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Page de couverture de LMC #49 | Dr. Gina D’Amato on Solitary Fibrous Tumor: Building a Registry and Racing Toward Targeted Therapies

LMC #49 | Dr. Gina D’Amato on Solitary Fibrous Tumor: Building a Registry and Racing Toward Targeted Therapies

LMC #49 | Dr. Gina D’Amato on Solitary Fibrous Tumor: Building a Registry and Racing Toward Targeted Therapies

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 SFT is rare—~17,000 sarcomas/year vs. ~300,000 breast cancers—and SFT is only a small slice of that number. Dr. D’Amato traces her path into sarcoma, the unmet need that drew her from lymphoma research, and the mentorship network that helped her build programs at Moffitt, Emory, and now Sylvester.We talk about modeling SFT in the lab (cell lines and engineered mouse models), why NAB2-STAT6 matters, promising signals (e.g., HDAC inhibition) being vetted before a first-in-disease trial, and the new Solitary Fibrous Tumor Patient Registry—a global effort to connect patient stories, molecular profiles, and outcomes so care teams can match treatments to tumor biology.Bottom line: more data → smarter trials → targeted options. Patients and clinicians can help—by enrolling in the registry, sharing pathology reports, and amplifying the work.HighlightsOrigin story: Family experiences with cancer → oncology → pivot to sarcoma to meet a critical gap.Why SFT is hard: 100+ sarcoma subtypes; each one rare within rare. Evidence takes time.What’s new: Building SFT cell lines, NAB2-STAT6 mouse models; early drug-screen “hits” under validation; HDAC inhibitors on the shortlist for a clinical trial.Registry mission: Capture diagnosis journeys, exposures, germline testing, pathology, and treatment response to map variant → site → therapy sensitivity and guide care.Real talk on research: Slow-growing tumors = slow cell-line culture; IRB, protocols, and flyers take time; philanthropy + institutional support bridge to federal grants.Top 3 TakeawaysPrecision beats guesswork. Understanding SFT at molecular and epigenetic levels enables targeted therapy—beyond “try this, then that.”Data is the accelerator. A well-run registry connects the dots between variants (NAB2-STAT6 subtypes), sites of origin, and treatment response.Everyone can help. Patients, families, and clinicians can enroll, refer, and share records to speed breakthroughs.How to Help (Registry + Research Fund)Join or Refer to the SFT Patient Registry (University of Miami – Sylvester)Who can join: Adults (18+) diagnosed with solitary fibrous tumor or hemangiopericytoma, fluent in English, Spanish, or Haitian Creole, willing/able to consent.What it involves: Short surveys about diagnosis/treatment, optional donation of blood/tissue, and sharing pathology reports.Contact: Peggy Gonzalez – 305-243-8091, pgonzalez@miami.edu.Show Notes (2)This registry helps researchers understand causes, biology, and treatment response in SFT—accelerating trials and future targeted options.Support the Horowitz Sarcoma Research Fundhttps://development.miami.edu/page.aspx?pid=383&id=ec01162f-1d17-4c44-89d6-addb185e07b5Funds support SFT research at Sylvester, including lab models, drug screening, and clinical translation.Additional ResourcesSFT Patient Registry – Sylvester (contact): 305-243-8091 | pgonzalez@miami.edu.20241120_D'Amato_Solitary Tumor…Horowitz Sarcoma Research Fund (donate): see link above.Key terms: NAB2-STAT6 fusion, HDAC inhibitors, epigenetics, sarcoma subtypes.About the GuestDr. Gina D’Amato is the Medical Director of the Comprehensive Treatment Unit, Assistant Director of Clinical Research, Sarcoma Medical Oncologist, and Professor of Clinical Medicine at the Sylvester Comprehensive Cancer Center, Leonard M. Miller School of Medicine, University of Miami.She earned her undergraduate degree in biology and her M.D. from the University of Miami, followed by internal medicine residency at Jackson Memorial Hospital. During her fellowship at Moffitt Cancer Center, she pivoted into sarcoma under the mentorship of global experts such as Dr. Jonathan Trent.Dr. D’Amato has since built sarcoma programs at Moffitt, Emory, and Sylvester, where she is Clinical Lead for Sarcoma Medical Oncology. She is extensively involved in Phase I–III clinical trials sponsored by NCI and industry partners, co-directs oncology curriculum at the Miller School, and serves as Medical Director for Sylvester patient education programs. She also administers the Horowitz Solitary Fibrous Tumor Initiative Fund.She has authored or co-authored more than 25 peer-reviewed publications on sarcoma and connective tissue oncology, reviewed for leading journals (Cancer Medicine, Clinical Cancer Research), and received multiple NIH research grants, including a prestigious NIH Career Development Award (2002–2005).Dr. D’Amato is board-certified in Internal Medicine and Medical Oncology by the American Board of Internal Medicine.About the Host:Dr. Peter Crane is a board-certified physician, educator, and storyteller with a heart for service and a calling to spotlight doctors who make a difference—in their communities, in medicine, and in the lives they touch.Through Doctors Making a Difference, he brings you into intimate conversations with physicians who have overcome challenges, redefined success, and found purpose in and beyond the clinic. His goal is simple: to...
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