In a breakthrough poised to reshape cancer treatment, Houston Methodist has developed the first personalized mRNA cancer vaccine designed to prevent recurrence of osteosarcoma, a rare and aggressive type of bone cancer.

 

This first-of-its-kind treatment was made possible through a compassionate use allowance from the U.S. Food and Drug Administration (FDA) and represents a major step in personalized medicine and cancer care.

 

Unlike conventional cancer treatments, the vaccine was custom-built to target newly formed abnormal proteins unique to the patient’s cancer, with the goal of preventing its recurrence. The vaccine uses messenger RNA (mRNA) technology to instruct the patient’s immune system to target tumor-specific proteins. This same technology, which revolutionized COVID-19 vaccines, is now being applied to oncology in a way that could prevent cancer recurrence—a critical challenge for patients with aggressive forms of the disease. Osteosarcoma has a recurrence rate of up to 40% and 5-year survival estimates are only 20-30% following relapse.

 

“Safety is our priority with this first-of-its-kind vaccine, which is being administered at low doses,” said Houston Methodist oncologist Dr. Kai Sun. “The patient is tolerating the treatment very well, and we are already observing promising immune responses in blood biomarkers even after the first dose.”

 

Houston Methodist is the first hospital system to leverage its infrastructure and expertise to develop and deliver this highly personalized therapy, with a multidisciplinary team harnessing the institution’s research and clinical capabilities to create a treatment tailored to both the patient and the biology of their cancer.

 

“This approach could transform cancer care,” said Dr. John Cooke, medical director for the Houston Methodist Center for RNA Therapeutics. Cooke led the multidisciplinary team responsible for the development of the treatment. “The ability to design a vaccine that trains the immune system to recognize and attack cancer cells unique to each patient opens the door to treating many other cancer types.”

 

The project exemplifies Houston Methodist’s commitment to innovation and collaboration. From sequencing the patient’s tumor to identifying neoantigens and manufacturing the vaccine, every step was completed within Houston Methodist’s facilities – and in just four months' time. The treatment leveraged existing studies of similar methods that could eventually prevent the recurrence of triple-negative breast cancer. In time, the researchers hope the treatment will be scalable so that medical centers around the world can personalize vaccines to treat other tumor-based cancers.

 

“What we’re doing for cancer now builds on what we’ve spent the last four years developing for infectious diseases—working to create effective vaccine candidates in an accelerated time frame,” said Jimmy Gollihar, head of the Houston Methodist Antibody Discovery & Accelerated Protein Therapeutics Laboratory and chief translational science officer. “The vaccine design wasn’t a single best guess that was rushed forward. We designed multiple candidates, synthesized them and tested them in human cells first, before moving forward with RNA synthesis.”