New Treatment Eradicates Rare Type of Cancer

By inhibiting the growth of a specific protein in laboratory mice, scientists were able to kill off cells responsible for a rare type of cancer that affects the connective tissue surrounding nerves.

The cancer, known as malignant peripheral nerve sheath tumors (MPNSTs), was eradicated in mice that had the protein BRD4 inhibited, according to a new study published in the journal Cell Reports.

"This study identifies a potential new therapeutic target to combat MPNST, an incurable type of cancer that is typically fatal," said senior study author Dr. Lu Le, an assistant professor of dermatology at UT Southwestern in Dallas, Texas. "The findings also provide important insight into what causes these tumors to develop."

MPNSTs are considered to be highly aggressive sarcomas, a type of malignant tumor, but they are quite rare and most commonly found in patients with a genetic disorder called neurofibromatosis type 1 (NF1), which affects 1 in 3,500 people. About 10 percent of NF1 patients will develop MPNST. Although the tumors are frequently benign, the tumors are large and often disfiguring.

Removing these tumors is difficult because of their location around the nerves. Radiation and chemotherapy are other treatment options, but their effectiveness is limited. The five-year survival rate of patients with MPNSTs is about 50 percent.

The new treatment for MPNSTs developed by Dr. Le and his colleagues may lead to better survival rates for human patients with this rare type of cancer.

"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," Dr. Le said.

The same class of drugs used in the MPNST tests are currently being used in experimental treatments for other cancer types, including leukemia and a type of lung cancer.