Human Trials

.           It is estimated that there are currently 14,000 human trials being conducted at research institutions, universities and hospitals, and that each year approximately 100,000 new volunteers enroll in clinical trials (Maureen Milford, The National Law Journal August 20, 2001).  In support of this enormous growth in medical or health-related research, there are approximately 48,000 clinical researchers practicing today.  In Human Trials: Scientists, Investors and Patients in the Quest for a Cure, Susan Quinn focuses on human subject research on debilitating autoimmune diseases, especially multiple sclerosis and rheumatoid arthritis, and introduces her reader to a brave new world of human experimentation.

As we might expect, Quinn’s title expresses a double entendre.  On the one hand, “human trials” refers to therapeutic research involving human beings that is an essential and necessary part of the process of determining if a new drug or therapy will prove effective in curing disease or saving lives.  Our author focuses on the struggles of polymath physician Howard Weiner, a Harvard Medical School professor and researcher, to demonstrate that a particular therapeutic approach, oral tolerance, will provide a viable treatment for people suffering from autoimmune diseases such as multiple sclerosis (MS).  MS is not just a disease of the nervous system that affects 350,000 people in the United States alone, but one of a family of autoimmune diseases, such as rheumatoid arthritis and lupus, that affect millions. 

Weiner believes that he has discovered a new drug that will bring relief to sufferers of autoimmune disease, particularly MS, for which there is no viable treatment.  But to move from scientific discovery to clinical application will require a series of make-or-break human trials “designed to determine whether the drug has an effect that is statistically significant” (94).   According to Weiner, “It takes as long as 15-30 years...from the time of a basic science observation for that finding to be reduced to practice in the clinic. (216)” Human trials, as partially described in this book, are the process through which a promising drug or therapy must go before it ends up in the medical cabinet.

Such clinical research will also be quite expensive.  Weiner appears to have discovered, in the language of the business world, “‘a platform technology’, in which researchers might be able to build approaches to a range of autoimmune diseases” (58).  But such “technology,” as research is usually called in the corporate world, is quite costly both in terms of time and money “[s]ince it takes ten to twenty years, and from $100 million to $300 million to develop a drug” (69).  This forces Weiner to move his research away from non-profit academic “benchwork” and into, for him, the unchartered waters of the investment community.  Here we witness Weiner joining with other venture capitalists to put together, and attempt to grow, a biotechnology company “into a viable entity so that its shares could be sold to the public” (83).  And grow it does as soon discover ourselves following the ‘true life adventure’ of a drug on its journey from synthesis in the laboratory to our medicine cabinet, a journey that takes it through “human trials.”  But the story is also a tragedy as the book ends with the company founded on Weiner’s ideas and research, AutoImmune, barely on life support (capital-support) and struggling to survive.

However, there is a second meaning to Quinn’s title.  She wants to move beyond the discussion of the role of human experimentation in phase 3 research, human trials, to an examination of the human trials involved in such research. Throughout seventeen crisp chapters, we meet patients, physicians, scientists, and investors whose careers and very lives depend on the results of human trials.  However, her focus is on Weiner’s faith in oral tolerance and his patience as he attempts to move from passive observation of the disease in his clinic patients to some kind of active intervention or therapy for MS.

At first we discover Weiner living a kind of double life as he moves back and forth between the clinic and the lab.  Soon we see him enter a third world, that of academic politics, and then a fourth as an entrepreneur, quick to seize the commercial possibilities of his discovery. Sometimes these worlds rest in conjunction; sometimes they move in parallel fashion rarely touching; but often they collide.  Throughout the book we get quick glimpses of “a man who didn’t hold back”(124) shuttling between various obligations - the lab, the clinic, scientific meetings, and Wall Street, but always obsessed with finding a cure for MS.  However, we never see someone able to meld his scientific interests, “his unabashed bid for immortality” (64), his wish to take care of patients, and his ambition for profit.

In her introductory “Note to the Reader,” Quinn promises us “a window into a world that is usually inaccessible”(XI).  However, the world opened for viewing is not really the one that we initially expect. Yes, there is discussion of human trials in medicine, especially a somewhat detailed following of a phase 3 trial.  (Unfortunately, there is little explanation or clarification of the three phases of testing or human trials usually involved in medical research.)  Yes, there is a narrative nod here and there to the trials of patients, the researchers, and the clinicians who are engaged in the clinical studies.  However, our initial concern with the MS patient, and later the rheumatoid arthritis (RA) patent, is soon replaced with a new patient - AutoImmune, Weiner’s biotech startup company.  Instead of following the vital signs of human patients or research subjects, we find ourselves monitoring the stock of AutoImmune as it loses 69% of its value after human trials with the MS drug and another 42% after the announcement of the results of human trials with an RA drug.  By the book’s end, we have traveled from clinical experiences with a”a frightening and capricious disease,” from the academic triumphs of Howard Weiner in the lab, to a new realism about biotechnology.  As one of the venture capitalists in the industry concludes, “biotechnology has been good for innovation but bad for investment” (244).  Only slight mention is made of the effect of all of this on patients.

In her writing, Quinn always seeks to inform, to eschew heated controversy about human trials.  Thus, some readers will be relieved that the banner for this book or subject will not read: Watch as scientists gamble their reputations!  Thrill as capitalists venture their capital!  Shudder in horror as patients risk their very lives in human trials!  However, other readers will be disappointed to discover that the author neglects to follow up on any of the moral issues and ethical conflicts that emerge from her chronicle of the role of human trials in the life cycle of a startup biotech company.  Although she acknowledges that we have entered the “age of informed consent,” she never really explores the concept or its work in human trials.  There is little comment on research activities where patients seem to be mere ends to scientist’s and AuthImmune’s ends, rather than collaborators with investigators.  Thus possible controversy over patients who are never told about risks, or informed that the trial will involve placebo, or apprised of their physician’s possible conflict of interest seems glossed over.  As one physician puts it, no doubt out of hearing range of the patient, “the focus one has to keep in this game is that what I am doing is not trying to help individual patients, what I’m trying to do is to help populations who have a disease” (201).

Human Trials emerges from Quinn’s close reading of Howard Weiner’s carefully kept journals, extensive interviews with patients and physicians in the clinic, and researchers in the lab, and her observations of key public and closed-door meetings with investors at AutoImmune.  She writes clearly of the major problems and lessons learned in one important quest for a cure for debilitating autoimmune diseases.  Although not mentioned in her book, in the past four years government regulators have restricted or shut down human trials at twenty institutions for not following standards of good clinical practice.  This is book that speaks to such issues and thus needs to be read by patients, physicians, researchers, investors, and especially potential research subjects who must decide whether or not to enter a human trial.

© 2002  Larry D. Hultgren

Larry Hultgren describes himself as follows:

A.B. Grinnell College majoring in Philosophy and Religion; Ph.D. Vanderbilt University in Philosophy. Currently Professor of Philosophy at Virginia Wesleyan College, Norfolk, VA. Since I am at a liberal arts college, my teaching runs the gamut of philosophy offerings. I am especially interested in interdisciplinary pursuits, and I direct the college's Social Ecology Program and our innovative PORTfolio Project, which attempts to bring the liberal arts to life for our students by connecting the classroom with real world experiences. I also serve on the Bioethics Committee of the Children's Hospital of the King's Daughters in Norfolk, VA, and serve on the Board of Directors of the Bioethics Network of Southeast Virginia.