Cancer Research Top Stories: September-October 2013

Are you interested in following developments in cancer research, but feeling a bit challenged to (a) find the time to do a lot of reading and (b) sort out what’s really important? Me too! Nevertheless, I do try to scan through the cancer research news headlines each day.

This is the first post, in what I expect will be a bi-monthly feature, where I’ll briefly review several of the most interesting cancer research stories that have come out over the last two months. These are stories that I know I’ll want to follow as they develop further.

Here are my personal picks for three of the top cancer research stories for September – October 2013.

1) A preventive breast cancer vaccine is expected to move to the first stage of clinical testing

The Cleveland Clinic announced that it will seek Food and Drug Administration (FDA) approval to begin Phase 1 testing of a preventive breast cancer vaccine. This development follows from a study that was reported in the journal Nature Medicine in 2010 showing the vaccine to be safe and effective in animal models.

The vaccine would be used to prevent breast cancer in women at high risk for the disease, such as those with BRCA mutations.  And it could also be used as part of therapy for patients who have received treatment for triple negative breast cancer (TNBC) to prevent recurrence of the disease. There are currently fewer treatment options for TNBC, which also tends to be more aggressive than other breast cancers.

A critical challenge in developing a preventive vaccine has been to find a way to zero in on drivers of a potential cancer without harming normal tissue. This vaccine works by inhibiting a protein that is normally only expressed in lactating mammary cells, but happens to be also highly expressed in triple negative breast cancer cells. It would be appropriate for any woman who is no longer bearing children.

The timeline, however, is not short. Phase 1 trials to determine dosage and safety are expected to begin in about 2 years and take 3 years to complete. These would be followed by larger clinical trials to be conducted over about 5 years.

This post by Kathleen T. Ruddy, MD in The Health Care Blog is a good resource for more detail on the story.

2) New drugs that are designed to remove “brakes” on the immune system are showing long-term benefit for some cancer patients

New findings from a pooled analysis of about 4,800 patients treated with the drug Yervoy (ipilimumab) were presented at the European Cancer Congress 2013 in September. Yervoy was approved for the treatment of advanced melanoma in 2011 both in the United States and in Europe.  The drug works by removing an “adaptation” by the cancer that “brakes” what would be the immune system’s normal attack on cancer cells.

This particular drug only in works in about 15% of melanoma patients, but for those in whom it does work the effect is very significant. The data presented at the conference showed that about 21% of patients who responded to the drug were still alive after 3 years, and 17% were still alive after 7 years. This is very striking for an advanced cancer with average survival after diagnosis previously measured only in months.

This research seems most interesting not just for what it may mean for a very small percentage of melanoma patients right now, but for how it and other related research may be expanded to help a wider range of patients in the future.  The hope is that combining drugs that act in different ways to free the immune system to attack cancer cells would lead to significant benefit for larger numbers of patients with different types of cancer.

“Breaking Through Cancer’s Shield” in the New York Times provides a general overview of current efforts to develop immune system therapies to treat cancer. Additional helpful background can be found in a special section on Immunology in the recently released AACR Cancer Progress Report 2013.

3) The FDA issued  its first approval of a drug as a pre-surgery cancer treatment

The FDA has, for the first time, approved the use of an anti-cancer drug for patient treatment prior to surgery. On September 30, it approved the use of Perjeta (pertuzumab) as part of pre-surgery treatment for patients with HER2+ breast cancer with relatively high risk of recurrence. Perjeta had been approved in 2012 for treatment of patients with advanced breast cancer.

Approving anti-cancer drugs as pre-surgical treatment first, prior to their approval as standard treatment following surgery, offers a possible route to quicker approval of new therapies for treating early stage cancers.  New drugs traditionally have been approved first for advanced cancer, and then tested in large clinical trials over many years in order to evaluate how well they work in the treatment of early stage cancers.

This was also the first time the FDA has approved a new use for a drug based on the extent to which patients experience a so-called “pathologic complete response” in a clinical trial. Patients have a pathologic complete response (PCR) when, as a result of pre-surgery treatment, there is no cancer present in the tissue that is removed surgically or in any sampled lymph nodes.

In this case, a small clinical trial of 417 patients showed that 39% of patients attained PCR with Perjeta and the standard treatment (chemotherapy and Herceptin) compared to 21% with the standard treatment alone. These results will need to be confirmed in a larger ongoing Phase III trial involving 4,800 patients, results of which will be available in 2016.

For more information on this story, take a look at this very informative blog post by Debra Madden, an advocate who served as the patient representative on the FDA advisory panel that recommended the approval earlier in September.

Other Reading

A very pertinent article entitled “The Cost of Living” in New York Magazine discusses the steeply rising prices of new cancer drugs such as Yervoy. It also describes how some of the newer, much more expensive drugs do not necessarily offer correspondingly better treatment outcomes for patients than existing therapies. Some doctors are speaking out, more publicly now than in the past, about the problems of extremely high drug prices.

“How Science Goes Wrong” in The Economist argues that the old adage “trust, but verify” is not being followed adequately by modern scientists and that too many academic studies result from shoddy experiments or poor analysis. It argues for such measures as increased transparency around research protocols and clinical trials data, as well as tightening of peer review.

That’s it for this time. If there was another recent cancer research story or article that really caught your attention, please mention it in the comments!

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