Fibrolamellar Registry

Last updated
Fibrolamellar Registry
FormationJune 1, 2014;10 years ago (2014-06-01)
FounderBarbara Lyons, Rachael Migler, Elana Simon, Sanford Simon, Gail Trecosta
Legal status 501(c)(3) nonprofit organization
Focus Fibrolamellar hepatocellular carcinoma
Dr. Sandy Simon
Website fibroregistry.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.[ citation needed ] The Fibrolamellar Registry does not fundraise for research.

Contents

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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