Myeloproliferative neoplasms (MPN) are a rare group of disorders of the bone marrow that cause an increase in the number of blood cells. They are also called myeloproliferative disorders. MPNs includes: polycythaemia vera (PV), essential thrombocythaemia (ET) and myelofibrosis (MF).
People with PV produce too many red blood cells, causing the blood to become thicker than normal. ET occurs when there are too many platelets in the blood, which can cause the blood to clot. People with MF have an overactive bone marrow, which develops scar tissue (known as fibrosis). The scar tissue builds up inside the bone marrow and blood cells can’t develop properly causing other complications including anaemia.
Researchers want to see if two drugs called azacitidine and ruxolitinib can help people with MPN. Both drugs are already used on their own to treat this group of people, but the trial wants to test how well they work when given together.
Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.
Ruxolitinib is a JAK inhibitor, and works by blocking the abnormal JAK signalling associated with changes to the JAK2 gene found in blood cells in people with MPN. By inhibiting this pathway, ruxolitinib can stop cancer cells from growing and dividing.
The aims of the trial are to:
- Find a safe dose of azactidine and ruxolitinib to have together
- See how well treatment works
- Find out more about the side effects
You may be eligible to join this trial if:
- You have a myeloproliferative neoplasm that includes essential thrombocythaemia, polycythaemia vera or myelofibrosis and you have a lot of immature cells called blasts in your blood and bone marrow
- You need treatment for your blood disorder
- You are at least 16 years old