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Study finds potential target for rare tissue cancer

Category: Cancer Detection and Tumor Markers

Research published in the online journal Cell Reports reveals that a team of scientists from the University of Texas-Southwestern Medical Center discovered a new method through lab tests to treat an incurable type of tissue cancer. The researchers found that removing a specific protein may completely eliminate the cancer.

Led by Lu Le, Ph.D., the team discovered that impeding the function of the protein BRD4 resulted in the death of cancer cells in an animal model of malignant peripheral nerve sheath tumors, or MPNSTs. These growths are aggressive sarcomas that develop sporadically around the nerves. Half of MPNST cases occur in patients with neurofibromatosis type 1 (NF1), a genetic disorder that affects 1 in 3,500 people. Typically, surgical removal has been the preferred treatment method for MPNST despite its level of difficulty due to the location of the tumors near the nerves. While chemotherapy and radiation therapy are alternative options, their effectiveness is restricted.

Learning about NF1
NF1 is characterized by changes in the coloring of the skin. Patients with this condition experience tumor growth along the nerves of the brain, skin and other parts of the body. Almost all people with NF1 have flat patches of skin that are darker than the area around them. Most adults develop noncancerous tumors underneath the skin, however, cancerous tumors do sometimes grow along the nerves in some cases. Patients with NF1 are at a higher risk for developing brain tumors and leukemia than others.

Due to the rarity of MPNST, studies such as Le's are needed to further research improved treatments for patients.

"This study identifies a potential new therapeutic target to combat MPNST, an incurable type of cancer that is typically fatal. The findings also provide important insight into what causes these tumors to develop," explained Le, senior author of the study and assistant professor of Dermatology at UTSW.

In MPNST, the levels of BRD4 are unusually high and this prevents the cancer cells from dying. To fight his reaction, the scientists administered a compound to impede BRD4 and found that the tumors shrank.

"These treatments suppressed tumor growth and caused the cancer cells to undergo apoptosis, or cell death. This is why BRD4 inhibition is exquisitely effective against MPNSTs and may represent a paradigm shift in therapy for these patients," Le continued.

Working with similar BRD4 drugs
The university is currently working with a pharmaceutical company to develop a drug similar to the BRD4 inhibitor in order to run clinical trials with patients who have MPNST. In trials for treatment of leukemia and another type of lung cancer, the same class of drug is being experimented with.

With constant advancements in cancer screening and blood testing, doctors hope to continually improve the quality of care for cancer patients.