Turning hope into reality through breakthrough research and technology
Rapid advancements in AI, genetic engineering and drug development are giving us hope. The technology to develop treatments for AxD exists today.
But to turn technology into treatments requires dedicated research, and research requires resources that academic labs often do not have.
Simply put, research is expensive. A small study to test a promising drug in rats with Alexander Disease can cost US$100,000. But without this, drugs cannot ever be given to children like Max. This is why we have dedicated ourselves to connecting scientists to AxD and with each other, and raising funds to support promising research projects.
Support Critical Research and Researchers
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Harvard Medical School
Support Dr. Mel Feany M.D, Ph.D., a professor of Pathology at Harvard Medical School and Brigham and Women's Hospital.
Dr Feany has identified ways in which Alexander Disease causes brain damage, and drugs that could potentially reduce it.
Donations will enable lab experiments test these new approaches to treating Alexander Disease.
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Waisman Center, University of Wisconsin-Madison
Support Prof. Tracy Hagemann, Ph.D at Alexander Disease Lab Waisman Center, University of Wisconsin-Madison
Dr Hagemann has uncovered mechanisms that could be targeted to - potentially - slow down disease progression and improve symptoms.
Donations will help conduct critical experiments to understand the disease and how it is affected by potential treatments.
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End AxD
Support our dedicated team of parents, united in our mission to find a cure for this devastating disease and support all those affected.
Partnering with an esteemed Scientific Advisory Committee and End AxD we are prioritizing research priorities and bundling our resources.
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UNC School of Medicine
Support Dr Natasha Snider, Ph.D., Professor of Cell Biology and Physiology UNC Chapel Hill School of Medicine.
Donations could support building a library of the specific genetic typos causing Alexander Disease. This helps us see how these errors make the key GFAP protein go wrong and find ways to stop it, aiming for new treatments.
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This list is not exhaustive...
We are in the soul crushing position where more promising treatment mechanisms have been identified than scientists can pursue, simply because it costs too much.
Your donations could remove that barrier by enabling critical experiments.
Where Your Donations Already Make a Difference
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UMass Chan
We donated US$75,000 to Prof Gao, Prof Xie to support research and development of a AAV Gene Therapy for Alexander Disease. This has helped keep the project alive. Nevertheless, it may take years longer for this potential treatments to be ready for human use than we hoped.
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University of Wisconsin
To Prof Hagemann at the Alexander Disease Lab we donated US$47,389 to support research and development of potential therapeutics for Alexander Disease. While helpful, this is not enough to conduct all the experiments required.
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Western Sydney University
To Prof Gyengesi we donated AUD$47,250.00 (about US$29,777) to support a 3 year PhD Scholarship to research a potential therapeutic for Alexander Disease. This donation was matched 100% with University funds, doubling the impact. Unfortunately, bureaucratic hurdles have delayed the start of research by more than 1 year
Connecting Researchers, Empowering Discovery
In addition, with the help of Google Gemini, we connected with scientists from different fields and biotechnology companies, in the hope that they could help in our quest to end Alexander Disease. Since then, new brilliant and kind scientists have gotten involved in Alexander Disease (by alphabet), to varying degrees.
Prof Corina Amor (Cold Spring Harbour Laboratory)
Prof Pranam Chatterjee (Duke)
Prof Lenore Cowen (Tufts)
Prof Avi Ma’ayan (Mount Sinai)
Prof Alberto Serrano-Pozo and Ayush Noori (Harvard Medical School)
Prof Rohit Singh and Kapil Devkota, Ph.D. (Duke)
Prof Michele Vendruscolo (University of Cambridge)
Prof Marinka Zitnik, Dr Yasha Ektefa, Ayush Noori, Dr. Shanghua Gao (Harvard Medical School) with Dr Owen Queen (Stanford)
Dr Morgan Zhang (NYU)
Furthermore, we are grateful for the support of experienced scientists who have provided input or welcomed new collaborations, such as Prof Natasha Snider (UNC), Prof Mel Feany (Harvard Medical School), Prof Ming-Der Perng (National Tsing Hua University) and Prof Perez-Sala.
Last, but certainly not least, we are grateful for the leadership, support and tireless advice by Prof Albee Messing who has dedicated his career to understanding and finding therapeutics to Alexander Disease.
Make a donation
Support our mission by contributing today.
