Pembrolizumab shows promise for some advanced, hard-to-treat rare cancers

Share on facebook
Share on twitter
Share on linkedin
Share on email
Share on print

A study conducted by researchers at MD Anderson Cancer Center demonstrated acceptable toxicity and anti-tumor activity in patients with four types of advanced, hard-to-treat rare cancers. Study findings were published in the March 17 online issue of the Journal for ImmunoTherapy of Cancer.

The open-label, phase II study followed 127 patients who had advanced rare cancers: squamous cell carcinoma of the skin, carcinoma of unknown primary, adrenocortical carcinoma, and paraganglioma-pheochromocytoma. All patients had tumors that had progressed on standard therapies.

The primary objective of the study was to find the proportion of patients who were alive and progression-free at 27 weeks on treatment with pembrolizumab. The median non-progression rate at that time was 28% for 127 patients with advanced rare cancers. Complete response, partial response or stable disease after four months was observed in 38% of the patients. Non-progression rates for each cancer group were: 36% for cSCC, 33% for CUP, 31% for ACC, and 43% for paraganglioma-pheochromocytoma. Treatment-related adverse events occurred in 52% of patients, with the most common side effects being fatigue and rash, with six deaths reported that were unrelated to treatment.

“Our findings that pembrolizumab has a favorable toxicity profile and anti-tumor activity in patients with these rare cancers supports further evaluation in these populations,” Aung Naing, associate professor of Investigational Cancer Therapeutics, said in a statement. “Finding solutions for treatment is vital given that patients with advanced rare cancers have poor prognosis and few treatment options.”

CUP is a type of cancer in which the primary cancer site is not always known, but has spread to other areas within the body, while ACC occurs when malignant cells form in the outer layer of the adrenal glands.

Paraganglioma-pheochromocytoma are tumors formed in nerve-like cells near the adrenal glands and near blood vessels or nerves in the head, neck, chest, abdomen, and pelvis.

YOU MAY BE INTERESTED IN

Just consider for a minute if this was the first year of running your lab, if you were on the job market as a physician or scientist right now, if you were a resident contemplating a career in cancer research after fellowship, if you were a graduate student or postdoc, if you were an undergraduate or a technician who was looking toward graduate school.
The immune system can be a powerful tool to control cancer. Immune cells within our body detect cancer cells and release payloads that kill them. Transformative science in the last decade has led to the development of therapies that enhance the ability of our immune cells to carry out this function. These therapies, including checkpoint blockade and CAR-T cells, have been lifesaving for many patients that before had untreatable cancer. But, sadly, a majority of patients with advanced solid tumors still succumb to their disease. 

Never miss an issue!

Get alerts for our award-winning coverage in your inbox.

Login