HUMAN TRIALS: Scientists, Investors, and Patients in the Quest for a Cure.

• Author: Brownlee, Shannon
• Position: Brief Article

THE VAST MAJORITY OF BOOKS about biotechnology spend endless pages trumpeting the triumphs of genetic medicine and the coming age of wonder drugs. Susan Quinn wants to try something different and instead chronicles the heartbreak of a biotech company that fails, and the dashed hopes of patients in desperate need of its drugs. The result, Human Trials, provides a sobering view of why the search for medical miracles involves enormous risk.

Autoimmune, Inc., was founded in 1988 to develop a new class of drugs for treating autoimmune diseases such as multiple sclerosis (MS), diabetes, and rheumatoid arthritis. Autoimmune disorders are caused by a flaw in the immune system, leading it to mistakenly attack parts of the body. The paralysis and numbness of MS, for instance, results when immune cells destroy myelin, the fibrous sheath that surrounds nerve cells and helps them conduct signals. In rheumatoid arthritis, the immune system methodically annihilates the connective tissue and cartilage in joints. All told, several dozen different autoimmune disorders afflict 8.5 million Americans, a gigantic potential market for the company that can produce a treatment.

Autoimmune's founder was Howard Weiner, a prominent neurologist and researcher at Harvard University who specializes in treating MS patients. Fed up with writing grant proposals that weren't getting funded, Weiner turned to investors to support his research after achieving spectacular results in two experiments in animals demonstrating a theory known as oral tolerance. For each autoimmune disease, the immune system targets a specific set of proteins. In MS, it is proteins found only in myelin; in diabetes, proteins in the pancreas; in rheumatoid arthritis, proteins in cartilage. Weiner reasoned that feeding patients tiny doses of the specific proteins in question might retrain the immune system to tolerate, or grow accustomed to them and stop the attacks. When the idea worked in rodents, Weiner was ready to try oral tolerance on human patients.

So far so good, except that Weiner has hardly a clue how oral tolerance actually works, or at least Quinn fails to explain it adequately, leaving the reader to wonder whether this is just some cockamamie idea cooked up by a bright, but overeager doctor and then marketed ferociously to Wall Street. After all, other biotech companies have been founded on even less science (my personal favorite: Shaman Pharmaceuticals, a company that raised millions for...