June 2014

Patient’s Own Cells Helped Fight Cancer

An experimental therapy developed at NIH used a patient’s own immune system to attack and shrink her tumors. With further research, this type of immunotherapy might be used to treat many common cancers.

The 43-year-old woman has cholangiocarcinoma, a rare and often-deadly cancer that develops in the bile duct (a tube that goes from the liver to the intestine). She was enrolled in an NIH clinical trial for patients with digestive system cancers. Her cancer had spread to her lung and liver. Standard chemotherapy didn’t help.

The scientists first removed some of the woman’s cancerous lung tissue, which along with tumor cells also had some of the patient’s own immune cells. These immune cells had been fighting a losing battle against the tumor.

Analysis showed that the tumor’s DNA contained several abnormal regions, or mutations. Some of these mutations created mutant proteins that could trigger an immune response.

The researchers then tested the patient’s immune cells. A few could specifically recognize and trigger an attack on a particular mutant protein found on the tumor cells. The scientists then grew billions of the anti-tumor immune cells in the lab and infused them back into the patient.

After this treatment, the woman’s tumors stopped growing in the lung and liver. When her disease eventually progressed, after about 13 months, she was re-treated with a more purified collection of the immune cells, and her tumors shrank. This regression continued as of the last follow-up exam 6 months later.

The results show that the immune system's response against a mutant protein can be harnessed to fight a hard-to-treat type of cancer. 

“The method we have developed provides a blueprint for using immunotherapy to specifically attack … mutations unique to a patient’s individual cancer,” says NIH’s Dr. Steven Rosenberg, who led the research. He and his colleagues are continuing to assess their experimental immunotherapy in a clinical trial.

 

Reference: Cancer immunotherapy based on mutation-specific CD4+ T cells in a patient with epithelial cancer. Tran E, et al. Science. 2014 May 9;344(6184):641-5. doi: 10.1126/science.1251102. PMID: 24812403.