Are Cancer Clinical Trials becoming more successful?

First you’ll need to define ‘SUCCESS.’
 

According to numerous studies and reports, Cancer Clinical Trials have a very low success rate—somewhere in the 5% to 7% range of all trials that are proposed. This is from Phase 1 (or pre-phase 1) to eventual FDA approval. This is not so good except that without even that modest success rate many of us Cancer Survivors would not be surviving.

Success is often defined in terms of producing an actual positive impact in treating and managing cancer and increasing cancer survival. Check out the National Cancer Institute site for more details. 

So are Cancer Clinical Trials Getting More SUCCESSFUL? YES or NO? 

If SUCCESS means more experimental results leading to new and effective drugs—Then NO. (Success rates have not improved in recent years­­­­­­—they have actually decreased)

If SUCCESS means more people are volunteering to participate in trials—Also NO. (Some potentially successful trials never even get started due to lack of participants)

If SUCCESS means the trial stages and FDA approval procedures are progressing more quickly—Definitely NO. (Final FDA approval can take many years)

If SUCCESS means ‘affordable’ prescriptions for cancer patients—Still NO, you’ve got to be kidding (Some well-insured patients are not even able to afford the co-pays)

If SUCCESS means weeding out the least promising drugs in the early stages—Mostly YES. (Ineffective or dangerous drugs can be replaced with new, more promising experimental drugs)

If SUCCESS means there will be some effective new drugs approved for patients—YES, but at a rather slow pace.

Over the years we have encouraged our readers to volunteer for clinical trials when given the opportunity. Your participation could help you, others, and add to the cancer information base. Many different types of information can prove to be useful in the study of cancer.

For example, this author, now with Metastatic-late stage prostate cancer and in a Chemotherapy regimen, is still participating in several clinical trials including a study of  the effect of pain in cancer patients, how cancer affects quality of life, relationships, etc.

Cancer Clinical Trials information and data can be useful even when a particular trial is not successful in curing, treating, or managing cancer. For example, side-effects you experience may provide information to help others avoid or manage the same side-effects, allowing a promising trial to continue. 

Progress in the whole Cancer Clinical Trials world depends on multiple factors. You have the chance to influence some of these factors by participating in trials and surveys, sharing medical records, sharing your cancer experiences, and encouraging and supporting others. Keep on keeping on and never lose your sense of humor.

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Advanced Prostate Cancer--Trials Don't Live Up to Expectations

 Dr. Tom Beer and Dr. Joshi Alumkal, of Oregon Health and Science University,  published an editorial in the July 12, 2016 issue of the Journal of Clinical Oncology questioning the value and accuracy of recent Clinical Trials using Docetaxel (a chemotherapy drug) to control symptoms and prolong life in metastatic castration-resistant prostate cancer (mCRPC) patients. Here are some of the topics they covered.

Since 2004 numerous Phase III clinical trials have used Docetaxel as one part of clinical trials looking for survival advantage in men with advanced prostate cancer. More than half of the trials failed to produce positive results and others produced very weak results. The authors explore how and why this has happened.

Some trials produce 'statistical' success (e.g. significance)  but not much 'clinical' benefit (e.g. a survival benefit of only a month or two). With such a high rate of Phase III failures, it makes sense to change the design of these clinical trials. The authors suggest:

1    Include the patient in the planning process. What do these men want and expect? What would be a positive outcome for them? What would make them want to volunteer for a clinical trial?

2    Identify and focus on patients most likely to benefit from a particular drug or treatment. Then design small studies including only those patients.

3    Determine what success is--improvement in survival, better quality of life, lower risk of toxicity, etc.

There have been too many failures in mCRPC phase III clinical trials to justify continuing the same procedures again and again. Trials are costly, patients are exposed to potentially toxic agents with little benefit, and marginally effective drugs may be moved toward approval. Studies should focus on providing the greatest likelihood of significantly improving the lives of patients.



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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

The World’s Largest Cancer Clinical Trial

The creation of cancer treatment drugs often follows a similar path. Somewhere a researcher or research team independently or as part of a drug company invents a potentially effective drug and a drug company sponsors clinical trials. After many trials at several levels, involving hundreds and maybe thousands of patients, some of the drugs are FDA approved. Conducting clinical trials is expensive and the successful drugs are eventually sold to patients at very high prices. You already know this, of course. But this new clinical trial from the UK has a different twist.

Clinical trials on the cheap

The BBC has reported that Cancer Research UK and NIHR (part of the National Health Service) will be sponsoring a 12-year study of the cancer prevention benefits of ASPIRIN. Yes, common aspirin. It is called the Add-Aspirin Trial. 

There has been some research to suggest that aspirin can delay or slow the growth of some cancers. This trial will help determine if aspirin can be an effective cancer treatment. A proven inexpensive cancer treatment would be a major benefit to millions. 

11,000 people with early stage cancer—the largest number ever in a clinical trial‑‑will be randomly assigned to one of four groups. One group will take a daily placebo and the other three groups will each take aspirin at a different dosage level. This part of the study will last five years. Participants will be monitored for as long as 12 years. Patients with early stage bowel, breast, prostate, stomach, and esophageal cancers will be included in the study. 

Aspirin is one of the cheapest meds on the market. It was originally developed as a painkiller but is now widely used to help reduce the risk of heart attack and stroke. Maybe there is one more chapter in the aspirin story. 

Aspirin has several documented side effects—as do all drugs‑‑ and would not be appropriate for everyone.

Keep an eye on this study. Not all clinical studies have a good outcome but, if successful, this one could be a ‘game changer.’

Post Text Here
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Cancer Clinical Trials and the Elderly—an Unlikely Combination

Did you know?

 

Q         What age group is most likely to have Cancer?

A         People older than 65. They represent 13% of the population and more than 60% of all those with cancer.

Q         Who is most likely to die of cancer?

A         Seniors. About 80% of all cancer deaths occur in those older than 65.

Q         What age group is least likely to be represented in cancer clinical trials?

A         Seniors. 

Q         Can seniors respond to cancer medications in a different way than younger patients? (For example, having more toxic side effects.)

A         Yes

Q         So, WHY are seniors with cancer not included more in clinical trials?

A         It’s a long story……

Improving the research base for treating older adults with cancer was a focus of the 2015 ASCO conference. An expert panel presented the problem and possible solutions. The Journal of Clinical Oncology reprinted the complete report and recommendations.

Factors such as meeting eligibility requirements can create hurdles. Many older patients are limited in mobility, have numerous previous treatment regimens, have a more limited predicted lifespan, and take multiple medications (the average is more than 9 for patients over 65). Multiple medications can interfere with trial medications. More than 80% of older patients have a chronic condition (such as diabetes or arthritis) which can complicate cancer treatment. 

Doctors often do not even recommend older patients for inclusion in clinical trials, although Doctor recommendation is the number one predictor of older people choosing to participate in clinical trials.

Many oncologists, such as Stuart Lichtman, MD, Memorial Sloan Kettering Cancer Center in NY, believes that seniors with cancer are being short changed when it comes to cancer treatment because they are under-represented in research.

ASCO makes five recommendations to improve evidence generation in the 65+ population:

(1) Use clinical trials to improve the evidence base for treating older adults (older than 65) with cancer,

(2) Leverage research designs and infrastructure for generating evidence on older adults with cancer (change trial requirements to fit older patients),

(3) Increase US Food and Drug Administration authority to incentivize and require research involving older adults with cancer,

(4) Increase clinicians' recruitment of older adults with cancer to clinical trials (educate doctors to recommend older patients for trials), and

(5) Use journal policies to improve researchers' reporting on the age distribution and health risk profiles of research participants.

Doctors currently have no choice but to use results from clinical trials conducted with people in their 50s to treat people in their 80s.

Research results from clinical trials with older patients could provide a better scientific basis for treating cancer in the elderly. As someone who is elderly and has cancer, this author wholeheartedly supports these recommendations.

Click on the links in this article for a more comprehensive discussion of this issue.

 

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

A Blog is a Blog is a Blog…

Or is it??? 

This blog was started in 2012 just after our book, Cancer Clinical Trials, was published. Our goal was to provide information about clinical trials, new meds, FDA approvals, how to participate in clinical trials, and other information related to cancer clinical trials. We have tried to be positive, encouraging, and occasionally make you smile. This reflects our outlook on life. Our blog entries are primarily aimed at those with cancer, those thinking about participating in a clinical trial, and the family and friends of those with cancer.

Some have questioned our inclusion of humor. Is there a place for humor in this serious subject? Cancer is not funny, of course, but a positive attitude, sense of humor, and ability to laugh may make life a little easier. This author, Larry, has been living with prostate cancer for 12 years. Being able to laugh at myself, the idiosyncrasies of the medical world, high drug prices, endless forms to fill out, strange side effects, etc. has made my quality of life a little better. We have been pleased to receive positive feedback from many blog followers.

There are literally millions and millions of blogs out there somewhere. There is something for everybody. They run the range of the very scientific, factual and useful information of all sorts, entertainment, political (not necessarily factual), personal diaries, pictures of cute kittens, and probably more than a few X-rated and nefarious ones. The odds are good that you or someone you know has a blog.

Cancer is the topic of a lot of these blogs. Many are dedicated to specific cancers (breast, lung, prostate, skin, and on and on). Then there are the personal information and experience blogs which are not always factual (we hope ours is), and all are infinitely varied. We hope you will follow our blog, but you may find others you prefer. The advantage of having so many choices is that you’ll surely find just what you’re looking for. 

We will continue to provide cutting edge cancer and clinical trial information, a few personal anecdotes, and a bit of humor‑‑often in the form of G-rated cartoons.

 Post Text Here
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

One Size Fits All—Right Now, But, What If…

The typical clinical trial today involves hundreds or even thousands of volunteers who are fairly similar (have the same cancer with similar stage and similar prior therapy and maybe even similarities in general health). Then two treatments are compared to each other in this large group of patients. If one treatment fares better than the other, that better treatment becomes the new gold standard treatment. That’s good. That’s progress! But it’s certainly not perfect.

So what about the individual patient?

What is lost in this process is knowledge about how these treatments affect individual patients; for example, you. The average benefit seen in a clinical trial is not shared equally by all participants. Some patients are fortunate to have cancers that are particularly responsive to available treatments and these patients may benefit a great deal. Others may benefit a little less and some may not benefit at all. A few may even be harmed by the treatment as they experience unpleasant side effects along with little or no effectiveness.

A clinical trial treatment is judged by how it performed in the entire group of participants and not as much by how each individual patient responded. Over the years, this approach has saved countless lives. In most cases it’s the best we have at the present time. What about the future? What’s on the horizon?

The Big Three New Directions

1 Targeted Therapy. More and more cancer drugs are designed to target specific defects that occur only in cancer cells and not in the normal human body. This is the same process used in creating antibiotics that attack the bacteria and not the human. As a result, antibiotics have been enormously successful. It has been far more difficult to figure out what makes a human cancer cell uniquely different from the regular human cells that it came from. But drugs are now being designed to exploit these newly discovered Achilles’ heels of cancer. For example, Imatinib Mesylate (aka Gleevec) (developed at OHSU) was one of the first such drugs to become mainstream, revolutionizing the treatment of Chronic Myelogenous Leukemia as well as several other cancers.

Targeted drugs have a good chance of being both more effective and less toxic than most drugs currently in use. Targeted drugs are also more amenable to individualized therapy. Since these drugs have a specific target, it is possible to develop tests that examine the cancer and determine if that particular target is present in a specific, individual patient.

2 Personalized Therapy. More clinical trials than ever before are asking patients to consider a biopsy of their cancer as part of the research. For cancers that circulate in the blood this may be possible with just a simple blood sample. In other cases a needle biopsy might be needed to get a sample. Don’t be surprised if you get such a request when you’re contemplating a clinical trial. These tumor samples are being used to better understand which cancers respond to which treatments. Fear of needles aside, this is a good and progressive step in conducting clinical trials and providing benefits for all those with cancer!

It is likely that in the future the multi-thousand patient clinical trials that seek to measure benefit in the entire group may be replaced by smaller studies that focus on subgroups of cancers that have specific treatable defects.

3 Pharmacogenomics. Not only can we not now tell in advance who will benefit from which treatment, we are equally unable to predict who will develop serious side effects. Matching drugs to cancers will require careful biologic analysis of the tumors. Predicting, and therefore avoiding, side effects will require a careful biologic analysis of the whole human being.

While our individual differences are not due only to genetics, many of our differences and those things that make us unique are coded into our DNA. We expect that hidden within that code is the ability to predict how the body will react to various medications and treatments. In the future, we hope to be able to perform very sophisticated laboratory tests that will enable us to predict which treatments will result in the greatest benefit and do the least harm to each individual patient‑‑and for minimal cost. Let’s hope this all happens sooner rather than later!

Post Text Here
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker