Fibrolamellar Registry

Fibrolamellar Registry
FormationJune 1, 2014 (2014-06-01)
FounderBarbara Lyons, Rachael Migler, Elana Simon, Sanford Simon, Gail Trecosta
Legal status501(c)(3) nonprofit organization
FocusFibrolamellar hepatocellular carcinoma
Dr. Sandy Simon
Websitefibroregistry.org//

The Fibrolamellar Registry is a 501(c)(3) non-profit organization in the United States established to bring together patients with Fibrolamellar carcinoma (FLC) and their families along with scientists and clinicians to achieve the goal of developing a diagnostic test and cure. Since FLC is a rare pediatric liver cancer, the Fibrolamellar Registry helps connect data across institutions and hospitals. The Fibrolamellar Registry does not fundraise for research.

History

The Registry was established in 2014 by Elana Simon along with other FLC survivors as an open-sourced data repository.[1] The Registry is governed by patients and their families. Researchers and clinicians are allowed to use the collected data for free to advance understanding of FLC.

The Registry uses a questionnaire with 600 questions, which go beyond the standard medical record to supply a rich data set for researchers and clinicians to use. The data from the Registry was used to support three research articles published in 2022[2][3][4] and another published in 2023.[5]

In addition to providing data to support new research, the Registry helps patients with FLC understand their disease through plain language summaries of new research papers[6] and tutorials on how to properly search the online biomedical database PubMed.[7]

As of 2024, the Registry has 250 participants from 21 countries which represents over 100,000 data points.[8]

Significance

The Fibrolamellar Registry is one of the first registries run by patients and their families rather than hospitals or universities.[9] As such, the Registry has served as a model for patient-run registries for other rare cancers such as uveal melanoma.[10] The Registry has also connected patients directly with researchers, which has allowed some patients to research their own cancers in the lab.[11][12]

References

  1. ^ Marcus, Gary (2014-02-27). "Open-Sourcing a Treatment for Cancer". The New Yorker. ISSN 0028-792X. Retrieved 2024-09-24.
  2. ^ Shebl, Bassem; Ng, Denise; Lalazar, Gadi; Rosemore, Carly; Finkelstein, Tova M.; Migler, Rachael D.; Zheng, Guangrong; Zhang, Peiyi; Jiang, Caroline S.; Qureshi, Adam; Vaughan, Roger; Yarchoan, Mark; Jong, Ype P. de; Rice, Charles M.; Coffino, Philip (2022-09-08). "Targeting BCL-XL in fibrolamellar hepatocellular carcinoma". JCI Insight. 7 (17). doi:10.1172/jci.insight.161820. ISSN 0021-9738. PMC 9536265. PMID 36073545.
  3. ^ Berkovitz, Amichai; Migler, Rachael D.; Qureshi, Adam; Rosemore, Carly; Torbenson, Michael S.; Vaughan, Roger; Marcotte, Erin; Simon, Sanford M. (December 2022). "Clinical and demographic predictors of survival for fibrolamellar carcinoma patients—A patient community, registry-based study". Hepatology Communications. 6 (12): 3539–3549. doi:10.1002/hep4.2105. ISSN 2471-254X. PMC 9701473. PMID 36245434.
  4. ^ Chen, Krista Y.; Popovic, Aleksandra; Hsiehchen, David; Baretti, Marina; Griffith, Paige; Bista, Ranjan; Baghdadi, Azarakhsh; Kamel, Ihab R.; Simon, Sanford M.; Migler, Rachael D.; Yarchoan, Mark (January 2022). "Clinical Outcomes in Fibrolamellar Hepatocellular Carcinoma Treated with Immune Checkpoint Inhibitors". Cancers. 14 (21): 5347. doi:10.3390/cancers14215347. ISSN 2072-6694. PMC 9655068. PMID 36358766.
  5. ^ Levin, Solomon N.; Tomasini, Michael D.; Knox, James; Shirani, Mahsa; Shebl, Bassem; Requena, David; Clark, Jackson; Heissel, Søren; Alwaseem, Hanan; Surjan, Rodrigo; Lahasky, Ron; Molina, Henrik; Torbenson, Michael S.; Lyons, Barbara; Migler, Rachael D. (2023-06-23). "Disruption of proteome by an oncogenic fusion kinase alters metabolism in fibrolamellar hepatocellular carcinoma". Science Advances. 9 (25): eadg7038. Bibcode:2023SciA....9G7038L. doi:10.1126/sciadv.adg7038. ISSN 2375-2548. PMC 10284549. PMID 37343102.
  6. ^ "Published Papers". The Fibrolamellar Registry. Retrieved 2024-09-24.
  7. ^ "Browse PubMed". The Fibrolamellar Registry. Retrieved 2024-09-24.
  8. ^ "Home". The Fibrolamellar Registry. Retrieved 2024-09-24.
  9. ^ Marcus, Gary (2014-02-27). "Open-Sourcing a Treatment for Cancer". The New Yorker. ISSN 0028-792X. Retrieved 2025-01-19.
  10. ^ "Progress in Rare Cancers". Cancer Today. Retrieved 2025-01-19.
  11. ^ Simon, Sanford M. (May 2023). "Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma". Nature Reviews Cancer. 23 (5): 335–346. doi:10.1038/s41568-023-00554-w. ISSN 1474-1768. PMC 10022574. PMID 36932129.
  12. ^ "Seek - Lab Partners". seek.rockefeller.edu. 11 December 2024. Retrieved 2025-01-19.

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