The Georgetown study was published in the American Journal of Gastroenterology. The study won the Presidential award at the conference as it was the highest rated abstract in the liver category. This was great exposure for PSC and for OV as a treatment.
DHZ - there is plenty of scientific and anecdotal evidence providing support for the long-term use of OV for the treatment of PSC. While you may not appreciate the existing research or the experiences of the people posting in this forum, this does not mean as youāve suggested in multiple topics that this information ādoesnāt matter.ā We PSCers are a small group and we will never see the types of studies that you are seeking (even the biggest Urso studies are insufficiently powered and weāve resorted to aggregating studies for better data). The reality is that we must rely on smaller studies and our shared experiences to guide our decisions. These case reports, presentations, and trials do in fact influence the decisions of doctors and help to develop future research. This data is extremely relevant for any PSC patient making the decision to treat their disease.
I post here because I remember vividly the depths of hell that was symptomatic PSC (that itch!) and I donāt wish that on anyone. OV brought me back from there and has given me the healthiest years of my adult life. Like you, I wondered if it was the random variations of the disease or the drug leading to my improvement so I stopped treatmentā¦and got sick all over again (n=1, but was repeatable). For the younger set, the end game of PSC is concrete: cancer, transplant, death. In my opinion, OV treatment is a good alternative even accounting for as yet unseen or unknown side effects. Spreading awareness of this potential alternative to the natural course of PSC is, again in my opinion, a good thing.
As much as I genuinely like to help answer your questions on the topic, your responses that the information should immediately be dismissed doesnāt exactly lend to a lively discussion. Imperfect trials and format aside, what is the issue here? Have you thought about a three month trial yourself?
I might be harsh in my language. But itās nothing personal. Iām concerned about misinformation regarding PSC treatment. Iām not an expert so my opinion is always open to criticism, but itās needs to be in a reasonable way.
I have been active in biomedical research for almost two decades. Trust me, I know whatās real publication.
It really surprised me that single case report could win a Presidential Award. And then itās no surprise because I couldnāt find such information from ACG website.Did I miss it? Help me out here, please.
I did a little research, but couldn't find any information regarding Presidential Award at Annual Meeting of the American College of Gastroenterology, where that Georgetown poster was presented (2015). There are awards for Abstract/poster, but none of them labeled as Presidential Award. And I couldn't find that Georgetown study was listed as a winner. There is a ACG President's Special Recognition Award. But that's to honor someone's long-term achievements in the field.
Since it is there on that website, my guess is that could be something from a specialty committee, but not directly from ACG. Otherwise, it'll be listed for sure.
BTW, clinical trial/research is not that high-end. It's more about persistence and motivation. No clinical study is perfect. It's not like those studies with mouse or cultured cells or genes. In those you can try to have a perfect design. For clinical study, all you can do is to try to make it better, more convincing. One thing I don't understand about this Stanford group is that they have patients but seems have no concern about quality. They have been working on this for more than 2 decades and should have enough patients. For that Vancomycin project, they can just choose a simple case-control design, matching a similar PSC patient without taking vancomycin for each PSC patient receiving vancomycin, then do a comparison in the end. Case-control study is not a very strong design, but still way better than case report. It may not cost much. If patent insurance is willing to pay for the cost of vancomycin (everything else is standard treatment for PSC), then you only need a signed consent form
OV has been studied long time by Stanford University. The project does have much funding, and there are various issues in play eg different vanco brands etc.
OV is now studied in several other institutions too.
Stanford is, depending on the exact criteria (eg number of Nobel laureates etc) the number #1 university in the world (some rankings have it #3 after Oxford and Caltech). Researchers there are best in the world.
Readers in this forum may believe some anonymous internet troll or trust Stanford University led promising project.
Regarding the Stanford trials: Ultimately this is about money. The patent is dead. It is real hard to push this stuff through without a drug company making it happen (and no, this isn't like Urso which had drug company money help push it into the old PSC clinical guidelines). PSC is a small population so there isnāt a whole lot of public funding either. Each patient is paying for their drug and for the screening. If their insurance isnāt on board (this thing isnāt FDA approved, after all), that patient canāt be involved in the trial. The Stanford doctors originally involved are pediatric doctors so our small population just got smaller. Oh, and you have to be local because there isnāt any money to fly people around. Are you surprised that we have a little more than a dozen patients in the previous trials? Personally, Iām surprised they pulled in 40+ for the most recent trial (adding adults helped). So whatās going to provide better data, a trial with 14 treated patients or a trial with 7 treated patients and 7 on a placebo? At higher numbers the dynamic changes, but with a handful of participants you arenāt going to get a whole lot of beneficial data by having half the participants sit on the sidelines. Another thing: between 1993 and 2012 Dr. Cox treated 33 pediatric patients and all benefitted from the treatment. It turns out there are some pretty good ethical reasons why you donāt see many trials with controls for the pediatric set, particularly when the treatment has been effective every single time it has been applied. Would you sign your daughter up for a controlled study or would you rather treat your daughter off study to ensure she received the treatment? These trials are set up the way they are for very good reasons not because the doctors are sloppy and donāt understand science.
The Georgetown case study is about me. Those were my doctors at the conference. The award is not a fairy tale.
I receive a lot of my information by talking to doctors, not just by searching the internet. I encourage everyone to do the same. You may not be convinced by the existing research, however there are a lot of big name PSC doctors, Dr. Lindor included, who are convinced enough to pursue further research into the treatment and who are actively treating their patients.
There is nothing productive about what is going on here and this line of conversation is completely inappropriate for the topic (sorry Lisa!) and for a forum that is about providing support. Letās drop this.
I think Iām obligated to provide other side of the story. Iām a well-trained biomedical researcher and have both publications in prestigious journals in the field and funding for my research. Iām experienced in clinical research and I think Iām qualified to present my opinions here. Whether you accept my opinions is up to you. But itās about science, science only. If you do disagree, please present your own reasonable arguments. You donāt have to be a PhD or MD to do that. You just need to be reasonable.
Stanford of course is a top research institute in the world. But it does not mean each individual is top notch in the field and its top notch status is in each single research field. Academia isnāt as pure as snow white. For a chitchat story, search āDukeā and āAnil Pottiā.
Funding is only a valid excuse to regular patients. Around year 2000, your chance to successfully get a NIH funding is a little bit below 50%. For a MD, that number is even higher. Thatās golden years for biomedical research. How much money Iām talking about? One million for 4 years. And you can hold multiple grants. Whatās the chance now? Less than 10%.
Ted said:
OV has been studied long time by Stanford University. The project does have much funding, and there are various issues in play eg different vanco brands etc. OV is now studied in several other institutions too.
Stanford is, depending on the exact criteria (eg number of Nobel laureates etc) the number #1 university in the world (some rankings have it #3 after Oxford and Caltech). Researchers there are best in the world.
Readers in this forum may believe some anonymous internet troll or trust Stanford University led promising project.
Actually I find the discussion rather misleading as well and going nowhere and certainly NOT helpful to patients.
Abastracts and case reports are presented for DISCUSSION. As DHZ said "Most of the time, data presented at professional conference is not complete, or well-thought. The purpose of such presentation is to solicit scientific discussion on the subject, so the authors can go home and further work on the project, or write a grant proposal to seek financial support for future project." They are not research nor presented as such. Some are better than others.
In the case of Vanco, they are presented as a possible off label use. Vanco has long been studied as a DMARD medication. used to treat inflammation (such as inļ¬ammation and ļ¬brosis of both intrahepatic and extrahepatic bile ducts present in early PSC before it moves on and destroys ones liver) not as ABX. jtb indicated as much. Heres the thing the question is not whether Vanco works or not.
Sometimes it does sometimes its does not (as do other anti-inflamatory/DMARD treatments that have been studied that cost a lot less than 3 grand a month) The question is rather whether there is a difference between PSC and PSC overlap syndromes. These are conditions with diagnostic features of both PSC and other immune mediated liver diseases including autoimmune hepatitis and autoimmune pancreatitis. The former do not respond to DMARD treatment and the latter do (for a while)
As I read this I don't find an interesting discussion. I find some serious misunderstanding coming across that somehow and somewhere there is a miracle cure for PSC that is being withheld from patients. That is very far from the truth. DHZ presented a pretty good explanation. Just not sure it was understood. (I will disagree with her on one point Case management studies aren't worth the paper they are printed on. The evidence at BEST is anecdotal and they all have a poorly defined cohort. BTW it only takes 16 patients in a tightly defined cohort to be statistically significant
NOW that is the end of the science. I can't out science talk anyone. I'm well published as well. I find it incredibly disturbing that ANYONE would infer to a member of this forum facing a crisis with a loved one that critical treatment is being withheld from them based on case studies and abstracts they know nothing about used to treat patients that they have no clue what their charts contain or whether or not the treatment is even appropriate.
I apologize to Lisa that this thing got so out of hand. So please members cool it. There is some good stuff here and I would hate to poof it or close this thread. Lets see if we can step up and offer Lisa some support.
I agree with most of what you said. I do agree that it seems some members did not really read my posts. This will be my last post on this subject, unless another time Vanco is presented as a miracle drug.
Here is the summary:
Short-term Vanco worked in some PSC patients, as has been shown in some IBD patients. But we still don't know the effect of long-term use.
Vanco may work on PSC as a immunomodulator. But at first, it is still a strong antibiotics. Lone-term use may affect other people in the community, not just PSC patients. Even for PSC patients, active UC may increase the chance of vanco entering into blood.
As a PSC patient myself, I'm frustrated by the lost opportunities. Stanford group should do better, could do better, and is able to do better. But they didn't deliver. New trial won't do much better.
Vanco is still in its exploratory phase, even after so many years. There are too much exaggerated information regarding this drug and people involved. I just want to remind people don't raise your expectation too high. That does not mean that you should not try it. But just don't expect too much. There is no sign yet that it's the miracle we are looking for. Mayo has a good reason not to pursue in this direction.
At this time we would like to request no further discussion to this post. We will leave the post up for now but for the record the moderators of this forum are in agreement that we do not like the way this discussion has gone. We are here to provide helpful and needful information to patients and families living with this incurable disease called PSC. We are to give them hope, not to debate on the merits of this organization or that organization. None of us are here to proclaim to you that we are experts, doctors, etc., but we are patients who have lived with this disease and are here to provide an avenue whereby the members of this group can find information. If we will all go back and search our hearts and remember the day we were given our diagnosis, it was a terrifying experience. We thought we were going to die and what would become of our families. How would are spouse and children go on without us. But the more we were educated about PSC the more hope we found. This forum has been one small way we could share that hope with others. It's not always a death sentence, there is time, there is hope. Without hope what shall we do. May we all have learned something from this and endeavor to do more in the future to stay with the original intent of why Ben's Friend's was started in the first place. Thanks for listening.