You thought botox was all about looks? it's also a salve for everything from cerebral palsy to writer's cramp
The neurological disorder that plagues Art Kessler arches his spine painfully backward and swivels his neck sideways. Every three months for the past four years, the 39-year-old private-equity manager from Chicago has gotten injections in his neck and along his spine that relax his tightened muscles and allow him to work, play with his young son, and "live a normal life. It's been huge for me in terms of keeping me mobile," Kessler says. The shots responsible? Botox.
In the past decade, since its war on wrinkles began, Botox has gained a massive following, from soccer moms to movie stars, who refuse to accept the evidence that they're getting on in years. Sales for cosmetic use of the drug, which causes temporary muscle paralysis and prevents the grimacing that leads to crow's feet and frown lines, were $357 million in 2005, according to Allergan, the drug's manufacturer. Meantime, sales for far-less-publicized therapeutic uses reached $473 million, as doctors have wielded it against everything from cerebral palsy to headache to Parkinson's disease and crossed eyes. "I don't know of a single treatment that has more applications," says Joseph Jankovic, director of the Parkinson's Disease Center and Movement Disorders Clinic at Baylor College of Medicine in Texas and a pioneer in Botox research.
It was Jankovic's work that led the Food and Drug Administration to approve Botox back in 1989 as a treatment for blepharospasm, an eye-muscle disorder that causes abnormal blinking. Injecting the drug-diluted botulinum toxin, a poison-directly into an affected muscle inhibits the neurotransmitter acetylcholine from relaying messages from the brain, which relaxes the clenching and thereby eases pain. It's the overactive signaling from the brain that's common to the conditions the drug treats best. Botox paralyzes only at the site of the injection, but patients must endure needle jabs directly into the problem area-again and again, since the drug's effect wears off in a few months.
Off-label use. Besides brow-wrinkling and blepharospasm, Botox has been approved to treat crossed eyes, cervical dystonia (involuntary muscle contractions in the neck and shoulder), and-most recently-excessive underarm sweating. (Acetylcholine also stimulates sweat glands.) But the list of "off label" targets has been growing fast and now includes lower back pain, constipation, epilepsy, tennis elbow, and fibromyalgia, to name a few. Jankovic considers the injections to be quality-of-life-saving to a number of patients with disorders that cause uncontrollable movements, including Parkinson's disease, tremors, and Tourette's syndrome. (With research support from the drug companies, Jankovic has been studying the effectiveness of two competing drugs as well as Botox, in hopes of ultimately lowering the cost of treatment.)
"Without it, I would not be on the air," says National Public Radio talk show host Diane Rehm, who has spasmodic dysphonia. The condition clenches her vocal cords, causing her voice to quiver and crack. Shots into her vocal cords every four months have saved her career, she says. More than 50 clinical trials of botulinum toxin are currently underway, including Allergan-sponsored efforts to rack up FDA approvals for headache, overactive bladder, and spasticity caused by stroke.
"There's a lot of experimentation going on," says Christine Cheng, assistant clinical professor in the School of Pharmacy at the University of California-San Francisco, who has studied the body of research on Botox's off-label uses. While there's mounting excitement about the drug's potential, Cheng thinks patients need to be cautious about off-label use, since its effectiveness and safety are yet to be proved in many cases. "Don't demand a certain treatment just because you saw a headline," she advises.
Possible side effects include headache, flulike symptoms, and greater-than-intended muscle weakness-causing a droopy eyelid after treatment for a blinking disorder, for example. Because Botox is not permanent, neither is the weakness, but the comeback can take weeks. No one knows what the effect might be of long-term use. And it's been observed that a small percentage of people develop antibodies to the Botox, making it less effective.
Botox offers "an alternative to drugs in difficult patients" when other therapies have failed, says Seymour Diamond, director of the inpatient headache unit at St. Joseph Hospital in Chicago. He's conducting Allergan-funded research on the use of Botox for headache. "But I'd be reluctant about its generalized use until proven useful," he says.
Where the science is sound, some insurance plans will cover Botox treatments even without the blessing of the FDA. Injections for writer's cramp, certain gastrointestinal disorders, and spasticity caused by brain injury, multiple sclerosis, or hereditary paraplegia are sometimes covered, for example. Aetna and Cigna, among other insurers, say that they may cover other uses if a doctor deems Botox medically necessary. "Where there's inconsistent literature, we'd give case-by-case consideration," says Daniel Winn, a medical director at CareFirst BlueCross BlueShield of greater Washington, D.C. So far, wrinkles don't clear the industry's bar.
Not a cure. Even when it works, Botox doesn't address the underlying condition. Kessler is now scheduled to get deep brain stimulation, a surgery that will implant electrodes in his brain to override the problematic signals caused by his condition, generalized dystonia; he hopes it will make the injections unnecessary. But sometimes just managing symptoms improves a patient's prognosis. In people with dystonias or spasticity, for example, Jankovic believes the drug can alter the disease's progression: Early treatments may head off the permanent contortions that can occur when muscles are contracted for a prolonged period and tendons shorten.
"I wanted to function," says Edward Rosa, 70, who started treatments after a stroke six years ago left him with clenched muscles on his left side. The retired Montville, N.J., businessman, who still does some consulting, says the therapy allowed him to exercise other muscles, improving his range of motion. At first, he went in four times a year for injections in his arm and leg. As he's progressed, the interval has lengthened; his last appointment was a year ago.
Because the delivery of the drug is localized and its effect wears off, some doctors like it as a possible alternative to more systemic and permanent treatments. Compared with drugs or surgery, "this is so minimally invasive," says Michael Chancellor, director of neurourology and female urology programs at the University of Pittsburgh Medical Center. Chancellor uses Botox off label to relieve muscle spasms in the pelvic floor and to manage enlarged prostate. By relaxing muscle tissue that permeates the gland, he says, the drug seems to reduce pressure on other parts of the urinary tract. Chancellor, who has received research funding from Allergan, is now investigating the treatment for overactive bladder.
Ironically, Botox's power is derived from a substance that the federal government classifies as capable of inflicting mass casualties. While using it as a weapon would be extremely difficult technically, botulinum toxin is "the most poisonous substance known," says Zygmunt Dembek, a biochemist and epidemiologist at the U.S. Army Medical Research Institute of Infectious Diseases. "And it has great potential for good."
This story appears in the January 22, 2007 print edition of U.S. News & World Report.