Novel treatments
Pro-survival inhibitors
As the name suggests, pro-survival inhibitors work by preventing the action of proteins whose usual function is to keep myeloma cells alive. BCL-2 is one such protein that is expressed at high levels by myeloma cells and ensures their survival by inhibiting cell death. Venetoclax (Venclyxto®) is a drug currently in late phase trials in the UK which inhibits the action of BCL-2, leading to myeloma cell death.
It is administered orally and has most promise as a combination treatment with for example, dexamethasone and bortezomib as demonstrated in the BELLINI trial. Due to early data looking at risk/benefit, it is currently only being developed for use in myeloma patients with t(11;14) myeloma or high expressors of BCL-2.
Side effects of venetoclax treatment can be serious and include diarrhoea, nausea, fatigue, neutropenia leading to an increased risk of infections, thrombocytopenia and anaemia.