1. Harpham, Wendy S. MD, FACP

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Understanding Promising Unproven Cancer Therapies

Dear Patient,

Wendy S. Harpham, MD... - Click to enlarge in new windowWendy S. Harpham, MD, FACP. WENDY S. HARPHAM, MD, FACP, is an internist, cancer survivor, and author. Her books include newly published

This handout reviews a few key facts about promising-but-unproven therapies. Knowing what those therapies offer-and don't offer-enables you to determine whether to consider them as options for you. Then you can make the best decision for you.


What are "promising unproven therapies"?

"Promising" treatments have shown the desired results in experiments done in test tubes and in animals. Some new therapies have shown effectiveness in studies done on people who had different diseases than yours. "Unproven" therapies have not yet been tested in patients with your type of cancer. Until those studies are done, nobody knows whether they help, harm, or make no difference in patients like you.


Why should you consider standard cancer treatments first?

Any standard therapy with FDA approval for treating your type of cancer has been proven effective in patients like you. In general, standard cancer therapies offer the best chance for improvement. We can determine which standard therapies, if any, are options to treat your cancer by assessing the risks and benefits of each in your situation.


What's the role of clinical trials?

Clinical trials are research studies in people. Trials are the only way to find safer, more effective treatments. Clinical trials can be considered as an option at every stage, including while patients have standard therapy options. To learn what they are and whether they might be right for you, go to


We can find out if any clinical trials are available to consider. Talking about trials does not obligate you to enroll in one. Clinical trials are not for everyone. For some patients, they are the best option.


Will enrolling in a trial increase your chance of recovery?

We don't know. Only after the studies are completed can anyone know if a promising new treatment helps, harms, or makes no difference in treating the condition under study.


The promising treatments used in trials are based on sound science, which gives everyone reason to feel hopeful of your recovery. That said, feeling hopeful is different than knowing you've increased your chance of recovery.


What about trying a promising-but-unproven therapy outside a clinical trial?

It's not the same. For one thing, patients treated outside trials are not contributing to progress in cancer care, even if the new treatments are administered the exact same way by the same physicians who run the trials. Outside a trial, what happens to you is not included in any useful analysis and does not help new therapies through the FDA-approval process.


For another, patients treated outside trials by physicians not involved in the research may provide care that differs in important ways because they would not be subject to the stringent review-board oversight required in trials.


How does uncertainty fit in?

Every patient's outcome is uncertain. If you receive a treatment that helps 90 percent of patients like you, we cannot predict whether you will improve or be one of the 10 percent who are not helped.


Is the uncertainty of standard therapies different than that of promising-but-unproven therapies?

Yes. When you try a standard therapy, we know it helps a certain percentage of patients like you. The problem is we can't predict if it will work for you. In contrast, if you try a promising-but-unproven therapy, nobody knows whether it helps, harms, or makes no difference for any patients like you.


How might emotions keep you from making wise treatment decisions?

Normal fear and sadness may make it more difficult to learn what you need to know about all your options. Maybe you're feeling pressure from family and friends to try (or not try) a new therapy.


If you are a "fighter," declining unproven therapies may feel like "giving up." It's not. There are many ways you can fight cancer-including optimizing your quality of life in whatever time you have.


If you feel the urge to "do something," keep this in mind: "Doing something" really means "doing something to help." With unproven therapies, you don't know if you are helping your chance to recover. With hospice, you know you're helping you live your best quality of life today.


How do you decide if you want to consider a promising-but-unproven therapy?

No matter which treatments you're talking about, wise treatment decisions are made the same way:


* List all your options.


* List the pros and cons of each option.


* Weigh the pros and cons by thinking about what matters to you.



What are pros and cons of trying an unproven therapy, if the only other option is hospice?

Two major pros are 1) it may work and lengthen your life, and 2) you can avoid talking about hospice if you want. The two big cons are 1) it may cause harm that shortens your life, and 2) you will deal with scans and blood tests, side effects of treatment, and doctor visits. It's important to consider all the big and little pros and cons of unproven treatment and of hospice.


What steps can help you make the best decision?

Decision tools and professionals (such as social workers) can help you work your way to the best decision. These two mental exercises may help, too:


* Think about each option. Which choice would you regret more if you had the most likely outcome for each option? What if you had the worst outcome for each option?


* Prioritize your many hopes. Naturally, you want to hold on to hope of treatment helping and hope of optimizing your quality of life. Only one of those hopes can drive your decision, so you have to prioritize: which hope do you hope for more?