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The Multiple Myeloma Landscape

Editor’s Note: This article originally appeared in Biotech Primer Weekly. For more of the science behind the headlines, please subscribe.

Emily Burke, BiotechPrimer.com

Multiple myeloma is a cancer formed by a type of white blood cell called a plasma cell. These cells are the antibody-producing cells of our immune system and play a critical role in our defense against infections. If they begin to grow and divide in an uncontrolled manner, however, they form a plasmacytoma – a mass of cells within the bone marrow that no longer function in our defense but instead simply take up space and interfere with the functions of healthy cells. Instead of producing normal disease-fighting antibodies, plasmacytoma cells produce abnormal antibodies called M proteins, which don’t provide any benefit to the body and crowd out normally functioning antibodies.

Easily Confused: Plasma Cells vs Blood Plasma

Plasma cells are specialized white blood cells that produce infection-fighting antibody proteins. Most plasma cells are found in the bone marrow. Blood plasma is the straw-colored liquid component of blood that holds blood cells in suspension, made up of water (95%), proteins, glucose, clotting factors, electrolytes, hormones, carbon dioxide, and oxygen.

Picking Apart Plasmacytoma

Plasmacytoma formation can lead to a host of problems with recognizable clinical symptoms. Because all blood cells are formed in the bone marrow, over-production of plasma cells can essentially “crowd out” normal blood-forming cells. This can lead to anemia, caused by a shortage of oxygen-carrying red blood cells; increased bruising and bleeding due to a reduction in clot-promoting platelets; and an increased risk of infections due to lower levels of healthy infection-fighting white blood cells.

Although multiple myeloma is classified as a blood cancer, it has a significant impact on bone health. As the plasmacytoma grows, bone-forming cells called osteoblasts are suppressed. At the same time, production of a substance that activates bone-reabsorbing cells, osteoclasts, is increased. The resultant damage to the bone structure results in soft spots or lesions which may extend from the inner bone marrow to the outside surface of the bone. Bone lesions result in significant pain and increase the risk of fracture. Bone destruction also releases excessive calcium into the bloodstream, which leads to a range of symptoms including changes in urination, restlessness, confusion, increased thirst, nausea, and loss of appetite. Excess blood calcium, combined with high levels of M protein, also contributes to the impaired kidney function seen in multiple myeloma patients.

Unmasking Multiple Myeloma

There is no one diagnostic test for multiple myeloma. Blood and urine tests to detect some of the symptoms listed above such as low blood cell counts, elevated blood calcium levels, and impaired kidney function may suggest multiple myeloma. These tests can be followed by a bone marrow biopsy for confirmation.

Most cases of multiple myeloma have no known cause, although some research suggests that regular exposure to herbicides, insecticides, petroleum products, heavy metals, and asbestos increases the risk of developing the disease. And although there is not a specific gene yet associated with multiple myeloma, abnormalities in chromosome structure or number are associated with the disease.

Multiple Myeloma Treatments

Once considered incurable, there are now a number of effective treatments for multiple myeloma, and several more are in the pipeline.

Currently, there are two FDA-approved monoclonal antibody therapeutics approved to treat multiple myeloma.  They work by recognizing and binding to proteins on the surface of multiple myeloma cells, activating the patient’s immune system to destroy those cells.

Another type of approved therapy for multiple myeloma is a small molecule proteasome inhibitor therapy. A proteasome is a specialized compartment within the cell that gets rid of damaged proteins by digesting them. If the proteasome is inhibited, damaged proteins build up within the cell. This triggers a process called apoptosis – essentially, cell suicide. In other words, the cancer cell kills itself.

Small molecule histone deacetylase (HDAC) inhibitors have also been shown to be safe and effective in treating multiple myeloma. HDACs are enzymes that modify chromosomes (strands of DNA that contain our genes) and influence how often specific genes are activated. Some cases of multiple myeloma are associated with changes in gene activation. By inhibiting HDACs, this faulty gene expression can be corrected.

In the Pipeline

Two novel drugs in the multiple myeloma pipeline are Mivebresib and Selinexor.

Mivebresib influences the activation of specific genes by inhibiting a group of proteins called Bromodomain and Extra Terminal motif (BET) proteins. In some types of cancer, genes are activated or deactivated inappropriately due to BET activity. By inhibiting BET, normal gene activity may be restored to these cells.

Selinexor helps to increase the number of tumor suppressor proteins present in the nucleus of cancer cells. These proteins help to protect against cancer by detecting DNA damage and promoting apoptosis in those cells that have high levels of DNA damage. In many types of cancer cells, tumor suppressor proteins are transported out of the nucleus, where they can no longer do their job of detecting DNA damage. Selinexor blocks this transport and enables tumor suppressor proteins to do their job of triggering apoptosis in cancer cells.

A number of CAR-T therapies are also in development for multiple myeloma, with several early stage clinical trials ongoing. 

Multiple myeloma is a complex type of cancer. In recent years, a better understanding of the disease has led to the approval of several new therapeutics. In the coming years, we can look forward to additional approvals as novel therapeutics move through the pipeline.

New Real World Health Care Series: Multiple Myeloma Research and Treatment

Krista Zodet, President, HealthWell Foundation

Susan, from Columbia, S.C., is among the millions of Americans struggling to manage a chronic and life-altering disease without the financial means to afford needed medication. Here at HealthWell, we’re honored to be able to help Susan and other patients like her. It always warms our hearts to receive letters like the one she sent:

“I have been a multiple myeloma patient since December 2007 and, unfortunately, there is presently no cure for this cancer. Thankfully, I am responding to treatment, but it is expensive. My oral maintenance medication costs over $11,000 per month and it gets more expensive every year. I am so very thankful for Medicare, but we have a $3,600 deductible that has to be met before insurance pays the claims. That’s a lot of money to come up with each year on a worship pastor’s salary. The charity I was receiving help from can no longer help multiple myeloma patients, but they referred me to HealthWell Foundation and it could not have been easier! The HealthWell representatives I have spoken with have been professional, caring, and efficient. I am thrilled with my experience. Thank you to the wonderful and generous donors who make this possible.” – Susan, Columbia, SC

Susan’s story is not unique. It is however, unfortunate, and one that we hear at the HealthWell Foundation all too often. Cancer is not easy and it’s not inexpensive. That’s why it’s important for us to continue to use this blog to highlight advances in current research, therapies, and what’s on the horizon for treating devastating diseases like Multiple Myeloma.

About Multiple Myeloma

Multiple myeloma is a type of blood cancer that affects the plasma cells found in bone marrow. It is the second most common blood cancer and is considered incurable. It is a treatable disease, however, thanks to recent advances in cancer research which are improving the life expectancy of multiple myeloma patients.

According to the American Cancer Society, more than 30,000 new cases of multiple myeloma are expected to be diagnosed in 2017 and more than 12,000 deaths are expected to occur. The disease is more prevalent among men than among women.

Promising New Therapies

Our new Real World Health Care series will shine a spotlight on the individuals and organizations driving research on new multiple myeloma therapies, including monoclonal antibodies, CAR-T cell therapy, checkpoint inhibitors and other immunotherapies. I share the hopes of these researchers that these new therapies, or others just coming to the drawing board, will allow multiple myeloma to be treated easily and effectively, and with the possibility of a cure.

Supporting Multiple Myeloma Patients

The HealthWell Foundation, sponsor of Real World Health Care, is proud to support the multiple myeloma patient community with copayment and premium assistance. We have helped more than 9,000 multiple myeloma patients afford their treatments since launching our Multiple Myeloma Medicare Access Fund in 2015 — thanks to the generous support of our donors. We invite corporations and individuals to help us by contributing to our Multiple Myeloma Medicare Access Fund, so no one goes without essential medications because they cannot afford them.