Multiple myeloma (MM) is a rare, incurable cancer of the plasma cells in the bone marrow. Bone marrow is the spongy tissue found at the centre of some bones, which produces blood cells for the body. Plasma cells are normally produced in a controlled way but in cases of MM, large amounts of abnormal plasma cells are produced. These fill the bone marrow and interfere with the production of other cells, including red and white blood cells and platelets. The cause of MM is unknown. Symptoms of MM varies but some may include bone pain, fractures, body weakness, malaise, bleeding, anaemia and infections. People with MM will experience periods of time without symptoms followed by periods when the illness comes back (‘relapsed’ MM). Eventually the periods without symptoms will shorten and the illness will become immune to the drugs given to treat it (‘refractory’ MM).
bb2121 is in development as a treatment option for relapsed and refractory MM. It is based on genetic therapies and targets the growth of specific proteins present in most MM cells. bb2121 is administered by injection and the unique way it acts may offer an additional treatment option for relapsed and refractory MM patients who have tried and failed to respond on current therapies.
Selinexor is the first treatment option that targets XPO1, a protein that is responsible for exporting tumour suppressor proteins from the cell nucleus. It belongs to a new family of therapies called selective inhibition of nuclear export (SINE) compounds that blocks XPO1 leading to controlled death of myeloma cells. Currently there is no standard of care for the fifth line treatment of MM. Selinexor and low-dose dexamethasone are being developed as an oral treatment. If licensed, this combination could be an effective treatment option for a patient group with clear unmet need.