His Brother’s Keeper: A Story from the Edge of Medicine by Jonathan Weiner. Ecco, $26.95, 356 pages.

The News & Observer

May 9, 2004

The Cure Hunter

By Phillip Manning

Stephen Heywood went to the neurology clinic at Massachusetts General Hospital just before Christmas in 1998. He was only 29 years old, but his right arm had weakened and become thinner during the past year. Earlier, a doctor had mentioned the possibility that Stephen had ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease). Stephen had ignored him. This time, though, the withering of the muscles of his right hand and arm was obvious. “There’s some weird stuff here,” the neurologist said. The doctor refused to put a name to the “weird stuff,” but Stephen knew what he meant: he had ALS. He also knew why the doctor was reluctant to tell him directly. The diagnosis is a death sentence; there is no cure for ALS, and most patients die within two years of diagnosis.

Doctors have been delivering devastating news of this sort to patients for centuries. Afterwards, families gather to console the victims who are likely facing long, debilitating illnesses followed by death. Stephen followed this ancient script and contacted his family to tell them the bad news. But technology has changed the way some people react to incurable diseases. When Stephen called his brother Jaime, an engineer in La Jolla, California, Jaime did not offer platitudes. Instead he did something that families couldn’t do a few decades ago. He turned to the computer on his desk and punched in “ALS.” He read abstract after abstract. Then, he resigned from his job and vowed to “find some way to pull Stephen back from the dead.”

In “His Brother’s Keeper,” the Pulitzer-prize-winning science writer Jonathan Weiner tells the gripping story of Jaime’s race to save his brother from ALS. The story vividly illustrates how science and technology have changed the relationship between patient and physician. Patients and caregivers can access web sites for almost every disease and get crucial information on experimental drugs and procedures, giving them far more insight about how their illness should be treated.

Jaime’s first job was to learn all he could about ALS. He discovered that it is an insidious disease that attacks the motor neurons, the nerve cells that carry messages from brain to muscles. Stephen’s motor neurons were dying, the messages sent by his brain to his right arm were not getting through, and his muscles were wasting away. The disease progression is inexorable and well documented. Stephen would get weaker, and his voice would fail. He would be confined to a wheelchair and forced to communicate with a voice synthesizer, as Stephen Hawking, the noted theoretical physicist and fellow ALS sufferer, does. Eventually, he might be unable to swallow and could drown in his own saliva. Why was this happening, wondered Jaime. And what could he — with his charm, his fast-talking intensity, his entrepreneurial spirit, and his supportive and well-connected family — do about it?

As he absorbed information about ALS, Jaime came across a paper by Dr. Jeffrey Rothstein of Johns Hopkins University. Rothstein suggested that the nerve death associated with the disease might be due to unusually low levels of a protein. Jaime jumped at the news. Why not insert the DNA that made the protein into Stephen’s dying nerves? Gene therapy, as it is called, was the rage in the late 1990s. Biotech companies promising gene therapy miracles were springing up all over, even though
its successes were almost nil. “I’m going to find a cure,” Jaime told his wife.

Jaime did not limit himself to investigating gene therapy. In a frenzy of activity, he found a scientist working on a neurological vaccine. Might this work for Stephen, he asked himself. Let’s pursue that lead, too. A stem cell injection of the patient’s own blood at the base of the brain might make new nerve cells. Could work, thought Jaime. As Jaime worked, Stephen tried to forget his ailment. He wanted to live whatever life he had left to the fullest. He considered buying a Harley and sowing “wild oats for a few years.” Instead, he decided to marry his girlfriend.

Meanwhile, Jaime was moving fast. He formed a nonprofit organization devoted to finding a cure for ALS and raised $240,000 to fund it. Weiner, who was shadowing Jaime’s every move to write a story about him for the New Yorker magazine, begins to believe Jaime might be able to pull it off. Weiner then becomes part of the story himself. His mother is suffering from a deadly neurological disorder, later identified as supranuclear palsy. Weiner had long ago concluded she was incurable. But Jaime’s vaccine might help her. Dare he hope again?

Jaime’s frenetic search takes Weiner and the reader on a roller coaster ride from hopeful highs to despondent lows. The ride takes a terrible toll on both men. Weiner dreams of returning to the simple, peaceful life he led while living in a tent in the Galapagos, the setting for his book “The Beak of the Finch.” Jaime fantasizes about using his newly acquired knowledge of vaccines and gene therapy to cash in on the biotech boom. He and a friend start a small biotech company. Why not save Stephen — and get rich, too?

One by one, the pipe dreams of both men are dashed. On September 17, 1999, Jesse Gelsinger, one of the first volunteers in a gene-therapy trial, died of complications from the treatment, and the FDA and other sponsoring organizations put a hold on gene-therapy trials. The neurovaccine turns out to be too unproven to try on Stephen, and Weiner’s mother’s condition deteriorates to the point that he realizes nothing can help her. Finally, the stock market boom that fueled the Internet and biotech bubbles goes bust. Jaime, the entrepreneur who wants to save ALS patients and get rich, realizes that his fledging business is never going to get off the ground.

In the end, Jaime selects stem cell injections as the best chance for his brother. Stephen undergoes the difficult procedure without harm but realizes no benefit from the treatment. However, despite setbacks and defeats, this is a story about hope, and though I will not reveal the book’s ending, it concludes on a heartening note. It is a note that doesn’t depend on miracle cures but arises from the resilience of the human spirit.
####