Multiple myeloma is an incurable cancer characterized by a proliferation of plasma cells in the bone marrow. The disease is associated with substantial morbidity and mortality, and predominantly affects older patients. There is an unmet need for more accessible treatments.
Multiple myeloma is an incurable malignant plasma cell disorder, characterized by clonal proliferation of plasma cells in the bone marrow and the production of excessive amounts of immunoglobulin. The disease predominantly affects older patients, with a median age for onset of disease of 72 years. These patients have often many comorbidities and consequently receive multiple concomitant medications.
Most patients with multiple myeloma have symptoms that reduce quality of life, including bone pain, fatigue, anemia, and infections. Patients may have symptom-free periods, but the disease always relapses, and become refractory to all available treatment options due to new mutations of the tumor cells.
Incidence of Multiple Myeloma
Multiple myeloma is the second most common hematologic disease, and accounts for approximately 1–2% of all new cancer cases, with a global incidence of 1.7 per 100,000 and an incidence of 2.1–3.4 per 100,000 in France, Germany, Italy, Spain and the UK. An estimated 40,000 patients were diagnosed in the EU in 2020, with an estimated 23,500 deaths due to the disease. Multiple myeloma is more common in men than in women.
Unmet Medical Need
The introduction of new drugs during the last decade has improved treatment outcomes. However, multiple myeloma is ultimately fatal, with a 5-year survival around 50 %. Patients with relapsed disease can respond to subsequent therapies, but the duration of response decreases with successive relapses until resistance is developed.
Treatment of relapsed, refractory multiple myeloma (RRMM) is challenging, since patients have continuing symptoms, complications, and decreased quality of life. The patients receive a therapy until the next relapse, progression or the development of intolerable toxicity and then move on to the next option.
Treatment of Multiple Myeloma
The treatment goal is ultimately to control disease progression and prolong survival. The major drug classes include steroids, alkylators, proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies. Recently, three new classes have been approved for use in triple class refractory patients: a BCMA-targeted antibody, a selective inhibitor of nuclear export, and an anti-BCMA CAR-T cell therapy.
Triple-class refractory patients have a disease that is refractory to immunomodulatory drugs, proteasome inhibitors, and CD38-targeting monoclonal antibodies. The patients have often been exposed to all major drug classes. The patients have a poor prognosis with an expected overall survival of just a few months.
None of the newly approved medicinal products has provided a cure. Ultimately patients relapse and treatment options are exhausted. There is a continued unmet need, particularly in elderly patients who require accessible treatment options.