Questioning clinical trials
I received a note elsewhere this morning from someone who had lost her brother to cancer, but felt it was the treatment that killed him, not the cancer. She questioned whether clinical trials did anything beyond making the medical establishment wealthy–and wasn’t that a cause for concern?
I have a friend who has the same cancer Jane did.
She also felt that the number of cases of cancer was on the rise and that, despite all the trials and effort, we had made few strides toward finding a cure–that, if anything, we were worse off today than we had been in the past.
Three levels of trials
Trials are experiments. By their nature, they involve some risk. This is why they are not generally offered to people until after they have undergone the standard treatments–if they exist–and have not been cured.
She had lost her brother to cancer…
There are three levels of trials before a drug is approved for use in the US. Phase I trials are very small and are done in part to demonstrate that the drug does less harm than the disease. Phase II trials are slightly larger and are about trying to find out if the treatment works. Phase III trials are the final step before going to the FDA for final approval. They exist to demonstrate the treatment works in a significant number of cases and to find out what the common side effects are.
Impact of trials
My sister is alive today because she was able to get into a drug trial for her aggressive, late Stage 3 breast cancer more than 15 years ago. My sister-in-law benefitted from those experiments when she was diagnosed with an equally aggressive breast cancer five years ago. She, too, is still alive and cancer-free. Today, there are forms of breast cancer we can cure because people took those risks; there is a form of lung cancer we can cure because some people took those risks.
Trials are experiments.
Childhood leukemia in the 1940s was nearly always fatal. Even in 1980, 80 percent of those diagnosed with it died. Today, close to 90 percent go on to live full and productive lives. That change is a direct result of people going through trials–not all of which worked, unfortunately.
Persistent problems
The number of cancer cases does seem to be increasing. Part of that increase is that we are getting better at diagnosing some forms of cancer. Part of that is the increasing number of toxins we pump into the environment that mutate our DNA. Sometimes, a particular treatment does lead to new cancer some years down the line–but that appears to be relatively rare.
Childhood leukemia in the 1940s was nearly always fatal.
When I graduated from high school, if you got a cancer diagnosis, about all you could do was get your affairs in order and prepare to die. Sometimes surgery, radiation or chemo worked–but it was relatively rare. Today, more than 60 percent of those diagnosed are still going concerns five years or more later. That is a direct result of people being willing to take part in trials–even though those trials were sometimes painful and often carried their own risks.
The truth about progress
When Jane was in the hospital for her heart surgery, the woman in the next room was there for the same operation. The drugs used in the mid ’90s had eventually destroyed the valves in the right side of her heart. In 2010, they replaced those valves. Her recovery from that operation was still uncertain, but without that therapy, she would not have had those 15 good years–not seen her children graduate from high school, not seen them marry, not have gotten to hold her grandchildren in her arms.
Sometimes surgery, radiation or chemo worked…
I’m sorry for what happened with my correspondent’s brother. I know what chemo’s immediate aftermath can look like–and I would wish that on no one. But his heroism is part of why we can say to many patients today there is hope for them.
The truth about trials
Not every trial is successful–and not every successful trial results in a cure for every patient. Every cancer, it turns out, is genetically different–and that complicates the search for cures in ways no one could imagine even 20 years ago. What works on one form of lung cancer doesn’t work at all on other forms. What works on one form of breast cancer doesn’t work well–or at all–on others. And what works on the breast cancers we can cure, generally doesn’t work at all on other forms of cancer.
I’m sorry for what happened with her brother.
Jane died 47 months and three days ago. We knew from fairly early on that the only hope she had was entirely experimental and would involve–potentially–four major operations over a two-year period–one of which had never been done on a carcinoid patient. No one has ever beaten her form of cancer–but she was determined she would be the first.
Taking risks
Jane was a woman of both science and compassion. She knew she was facing long odds, but if her efforts could save even one other life, she was willing to endure whatever she needed to. In the event, she never came out of the hospital after her heart operation–not because the heart operation failed but because of complications no one saw coming. The result was that the medical community has now changed the protocols for carcinoid patients undergoing heart surgery. Her doctors told me a year ago that what they learned from Jane’s case had saved at least three lives in their operations already–and who knows how many more elsewhere.
…not every successful trial results in a cure for every patient.
The liver ablation that was next on the docket is now being used frequently on patients with her form of cancer because others were willing to take the risk to pursue that treatment. It is not a cure–by any means–but it buys patients time and eases the horrific symptoms of carcinoid/NETs.
The value of trials
Medicine offers no miracle cures. Everything we learn comes from years of research–and from patients willing to take the risk of being human guinea pigs on the front lines of the war on disease. In broad terms, the results are staggering: in our lifetime, polio has gone from an annual terror to a disease virtually unheard of in the US; small pox is dead on a global scale; life expectancy in the US has increased by nearly 20 years; globally, we have a handle on AIDS; and cancer, while still a deadly killer, is no longer the death sentence it nearly always was in every case.
She was a woman of both science and compassion.
We have a long way to go in the fight against cancer. While we have cures for some, there are too many we don’t have cures for–or even a good idea of how to proceed.
There are moral problems…
And there are problems with unscrupulous drug companies and researchers more concerned with profits than with human beings and the diseases they face. And, yes, that concerns me.
Medicine offers no miracle cures.
But every time I meet someone with a currently incurable cancer–hear the story of what they are going through, and what their families are going through–I know that we have to keep trying: keep researching, keep learning, keep experimenting, keep doing the trials and keep taking the risks.
What heroes fight for
I have a friend who has the same cancer Jane did. She has two little boys and a husband she loves and who love her. She has enormous courage. And she is just one of tens of thousands who faces what Jane faced every day.
…I know that we have to keep trying…
That will not change except through the work of doctors and researchers and patients like Jane who are willing to take the risks so that others can live full lives.
