Primary sclerosing cholangitis is a serious rare disease for which there is no recognized effective treatment. Oral vancomycin has shown substantial promise, but the American Association for the Study of Liver Disease is just about to issue guidance that it should be used only in clinical trials. Why this is happening is unclear.
My daughter was diagnosed at age 17 and suffered for 10 years from fatigue, persistent diarrhea, terrible itching, elevated liver enzymes, and abnormal MRI of her bile ducts. We heard from another parent about oral vancomycin, and convinced her doctor to try it. Within a few weeks, she had the first formed stools she had had in 10 years. Her itching resolved, and eventually her liver enzymes returned to normal, her colitis resolved completely, and even her MRCP returned to almost normal. I am personally aware of multiple other such stories. She has ben asymptomatic now for 6 years.
Vancomycin doesn't work for everybody, and doesn't work for most patients who have IBD without PSC.
But to restrict it to clinical trials, as the new AASLD guidance does, when there is no demonstrated harm, is misguided. Insurance companies are already starting to deny coverage. Physicians should be allowed to use their judgment, with consent of the patient.
Please consider signing the petition. If you care, you're a patient advocate.
What do you think it might take to satisfy the criteria of the AASLD guidance by forming a new study that is more patient-friendly than the Mayo study mentioned in the article?
I wonder what it would take for patients affected by this to organize such a study themselves. I imagine that paying for it would be a big hurdle.
It is frightening for patients who are already on vancomycin, and makes it harder for new patients to try it. The AASLD wants to see randomized controlled trial data; there have in fact already been two small such trials, but obviously not enough to satisfy them. I do think a better trial could be designed, and I'm actually trying to do that now. Finding the funding is a challenge because vancomycin is a generic drug. There are entities who would be willing to carry out such a trial if funds can be found.
I wonder if it would be possible for the participants themselves or their families to help pay to fund the study. Maybe not as a pay-to-play, but more like a gofundme: "If we manage to raise this much in donations, then the study can move forward."
Would you be able to say what kind of funding is needed for a study like this? Like, how large a budget is required, and what does it pay for?
My spouse also has PSC w/o IBD, 13 years now since diagnosis. We were given a poor prognosis 5 years ago but Vanco has stabilized her MELD score (indicator for transplant) and has been somewhat stable since. The Mayo Clinic in AZ has been very helpful after many hospitals rejected our desire to use experimental treatment.
NOTE: There are some generic equivalence issues having to do with the coating, make sure you research the manufacturer (ANI seems well tolerated, Alvogen not so much).
I'm happy to hear about your spouse. I actually published a case report that included observations about the importance of brand. One of the capsules tested didn't dissolve until the ph reached 9, clearly not physiologic. ANI, Lupin and Firvanq (liquid) seem to work.
I’m thankful you posted this. As a male with ulcerative colitis, I have a continuous fear that I will get PSC some time in my life.
I wish all the best for your daughter and while I’m not a US citizen, I wish I could support your cause.
Thank you and I wish you the best. There is a lot of exciting research being done on IBD, especially on the gut microbiome. I am optimistic that the coming years will bring significant advances in treatment.
My daughter was diagnosed at age 17 and suffered for 10 years from fatigue, persistent diarrhea, terrible itching, elevated liver enzymes, and abnormal MRI of her bile ducts. We heard from another parent about oral vancomycin, and convinced her doctor to try it. Within a few weeks, she had the first formed stools she had had in 10 years. Her itching resolved, and eventually her liver enzymes returned to normal, her colitis resolved completely, and even her MRCP returned to almost normal. I am personally aware of multiple other such stories. She has ben asymptomatic now for 6 years.
Vancomycin doesn't work for everybody, and doesn't work for most patients who have IBD without PSC.
But to restrict it to clinical trials, as the new AASLD guidance does, when there is no demonstrated harm, is misguided. Insurance companies are already starting to deny coverage. Physicians should be allowed to use their judgment, with consent of the patient.
Please consider signing the petition. If you care, you're a patient advocate.