Researchers have now uncovered a new approach for treating thrombocytopenia, a condition with a low blood platelet count.
Blood platelets are like the sandbags of the body. Got a cut? Platelets pile in to clog the hole and stop the bleeding.
But genetic mutations, infections and even radiation from cancer treatments can slash platelet numbers, leading to a condition called thrombocytopenia and putting people at risk for internal bleeding.
Researchers at Scripps Research found an enzyme that can boost platelet production and may work as a future therapeutic.
The study is the first to show that YRSACT is the key to one method of platelet production. Scientists worked with a platelet-deficient mouse model. Animals injected with YRSACT showed a dramatic increase in platelet production, especially under stressful conditions, such as radiation similar to what cancer patients face.
So how does YRSACT work? The researchers found that YRSACT increases the production of large bone marrow cells called megakaryocytes, which are the precursors to platelets.
Until this discovery, thrombopoietin (TPO) was previously the only other protein known to increase platelets. Schimmel says a version of TPO is currently used as a drug to treat some cases of thrombocytopenia. However, TPO has limitations, making it unsuitable and hazardous in some clinical settings.
Showing that YRSACT could be useful in human patients posed its own challenges. Congenital thrombocytopenia is rare, and even finding the right blood cells to test in thrombocytopenia patients is like finding a needle in a haystack.
The findings appeared in the Journal of Proceedings of the National Academy of Sciences.