Secret Behind Off Label or Unlicensed Medications

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As a private practice, we are frequently approached by patients who require certain medications “off label” or “unlicensed”. This can sound quite scary for the average person who is deciding to undergo this route or for carers of the patient who may be nervous about what this may entail.

Here's a little background on these off label medications....

Every drug is developed and tested for treating certain conditions. Clinical trials are conducted and the drug is deemed “safe” to use for its intended purpose. Occasionally, the initial purpose a drug is being developed becomes null and void when one of their “side effects” then becomes the more interesting feature of the drug. A good example is Viagra. Viagra was originally developed to treat high blood pressure and angina (chest pain). Its side effect- causing erections- became an unexpected and highly lucrative venture for the manufacturers. Another good example is Botox- initially developed to treat facial tics and spasms. When they had a side effect of decreasing wrinkles, this became its main and highly lucrative use.

Pharmaceutical companies are then allowed to patent the drug for a certain number of years. This is to allow them to claw back their investment in research and development that has gone into making those drugs. It is also to enable them to make a profit- they are a business after all. This system works well. If you consider the number of drugs that don’t make the cut for human use- which translates into millions of dollars of lost revenue for the drug manufacturer, its only fair that the risk they take gets rewarded when one of their experimental drugs do work.

So let’s say that viagra was indeed licensed for high blood pressure. Now according to the medical guidelines, we as doctors, should only prescribe them for that purpose. But we are highly trained and frequently have to use our judgement in every individual case to decide the best course of action for that particular person. Guidelines exist for a reason but at the end of the day, the reason our job is not done by a computer is because of the human quality, experience and instinct that we are able to hone over the years- that beats the computer. This means that I can now decide that for my patient with an erection issue, I can give him viagra in a suitable dose to help with his erection, but not any blood pressure issues (which he may not have). That would be a classic off label use of a drug.

Some examples of off label use medications are low dose naltrexone, clemastine, various hormones, aspirin, cholestyramine and metformin amongst others.

Now lets say that a drug that has been long approved and deemed safe for a purpose has now been found to have another use. A good example is LDN (low dose naltrexone). Initially developed for addictions, it is now used in a very low dose for immuno-modulation amongst other things. This benefit has been stumbled upon. Our challenges as practitioners are listed below:

  • Someone needs to inform other practitioners about this

  • Other practitioners may well be quite sceptical as there are no large trials to “prove” this fact

  • Big pharma are not very interested in conducting large trials. These things cost money and since the initial drug has already lost its patent, there’s no money to be made.

  • So its up to individual practitioners, happy patients and word of mouth to get the word out

  • There are risks and we know it. And there’s a lot we don’t know regarding potential side effects, other uses, risks and benefits. But we’re willing to take the chance and happy to monitor closely

  • In an ideal world, we will all do research and audits on all our patients. Sample sizes will be small but something is better than nothing. However, this does mean tons of paperwork and potentially increase in staff size which unfortunately translates into increased costs for the practitioner. One reason why there are so few trials for off label medications. Its simply a few good doctors and too much work.

  • We as practitioners, also take the risk of being thought to be unsafe by fellow doctors who may not understand our reasons for going off label. This is a big issue and concerns us gravely as it can lead to suspension of our practising privileges.

I hope that this article puts things into perspective a little so that it can be more clearly understood where we- as practitioners- stand when we take the decision to use an unlicensed medication and so that you- as a patient or carer of a patient- will be able to make an informed decision as to how to decide if its the right option for you. It will probably mean more paperwork as we try to safeguard ourselves, we may ask you to participate in trials and we may even ask you to volunteer your time to help us analyse data and eventually make things more available in the future.

Do let us know if you would like to contribute anything at all ie ideas, time, money to help towards pushing these ideas forward. We have recently registered our charity Fighting Chance Foundation. But I envisage that its going to take quite a long time to get anything going mainly due to lack of time and staff at present. We would however, love to hear from you and know that we have some support.

LDN Stories- Down and Depressed Amir

Amir, 43, came to see me after being referred by his nutritionist who was seeing him for adrenal fatigue. He is a professional who came upon some hard times which affected him badly.

He was on quite a few supplements for his fatigue and showed some signs of hormonal imbalance. I also thought that his diet was quite poor which would affect him quite significantly. He had brain fog, constant fatigue, struggled to recover from exercise, was moody and depressed and could not sleep well. Here's his story in his words.....

"Hi Dr Bains,

Thank you very much for your help and support. It has been a real life saver.

Prior to this adrenal fatigue, I was very fit and healthy, exercised 4 times a week, and was successful. An abusive marriage, mom dying and extreme stress caused me to become ill and life has been a struggle since 2003. Three years ago, after finishing an MBA,  I crashed completely and started work with a nutrionist making slow progress.  Prior to the LDN, my energy levels were very very low to non existent,  my mood was very low to depressed, I struggled to get 5-6 hours very poor quality sleep, and woke up feeling unrefreshed with high adrenaline levels. I felt like a zombie ,put on weight, was unable to think straight, had very bad brain fog, and was unable to do anything due to very low energy levels.

The turnaround with LDN has been nothing short of miraculous, and a real game changer.

We started the dose at 3mg, and things got worse before they got better. The LDN really ramped up an already primed immune system, increased my adrenaline, caused flu and cold like symptoms, i.e. phlegm from nose and chest, aches and pains, especially back ache, skin was horrendous and sleep was very poor due to the increased adrenaline.  However, 2 weeks after this, the immune system settled and my sleep went from 5 hours to 7 hours, and sleep quality improved from 5 to 6 out of 10. After letting the body rest and recover for 2 weeks, we increased the LDN again, causing the same cycle, but each time resulting in slightly improved sleep. Now at 5mg and after 4 months, the turnaround has been dramatic. I can sleep for 9 - 11 hours, of good quality sleep ( 8 out of 10 ), and things are improving at a faster rate. Energy levels are higher, brain fog has lifted, and most dramatic of all, my mood has improved 100%. I no longer feel depressed, despite being sub-optimal. I wake up happier, relaxed, and with a positive mindset, excited about the future. 

The key to LDN was definitely perseverance. Each stage caused initial upset, and then the reward phase followed. The big pay off being 4 months down the line. Definitely worth the wait.

Without your help, I would not have made this breakthrough.

I am now hoping to proceed to the next level of the program with my nutritionalist, and start the anti-microbial phase, and to be able to tolerate it well, due to the LDN and enhanced sleep. 

I have taken the advice you provided 4 months ago and incorporated healthy fats into my diet, started cooking with coconut oil,  watched YouTube videos and vastly improved my cooking skills. I'm struggling to add in fermented foods, and this is something my nutritionist is keen to do at a later stage.

I will now look into SIBO and get back to you."

LDN Stories- Laura and her Mood Swings, Menstrual Problems, Back Pain and Fatigue

I decided to start this series of LDN stories as I felt that people interested in starting it needed to hear about it- as and when patients felt the need to share. It is also an important lesson in persevering as some stages of this journey can feel very difficult. 

Names will be changed but I will give a brief history of why they came to me. I will then put forth their stories in their own words which, in my opinion, is more powerful.

Laura, 36, came to see me on recommendation from her nutritionist. In summary, this is her history:

  • drug addiction for 5 years in late teens

  • Chronic anxiety and mood swing with a diagnosis of 'cyclical mood disorder' from early teen

  • adrenal fatigue

  • menstrual irregularities later on

Despite a difficult and protracted ill health history, she is currently doing her masters in a difficult 'sciency' subject and working as a research assistant in the meantime. She also has a 4 year old child. So here is a smart and motivated person, feeling like there's no light at the end of the tunnel but wanting desperately to just be normal and be a mum to her child.

Here's her story:

I was referred to Harpal by my nutritionist, as he felt this would be a useful therapy for me in the aftermath of adrenal fatigue and multiple recurring health problems. Harpal prescribed the LDN sublingual drops as they give you a method to introduce the drug slowly and minimise potential side effects - and I was glad we took this approach. I was optimistic about the LDN, but the initiation process has not been without its challenges! Within a week of being on 1mg I came out in a spectacular rash on my back - a collection of bright red, itchy circles. I contacted the clinic and they assured me that this could be a side effect of the LDN, so I stuck on 1mg and didn't increase my dose until they had settled down - which was about 8 or 9 days. After that, increasing to 1.5 and 2mg did not cause the emergence of any new circles.

 

And at this point I realised some quite important things: the chronic pains I'd had for nearly a year in my back and knee had lessened, suggesting their root cause was auto-immune rather than a simple misaligned joint - and just a week before my osteopath had admitted she really wasn't sure how to treat me as she couldn't do any more than she already had. I'd previously experienced terrible 'flare-ups' of this back pain which would leave me on crutches, and left with months of weakness in my core muscles. On LDN this residual weakness had completely disappeared. I felt fitter and had dispensed with the nagging muscle fatigue that had become normal over the last year. So I learnt that maybe there was more to this situation than I had considered, but I didn't mind because I was clearly doing the right things to deal with it.

 

However, I had been dosing in the morning as I wasn't brave enough to try at night - as soon as someone mentioned the possibility of insomnia I was pretty determined to be a morning doser! But it turned out the mornings weren't the right time for me - although I had begun to see some pretty impressive benefits, I noticed my mood was low for quite a few hours after my 9am dose - to the point even I was convinced to try evening dosing. The first few nights my sleep was a bit messed up - shallow with vivid dreams - but I was like a different person the next morning. I felt happy, together - and my thinking seemed clearer than usual despite being pretty tired. Over the next week I adjusted to the evening dosing and was waking up feeling I'd had enough sleep. I was feeling better than I have in a long time - more emotionally connected, and more effective in my day to day life.

 

Then a combination of things happened - I put my dose up 1mg in 2 weeks, and started holding the sublingual liquid in my mouth for longer. I'd realised I hadn't been doing it as well as I could have, and started timing myself to keep it there 5 minutes. I guess this meant I was jumping up more than I was used to, and within a week all the positive benefits just disappeared. I had pains again, terrible fatigue, muscle twitches, foggy thinking and bad sleep. I felt pretty disappointed! After a couple of weeks feeling sorry for myself and assuming this was just how I was now, I realised it could be the LDN and contacted Harpal for advice. I followed her suggestion of dropping my dose back 1mg, and within a day I started feeling better. 3 days later I'm feeling really good again - pretty much back to where I was before. So the moral of the story was take it slow....LDN works in mysterious ways and it definitely doesn't like being rushed!

Oh and I forgot to add - I used to have to get up and pee every night, but since the LDN that has just stopped. Not sure why but I'm pleased it has!