A question for MacGyver and McKenzie
Posted: 01 December 2014 01:46 PM   [ Ignore ]
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Like you, I am on the side of using only peer reviewed and tested medicines in most cases . However, there was a segment on 60 Minutes yesterday, showing how marijuana injections helped a child who had been having 50 or more epileptic seizures a day. After receiving the marijuana injections, the seizures were reduced to one a week, allowing the child a normal life. According to the report, no other medicine had anything like this effect and also caused undesirable side effects.

My question to both of you is, What would you do if you had a child who had multiple seizures a day and discovered that marijuana—which has not been tested as a treatment for seizures—was shown to be highly effective in stopping them? Would you refuse to try it because it has not yet been tested for efficacy and safety? If you tried it once and it prevented seizures in your child as reported on 60 Minutes and elsewhere,  would you continue it or would you wait until scientific tests were done?

I don’t see a clip of yesterday’s 60 minutes online, but you can see articles from other sources by doing a search for “marijuana for seizures.”

Lois

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Posted: 01 December 2014 02:47 PM   [ Ignore ]   [ # 1 ]
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Lois you raise a couple of important issues here

1) What qualifies a treatment as proven safe and effective

2) When if ever should a physician recommend a treatment that is not FDA approved?

3) Should a parent use a treatment that is not FDA approved?


I did catch a portion of this story on TV and while it is compelling we always have to be aware of the pitfalls of anecdotal reporting especially when desperate individuals are involved. The news and the medical literature are replete with testimonials of miracle treatments and cures which turned out not to hold up to scrutiny. That doesn’t mean we should discount such reports entirely. In fact anecdotal reports are often the impetus for further research that leads to new discoveries but the vast majority turn into dead ends. This is why randomized controlled trials are essential and why we need to be especially careful not to allow heartbreaking stories like these lead to a situation that becomes a free for all. We don’t want people using all sorts of unproven treatments based solely on testimonials.

There is of course a place for compassionate use of unproven and unapproved treatments but we need to be careful about how we do this or it becomes a loophole that allows every quack treatment to be used. In general compassionate use applications generally apply to drugs that are under study but have not yet accrued enough scientific evidence to be approved for use. The recent Ebola outbreak is a good example as ZMapp was given compassionately to several victims despite not having been approved yet. The key here is that the drug has to be under investigation and there has to be a compelling need. That’s important for two reasons. It puts the breaks on a system that could otherwise become a compete free for all and it ensures that if the drug does indeed have promise there will be actual studies to answer the question of whether it is effective and safe. If ZMapp or in the case of this story, marijuana, were allowed to be used for a particular indication in an unlimited fashion without FDA approval or controlled studies then what incentive would there be for any company to ever do those expensive studies and how would we ever know if these things actually worked?

So to answer question number 2, if there is a drug that’s available for compassionate use and nothing else has worked then there are times when it may be reasonable to use that even if the evidence for its safety and efficacy is not complete yet. There may also be situations where a patient has a terminal disease with no available alternative when treatments might be considered even if the evidence is not available for safety or efficacy. Intractable and disabling conditions like seizures are another story since a treatment could in theory make the condition worse and if there is no data to inform the decision then I think from an ethical standpoint physicians would need to refrain from recommending such treatments but it would depend on what was known about the safety profile.

As a parent the considerations are perhaps different but maybe not as much as one would think. Your duty is to do whats best for your child, evaluate risk and benefit and do no harm. Not unlike the goals of a physician. The difference is that its easier for a physician to remain dispassionate and not so easy for a parent. Parents are living with the illness every day and watching the suffering so they may be biased in wanting to treat even if the treatment may be ineffective. If they are distraught enough they may even be willing to overlook or deny safety issues. A physician doesn’t and shouldn’t have that luxury. On the other hand a parent may see things that a physician may miss since they don’t live with the child full time. Perhaps there is a benefit that is only noticeable when you see the child 24 hours a day.

We really don’t know all the details here. Was this child given every possible treatment available? Did the parent refuse certain options? I am not a pediatrician or a neurologist so this particular issue is not one that I am ever in a position to decide, but as a physician I would not feel comfortable recommending that a parent use marijuana to treat a child. Its well known that marijuana can have lief long effects on brain function in adolescents and I would be surprised if that were not also the case in children. If it were my own child and I new all the facts I might make a different decision but its hard to know without those facts.

[ Edited: 01 December 2014 06:29 PM by macgyver ]
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Posted: 01 December 2014 06:36 PM   [ Ignore ]   [ # 2 ]
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Hi Lois,

I certainly agree with MacGyver’s take on these questions. Here’s what I would say in addition.

Medicine is about balancing the risks and benefits of our therapies. This balance is influenced by how much uncertainty there is about the risks and benefits of a specific treatment. It is also influenced by the urgency with which we need to act. The value of scientific research is that it reduces uncertainty, allowing us to more effectively balance risks and benefits. The problem with anecdotes, such as this one, is that they appear to reduce this uncertainty, but they really don’t. The exact same kind of experience that made it seem marijuana helped this child has made people believe in the healing power of prayer, Lourdes water, homeopathy, and many other therapies we know can’t and don’t actually work.

So it’s not a simple question of saying, “What would you do if you knew something worked even if it hadn’t been demonstrated scientifically,” because you can’t know it works without scientific evidence.  The real question is how do you balance the need to act against the uncertainty about the effects of your actions in each case. Let me illustrate with a couple of examples.

I am often asked about unproven remedies for tear staining in white dogs. This is a benign condition in which a natural pigment in the tears causes the hair under the eye to turn reddish brown. People with show dogs hate this, and they are often willing to try any kind of remedy that promises to fix it. Since none of these have been studied scientifically, we don’t know if they work or if they are safe. I tell people that since the condition is purely cosmetic, the need to act isn’t very great. Balancing this against the great uncertainty about safety and efficacy, I wouldn’t use these remedies.

Another easy example is when a dog comes in having lost a great deal of blood from some acute trauma. The need to act is very great. And there is tremendous evidence supporting the safety and effectiveness of a blood transfusion in this situation, so there is not much uncertainty. It is easy to decide to use the therapy.

A situation like the child you describe is much more ambiguous. Obviously, a high frequency of seizures does not permit an acceptable quality of life, so there is clearly a strong urgency to act. I am not a neurologist nor a physician, so I don’t know anything about the treatment options, or how much uncertainty there is about their effects. Presumably, the best choice would be to begin with the therapies that have the most scientific evidence to support them. If these fail, it would be appropriate to consider therapies with less evidence.

In the case of marijuana, I’m not aware of any evidence to suggest it ought to be a good therapy for seizures, so I don’t know why it was tried in this case. But for the sake of argument, if there are only anecdotes to suggest it might be useful, I would expect the child’s doctors to have a very clear conversation with the parents about the great uncertainty. Unproven remedies don’t just fail to work sometimes, they also sometimes cause real harm. This is the risk we take when we act without good evidence. Sometimes, if the situation is desperate enough, it may be right to take this risk, but it should always be done with a clear understanding that we are, to a large extent, rolling the dice with our patient’s health.

Finally, if we use such an unproven remedy and the patient improves, do we conclude the remedy works? No, because again this same logic supports every magical therapy out there. Instead, we add the information to our list of anecdotes, which would support more controlled research to find out if it really does predictably help patients with this problem without unacceptable side effects. If the patient is doing well, it is reasonable to consider continuing the therapy with close monitoring for potential harm. But it wouldn’t be appropriate to conclude the therapy is effective and recommend it freely for others.

So there isn’t a simple yes or no answer. It’s a fluid, subtle balancing of risks/benefits/uncertainty that has to be individualized for every patient/problem/therapy.

I hope this helps.

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Posted: 01 December 2014 07:27 PM   [ Ignore ]   [ # 3 ]
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I saw a different show where a young child in New Jersey was having 75 seizures a day.  From my days working in Early Childhood Intervention, I am acutely aware of the devastating effects of such frequent seizures in very young children.  If not controlled, the child that survives will become increasingly developmentally delayed. Some don’t respond to traditional treatments.  The side effects of traditional treatments are often quite debilitating as well.  There have even been some children who have even had 1/2 of their brains removed as a treatment. 

Anyway, the family had heard about “Charlotte’s Web”.  This is a strain of marijuana that one legitimate marijuana producer has developed in Colorado.  Charlotte’s Web is not a strain that would have been expected to be marketable, as it has very high CBD, and very low THC (IOW, you can’t get high from it).  It was named after another little girl, Charlotte, who was helped by it.  Long story shirt, even though medicinal marijuana is “legal” in New Jersey, the fat governor there, severely limited the available forms.  So the family had to uproot and move to Colorado.  In the TV piece, their little girl went from 75 seizures a day, to (I think they said 7) despite the family now putting her in everyday circumstances,  that were, previously, almost guaranteed to induce a seizure (e.g., the simple stimulations one would encounter going to a park).  The girl continues to get traditional heavy duty meds, when she has a seizure, and wobbles about for a while afterward, apparently from the effects of the medicine.  She gets the oil extracted from Charlotte’s Web, orally. No effects on her functioning (other than decreased seizure frequency) are noted. I strongly suspect that if this girl is able to develop normally in these critical early years, it will be due to this marijuana extract. 

In an ideal world, there would be no classification of marijuana as a Schedule 1 narcotic, and, thus, no ubiquitous, pervasive, and relatively intransigent federal roadblocks to appropriately designed studies for medicines that may, someday, come from marijuana.

We don’t live in that ideal world, presently.

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Posted: 01 December 2014 11:03 PM   [ Ignore ]   [ # 4 ]
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Thanks to MavGyver and McKenzie for your honest appraisals. I thinkmif I were in the positiin of a parent with such a debilitated child whoose life was being destroyed by seizures, I’d be tempted to try almost anything within reason to see if it could help. Of course, this is a rare situation and I wouldn’t take such a risk under ordinary circumstances. I hope it does work for this child and others like her, and I hope scientific trials are underway. In this case, even if only one child showed such dramatic improvement as was shown on the program, I would probably throw caution to the winds and use it.

Lois

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Posted: 01 December 2014 11:09 PM   [ Ignore ]   [ # 5 ]
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TimB - 01 December 2014 07:27 PM

I saw a different show where a young child in New Jersey was having 75 seizures a day. 


It was probably the same case.

From my days working in Early Childhood Intervention, I am acutely aware of the devastating effects of such frequent seizures in very young children.  If not controlled, the child that survives will become increasingly developmentally delayed. Some don’t respond to traditional treatments.  The side effects of traditional treatments are often quite debilitating as well.  There have even been some children who have even had 1/2 of their brains removed as a treatment. 

Anyway, the family had heard about “Charlotte’s Web”.  This is a strain of marijuana that one legitimate marijuana producer has developed in Colorado.  Charlotte’s Web is not a strain that would have been expected to be marketable, as it has very high CBD, and very low THC (IOW, you can’t get high from it).  It was named after another little girl, Charlotte, who was helped by it.  Long story shirt, even though medicinal marijuana is “legal” in New Jersey, the fat governor there, severely limited the available forms.  So the family had to uproot and move to Colorado.  In the TV piece, their little girl went from 75 seizures a day, to (I think they said 7) despite the family now putting her in everyday circumstances,  that were, previously, almost guaranteed to induce a seizure (e.g., the simple stimulations one would encounter going to a park).  The girl continues to get traditional heavy duty meds, when she has a seizure, and wobbles about for a while afterward, apparently from the effects of the medicine.  She gets the oil extracted from Charlotte’s Web, orally. No effects on her functioning (other than decreased seizure frequency) are noted. I strongly suspect that if this girl is able to develop normally in these critical early years, it will be due to this marijuana extract. 

In an ideal world, there would be no classification of marijuana as a Schedule 1 narcotic, and, thus, no ubiquitous, pervasive, and relatively intransigent federal roadblocks to appropriately designed studies for medicines that may, someday, come from marijuana.

We don’t live in that ideal world, presently.

I agree, but the time will probably come. In the meantime, I’d probably do what those parents are doing. Desperate cases call for desperate measures.

Lois

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Posted: 01 December 2014 11:41 PM   [ Ignore ]   [ # 6 ]
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Also, it is oh so gradually becoming common knowledge that the US govt (dept of Health and Human Services) got a patent on CBD for its anti-inflammatory and neuro-protective qualities back in 2003.

http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO2&Sect2=HITOFF&p=1&u=/netahtml/PTO/search-bool.html&r=11&f=G&l=50&co1=AND&d=PTXT&s1=6,630,507&OS=6,630,507&RS=6,630,507

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