FDA Clears Breast Cancer Vaccine For Clinical Trials

Doctor giving a patient a vaccine on their arm.

PhotoAlto/Michele Constantini / Getty Images

Key Takeaways

  • The FDA has approved a triple-negative breast cancer vaccine for use in human clinical trials. 
  • The vaccine was developed by researchers at the Cleveland Clinic in Ohio and works by training the immune system to kill cells that express the protein alpha-lactalbumin. 
  • The technology used to develop the vaccine could be applicable to cancers beyond breast cancer as well. 

On December 21, the Food and Drug Administration (FDA) approved an investigational drug application for a triple-negative breast cancer vaccine that could reduce the incidence of advanced disease. The brainchild of Vincent Tuohy, PhD, a researcher in the department of inflammation and immunity at the Cleveland Clinic’s Lerner Research Institute, the vaccine has been preemptively licensed to the biotechnology company Anixa Biosciences.

Triple-negative breast cancer—so named because it does not produce receptors for the hormones estrogen and progesterone and only produces HER2, the receptor for the protein human epidermal growth factor, in small amounts—accounts for about 10-15% of all breast cancers, making it one of the disease’s rarer incarnations.

Medically, it is differentiated from other breast cancers by its aggression and its target demographics: women under the age of 40, Black women, and women who have a BRCA1 mutation.

The vaccine, Tuohy tells Verywell, essentially works by nipping tumor development in the bud. By imbuing immune cells with the ability to recognize, attack, and kill cancer cells, it prevents them from ever reaching a size at which they could inflict lethal damage on the body. Triple-negative breast cancer cells are distinctive because they express the protein alpha-lactalbumin, a major component of breast milk, even when their host is not pregnant or breastfeeding.

“Most triple-negative breast cancers express alpha-lactalbumin," Tuohy says. "It is a mistake that the tumors make because they have no default inhibition mechanisms through progesterone and estrogen signaling that would ordinarily prevent the expression of this protein." He describes the vaccine mechanism as “simply taking advantage of this mistake.”

What This Means For You

It's not currently known when this vaccine may be available to the general public. If it proves to be effective and approved for use several years down the line, you will not be eligible to receive the vaccine if you are breastfeeding or plan to breastfeed in the future. 

What Happens Next?

The FDA’s approval clears the way for the Cleveland Clinic to start Phase I clinical trials of the vaccine. Set to be helmed by George Thomas Budd, MD, staff physician at the Cleveland Clinic’s Taussig Cancer Center, the trials will also include men, who constitute about 1% of all breast cancer patients, Tuohy reports.

Ideally, the trials will help doctors determine whether the vaccine can be safely administered to human beings by providing insight into dose size, dose number, and immunity duration. But it is hard to know exactly when the results might arrive. 

“My team takes [it] one day at a time," Tuohy says. "We hope to start the trials by the end of [the second quarter of] 2021, but we have been wrong so many times with such anticipated timelines. We hope to finish Phase I trials within two years after starting them." He adds that he has "no idea” when the vaccine might become widely available. 

The preliminary results of several animal studies conducted by the clinic have been promising. In one study of female mice, every single one of those that did not receive the vaccine subsequently developed breast cancer and died.         

The trials will be funded by the U.S. Department of Defense and conducted in people who do not intend to breastfeed, Tuohy says. Because the vaccine conditions the immune system to wipe out all alpha-lactalbumin-expressing cells, it would have adverse effects on human milk production. 

The Future of Cancer Vaccine Development 

Cancer vaccines can be divided into two camps: those designed to prevent cancer, like Tuohy’s, and those designed to treat it. The only direct cancer treatment vaccines currently available are sipuleucel-T/Provenge (for the treatment of advanced prostate cancer) and talimogene laherparepvec/T-VEC (for the treatment of advanced melanoma, an aggressive form of skin cancer).

Many breast cancer treatment vaccines are in development, including peptide-based vaccines, whole-cell vaccines, gene-based vaccines, and dendritic cell-based vaccines, but none have yet advanced past Phase II clinical trials.

The candidates hail from organizations all around the country, including the Mayo Clinic in Jacksonville, Florida, the University of Washington in Seattle, and the University of Texas M.D. Cancer Center in Houston, according to Pravin Kaumaya, PhD, professor of translational therapeutics at the Ohio State University Comprehensive Cancer Center.

Kaumaya and his research team are working on several breast cancer treatment vaccines of their own. The furthest along is a HER2-based peptide vaccine that works by manipulating the gene that codes for human epidermal growth factor, which enhances tumor growth, invasion, and metastasis. The vaccine is effective against roughly 30% of breast cancers, but triple-negative breast cancer is not one of them, he says.

Looking Ahead

If the Cleveland Clinic vaccine proves safe and effective, it could “inhibit the emergence of triple-negative breast cancer” either by itself or in combination with what Tuohy calls “other compatible prevention strategies.” That’s not to say, however, that it is a miracle cure for the disease. In fact, it would be naive to think that one vaccine alone would be sufficient to “control [the] emergence and growth” of a cancer of a “particularly aggressive and lethal” variety, Tuohy says.

But he does say that the vaccine could potentially pave the way for further advances in the field of oncology—namely, by pioneering medical technology that could eventually be used to develop vaccines for other cancers. 

“So we envision a 21st-century vaccine program here at the Cleveland Clinic that prevents diseases that we confront with age that we think are completely preventable,” Tuohy told Fox 8. “We think breast cancer, ovarian cancer, [and] perhaps prostate cancer are all preventable diseases, and that’s why we want to bring our vaccine program up to the 21st century.”

For his part, Kaumaya believes that any successful breast cancer treatment vaccine will need to include checkpoint inhibitors—drugs that prevent cancer cells from acting on the immune system.

“The bottom line is for a vaccine to be successful, you’re going to have to incorporate multiple modalities," he says. "And to me, what is most important at this stage is the inclusion of checkpoint inhibitors. That combination of immunotherapies is going to be essential for any of us developing breast cancer vaccines to be successful."

7 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
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By Caroline Tien
Caroline Tien is a journalist with degrees in English and biology. She has previously written for publications including Insider and Cancer Health.