Treatment for Rare Form of Non-Hodgkin Lymphoma Gets FDA Approval

Ibrutinib – a drug that targets the molecule that fuels the growth of B cells in Waldenstrom macroglobulinemia and other cancers – is approved for use in the U.S.

In January 2015, the U.S. Food and Drug Administration (FDA) announced the first ever approval of a treatment for Waldenström’s macroglobulinemia, a rare form of non-Hodgkin lymphoma that is diagnosed in about 1,500 people a year in the United States.

Waldenström’s macroglobulinemia, which is also known as lymphoplasmacytic lymphoma, is a rare and incurable type of non-Hodgkin lymphoma that arises in immune cells called B cells. Ibrutinib, sold under the brand name Imbruvica, had already received FDA approval for treatment of other, more common forms of B-cell lymphoma and leukemia, including chronic lymphocytic leukemia (CLL).

One patient with rare lymphoma, Shelley Lehrman of Solon, Ohio, was benefiting from ibrutinib for more than two years when she was featured in the AACR Cancer Progress Report 2014.

Shelley, who was enrolled in a clinical trial testing ibrutinib, said that the molecularly targeted drug allowed her to live a life as busy and full as it was before her diagnosis.

This is common among patients taking molecularly targeted therapeutics, which hone in on the Achilles’ heel of a patient’s cancer and are therefore often more effective and less toxic than the treatments that have been the mainstay of patient care for decades.

“Participating in clinical trials was an easy decision for me,” Shelley explained. “I feared my loved ones could be stricken with the same disease in the future, and I knew that by taking part I was doing something that could potentially benefit them. I also felt that I had more control over my health care because the clinical trials gave me choices fueled by the most recent research.”

Ibrutinib specifically blocks the function of a molecule called BTK. This molecule is a central component of a molecular chain of command that fuels the expansion and survival of Waldenström’s macroglobulinemia B cells.

The BTK signaling pathway also fuels the expansion and survival of a number of other blood cancers that arise in B cells, including CLL and a type of non-Hodgkin lymphoma called mantle cell lymphoma.

In fact, ibrutinib was approved by the FDA for treating patients with mantle cell lymphoma in 2013, and for treating certain groups of patients with CLL in 2014.

The medication is also being tested in clinical trials as a treatment for several other B cell-originating blood cancers that depend on BTK signaling, including multiple myeloma and diffuse large B-cell lymphoma.

The FDA decision to approve ibrutinib as a treatment for Waldenström’s macroglobulinemia, which came two months earlier than expected, was based on a phase II clinical trial that showed that 62 percent of patients taking the drug had their cancer shrink.

The FDA approval was rendered on Jan. 29, 2015. Find out more about the decision.