Advances in medicine never cease to amaze. Last Wednesday (12/21), a medical team from Israel published in the journal Science Translational Medicine a successful report on an unprecedented procedure for transplanting mitochondria in children. The new surgery is capable of helping patients with mitochondrial DNA depletion disorder, a currently incurable disease.
Generally, the condition manifests itself during the second decade of the patient’s life with progressive multisystemic failures. Problems include hearing and vision loss, muscle weakness, heart and gastrointestinal problems, as well as dementia and early death.
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The new transplant involves augmenting the patients’ hematopoietic stem cells – the units that give rise to all the different types of blood cells – with healthy mitochondria donated by their mothers. The surgery, which has already been performed on six children, proved to be safe, in addition to reducing symptoms.
the transplant
To perform the transplant, doctors collect hematopoietic stem cells from the sick child’s bone marrow and the mother’s blood. Scientists open the walls of white blood cells and use a centrifuge to separate the mitochondria from the cytoplasm.
They then trick the patient’s stem cells into taking up the mitochondria through a natural process called pinocytosis. Once the cells are enriched with the extra mitochondria, the units are infused back into the child.
The patients who had already received the transplant were extremely weak compared to other healthy children. One of the participants, who arrived at the hospital weakened, being pushed in a cart, managed to run and put on weight a few weeks after the procedure. Another girl, who was bedridden and unable to remain seated, managed to stand up.
New clinical trials
The mitochondria transplantation method is still being tested. A new clinical trial is planned for 2023 and, if the results remain positive, the therapy could provide a new way to treat not only rare genetic diseases, but also other conditions and injuries resulting from mitochondrial damage.
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