This stage, Phase I, is designed to determine the maximum tolerated dose of the vaccine in patients with early-stage triple-negative breast cancer and to characterize and optimize the body's immune response. The U.S. Food and Drug Administration recently approved an investigational new drug application for the vaccine, allowing Cleveland Clinic and its partner Anixa Biosciences, Inc. (ANIX: NASDAQ) to begin the study.

"We are hopeful that this research will lead to more advanced trials to determine the vaccine's efficacy against this highly aggressive type of breast cancer", said G. Thomas Budd, M.D., of Cleveland Clinic's Taussig Cancer Institute and principal investigator of the study. "In the long term, we hope this could be a true preventive vaccine that would be given to healthy women to keep them from developing triple-negative breast cancer, the form of breast cancer for which we have the least effective treatments".

There is a great need for improved treatments for triple-negative breast cancer, which does not have biological characteristics that typically respond to hormonal or targeted therapies.

Despite accounting for only about 12-15% of all breast cancers, triple-negative breast cancer accounts for a disproportionately higher percentage of breast cancer deaths and has a higher rate of recurrence. This form of breast cancer is twice as likely to occur in African-American women, and approximately 70% to 80% of breast tumors occurring in women with BRCA1 gene mutations are triple-negative breast cancer.

"The vaccine approach represents a potential new way to control breast cancer," said Vincent Tuohy, Ph.D., the lead inventor of the vaccine and a staff immunologist at Cleveland Clinic's Lerner Research Institute. "The long-term goal of this research is to determine whether this vaccine can prevent breast cancer before it occurs, particularly the more aggressive forms of this disease that predominate in high-risk women."

Dr. Tuohy is named inventor of the technology, which Cleveland Clinic licensed exclusively to Anixa Biosciences, Inc. He will receive a portion of the commercialization revenues received by Cleveland Clinic for this technology and also have personal equity in the company.

Cleveland Clinic for this technology and also has personal equity in the company. The investigational vaccine targets a breast-specific lactation protein, α-lactalbumin, which is no longer found after lactation in normal and aged tissues, but is present in most triple-negative breast cancers.

Activation of the immune system against this "retired" protein provides preventive immune protection against emerging breast tumors expressing α-lactalbumin.

The vaccine also contains an adjuvant that activates an innate immune response that allows the immune system to mount a response against emerging tumors to prevent them from growing.

The study builds on preclinical research led by Dr. Tuohy that demonstrated that activation of the immune system against α-lactoalbumin protein was safe and effective in preventing breast tumors in mice. The research also found that a single vaccine could prevent the occurrence of breast tumors in mouse models while inhibiting the growth of existing breast tumors. The research was originally published in Nature Medicine, was funded in part by philanthropic donations from more than 20,000 individuals over the past 12 years.

Funded by the U.S. Department of Defense, the new Cleveland Clinic study will enroll 18 to 24 patients who completed treatment for early-stage triple-negative breast cancer within the past three years and are currently tumor-free but at high risk for recurrence. Over the course of the study, participants will receive three vaccines, each two weeks apart, and will be closely monitored for side effects and immune response. The study is estimated to be completed in september 2022.

The researchers anticipate that a subsequent trial will involve healthy, cancer-free women at high risk of developing breast cancer who have decided to undergo a voluntary bilateral mastectomy to reduce their risk. Typically, such women carry mutations in the BRCA1 or BRCA2 gene and are therefore at risk of developing triple-negative breast cancer or have a high familial risk of any form of breast cancer.

"This vaccine strategy has the potential to be applied to other tumor types", Dr. Tuohy said. "Our translational research program is focused on developing vaccines that prevent diseases we face as we age, such as breast, ovarian and endometrial cancers. If successful, these vaccines have the potential to transform the way we manage adult-onset cancers and improve life expectancy in a manner similar to the impact that the childhood vaccination program has had".