Updated April 2026
Research Roadmap
Our strategic priorities through 2027: clinical trials, novel drug development, and disease tracking running in parallel.
Updated April 2026
Our strategic priorities through 2027: clinical trials, novel drug development, and disease tracking running in parallel.
The ADLD Center runs five parallel programs aimed at the same goal: a treatment, and eventually a cure, for Autosomal Dominant Leukodystrophy. Clinical trials, novel drug development, and the Natural History Study all feed into one another, so progress on any one track accelerates the others.
Timeline at a Glance
Timeline at a Glance
Antisense oligonucleotides are synthetic molecules designed to silence the overexpressed LMNB1 gene that causes ADLD. Our experimental ASO trial at Mayo Clinic is the first therapeutic intervention ever attempted for this disease, and our work through 2026 focuses on deepening the evidence base and evaluating additional patients over time.
Completed
Preclinical Validation
ASO efficacy confirmed in mouse models
Underway
Experimental ASO Trial: Active
Intrathecal dosing and safety monitoring at Mayo
Next
Expanding Enrollment
Evaluate additional patients as trial matures
A global, remote study of ADLD patients tracking symptoms, imaging, and blood samples over time. The dataset serves as a synthetic control arm for future trials, and the blood samples power our biomarker program so we can measure, in real time, whether a treatment is working.
Live
Global Patient Registry
35+ patients consented across multiple countries, aiming for 50+
Underway
Longitudinal Data and Biosamples
MRI, functional assessments, and blood collection for the miRNA biomarker panel
Goal
FDA-Ready Endpoints
Synthetic control arm and validated biomarker panel to support trial endpoints
A parallel genetic approach uses short hairpin RNA delivered by adeno-associated virus to reduce LMNB1 expression in brain cells. This program is designed as a second shot on goal, so that if one approach stalls, another is ready to move forward.
Completed
Lead Candidates Selected
Two oligonucleotide constructs advanced from screening
Underway
In Vitro Knockdown Testing
Cell-line assays and off-target analysis
Next
In Vivo Efficacy Testing
Transition to patient-derived cells and in vivo studies
The fastest path to an approved treatment often starts with drugs that are already FDA-approved for other diseases. The ADLD Center pursues two complementary repurposing strategies: partner-led functional screening of compounds in ADLD cell models, and targeted repurposing based on shared disease biology.
Completed
Functional Screening Platform
Past compound screens with a rare-disease drug-repurposing partner on engineered oligodendrocyte models surfaced candidates that normalize the LMNB1 disease signature.
Underway
Targeted Repurposing
Farnesyltransferase inhibitors, already FDA-approved for Progeria, reduce Lamin B1 in ADLD cell models; additional ongoing screens at academic collaborator labs in the US and Italy.
Next
Preclinical Validation
Advance the most promising candidates into preclinical animal studies in 2026
Beyond our in-house programs, we are in active conversations with biotech companies whose platforms could be rapidly adapted to ADLD. Each candidate partnership is evaluated for mechanism, delivery, and how quickly it could reach patients.
Completed
Partner Identification
Three candidate platforms mapped across remyelination, siRNA, and lamin biology
Underway
Technical and Regulatory Diligence
Reviewing published safety data and fit with our preclinical pipeline
Next
Partner-Specific Pilot Studies
Move the most ready candidate into a joint preclinical study in 2026-2027
Community Research Studies
One-on-one interviews with ADLD patients and family members exploring experiences and considerations around reproductive options. Led by Dr. Quasar Padiath at the University of Pittsburgh.
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