Discovery could help address platelet shortage issues
CHARLOTTESVILLE, Va. (CBS19 NEWS) -- The American Red Cross has said the United States is suffering from a national blood crisis due to a shortage in the supply.
However, a new University of Virginia School of Medicine discovery may help ease the shortage of platelets.
According to a release, researchers have identified an enzyme that controls the production platelets or of platelet makers.
They say this discovery could also help treat a potentially dangerous clotting disorder called thrombocytopenia, which affect almost a third of newborns in intensive care, and cancer patients who need to get cord-blood transplants.
“Because of worsening shortages of donor-derived platelet units, there has been a big push within both public and private sectors for cell culture-based methods of generating platelets,” said Adam N. Goldfarb, MD, the chief of the UVA Health Division of Experimental Pathology. “In addition to alleviating platelet shortages, the cell culture approach affords the opportunity for creating ‘designer platelets.’ For example, platelets that do not elicit an immune response, which is a major problem in cancer patients.”
The release says the findings provide insights into platelet-producing cells called megakaryocytes, which are better at making more of themselves when they are “babies” and then produce platelets as “adults.”
When they are young, these types of cells appear to be more focused on building platelet-production factories, and then they begin to crank out platelets in time.
When they make the transition to producing platelets, the ability to create new megakaryocytes drops dramatically.
Goldfarb and the researchers learned they could toggle between the young and adult modes of these cells, making them either produce more of themselves or platelets at will.
To do so, they blocked an enzyme called Dyrk1a.
It is possible that this discovery could relieve a bottleneck in the production of platelets outside of the body that are then used for transfusion.
The release says cancer patients who get cord-blood transplants may be helped by overcoming platelet problems slowing the immune system’s recovery and raising the risk of contracting dangerous infections.
Cord-blood transplants are frequently performed on patients who cannot find a suitable donor for a stem-cell transplant, which is especially challenging for people of African, Asian, Hispanic, Middle Eastern, southern European or mixed ethnic backgrounds.
Regarding infants with thrombocytopenia, the body has too few platelets, which can lead to immune system problems, a bone marrow disorder such as leukemia, and other issues.
Premature babies are more likely to have an issue with this disorder. The more premature the baby is, the more severe the condition tends to be, with many premature infants needing platelet transfusions to help reduce the risk of uncontrolled bleeding inside their body.
The release says drugs to inhibit Dyrk1a already exist and are currently being evaluated to fight various diseases such as Alzheimer’s and diabetes.
Since these drugs are currently available, this could speed up the process to get to human trials testing the clinical benefits of controlling megakaryocyte production.
Goldfarb says this discovery could have far-reaching benefits.
“In the short term, we hope to improve the efficiency of donor-independent platelet production to the point where it could be scaled up for routine clinical use. In the long term, we hope to identify new patient treatments that could stimulate rapid platelet recovery,” he said. “Our findings offer a perfect example of how studying infantile versus adult cell development can yield clinical benefits.”
These findings have been published in the Journal of Clinical Investigation.