Year of Grant: 2013

Location: Switzerland

Myeloproliferative neoplasms (MPN) is a type of blood cancer in which patients produce too many blood cells. These increased blood cell numbers cause problems to the patient such as bleedings or thrombosis and some patients may progress to acute leukemia, a life threatening condition. There is currently no cure available for patients suffering from MPN.

Most MPN patients (>80% overall) have a gene mutation called JAK2-V617F. The disease is maintained by mutant MPN stem cells that reside in the bone marrow in specialized locations called “niches”. These niches need connections to the nervous system. New findings show that these connections are destroyed by the presence of the mutated MPN stem cells.

This phase II study will test the effects of the beta-3-sympathicomimetic drug Mirabegron (Betmiga®) on MPN disease in 36 patients that carry a JAK2-V617F mutation. Betmiga is currently approved for the treatment of hyperactive bladder and have shown to have only few side effects.

The researchers expect that Mirabegron will have a beneficial effect on bone marrow niche cells and will thereby improve the disease manifestation in MPN patients.

Once completed, the study should provide a rapid answer whether targeting the nervous system of the niche cells could be useful for patients with MPN and warrants to be tested in larger and more long-term studies. It could also potentially create a novel treatment option with reasonable chance for clinical activity for these patients without alternative therapy option.