Early next year, a new clinical trial will be conducted to test an immunotherapy treatment that has a new and unique delivery system. The study will be conducted at the University of Pennsylvania. The Food and Drug Administration gave orphan drug status to SynKIR-110, a CAR T-cell therapy utilized for solid tumors (including mesothelioma) that express a specific protein. Orphan drug status allows the FDA to give financial incentives like tax credits for clinical trials and market exclusivity for seven years. Orphan drug status is meant to help find new treatments for diseases that occur in less than 200,000 people in the United States.
The clinical trial, known as STAR-101, will be the first in human test. It will help determine efficacy, tolerability, and safety in humans. It was previously tested in mice and had great results including complete remission. SynKIR-110 will also be used on bile duct and ovarian cancer patients whose cancers express the protein mesothelin. Verismo Therapeutics, a biotechnology company from Philadelphia, created SynKIR-110. It created the KIR-CAR delivery platform, a modified killer-like receptor meant to improve efficacy and persistence when used against aggressive solid tumors. SynKIR-110 will be the first treatment to use the KIR-CAR treatment delivery platform.
CAR T-cell therapy is very effective on blood cancers including leukemia and lymphoma, but it is not effective on advanced solid cancers like mesothelioma. T-cell therapy utilizes the unique genetic profile of a patient as well as modified T cells, which are white blood cells that help the immune system fight disease and infection. Cancer cells can avoid detection of T cells by expressing mesothelin. Doctors can fix this by altering the cells in a lab to recognize and target cancer cells that express a specific protein like mesothelin. The company made more effective T cells by putting them in a multichain structure.
There is a clear need for treatments that are effective on solid cancers that express mesothelin. Ovarian cancer’s survival rate is 50 percent, while other cancers like cholangiocarcinoma and mesothelioma have five-year survival rates of 5 percent and 30 percent respectively. Mesothelioma is a rare cancer with only around 3,000 cases diagnosed in the United States every year. The best option to treat mesothelioma is usually a combination of surgery, chemotherapy, and radiation, but not many patients are able to receive this treatment regimen because less than a third qualify for surgery. Once people are diagnosed, the disease has usually progressed to a late stage and chemotherapy is the only option.
