The (not so happy) Story of M (Part I)

Or… when is enough treatment enough?

A couple of weeks ago, I shared the story of my patient, K . Today’s story does not have the same happy ending. The case caused me to feel uncomfortable trying to balance my own beliefs against the wishes of two clearly loving parents, and I thought that I would share it with everyone who is reading this, and generate a bit of discussion about when to stop treating.

M is a 12 year old boy, and he was diagnosed with osteosarcoma of the right tibia in Kobe, Japan in 2005.

He was treated in the usual way, with chemotherapy followed by surgery to remove his tumor and replace his knee. Unfortunately, during the 2 months he was not receiving chemotherapy because of his surgery, he developed numerous metastases to other bones. He received 4 courses of intensive chemotherapy followed by a bone marrow transplant, but his disease did not respond. His doctors tried a different chemotherapy regimen, also without success. In October 2006 he was enrolled on a tumor vaccine trial in Osaka, but his cancer grew despite this treatment. Two further cycles of chemotherapy accomplished nothing. Desperate, his parents began to scour the world for novel therapies to try, and they stumbled onto a clinical trial that I am running. That’s how M came to be my patient in May of this year. Sadly, his response to my clinical trial was no better than his response to anything else he has been treated with.

Here is where the ethical difficulties begin.

M is now in the US, thousands of miles from home. His family has been told that there is nothing curative to offer him, and that they should take him back home to Japan for hospice care near his home, surrounded by friends and family. That advice, of course, comes from the medical team, and is certainly not what the family wanted to hear. The family has already shown a willingness to go to any lengths to find a treatment for their son. They are asking if there are any Phase I trials going on that he would be eligible for, because if so, they want to stay in the US and participate.

Let me step back and explain Phase I trials. Drug development in the US takes place in phases. In Phase I, a clinical trial is conducted to learn what is the best dose of the drug to give a patient. In Phase II, the goal of the trial is to learn what tumors are affected by the drug, and in Phase III the goal is to compare a new drug to either placebo or an old drug to see which works best. Because of the way Phase I trials are designed, most patients will not get the optimal dose (when the trial begins, the optimal dose is not known), and no one knows which types of cancer are affected by the drug. Because of this, it is estimated that only 3% of patients enrolled in a Phase I study get any clinical benefit. That’s great if you’re in the lucky 3%, but the overwhelming 97% of patients do not benefit from being in Phase I study.

So… the dilemma becomes what to do with M.

His family wants to keep trying new things. His doctor at home and I both think the time has come for hospice care. He is thousands of miles from home, in a foreign country, and he doesn’t speak the language. Should I do what the parents want, and enroll him on a Phase I trial, knowing how unlikely it is that he will benefit? Or should I refuse, essentially over-riding the family’s request, and not enroll him? If I do that, will they go home and get hospice care? Or will they take him to another American center for a trial that is unlikely to benefit him, but will increase the chance of him dying in pain?

There is a cross-cultural dimension to this case that makes it complicated. Here in the US, we are used to the idea that the physician’s role is to present options to a patient or family, and help them decide what the best treatment is. The physician’s role in Japan is different. Through the help of an interpreter, they were very explicit that they wanted me to tell them what they should do (as long as my suggestion wasn’t hospice care), because that is what they are used to. This, then, is the cross-cultural dilemma – I was trained to present patients and their families with options, but to leave the decision-making to them, but this family is used to a different model, where the doctor makes the final decision about what treatment will be pursued. So I was uncomfortable being asked to decide a treatment course for them, and they were uncomfortable having this decision left up to them.

Please check back in a day or two to see how the story ends.