As I noted 12 weeks ago, cancer statistics are both staggering and disappointing. Although cancer deaths per 100,000 people have been on the downswing since 1991 thanks to access to more effective medications and better awareness about the negative health effects of smoking, there is still a lot of research and progress yet to achieve. My focus in this 12-week series is to bring to light both the need for continued research in these fields, as well as highlight ways you can profit from the biggest current and upcoming players in each area.

Over the past 11 weeks, we've looked at the 11 cancer types most expected to be diagnosed this year:

Today, we'll turn our attention to the projected 12th-most diagnosed cancer: pancreatic cancer.

The skinny on pancreatic cancer
Last, but certainly not least, in this final installment of "Tackling Cancer" is pancreatic cancer, which just might be the most virulent of all the cancer's we've discussed. Although it will be diagnosed in "only" 45,220 people this year, pancreatic cancer is forecast to claim the life of 38,460 people, making it the fourth most-deadly cancer for both men and women. Although there are two types of pancreatic cancer -- that of the exocrine glands (those that make digestive enzymes) and endocrine glands (the glands that produce hormones like insulin) -- exocrine tumors make up about 95% of all diagnoses.

While pancreatic research funds are booming, the long-term survival results clearly aren't. In 1975-1977 the five-year survival rate for pancreatic cancer was a paltry 2%. In the three decades since then, the five-year survival rate has climbed to only 6% -- and that has more to do with awareness over the risk factors than actual improvements in the quality of care!

The troubling aspect of pancreatic cancer is that there are no diagnostic tools beyond CTs and MRIs that help screen for this type of cancer. Researchers have determined that obesity, smoking, and genetic mutations (such as testing positive for the BRCA2 gene) are three factors that can, without question, increase your risk of getting pancreatic cancer. Another factor is simply getting older as age increases the likelihood of getting this disease. The disease has also been shown to afflict men more often than women and appears to be more prominent in persons of African-American descent according to the American Cancer Society (link opens PDF file).


Sources: Surveillance, Epidemiology, and End Results Program, and National Center for Health Statistics. 

Without any reasonable diagnostic tests available, only 20% to 25% of all pancreatic cancer is caught in the localized stage. Even then -- when surgery is still an option -- the five-year survival rate is a meager 23%. As you can see from the staging of the disease, the treatment options need to get better because these five-year survival rates aren't the least bit encouraging:


Source: American Cancer Society; data from Bilimoria. 

Where investment dollars are headed
Surgery is the only outcome that offers any sort of curative option for pancreatic cancer patients -- although, as I mentioned, only 20%-25% of patients even fall into the eligible category. For the remaining patients, a combination of chemotherapy, radiation, or targeted therapy is often used to combat this difficult to treat disease. Here are some of the most commonly used therapies:

  • Sutent: The FDA approved Pfizer's (PFE -0.36%) oral capsule Sutent in 2011 to treat advanced pancreatic neuroendocrine, or pNET, tumors. In trials, Sutent delivered progression-free survival of 10.2 months, which was nearly double that of the placebo at 5.4 months, although no complete responses were observed. This is just one of three diseases Sutent is approved to treat. 
  • Afinitor: Developed by Novartis (NVS 0.63%) and also approved in 2011 to treat pNET tumors, Afinitor was tested in a 410 patient trial of which 207 received the new treatment. Afinitor delivered very comparable results with Sutent -- progression-free survival of 11 months, compared with just 4.6 months for the placebo. However, an interim analysis done on overall survival when Afinitor was approved in 2011 yielded no statistical significance. 
  • Tarceva + Gemzar: This drug combo of Roche and Astellas Pharma's Tarceva with Eli Lilly's Gemzar was approved in 2005 as a first-line treatment for metastatic pancreatic cancer. The combination of Tarceva with Gemzar, in trials, delivered a PFS of 6.4 months, compared with six months on the dot for the Gemzar plus placebo arm. In cases of exocrine tumors, this combination is used very frequently. 
  • Folfirinox: This potent chemotherapy mixture combines 5-FU, leucovorin, irinotecan, and oxaliplatin to treat advanced stages of pancreatic cancer. Folfirinox can be used to treat multiple types of difficult cancers, but low tolerability among patients with advanced disease negates many of the potential PFS benefits it can offer.

Just as we've witnessed in numerous other cancer types, while we've had successes there has also been quite a slew of failures. Perhaps the most notable in recent history is Clovis Oncology (CLVS), which reported in November that its experimental late-stage pancreatic cancer drug CO-101 didn't provide a statistical benefit over Eli Lilly's Gemzar. The drug was scrapped, and Clovis lost its leading drug candidate.

What's coming down the pipeline
Now that you have a better idea of what the current treatment picture looks like for pancreatic cancer patients, let's have a look at what's coming down the pipeline that could revolutionize treatment in the near future.

  • TH-302: Developed by Threshold Pharmaceuticals (NASDAQ: THLD) and being combined with Gemzar in trials, late-stage study enrollment is currently under way. TH-302 is a DNA alkylator that targets hypoxic cells (those with low oxygen). Healthy cells receive normal blood flow while tumor growth can vary leaving certain areas of the tumor "starved" for oxygen. TH-302 will hone in on these hypoxic cells and target the tumor this way. In mid-stage trials, TH-302 with Gemzar delivered a 41% reduction of risk for disease progression or death while also increasing median-PFS by 2.4 months.
  • Abraxane: Celgene's (CELG) wonder drug Abraxane may be on the path to yet another FDA-approved indication based on its trial results. When combined with Gemzar, Abraxane delivered a median PFS of 8.5 months compared with the Gemzar only placebo, which had a median PFS of 6.7 months. The risk of death was 28% lower for those taking Abraxane in trials, yet after two years, just 9% of patients in the Abraxane + Gemzar trial arm were still alive.

Your best investment
If we've learned anything from this romp through pancreatic cancer treatments, it's that we need all the new treatments and research dollars we can get. Clearly, PFS is improving with many ongoing trials, but we're still a long way from having any semblance of control over pancreatic cancer.

From an investment perspective, I think both experimental candidates hold the most promise in the sector, even though Pfizer, Novartis, and Roche all have an FDA-approved drug already on the market.

Threshold's TH-302 is certainly one of the most exciting cancer-fighting agents imaginable. Being able to target hypoxic cells TH-302 should leave healthy cells relatively intact, and it could hold promise across a bunch of different cancer types -- not just pancreatic cancer. Understandably, a lot is riding on TH-302 for Threshold, so the risk versus reward is incredibly high here, and only the most biotech savvy investors should even consider this as a viable investment.

Celgene, however, has a well-established pipeline of which Revlimid and Abraxane are its core. Abraxane, which is already approved as a first-line treatment for non-small-cell lung cancer and as a treatment for metastatic breast cancer, looks very likely to add pancreatic cancer to the list. Celgene offers one of the most balanced portfolios among biotechs and would be the conservative investor's best choice in this space.

Stay tuned next week, when I wrap up this "Tackling Cancer" series by selecting my favorite established and pipeline candidates among the 12 cancer types we've examined.