Targeting a brain enzyme (HDAC11) to treat Alzheimer's
New epigenetic inhibitors for Alzheimer's disease treatment
['FUNDING_R01'] · MASSACHUSETTS GENERAL HOSPITAL · NIH-11296896
Researchers are creating new drugs that block a specific brain enzyme called HDAC11 to try to slow or reverse Alzheimer's disease in people with AD-related brain changes.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MASSACHUSETTS GENERAL HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11296896 on ClinicalTrials.gov |
What this research studies
From a patient's point of view, the team will design and refine small-molecule drugs that specifically block the HDAC11 enzyme. They will test these molecules in cells and animal models that mimic Alzheimer's pathology to find ones that reduce disease markers and improve brain function. Promising compounds will be optimized for how the body absorbs and handles them (PK/PD) and for safety. The best candidates will be prepared for the next step toward first-in-human (IND-enabling) studies.
Who could benefit from this research
Good fit: Future clinical trials would likely enroll people diagnosed with Alzheimer's disease, especially those in earlier stages or with measurable amyloid-related changes.
Not a fit: People with non‑Alzheimer's dementias, very advanced Alzheimer's, or unrelated neurological conditions would be unlikely to benefit from these early drug candidates.
Why it matters
Potential benefit: If successful, this work could produce a new type of treatment that slows or reverses cognitive decline in people with Alzheimer's disease.
How similar studies have performed: Broad HDAC inhibitors have shown benefit in animal models but have not yet led to effective patient treatments, and HDAC11-selective inhibitors represent a newer and still largely untested approach.
Where this research is happening
BOSTON, UNITED STATES
- MASSACHUSETTS GENERAL HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WANG, CHANGNING — MASSACHUSETTS GENERAL HOSPITAL
- Study coordinator: WANG, CHANGNING
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.