Botulinum Toxin, or Botox

Imagine seeing your child with Cerebral Palsy raise his arm for the first time, carry groceries, or walk. Researchers report that botulinum toxin can give you that. But, the drug derived from bacteria that causes botulism, also has serious side effects. Among them, death.

Botox: The good, the bad and the ugly

The miracle wrinkle eraser, Botox, has recently been touted as a wonder drug in treating symptoms of children with Cerebral Palsy.

Don’t schedule an appointment with the doctor just yet.

Decades after Botox received government approval to treat specific eye conditions, the drug remains at the center of controversy.

Some say Botox has proven highly effective in relaxing stiff muscles, eliminating excessive blinking and reducing upper and lower limb spasticity.

Others call it a risky remedy that can cause botulism, respiratory failure, even death.

On both sides of the fence are parents who have watched their children with Cerebral Palsy walk for the first time after receiving Botox injections and families who have won settlements against the drug maker after a loved one got very sick, or died.

On April 9, 2013, the New York Daily News reported that a 3-year-old boy, Aiden Farrell, took his first steps thanks to Botox injections in his legs. Farrell was diagnosed with Cerebral Palsy at birth and was not able to straighten his legs without pain.

On May 24, 2013. The Miami Herald reported that 16-year-old Adam Leon was able to lift his arm after Botox injections at the University of Miami, Miller School of Medicine. Diagnosed with Cerebral Palsy at birth, Adam’s condition caused stiffness in his right arm and wrist.

And, the latest research reports positive results over a two-year period for patients with Cerebral Palsy. Dr. Marc DiFazio, who led the research at Walter Reed Army Medical Centre, Washington DC, said that 250 children with the disease, aged one to 16, received Botox injections. Of those, 86 percent experienced notable improvements in their symptoms, some lasting four months or more.

DiFazio also reported that adverse side effects were minor and were experienced by only 5 of the 148 children who were monitored and treated with Botox for more than two years.

Allergan, the Irvine, Calif.-based pharmaceutical company that produces Botox, also settled out of court with many families who claimed the drug caused severe illness, impairment, even death. Among them was a case filed in February 2013 by the family of an Oklahoma boy, 6, who allegedly developed botulism and severe respiratory problems after being treated for leg spasticity.

The company also paid $600 million to the government in 2010 for unlawfully promoting its product, that included using it to treat children with Cerebral Palsy.

In March 2010, an Orange County, California jury declined to hold Allergan liable in the death of a 7-year-old Texas girl being treated for Cerebral Palsy because it found the company’s warning labels adequate. The Texas family of Kristen Spears alleged that their daughter overdosed on Botox because the physician had not been properly educated about its potential dangers.

What is Botox?

Botox and Botox Cosmetic are brand name prescription drugs produced by the bacterium clostridium botulinum, the same toxin that causes a life-threatening type of botulism, or food poisoning. Allergan Pharmaceuticals, of Irvine, Calif., is the main manufacturer. Similar products with the same active ingredients are sold under different names.

Manufacturers don’t recommend that one brand or type of botulinum toxin be substituted for another. The main difference of each is how the drugs are used, the strength, and the dosage.

They are:

• Botox, or botulinum toxin Type B
• Myobloc, developed in Ireland and sold in the United States by Solstice Neurosciences, San Francisco
• Botox Cosmetic, or botulinum toxin Type A
• Dysport, developed in Europe, and sold in the US by Medicis Pharmaceutical Corporation, Bridgewater, New Jersey
• Xeomin, developed and sold by Merz Pharmaceuticals, based in Greensboro, North Carolina and Germany

How does Botox work?

Botox is injected into muscles to weaken or paralyze them by blocking the connection between nerves and muscles. Medical professionals report that treating muscle spasms with botulinum toxin can require up to 100 times the amount needed for frown lines. The effects of one treatment can last anywhere from three to six months.

Approved uses

Since Botox was first approved by the FDA to treat blepharospasm and strabimus, Botox and its competitive manufacturers were given the thumbs up to treat adults with spasticity in the flexor muscles of the elbow, wrist, and fingers, migraines and urinary incontinence to its list of approved uses. Botox Cosmetic was approved in 2002.

A list of FDA-approved uses include:

• Blepharospasm, characterized by uncontrollable blinking
• Cervical dystonia, or lack of control of the neck and shoulder
• Severe migraines with at least 15 episodes per month, each lasting 4 hours or longer
• Severe underarm perspiration
• Strabismus, or misaligned eyes
• Urinary continence, or overactive bladder
• Wrinkles, crow’s feet and frown lines

The controversy

Botox has not been approved in the United States to treat children with the exception of the following:

• Children 12 and older with blepharospasm
• Children 12 and older with strabismus
• Teens 16 and older with cervical dystonia

However, the FDA also allows medical providers to “use their best judgment” in using prescription drugs for off-label purposes. Off-label means the FDA hasn’t approved a drug for a specific use. By the same token, pharmaceutical companies cannot advertise their products for off-label uses. Botulinum toxin has been approved for use in 85 countries for both cosmetic and medical purposes, including the treatment of spasticity and muscle stiffness in children with Cerebral Palsy.

Risks and side effects

Allergan claims on its website that it does not know whether Botox is safe to treat increased stiffness in upper-limb muscles other than those in the elbow, wrist and fingers, or to treat increased stiffness in lower-limb muscles.

In addition, it reports that Botox has not helped people perform specific functions with their upper limbs or increase movement in joints that are permanently fixed.

The company also recommends that any use of Botox should not replace an existing physical therapy routine.

In 2008, health officials warned against using Botox to treat Cerebral Palsy and other muscle spasms, triggering a requirement by the FDA that the makers put an enhanced warning label on their packaging.

The warning states, in part, that botulinum toxin may spread from the area of injection to other areas of the body, causing symptoms immediately or up to three weeks later. Some of the symptoms can be life-altering or deadly.

The FDA reported that safety information provided by the manufacturer indicated that the most common symptoms reported by patients with upper limp spasticity were bronchitis, fatigue, muscle weakness and arm pain.

Other possible side effects include:

• Blurred vision
• Double vision
• Drooping eyelids
• Difficulty swallowing
• Fatigue
• Hoarseness or loss of voice
• Loss of bladder control
• Loss of strength
• Muscle weakness
• Nausea
• Respiratory problems
• Trouble speaking clearly

Other considerations include:

• Pre-existing conditions such as chronic respiratory infection
• Prescription drug use
• Street drug use
• Herbal remedies
• Allergies
• Recent surgeries

Life vs. quality of life: Is Botox worth the risks?

Is Botox worth the risks? Ask a mom who thought she would never see her son walk. Then talk to the parents whose child suffered greatly because of overexposure. Do more research, read statistics, talk with a medical practitioner with a history of legitimately using Botox for patients with Cerebral Palsy. Understand the risks, know the procedure, research the practitioner, and learn about alternatives. Then get a second opinion, perhaps a third. Make an informed decision with the help of your primary care physician and medical research teams.

Brief history of Botulinum Toxin

The first experimental injections to treat hyperactive muscles occurred in 1950’s, prompting new interest in Botulinum Toxin Type A as a potentially significant therapeutic agent.

In the late 1960s, researcher Alan B. Scott, M.D., of the Smith-Kettewell Eye Research Foundation in San Francisco injected a small amount of botulinum toxin into the hyperactive eye muscles of monkeys with strabismus, or crossed eyes.

Success led him to develop a product for humans. In the late 1970’s, he had his first human volunteers.

In 1988, Allergan, Inc., bought the rights to distribute Scott’s product, called Oculinum. A year later, the U.S. Food and Drug Administration approved Oculinum to treat adults with strabismus and blepharospasm. The company changed the drug’s name to Botox.

In 2000 and 2004, the FDA added cervical dystonia, or involuntary neck muscle contraction and primary axillary hyperhidrosis, or excessive underarm sweating to the approved uses.

After complaints by health officials that Botox had caused severe illness and death in some patients with Cerebral Palsy and other muscle spasm-related conditions, the FDA in 2009 required the manufacturers to put an enhanced warning label on the packaging.

In 2010, Botox gained approval to treat adults with spasticity in flexor muscles of the elbow, wrist and fingers.