Therapeutic potential of mitochondrial transplantation in the treatment of MDS

Myelodysplastic syndrome (MDS) is a disease where the blood cells in the bone marrow do not mature, and as a result, do not develop into healthy blood cells. Studies show that patients with MDS have dysfunctional mitochondria and that these diseased mitochondria affect the severity of MDS. Understanding and treating this disease is important as some patients with MDS may later develop acute myeloid leukemia.

Currently, the only cure for patients with MDS is bone marrow transplant, however, this treatment is not a good option for a majority patients. Omar's project seeks to understand the role that a new technology, mitochondrial augmentation, has in treating patients with early and low-risk but unresponsive MDS.

The therapy works like this: A patient’s bone marrow stem cells are collected and enriched with mitochondria from healthy donor sources such as the stem cells from placenta. The patient stem cells uptake the healthy mitochondria, becoming augmented. These augmented cells are then infused back into the patient. Initial clinical trials showed that patients who received mitochondrial augmentation treatment saw promising safety and clinical benefit.

In the upcoming year, Omar will be working in the Abdel-Wahab lab at Memorial Sloan Kettering, hoping to contribute to this ground-breaking work and improve treatment options for future patients!