What is Multiple Myeloma?
Multiple myeloma (MM) is a plasma cell malignancy characterized by a monoclonal expansion of malignant plasma cells in the bone marrow, which produce an immunoglobulin, called a monoclonal spike.
MM accounts for about 10% of all hematologic cancers and is responsible for 2 percent of all cancer-related deaths in western countries.
MM is characterized by a molecular heterogeneity, which impacts response to treatments and overall survival.
Despite significant advances in treatments, Multiple Myeloma remains incurable and needs novel therapeutic approaches.
Symptoms of Multiple Myeloma
Multiple Myeloma usually evolves from an asymptomatic premalignant stage termed MGUS (Monoclonal Gammopathy of Undetermined Significance) present in more than 3% of the population above the age of 50. It progresses to Multiple Myeloma at a rate of 1% per year.
Clinical signs and symptoms of Multiple Myeloma are an accumulation of high levels of monoclonal immunoglobulins in blood and/or urine, recurrent bacterial infections, anemia, osteolytic lesions, and renal insufficiency.
Treatment for Multiple Myeloma
Over the last decade, the treatment pattern for Multiple myeloma has changed with the use of autologous stem cell transplantation (ASCT) and the introduction of novel therapeutic agents such as proteasome inhibitors (Bortezomib and Carfilzomib) and immunomodulatory drugs (IMIDs: Thalidomide, Lenalidomide and Pomalidomide). The incorporation of these novel drugs has significantly prolonged the survival of newly diagnosed and relapsed multiple myeloma patients.
In spite of significant progress in understanding the biology of Multiple Myeloma and prognostic criteria, the choice of treatment is still based primarily on a patient’s age and their comorbidities. The main treatment today for young, fit Myeloma patients (under 65 years of age) consists of induction therapy with different combinations of proteasome inhibitors with IMIDs, corticosteroids (mostly Dexamethasone) and/or anti-mitotic agents (cyclophosphamide or doxorubicin), followed by high-dose Melphalan treatment, and ASCT. Followed by consolidation and/or maintenance therapy composed mainly of proteasome inhibitors- or a IMIDs-based regimen which are used to improve response and increase time of remission. For patients ineligible for transplantation, Bortezomib-Melphalan-Prednisone or Bortezomib-Prednisone-Thalidomide combination regimens are the standard-of-care.
Despite such advances a major problem in Multiple Myeloma treatment is the acquired or intrinsic drug resistance against current standard-of-care agents that leads to eventual relapse and fatal outcomes in the vast majority of patients.
Multiple Myeloma Research
Each year worldwide more than 115,000 people are newly diagnosed with Multiple Myeloma, a plasma cell malignancy. Recent advances in treatment mean that survival rates have significantly improved to between 5 and 9 years. However with time patients relapse as treatments fail to work over the long term meaning that for most patients the disease remains incurable. Over 220 people still die each day from Multiple Myeloma.
New treatments must be fully researched to be sure they work better than the treatments already in use and to know they are safe. For ethical and safety reasons treatments are first tested in the laboratory, for example here at Myelomax, before they can be tested in patients during clinical trials. Often it is the case that researchers look at drugs (chemotherapy, proteasome inhibitors, IMIDs, corticosteroids) in different combinations and at different myeloma subgroups.
Further research is required because as Multiple Myeloma progresses the biology of the disease changes, and so treatments must be carefully planned as treatment options because increasingly limited.
Consequently there is a real unmet need for more effective therapies and treatment strategies that can provide long term responses in patients at all stages of their disease in particular for those with relapsed or refractory disease.