As 2023 draws to a close, we are filled with a feeling of excitement. Thanks to your support, this year has been one of remarkable progress in our quest to find a cure for ADLD.
While we are very thankful for the $120,000 we have raised this year, we are $200,000 short of our goal to get the Natural History Study off the ground. Please watch the video, read below and most importantly, please donate now so we can continue this important work.
Milestones Achieved: A Year in Review and Next Steps
- Initiated the Discovery Process for an ADLD Drug: We’re working with strategic partners to discover a unique drug for ADLD. Currently, potential drug candidates are undergoing testing, which will be followed by a potential pilot trial with a single patient.
- If successful, we will approach regulators and initiate the process for a broader trial. This will require guidance and dedicated time from many experts.
- Advanced Drug Repurposing Studies: We kicked-off an effort to understand if existing, FDA-approved drugs can be repurposed to treat ADLD. An initial screen was done in cells that were engineered to mimic ADLD.
- Another screen, which we expect to be more informative, will be done on cells that were derived from patients. We expect to have results of that screen by mid-2024.
- Planned a Natural History Study: We’re working with world-class healthcare organizations to initiate a natural history which will be used to study ADLD progression across as many patients as possible. If we’re able to get enough participants, when we begin a clinical trial in the future, regulators may be able to use the natural history study as a control group, which means everyone who participates in the clinical trial could get the drug. In most circumstances, the FDA requires a placebo.
- In 2024, we will begin recruiting patients for this study and processing their data. In addition to time from patients, this will require continuous support from many clinical sites, clinical coordinators, physician partners, and beyond.
- Funded three pilot research grants: In partnership with the Orphan Disease Center at the University of Pennsylvania, we awarded $50,000 each to three grantees.
- Dr. Quasar Padiath at UPitt who is working towards understanding which specific processes lead to the demyelination in ADLD.
- Dr. Stefano Ratti at the University of Bologna who is working towards building 3D organoid models of ADLD.
- Dr. Margot Cousin at the Mayo Clinic who is working towards preparing for a clinical trial for ADLD.
- We will continue to identify priority research areas and fuel innovative discoveries that will help better understand how ADLD works and what we can do to stop it.
- Created a comprehensive ADLD Handbook: This valuable resource provides essential information and support for individuals and families navigating their journey with ADLD (available at adld.center)
- Fostered Scientific and Clinical Expert Collaboration: A consortium of ADLD-knowledgeable physicians have drafted a standard of care protocol for other physicians, which is expected to be finalized in early 2024. We will continue this work so every ADLD patient can get the best care possible.
- Enriching Community Engagement: Our events and the expansion of the ADLD Research Community have woven a stronger support network for researchers, patients and caregivers.
The roadmap for the upcoming year is ambitious, reflecting our commitment to you and the wider ADLD community. With several key projects on the horizon, including execution of a natural history study, groundwork towards potential clinical trials and the creation of an ADLD Center of Excellence, we are hopeful for a breakthrough in the near future.
View image
While we are very thankful for the $120,000 we have raised this year, we are $200,000 short of our goal to get the Natural History Study off the ground.
Please donate now.
Your support is critical to our success. Every contribution, no matter how big or small, brings us closer to finding a cure for ADLD. This year-end, please consider making a generous donation to fuel the above programs.
Together, we can make a real difference in 2024!
With heartfelt thanks,
ADLD Center Team
View image