Among the challenges with cell therapy in cancer treatment are the side effects caused by the body itself. While prompting the immune system’s T cells to target cancer has shown potential to be effective, these experimental approaches can also trigger immune reactions that harm patients, says Daniel Passeri, president and CEO of Cue Biopharma.
Cue aims to accomplish what cell therapy does, but in a different way—and without the same side effects. Rather than reintroducing T cells to the body that have been engineered to recognize tumors, Cambridge, MA-based Cue has developed what it says is a way to use a drug to activate the immune response. Passeri says Cue’s biological drug only triggers the T cells relevant to the cancer.
“You’re leaving the other T cells alone, and you don’t end up with these deleterious side effects,” he says.
Cue is now unveiling its immunotherapy approach, along with $16.4 million in new capital, led by MDB Capital Group. The investment follows $10 million in seed funding the company raised after it was founded in 2015. The technology underlying Cue’s approach was developed from research at the Albert Einstein College of Medicine in New York and was supported by National Institutes of Health grant funding.
Research over the last decade has helped scientists better understand how the immune system modulates itself, Passeri says. New drugs have been developed to trigger an immune response by activating T cells, a type of white blood cell that roams the body for scanning for problems like cancer. But Passeri says this broad approach of activating T cells throughout the body, without instructing them to hit a specific target, can prompt an overreaction by the immune system that leads to inflammation, causing fatigue, headache, nausea, and muscle and joint pain. These “cytokine storms” can be fatal. Last year, Kite Pharma (NASDAQ: KITE) said that two patients who died in a clinical trial testing its cellular therapy had experienced cytokine release syndrome. Two patients who died in a 2014 Juno Therapeutics (NASDAQ: JUNO) trial also reportedly experienced the cytokine side effect.
Passeri says Cue can design drugs that they emulate the way that the immune system selectively instructs T cells to find the right target. Cue says that its drug delivers molecules to the T cells that have a receptor protein for a specific type of cancer, which stimulates the more focused immune response. In animal studies, the Cue drug did not show inflammation or cytokine storms, Passeri says.
Furthermore, those early studies suggest that Cue’s drug would offer a broader “therapeutic window,” the range of doses that produce a therapeutic benefit without causing toxic effects, Passeri says. Some drugs currently used to treat cancer don’t have a very large therapeutic window, leading to patients to develop side effects. Ronald Seidel, co-founder of Cue and the company’s head of research and development, says that the selectivity of Cue’s approach is able to achieve therapeutic benefit at doses well below what would cause toxic side effects.
Cue’s pipeline includes two pre-clinical candidates: CUE-101 is being developed for cervical, head and neck, and anal cancers; CUE-102 is a potential treatment for melanoma, synovial sarcoma, prostate, and head and neck cancers. Passeri says the company’s approach of selectively modulating the immune system could also have applications in treating autoimmune disorders. But for now, cancer is the company’s main focus.
Cue still has to test its approach in humans, of course. Passeri says that the new capital allows Cue to continue developing its platform and working toward clinical trials. The company is about a year away from starting human studies, which will require additional funding, Passeri says. While Cue’s approach can be used alone, Passeri says the technology could also work in combination with other cancer treatments, such as checkpoint inhibitors. Passeri says Cue is exploring potential partnerships with companies that already have such drugs in their portfolios.