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Calliope

Off Label Prescriptions Approach to MDS

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New member here.  My husband had a recent tentative diagnosis, but as I learn more, he has all the first signs, although nothing noticeable except a light hand tremor and minimal arm swing and nothing that bothers him yet.  After reading this forum we decided to try TUDCA which helped everything right away, sleep, arm swing, mood, anxiety.  The tremor is still there but he says it takes the edge off his noticing it which makes all the difference.  Then one day after reading an anecdotal post he tried aloe vera juice from the grocery store just in case it could help.  The tremor stopped immediately.  I looked it up and the aloe vera study on mice seems preventative, in other words it produced recovery having to do with the tyrosine which sounds like it might something that helps stop progression like Ursodiol (UDCA) as opposed to dopa.  The aloe vera only stops the tremor when he takes it with the TUDCA.

So it's very comforting to have at hand the ability to hopefully slow it down from over the counter, but we are going to meet with our first MDS soon.  We would like to see if she is willing to give him ursodiol off label since the end of the study has been slightly delayed to replace the TUDCA.   We would also like to try the isradiprine which is off label, but my husband is already on blood pressure meds and a channel blocker.  So we are hoping we could convince the MDS to switch him to Isradipine instead of the other two meds.

I have read that people here present their doctor with things they print out from the internet which helps the doctor in this type of situation.  Can anyone tell me what kind of info might help them or what approach you might suggest that might make the doctor's decision easier?

 

CAVEAT:  Don't try either of these over the counters including aloe drinks without serious research or talking to a doctor.  Both can be dangerous under certain conditions.

 

 

 

 

 

 

 

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Hiker,  UDCA or Ursidiol is a drug nearing the end of its trials for Parkinson's that may stop or slow progression.  You can get a very similar compound over the counter called TUDCA, tauroursodeoxycholic acid, which has been found to work as well for some people with Parkinson's.  It's a chemical found in bile and is prescribed for some kind of stones, gall or kidney maybe.  Body builders use the over the counter compound TUDCA as a supplement to help their liver.  It's called TUDCA on the container.  For Parkinson's you can take 1250 mgs a day.  You can take it in five 250 pills throughout the day.  But don't do this on your own without making sure you don't have a condition that the combination could make it harmful for you.  

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17 hours ago, Calliope said:

Hiker,  UDCA or Ursidiol is a drug nearing the end of its trials for Parkinson's that may stop or slow progression.

That's not accurate. Phase 1 human trials have only just started for UDCA in Parkinson's; the trial is open-label (not placebo-controlled) and small. There have been a couple small human trials of TUDCA, but in patients with ALS and MS, not PD. More here: https://scienceofparkinsons.com/2018/04/16/udca/

UDCA/TUDCA *may* eventually prove to have benefits for people with PD, but it is far too early to say, one way or the other. The vast majority of drugs that show promise in animal models or early human trials fail more rigorous trials.

As miracleseeker suggests, there's at least one UDCA/TUDCA evangelist on these forums. If he turns out to be right, it's because of a lucky guess, not hard data.

Full disclosure: I take isradipine off-label (prescribed by my MDS), because there's a chance it will be shown to slow progression and I didn't qualify for the Phase III trial. But I didn't start taking it until it had already passed Phase II (large-scale testing for safety in PD patients) and Phase III (large-scale testing for efficacy) was underway. Phase III wrapped up last month, and results should be published sometime in 2019. If results are positive, I'll continue taking it; if not, I'll stop immediately and save a couple bucks every month.

Edit to add: It seems unlikely to me that readily-available and widely-used drugs or supplements will be game-changing PD treatments--if they were, we'd already have the data. And there's no reason to think a drug that slows progression will have symptomatic benefit (and vice versa). It's well known temporary improvement in PD symptoms is highly susceptible to placebo effects, hence the reason open-label studies should be viewed cautiously.

Edited by ShopGuy
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'Off-label' = prescribed for some use/condition other than that approved by the FDA. It's legal and common for doctors to prescribe drugs off-label, but insurance companies can (and do) refuse to pay for off-label rx. In my case, isradipine was prescribed by my MDS, after we figured out I wouldn't qualify for the STEADY PD-III trial.

Generic isradipine is cheap--my insurance has always covered it, no questions asked. Dose is 10 mg/day: (2) 5 mg capsules, each morning. Based on the Phase III protocol, I should prob. split up the dose, morning and evening, but haven't had any ill effects doing it this way. Edema is a common side effect, but I haven't had any.

Never experienced any effect on symptoms, but wasn't expecting any. Progression has been slow for me, to-date, but I exercise and also have high-normal blood urate levels (there's some evidence higher urate levels are associated with slower progression, and a urate-raising supplement is in Phase III trials). As you probably know, YOPD and tremor-dominance are associated with slower progression, so I tick those boxes, too.

I guess I feel pretty fortunate to have the 'right' kind of PD. Although not quite as fortunate as the other 499 out of 500, who don't have PD at all. (I'm joking--sort of.)

A few other people here are taking isradipine, including one person who was part of the STEADY PD III trial. Search the forums, and you should find more discussion.

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One more note regarding dosing of TUDCA. If you follow the link in my post above to the Science of Parkinson's blog, you'll find that of the few studies of TUDCA in humans, none of them recommend doses as high as 1250 mg/daily. In fact, one study (in liver patients) found side effects between 1000-1500 mg, and recommended doses of 10 mg/kg body weight for future studies. Under that guideline, 1250 mg would only be appropriate for someone 275 pound or heavier.

If I had ALS, chronic diarrhea from taking an unproven compound might be worth it, for the chance at slowing the progress of a fatal disease. But PD isn't ALS--not even close. 

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On 12/10/2018 at 9:13 AM, ShopGuy said:

One more note regarding dosing of TUDCA. If you follow the link in my post above to the Science of Parkinson's blog, you'll find that of the few studies of TUDCA in humans, none of them recommend doses as high as 1250 mg/daily. In fact, one study (in liver patients) found side effects between 1000-1500 mg, and recommended doses of 10 mg/kg body weight for future studies. Under that guideline, 1250 mg would only be appropriate for someone 275 pound or heavier.

If I had ALS, chronic diarrhea from taking an unproven compound might be worth it, for the chance at slowing the progress of a fatal disease. But PD isn't ALS--not even close. 

ShopGuy, You're right about the date for the Ursodiol.  I was mixing up the two studies.  But for the TUDCA, I'm confused.  My husband would be about 80 kilograms.  Can you redo the math for that 275 pounds?  I think your numbers may be off.  I am finding most sites say 20/mg/kg TUDC per day is safe and common dosage for fatty liver patients is 1750 mgs/day.  I'm not seeing any warnings or any toxicity at the levels my husband is taking in any studies anywhere.  There is one study that found problems with patients who had PSC, which is a rare chronic liver disease.  They did well on TUDCA until five years, and then they had real problems, but the studies say it seems to be PSC-related. 

Another thing, ShopGuy, that I wanted to mention.  Both times you have replied to my posts you have said something negative about how I am approaching PD, once in this post and once when I originally found out about my husband's diagnosis.  Although you may have been correct, the way you wrote made it sound  as though I was doing something wrong.  Since this has happened twice, I would just suggest that you might want to be a little more careful with how you come across to newcomers. This is a very tough diagnosis, and we're already pretty scared about doing the wrong thing.  

As far as some of the other comments by others on this post directed to members besides me, I would also mention that when we as newcomers hear people being sarcastic or denigrating to other forum members we do not feel we are in a welcome space.  It's actually kind of shocking when you think you have found an oasis of people who understand to hear fellow sufferers talking about other forum members that way.  

If anyone actually has an answer to my original question I would be very appreciative.  The question was what kind of print out would you bring to a doctor to help familiarize them with an off label medication that may be helpful for PD.  

 

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Unfortunately there are a  lot of pettiness that goes on here.  Sulk and move on.  I prefer to react to what I see and not so much who said what.  Maybe I didn't in the past but I do now.  If someone I had a problem with previously posts something in the present that I agree with I will applaud and give a shout out.  Same goes with not appreciating what I see and can have a discussion on that.  There is nothing worse than awkward pauses to stop the flow of communication.   We are all here to learn and contribute.  We have different personalities and sometimes we say things that may rub people the wrong way.  I like to think that we should respect people's lingo and take it at face value.  No one insults anyone on purpose or have an agenda to crush anyone's spirit.  At least I don't so I assume the same of everyone.    Sorry Calliope for hijacking your thread to make this point.   I hope you will get more answers to your particular questions and make lasting friendships with other forum members.  I know I have.

 

 

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On ‎12‎/‎11‎/‎2018 at 6:38 PM, Calliope said:

what kind of print out would you bring to a doctor to help familiarize them with an off label medication that may be helpful for PD.  

1. From now until the actual appointment with the MDS keep a chart of all medications and foods consumed daily and their effects. This is to establish a baseline.

2. In what way are the medications currently prescribed working or not working? The MDS will have to weigh interactions between existing treatments and any proposed "off label" intervention.

3. What research has been done using the proposed "off label" medication for its on label prescription? Is it really benign? If possible, send this information to the MDS' s office staff in advance of the appointment. It is likely that he/she has better access to scientific information than you do. In fact, if the MDS has a well staffed office (residents, PA's, nurse practitioner) he/she will likely assign one of them to research appropriate information prior to your scheduled appointment.

Your MDS is being asked for an unbiased opinion but is not doing so in an unbiased circumstance since you have already indicated some use of off label drugs (aloe vera juice). This would be the opportunity to have a frank discussion about what your MDS wants to know before you start any new supplements (by definition off label) and what results you hope to achieve by asking the doctor to prescribe something off label.

Hope this helps. I am not an expert on anyone's disease or progression except my own.

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On 12/9/2018 at 1:31 PM, ShopGuy said:

That's not accurate. Phase 1 human trials have only just started for UDCA in Parkinson's; the trial is open-label (not placebo-controlled) and small. There have been a couple small human trials of TUDCA, but in patients with ALS and MS, not PD. More here: https://scienceofparkinsons.com/2018/04/16/udca/

UDCA/TUDCA *may* eventually prove to have benefits for people with PD, but it is far too early to say, one way or the other. The vast majority of drugs that show promise in animal models or early human trials fail more rigorous trials.

As miracleseeker suggests, there's at least one UDCA/TUDCA evangelist on these forums. If he turns out to be right, it's because of a lucky guess, not hard data.

Full disclosure: I take isradipine off-label (prescribed by my MDS), because there's a chance it will be shown to slow progression and I didn't qualify for the Phase III trial. But I didn't start taking it until it had already passed Phase II (large-scale testing for safety in PD patients) and Phase III (large-scale testing for efficacy) was underway. Phase III wrapped up last month, and results should be published sometime in 2019. If results are positive, I'll continue taking it; if not, I'll stop immediately and save a couple bucks every month.

Edit to add: It seems unlikely to me that readily-available and widely-used drugs or supplements will be game-changing PD treatments--if they were, we'd already have the data. And there's no reason to think a drug that slows progression will have symptomatic benefit (and vice versa). It's well known temporary improvement in PD symptoms is highly susceptible to placebo effects, hence the reason open-label studies should be viewed cautiously.

May I ask, Shopguy, do you take any other medications for PD?  Do you feel as though the isradipine has helped arrest PD progression for you?  I’m very interested - as I’m sure lots of folks are - in seeing the phase III results next year.

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3 minutes ago, Rosencrantz said:

May I ask, Shopguy, do you take any other medications for PD?  Do you feel as though the isradipine has helped arrest PD progression for you?  I’m very interested - as I’m sure lots of folks are - in seeing the phase III results next year.

Oops...  if I’m understanding how to read tags, it appears you’re currently taking Azilect with the isradipine?

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fire 1 fl, thank you.  I guess I can print out the study for the isradipine that shows it's nearing the end and that it's presently in use so safety has already been tested.  I appreciate the help!

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Calliope, I would only add one more caveat. 

For many drugs following their clearance to a label:

(1) Long-term issues may till arise.

(2) The absence of evidence is not evidence (I.e., finding no contraindications or detrimental side effects  does not mean there are none, just still testing). 

I long ago stopped anti-cholesterol statin drugs because they made me sicker. I was able to achieve side effect free results with better diet and exercise.

As always, live by the rule-of-thumb, all drugs are poisons that occasionally have a beneficial side effect.

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fire1fl, So true.  Even supplements are very questionable.  My husband is taking aloe vera juice, and despite the fact that it's in every grocery store, it can be highly toxic.  I chose the only brand that has had a study done on it because it takes a very specific processing for it to be safe.  Even then, it's impossible to really know.  The other supplement my husband is taking, although it's tested and trials for off label use showed no toxicity, even on this forum when I did a search for a product that mostly contains TUDCA, the Parkinson's professional in that section said it was perfectly safe, neglecting to mention the two medical conditions that if you have make it unsafe to take because he didn't see the main ingredient in the label.  So even the best professionals can miss something.  

I truly wish this condition could be dealt with without using chemicals.  I was so hopeful in the beginning when I read about the people who only used exercise or meditation/Qigong/Tai Chi successfully.  And then saddened to find out that there were so many others who are true athletes or meditators and still need medication.  I am feeling hopeful about the blood plasma from young people doing so well in studies.  That method seems like a comfortable hybrid between medicine and nature, fingers crossed.

 

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I just wanted to report that we successfully got a prescription for israpidine (dynacirc) which is in Phase III trials now from our GP for my husband.  He is on regular blood pressure meds and we got her to switch to israpidine instead.  I ended up bringing a printout of the first page of the actual study of the Phase III trial as opposed to something less technical.  I gave the printout of the study with a handwritten note on it saying that the neurologist had recommended we switch blood pressure meds (which the neuro did recommend at our most recent visit).

Giving the printout to the assistant first so that the regular doctor could read it without the pressure of us in the room seemed to work well.  It seems like she looked it up before coming in to talk to us.  The good news is that not only is she willing to switch blood pressure meds to israpidine, but the dosage they are giving for the trials happened to be on the first page as well, and she said since the paper said 10 mgs that's what she would give him, which is hugely important.  Anyway, I'm very, very happy he gets to be on this drug in early stages of his PD!  

 

 

 

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Good for you.  I didn't think it would be a problem in the first place to basically switch brands.  My mom was on Dynacirc before she got PD but circumstances made her switch to Norvasc and that was that. 

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Just now, miracleseeker said:

Good for you.  I didn't think it would be a problem in the first place to basically switch brands.  My mom was on Dynacirc before she got PD but circumstances made her switch to Norvasc and that was that. 

Luckily the doctor told us if the Dynacirc doesn't work for his blood pressure he can just add something else and stay on the Dynacirc.  So unless for some reason the insurance doesn't cover and it costs some crazy amount, I think we're set.  Hopefully the drug will be on label in the next few years anyway.

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On ‎12‎/‎16‎/‎2018 at 10:56 AM, Calliope said:

fire1fl, So true.  Even supplements are very questionable.  My husband is taking aloe vera juice, and despite the fact that it's in every grocery store, it can be highly toxic.  I chose the only brand that has had a study done on it because it takes a very specific processing for it to be safe.  Even then, it's impossible to really know.  The other supplement my husband is taking, although it's tested and trials for off label use showed no toxicity, even on this forum when I did a search for a product that mostly contains TUDCA, the Parkinson's professional in that section said it was perfectly safe, neglecting to mention the two medical conditions that if you have make it unsafe to take because he didn't see the main ingredient in the label.  So even the best professionals can miss something.  

I truly wish this condition could be dealt with without using chemicals.  I was so hopeful in the beginning when I read about the people who only used exercise or meditation/Qigong/Tai Chi successfully.  And then saddened to find out that there were so many others who are true athletes or meditators and still need medication.  I am feeling hopeful about the blood plasma from young people doing so well in studies.  That method seems like a comfortable hybrid between medicine and nature, fingers crossed.

 

Has anyone heard about young plasma infusions? There’s a double-blind / placebo-based study in Houston that has released some pretty impressive one month results. www.YoungPlasmaStudy.com

 

According to the study, critical disease-conditions such as pain, facial expression, speech, handwriting, tremors, rigidity and falling showed dramatic improvements.

 

https://docs.wixstatic.com/ugd/3e0a14_8dc827840990442a9ceb0d1b371cfb2d.pdf

https://docs.wixstatic.com/ugd/3e0a14_3b0d176d57ec4ababc8699c5852939d7.pdf

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On 2/5/2019 at 2:07 PM, plasmaunitedtx said:

Has anyone heard about young plasma infusions? There’s a double-blind / placebo-based study in Houston that has released some pretty impressive one month results. www.YoungPlasmaStudy.com

 

According to the study, critical disease-conditions such as pain, facial expression, speech, handwriting, tremors, rigidity and falling showed dramatic improvements.

 

https://docs.wixstatic.com/ugd/3e0a14_8dc827840990442a9ceb0d1b371cfb2d.pdf

https://docs.wixstatic.com/ugd/3e0a14_3b0d176d57ec4ababc8699c5852939d7.pdf

That's too bad.

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On 12/15/2018 at 3:47 PM, fire1fl said:

1. From now until the actual appointment with the MDS keep a chart of all medications and foods consumed daily and their effects. This is to establish a baseline.

2. In what way are the medications currently prescribed working or not working? The MDS will have to weigh interactions between existing treatments and any proposed "off label" intervention.

3. What research has been done using the proposed "off label" medication for its on label prescription? Is it really benign? If possible, send this information to the MDS' s office staff in advance of the appointment. It is likely that he/she has better access to scientific information than you do. In fact, if the MDS has a well staffed office (residents, PA's, nurse practitioner) he/she will likely assign one of them to research appropriate information prior to your scheduled appointment.

Your MDS is being asked for an unbiased opinion but is not doing so in an unbiased circumstance since you have already indicated some use of off label drugs (aloe vera juice). This would be the opportunity to have a frank discussion about what your MDS wants to know before you start any new supplements (by definition off label) and what results you hope to achieve by asking the doctor to prescribe something off label.

Hope this helps. I am not an expert on anyone's disease or progression except my own.

I'm afraid I didn't see your response.  Thank you so much.  The appointment went very well and my husband is on the off label drug now.

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