For Rare Cancer Patients, More Treatment Options Are Needed

For someone with cancer, the right treatment option can mean the difference between life and death. While cancer research has made immense strides in the past decade and even the past few years, there is still much work to be done, especially for patients with rare cancers like Waldenstrom macroglobulinemia (WM).

What is WM?

WM is a rare cancer that begins in the white blood cells and is one of the many subtypes of non-Hodgkin lymphoma (NHL). With about three people diagnosed per million people per year in the U.S., it is uncommon, but approximately 1,500 Americans are diagnosed with WM annually. On average, people living with WM are 70 years old.

WM cells have changes in their DNA—or genetic mutations—that cause uncontrolled growth, but scientists and researchers are making advancements in understanding how these mutations can cause normal cells to develop into WM cells. Understanding genetic mutations is important because as researchers learn more about them, treatment options become more personalized.

With more personalized treatment, doctors can tailor a person’s treatment regimen to that individual’s specific genes and disease. Instead of giving the same treatment to everyone, doctors can pick the right medicine and the right dose at the right time.

Scientists now know that some patients’ WM cells have mutations in the CXCR4 gene. Treatments that are currently available for WM patients, such as ibrutinib, may not be as helpful for people with the CXCR4 variant, according to a recent study. This is one of the many reasons more treatment options are needed. Researchers are looking into treatments that target the WM cells with this genetic mutation, and some of these potential treatment options are available now through clinical trials.

What Is a Clinical Trial?

Clinical trials help researchers understand more about a disease and find new treatments. They are the primary way that researchers find out if a new treatment is safe and effective in people before it is approved by the FDA.

A clinical trial is now enrolling people living with WM who have a CXCR4 genetic mutation, as well as other characteristics. This WM clinical trial will help researchers answer questions about the safety of an investigational drug mavorixafor, an oral medication in capsules taken by mouth once a day. In the trial, mavorixafor will be taken along with ibrutinib, an FDA-approved treatment for WM.

If you are living with WM, talk to your doctor, and see if participating in a clinical trial is an option for you. There is no cost to join the study. Expenses for medical care and travel are reimbursed by X4 Pharmaceuticals, the study sponsor. To learn more, visit the study website at

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