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Exploring the cause of the blood cancer myelofibrosis

  • Researcher: Dr Golam Mohi
  • Institution: University of Virginia, Charlottesville, USA
  • Award Amount: £202,500 for 3 years from 1st June 2015
  • Cancer Type: Leukaemia
Exploring the cause of the blood cancer myelofibrosis
Myelofibrosis (MF) is a devastating blood cancer and is still not fully understood at the molecular level.  It’s characterized by abnormal scar tissues in the bone marrow, low levels of red blood cells (anemia) and enlargement of the spleen. Other associated symptoms include tiredness, itching, bleeding and bone pain. Sadly, the overall survival for MF patients is less than 6 years.A single mutation (V617F) in a gene called JAK2 has been found in half the cases of MF. Additional mutations in other genes called CALR, MPL, TET2, AXL1 and EZH2 have also been identified. However, the contribution of these mutations in the development of MF remains unclear.Dr Golam Mohi explains “Currently, there is no cure for MF. Ruxolitinib, a drug that works by blocking both JAK1 and JAK2, has been approved for the treatment of MF. Although Ruxolitinib can reduce the spleen size and other associated symptoms of MF, it failed to produce any remission in MF patients. Moreover, patients treated with the drug had toxic effects on their blood cells. Understanding MF at the molecular level is therefore critical for the development of new therapies.” He continued “In our initial studies, we found that removing the EZH2 gene accelerates the development of MF in mice with the V617F mutation. We are now further investigating the role of EZH2 in MF and hope to identify targets of EZH2 that are required to induce MF. Results from these studies will provide new insights into the mechanisms of MF and should identify new therapeutic targets for the future.” When asked about the grant he told us "Our ultimate goal is to find a cure for the deadly blood cancer myelofibrosis. A research grant from the Worldwide Cancer Research will be extremely helpful in reaching that goal."
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