Two years ago, the world lost an extraordinary person, my dear friend Dr. Brent Whitworth. He was a few days shy of his 42nd birthday when he learned of his diagnosis – stage IV kidney cancer – and he was devastated. He had surgery at a major cancer hospital and then entered a clinical trial for an oral chemotherapy drug. He responded well to treatment at first, but then the cancer grew. He lived for 19 months after his diagnosis, which is probably longer than he was expected to live. The medicine he took was rejected by the Food and Drug Administration (FDA) last year, a year after he died, because it had lower survival rates than the other possible treatments for kidney cancer. But it likely helped him to some degree.
I hear a lot of talk about the importance of “incremental improvements” in drug development, particularly for cancer – in other words, new drugs that provide relatively small improvements in survival over existing treatment options. I am not an expert on these arguments, and I’m not here to debate them. But I would like to put a human face on why incremental improvements are not enough.
One argument is that patients can string together different treatments, each of which may provide some number of months of response, to have a longer cumulative benefit. That may be true for some patients, but for others, like Brent, there is no opportunity for a second line of treatment. He was too weakened and debilitated by the treatment and the spread of the cancer to try another drug option.
I hear some patients say that they hope a drug will help them live long enough for the next advance that will give them more time. That works for some patients, in some diseases where the current treatments provide a long-term response. While the FDA has been approving drugs efficiently with new regulatory pathways, and in some cases the time to approval for new drugs is getting shorter, it’s still a long enough timeframe that small improvements in survival may not be enough to get to the “next big thing.”
Another argument is that treatments can help patients reach a certain milestone in their lives – attending a child’s graduation or wedding, being present for the birth of a grandchild, etc. A treatment that helps patients achieve an important life goal is important. But we have to remember that while someone may be grateful for the opportunity to enjoy these important life events, in the end what patients really want is to be cured or to have a long-term, manageable disease with a high quality of life. While some individuals with cancer may choose to accept the toxicities of cancer drugs for the hope of additional time, others choose less aggressive treatment and higher quality of life.
Brent left a successful business career to go to medical school. He knew that his calling was to be a doctor and care for patients. Being a doctor meant the world to him. He was brilliant and compassionate and thorough. He took the time to get to know his patients, understand their concerns, and get to the bottom of issues that may have gone undiagnosed previously. While he built a successful medical practice, he ensured that he had the time to devote to his patients. Being a doctor was more than just a job to Brent, and it was about more than just science. It was a labor of love. One of his patients said about him, “The time, care and concern you showed each of us made all of us feel like friends, not just patients. That is a gift you have, to make everyone feel special.”
When he was sick, he had to sell his medical practice, which was one of the hardest things he had ever done. He lost not only his livelihood but his identify as a physician and caregiver. He knew that when he was able to return to taking care of patients, he would be an even better doctor. Being a cancer patient had given him a new understanding of what it was like to be sick and place your trust in someone else to care for you. Unfortunately, Brent was never able to return to medicine.
In the 19 months he lived with cancer, he had some wonderful experiences – he found a church community and grew in his faith, he built and tended an amazing garden, he traveled with his partner and spent time with his family and friends. He was grateful for the time that he had.
But he would have much rather had more time and better quality of life. While I acknowledge that the pace of discovery can be slow and that incremental improvements are sometimes all we have, we can’t get complacent and celebrate, or even settle for, small improvements.
Post by NCCS CEO Shelley Fuld Nasso. Follow Shelley on Twitter @sfuldnasso.