Does anyone have any input about using Vancomycin for PSC? I’ve been reading many positive stories but my son’s diagnosis is very new so I don’t have much information yet.
It is helping many people with psc so you should try to find a doctor who is familiar with it. Most doctors are not familiar with it.
Maybe you can travel to Stanford University for an appointment and get the prescription for vanco. Mayo clinic may be ok too.
It is still in clinical trials but it will not be submitted for FDA approval because it is expensive process and vanco has no sponsor. So insurance may not cover it. In anycase, it has been used to treat children with psc over 20 years now. It is only known treatment at the moment.
This forum has lots of old discussions about vanco. Also google Stanford University and vancomycin.
My 2 cents. Nothing personal, just my personal opinion and some thoughts I wrote down when I read those materials.
Fox's paper has been out since 2008. Although the findings are supposed to be significant, there isn't any significant impact on treatment of PSC. I think there are some issues with that study. One thing particular, is the research design. There is no control group in that study, also no detail about the selection of patients. Thus, it's really difficult to interpret the result.
As to the new ongoing study, they claimed it as 'Phase 3', which is very confusing. In clinical tries for new drugs, Phase 3 is a large scale study before final approval. That seems not the nature of that study.
There is no way PSC is a simple disease. We still don't know much about it. It does seem to me that PSC is not a single disease. Patients are so different. Some people need transplant within a few years after diagnosis, while others have no obvious progress after years. Some responded to Urso and others don't. Some has UC. Some don't. It's hard for me to believe that at this stage, one treatment can have 100% positive effect.
I do believe vancomycin can work in some way to improve the clinical condition in some PSC patients. But its long-term effect is still unknown at this stage.
Ted said:
It is helping many people with psc so you should try to find a doctor who is familiar with it. Most doctors are not familiar with it.
Maybe you can travel to Stanford University for an appointment and get the prescription for vanco. Mayo clinic may be ok too.
It is still in clinical trials but it will not be submitted for FDA approval because it is expensive process and vanco has no sponsor. So insurance may not cover it. In anycase, it has been used to treat children with psc over 20 years now. It is only known treatment at the moment.
This forum has lots of old discussions about vanco. Also google Stanford University and vancomycin.
In rare diseases it is not possible (or required) to have large research groups (since it is impossible)…
Full scale clinical trial program may cost $100 million. Vanco has no corporate sponsor (patents expired) and Stanford U cannot afford full FDA process.
Stanford is going to publish the findings of the phase 3 soon. I’m optimistic.
I am going to choose to be optimistic too Ted. We meet with my son’s specialist on the 15th and I’ve already called in advance to have her look into it before we meet. Keep your fingers crossed my son is hopefully still in early stages. Thanks for the input and support:)
We know too little about PSC. All current studies are sort of like trying to win lottery. We might hit jackpot. It's always possible， but no need to keep our expectation too high.
Stanford group announced this new trial in early 2013. And they estimated its complete time at the end of 2016. So likely it's a two-year study (not really long-term). They want total 40 subjects, including 20 with both PSC and IBD, and 20 healthy control with positive IBD indexes. They want both children and adults. Naturally there should be 10 kids PSC and 10 adult kids. It's a very weak design again. What kind of results can you expect from this study? Or what can you expect more in addition to that old 2008 study? They will have ALP data for sure, and some immune function indexes. Anything new? I doubt it. Since the study will end at the end of 2016. Conference presentation could be reasonably expected at the end of 2017 and publication in 2018, if everything goes as expected.
I really don't expect too much from this group. But I still respect their efforts in looking for the cure for PSC. I just think there are better ways. For example, does anyone know whether they have follow-up with those pts in that 2008 study? That paper is submitted for publication in Jan. 2007. So the study should be done around 2005. It'd be very interesting to see the current condition of those kids. But they still need a control group.
Please don't feel offended by my post. I'm just disagree with your opinions, nothing else.
Great to hear your opinions here, thanks a lot. That’s the nicest part of the forum!
I agree that it would be nice to have larger study groups and follow up on earlier study group patients.
I’m not an expert in this area, but I did talk couple of times with the prople behind the study. My understanding is that one of the “new things” they are focusing here is the really sophisticated stool sample analysis. This might give new insight why vanco works for some people but for others.
As said earlier, Stanford team does not have much money. They cannot afford to do full FDA compliant trials leading to FDA approval. Also, they may not have access to all earlier patients (many were out of state etc).
There are some other psc vanco trials now. I’m think Minnesota has one now too…
The trial referenced ended last year and results are expected shortly. There was no control group; all patients were treated. The study followed each patient for 3 months and measured improvements by LFTs, imaging, and biopsy. The study is significant because of its size and because it is the first time the Stanford protocol has been applied to adult patients.
The Stanford group has released many prior studies regarding OV for PSC including their most recent from 2013. From 1995-2012 Dr. Cox had treated 33 pediatric patients. Long term, the patients with cirrhosis have shown improvement and the patients without cirrhosis have shown normalization in LFTs, biopsies, and imaging. Most have remission of IBD. There have been no significant side effects. The sample size is small but some of these people have been on treatment for 20 years. This data was pulled from a presentation Dr. Cox gave back in 2012. Dr. Cox is very approachable by phone or email and has answered many of my questions in the past.
There are nearly a dozen clinical trials and case studies regarding OV as a treatment for PSC at this point. A lot of people are cheerleading for OV because it's the best thing we've got right now to treat PSC. Worst case side effects are benign when compared to the side effects of PSC or typical IBD treatments and real world side effects are trivial. It quite literally doesn't hurt to give it a shot. If it doesn't work out, stop treatment and in a couple months your gut will return to "normal."
Redbrook - I've been using OV for about 2.5 years with great results. If you have any questions or need links to clinical trials, let me know.
Hi JTB,
Great information on the studies thank you. My 4 year old daughter was diagnosed with PSC in September of 2015. She has been on vanco for three months now. She has had great results so far. Her liver enzymes have normalized and her colitis symptoms have vanished she has gained some weight and height which had been stunted by the disease. She has her energy back, some days I forget she has this horrible disease. Besides the vanco she is also on pentasa for the colitis and was on Ursodiol. We are thinking of weening the pentasa and going exclusively with the vanco since she continues to do well without the Ursodiol. We are hopeful that we can reduce the prescriptions to just the vanco. If you don't mind me asking what is your regiment besides the vanco? We are giving my daughter folic acid and a multi vitamin as supplements. We seem to have caught the disease in its early stages as her bile ducts showed only mild damage. Thanks, David
jtb said:
The trial referenced ended last year and results are expected shortly. There was no control group; all patients were treated. The study followed each patient for 3 months and measured improvements by LFTs, imaging, and biopsy. The study is significant because of its size and because it is the first time the Stanford protocol has been applied to adult patients.
The Stanford group has released many prior studies regarding OV for PSC including their most recent from 2013. From 1995-2012 Dr. Cox had treated 33 pediatric patients. Long term, the patients with cirrhosis have shown improvement and the patients without cirrhosis have shown normalization in LFTs, biopsies, and imaging. Most have remission of IBD. There have been no significant side effects. The sample size is small but some of these people have been on treatment for 20 years. This data was pulled from a presentation Dr. Cox gave back in 2012. Dr. Cox is very approachable by phone or email and has answered many of my questions in the past.
There are nearly a dozen clinical trials and case studies regarding OV as a treatment for PSC at this point. A lot of people are cheerleading for OV because it's the best thing we've got right now to treat PSC. Worst case side effects are benign when compared to the side effects of PSC or typical IBD treatments and real world side effects are trivial. It quite literally doesn't hurt to give it a shot. If it doesn't work out, stop treatment and in a couple months your gut will return to "normal."
Redbrook - I've been using OV for about 2.5 years with great results. If you have any questions or need links to clinical trials, let me know.
Djamato - that's awesome she is doing so well and that you caught it early. I just take Vanco, Vitamin D and calcium supplements. I don't have IBD, so no drugs for that. I took high-dose Urso back when it was in style but stopped taking it about 10 years ago because it didn't normalize my LFTs and I didn't think it was doing anything to help.
Thanks JTB,
Glad to hear you are doing well and only have to take one prescription. My goal is the same for my daughter. Since she is so young and will likely have to be on vanco for years and years, I am trying to reduce the pills she must take. I am hoping more parents and patients can be made aware of vancomycin. It pains me to hear all the negativity. My wife and I made the decision to try vancomycin because the alternative was likely catastrophic. I don't know what the long term consequences will be for my daughter on vancomycin but I do know without this drug her health would be in serious decline. I only have three months experience but from the results I have seen personally and all the reading I have done, its a must step until we find a cure. For those thinking of trying vancomycin, my daughter has done well on the Akorn vancomycin generic. I eagerly await the new Stanford study. I think our best hope is if researchers can isolate the bacteria/microbe that is causing the liver problem, maybe then we can make progress for the cure. Thanks again JTB. Hearing from people who are having success against this disease, gives me and my wife strength and comfort. Good luck to you. Has vanco reversed any of your bile duct damage or only halted the progression? Either way that is good news.
jtb said:
Djamato - that's awesome she is doing so well and that you caught it early. I just take Vanco, Vitamin D and calcium supplements. I don't have IBD, so no drugs for that. I took high-dose Urso back when it was in style but stopped taking it about 10 years ago because it didn't normalize my LFTs and I didn't think it was doing anything to help.
Thanks, Djamato - I hope it continues to work for your daughter as well. My imaging has not improved after starting OV treatment, but for the first time my yearly scans have not gotten any worse. In hindsight I wish I would have done the research and started treatment a lot earlier, before I started to get really sick. It would have saved a lot of pain, money, and scar tissue.
1 Like
JTB - Do you know if once the liver enzymes have normalized do Dr. Cox or other Drs. recommend keeping the vancomycin dose the same indefinitely or do patients continue to take vancomycin but cut back on the amount of dosing?
The only good thing with my daughter;s situation is we caught it very early. I think it is easier to diagnose with children because the liver enzymes in children seem to get very elevated even in the early stages of the disease which I have read doesn't happen as much in adults. Sorry for all the questions, it's just great to have a resource who has been living with the disease for several years. I am just worried since she is so little and so young how the vanco will be on her kidneys. Currently she is on the dose used in Dr. Cox's research 50 mg/kg/day given orally 3 times a day. This equates to 250 mg 3 times a day for a total of 750 mg.
I went to see one of the Stanford docs recently and asked if I should start vanco. My LFTs have been close to normal (and recently fully normalized), imaging shows mild beading in liver. The recommendation was that no need. Main reason I recall was that they would have no way of knowing if it helps or not. So we agreed not to start vanco, but keep it in the reserve in case my condition gets worse.
A number of us have tapered down the dose successfully (I went from 1500mg/day to 500mg/day). My doctor looked at the recent OV for C-diff studies that found that lower doses were just as successful as higher doses and wanted to see if this also applied in our case. I still think it is a good idea to start at a higher dose until numbers normalize before moving to a lower dose. Dr. Cox has been experimenting with cycling his patients on and off OV for periods of time. Unfortunately I don't have specifics as to what has worked/not worked.
The handful of cases of OV permeating the gut that I've found all involved drug induced colitis (caused by drugs other than the OV). It is still a rare possibility but is thankfully easy to track. The metabolic function tests we get to track LFTs also keep tabs on kidney function. If creatinine is normal I wouldn't stress too much about there being appreciable Vanco in the blood. For added piece of mind you can also request the aptly named Vancomycin test that tests the amount of Vanco in the blood. Probably overkill for routine screening but wouldn't hurt if there are any suspect ear or kidney issues.
JTB - thanks for the information, I learned more in a couple of emails back and forth than I did in several days of research. I am sure I will have to hit you up with more questions in the coming months, so thanks again in advance.
Stool analysis won't tell us much. The sample size of this Stanford study is too small. Given the complex nature of PSC, variability among PSC patients would be big. Then a small study may not be powerful enough to detect the difference.
I saw that explanation on Stanford study somewhere else, not you, a different forum. I'm wondering whether that's the one from people there.
Ted said:
Great to hear your opinions here, thanks a lot. That's the nicest part of the forum!
I agree that it would be nice to have larger study groups and follow up on earlier study group patients.
I'm not an expert in this area, but I did talk couple of times with the prople behind the study. My understanding is that one of the "new things" they are focusing here is the really sophisticated stool sample analysis. This might give new insight why vanco works for some people but for others.
As said earlier, Stanford team does not have much money. They cannot afford to do full FDA compliant trials leading to FDA approval. Also, they may not have access to all earlier patients (many were out of state etc).
There are some other psc vanco trials now. I'm think Minnesota has one now too..
Only peer-reviewed publication matters. That's the key. Conference presentation or speech itself does not really have much impact, unless they do go through strict peer-review process, which usually is not the case at conference. I only saw two related publications from Cox group, no recent ones.
BTW, many times people on this forum mentioned that long-term OV has been used in IBD patients. I did my search with PubMed but only found publications with short-term uses. Anyone provide more information?
jtb said:
The trial referenced ended last year and results are expected shortly. There was no control group; all patients were treated. The study followed each patient for 3 months and measured improvements by LFTs, imaging, and biopsy. The study is significant because of its size and because it is the first time the Stanford protocol has been applied to adult patients.
The Stanford group has released many prior studies regarding OV for PSC including their most recent from 2013. From 1995-2012 Dr. Cox had treated 33 pediatric patients. Long term, the patients with cirrhosis have shown improvement and the patients without cirrhosis have shown normalization in LFTs, biopsies, and imaging. Most have remission of IBD. There have been no significant side effects. The sample size is small but some of these people have been on treatment for 20 years. This data was pulled from a presentation Dr. Cox gave back in 2012. Dr. Cox is very approachable by phone or email and has answered many of my questions in the past.
There are nearly a dozen clinical trials and case studies regarding OV as a treatment for PSC at this point. A lot of people are cheerleading for OV because it's the best thing we've got right now to treat PSC. Worst case side effects are benign when compared to the side effects of PSC or typical IBD treatments and real world side effects are trivial. It quite literally doesn't hurt to give it a shot. If it doesn't work out, stop treatment and in a couple months your gut will return to "normal."
Redbrook - I've been using OV for about 2.5 years with great results. If you have any questions or need links to clinical trials, let me know.
I should clarify my opinion. I'm not saying that Vanco is not working in PSC patients. What I mean is that there is no evidence supporting Vanco, particularly long-term use, is an effective treatment for PSC. And I don't think the new study will provide much new evidence.
Don’t go to the Mayo looking for vanco. My husband’s liver specialist is the head of the department in Jacksonville and every year that we see him he chews us out for taking it. Tells us there is no evidence to support it and we could be doing harm but his liver enzymes and function look good. Granted my husband is on a small dose 250 3xs a day. They only do ‘science’ based medicine there and are very by the book. We get it from a local GI in east Tennessee. So far my insurance has covered it for the past 2 years. I have a large copay 175.00/a month with United but blue cross blue shield covered it for 5$ a month.