Cure Interrupted?

Human Guinea Pigs Bewail Withdrawal Of Experimental Drug





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Human Guinea Pigs

Parkinson’s patients say a drug they took in a clinical trial improved their lives. Now, the drug’s maker says it’s unsafe to give them more. Lesley Stahl reports on 60 Minutes. | Share/Embed

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(CBS) Bob Green, a minister, is the man in the video that Slevin showed 60 Minutes. He says, "If the placebo effect was a week or two, I would understand that and say, 'Yes, you’re right.' You know. But two years later to be gaining ground and making real progress? That’s far from a placebo."

Phase one patients (a group in Kentucky plus five more in England) took GDNF for up to three years, and according to published reports, all improved dramatically.

So why did the data from the phase two trial fail to show greater benefits? Several of the doctors argue that the patients were on the drug for only six months (not long enough) and were given too low a dose. So convinced the drug was safe and effective, the doctors wanted Amgen to at least provide it under compassionate use.

Stahl asks Caplan, the bioethicist: "Does the company have some kind of an ethical obligation to at least continue compassionate use for the people they’ve put through these experiments?"

Caplan's answer, "Normally, you would say yes to that. The issue in this particular study, though, is they’ve got animals that are getting problems."

But Slevin, in Kentucky, suspects that those monkey lesions could have resulted from abrupt withdrawal from a toxic dose, ten times the concentration the patients got. To demonstrate that his patients were OK, he compared scans of their brains from before they took the drug, to after, and saw no damage. He took his evidence to the Food and Drug Administration (FDA).

"What the FDA told us," says Slevin, "they didn’t see any reason why they shouldn’t be allowed to continue having the drug as long as things were being monitored."

Stahl: "And still Amgen wouldn’t do it?"

Slevin: "No."

"You must have been frustrated."

Slevin: "Yes."

Amgen declined to give 60 Minutes an interview, but sent a statement saying it has an ethical obligation to “protect patients …against the possibility of drug-induced adverse effects.” Bioethicist Caplan says recent lawsuits involving the safety of drugs like Vioxx were a huge factor.

Explains Caplan, "We’ve made a very jittery pharmaceutical industry. And I will tell you, at the first sign of problems in animals, even if you’re giving them a million times the dose that you’re going to give a human being, they start to say, 'That’s it. We’re outta here. This is not something we can pursue.'"

Aware of the company’s fear of liability, the patients have made assurances they will never sue Amgen.

Stahl says to Linda Thacker, "You’re saying that even if your husband suffered – I’m thinking the worst case scenario, brain damage."

Linda Thacker: "Lesions."

Stahl: "Lesions, whatever. No suing, period. That’s the end of it. You accept."

Linda Thacker: "I’ll sign. My husband will sign. His daughter will sign. Right now, it’s the same mindset we had when we went into the study to begin with. We have nothing to lose."

Even if these particular patients promise in a kind of legal contract that they will not hold Amgen the company liable, even if they give away every chance they have to come back and sue.

The problem? "The companies don’t believe them," says Caplan. "We’re in a litigious society to the point where people will say, 'That won’t be worth anything. You can’t waive your rights to sue me.'”

Off the drug and getting worse by the day, Bob Suthers and eight of the 10 Kentucky patients decided to go to court to force Amgen to give them GDNF. Even though they all had signed a consent form acknowledging the company could stop the experiment, they hired attorney Allan Milstein to argue that the decision about continuing the drug should be in the hands of the principal investigators, the patients’ own doctors – not the company.

Attorney Milstein: "The principal investigators said, 'We think Amgen’s wrong. Keep the pumps in there and we’re going to try to get you the drug.'”

Stahl: "Have you ever seen doctors defy a company quite like that before?"

Attorney Milstein: "No."

Stahl: "Or heard of it?"

Attorney Milstein: "Never."

Stahl: "I spoke to somebody who said it would be unheard of, if a court ordered a company to give a drug. I mean, that the court really wouldn’t have the power to do that. And it would set a terrible precedent."

Attorney Milstein: "Well, I don’t think it would set a terrible precedent. This is an unusual case. "

That's because, he says, the patients went through so much. Roger Thacker has his own theory of why Amgen stopped the trial:

Thacker: They know it works. What we’re talking about here is money and delivery system. Our delivery system is not viable anymore.

Stahl: You’re saying the idea of having brain surgery, stomach surgery, that’s not going to work for everybody with Parkinson’s.

Thacker: You can’t afford it.

Stahl: Can’t afford it. So they need to come up with…

Thacker: What they’re gonna come up with, I believe, is an encapsulated form where you get up every morning and…

Stahl: Take the pill and that’s it.

Thacker: You’re done.

Stahl reports that 60 Minutes doesn't know whether Roger Thacker has it right. But they found this statement by Amgen's vice president of research, Roger Perlmutter, on the Internet. It's from a speech from when the phase two trial was still under way:

"There aren’t enough neurosurgeons in the country to actually do that procedure and there aren’t enough neurosurgical suites in which to actually do it. So that would limit you pretty dramatically. This is not a therapy from our perspective that is going to be a huge moneymaker for Amgen. It’s just, you’re never going to get there."

Eight days after Amgen stopped the trial, the company applied for another patent for GDNF, along with new ways to deliver it, including an encapsulated form.

For the patients, their only chance of getting GDNF is their court case, which won't go to trial for several months. Roger Thacker says he risked his life for Amgen with brain and stomach surgery. Now, he says, they owe him.

"Yeah," says Thacker. "My point is, I don’t feel like the guinea pig. I’m not a lab rat. I’m a human being. I signed a contract with them, and they have rights, and I recognize that. But in return, they should be doing the same thing for me."

Says Suthers, "It’s like being a racehorse. And when he’s no further use to them, they take him out and shoot him. And that’s just what it’s like."

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