CACNA1A Treatment: The Fastest Path Forward

Our bold new science strategy built around one central goal: moving the most promising CACNA1A treatment forward.

Picture a future where a CACNA1A-related disorder diagnosis comes with a real answer: a treatment built for this exact gene and for this exact community. That future is what we are building together, starting now.

In April 2026, our CACNA1A community launched The Fastest Path Forward, a bold new science strategy built around one central goal: moving the most promising CACNA1A treatment toward a clinical trial as efficiently and thoughtfully as possible.

At the center of that goal is a precision medicine approach aimed directly at the CACNA1A gene and the Cav2.1 channel it encodes. 

Our strategy focuses on three connected priorities: building CACNA1A Connect so families can shape research with their lived experience, learning everything we can from the data our community has already generated, and advancing the single most promising treatment approach targeting CACNA1A or the Cav2.1 channel.

Three Pillars of The Fastest Path Forward

Each pillar builds on the one before it. Together, they connect the lived experience of CACNA1A families to the scientific decisions that will shape future treatments.

  • CACNA1A Connect is a new platform (coming soon!) designed to make it easier for families to participate in research and share their lived experience. Families have told us that research needs to reflect real life: which symptoms matter most, how experiences change over time, and what meaningful progress actually looks like day to day.

    Bringing that information together in one place helps researchers and clinicians learn directly from the community, and helps ensure the path toward treatment stays grounded in the priorities of the families living with CACNA1A-related disorders every day.

  • Our community has already built an extraordinary foundation of data through Natural History studies, RARE-X, Citizen, and other research efforts. Rather than treating each of those as a separate project, we are now analyzing them together to identify patterns, close gaps, and surface insights that can strengthen research across the field.

    This work will help shape clinical care recommendations, support stronger standards of care, guide future research decisions, and make sure that future studies and clinical trials reflect the realities of families living with CACNA1A-related disorders.

  • This is the heart of The Fastest Path Forward: identifying and advancing the single most promising therapeutic approach targeting the CACNA1A gene or the Cav2.1 channel protein it encodes. 

    This work is led by Dr. Brandon Henry, our Chief Science Officer, a physician-scientist specializing in the clinical development of gene replacement, gene editing, and RNA therapies for rare and ultra-rare disease. His team is evaluating different therapeutic modalities and assessing where the science and the opportunity are strongest, with the goal of moving the single most promising approach toward the clinic as efficiently as possible.

    The chosen approach must also meet a second bar: it has to address the real priorities and needs of the CACNA1A community, not just what is scientifically interesting. Once a modality is selected, the Foundation will share that decision with the full community.

Gauging Our Clinical Trial Readiness

To ensure we choose a treatment strategy that aligns with our community's desires, we need to understand your perspectives. Please help us gauge interest in genetic medicines and measure our overall clinical trial readiness by completing our Clinical Trials Perspectives Survey. Your feedback is vital to helping us shape this next phase of CACNA1A treatment development, because elevating the patient's voice is how we ensure we are on the fastest path forward.

Meet the Science Team

An ambitious strategy requires dedicated, expert leadership. The Fastest Path Forward is led by a science team with clearly defined roles aligned to each pillar of the strategy.

Brandon Henry, MD — Chief Science Officer

Leads Pillar 3. Brandon is focused on identifying and evaluating potential therapeutic approaches targeting CACNA1A or the Cav2.1 channel, and on moving the most promising treatment forward as efficiently as possible.

Pangkong Fox, PhD — Science Director

Leads Pillars 1 and 2. Pangkong oversees the Foundation's research network, spearheads CACNA1A Connect and maximizes insights from the data the community has already contributed. She also brings a deep personal connection to the mission as she is a parent to a son with a CACNA1A-related disorder.

Michelle Kielhold, PhD — Science Program Manager

Supports Pillars 1 and 2 and helps translate complex science into updates families can actually use, drawing on a background in translational research.

How Families Can Help Move the CACNA1A Treatment Forward

We Are All In. Are You?

What comes next will require the same level of partnership and participation.

The steps we take now, together, will shape how quickly and effectively we can move toward a treatment.

Share Your Lived Experience.

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Coming soon CACNA1A Connect.

Fund the Path Forward.

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Help us fund this precision medicine approach.

Donate here

Stay Informed.

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Sign up for updates as we reveal the chosen therapeutic modality and more in the coming months.

Newsletter sign up here

Connect with Leadership.

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Have questions or comments about our new scientific direction? Reach out directly to Executive Director Shira Johnson at shira@cacna1a.org.

Your Questions, Answered

  • Not yet, but the CACNA1A Foundation is working to change that. Today, families manage CACNA1A-related disorders by treating individual symptoms, like seizures, ataxia, and hemiplegic migraine, through a team of specialists. Our science strategy, The Fastest Path Forward, is focused on advancing a targeted treatment for the CACNA1A gene and the Cav2.1 channel it encodes toward a clinical trial.

  • The Fastest Path Forward is the CACNA1A Foundation's science strategy, launched in April 2026. It centers on three priorities: building CACNA1A Connect to gather lived-experience data from families, learning from data the community has already contributed, and advancing the single most promising treatment approach toward clinical development.

  • A dedicated science team leads this work, each with a role tied to one of our three pillars. Dr. Brandon Henry, our Chief Science Officer, leads the search for the most promising treatment targeting CACNA1A or the Cav2.1 channel, drawing on his background in gene replacement, gene editing, and RNA therapies for rare disease. Pangkong Fox, PhD, our Science Director, oversees CACNA1A Connect, data analysis, and our research network, and Michelle Kielhold, PhD, our Science Program Manager, supports our science efforts and helps communicate the science to our families.


  • The Foundation is pursuing a precision medicine approach: a therapy that specifically modulates the CACNA1A gene or the Cav2.1 channel protein, chosen for its potential to reach the clinic as efficiently as possible while addressing the priorities of the CACNA1A community. At this point, we have not decided on a modality. We will update this page when we do.

  • Treatment currently depends on the specific symptoms a person has. Most families work with a team that may include a neurologist, an epileptologist, a neurogeneticist, an ophthalmologist, and a developmental pediatrician, depending on the symptoms involved. You can read more about CACNA1A-Related Disorders here.‍

  • There is no confirmed timeline yet. The Foundation is currently assessing therapeutic approaches and will share the chosen modality with the community once a decision is made. The Fastest Path Forward is designed as a 24-month strategy to move that decision and the work that follows forward as efficiently as possible.