2018 Research Workshop Attendees

Dr. Alman is an orthopaedic clinician-scientist, whose research focuses on understanding role of developmentally important processes in pathologic and reparative process involving the musculoskeletal system. The long-term goal of his work is to use this knowledge to identify improved therapeutic approaches to orthopaedic disorders. He makes extensive use of genetically modified mice to model human disease, and has used this approach to identify new drug therapies for musculoskeletal tumors and to improve the repair process in cartilage, skin, and bone. He also works on cellular heterogeneity in sarcomas, and has identified a subpopulation of tumor initiating cells in musculoskeletal tumors. In this work, he also has identified specific cell populations that are responsible for joint and bone development. He has was recently recruited from the University of Toronto to Duke University to chair the department of orthopaedics, which was established in 2010, and includes a large musculoskeletal research component. He has half his time protected for his research work.  Dr. Alman is the Principal Investigator in the DTRF-funded collaborative project, “Collaboration for a Cure: Identifying new therapeutic targets for desmoid tumors.” Profile here.

Mushriq received his bachelor of science at the University of Toronto in Genes, Genetics, and Biotechnology. He is currently a PhD candidate in the Department of Laboratory Medicine & Pathobiology, University of Toronto in Dr. Benjamin Alman lab, studying the role of platelet-derived growth factor signaling and microRNAs in desmoid tumors.

Dr. Aras has been the President & CEO for Beta Cat Pharmaceuticals since 2018. He is an experienced biotechnology executive with an established track record of raising capital for early and mid-stage companies and advancing product through key development milestones. Dr. Aras brings deep international business development experience in all aspects of deal sourcing, negotiation, and strategic alliance management. Prior to Beta Cat, he was the founding CEO for Juventas Therapeutics, a gene therapy company developing novel regenerative therapies for treatment of cardiovascular disease. He led Juventas since its inception in 2007, successfully advancing their lead program from the concept through Phase 2b clinical studies. Prior to leading Juventas, Dr. Aras was the Director of Life Science Commercialization at Cleveland Clinic Innovations where he managed commercialization of all biotechnology and pharmaceutical related technologies developed at Cleveland Clinic. Dr. Aras has held research positions at Vanderbilt University Medical Center and Massachusetts General Hospital and has published articles in several leading scientific journals. He received a B.S. from Tufts University and a Ph.D. in biomedical research from New York University.

Christina Baumgarten started her work for desmoid patients 2005. In 2009 she founded sos-desmoid e.V the german association for desmoid patients and co-founded SPAEN the european association for GIST, desmoid and sarcoma patients. The aim of the european organisation is to extend information services, patient support advocacy so that patients across the whole Europe benefit. SPAEN is acting in partnership with experts, the healthcare industry and other stakeholders. SPAEN will  work to improve treatment and care of GIST, desmoid and sarcoma patients in Europe through improving information and support and by increasing the visibility of sarcoma, desmoid and GIST patients supported by leading european sarcoma, desmoid and GIST experts.

Together with her colleagues from the board of SPAEN and Prof. Kasper and Prof Hohenberger from Germany he initiated a “round table” meeting to create a consensus paper for the management of desmoid tumors based on patients and professionals expertise. The paper was published by Prof Kasper in the EJC January 2015.

Jesse is the Associate Director of the Broad Institute’s Cancer Program and an Institute Scientist at the Broad. He leads a research laboratory focused on developing patient models from rare tumor types and genotypes (Cancer Cell Line Factory; CCLF) to support the Broad’s Dependency Map Initiative and assessing the function of novel cancer mutations as part of the Variant to Function (V2F) initiative. The Boehm Lab has an ultimate goal of making “precision functional genomics” a reality for cancer patients. Jesse received his B.S. in biology from MIT and his Ph.D. from Harvard University, Division of Medical Sciences. Profile here.

Dr. Cunningham received his fellowship training in oncology and hematology at the Harvard Medical School, where he subsequently served on the faculty. He was also one of the founding members of the Division of Experimental Medicine at the Brigham & Women’s Hospital in Boston, where he established a basic research laboratory in Cell and Molecular Biology.

Casey received his medical degree from the University of Texas Southwestern Medical School with a residency in Internal Medicine at the Medical College of Wisconsin. Casey returned to Texas in 1999 as the Associate Director of the Mary Crowley Cancer Research Center in Dallas. The MCCRC performs early phase drug development and Casey was the principal investigator on numerous trials, with a particular emphasis on gene and cell-based therapies. He left this position in 2007 to found a biotechnology company and shortly thereafter also joined Sante Ventures as Chiel Scientific Officer. In addition to performing scientific and technical assessments for the fund, Casey has served in operating roles as Chief Medical Officer of three different portfolio companies, Terapio, Molecular Templates and currently, Iterion Pharmaceuticals (formerly Beta Cat Pharmaceuticals).

Dr. Palma Dileo is Consultant Medical Oncologist on the Sarcoma Unit at University College Hospital, UCLH NHS Trust, specialising exclusively in the management of soft tissue and bone sarcomas. The Sarcoma Unit at UCLH, together with the Sarcoma Unit at the Royal National Orthopaedic Hospital, is the London Sarcoma Service (www.londonsarcoma.org) which offers a comprehensive clinical service to patients of all ages with sarcoma at all sites of the body. She continued to provide patient care since 1996 (general oncology), with more emphasis on sarcoma and rare tumours starting from 1998. She completed her training at the Istituto Nazionale Tumori of Milan, Italy and at Dana-Farber Cancer Institute, Boston, USA. From 2006 to 2010 she has been Associate Physician, Medical Oncology, Istituto Nazionale Tumori, Milan working in the Sarcoma Team. She has a strong interest in early phase clinical trials and is a co-investigator for a number of clinical trials in sarcoma. She is a member of the EORTC Soft Tissue and Bone Sarcoma Group and ASCO. 

Dr. Eldad Elnekave, MD serves as the director of the Clinic for Interventional Oncology at the Davidoff Cancer Institute, Rabin Medical Center, Israel. He obtained his medical degree from Tufts University in Boston, Massachusetts and spent two years as a Howard Hughes Medical Institute Research Scholar at the National Institutes of Health in 2003-2005. Dr. Elnekave completed radiology training at Albert Einstein Medical Center and trained in Vascular and Interventional Radiology at Memorial Sloan-Kettering Cancer Center. His clinical and research focus is on the combination of anatomical and molecular targeting to treat disease in the most precise and minimally invasive method possible. His primary clinical focus is treatment of mesenchymal tumors and sarcoma. Dr. Elnekave also serves as the founding Chief Medical Officer of Zebra Medical Vision, LTD.

Jessica Foley, PhD is the Chief Scientific Officer for the Focused Ultrasound Foundation, whose mission is to accelerate the development and adoption of focused ultrasound, a breakthrough non-invasive medical technology. As Chief Scientific Officer, Foley oversees the Foundation’s scientific and research programs and activities, and aligns these with communications and development efforts. She also leads alliance-building efforts with external stakeholders including (but not limited to) governmental policymakers, regulatory agencies, and disease-specific foundations whose interests are complementary to those of the Foundation. She is a representative and advocate for the Foundation and the focused ultrasound community among a wide variety of stakeholders. Dr. Foley joined the Foundation in 2012 after completing a one-year AAAS Science and Technology Policy Fellowship at the National Science Foundation. Prior to that, she was Neuro Projects Manager and Clinical Marketing Manager at InSightec, one of the leading focused ultrasound medical device manufacturers. Other previous experience included Senior Scientist at Medtronic. She holds a BSE in Biomedical Engineering from Duke University and a PhD in Bioengineering from the University of Washington. Profile here.

Dr. Rebecca Gladdy is a Surgeon Scientist and Assistant Professor at the University of Toronto, who joined the faculty in 2008 after completing a Surgical Oncology Fellowship at Memorial Sloan-Kettering Cancer Center in New York. Dr. Gladdy trained in the General Surgery residency program at the University of Toronto and attained a PhD in cancer genetics in 2003 as member of the Surgical Scientist Training Program. Her independent research program at the Lunenfeld-Tanenbaum Research Institute is focused on developing functional genomics platforms to inform the development of much-needed effective therapies for sarcoma patients. Her clinical expertise is in the surgical management of soft tissue sarcoma. Dr. Gladdy is a member of the Sarcoma Program, which is the Centre of Excellence for Sarcoma Care in Canada and is affiliated with Princess Margaret Cancer Centre, Mount Sinai Hospital (MSH) and the Hospital for Sick Children. In addition to heading a translational research lab, she is the Director of the GI Sarcoma Clinical Research Group at MSH, which is dedicated to advancing clinical care through engaging in clinical trials and improving quality of life for sarcoma patients. Profile here.

Dr. Gounder is a DTRF grant recipient and is the Foundation’s Scientific Director. He is an Assistant Professor and medical oncologist at Memorial Sloan-Kettering Cancer Center specializing in the care of patients with sarcomas of soft tissue and bone and in developing new drugs in all cancers. He has a special clinical and research interest in desmoid tumors and recently showed for the first time that sorafenib is an active drug in desmoid tumors.  Dr. Gounder is the Principal Investigator in a trial partially funded by DTRF studying Nexavar/ Sorafinib in desmoid tumors. Profile here.

Nadia Hindi is a Medical Oncologist focused on sarcoma. She currently works the Sarcoma Unit of Hospital Universitario Virgen del Rocio, in Seville, Spain. This center is a National Reference Center for Sarcoma and also part of the European Reference Network in Adult Cancers (EURACAN). She develops both clinical and traslational research in soft-tissue, bone sarcomas and GIST. She also actively collaborates with Spanish Group for Research on Sarcoma (GEIS) and with sarcoma group of EORTC.

Prof. Peter Hohenberger heads the Division of Surgical Oncology &Thoracic Surgery at the Medical Faculty Mannheim, University of Heidelberg (UMM). He is a boarded surgeon in visceral and vascular surgery as well as in thoracic surgery and surgical intensive care. Professional education was received at the University of Erlangen including training in psychology and pathology. Surgical qualifications were received at the University of Heidelberg including senior position at the Department of Surgery followed by a Professorship on Surgical Oncology at Charité, Berlin. Being now specialized in the treatment of soft tissue tumors he was Chairman of the EORTC Soft Tissue and Bone Sarcoma Group. As a collaborator to EORTC (GI, melanoma and STBSG) since the 1980s he was member of tPRC and Board and also served on the faculty of the AACR/ASCO/FECS/ESMO course on Methods of Clinical Cancer Research. In patient relationship he is member to the advisory board of SPAEN (Sarcoma Patients Euronet), Das LEBENSHAUS and SOS-DESMOID. He chairs the German Interdisciplinary Sarcoma Group (GISG) and the Interdisciplinary Working Party on Sarcoma (IAWS) of the German Cancer Society (DKG). He also leads the Sarcoma Guideline Development group funded by the German Cancer Aid (DKH) the as well as the initiativeto establish a certification system for sarcoma centers in Germany.
The Mannheim sarcoma center is a major contributor to multinational randomized studies on the treatment of sarcoma and GIST, hosts the Data Center of GISG was co-founder of the German Sarcoma Conference. UMM is part of EURACAN for five domains and is leading a working package in Joint Action on Rare Cancer (JARC). Trial activities also included EU-funded EUROSARC and MITIGATE pursuing the treatment of imatinib-resistant GIST. Profile here.

Dr. Hans Iwenofu is an Associate Professor of Pathology and the Chief of Division of Soft Tissue and Bone Pathology, Department of Pathology, The Ohio State University Wexner Medical Center. He earned his MBBS at the University of Nigeria and did his residency in Anatomic and Clinical Pathology at the University of Oklahoma Health Sciences Center, OK. He subsequently completed oncologic surgical pathology and soft tissue and bone pathology fellowships at Roswell Park Cancer Institute, Buffalo, NY and the University of Pennsylvania respectively. Dr. Iwenofu’s research interest is focused on understanding the underpinnings of sarcomagenesis, biomarker discovery and personalized medicine as it applies to soft issue and bone tumors. In a collaborative seminal study titled: “miR-29 acts as a decoy in sarcomas to protect the tumor suppressor A20 mRNA from degradation by HuR” they discovered a unique regulatory circuitry of the decoy function of miR-29 on HuR in the regulation of A20 tumor suppressor activity. This finding uncovers a completely new role of the microRNA-29 besides its tumor suppressor function and provides insightful clues as to why NF-kB is constitutively elevated in different sarcoma subtypes thus maintaining cells in a perpetual primitive state. He has published many papers focused on clinical and translational aspects of soft tissue and bone bones and serves on the editorial boards of Human Pathology, Journal of Surgical Oncology and BMC Cancer. He is a recent recipient of the DTRF grant titled: “Quantitative Proteomic Analysis of Desmoid-Type Fibromatosis”. 

Prof. Bernd Kasper studied Medicine at the University of Heidelberg. In 2001, he finalised his thesis at the German Cancer Research Centre (DKFZ) dealing with new treatment strategies for chronic myelogenous leukaemia patients using the tyrosine kinase inhibitor imatinib. To deepen his training, he stayed in London (Imperial College School of Medicine, Hammersmith Hospital, Department of Haematology) and Brussels (Jules Bordet Institute, Medical Oncology Clinic). In 2007 and 2008, he specialized in Internal Medicine and Medical Haematology/Oncology at the Department of Internal Medicine V at the University of Heidelberg. Currently, he works together with Prof. Peter Hohenberger at the Sarcoma Unit at the Interdisciplinary Tumor Center Mannheim (ITM) at the Mannheim University Medical Center, University of Heidelberg. Since 2011, he is leading the ITM. His special interest lies in the treatment of patients with bone and soft tissue sarcomas including GIST and desmoids. He is head of the study center of the German Interdisciplinary Sarcoma Group (GISG) and Secretary of the Soft Tissue and Bone Sarcoma Group (STBSG) of the European Organisation for Research and Treatment of Cancer (EORTC). Bernd Kasper is actively involved in patient advocacy work on the national as well as international level as a board member of Sarcoma Patients EuroNet (SPAEN). Profile here.

Dr. Krupnick oversees and directs all qualitative research conducted by the patient-centered endpoints strategy team. He is a psychologist with over 20 years of experience as a life sciences research and consulting professional.  Bob specializes in qualitative research design and interview moderation, especially as these relate to patient-reported outcomes development and validation, market opportunity assessment, and brand communications development.  His experience covers a wide range of therapeutic categories in both primary and rare diseases, with particular expertise in psychiatry, neuromuscular disorders, respiratory, renal and metabolic diseases, and men’s health.  Bob holds a B.A. in Psychology from Pennsylvania State University, a M.A. in Human Development & Family Relations from University of Connecticut, and a Ph.D. in Industrial/Organizational Psychology from New York University. In addition to his career in market research, his work experience includes years as a marital and family therapist, alcohol and drug abuse researcher, and human resources consultant.

Alexander Lazar MD/PhD is a practicing academic pathologist at The University of Texas MD Anderson Cancer Center where his clinical, academic and research interests are focused on sarcoma and the genomics of solid tumors. Working within a multidisciplinary team at a high volume treatment center for desmoid tumors, over the last decade he has participated with colleagues on multiple projects involving these tumors. Profile here.

Nicole Leedom is Director of Clinical Operations at SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities.  She joined SpringWorks Therapeutics at launch in 2017 and brings 15 years of clinical operations experience from both biotechnology and CRO companies, with an expertise in clinical trial conduct and execution.  Most recently, Nicole spent 10 years at United Therapeutics as a Senior Clinical Trial Lead where she oversaw all aspects of study start-up, execution, and close-out for adult and pediatric, Phase 2-4 studies in the orphan indication of Pulmonary Hypertension.

Dr. Lefkowitz is a radiologist specializing in abdominal and musculoskeletal imaging and he has a particular interest in imaging of soft tissue tumors. Currently, he is involved in several prospective and retrospective research projects evaluating the treatment response of desmoid tumors to medical therapy and the associated changes seen on MRI. Profile here.

Dr. Maki is a medical oncologist with interests in desmoid tumors, sarcoma clinical trials and translational biology. 

Dr. Kelly Mercier is a DTRF grant recipient, the DTRF Natural History Study and Patient Registry Principal Investigator, a lead clinical trial scientist at bioMerieux, and an adjunct associate professor at Duke University. She specializes in designing and implementing novel diagnostic tools for clinical care. She has received grants to understand the metabolic differences between desmoid tumors and normal adjacent tissue in the same patient with Dr. Ben Alman. Dr. Mercier is passionate about desmoid tumor research and is thrilled to be able to contribute her scientific knowledge to better understand and treat desmoid tumors.

Dr. Meyer serves as the lead medical oncologist for adult sarcoma patients at Johns Hopkins. Dr. Meyer received his bachelors and masters degrees in cell and molecular biology from Stanford University. From there, he joined the Medical Scientist Training Program at Baylor College of Medicine and earned his medical degree followed by a doctorate in immunology. Meyer completed his residency at the University of Maryland and served as chief resident in medicine from 2004-2005. He joined the Hopkins Fellowship program in medical oncology and has been worked in the laboratory of Jonathan Powell, M.D., Ph.D., on projects relating to the characterization of molecular regulatory pathways involved in T-cell activation and energy. As a third-year fellow, Meyer served as the chief fellow for our Training Programs in Hematology and Medical Oncology and he received a Young Investigator Award from the American Society of Clinical Oncology in 2010. His current work focuses on building the Adult Sarcoma and Connective Tissue Oncology Program through the Hopkins sarcoma multidisciplinary clinic. In collaboration with pediatric oncologist Christine Pratalis, M.D., Ph.D., orthopaedic surgeon Carol Morris, MD, and general surgeon Nita Ahuja, MD, Dr. Meyer works to bring novel therapies to the clinic for sarcoma patients.

Dr. Domingo-Musibay is an Assistant Professor and Attending Physician in the Division of Hematology, Oncology and Transplantation. He received his Bachelor of Arts degree from Columbia University in New York and was awarded his Medical Degree from the University of Minnesota Medical School in 2009. He completed Internal Medicine residency training and Hematology/Oncology Fellowship at Mayo Clinic in Rochester, MN as part of the Clinician-Investigator Program. His research focuses on defining optimal immunotherapy sequencing strategies and identifying mechanisms of resistance to standard immunotherapy agents and novel in situ vaccination and adoptive cell therapy based immunotherapeutic treatments.

Dr. Fariba Navid is pediatric oncologist with expertise and research focus in early-phase drug development and pediatric sarcomas and melanoma. She is an Associate Professor at the Keck School of Medicine and Assistant Director of Clinical Trials Office of the Children’s Center for Cancer and Blood Diseases at the Children’s Hospital Los Angeles. Profile here.

Dr. Yoshihiro Nishida is Associate Professor, Chairman, Department of Orthopaedic Surgery, Nagoya University Graduate School and School of Medicine. He has published more than 100 articles on Orthopaedic Oncology and basic research area. His present specialty is surgical and conservative treatment for patients with bone and soft tissue tumors, and doing translational and clinical research. He graduated from Nagoya University School of Medicine, Japan, in 1988. He was selected as a traveling fellow of Japanese Orthopaedic Association—American Orthopaedic association in 2005. He has been a PI of “Study for understanding of current status and established of treatment guideline for patients with extra-peritoneal desmoid tumors” which is selected by Health, Labour, and Welfare Ministry of Japan.

Dr. Okuno is a Professor of Oncology at Mayo Clinic in Rochester Minnesota where he is also the Sarcoma Tumor Chair in the Division of Medical Oncology. In addition, Dr. Okuno is the Medical Director of the Oncology Department for Mayo Clinic Health System for Northwest Wisconsin, and Chair of the Cancer Coordinating Committee for the entire Mayo Clinic Health System. Dr. Okuno’s work focuses on clinical trials for bone and soft tissue sarcoma. He is the Medical Officer for SARC and collaborates with sarcoma researchers in the Midwest Sarcoma Trials Partnership as well as the Sarcoma Working Group of the NRG. Dr. Okuno earned is MD degree at the University of Illinois at Chicago and completed his residency in internal medicine and hematology and oncology fellowship at Mayo Clinic Graduate School of Medicine in Rochester, Minnesota. Profile here.

Dr. Raphael Pollock is Professor and Director of the Division of Surgical Oncology at the Ohio State University Wexner Medical Center, and holds the Kathleen Wellenreiter Klotz Chair in Cancer Research. He also serves at Surgeon in Chief for the James Comprehensive Cancer Center and the Ohio State University Health System. Dr. Pollock’s work focuses on soft tissue sarcoma. His laboratory research activities are examining multiple facets of the molecular drivers underlying soft tissue sarcoma inception focus on soft tissue sarcoma, a rare cancer in adults but rather prevalent in children. He has published widely on sarcoma surgery and treatment, and his funded research includes sarcoma molecular biology and the development of novel therapeutics for this group of diseases. His laboratory work involves the discovery of oncogenes and tumor suppressor genes in soft tissue sarcoma. He is principal investigator of an $11.5 million National Cancer Institute (NCI) grant to support collaborative sarcoma translational research. The NCI Specialized Programs of Research Excellence (SPORE) grant, awarded to the Sarcoma Alliance for Research for Collaboration, represents the largest award ever to study sarcoma. Profile here.

Marlene Portnoy co-founded DTRF because of the frustration she experienced with the lack of information available and the lack of research on this rare disease. Her husband, Steve, was diagnosed in August 2004 with a retroperitoneal desmoid and has since been treated with surgery and radiation.

After graduating magna cum laude from Albany State University in 1981, Marlene pursued a career in sales, which she started at Corning Glass Works from 1982-1985 in Chicago, Illinois. She then moved to New York and worked for Playskool Toys, a division of Hasbro Toys, first as a sales representative and then as a District Sales Manager for the Middle Atlantic territory from 1986-1993. She then took time out to raise her two young children and became active in community service which has included activities such as running various social action programs, participating in the local soup kitchen, and working on golf outings and walkathons for local community interests.

She resides in Suffern, New York with her husband, Steve, and two daughters Brittany and Danielle.

Dr. Przybyl obtained a joint PhD degree from the Catholic University of Leuven, Belgium and Maria Sklodowska-Curie Memorial Cancer Center – Institute of Oncology in Warsaw, Poland. Her doctoral research was focused on new prognostic and predictive markers for selected soft tissue tumors including synovial sarcoma, Ewing sarcoma and endometrial stromal tumors. She also worked in Dr. James E. Darnell Jr. lab at the Rockefeller University (New York, USA) and Dr. Montse Sanchez-Cespedes lab at the Spanish National Cancer Research Center (CNIO) (Madrid, Spain). She is a member of AACR and ASCO.

Dr. Pryzbyl’s research interest involves the development of a comprehensive understanding of molecular mechanisms contributing to soft tissue sarcomas and improvement of the treatment outcomes for patients diagnosed with these tumors. Her academic training and research experience have provided me with expertise in multiple disciplines including molecular biology, biotechnology, and computational biology. Currently she works as a postdoctoral research fellow in Dr. Matt van de Rijn laboratory at Stanford University. My research is focused on the development of novel prognostic/predictive markers and discovery of novel therapeutic targets for selected histological types of sarcomas, including desmoid tumors, using next generation sequencing. Profile here.

Denise has been the President and CEO of SARC since 2002. Denise was a Nurse Practitioner for over 30 years, mostly caring for people with cancer in a variety of settings. She received her diploma in nursing from Milwaukee County School of Nursing and a Bachelor of Science in Nursing from Alverno College in Milwaukee, Wisconsin. She went on to get her Master of Science from University of Michigan where she graduated with honors. Denise also earned her Master of Business Administration from University of Michigan, Ross School of Business.  She has also served as the administrative director of Southwest Oncology Group (SWOG) and is a member of ASCO (American Society of Clinical Oncology), the American Academy of Nurse Practitioners, and CTOS (Connective Tissue Oncology Society). Denise has coauthored numerous publications and been a frequent guest lecturer at major conferences across the country providing information and updates on sarcoma diagnosis and treatment. Profile here.

Dr. Renstrup is a strategic & operationally-focused bio-pharma executive and business leader with oversight of multimillion dollar P&L and a successful track record of transforming regional/global pharma & biotech giants into effective, client-centric organizations with robust medical affairs footprints, accelerated time to market and new drug approvals. His expertise in leading organizations, business development and change management initiatives informs all his work. Dr, Renstrup serves as the Chief Medical Officer at SpringWorks. He joined the company in August 2018 and oversees Clinical Development and Medical Affairs. Prior to joining SpringWorks Dr. Renstrup served as SVP & Global Medical Affairs Head at Alexion (2015-2018) where he helped shape early/late stage development pipeline; upgraded the leadership team; and transformed Medical Affairs into a forward-looking organization with a cross-functional business platform.  Before, Dr. Renstrup was VP & Global Medical Affairs Head at GSK Vaccines (2010-2015). Tasked with enhancing the organization’s structure/culture in collaboration with cross-functional partners, he reshaped the business model of building medical strategy, evidence & engagement programs & helped shape early/late stage development pipeline, supporting rapid drug applications in Europe, the US & emerging markets. Earlier, Dr. Renstrup served in progressive leadership positions at Merck. He started his career at IPSEN Scandinavia A/S. Dr. Renstrup has an MD & Specialist Medical Degree (Anesthesiology & Intensive Care Medicine), University of Copenhagen, and an MBA from the Copenhagen Business School.

Mary Smith joined SpringWorks Therapeutics at launch in 2017 to lead Clinical Research and Drug Development. She brings over 20 years of research and clinical development experience from both pharmaceutical-and biotechnology-focused companies. She has experience in all stages of drug development from target discovery through all phases of clinical development and commercial launch. Most recently, Mary was the executive director of gene therapy at Bamboo Therapeutics, a wholly owned subsidiary of Pfizer where she led several key gene transfer programs for rare genetic diseases. Prior to joining Bamboo, Mary spent 12 years at United Therapeutics as the vice president of product development. In that role, she was head of biologics development in oncology, with additional responsibility for regenerative medicine and virology. Specifically, she led the multi-disciplinary team responsible for the clinical development and approval of Unituxin, the first approved immunotherapy for the treatment of high-risk neuroblastoma in the US and EU. She also managed the regenerative medicine program, which included mesenchymal stem cell therapies and lung regenerative efforts. Mary earned a PhD in microbiology/immunology from the University of New Hampshire followed by post-doctoral training at Emory Unversity.

A medical oncologist, Dr. S. Stacchiotti, works in the Adult mesenchymal and rare tumor medical treatment unit, Cancer Medicine Department, Fondazione IRCCS Istituto Nazionale Tumori (INT), Milano, Italy.
Dr. Stacchiotti clinical and research activities focus on adult soft tissue and bone sarcomas, including gastrointestinal stromal tumors (GIST). She is involved in all institutional research activities on sarcoma, with a special focus on very uncommon subtypes such as chordoma, chondrosarcoma, giant cell tumor of the bone, alveolar soft part sarcoma, clear cell sarcoma, DFSP, desmoid, solitary fibrous tumor, vascular tumor, PVNS, PEComa. She is the Principal Investigator and Coinvestigator of several trials on Sarcoma and GIST. She is a member of the Italian Sarcoma Group, a national cooperative group for clinical and translational research on soft tissue and bone sarcomas, and is a member of the EORTC Soft Tissue & Bone Sarcoma Group. She collaborates to the Italian Network on Rare Tumors, a collaborative effort among Italian cancer centers, which tries to exploit distant patient sharing in order to improve quality of care and diminish health migration for rare solid cancers. She is a member of ESMO (European Society for Medical Oncology), Connective Tissue Oncology Society (CTOS) and of ASCO (America Society of Medical Oncology). She is a member of the advisory board of the Chordoma Foundation and of Desmoid Tumor Research Foundation. She is included in the list of European Medical Agency (EMA) external expert. She serves as associate Editor of the European Journal of Cancer and of Sarcoma Journal.

She has authored more than 100 scientific publications on sarcoma.

Born in 1968, dr Silvia Stacchiotti received his medical degree in 1993 in Milan, and trained at the INT. She is certified in Clinical Oncology.

Mona is Director of Medical Affairs at SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved rare disease patient communities. She joined SpringWorks in May 2018 and is experienced with medical affairs strategy and medical communication activities across several drug and biologic launches. Mona received her PhD in Immunology from Thomas Jefferson University, followed by a Postdoctoral Research Fellowship in Virology at Schering-Plough. She also served as Adjunct Professor with New York City College of Technology.

Dr. Timbergen started medical school in Maastricht, the Netherlands in 2009. After graduation in 2015 she started working as a medical doctor in the surgical ward of the Erasmus MC, Rotterdam. In 2017 she started her PhD with desmoid tumors as a specific interest. Today her research involves a broad spectrum of topics regarding desmoid tumors including pre-clinical experimental work and both retro- and prospective clinical studies.

Dr. Keila Torres (a graduate from Albert Einstein College of Medicine, of Yeshiva University in New York) decided to make a difference in the lives of those affected by cancer after losing a loved one to cancer. As a surgeon at MD Anderson Cancer Center, Dr. Torres specializes in providing surgical care to patients with sarcoma. As a scientist, she also leads a laboratory whose primary goal is to understand how sarcomas grow & how they can be treated more effectively. Her efforts have culminated in several scientific publications, as well as clinical articles & book chapters that have advanced the standard of care for sarcoma patients. Dr. Torres has received numerous awards including the 2011 SARC Career Development Award, the National Cancer Institute Mentored Clinical Scientist Research Career Development Award (2012-2016),& the 2015 American Society or Clinical Investigation Young Physician-Scientist Award. Dr. Torres’ commitment to sarcoma patients does not stop at her professional calling. She serves on the Board of Directors of the Amschwand Sarcoma Cancer Foundation & actively supports the Sally M. Kingsbury Sarcoma Cancer Research Foundation. In fact, much of Keila’s passion & charity work revolves around increasing sarcoma education & awareness. One of her most recent projects was a sarcoma awareness campaign using canine companions as advocates. She is the founder and CEO of Sniffing for the Cure, LLC, an independent publishing company founded with the primary intent to empower people with knowledge, hope and inspiration by incorporating their pets into everyday activity. With sarcoma being the most common form of cancer in dogs, Sniffing for the Cure is committed to increase sarcoma awareness in both canines and humans and advance canine cancer research & related clinical trials that may also offer insight into treatment for human sarcoma patients. Profile here.

Representatives of  Contactgroup Desmoid for Dutch patients.

Dr. Van Mater is a pediatric oncologist at Duke University with an interest in sarcomas, cancer predisposition syndromes, and desmoid tumors. He is interested in expanding clinical trial options for children desmoid tumors. Profile here.

Dr. Weiss graduated from the University of Rochester in 1994 and subsequently earned his medical degree from the Philadelphia College of Osteopathic Medicine in 1999. He completed a pediatric emphasis internship at the Philadelphia College of Osteopathic Medicine/Albert Einstein Medical Center in 2000 followed by a pediatric residency at the AI duPont Hospital for Children in Wilmington, DE in 2003. He then went on to complete a pediatric hematology-oncology fellowship at St. Jude Children’s Research Hospital in Memphis, TN in 2006. He subsequently spent six years as an attending pediatric hematologist-oncologist at the Cancer Institute of New Jersey/University of Medicine and Dentistry of New Jersey and Jersey Shore University Medical Center. In 2012, Dr. Weiss joined the Maine Children’s Cancer Program at Maine Medical Center in Portland, ME. Dr. Weiss has particular interest in pediatric sarcomas. He has co-authored a number of publications on this subject and is currently involved in conducting pediatric clinical trials both locally and nationally in the fields of desmoid tumor and non-rhabdomyosarcoma soft tissue sarcoma. He is the Principal Investigator of the DTFR-funded project: Deregulated mTOR in Desmoid-type Fibromatosis: Identification and Validation of a New Therapeutic Target. Profile here.

Dr. Gerlinde Wernig is an Assistant Professor of Pathology at Stanford University Medical Center. After receiving her residency training in internal medicine (Germany) and pathology (US) specializing in hematology/oncology and hematopathology, she sought postdoctoral training in leukemia research and joined Gary Gilliland’s lab where she was involved in the discovery of the now famous JAKV617F mutation. She then joined Irv Weissman’s laboratory for her second postdoc and also started her pathology residency training at Stanford University. Now, Dr. Wernig has her own lab which focuses onunderstanding the pathomechanisms of end stage organ fibrosis with the ultimate goal to help identify new targets for effective therapies. They have found that the transcription factor c-JUN is specifically activated in human idiopathic pulmonary fibrosis and many other human fibrotic diseases and that induction of c-Jun in mice mimics the pathological hallmarks of these diseases. Profile here.

In 2005, Jeanne Whiting and Marlene Portnoy founded The Desmoid Tumor Research Foundation to facilitate and fund desmoid tumor research and support patients with access to information. Jeanne’s personal journey with a retroperitoneal desmoid tumor involved resection, recurrence, 2 1/2 years of two different chemotherapies and eventual loss of her right kidney. She is keenly aware of the needs of the desmoid community, and is dedicated to accelerating the search for a cure.

Prior to co-founding DTRF, Jeanne spent most of her career as an attorney. After attending University of Michigan Law School and J. Reuben Clark Law School, she received a Juris Doctor degree, magna cum laude, in 1980. She then clerked for the Michigan Court of Appeals in Detroit, Michigan, and in succeeding years practiced with Sidley & Austin in Cairo, Egypt and in Washington, D.C.; with Shearman & Sterling in New York, New York; with Stern, Dubrow & Marcus in Maplewood, NJ; and, as a sole practitioner.

After retiring from the practice of law to raise her family, Jeanne became involved in various nonprofit organizations, including serving as Northeast Director for Choice Humanitarian (1997-1998) and Executive Director of FamilyCares (2000). She feels that getting a desmoid tumor was both a wake-up call and a calling to a new endeavor as it became clear to her as a patient that this was a field that needed jumpstarting into a new level of accelerated research to develop new treatments.

Jeanne resides in Salt Lake City, Utah, with her husband Steve. They are the parents of four sons and a daughter.

Dr. Wilky is medical oncologist specializing in benign and malignant disorders of bone and soft tissue, including sarcomas. In addition to clinical care of patients suffering from these rare cancers, she is a clinical and translational researcher, working to develop new therapies apart from traditional chemotherapy approaches. Dr. Wilky is particularly interested in the role of immunotherapy for sarcomas, and is currently leading three clinical trials at Sylvester Comprehensive Cancer Center investigating immune checkpoint inhibitors and patient-derived vaccines. In addition to studying whether these treatment approaches can help patients with sarcoma, she also works with laboratory investigators to study the immune cells from patients treated on these trials, to understand how to help the immune system fight cancer more effectively. In addition to her research for sarcoma patients, I am an active member of Sylvester’s Phase I Site Disease Group and also participate in Sylvester’s Personalized Medicine Initiatives, which aim to match patients to the best molecularly targeted treatments available based on the patient’s own genetic sequencing results from tumor tissue. Dr. Wilky’s work with the Desmoid Tumor Research Foundation aims to utilize the specific genetic mutations identified in a patient’s desmoid to help predict the most effective and least toxic systemic treatments, including the identification of patients likely to benefit from observation only.

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