Research

At MitoCure, we believe that funding cutting-edge research is key to unlocking breakthroughs for mitochondrial disease patients. We proudly support research that advances new treatments for mitochondrial disease, including efforts highlighted below.

Together, we’re changing the future of mitochondrial disease.

Tackling Mitochondrial Damage in the Eye: A New Frontier in Transplantation Therapy

MitoCure is proud to support a groundbreaking research collaboration between Mass Eye and Ear and Boston Children’s Hospital, focused on developing a novel mitochondrial transplantation (MT) approach to treat retinal damage caused by mitochondrial diseases like MELAS, KSS, and LHON.MT is a promising and emerging technology with remarkable potential. This innovative approach serves as a valuable model for drug screening, both in vitro and in vivo, specifically addressing mitochondrial dysfunction.

In these conditions, retinal ischemia — or inadequate blood flow to the eye — can lead to excessive production of toxic molecules known as reactive oxygen species (ROS). This results in significant oxidative damage during reperfusion, when blood flow returns to the tissue, causing further cell death and vision loss.

Researchers Demetrios Vavvas, MD/PhD, and Maria LosCertales, PhD, of Mass Eye and Ear (affiliated with Harvard Medical School and Mass General Brigham), together with James McCully, PhD, and Aybuke Çelik, PhD, of Boston Children’s Hospital, are refining and validating an innovative procedure for mitochondrial transplantation (MT). Their research is exploring how transplanting healthy mitochondria into damaged eye tissue may protect cells, reduce injury, and promote recovery. In tandem, the team is testing a synthetic, mitochondria-targeted drug (PMX500FI) to enhance these protective effects.

This promising work not only holds potential for treating retinal disorders but also offers insight into protecting other high-energy organs — including the brain and heart — from mitochondrial dysfunction and oxidative stress.

See more here.

Advancing Hope Through Research: Dr. B. Rita Alevriadou and the University at Buffalo

We are proud to support the innovative work of Dr. B. Rita Alevriadou and her team at the University at Buffalo – SUNY, whose research is paving the way for new treatment possibilities for individuals living with MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes) and other mitochondrial diseases.

Dr. Alevriadou’s lab is focused on the role of vascular endothelial cells (ECs) — the cells that line our blood vessels — and how mitochondrial dysfunction in these cells contributes to inflammation and stroke-like episodes in MELAS patients. One of the lab’s most promising areas of exploration is the use of extracellular vesicles (EVs) as a potential therapeutic tool. These tiny particles, naturally released by cells, can carry and transfer mitochondria between cells — offering the potential to restore energy function in damaged or dysfunctional cells.

The team’s goal is to harness this natural cellular mechanism to deliver healthy mitochondria to MELAS-affected cells, potentially improving vascular function and reducing symptoms. This groundbreaking work is still in the laboratory phase, but it represents a bold step forward in our collective effort to find a cure.

We are deeply grateful for Dr. Alevriadou’s leadership, vision, and dedication — and for all the researchers who are working tirelessly to improve the lives of those affected by mitochondrial disease.