Hope collides with reality…the promise #fail of new drug research

Research promises, but when does the promise fail?

The hype started in fall, 2011 with a press release from Bayer Pharmaceutical, a German-based pharmaceutical company. Their new drug for colorectal cancer, a multi-kinase inhibitor that was in a randomized Phase III clinical trial for Stage IV patients whose cancer had progressed on all prior chemo regimens, had done so much better than expected that at the interim report phase, the trial was ended early. The participant groups were unblinded, and the patients receiving only placebo and “best supportive care” (symptom management) were offered the drug, called Regorafenib.

The FDA gave this new-hope drug fast-track status based on these results, meaning that if the company’s reporting on the drug was lined up, approval could come much, must faster. And patients who had run out of chemotherapy options, or who, like me, were watching their options dwindle, waited anxiously for the formal reports to spell out the specifics only hinted at in the press releases.

Well, this week the specifics of those preliminary, interim report results were released as part of ASCO’s Gastrointestinal Cancers Symposium 2012 meeting this weekend in San Francisco. Yes, the results are promising. But unfortunately, the results also force hope to collide with reality – an epic promise #fail.

Dr. Axel Grothey, co-principal investigator of the Regorafenib CORRECT trial and professor of Oncology at the Mayo Clinic is going to present the oral abstract session. Over 700 people were randomized onto the Phase III trial, so the numbers looked promising. Hope rose – not only for me, but for many other stage IV patients who are facing dwindling treatment options.

But this week those “statistically significant” and “high” percentages of improvement have been translated into this collision with reality:

Stage IV patients taking Regorafenib experienced approximately 1.4 month longer overall survival (OS) than stage IV patients given only “best supportive care” (BSC) that was designed to manage their symptoms without changing the course of their disease. Stage IV patients taking Regorafenib experienced approximately one week longer progression-free survival (PFS) than stage IV patients on BSC.

When all other drugs have failed, Regorafenib makes it possible to live one more month than people who aren’t taking any chemo at all? One month of increased OS is the hope that has been hyped for six months? One more month of life – and by the way, life where patients on Regorafenib experienced these side effects that were significantly higher than patients on placebo: fatigue 47.4%, hand-foot skin reaction 46.6%, diarrhea 33.8%, anorexia 30.4% , hypertension 27.8%, oral mucositis 27.2%, and rash/skin desquamation 26.0%.

This is the state of hope hyped to Stage IV patients who are running out of options? Really, Bayer? Really?

Yes, it’s a new drug. It works along different (multiple) pathways at the same time, so its chances of having an effect are greater than drugs that work on only one pathway. And as Bayer is now testing in a Phase II trial, using it before the patient has progressed on all other approved drugs, may show even greater benefits in overall survival.

But Bayer, as a Stage IV patient who is now officially “allergic” to oxaliplatin and irinotecan, and who is growing increasingly sensitive to Erbitux – the heavy hitters usually paired up with 5FU or Xeloda in the currently approved CRC chemotherapy cocktails – I really wanted more from this study than a month, or a week, or a boatload of debilitating side effects. Call me crazy, but I’m not willing to sell what little time I may have left without side effects for a whopping month of overall survival, or a whole week before my cancer progresses.

Here’s the thing, Bayer, from a stage IV rectal cancer patient who spent 24 years in pharma research to a company engaged in that research. I want more than one month of extended overall survival. I want more than a week before my cancer outsmarts the drug and progresses. I want a drug that can step up to the plate for me when my sensitivities to Erbitux mean I have to stop taking it. I want meaningful extension of my life without debilitating side effects.

And all of those hopes and wants add up to more than a few days or weeks, Bayer. Just sayin’.

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  1. Eric says:

    Hi Pat, I am husband of a young rectal cancer patient and I follow you closely here as well as on ColonClub.com (slowly opening up to other sources of support as well).

    I do agree the one month is not much, but is that an average? I read recently Avastin increases life expectancy of stage IV patients by 4.7 months and I find it hard to believe anyone would risk all the side effects of Avastin for a mere 4 months of extended life.

    Thinking of you always,


    • PAS says:

      Hi, Eric, thanks for stopping by.
      The numbers that have been released so far for regorafenib, and which I mention in my post, are medians…so yes, sort of an average. Unfortunately a median fails in some ways because it only shows the top of the bell curve. It doesn’t tell you either the low point (least amount of survival time) or the longest point (most amount of survival time.)
      Hopefully, at Feb. 8th’s webinar featuring updates from the 2012 Gastroenterology conference, some of the people who attended and got to see the presentations in person will have some more information to add to what was published so far about this study.
      Be well.

  2. Julia Gallagher says:

    Thank you for your synopsis and opinion. I’m in complete agreement with you. So unbelievably dissappointing.

    I think of you often, Pat, and I sure hope something better comes along for you and my husband before it’s too late.