PFIC Scientific Conference: Wrap-Up and Next Steps

Last month the PFIC Network hosted its first ever PFIC Scientific Conference in Pittsburgh, bringing researchers and physicians from around the globe to present their findings as it related to different aspects of PFIC and PFIC related diseases. The conference hosted twelve presentations, five patient stories, and two roundtable discussions, with a total of fifteen different presenters over the two days. We not only had researchers and physicians at the conference, but many of the parents and patients showed up in Pittsburgh to learn, connect, and build lasting relationships. See the photos from the conference at the end of this article!

What did the presentations cover?

The presentations at the conference covered a wide variety of research on PFIC and PFIC related diseases. The start of the conference saw an introduction to the NAPPED consortium and the ChilDReN Network after Dr. Gitta Lubke presented our new patient registry powered by REDCAP. Presentations regarding Bylvay and Maralixibat were a part of the conference to give an update on the safety and efficacy of the drugs, and the effects on bile acids and pruritus. 

The conference presentations closed out with a presentation followed by discussion of Autoimmunity Post Gene Therapy, what gene therapy is, and how it could be applied to PFIC in the future as the technology and knowledge regarding gene therapy progresses. To see a full list of presentations from the PFIC Scientific Conference, go to the Scientific Conference agenda page. Here you will find a list of the presenters and topics covered.

For a breakdown of what was presented at the conference Dr. Jim Squires will give a zoom webinar conference overview presentation scheduled for June 28, 2022. During this call he will give an overview and breakdown of the science presented during the conference. 

Who shared their stories at the conference?

Five parents spoke on the patient journey at the conference. We felt that the patient voice needed to be heard while the people who are researching for a better future were in attendance. The conference opened with a very powerful story from and anonymous caregiver, which set the patient centric tone of the conference. Three of the patient stories were pre-recorded from different countries around the world. Francesca Lombardozzi from PFIC Italia Network, Bernadetta Skora from PFIC Polska, and Zunera Dilshad from Pakistan all shared their family’s story over recording. Two of these stories were told in their native language with a live interpreter present to translate for the audience at the conference. These presentations were a truly powerful addition to the agenda.

What did the roundtable discussions cover? 

We held two roundtable discussions to close out each day of the conference. Day one saw a roundtable that covered “current management pathways”. The roundtables were a place for parents and clinicians/researchers to come together and ask each other important questions, learn from one another, and come up with steps for the vision of what to take on next for clinicians and the PFIC Network. The conversation around treatment pathways led to the discussion about all treatments not being the same, physician 1 doing this while physician 2 would recommend that, and the obscurity behind what treatment is appropriate and when. Overall the discussion posed the question for us “Can we create a resource, a webpage/platform/brochure/etc. that brings together all treatments, best use cases for treatments, and how to standardize these treatments?” 

Day two hosted a roundtable centered around genetic therapy, what it is, and how it could apply to PFIC in the future. The presentation from Dr. Richard Thompson over gene therapy led into a conversation around the possibilities of gene therapy in PFIC patients in the coming years. It is now a real possibility that this can happen, though it needs to happen for other liver diseases first. There are still too many variables and unknowns in changing the genetic makeup of a person and what potential side effects there may be. This roundtable was the conference favorite conversation, rightfully so, based off of our post-conference survey.

Final Remarks

Again, we want to thank everyone who joined us at the conference or supported in any way. A big thank you to Jeff Lord and his family from the DASH Alliance as they hosted the virtual platform and the technical portion of the in-person conference. 

We also want to thank our sponsors for the conference Albireo Pharmaceuticals, Mirum Pharmaceuticals, Sanguine Bio, Cincinnati Childrens Hospital, and Optum Frontier Therapeutics. To learn more about our sponsors, go to our sponsor page here: https://events.pfic.org/scientific-conference-agenda/ 

To register for the conference debrief call mentioned above where Dr. Jim Squires will go over what was presented at the conference for those that were not able to attend, click the link below. 

https://us06web.zoom.us/webinar/register/WN_689h7IeIRa6Uog_vWGQihA

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About the PFIC Network

In September 2018 pfic.org became the PFIC Network, Inc., a 501c3 charitable organization. PFIC Network is run by patients and caregivers with a strong connection to families all over the world. Our mission is to improve the lives of patients and families worldwide affected by Progressive Familial Intrahepatic Cholestasis, PFIC. For more information, please visit us at www.pfic.org.