Fast-Track The Pink Vaccine

By Dr. Kathleen T. Ruddy

On September 17, 2013, after three years of fervent awareness-raising and pleading for funds, the Cleveland Clinic announced that it has secured the money necessary to bring the preventive breast cancer vaccine developed by Professor Vincent Tuohy of the Cleveland Clinic in 2010 forward to the FDA for permission to begin clinical trials to see if it is safe and effective for use in women.

The vaccine was shown to be completely safe and 100% effective in preventing breast cancer in three animal models (Nature Medicine, May 2010), and was also found to slow the growth of tumors that had already formed.  The vaccine is especially powerful in inhibiting the growth of triple-negative breast cancer, the most aggressive form of the disease with the lowest survival rate. Triple-negative breast cancer lacks estrogen, progesterone and Her2 receptors.  It occurs in approximately 15% of cases and is the kind of breast cancer commonly found in women who carry a BRCA mutation.

The initial clinical trials, called Phase I studies, will be conducted in two groups of volunteers, women with triple-negative breast cancer who have completed their treatment and are free of disease, and women who will be vaccinated shortly before undergoing bilateral prophylactic mastectomy. (The second group will likely be comprised of those, like Angelina Jolie, who carry a BRCA mutation and elect to remove their breasts to lower their risk for cancer.)  The first group of women will be studied to determine the dose and effectiveness of the vaccine; the second group will be studied to make sure the vaccine does not trigger an untoward immune response in their breast tissue.

The vaccine targets an unique protein normally made only by women who are breastfeeding, alpha lactalbumin (ALA).  In the 12 years Tuohy spent developing and researching his vaccine, he discovered that the majority of breast tumors express, or make, ALA.  Priming the immune system with a vaccine so that it attacks any cell that makes ALA is the method by which Tuohy’s vaccine works.  Because the vaccine targets ALA, a protein necessary for successful lactation in healthy women, the vaccine would not be appropriate for use in women who are still in their childbearing years.  However, the majority of women diagnosed with breast cancer in the United States and other western countries are post-menopausal:  at least 60% of the cases of breast cancer in the United States occur in women over 55; thus, Tuohy’s vaccine holds great potential as a preventive vaccine for the majority of women in this country and elsewhere.  Because the vaccine has also demonstrated therapeutic potential (in slowing the growth of tumors that have already formed), it may also prove useful in treating women who already have the disease, particularly women with triple-negative breast cancer for which there are presently no available targeted therapies.

Other scientists have joined the movement toward creating preventive as well as therapeutic vaccines.  Dr. Brian Czerniecki of the University of Pennsylvania has a very effective vaccine to treat women with ductal carcinoma in-situ whose tumors express the growth receptor, Her2.  And two years ago, Dr. Susan Love and the National Breast Cancer Coalition began working with a panel of scientists on the Artemis Project, whose goal is to end all breast cancer in seven years, hopefully by way of a vaccine.  However, only Tuohy’s preventive breast cancer vaccine and Dr. Czerniecki therapeutic breast cancer vaccine have reached bench-to-bedside potential, ready to begin clinical trials in human subjects.

While it’s true that not everything that works in mice works in women, everything that currently works in women first worked in mice.  Therefore, hope for this vaccine must be soberly balanced with skepticism that it will not fulfill its promise.  (Alas, we woman are used to that!)   But since so much is at stake – millions of lives, millions of breasts, billions of dollars – the possibility of developing an effective preventive breast cancer vaccine has rightfully captured the imagination of scientists, the financial commitment of institutions like the Cleveland Clinic, and the growing support from a groundswell of women who clearly prefer prevention to the rigors of a cure.

The Pure Cure for breast cancer (prevention), as I like to call it, is my mission.  Let’s see if we can hit our target with this vaccine.  Fast-tracking Tuohy’s vaccine through the FDA so that clinical trials can begin as soon as possible is now my objective – to see if it’s safe and, if safe, to see if it works.  Opinions may vary about its potential – they always do – but fortunately, clinical trials are the arena in which final arbitration of a scientific hypothesis takes place, and now we have the money to do just that.

The gruesome data on the global burden of breast cancer (in 2013 alone, 1.5 million new cases and 500,000 deaths) provide the most compelling arithmetic for moving forward, reasonably but quickly.  For those who have no head for figures, allow me to simplify this mountain of dreadful data:  somewhere in the world a woman is diagnosed with breast cancer every 20 seconds and another one dies every minute.   On the other side of the equation we have this dismal reality:  under normal conditions, it takes two years to obtain approval from the FDA for permission to test a new vaccine in Phase I studies.  And then it takes another three years to complete them.   So, let’s multiply by 5, the number of years (under normal conditions) it takes to know if a vaccine is even safe, let alone effective:  7.5 million new cases of breast cancer worldwide and 2.5 million deaths.  That’s what happens worldwide while we’re waiting.  I say, given the crushing weight of this pandemic of breast cancer pressing in upon us from every landmass on the globe, Tuohy’s vaccine should be fast-tracked through the FDA in the same manner HIV drugs were in the 1980’s.

In the meantime, there’s no further need to argue about the potential value of this preventive vaccine.  It’s time to allow the harangue to give way to the scientific method, the final arbiter of what we propose to be true – in this case, that Tuohy’s vaccine is safe and effective for use in women.

Let the vaccine speak for itself.  Put it to the test, and “let slip the dogs of war” (Julius Caesar, Shakespeare) to prevent breast cancer if we can, and as soon as we can.