A new drug looks poised to become the first effective treatment for liver cancer, one of the most common and deadliest cancers in the world and one whose incidence has been rising in the United States.
Today at the annual ASCO meeting the results of a clinical trial of 602 patients with hepatocellular carcinoma will be released. The trial compared the oral targeted agent sorafenib to placebo. Not surprisingly, sorafenib was more effective than placebo in producing a benefit for patients. What was this benefit that is getting all the publicity today?
Was it shrinkage of the tumor? Nope - neither the sorafenib nor the placebo pill significantly shrunk the primary liver cancer.
Was the benefit improvement in symptoms? Nope - the incidence of side effects (mainly rash and diarrhea) were to same in both study groups.
What does sorafenib do then, to bring such tears of joy to researchers, if not the company that makes the product?
Survival, man, survival - that's the name of the game when looking for salutary outcomes with targeted agents that don't have much effect on tumor cell kill.
To put it plainly, the group of patients that took sorafenib lived on average 10.7 months (called median overall survival) and the cohort that took a placebo pill lived 7.9 months, a result that was statistically significant and led to the placebo patients being crossed over to receive sorafenib. Here's another outcome that was reported - time to progression of disease:
Time to cancer progression was 5.5 months in the Nexavar group, versus only 2.8 months in the placebo group. The findings were so positive that the study was terminated early. "We recommended ending the trial early because of survival advantages favoring the sorafenib group," said Dr. Joseph Llovet, lead author of the study and director of research in liver cancer at Mount Sinai School of Medicine.
Hepatocellular carcinoma is not a malignancy that tends to appear in healthy people without warning. It is associated with certain conditions, and its incidence is rising:
Most cases of liver cancer occur in people who already have cirrhosis of the liver, a scarring of the organ that in turn can be caused by hepatitis B or C, alcohol abuse, or obesity. Liver cancer has been fairly uncommon in the United States, with about 19,000 new cases expected this year, with nearly 17,000 deaths. But the number of cases has been rising because of an increasing incidence of hepatitis C. And worldwide, there are about 600,000 new cases a year, making liver cancer one of the most common cancers and cancer deaths globally. That is mainly because of the large number of people in the Far East with hepatitis B and C.
600,000 people a year - and the majority of them will die of their cancer. No wonder experts are hailing the onset of targeted therapy as a breakthrough in the treatment of the most intractable malignancies. Let's hope that the geniuses trying to run this country get it through their skulls that progress in the fight against cancer is intimately linked to freedom and democracy, not the converse.
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So the new drug increased the lifespan of the patient on average a little less than 3 months? At what cost?
Three months is the median. Many of those patients lived longer. Perhaps significantly so. A 44 percent increase in survival sounds awesome to me.
So, how many people have to see no benefit whatsoever for one person to see a significant increase? And what is a significant increase? Is it 6 months, or a year? And what is the cost? How much of that time is spent in treatment? What are the side effects? In terms of finance and quality of life what is this less than three moonths costing? Remember, it's costing the person receiving no benefit the same. It just doesn't seem like much to throw a party over.
Are you really expecting to find a medication that works 100% of the time for 100% of the affected population with few or little side effects? BTW: toxicity with sorafenib was shown to be similar to that of placebo in this study. If so, you're going to be disappointed in every study that comes across.
This is a patient population that essentially has NOTHING that significantly improves survival. Based on these findings, not only is there finally SOMETHING, but also a comparator for future meds that could possibly boost survival even higher.
If the investigators saw fit to terminate the study because the results were significantly positive in the sorafenib arm, I'm going to trust that they're on to something.
Hope. There is always hope.
I'm not the one who brought up the spector of 100%, you are. My point is that less than three months on average isn't a whole lot of time for the majority of people who are taking it. The financial costs involved in treatment with cancer drugs is astronomical, and then you have to accept whatever side effects the treatments have. Is an expected 3 months really worth it. It just doesn't seem like a whole lot to get excited about in terms of survival versus costs.
Your reduction of a study that many investigators are finding encouraging implies your expectations of a successful medication are greater than their collective knowledge can deliver.
Yes, the drugs are expensive. Yes, there are often side effects. Yes, the survival is incremental and not across the board. Welcome to the current environment of oncologic research. If you've got some better ideas, I'm sure folks would be interested to hear them.
Again, what is your realistic idea of a treatment success? What is worth getting excited about?
welcome? I've been living survivorship for three years and I do ask if the costs are worth the gain. Suvivorship at any and all costs will sink me and my family. The question is what is to be gained and is it worth the cost?
I think it's a legitimate question to ask about the cost versus survival benefit of the drug, albeit it's a question with no easy answers.
The NY Times article linked to above says the new drug costs $4500 a month. A year's treatment will cost $54000, for an average survival benefit of 3 months. I'm sure for some it's worth it, but I also hope that a person who chooses not to take this drug or who cannot afford it, is not made to feel guilty over this.
I don't care for the NY Times article saying that the drug increases survival time by 44%. I believe these relative comparisons are misleading. Hearing 44% on it's own, without reference to the absolute number of months, is disingenuous. When a person reading the NY Times reads '44% better', they're going to get the impression that this means something along the lines of years, not months.
I'm not trying to belittle the efforts of cancer researchers; I certainly acknowledge that their work is very hard. I am concerned about how these efforts are portraying in the popular media. There are constant announcements of 'a new revolutionary treatment for Cancer X', which are breathlessly portrayed as quasi-miracles that are cures for Cancer X. Then you read/hear further, and it's a $54000 drug that increases survival by 3 months, not a cure at all.
If it's possible, I'd like to see the C.O. give us his thoughts in a post on the costs of cancer drugs, particularly the newer ones.
It's all very well to talk about survival and percentages, but to the person with the cancer it comes down to "will this work for me"? I saw this lady yesterday who has lived with liver cancer for over a year; she's had surgery, radioisotope therapy to the liver lesions, and still the cancer continued its relentless progress. Last month she started sorafenib - and yes it does cost USD4,500 - and it hasn't worked. And she feels guilty for having spent the money, her children's money. But what if the therapy had worked? Then she would have the guilt of having her children find the funds to continue treatment to let her live a few months more. The eventual outcome is the same, she would've succumbed to her cancer anyway, and right now she feels like somehow it's her fault she didn't get better.
Don't get me wrong, discoveries of this nature are very important, as small steps towards eventual big improvements. Look at how the treatment of cancers of the breast, lung and colorectum have changed in the last ten years. But in the real world, where there is no equity in access to healthcare, and finite resources make rationalisation necessary, optimism needs to be tempered by reality. Researchers will say it's not their problem, they provide the evidence, it's up to governments and policy-makers to deal with the rest. And what about the patients?
There was an interesting interview with the head of Genentech in the Wall Street Journal in which he discussed the cost of cancer drugs.
http://online.wsj.com/article/SB118100806142024548-search.html?EYWORDS=…
While the costs of these drugs to patients/ins. cos. are staggering (I was an enthusiastic user of Herceptin) I think the real outrage should be directed at the federal government and their tepid support for basic research. As it stands, these biotech companies have to recoup the costs of their research and yes, it is very expensive. But without the profit motive, I doubt there would have been a Herceptin or a Rituxi, or any of the other nifty drugs that are changing the face of cancer treatment.
The real story on the liver cancer treatment is that after 100 unsuccessful clinical trials, the reserachers finally got something that works. It may not be perfect, three months average survival may not be great, but now the researchers have a new avenue to discover, success to build upon and hopefully, if the stars and financing align, a potentially truly efficacious new treatment for this horrible disease. In five or ten years.
HOWEVER, it also seems that there should be a greater world wide focus on public health, which will never happen, given that the incidence of this disease could probably be lessened dramatically through prevention.
RLR, NC, Jen --
You've all made really clear and valid points. Cost versus survival benefit is a very important issue that is complicated and forces too many people to make difficult choices during a very vulnerable period in their lives.
My pressing responses to emmy's initial comment came from what I perceived to be an outright dismissal of the study findings as insignificant. What is encouraging to researchers is certainly not necessarily, and frequently isn't, encouraging to the suffering patient population.
Further, as RLR points out, the media throwing up headlines that are bound to be misinterpreted by the general public is frustrating to those of us who are living with or have a loved one living with cancer. We want cures now. Not 10 years from now after the disease has already consumed us.
I would love to wake up tomorrow (today would have been good, too) and find out that my disease has a cure. But, I am well aware that there are no homeruns on this field. So, yeah, I applaud the singles. And I hope; it's how I live. My apologies if my postings have offended or hurt anyone.