sodium phenylbutyrate

[kellysal]

I just started SP last week. I read the same thing about limiting protein. I have an appointment with my Dr. tomorrow and I was going to ask the same question

 

[kellysal]

I asked my Dr today . She said limiting protein only applies to people taking it for Urea cycle conditions

 

[Samkl]

I hope this doesn’t count as hijacking. Our PALS had gotten a prescription for sodium phenybutyrate and had taken it to a compounding pharmacist to get made up, but the pharmacist hadn’t prescribed it, or the brand that is dispensed in Australia even for urea cycle disorder. They’ll probably work it out but I wondered if this form (granules?) of the substance is familiar to anyone, and what dosage is appropriate and in line with what was prescribed in the AMX0035 trial. Will be off label unfortunately so will cost an
arm and a leg. I’ve attached a photo of the script. Many thanks x

 

[lgelb]

The CENTAUR trial used 3 g twice daily. Since the granules are 483mg/g, that would mean if the granules are equally bioavailable as whatever form the trial used, that you would want to administer 6.2g twice daily. Of course, the absorption and effectiveness will be different without the TUDCA that is the second ingredient in the Amylyx compound.

For safety, I would also make sure you read all the cautions and contraindications as regards your PALS' medical history, and note the lab tests recommended for monitoring. --Laurie

 

[Samkl]

Thanks Laurie, I’ve passed that on, and our PALS is most grateful to you. X

 

[Nikki J]

On a webinar last night Dr Brown from UMass said he was having good luck with insurance appeals by sending the reviewer the NEJM article Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis - PubMed

 

[lisa g]

Thanks Nikki, I watched Dr Brown last night. Anyone trying to get Sodium Phenylbutyrate should print that article in case insurance denies coverage. It was interesting to hear him say that he uses that article on appeals and he has good success with it.

 

[kellysal]

Rea
Have you had any side effects from it? I have been on it for 12 days and my face broke out in cysts ( similar to large pimples) I stopped it for 4 days and they went away. Just wondered if anyone else experienced this
Kelly

 

[Samkl]

Our PALS is still having issues with the supply of sodium PB. The main problem is the pharmacist not being sure about how to prescribe it. He has all the study data and any information that I have been able to gain from Laurie and anyone else, I have passed on. Could I ask anyone who has it prescribed by a pharmacist to let me know what the actual prescription is for? Or what is actually written on the bottle of tablets or granules? Many thanks

 

[lisa g]

I haven't received mine yet but this is how my neurologist wrote the prescription:
Take 3grams by mouth 2 times daily.
Start with once a day for three weeks. Increase to twice daily if well tolerated.
Are you also using Tudca? I started using 250mg and worked my way up to 2,000mg a day. Taking it in the dosage as follows: 750mg in the morning, 750mg in the afternoon than 500mg in the evening.

 

[GXTrex]

@lisa g

Hey Lisa,
How long Did it take you to go up from 250 mg to 2000 mg of Tudca? My PALS is only able to handle 500 mg every other day (he got 500 mg tablets instead of 250 mg by accident) and he has been on that dosage for a couple weeks now.

 

[lisa g]

It took me around 3 weeks some people expierence stomach upset so be careful and take it slow.

 

[Samkl]

There is still a holdup for our PALS getting a PB prescription. Both his doctor and pharmacist are on board but not sure of what to prescribe. I don’t know what the issue is - I’ve passed on all information I’ve gathered here. I’ve emailed, and had a response from the Hopewell Pharmacy in NJ, but the person who responded was not able to give me specifics, but did tell me about the service available for international patients. As we have a doctor and pharmacist we haven’t pursued that Avenue, but can anyone tell me what is actually on their script so I can pass that on? I’m not sure whether anyone on Australia is on this medication.

 

[McCamy]

Can a biochemist explain why the trials are using sodium phenylbutyrate an expensive orphan drug and not sodium butyrate a relatively cheap nutritional supplement which is also a histone decacytelase inhibitor both of which target HDAC1? Is there something about binding excess nitrogen and allowing it to be excreted that is beneficial for ALS? I bring this up because of the studies in mice models of ALS that show benefit from adding sodium butyrate to drinking water. Butyric acid is the chemical that healthy bowel bacteria create from legumes and fiber so it is natural to the human GI tract and might have fewer side effects. Does the phenylbutyrate have different properties that make it better than plain butyrate?

 

[KimT]

I bet someone can explain it over at ALSTDI forum. I've been taking sodium butyrate for about three years.