Myelofibrosis is a cancer of the bone marrow in which the marrow is replaced by scar tissue. The early stages of myelofibrosis may be asymptomatic in some people while others may have severe symptoms from the onset. As the bone marrow becomes more scarred, it is less able to produce blood cells leading to enlargement of spleen and liver. Enlargement of spleen may cause abdominal pain, shortness of breath, early satiety and faecal incontinence, along with progressive anaemia. Other symptoms include incurable itch, general malaise, weight loss, night sweats, low grade fever, anaemia, fatigue, and pallor. Many people with myelofibrosis have mutations in a gene known as JAK2 gene.
Fedratinib is in development for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis, including patients who have been previously exposed with ruxolitinib. Fedratinib is an inhibitor of JAK2 with potential to kill cancer cells. Fedratinib is given by mouth as capsule. If licensed fedratinib will offer an additional option for the treatment for patients with myelofibrosis.
Nivolumab works by improving the activity of white blood cells thereby increasing the ability of the immune system to kill cancer cells. Cabozantinib works to stop signals that cancer cells use to divide and grow. It is thought that when used in combination, both drugs may be more effective than each drug on its own. If licenced, nivolumab in combination with cabozantinib may improve long-term outcomes in mRCC patients who currently have limited treatment options.