The cases that bother Allison Brashear, MD are the ones that come to her office too late. The ones who did not have to suffer for so long; the patients who went months, maybe years, thinking that their rehabilitation had gone as far as it could.
Brashear, professor and chair of the Neurology department at Wake Forest University Baptist Medical Center in Winston Salem, N. C., hates to see those kinds of patients, because she knows that she has a treatment that could have helped them a long time ago. A treatment that despite growing acceptance is still not reaching as many patients as it could.
The treatment she can offer is injections of botulinum toxin type A, purified and marketed commercially as Botox, and what it can do is help ease the relentlessly clinched, over taut muscles often left in the wake of a serious neurological event such as a stroke.
What Brashear has is not a magic, cure all elixir – she can not make marathon runners out of those who can not walk unaided – but what she does have can make a dramatic difference in the lives of patients with post-stroke spasticity. Combined with rehabilitation therapy and good orthotic work, the gains can be magnified.
Brashear, who has been at the forefront of the research and use of Botox to treat spasticity, and the many other doctors who use the injections, can not work miracles, but they can help patients regain vital and dignity-restoring functions.
“Each patient tends to have something that is really important to them,” Brashear said. “We don’t treat without first focusing on what activity they want to do; what is important to them.”
In one of the most common cases Brashear sees, that means using injections to help unclench a closed fist. Something that seems on the surface like a minor gain opens a world of possibilities for the patient: from being a stepping stone to major life-altering goals such as dressing independently or feeding oneself, to basic hygienic goals such as cleaning the hand and trimming the nails.
“Those are the kinds of functions we’re honing in on,” Brashear said.
These are the functions that, in Brashear’s experience, Botox safely and consistently delivers.
Botulinum toxin type A contains the same toxin that causes deadly or paralyzing food poisoning if ingested. Botox, a weakened and sterile form of the toxin, blocks a chemical compound released by nerve cells that signals the muscle to contract.
“I think, really, no,” Brashear said when asked if there was a downside or negative side effects to the treatment. “The benefit of botulinum toxin is that the safety of it is very well documented. The medication goes in the muscle and stays in the muscle. That’s why it’s really the treatment of choice for spasticity.”
Brashear would know. The doctor and researcher, professor and chair of the department of neurology at the Wake Forest University School of Medicine, has led two national studies this decade on the subject, offering evidence that Botox is an effective weapon against post-stroke spasticity.
Yet, somewhat to her disappointment, the U.S. Food and Drug Administration (FDA) has not approved the treatment, rendering it an off label use. Because of that, in some cases insurance may not pick up the cost – though in many instances they will – and the company behind Botox can not directly market it as a treatment for spasticity.
“I wish there was a way to communicate (the benefit) more directly to patients and their families,” Brashear said.
Every time a person pulls on a car handle or squeezes a hand for a shake, their central nervous system sends out a pulse asking for the muscle to contract. The same thing happens when they curl their toes, walk or make thousands of other voluntary and involuntary motions.
Now, imagine if that request for contraction, that pulse, was perpetually set in the on position. That is spasticity.
The continual contraction causes muscle tightness and stiffness, such as a hand that will not come unclenched or a joint that does not hinge. It can present as spasms, or too much muscle tone, called spasticity or rapid uncontrolled contractions called clonus.
Stroke is not the only cause. Any traumatic injury to the spinal cord or brain, along with neurological diseases can create a spastic condition.
Spasticity can effect anywhere between 15% to 30% of stroke survivors. According to the American Stroke Association, 780,000 Americans suffer a stroke each year. At the low end, that calculates to 115,000 Americans a year who could experience spasticity of some degree.
Depending on the severity, spasticity can range from minor pain and movement loss to a debilitating condition. In any case, it undermines one of the main principles of rehabilitation therapy – movement. Over long periods of time, muscles and tendons can become permanently weakened and shortened.
The case for Botox
In 2005 in Philadelphia, at the annual meeting of the American Association of Physical Medicine, Brashear presented the findings of the first long-term study of the repeated use of botulinum toxin type A for the treatment of post-stroke spasticity.
Led by Brashear, then at Indiana, the study focused on 279 patients at 35 rehabilitation centers lasting a year. The research was funded by Allergen Inc., the company that manufactures Botox.
All of the study participants had hand, wrist or elbow spasticity and received up to five doses. The injections were given to targeted muscles in the wrist, elbows and fingers. Six weeks into the study researchers discovered a notable improvement in patients’ muscle tone from the onset of treatment.
Researchers asked the patients to grade their improvement in four areas: pain, hygiene, dressing and limb posture. Patients were asked beforehand which of the areas meant the most to them, and to grade their condition on a four-point scale from “no disability” to “severe disability.” By year’s end, at least half the participants said that they had improved by one-point in the area they deemed most significant. No major side effects were reported.
Brashear also directed the first major study in 2002, which led to a report in the New England Journal of Medicine. In that multi-center study, half of the 126 patients received Botox and the other half took a placebo. Sixty-two percent of the Botox group reported improvement in the area they desired as opposed to just 27% of the placebo group. In that study, patients received just one injection.
“In the last five years acceptance has been dramatic,” Brashear said. “There have been several large clinical trials that show the benefits and…it is a very well-tolerated treatment.”
Brashear said in her use with patients the effect of the Botox gradually wore off over a 3 to 6 month period, and to maintain maximum benefits, the injection needs to be repeated. In her experience, most continue the injections, which she said are no more painful than getting a typical needle.
“Patients don’t generally stop treatment that is working,” Brashear said.
In fact, Brashear has seen some of the greatest improvement come in patients with 10 to 15 treatments. As the injections continue the doctor can hone in on a dosage that works best for the patient.
Communication between patient, physician and therapist is also key, as the treatment does not stand alone but comes within a course of therapy.
“There’s definitely and additive effect between the patient and the therapist,” Brashear said.
Under the brand name Botox, the FDA has approved botulinum toxin type A to treat everything from cross-eyed vision – Botox’s first approval in 1989 – to excessive underarm sweat, to the wrinkle eraser that has made it famous in pop culture. Yet despite the studies Brashear and others have produced, Botox to control spasticity remains an off-label use.
“You’d have to ask the FDA why,” Brashear said.
Despite the lack of the government’s approval seal, the treatment has become mainstream.
“It’s become very accepted as a first line treatment,” Brashear said. “The Academy of Neurology has again endorsed it as a first line treatment.”
As long as the use remains off-label, there is a danger that insurance companies will not pay for the treatment, although anecdotally, Brashear does not see it as a huge problem in her patients. However, where it does create a problem in Brashear’s mind is the lack of the ability to pitch the treatment directly to patients.
Without a full-scale marketing campaign, news of the treatment’s success is passed through the media and word of mouth.
“I wish there was a way to communicate directly to the support groups,” Brashear said.
Brashear has run across patients who were only aware of the treatment because a neighbor told them. She also finds patients who could have been helped by the treatment long ago, but have only recently become aware of the Botox option.
“It’d be wonderful if we could get that word out,” Brashear said.
So Brashear does what she can, hitting the conference circuit, training physicians and trying to spread the prevalence of the practice.
The O&P side
Vincent Benenati is the chief executive officer and co-founder of East Coast Orthotic & Prosthetic Corp., based out of Deer Park, New York. Benenati’s company provides the O&P staff at the spasticity clinic at New York-Presbyterian Hospital/Columbia University in New York. East Coast has 18 full-time employees in various sites at the hospital.
Most of East Coast’s work at the spasticity clinic is with pediatric patients, some suffering from the effects of a stroke, some from other diseases or injuries. Botox is a common treatment by the physicians at the clinic.
Benenati, a certified orthotist, said he sees the benefits of using Botox in conjunction with therapy and solid orthotic work.
“It’s been very successful, what we’ve seen far,” Benenati said.
Benenati believes there is a great benefit to the clinic setting, because the orthotists can consult with patients on site before and after Botox or other treatments. The bracing is almost immediate. With the Botox injections wearing off after a few months, the sooner a patient can get into an orthotic, the better.
“If they don’t get a brace until six weeks later, they’ve lost a lot of time,” Benenati said. “If you do it immediately you see greater gain. The thing we look at is range of motion.”
The greatest advantage of the clinic, according to Benenati, is the team approach. Under one roof, the patient, orthotist, neurologist and any other physician can all meet at the same time. Not only is time saved that would have been lost while the patient is ferried and referred from one doctor and specialist to the next, the lines of communication are much clearer.
Benenati said an orthotist seeing a patient outside of the clinic setting can be at a disadvantage because they are not as aware of what the goals of the rehabilitation are and they may not know the extent of the patient’s condition. But when the orthotist can interact with the neurologist about the medical issues and work with the therapist all in the same office, the odds of setting and meeting realistic goals are much higher.
“The team approach has been a very good one for the patient,” Benenati said. “Not only does it speed [the process] up, but hearing other parts of the puzzle helps things make sense. It helps you do more.”
The best outcomes, for patient and orthotist, come as part of a multi-disciplinary team, Benenati said. It’s not just the orthotist listening to the neurologist. He’s found the doctors at Columbia value the opinion of his clinicians, whether it’s in a rehab role like the clinic or in trauma situations such as an amputation.
“To be on the corner and have a storefront, that’s fine. It has its value. But I think there is more value where we interact with the physicians as part of a clinic,” Benenati said. “That’s why I do this.”
Tim McManus is a correspondent for O&P Business News.