The cells of children who have faulty mitochondria could be rejuvenated by a gift from their mothers. A research team is testing a strategy that involves soaking patients' blood cells in a large amount of healthy mitochondria from their mothers. There are early signs that the intervention is safe and may improve the children's health and development.
"This approach is different from what anyone else is doing," says Mary Kay Koenig, a child neurologist at the University of Texas Health Science Center, Houston, who wasn't involved in the study. She says that the results are very exciting. There is a clinical neurologist at the University College London Queen Square Institute of Neurology. The data is very preliminary.
Most of the adenosine triphosphate (ATP) that fuels cells comes from the Mitochondria, which originated early in the evolution of the organisms. About one in 5000 babies are born with some sort of defect.
When isolated mitochondria are mixed with cells, the organelles begin to work. The researchers realized they could use this behavior to increase the number of healthy mitochondria.
Hematopoietic stem and progenitor cells are found in bone marrow and give rise to a variety of blood cells. The dispersal of HSPCs may suppress disease effects in other tissues.
There is a regulatory path for testing experimental approaches in people with untreatable illnesses under compassionate use. The children's cells were working on a low battery which resulted in many problems. They were shorter than almost all of their peers due to impaired growth.
The researchers mixed the healthy mitochondria from the patients with the healthy ones from the mother. Shake it and put them in a tube. The cells were returned to the patient after 24 hours.
Some of the cells had absorbed the part of the body. After reinfusion, the patients' blood cells held 30% moreMitochondrial DNA and produced 1/3 moreATP than before. Two patients showed improvements after being tested for strength and endurance. One child who underwent the treatment five years ago is still alive.
She believes that they are showing some improvements. It's hard to confirm the treatment was responsible. Scientists don't understand how the symptoms of the diseases evolve as children age because the study didn't have a control group to compare it to. Every patient is supposed to decline according to Koenig.
He doesn't believe that cells with faulty mitochondria will have much of an impact outside the blood.
The results of a clinical trial of the treatment on five more patients with the two syndromes will be analyzed by the researchers and company they founded to commercialise their work. There are a lot of mitochondrial disorders that this could work for.
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