Mitochondria Transplantation to Improve Transfusion-Dependent Low-Risk MDS 

New early-stage research suggests that restoring mitochondria may help improve blood cell production and slow disease progression in people with myelodysplastic syndromes (MDS). MDS is a group of blood cancers affecting how blood cells are formed in the bone marrow.

Low-risk MDS patients who no longer respond to standard treatments face challenges when trying to find a therapy that effectively controls their disease. Mitochondria transplantation is a new cell therapy proposed to address these challenges. 

The importance of mitochondria in MDS

Mitochondria are often called the “power plants” of cells because they produce energy needed for cells to function and grow. In MDS, mitochondria do not work properly. When this occurs, blood-forming stem cells struggle to mature into healthy blood cells. This may play a role in anemia and disease progression in MDS.

What is mitochondrial transplantation?

Mitochondrial transplantation is a process where a patient’s own blood-forming stem cells are collected and enriched with healthy mitochondria from a donor source. Then, the enhanced cells are returned to the patient.

Because the cells come from the patient, this is considered an autologous cell therapy, meaning it uses the patient’s own cells.

What did the preclinical studies show?

Researchers tested mitochondrial transplantation in a mouse that closely mimics human MDS. Mice that received mitochondria-enhanced stem cells showed longer overall survival, a lower risk of progressing to acute myeloid leukemia (AML), and importantly, no harmful effects on normal blood cells

How did human MDS cells respond in the laboratory?

When stem cells from patients with low-risk MDS were treated with healthy mitochondria in the lab:

• Red blood cell development increased about two-fold
• More mature red blood cells were produced
• Signs of improved erythroid (red cell) differentiation were confirmed

This suggests mitochondrial transplantation may directly help address anemia, one of the most common and burdensome symptoms of MDS.

A phase 1 clinical trial studying mitochondrial transplantation in MDS

A Phase 1 clinical trial (NCT06465160) is currently studying this therapy, called MNV-201, in adults with transfusion-dependent, low-risk MDS who no longer respond to approved treatments.

Early findings in the first five treated patients showed no serious treatment-related side effects and long-lasting transfusion independence for over 10 months. 

Why is this research important for patients with MDS?

This approach is different from traditional treatments. Instead of killing abnormal cells, it aims to restore healthy cell function by fixing the energy problem inside bone marrow stem cells.

Mitochondrial transplantation offers the possibility to:

• Reduce transfusion needs
• Improve quality of life
• Delay progression to leukemia

Mitochondrial transplantation is still experimental and only available through clinical trials. Larger studies are needed to confirm who benefits most and how long the effects last.

If you want to learn more about ongoing clinical trials, read more news, or explore our clinical trial finder, where you can use our filters to personalize your search. 

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Source:

• Therapeutic potential of mitochondrial transplantation in the treatment of MDS