Using mitochondrial transfer to treat motor neuron degeneration in SBMA

Mitochondrial Transfer to Treat SBMA Motor Neurons

NIH-funded research Massachusetts General Hospital · NIH-10985401

This study is exploring a new way to help people with spinal bulbar muscular atrophy (SBMA) by using special cells to deliver healthy mitochondria to the damaged nerve cells, with the hope of improving their health and function.

Quick facts

Grant typeR21 grant
Study typeNIH-funded research
Funding institutionMassachusetts General Hospital NIH-funded
Lab location1 site (Boston, United States)
Project IDNIH-10985401 on NIH RePORTER

What this research studies

This research investigates the use of mitochondrial transfer as a potential treatment for spinal bulbar muscular atrophy (SBMA), a condition characterized by motor neuron degeneration. The approach involves developing human neural stem cell models to isolate extracellular vesicles that contain mitochondria, which are then transferred to affected motor neurons. By examining how these transferred mitochondria affect the health and function of the motor neurons, the research aims to establish a new therapeutic strategy for SBMA and similar neurodegenerative disorders.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with spinal bulbar muscular atrophy (SBMA) who are experiencing motor neuron degeneration.

Not a fit: Patients with other types of motor neuron diseases or those without mitochondrial dysfunction may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to innovative treatments that improve motor neuron health and function in patients with SBMA.

How similar studies have performed: While mitochondrial transfer is a novel approach, preliminary studies have shown promise in similar neurodegenerative conditions, suggesting potential for success.

Where this research is happening

Boston, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.