The Ultimate Cure

The couple’s new push is to get more federal dollars channeled toward the industry. Zack has been traveling back and forth to Washington, sometimes taking along neurotech C.E.O.’s, to promote a $1 billion “national neurotechnology initiative” that Representative Patrick Kennedy, a Rhode Island Democrat, recently announced he will introduce in Congress. The legislation asks the federal government to spend $200 million a year for five years on neurotech, including $30 million for the Food and Drug Administration to train more experts, $80 million for the National Institutes of Health to coordinate the neuroresearch efforts that are now run by 16 different institutes, and $75 million to increase small-business grants for neurotech companies.

Treatments for the mind are hardly new. Before modern times, remedies included the exorcism of evil spirits, bleedings to rid the body of bad humors, and opium smoking to alleviate “melancholy.” In the mid-20th century, physicians tried crude and often destructive “cures,” now discredited, such as lobotomy—removing sections of the brain believed to be causing neuroses.

In the 1950s and ’60s, psychiatry was revolutionized by the invention of antidepressants and tranquilizers. The progression of new drugs continued into the ’70s and ’80s, especially with the development of the blockbuster class of antidepressants called selective serotonin reuptake inhibitors, which includes Prozac. Approved by the F.D.A. in 1987, Prozac, as well as other S.S.R.I.’s—like Zoloft and Paxil—prolongs the action of serotonin, a neurotransmitter, which has beneficial effects on such problems as depression, attention-deficit disorder, and anxiety. Critics of S.S.R.I.’s argue that they don’t work for many patients, that they are being overprescribed, and that they can cause side effects such as loss of libido and (according to controversial findings) suicidal thoughts in teenagers.
But since the late ’80s, few new classes of drugs to treat brain maladies have made it to market, and many diseases remain either undertreated or, like Alzheimer’s and Huntington’s disease, not treated effectively. The industry now faces the challenge of parlaying two decades’ worth of breakthrough research on the basic workings of the brain into new and better treatments—a process that is often thwarted by the complexity of the brain. “We target a drug that is supposed to do one thing, and we find out it does five more things we didn’t expect,” says Sam Barondes, director of the Center for Neurobiology and Psychiatry at the University of California at San Francisco and the author of Mood Genes.

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Much of the industry’s financial success in recent years has come from drugs that differ only slightly from longtime neuro-blockbusters, some of which are losing their patent protection. Patents expiring in 2008 include Risperdal, a schizophrenia drug from J&J’s Janssen unit, annual sales of which are approaching $4.2 billion. “The new products are coming, but the big numbers are still in the Prozac category,” says Martha Farah, director of the Center for Cognitive Neuroscience at the University of Pennsylvania.

Some of the most interesting advances are being made not in drugs but in devices and other treatments. Late last year, StemCells Inc., a Palo Alto, California, company, announced that it had successfully transplanted stem cells into a human brain and that the patient had recently completed a one-year follow-up exam. Five other patients have also been injected with HuCNS-SC, as the company calls its human stem-cell product. All of the patients suffer from Batten disease, a genetic malady that leaves children’s brain cells without a critical enzyme and eventually causes seizures, loss of motor skills and mental capacity, blindness, and finally death. In previous studies with mice, the stem cells took hold and produced the missing enzyme. “These trials are just the beginning for stem-cell therapies in the brain and elsewhere,” says Antoun Nabhan, a former venture capitalist for Sagamore Bioventures who has invested in and sits on the board of Cellerant Therapeutics, one of StemCells’ competitors. But stem-cell treatments for more-common brain diseases are at least five years away, Nabhan says.

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