Adult-onset Autosomal Dominant Leukodystrophy (ADLD) Pilot Grants

The ADLD Center, in collaboration with Orphan Disease Center, is seeking grant applications for multidisciplinary teams of scientists that aim to further progress our understanding of ADLD,  the available therapeutic options, and investigating strategies to establish outcome measurements.

The RFA could focus on one, or several, of the following aims to further advance ADLD research and therapeutic approaches:

1. Identification of short-term biomarkers that can monitor disease activity and treatment response.

2. Establishment of outcome measures for future clinical trials.

3. Development of therapeutic approaches in early symptomatic patients.

4. Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.

5. Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.

6. Development of cellular models (i.e., oligodendrocytes) for evaluation of therapeutic options to translate for clinical use.

7. Evaluate pre-clinical patients (MRIs, genetic testing).

Funding: $50,000

Duration: 1 year

Research Authority due date: 5.1.23

LOI (required) due date: 9.1.23

Full proposal due date: 24.2.23