Botox and Cerebral Palsy: Is it for me?

I am not quite sure why it has taken so long to write about this subject, given that it is definitely within the top 3 topics that come up in my discussions with parents of children with Cerebral Palsy (CP) and other disorders of movement and posture.  At any rate, I will step into this popular intervention with definite interest and plenty of debate experience behind me.

In order to properly frame this rant, I will need to provide some initial feedback on my own personal and professional perspective on the matter.  I do not make any claims that this drug is either good or bad, nor do I have any invested interest in it's use or "non-use".  The consistent mandate of this blog is to provide extra insight and some additional perspective so that the decision as to "is this right for my child" can be made with as much conviction and confidence as possible.  Although my general feeling on the issue may be obvious, the relevant message is not to fall into a overly simplistic "good or bad" mentality.  Many spend more time and effort researching a home mortgage or reviewing their stock portfolio than they do researching an injectable drug.  Again, the appropriate question isn't "is Botox good or bad?"...rather, "is it the right thing for my child and my family".  Once this fundamental question has been asked, then the resultant answer is irrelevant...it has been put through the internal "prism" of the family unit and the appropriate answer has come out of the other side.

Perhaps the most "diplomatic" and productive way to engage in this discussion is to simply convey the fundamental issues that I typically put forth to any and all families that ask me about Botox.  Although there are many different debates that can take place regarding its merit, the main goal of this post is to shed light as opposed to polarize.

1. Botox Defined: 

--- a neurotoxin (trade name Botox) that is used clinically in small quantities to treat strabismus and facial spasms and other neurological disorders characterized by abnormal muscle contractions; is also used by cosmetic surgeons to smooth frown lines temporarily. ---

During many of my discussions with parents, when the generic name for Botox (Botulinum Toxin Type A, for example) is actually spoken out in the open, it generates an initial sense of apprehension.  It should not be forgotten that this particular drug is indeed a toxin (or as the definition explains, a neurotoxin).  Therefore it is important to always keep this fundamental understanding in mind.

2. Big Pharma:

I certainly would not be characterized as an activist...however, the unfortunate reality in todays medical system is that "Big Pharma" are an extremely powerful and influencial group.  This idea is not new and, paradoxically, well understood and accepted.  It is also well known that a large number of the studies on the use of Botox are actually funded by the same pahrmaceutical companies that produce it.  Therefore, it is in the best interest of the pharmaceutical industry to have Botox dispensed as much as possible...which inevitably leaves room for misuse and irresponsible behaviour.

If these first two phases are understood, then the path becomes relatively more straightforward. Any use of prescription drug comes with its own set of risks, therefore full disclosure of the risks involved always create a clearer path.  In 2009, the FDA ruled to include boxed warnings on Botox products:

FDA NOTE TO CORRESPONDENTS

For Immediate Release: August 3, 2009
Media Inquiries: Sandy Walsh, 301-796-4669; sandy.walsh@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

FDA Gives Update on Botulinum Toxin Safety Warnings; Established Names of Drugs Changed

The U.S. Food and Drug Administration today announced an update to a previous safety alert on four botulinum toxin drug products, noting that all of them now have boxed warnings on their labels and have developed Medication Guides for patients, as directed by the agency in April 2009.

The boxed warning cautions that the effects of the botulinum toxin may spread from the area of injection to other areas of the body, causing symptoms similar to those of botulism. Those symptoms include potentially life-threatening swallowing and breathing difficulties and even death.

These symptoms have mostly been reported in children with cerebral palsy being treated with botulinum toxin for muscle spasticity, a use of the drugs that has not been approved by FDA. Symptoms have also been reported in adults treated both for approved and unapproved uses.
The affected products are:

- Botox (new established name: onabotulinumtoxinA)
- Botox Cosmetic (new established name: onabotulinumtoxinA)
- Myobloc (new established name: rimabotulinumtoxinB)
- Dysport (abobotulinumtoxinA) was approved in April 2009 with the boxed warning and is not making any name or label changes at this time.

No definitive serious adverse event reports of distant spread of toxin effect have been associated with dermatologic use of Botox/Botox Cosmetic at the recommended doses (for frown lines between the eyebrows or severe underarm sweating). As well, no definitive serious adverse event reports of distant spread of toxin effect have been associated with Botox when used at approved doses for eyelid twitches or for crossed eyes.

The revised labels also emphasize that the different botulinum toxin products are not interchangeable, because the units used to measure the products are different. To help reduce the potential for dosing errors, the botulinum toxin products have changed their established drug names (often referred to as the drug’s “generic” name). Neither the brand names nor the formulations of the products have changed.

The portion in red is understandibly quite alarming...however, it is not intended as a "scare tactic".  As mentioned before, full disclosure of the potential risk is the responsible path which has lead to the boxed warnings and the further clarification by the FDA that Botox has not been approved for the use of muscle spasticity.  

3.  "Relaxation" VS "Paralysis" 

I have often heard the use of Botox described as something that will "induce relaxation of muscles".  Although it could be argued that this is an accurate statement, it is undeniably misleading.  The reality is that it essentially paralizes the tissue at the injection site...which could be equated with "relaxation" but that would be an gross over-simplification.  I agree that both terms tend to elicit two different extremes (one being very pleasant and the other more frightening), but the reality is none-the-less present.

4. Local VS Systemic:

As mentioned in the FDA report, the effect of Botox is not only local but systemic.  The overwhelming assumption that the drug "only goes where it is needed"...as if it were a "smart drug"...but it is only a simple liquid, therefore it will behave like any other liquid that is injected into the body.  It will leech into adjacent tissues (muscles) and will also enter the bloodstream.  It has been said that the amount that actually enters the bloodstream is negligible, but the reality is that there is no conclusive evidence that supports this.  The effects of free flowing Botox through the heart, lungs, and brain have yet to be determined.

5. Practical solutions to the overwhelming reality:

Having been fortunate enough to interact with a large number of families and family units, I am very much aware that parents are consistently under the heavy burden of information overload and the fundamental challenges that come from competing philosophies and schools of thought.  All of this information may potentially serve only to confuse and frustrate even more.  Therefore it is important to boil everything down into something that is more practical, productive, and user-friendly.

The use of Botox (and other interventions) cannot be given a simplistic "good or bad" designation. Their role is completely determined by the circumstances present within the individual and the undividual family unit.  There are some cases where spasticity is so profound that it generates considerable pain and discomfort which inevitably contributes tremendous stress to the childs quality of life and therefore extends into the well-being of the parents.  In this situation, Botox is an understandable (and likely the most recommendable) tactical solution.  To be precise, the overwhelming priority of comfort and well-being outweigh the underlying risks.

Outside of these extreme cases, a fundamental examination needs to take place.  It is important to remember that muscle spasticity is symptomatic...it is a reflection of the underlying biomechanical / architectural weaknesses.  Therefore addressing the symptomatic manifestations is a productive short-term strategy, the long term strategy requires some focused attention as well.  The typical scenario is that spasticity increases as the child grows and is characterized by progressive muscular tension and imbalance.  This is analogous to a growing tree whose limbs continue to grow while the trunk remains relatively small...the mechanical stress will inevitably contribute to distortions within the trunk which only serves to perpetuate a vicious cycle of degeneration.

In summary, there is no intended condemnation of the use of Botox, rather a presentation of what I consider to be very relevant points of consideration.  They are not subjective statements...they are objective realities.  Therefore it should be considered in the same "vein" as food labelling:  knowing "whats in it" contributes a great deal as to whether or not you choose to buy it".  If, after "reading the labelled ingredients" it is decided that it fits within the value system of a particular family, then it is most certainly the right choice for them.

To conclude, I fully agree with the availability of this option...but it requires a higher level of responsibility and accountability on the part of Big Pharma as well as an acceptable level of formulation and consideration from individual families.

Cheers!