Human Trials: Scientists, Investors, and Patients in the Quest for a Cure - Hardcover

Quinn, Susan

Hardcover

ISBN 10: 0738201820 ISBN 13: 9780738201825

Publisher: Da Capo Press, 2001

Synopsis

Over fifty million people suffer from some form of autoimmune disease-multiple sclerosis, arthritis, lupus, and other afflictions in which the body attacks itself-none of them with a lasting cure. Susan Quinn has investigated the worlds where new autoimmune drugs are being developed: the research labs, the drug-company boardrooms, and the clinics where patients become "subjects" in the search for new medicines and treatments. Her exciting story is one of real people: fiercely competing scientists, ambitious venture capitalists, and, above all, anxious, sick human beings. She takes the reader inside these otherwise closed worlds, into the lead investigator's diaries, the tense closed-door meetings with investors, and the hopeful or heart-rending encounters in doctor's offices. Hers is the archetypal story of all medical research: the roller-coaster trip from the lab bench to the medicine cabinet, in which only a very few new drugs and treatments survive. Susan Quinn, author of the acclaimed biography Marie Curie, catches the hopes, triumphs, and crushing failures, the greed and the idealism in these dramatic human trials.

About this title

Review

Human Trials tells the life story of an unusually dedicated contemporary scientist who strove to revolutionize his field with his innovative ideas, and whose story is far from over.

Susan Quinn, biographer of Marie Curie and Karen Horney, focuses here on Dr. Howard Weiner and his belief in oral tolerance--"the long-held observation of systemic hyporesponsiveness to an antigen fed prior to immunization." He believes that compounds based on oral tolerance can be used to successfully treat autoimmune diseases, particularly multiple sclerosis. His attempts to prove this belief and bring such a compound to market are told as representative of what all scientists, investors, and patients involved in drug discovery must endure. This approach yields interesting observations regarding clinical trials in general. Most notable among them is that the trials are designed to treat large populations in the future rather than the individuals enrolled in them today.

Human Trials is a heartbreaking book. All the characters--the researchers in Dr. Weiner's laboratory, the executives in his fledgling biotech company, and especially the patients he treats--are sympathetic, and there are no happy endings for any of them. But Dr. Weiner still believes in his idea and is still toiling to prove it. Let's hope that one day we will read about how he fulfilled his life's ambition and cured MS. --Diana M. Gitig

From the Publisher

An Interview with Susan Quinn:

Why did you write this book, and why did you title it Human Trials?

I wanted to tell the inside story of drug development. When I heard Harvard Medical School’s Dr. Howard Weiner talk about his research into a potential drug for autoimmune disease, I knew I had a rare opportunity. He had just raised millions for a biotech startup to develop his dream drug, and he was willing to let me in on virtually everything: his journals, the deliberations of the new company, and his intimate encounters with his MS patients. As for the title, I was attracted by the many layers of meaning in “human trials.” The success or failure of a drug depends on transferring results from animals to humans. At the same time, the whole process—with its moments of ecstasy and agony—was itself a trial to all the humans involved: the researchers, the investors and company executives, the doctors, and above all the patients who were trial subjects.

What exactly are clinical trials?

Clinical trials are trials in humans, conducted by physicians under the supervision of private drug companies and the government-run National Institutes of Health (NIH), both under the scrutiny of the Food and Drug Administration (FDA). Despite extensive animal research, drugs are only as good as human trials show them to be. First, new drugs are tested for safety, then for efficacy. It’s a long and costly process: the average time from lab bench to medicine cabinet is 17 years. Hundreds of thousands of people participate in clinical trials every year.

What should a patient know before entering a clinical trial, and why do so many people enter them with a misconception about what is going to happen?

There are three important things for every person entering a clinical trial to know.

The first is that, in many cases, the new treatment in question is being tested against a placebo, an inactive sugar pill. Sometimes, half the patients in the trial will be on placebo. That means there is a very good chance that the trial candidate will not be getting the new drug. Secondly, and very importantly, the trial drug may not work and may have side effects that outweigh its benefits. This is an obvious fact, but it is very difficult, clinicians have discovered, to convince patients of this, particularly if the trial drug is their only hope. The truth is, however, that most experimental drugs don’t pan out. A patient entering a trial should do so only if other available treatments have failed.

The third fact is the one that, in some ways, gainsays the previous two. Many patients benefit from their participation in clinical trials, whether they are on the trial drug or the placebo! They receive regular attention to their disease, and they receive hope for improvement. Everyone who conducts clinical trials knows that the care and the hope result in a “placebo effect”—sometimes of astonishing proportions.

What happens to the interests of individual patients in clinical trials?

In Human Trials, I quote a clinical trial director who says, “the focus one has to keep in this game is on helping populations who have a disease, not individual patients. The minute a patient enters a clinical trial they become part of something that’s more important than they are.” But a patient, suffering from a chronic disease, feels differently. So do doctors who treat patients, and who are trained to put their patients’ interests first.

Ideally, when a trial drug is working, patient interests and the trial director’s interests overlap. Often, though, there is a clash between the two. What happens, for instance, when a trial patient is in pain and begs his doctor for a drug that will muddy the trial results? Patients are always free to drop out of clinical trials. Drug companies, however, compensate doctors only as long as patients stay in.

In your book, you follow the trial process for one particular drug. Why did you choose this drug over others?

Autoimmune disease, along with cancer and AIDS, is an area of research in which there are no cures but many promising recent breakthroughs. That certainly made me want to write about a drug for it. But much of my choice also had to do with access. Dr. Weiner allowed me to be present not just in happy moments but also in desperate ones. He was also willing to grant me complete editorial freedom: he agreed that his only input into my book would be to make sure that the facts were correct. What changes would you like to see in the way clinical trials are conducted here in the United States?

The most stunning and obvious problem I saw was the way patients were treated when the trial they were participating in failed. Despite the very large placebo effects observed by the investigators, no one gave any thought to the fact that a reverse placebo effect was likely when the trial didn’t succeed. If hope made people feel better, then the sudden death of hope was bound to have the opposite effect. And sure enough, in the patients I was in touch with, there were bad flare-ups after the trial failure. Thought should be given to ways to help patients deal with the disappointment. A cold phone call telling a patient to return the drug in question is not enough.

While venture capital is essential to drug development, there has been a tendency in biotech, as there has more recently in the computer industry, to hurry the process in order to justify large initial investments and sustain viability in the stock market. As Genzyme CEO Henri Termeer suggests in the final chapter of Human Trials, research is too difficult and mysterious to be subjected to market forces until the late stages of development. At that point, only the market can foot the bill. Timing is everything.

The next to last chapter of your book is entitled “The End,” and the last chapter is titled “The Beginning.” Explain.

After spending five years in the company of researchers, I finally got it: research is mostly about trying and not succeeding. But the trying matters. As one distinguished AIDS researcher pointed out to me, “the only real failure in science is the experiment that you don’t learn from.”

When I began to work on this book five years ago, I fully expected that the trials I was writing about would succeed, and I would write a final chapter about the FDA approval of a fabulous new drug for autoimmune disease. That’s what all the investors, and patients, and researchers in my story believed too. When the trials failed, many doubted that I would have anything to write about. I doubted it myself for a month or so. But then it gradually dawned on me that I had something more valuable than a slam-dunk. I had a story of how research really works. All the big breakthroughs come at the end of a long series of failures. In this case, Howard Weiner’s prediction that somebody was going to make his idea work somewhere began to come true. An Israeli drug company is now engaged in a second major clinical trial, involving thousands of MS patients, which is based on Weiner’s concept.

In addition—and this is also typical of the discovery process—Weiner’s idea for treating autoimmune disease may turn out to have some of its most important applications in other realms. Clinical trials are about to get underway to treat Alzheimer’s disease using the concept, and it looks as though it may be useful in the treatment of atherosclerosis as well. Those are the “beginnings” I’m referring to in the last chapter of Human Trials. There will be others.

About this edition

• Publisher: Da Capo Press
• Publication date: 2001
• ISBN 10: 0738201820
• ISBN 13: 9780738201825
• Binding: Hardcover
• Edition number: 1
• Number of pages: 288