4. Positive results from targeted therapies
There are many medicines available to treat multiple myeloma, with chemotherapy and autologous stem cell transplants (when stem cells are collected from the patient) are the most common, but several of the most recent and exciting treatments to become available are two medications called daratumumab and ixazomib, and a form of treatment known as immunotherapy.
Darzalex (daratumumab): In November 2015, the FDA granted ‘accelerated approval’ for daratumumab injections in the treatment of multiple myeloma. The medicine may only be used by individuals who have already undergone at least three other types of therapy.
Darzalex is part of a category of medicine called monoclonal antibodies. It works by binding to a protein called CD38, which is typically found on the surface of myeloma cells. Once it is attached to the cell, the medicine attacks the cell while simultaneously signaling to the immune system to fight against the cells.
Almost one-third of clinical trial participants (with a median of five previous therapies) responded positively to the medicine.
Ninlaro (ixazomib): Recently approved by the FDA, this completely oral treatment is used in combination with standard myeloma drugs to treat people who have already undergone at least one previous therapy. Clinical study results showed that the drug taken in combination with lenalidomide and dexamethasone increased ‘progression-free survival (PFS) in patients with relapsed/refractory multiple myeloma’.
Immunotherapy: Immunotherapy is when a person’s immune system is used to treat an infection or disease. In a recent study, scientists discovered that 70 percent of people with multiple myeloma who were treated with immunotherapy had a ‘significant clinical response’ to the disease. In the study, 14 of the 20 participants with an advanced form of multiple myeloma had a ‘near-complete or complete response three months after treatment; median progression-free survival was 91.1 months, while the overall survival lasted 32.1 months’. Furthermore, no significant side effects were reported. This is an important advance, given that current treatments such as chemotherapy and autologous stem cell transplants have low long-term responses and an average survival rate between three and five years. *Update January 2018 – Empliciti (elotuzumab), is a type of immunotherapy that was approved in the US, Europe, and Australia, but has still not been approved in many other parts of the world and is therefore not directly available there.
Despite advancements in the treatment of multiple myeloma, not all medicines are available in the same countries at the same time. This can be due to delays in initial approval by one regulatory body and approvals within a certain country — if the manufacturer has filed for approval in that country. There is no global, harmonised approval system and it’s up to manufacturers to decide where to go to market first (also known as applying for market authorisation). Regulatory bodies also differ in speed, which can cause delays.
Almost any country in the world allows individuals to import elsewhere approved medicines for personal use, which may give multiple myeloma patients access to new-to-market medicines.
5. Tom Brokaw is living with the disease and has written a book about it
NBC News anchor Tom Brokaw was diagnosed with multiple myeloma in August 2013 following a bout of severe back pain. Though he originally wanted to keep the diagnosis private, he eventually announced his fight against the disease. His memoir, A Lucky Life Interrupted, was published last year and details his journey following his diagnosis. In it, Brokaw discusses the challenges he faced from the disease: weight loss, the inability to sometimes walk without help, the side effects from his medications, and the moment when he learned that the disease was affecting 60 percent of his blood. After 16 months of treatment, his cancer went into remission.
6. How it’s diagnosed
Several different diagnostic tests must be used to confirm a multiple myeloma diagnosis because it is challenging to diagnose based on a single laboratory result. A physical evaluation will be done alongside a review of the individual’s history, symptoms, blood and urine tests, and a bone marrow biopsy. Other tests might include an MRI, CT scan, PET scan and X-rays.
In order to definitively diagnose multiple myeloma, a person must meet at least one major and one minor or three minor criteria. Those criteria are:
- Plasmacytoma (based on a biopsy)
- The existence of 30 percent plasma cells in a bone marrow sample
- Increased levels of M protein in either blood or urine
- 10 percent to 30 percent plasma cells in a bone marrow sample
- Osteolytic lesions
- A minor elevation in M protein levels in blood or urine
- Low levels of antibodies (that are not produced by cancer cells) in the blood.
7. Stages and classifications
The criteria as discussed above helps doctors determine not only whether a person has the disease, but also under which classification the disease falls. Those classifications are:
- Monoclonal gammopathy of undetermined significance (MGUS)
- Asymptomatic myeloma (also called smoldering myeloma or indolent myeloma)
- Symptomatic myeloma.
Once the classification is known, a doctor will then determine which stage of the disease exists, which will help establish the prognosis and treatment options.
The most common way to diagnose the stage of the disease is through the International Staging System (ISS), which is based on two different blood test results: the beta 2-microglobulin (β2-M) and the albumin. There are three stages of classification under the ISS:
- Stage I: β2-M less than 3.5 mg/L and albumin greater than or equal to 3.5 gm/dL
- Stage II: Either β2-M greater than 3.5 mg/L but not greater than 5.5 mg/dL and/or albumin less than 3.5 g/dL
- Stage III: β2-M greater than 5.5 mg/L
The Durie-Salmon Staging System is an older system of diagnosis. This uses four measurements to determine which stage of the disease exists: 1) the amount of haemoglobin in the blood, 2) the amount of calcium in the blood, 3) the production rate of M protein, and 4) the number of bone lesions. The disease’s stage is then further subdivided based on kidney function.
The three stages of the disease as determined by the Durie-Salmon Staging System are: Stages I, II and III. Each of these stages is then subdivided into either Stage A or Stage B based on whether kidney function is affected. (Stage B means there is significant kidney damage.)
- Stage I: Though a person with Stage I often shows no symptoms of the disease because there are fewer cancer cells present in the body, other signs will be present, such as: amount of red blood cells within or a little below the normal range, a normal amount of calcium in the blood, low levels of M protein in the urine or blood.
- Stage II: More cancer cells are present in the body than in Stage I. An individual who does not fit into either Stage I or Stage III is said to have Stage II.
- Stage III: There are many cancer cells present. Other characteristics of this stage include; hypercalcemia, high levels of M protein, anaemia, and significant bone damage.
Note: In any stage, if kidney function is affected, the prognosis will be worse.