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New Mitochondrial Transplant Research Offers Hope for Children with Rare Genetic Diseases

Researchers at Sheba have reported promising results following the first-ever mitochondrial transplants in children with rare genetic diseases. In a groundbreaking study published in Science Translational Medicine, six patients showed small yet significant improvements in their symptoms after receiving healthy mitochondria from their mothers.

Mitochondria play a crucial role in the functioning of nearly every cell in the human body, providing energy and metabolizing cholesterol while synthesizing hormones and neurotransmitters. When an individual has a mitochondrial deletion disorder, such as SLSMDs, they suffer from progressive multisystem failures. These can lead to hearing and vision loss, muscle weakness, gastrointestinal and cardiac issues, dementia and early death.

Led by Dr. Elad Jacoby, a pediatric hematologist at Sheba, the Israeli team of doctors and researchers have been working on mitochondrial augmentation therapy for the past seven years. The treatment involves augmenting a patient’s hematopoietic stem cells with healthy mitochondria donated by their mother. This innovative approach was tested in six children and proved safe, with improvements observed in their quality of life.

Mitochondrial transplantation could potentially provide a way to treat not only rare genetic diseases but also a long list of other conditions and injuries arising from mitochondrial damage. The positive results of this study mark a significant step forward in the field of mitochondrial transplantation.

Sheba is renowned for specializing in Pearson syndrome and other mitochondrial diseases. The medical center has been working closely with Minovia, an Israeli biotech company focused on developing mitochondria-based medicines, to push forward the idea of using mitochondria to treat such disorders.

In collaboration with Minovia, Sheba is planning to launch a new trial in 2023 using an off-the-shelf version of the treatment, which involves hematopoietic stem cells enriched with mitochondria extracted from donated placentas. The medical center is also seeking to expand its clinical and research collaborations into the United States, with a manufacturing facility set to open outside of Boston next year.

While many questions remain about the treatment’s long-term effects and dosing, the early results from Sheba’s research offer hope to patients with rare genetic diseases and highlight the potential of mitochondrial transplantation as a revolutionary therapy for various conditions.

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