Our Strategy to Help PSC Patients
Children's PSC Foundation is focused on pursuing and supporting three core areas of research that will help PSC patients and ultimately lead to a cure:
- Better understanding why oral vancomycin is an effective treatment for some PSC patients. We are interested in exploring and understanding the mechanisms of action of oral vancomycin for PSC patients to essentially "reverse engineer" what oral vancomycin is doing to make patients better. Through this understanding, we believe we can help find ways to identify and better target the underlying cause of PSC.
- Identifying additional or better treatments for PSC to optimize the long term health of PSC patients.
- Exploring new approaches to address PSC that might lead to a cure.
Current Research Projects
Our strategy has helped us to identify or help create numerous current or potential projects that we are directly supporting and pursuing. These projects include:
A Double Blind Placebo Controlled Trial of Oral Vancomycin for PSC
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A team at University of Milano-Bicocca in Milan, Italy, led by Dr. Marco Carbone as Primary Investigator, have developed a protocol for a randomized trial of oral vancomycin for PSC patients throughout Italy.
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It is broadly agreed in the PSC community that a double blind placebo controlled trial is the best way to confirm the effectiveness of OV for PSC. A successful trial is critical to achieving broad support for OV therapy for PSC.
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Additionally, this work will provide insights into the mechanisms of action of OV on the microbiome of PSC patients to help gain a better understanding of what is going on in the microbiome and what OV is doing to make patients better. Significant tissue and serum analysis, as well as liver and biliary tree scans, will be done on the patients who enroll in this trial in order to better understand the mechanisms of impact of oral vancomycin.
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The Children’s PSC Foundation has committed to provide funding for this trial, but more funding is needed to complete the full analysis of the tissue samples that are collected. Advanced genome sequencing and the analysis of those results, which are not yet funded, are critical to fully understand the changes in the microbiome of patients before, during and after treatment.
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This trial is currently recruiting patients.
A Prospective Clinical Trial of Hymecromone for PSC
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Stanford University, under leadership of co-Investigators Dr. Leina Alrabadi and Dr. Aparna Goel, have developed a protocol for a clinical trial of Hymecromone as a potential immune system modulator to help address latent or ongoing inflammation and possible fibrosis in PSC patients. If effective, this could be an additional therapy for reducing long term potential issues for PSC patients.
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The trial will combine Hymecromone with oral vancomycin, the current standard of care therapy for PSC patients at Stanford, to track the impact of hymecromone on inflammation.
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The Children’s PSC Foundation has recently provided funding to support this trial which has just begun recruiting patients. However, if successful, more funding will be needed to support follow-up studies.
Two Separate Pilot Clinical Trials of Microbiota Transplant Therapy (MTT) for PSC
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There is a known correlation between a dysbiosis (imbalance) in the microbiome and PSC. The microbiome of PSC patients is lacking diversity compared to a "normal" microbiome. There are also research papers suggesting that bacteria in the gut may be a contributing cause of PSC. Additionally, oral vancomycin, an antibiotic which is an effective treatment for PSC for some patients, only acts in the GI tract (it is not absorbed into the bloodstream), which is another indicator that bacteria in the gut are a causal component to PSC.
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Fecal Microbiota Transplant (FMT) therapy has been effectively used to treat Clostridium Difficile infections. In this process, a C. Diff. patient's microbiome is replaced or rebalanced by introducing a healthy microbiome into the patient's GI tract. If this therapy is possible and works in C. Diff. patients, this raises the question: "Can microbiota therapy be used to re-balance or replace the microbiome of PSC patients and resolve their PSC?"
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CPSCF is funding two separate pilot trials of Microbiota Transplant Therapy (MTT): one at the University of Minnesota (UM), and one with a company called Biomebank in Adelaide, Australia. MTT is accomplished by encapsulating bacteria from the stool of healthy donors and providing these capsules to patients who are ‘prepped’ to receive these bacteria. MTT is a more patient friendly therapy than Fecal Microbiota Therapy (FMT).
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UM has developed expertise and experience in developing oral MTT capsules using donated microbiota for the successful treatment of C. Difficile infections. Working with UM's MTT capsule experts and UM's PSC clinic, CPSCF is funding a pilot trial of MTT for PSC patients. This trial is in the recruitment phase.
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Biombank has developed a similar, but different, encapsulated oral microbiota transplant therapy, and CPSCF is funding the pilot trial of their therapy in PSC patients. This trial is also in the patient recruitment phase.
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While these two studies are quite similar, they do take slightly different approaches to MTT therapy, and we are hopeful that the combined results will provide excellent insights into MTT as a potential new approach to PSC. This is an exciting new area of exploration toward a potential cure for PSC. It is very early, and the impact is unknown, which is why we are focusing on small, relatively quick pilot trials.
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More funding is needed to fully support these trials. Your support is critical to explore this new area.
Other areas of interest that the Children’s PSC Foundation is exploring include:
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More detailed understanding the microbiome of PSC patients as compared to normal gut microbiota using an innovative new, non-invasive biotech device under development.
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Compiling a database of PSC patients successfully being treated with oral vancomycin along with their histories, evolution of symptoms, and lab test and imaging results over time.
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The potential for MTT in combination with oral vancomycin and possibly a passive immune system modulator to resolve PSC.
NIH Observational Study of PSC in Children
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We are interested in encouraging and recruiting pediatric patients to participate in an observational study of PSC patients supported by the NIH: Click here for more
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The goal of this study is to collect medical and other information about children with PSC to learn more about the disease, how it progresses and to learn more about factors that may impact disease progression.
Research suggests that there are at least two elements at play in PSC: 1) a dysbiosis of the microbiome and 2) an auto-immune system response in the liver. The likely combination of these two elements makes PSC complex, and both elements of PSC need further study.
Our goal is to find a cure for PSC. We believe the best path to achieve this goal is to support focused, targeted research and trials that will provide insights about PSC. Ultimately, these insights will lead to better treatments and a cure.
Your contribution will make a difference in the lives of people living with Primary Sclerosing Cholangitis (PSC). The Children's PSC Foundation is an all volunteer organization, and other than required, minor administration expenses all funds raised by the Children's PSC Foundation go directly to research. Any personal expenses in support of our work (travel to conferences, etc.) are paid for by our volunteers, not your contributions. All of our volunteers are also contributors to the foundation.
Your gift is tax deductible. Children’s PSC Foundation is a non-profit, tax-exempt entity pursuant to Section 501(c)3 Federal Tax ID # 26-4814733.
Past Research To Help PSC Patients
Your past donations to Children’s PSC Foundation have provided funding for numerous clinical studies and research related to PSC and oral vancomycin, including:
Because Stanford has been the pioneer in oral vancomycin for PSC, we have worked closely with the team at Stanford over time. However, in addition to continuing to work closely with and support the teams at Stanford, we continuously look for opportunities to outreach to other areas and other studies related to PSC, including internationally.