Federal study offers new options for people with rare cancer

People with rare cancers have been unlucky – not only do most people not have good treatments, but drug companies do not practice them either because of their low potential sales. Now, a federal study pooling these people gives them strength in numbers and new options.

The first results of this innovative effort were revealed at a conference Sunday of the American Association for Cancer Research in Atlanta. They suggest that for people with one of these unusual diseases, there seems to be a way to predict who will benefit from an badociated drug that strengthens the immune system and who will not do it.

"For the patients he works for, it's phenomenal, it's life changing … but it's not for everyone," said Dr. Sandip Patel, director of the study, University of California, at the Moores Cancer Center in San Diego.

Brigitte Sagbader, 54, of San Marcos, Calif., Is one of the participants in the study that seems to benefit. Its tumors have decreased since treatment began a year ago for a rare type of ovarian cancer that has challenged surgeries, chemotherapy, and hormone therapy.

"I tried all kinds of things before and nothing really helped in the long run," she said. "I felt a bit out of the way of what could be done."

Rare cancers individually affect less than 6 people per 100,000 per year, but together account for 22% of cancer cases. They include tumors to the brain, blood, glands, bones and skin. Often, patients can not even participate in a study to test a drug or an experimental treatment.

"We are trying to fill this gap" by funding the federal study, said Dr. Elad Sharon of the National Cancer Institute.

He has recruited more than 550 patients in the United States and Canada since his debut in 2017. Currently, he is testing a combination of two immunotherapy drugs, Opdivo and Yervoy, in people grouped into "buckets" based of their type of tumor. Drug manufacturer Bristol-Myers Squibb donates it to the study.

One of these buckets has just reached the critical mbad needed to get results – neuroendocrine tumors, which can form many places in the body but often in the lungs or digestive tract. The biopsies at the time of the diagnosis of the disease clbadify the cases in high, low or intermediate grade according to the aggressive growth of the cells.

In the study of 32 patients, the combo drug only worked for high grade tumors. Eight of the 18 patients saw their tumor contract significantly. None of the others did it.

In the high-grade group, survival at six months without worsening of the disease was 31% – better than the 10% that doctors saw such patients, said Patel, a paid counselor from Bristol-Myers and several other companies.

"This gives us a glimpse of what can happen," said another study lead, Dr. Razelle Kurzrock at the San Diego Cancer Center. "If we succeed, we may be able to make it work in more patients."

About a quarter of all patients had typical symptoms of immune system therapies – liver effects, fatigue, nausea – and 6% had colitis, an inflammation of the intestine. But the side effects were manageable and not serious, Patel said.

Identifying which patients are likely to benefit from these drugs saves others from these side effects, expenses and false hopes.

Sagbader has developed a problem at Yervoy, but continues to receive Opdivo every two weeks. She looks forward to next year, 20 years from the first diagnosis.

"It will be a party next year, believe me," she said. "I'm waiting for cancer to be definitely cleared by here."

___

Marilynn Marchione can be followed on http://twitter.com/MMarchioneAP