Emerging therapies for rare maternal mitochondrial diseases with shared causes
Official title: Emerging therapeutic candidates for rare maternally inherited mitochondrial diseases with shared etiologies
Funded at George Washington University · NIH-11536511 · Data updated 2026-11-12
A basket clinical trial will test new drug candidates for two ultra-rare mitochondrial diseases (MELAS and LHON-Plus) that share the same underlying energy-making problem in people who could be affected.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | George Washington University NIH-funded |
| Lab location | 1 site (Washington, United States) |
| Project start | May 7, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $649,230 |
| NIH project number | 3UH3TR003897-05S1 |
| Project ID | NIH-11536511 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with MELAS or LHON-Plus who have the maternal mitochondrial DNA defect and related energy-deficiency symptoms.
✕Less likely to help
Patients without the specific mitochondrial energy deficit or who cannot participate in a basket trial design may not benefit.
What this research studies
This project aims to combine MELAS and LHON-Plus patient groups into one basket trial to see if new therapies can help improve energy production in cells and tissues. It focuses on drugs that target the shared defect in oxidative phosphorylation and ATP synthesis, hoping to show proof-of-concept for these approaches. The study will work with patients and/or patient-derived samples to evaluate safety and signals of efficacy in a feasible, collaborative framework. If successful, it could pave the way for larger trials and faster access to therapies for these devastating conditions.
Why it matters
Potential benefit: If successful, patients may gain access to new therapeutic options that improve energy production and reduce disability.
How similar studies have gone: Other rare-disease basket trials have shown feasibility in combining small, similar patient groups to test therapies.
Where this research is happening
-
George Washington University
Washington, United States Active
Researchers
- Principal investigator: Chiaramello, Anne Eliane — George Washington University
- Study coordinator: Chiaramello, Anne Eliane
Related research
Other NIH-funded projects on the same topics.
-
Drosophila models to study human mitochondrial diseases
A scientist at Harvard Medical School is creating fly-based tools to study how mitochondrial problems cause human diseases.
-
First MTATP6 mitochondrial disease mouse model and AAV-based gene therapy approach
A new grant aims to create a mouse model of MTATP6 mitochondrial disease and test an AAV-based gene therapy approach to treat it.
-
New treatments for maternally inherited mitochondrial diseases MELAS and LHON-Plus
New drug candidates aim to boost cellular energy for people with MELAS or LHON-Plus, rare maternally inherited mitochondrial diseases.
-
Developing a gene therapy for mitochondrial diseases using a mouse model
This study is working on a new way to help people with mitochondrial diseases caused by a specific gene problem by using a special virus to deliver a healthy version of that gene, which could lead to better treatments for those affected.
-
New treatments for rare mitochondrial diseases passed from mother to child
This study is looking for new treatment options for people with two rare inherited mitochondrial diseases, MELAS and LHON-Plus, which cause serious health issues, and it aims to see if a shared treatment can help both groups of patients feel better.
-
Using fruit flies to study human mitochondrial diseases
This study is looking at how mitochondrial diseases work by using fruit flies to learn more about the problems they cause, which can lead to muscle weakness and organ issues, and it aims to find new ways to detect and treat these conditions.
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.