The Samuel J. Heyman Service to America Medals®, or the Sammies for short, are known as the “Oscars” of government service and are awarded to outstanding federal employees each year. This year, Dr. Widemann and her team are finalists for their work on selumetinib – the first FDA approved treatment for children with inoperable tumors and neurofibromatosis type 1. Neurofibromatosis is a genetic condition that affects about 1 in 3,000 people and can lead to tumors on the nerves throughout the body and a variety of other health conditions. Several other NIH employees are also finalists. Last year, Dr. Anthony Fauci was a 2020 Sammies award winner. The Sammies are hosted by Partnership for Public Service, a non-profit dedicated to improving the efficiency and efficacy of government for the American public.
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The National Leiomyosarcoma Foundation (NLMSF) and Sarcoma Patients Euronet (SPAEN) recently published a position paper about the status of leiomyosarcoma (LMS) research and clinical trials. LMS is a rare soft tissue tumor that grows from smooth muscle cells and can occur anywhere in the body. LMS is more common in older adults and females, but younger patients do exist. The position paper, entitled “Unmet Medical Needs and Future Perspectives for Leiomyosarcoma Patients—A Position Paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN),” provides a thorough overview of the pathological and clinical features of the two subtypes of leiomyosarcoma: extrauterine LMS and uterine LMS, and discusses issues important to LMS patients.
The paper outlines current treatment options for localized and advanced and/or metastatic disease and highlights that patients should seek care from experts at a sarcoma treatment center. Surgery to remove the tumor and achieve negative margins is the standard procedure. Adjuvant or neoadjuvant therapy (radiation and/or chemotherapy) may be an option for patients at high risk of recurrence and/or when clean borders cannot be achieved with surgery. Because cases of advanced and/or metastatic LMS are challenging to treat, patients’ quality of life and symptom management are often the priority in these cases. The authors highlight that overall LMS patient prognosis is low: in cases of localized LMS, adjuvant therapy requires further study and in cases of advanced and/or metastatic disease, few treatment options exist. The authors also present a landscape of current clinical trials for LMS and discuss recommendations for future LMS clinical research.
Finally, the paper explores the results of a survey conducted by SPAEN in 2019, which collected responses from 265 international sarcoma patients about their thoughts on a range of topics from diagnosis, treatment, quality of life, and more. The authors organize these responses into 12 main topics and discuss their importance and the opportunity for growth for LMS research and patients (see Figure 2 from the paper, below).
NLMSF, one of MyPART’s advocacy partners, has a variety of resources available for patients and researchers. NLMSF hosts three websites:
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Support: provides an overview of available programs for patients and offers patients and researchers the opportunity to connect with the LMS community; supports LMS-specific research efforts
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Education: offers access to workshops, educational symposia, and current LMS research; facilitates collaboration with organizations to amplify patient and family access to resources and increase advocacy
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Engagement: allows LMS community members to exchange stories, discuss the “new normal” of living with LMS, and share emotional support resources
 Figure 2. Future perspectives for the management of patients diagnosed with LMS.
Dr. Karlyne Reilly is the co-leader of MyPART and leads her own research lab in the Pediatric Oncology Branch. Dr. Reilly grew up in Berkeley, California and knew she was interested in science from a young age. Although she moved across the country for college, she returned home for the summers and work at Oakland Children’s Hospital helping with experiments on heart disease and sickle cell anemia. For graduate school, she focused on answering a fundamental question in developmental biology: How does a single cell egg reliably divide to form the front, back, top, and bottom of an animal? After graduate school, she switched her area of research focus to cancer biology. She says, “It still amazes me that evolution has led to ‘signaling pathways’ that get used over and over in biology with such precise outcomes… I think this is what makes cancer research so challenging—many times cancer is reusing a pathway the body is already [using] for another important function.”
At NCI, Dr. Reilly's laboratory is focused on rare nervous system cancers that are associated with the genetic condition neurofibromatosis type 1. Her lab analyzes biospecimens and genetic data from these rare cancers to learn more about these cancers and find potential new treatments. Dr. Reilly also helped establish the Rare Tumors Initiative in the NCI Center for Cancer Research. She explains, “The Rare Tumors Initiative was founded to increase communication and collaboration between clinical and basic researchers, two groups that often approach medical research with different perspectives, terminologies, and paradigms.” She believes that diversity in training and perspectives can drive scientific discovery. She says, “I have always been interested in the idea that people with different training (and thus perspectives) can ‘shake up’ a scientific field, leading to scientific revolutions.”
Through her work with the Rare Tumor Initiative, Dr. Reilly began to work closely with Dr. Brigitte Widemann, Chief of the Pediatric Oncology Branch and co-leader of MyPART. Dr. Reilly explains that this collaboration ultimately led to the development of MyPART: “Dr. Widemann and I developed the idea for MyPART through her expertise in natural history studies and clinical trial development and my expertise in cancer genetics and cancer model development.” As the MyPART team has grown, Dr. Reilly’s role has shifted to focus on the preclinical research activities, including biospecimen analysis and development of rare tumor models, in addition to overseeing MyPART operations with Dr. Widemann and Dr. Abby Sandler.
When asked about her favorite part of MyPART, Dr. Reilly says, “first and foremost, [it’s] the people on our team. Everyone brings different talents and different perspectives, and I firmly believe that MyPART is greater than the sum of the parts.” She also reflects on the generous contributions from patients to cancer research. She explains, “As I work with the biospecimens, I think about the people who donated them to make the research possible and I’m humbled by all that they go through to battle their tumors, for example, when I see multiple surgical specimens from a teenager or young child. The data we collect represents the story of many people’s journey to fight cancer; it is so valuable for our increased understanding of the cancer but requires a sacrifice of time and reliving painful memories from the people telling us their story. Participating in MyPART has [been] one of the most fulfilling experiences in my scientific career thus far.”
Learn more about Dr. Reilly and her research here.
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