A Guest Perspective for Gratitude Day: Why Huntington’s Disease May Be Neuroscience’s Best Investment

Every year on Gratitude Day – March 23rd – the Huntington’s disease (HD) community pauses to reflect on the researchers, clinicians, families, and advocates who make this work possible.

This year, we’re marking the occasion by sharing a guest piece from Roy Maimon, PhD, Assistant Professor of Biomedical Engineering at NYU and an HD researcher specializing in stem cells and brain regeneration. Roy recently published an opinion piece suggesting that HD, with its singular genetic clarity, predictable disease course, and deeply united community, may be one of the most powerful entry points for understanding and repairing the brain.

We thought there was no better day than Gratitude Day to share it. HD has given neuroscience so much. Roy’s piece says why.

Here, we’re proud to share a guest piece by Roy in his own words. What follows is his perspective, offered here as the kind of voice we want to amplify: a researcher who came to HD science and found not just a compelling problem, but a community worth being part of.

A rare disease with unusual clarity

Neuroscience rarely enjoys clean experiments.

Most brain disorders are mosaics of risk genes, aging, lifestyle, and chance that leave their origins obscured. 

HD is different.

It begins with a single genetic expansion, a repeated stretch of DNA letters in the huntingtin (HTT) gene. If you inherit a sufficiently long repeat, you will develop the disease. That stark clarity makes HD scientifically invaluable.

That is the case I made in a recent essay in Trends in Molecular Medicine, titled “Huntington’s Disease Is the Best Investment in Neuroscience Today.” The article headline was the cover of the volume, reflecting a simple idea: HD may be one of the most powerful entry points for understanding and repairing the brain.

The power of a molecular clock

The HTT expansion acts like a molecular clock.

Decades before the first involuntary movement or subtle cognitive change, a blood test can reveal who carries the expansion.

Few neurological diseases offer such foresight.

That predictability allows researchers to ask a question that is nearly impossible elsewhere. For example, what happens if we intervene before neurons begin to die?

A disease with a predictable path

The brain changes in HD follow a surprisingly consistent pattern.

Early damage centers on the striatum, a deep brain structure involved in movement and decision making. Over time, connected regions of the outer wrinkled part of the brain, called the cortex, become involved.

Even within the striatum, some neurons are especially vulnerable while their neighbors remain relatively resilient.

Why some cells are fragile and others hardy is a major puzzle. HD offers a controlled system to investigate it.

A proving ground for new therapies

Because the genetic cause of HD is so precise, the disease has become a testing ground for new therapies.

Antisense oligonucleotides (ASOs) aim to lower production of the harmful huntingtin protein. Gene therapies attempt to deliver long lasting genetic instructions that modify the gene’s output. Other approaches target the DNA repair machinery thought to drive repeat expansion.

Not every clinical trial has succeeded. But each has sharpened our understanding of biomarkers, drug delivery, and how to measure change in the human brain.

Can the brain rebuild itself?

HD also raises an even more ambitious question.

Can the adult brain regenerate?

The striatum sits near the subventricular zone, one of the few places in the adult brain capable of generating new neurons.

We and others showed in animal studies that boosting this process of creating new neurons, called neurogenesis, can partially rebuild damaged circuits. Other experimental strategies involve transplanting healthy cells in the disease brain regions.

These ideas remain early, but HD provides a uniquely measurable testing ground. It provides a known mutation, defined target cells, and a predictable timeline to disease onset.

If regeneration works in HD, it could reshape how we think about treating neurodegeneration more broadly. 

In my lab, we push this idea forward, and we use HD as a model. 

The people behind the science

What makes HD unique is not only the biology. It is also the people.

The HD world is unusually united.

It is among the few fields in biomedicine where people with HD, scientists, and clinicians share the same space. We meet families regularly. We see their courage, and their humor.

We celebrate together and we grieve together.

It is this culture that makes HD research not just productive, but deeply personal. When you join this community, you become part of something that truly matters.

At HD meetings the science rarely stops when the talks end.

Ideas continue in hallways, at dinners, and sometimes in unexpected places. At one recent meeting, me and my colleagues and dear friends, also HD researchers, Carlos Chillon Marinas and Sonia Vázquez-Sánchez, even organized an impromptu party in a camper van we rented during the conference, where conversations between scientists, clinicians, and families continued long into the evening.

Moments like these often spark new collaborations.

They are part of the reason the field moves so quickly.

Why investment in HD benefits everyone

HD affects roughly one in every ~4,000 people worldwide.

That number may appear small, but its scientific impact is enormous.

Every dollar invested in HD produces tools, models, and biomarkers that accelerate discoveries throughout neuroscience.

For students, it is one of the fastest ways to learn science with real translation to therapeutics.

For funders and investors, it is an unusually efficient place to deploy resources.

Today HD research spans many disciplines. Stem cell lines created from patients, careful tracking of symptoms against disease progression, large natural history studies, advanced imaging, and gene therapy all contribute to a growing pipeline of translating science into drugs.

Few diseases offer such a complete bridge between molecular biology and clinical medicine.

A hub for future discovery

I believe that New York University (NYU) is uniquely positioned to serve as a national hub for Huntington’s disease research.

The university bridges engineering, neuroscience, clinical medicine, and data science within a single ecosystem. Discoveries can move rapidly from molecular insight to patient-facing trials.

NYU also hosts strong programs in gene therapy, RNA therapeutics, biomarker development, and computational modeling.

Equally important, its proximity to major clinical centers and HD communities allows sustained engagement with families living with HD.

A blueprint for repairing the brain

If the ultimate goal of neuroscience is to understand the brain well enough to repair it, Huntington’s disease may offer the most direct path forward.

Its genetic clarity turns complexity into opportunity.

Its research infrastructure turns investment into impact.

And its community turns science into belonging.

For me, HD is more than a disease to study.

It is a blueprint for how neuroscience can move from discovery to cure.