Announcing a New Fundraising Campaign to Support PSC Patients
Children's PSC Foundation has identified numerous research opportunities that could meaningfully help PSC patients, and we are moving to support as many of those opportunities as possible. An anonymous donor who is aware of our work has offered a fundraising challenge: this donor will match every dollar raised by Children's PSC Foundation through the end of May 2023 up to $250,000!! This is an extremely generous and exciting level of support that could change the outcome for PSC patients. Every dollar you provide will be worth two dollars to the foundation. But we need to move quickly to achieve the full level of donor matching before the end of May 2023.
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.
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.
This strategy has helped us to identify or help create numerous current or potential projects that we are directly supporting or hope to support. These projects include:
A double blind placebo controlled trial of oral vancomycin for PSC
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.
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.
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. Signficant 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.
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.
This trial is currently recruiting patients.
A prospective clinical trial of Hymecromone for PSC
Stanford University, under leadership of co-Investigators Dr. Leina Alribadi 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.
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.
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.
A Pilot Clinical Trial of Microbiota Transplant Therapy (MTT) for PSC
There is a known corrolation 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.
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?"
We are currently in discussions with a major medical institution that has advanced expertise in Microbiota Transplant Therapy (MTT) about a possible clinical trial for PSC patients.
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 a small, relatively quick pilot trial.
More funding is needed to fully support this trial. Your support is critical to explore this new area.
Other areas of interest that the Children’s PSC Foundation is exploring include:
More detailed understanding the microbiome of PSC patients as compared to normal gut microbiota using a new biotech device under development.
The potential for microbiota transplant therapy (MTT) to restore the microbiome of PSC patients, possibly in combination with oral vancomycin therapy
The potential for MTT in combination with oral vancomycin and possibly a passive immune system modulator to resolve PSC.
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 children 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.
100% of funds raised will support Primary Sclerosing Cholangitis (PSC) research. Our foundation is completely volunteer, and our personal expenses in support of our work (travel to conferences, etc.) are paid for by our volunteers, not your contributions. 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.