High risk research to find transplant tolerance

Scientists at Penn seek out people who can live without anti-rejection meds, to figure why.

Medications can be expensive — even for people with health insurance. A recent survey from Consumer Reports finds that a quarter of people skip doses to save money. In some cases the stakes are high – even life threatening. In other cases, self-experimentation has led doctors to discover that some medications aren’t necessary. From WHYY’s health and science desk, Kerry Grens reports on high-risk research to find people who can live without life-saving drugs. [audio:100901kgorgans.mp3]

Several years ago, Art Caplan, a bioethicist at the University of Pennsylvania, got pulled in to the middle of a complicated problem. A kidney transplant recipient was rejecting his donated organ. Voluntarily, he had taken himself off the immunosuppressant drugs that stop his body from attacking the foreign kidney. The transplant team was mad.

Caplan: It’s hard to get kidneys…and he clearly had not been taking his medicine. And the ethics issue was, were they going to relist him to get another transplant because he was rejecting this kidney?

Caplan was asked to advise on this question. He interviewed the patient, and asked why he stopped his meds — even though it was sure to kill him.

Caplan: And he said, well, it costs too much. I said, what do you mean? And he said, my drugs cost too much.

It’s the reality for many people who don’t have insurance or who do, but have co-pays — especially for expensive regimens like anti-rejection meds that last a lifetime. They cost thousands of dollars per year.

Grens: What happened with him, do you know?
Caplan: I do. He got retransplanted.

And he got assistance to keep him on his medications. A fortuitous discovery came out of his self-experimentation: it turns out this patient was able to tolerate his organ with just half the dose of the immunosuppressant drugs.

Caplan: And it is that kind of case that is the preliminary experimental basis for finding out that some people can use less drug or some people may not need any drugs.

He wasn’t the first patient to successfully reduce his immunosuppresant drugs. But he’s still a rare mystery.

Shaked: It’s kind of considered the holy grail of tranplantation: tolerance.

Abraham Shaked is the director of the University of Pennsylvania’s Transplant Institute. He specializes in livers.

He’s leading a study among his patients to find those who are naturally able to tolerate their new organs without drugs — and figure out why. Shaked is collecting genetic and immune response data from people in the experiment to uncover their secret.

Eighty percent of those invited to participate agree to do so, even though the risk is enormous.

Shaked: Which is amazing to me. They really volunteer to do that. They want to get rid of the immunesuppression. They know the risk very well of rejection, but they do volunteer to do that.

The drugs increase the risk of infections, cancers, diabetes and kidney damage. But the majority of people need them to keep their new organs safe.

Glenn Hazel was willing to take the risk and see if he was in the minority.

Hazel: Couple days after I had my liver, Debbie came in and said, would you like to join our program? I said, you kept me living, I’ll join anything you want me to.

Hazel had a liver transplant in 2006, after suffering from cirrhosis. His wife, Joyce Wise, remembers the period following surgery.

Wise: He probably was taking 70-80 pills a day. It was massive…We were fortunate that his insurance covers most, the majority.

Doctor Shaked’s team tapered Hazel’s medication over the course of years, constantly monitoring his progress. At the first sign of rejection, he would have been put back on the pills. But it’s been more than a year, and Hazel is completely free of the immunosupressants.

Hazel: The best decision I ever made in my life was joining that study.

Fewer than twenty five percent of people can tolerate transplanted organs. Yet, self-weaning because of cost is a big problem, says Maryl Johnson. She’s the president of the American Society for Transplantation, and the director of heart transplants at the University of Wisconsin Medical Center.

What’s troubling to her is that Medicare — which pays for most of the kidney transplants in the US — only covers three years of anti-rejection medications.

Johnson: It makes no sense to pay for a transplant, the patient’s doing well, and then if we can’t provide ongoing care of the organ that the transplant can fail.

Her group is lobbying to extend coverage for a lifetime. When the new health insurance law was developing in Congress last year, the House had included more benefits for immunosuppresant drugs, but ultimately that provision was rejected.