From Hope to Action:

Transforming the Trajectory of Neurodegeneration, Starting With BPAN


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​Juliet Knowles, MD, PhD, leads a team of Stanford scientists working to uncover the mechanisms of how BPAN impacts children, with implications for other neurodegenerative conditions.

Innovating for Children With BPAN

BPAN (beta-propeller protein-associated neurodegeneration) is a life-threatening pediatric disease that can have a devastating impact on children, including diagnoses such as epilepsy, developmental delay, and autism. In some cases, the disease ultimately strips away hard-won skills, leading to movement abnormalities resembling Parkinson's disease, dementia similar to that seen in Alzheimer's disease, and—heartbreakingly—a life cut short.

BPAN is currently untreatable, and there is no way to predict who will develop severe decline. Yet there is reason for hope. A critical window, often lasting years, exists between early symptoms and later severe decline—giving us the chance to intervene before irreversible damage is done. The key to cures lies in understanding the underlying biology.

Led by pediatric neurologist Dr. Juliet Knowles, our team will launch a first-of-its-kind effort to uncover BPAN disease signatures and discover new therapeutic candidates using animal and patient-derived models. This bold, collaborative mission unites many of Stanford’s top minds across disciplines and leverages the most advanced technologies available to change the future for children with BPAN. Furthermore, this work opens new doors to improve brain health across the life span. The insights uncovered by our work on BPAN will have broad impact: the same biological mechanisms at the root of BPAN (related to autophagy and cellular metabolism) are thought to contribute substantially to common neurodegenerative diseases encountered in adults, such as Alzheimer's and Parkinson's diseases.

Harnessing Stanford's
​Brightest Minds

With groundbreaking neuroscience research and a top-ranked clinical program in pediatric neurology, Stanford is uniquely positioned to accelerate progress in BPAN research. Our experts bring a combination of basic, translational, and clinical expertise relating to neurodevelopment, neurodegeneration, and pediatric epilepsy—all at the root of BPAN. This expertise is supported by the Stanford Wu Tsai Neurosciences Institute, the Stanford Alzheimer's Disease Research Center, and the Stanford Phil and Penny Knight Initiative for Brain Resilience, as well as the West Coast's leading pediatric epilepsy center at Lucile Packard Children's Hospital Stanford, our Level 4 facility offering cutting-edge diagnostics and technologies.

One of our team's greatest assets is its rare integration of pediatric and adult expertise—bringing together complementary insights from both early-life neurodevelopmental disorders and later–onset neurodegenerative diseases. We believe that BPAN's emergence early in life may offer a rare opportunity to understand the origins of neurodegeneration, with far-reaching implications for diseases affecting millions, like Alzheimer's and Parkinson's.

By channeling Stanford's deep bench of expertise, all within walking distance for novel collaborations, we will accelerate discovery, build a dedicated research community, and create a pipeline of treatments that could transform the future for children with BPAN while deepening our understanding of adult-onset neurodegenerative diseases.

Meet Our Dedicated Team

Our extraordinary team of investigators is further strengthened by close collaboration between scientists and clinicians, accelerating the path from the lab bench to patient care. Together, these partnerships expand our understanding of BPAN and open new possibilities across the full neurodegenerative disease spectrum.

Juliet Knowles, MD, PhD

Frank Longo, MD, PhD

Tony Wyss-Coray, PhD

Rebecca Levy, MD, PhD

Sergiu Pasca, MD

Jonathan Long, PhD

Jennifer McNab, PhD

Christopher Lee-Messer, MD, PhD


Advancing Critical Research Pathways
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BPAN Science:
​A Bridge Across Brain Disorders
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In partnership with Alzheimer’s and Parkinson’s expert Frank Longo, MD, PhD, and Tony Wyss-Coray, PhD, a leader in research on aging and the systemic factors that shape brain function, Dr. Knowles is leading a groundbreaking effort to understand BPAN, which like Alzheimer's and Parkinson's, is driven by impaired autophagy (the cell's waste-clearance system). Their work on this rare disorder holds critical clues not only for affected children, but also for millions living with more common neurodegenerative diseases.


Why Stanford, Why Now

Our approach—using a rare pediatric neurodegenerative disease to advance understanding of more common adult-onset diseases—is unique and innovative, bringing together Stanford’s strengths across pediatric and adult medicine in a new way. This work forms the foundation of a novel Stanford-led initiative with the potential to improve neurological health across the lifespan.

We have brought together experts and world-class resources from across campus to develop the first treatments for the devastating pediatric neurodegenerative disease BPAN. We will also be the first to use BPAN as a unique—and, until now, overlooked—lens to probe the underlying biology and identify novel therapeutics for Alzheimer’s and Parkinson’s.

To learn more about our vision and the momentum behind it, we invite you to hear directly from Dr. Knowles and from Sherri Hansen, whose child Zoe lives with BPAN. Their perspectives, shared at an event, reflect both the scientific ambition and the deeply personal commitment driving this effort forward.


Stanford’s uniquely integrated ecosystem—uniting the university’s seven schools, our adult and children’s hospitals, and Silicon Valley partnerships—empowers us to take on the most complex health challenges with unparalleled speed and precision. We are leading the biomedical revolution in precision health to make care more preventive, more personalized, and more effective for children and adults with challenging conditions."
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​- Lloyd B. Minor, MD, Carl and Elizabeth Naumann Dean of the Stanford University School of Medicine, Vice President for Medical Affairs, Stanford University

What began as a few determined voices has become a united effort to change the future of BPAN. With every gift, our community brings new hope to families living with this rare disease—and brings us one step closer to fulfilling our bold BPAN program goals.
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Isa—possibly the youngest person ever diagnosed with BPAN—is inspiring the search for answers.

Philanthropy is the catalyst. With your partnership, we have an opportunity to act at a time when expertise, resolve, and possibility are converging at Stanford. Together, we can take the next bold step toward a future where children with BPAN have every chance to thrive.
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Thanks to our generous donors, we are already making incredible progress.
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Click the buttons below to read about how we're advancing our shared goal to improve the lives of children and families affected by BPAN.

Science is the cavalry coming over the hill. That image gives me so much hope, and I repeat this phrase often. But I have come to realize that the cavalry’s arrival is not an inevitability. You need to call the cavalry, and you need to do so with urgency if you want it to arrive in time. The cavalry is made up of scientists at Stanford. They have the technology. They have the intellect. They have the raw capacity to pursue this groundbreaking work. But they also need our support to fuel them.”
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​- Sherri Hansen, parent advocate for BPAN research and mom to Zoe

Our team has a bold and ambitious plan.

With visionary philanthropy, we can push the boundaries of what’s possible. Together, we have the opportunity to transform how we understand and treat BPAN and other neurodegenerative diseases.

To make a gift today:

Visit the donation page for Dr. Knowles' BPAN research.


​For more information, contact:
​Melisa Addison
​Director of Development, Major Gifts
​(650) 723-0072; Melisa.Addison@LPFCH.org

Maura McGinnity
​VP, Principal Gifts & Strategic Initiatives
​(415) 225-8865; Maura.McGinnity@LPFCH.org
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​True to her nickname, "Spark," Zoe meets BPAN with fierce courage and optimism.