Pembrolizumab for Sarcoma

This trial tests pembrolizumab, a drug that aids the immune system in fighting cancer, as a treatment for certain types of sarcomas, which are rare cancers. Researchers aim to determine if the drug is effective and causes only mild side effects. Participants must have a confirmed diagnosis of pleomorphic liposarcoma, PEComa, epithelioid sarcoma, CIC-rearranged sarcoma, or sclerosing epithelioid fibrosarcoma, with cancer that has recurred or spread and lacks other treatment options. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

The trial does not specify if you need to stop taking your current medications. However, you cannot be on certain treatments like systemic steroids or have had recent cancer therapies. It's best to discuss your specific medications with the trial team.

A previous study found that pembrolizumab had a manageable safety profile in patients with rare and ultra-rare types of sarcoma. Common serious side effects included anemia (low red blood cell count), affecting about 8% of patients, and increased liver enzymes, seen in 6% of patients. These side effects, while significant, are considered manageable.

For those with epithelioid sarcoma, pembrolizumab proved effective and maintained a similar safety profile. Research on sclerosing epithelioid fibrosarcoma and low-grade fibromyxoid sarcoma shows that pembrolizumab is generally well-tolerated, with mostly mild to moderate side effects.

In PEComa (a type of tumor), pembrolizumab has been associated with stable disease over a long time, suggesting it works well without causing severe side effects. For CIC-rearranged sarcoma, the treatment's safety was consistent with previous trials, with only 5% of patients experiencing serious issues.

Finally, in pleomorphic liposarcoma, pembrolizumab, when used with other treatments, improved outcomes and was generally safe, indicating it can be a valid option without major risks.

Pembrolizumab stands out because it harnesses the power of the immune system to tackle sarcoma, unlike traditional chemotherapy or radiation treatments. It works by blocking a protein called PD-1, which can prevent the immune system from attacking cancer cells. This mechanism helps the body’s natural defenses recognize and fight the cancer more effectively. Researchers are excited about pembrolizumab's potential to offer a more targeted approach with possibly fewer side effects, providing new hope for patients with challenging sarcomas.

Research has shown that pembrolizumab can help treat certain types of sarcomas, which are rare cancers. In this trial, participants will receive pembrolizumab for various sarcoma types. For epithelioid sarcoma, about 6.2% of patients respond to the treatment, with an average survival time of 19.7 months. In sclerosing epithelioid fibrosarcoma and low-grade fibromyxoid sarcoma, some patients have responded well, experiencing different durations before disease progression. Pembrolizumab has also shown promise in treating PEComa, with some patients experiencing stable disease after treatment. In CIC-rearranged sarcoma, about 34.3% of patients respond to the treatment, with some experiencing survival benefits. For pleomorphic liposarcoma, combining pembrolizumab with other treatments has improved the time patients remain disease-free. These findings suggest that pembrolizumab may enhance the immune system's ability to fight these rare sarcomas.²³⁶⁷⁸