Thursday, July 12, 2007

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

I have to confess, I was a bit misleading in my last post .

I asked you to come back in a few days to see how the story ends. Although decisions have been made, there is no ending yet (and I think that’s a good thing). M’s father and I have been in near daily communication about his treatment, the family’s goals, their hopes, and their expectations. They realize that nothing I have to offer has any realistic hope of curing their son. They do, however, hope to find a treatment that is available in the US but not in Japan that might slow the progression of his disease, and perhaps even offer some palliation. They also seek the comfort of knowing that they have given their son every chance and tried every possible option to treat him.

Next Monday M and his family will return to my clinic and we will enroll him on a Phase I trial of a drug that is related to one of the more active osteosarcoma drugs, cisplatin. We hope that M will be one of the lucky ones who benefits from participation in a Phase I trial.

The statistic that I quoted the other day, that only 3% of patients benefit from being enrolled on a Phase I trial, uses a very narrow definition of “benefit.” They define “benefit” as the tumor having an objective response to the treatment (meaning the tumor shrinks or goes away). Using a broader definition of “benefit” would yield a very different statistic. M and his family will benefit from this trial, even if his tumors continue to grow. How will they benefit? They will have achieved a feeling of closure. They will know that they tried everything, that they went everywhere, and that they never gave up the fight. In the family’s eyes, this psychological benefit is worth another 6 weeks away from home.

And if the family sees this as beneficial, that’s good enough for me!


bint alshamsa said...

I agree completely. Evidently, some people might define "benefit" a lot differently than you or I might. If it were me (as the patient), I think I'd prefer to try the Phase I trial. My hope is that even if it doesn't benefit me medically, knowing that my last days were spent doing something that might contribute to someone else's care seems quite meaningful to me.

Doctor David said...

You hit on one of the most difficult issues relating to "early phase" trials... who is the right person to enroll on such a trial, and how do you ethically get informed consent? I think the key point is to make sure the patient (or in the case of a child, the patient's family) realizes how unlikely the trial is to result in a "direct medical benefit" (like shrinking a tumor). If, knowing that, they wish to continue, for either the psychological benefit of knowing they had "tried everything" or the benefit of knowing they might contribute to someone else's care, or for whatever reason they choose... that's OK by me. What we onc docs need to worry about, though, is the patients who want a phase I study because they think it will cure them. In my mind, it's important that the patient go into the study with their eyes wide open, knowing what the potential benefits might be and knowing what risks they face so that they can make the decision that's right for them.