More genomes means more statistical power, more confidence in findings, and a better chance of identifying the genetic modifiers that determine disease outcomes. A dataset this size gives researchers a better chance of finding meaningful answers.

The Leverage Effect of Your Support

When we funded Dr. Amena Smith Fine at Kennedy Krieger Institute to study how ALD progresses in adults, she had already submitted multiple NIH grant applications. One recurring challenge was funding for the MRI scans needed to support her research.

Our grant filled that gap.

This spring, she was awarded a $1 million NIH Career Development Award that builds directly on the work your support helped launch.

"I made several attempts over a few years to obtain an NIH Career Development Grant. Reviewers rejected my application in part because there was no separate source of funding to pay for the MRI scans needed. The generous Will to Cure ALD gift resolved this problem, and this critical seed money has now secured an additional $1 million to support ALD research."

— Amena Smith Fine, MD, PhD, Kennedy Krieger Institute / Johns Hopkins University

This is exactly how we hope philanthropy works. Strategic funding can help researchers generate the data they need to compete for much larger sources of support, multiplying the impact of every donor dollar.

In May, we hosted our first formal donor dinner at Brook Hollow Golf Club in Dallas. Nearly 100 people joined us, including researchers, donors, and ALD families, some meeting one another for the first time.

Dr. Keith Van Haren's flight was diverted on his way from Stanford, but he still joined us by video from the tarmac. That commitment says a lot about the people we’re fortunate to partner with.

Morgan Phillips, whose son Jude was diagnosed with ALD in 2024, shared her family's story with the room. Her husband, Stephen Phillips, spoke from his experience as a biotech investor on why this research matters and why it can't wait. It was one of the most powerful moments of the evening.

Together, the evening raised $200,000, which was matched dollar for dollar, bringing the total impact of this single night to $400,000.

But what we’ll remember most wasn’t the fundraising. It was looking around that room and seeing newborn screening families, carriers, AMN patients, and researchers all in the same place, all fighting for the same thing.

That’s the community we’re trying to build.

We received 501(c)(3) approval this spring as a standalone entity. We are currently planning our migration to that structure, which will give us more flexibility as we grow. We will continue to work with The Dallas Foundation during the transition. Follow our website for updates as the migration progresses.

Tyler was also appointed to the ALD Connect board of directors. ALD Connect is the leading national advocacy organization for the ALD community. His focus will be on national fundraising strategy and shaping how the organization deploys its research investment dollars. This keeps us connected to the broader ALD research community and reflects our commitment to supporting the field beyond our own portfolio.

We have also started to use social media to share more real-time updates. Follow along on Facebook or Instagram.

We know there is still a long way to go, but the path forward is becoming clearer. Our goal isn’t simply to fund great science. It’s to help bring together the researchers, ideas, and resources needed to move the entire field forward.

If you’d like to continue supporting our work, every dollar goes directly toward funding the next generation of ALD research. You can give at willtocureald.org.

We have more to share soon. Thank you for being part of this.

The Hall Family