|
The Natural History of Rare Solid Tumors opened for enrollment in January 2019. As of September 2023, the study has enrolled 571 participants from 6 continents, including 46 US states, the District of Columbia, Puerto Rico, and 27 countries outside the US. So far, the study has enrolled participants with nearly 70 different rare cancer histologies. View the graphs below to learn about the age distribution of participants and the histologies represented. Learn more about the study here, including videos about how to participate in the study and infographics describing how we study rare tumors.
MyPART welcomes SMARCB1 Hope, a non-profit organization with the mission to find a cure for SMARCB1-deficient cancers, as a MyPART Network advocacy partner. SMARCB1 is a protein that is important for DNA organization, and loss of this protein in cells has been associated with the development of several types of rare and aggressive cancers, such as malignant rhabdoid tumor and atypical teratoid rhabdoid tumor (ATRT). Collectively, SMARCB1-deficient cancers most frequently affect children and young adults and have a poor prognosis with few treatment options available.
SMARCB1 Hope was founded by Cassie and Jean-Baptiste Toulouse, who lost their 23-year-old daughter Ella to a SMARCB1-deficient sinonasal tumor in 2020. After witnessing the lack of awareness in the scientific and healthcare communities of this family of rare aggressive tumors, Cassie and Jean-Baptiste founded SMARCB1 Hope to raise awareness and accelerate progress to find a cure.
“When a SMARCB1-deficient cancer diagnosis falls on your lap, you don’t understand, or know where to turn. You are dependent on the oncologists you have at hand, whether or not they have heard of SMARCB1-deficiency. Many scientific and healthcare communities are misdiagnosing them, and subjecting patients to brutal yet ineffective treatments. We want to change that, to help connect patients and specialists, to give patients a chance to learn about their SMARCB1-deficient cancer, to point them towards cutting-edge research and relevant clinical trials…” explain Cassie and Jean-Baptiste.
In one of the organization’s first efforts to spark research progress in SMARCB1-deficient cancers, SMARCB1 Hope hosted an international symposium in May 2023, which included over 30 internationally recognized experts. The goal of the symposium was to “help the right people ask the right questions.” Researchers and oncologists spent 3 days engaging in discussions about the latest research on SMARCB1-deficient cancers and potential therapeutic avenues. In addition to catalyzing research collaborations towards finding treatments for SMARCB1-deficient cancers, Cassie and Jean-Baptiste connect with other families experiencing similar cancer diagnoses, share information and resources, and increase awareness about these rare tumors. “These cancers are rare, but if science could understand their mechanisms and find effective treatments, this could open the door to new treatments for a far greater number of cancers,” Cassie and Jean Baptiste say.
MyPART researchers also share an interest in SMARCB1-deficient cancers. MyPART physician, Dr. Mary Frances Wedekind, is the Principal Investigator for a new clinical trial through the Pediatric Early Phase Clinical Trials Network (PEP-CTN). The study, “Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors,” is a Phase I/II trial open to children and adults with relapsed or refractory disease, or newly diagnosed disease for which there are no known effective therapies. Children must be above the age of 1 year old to participate. In addition to the NIH Clinical Center, there are over 25 additional sites in the US and one location in Australia open to recruit for this study. The study will evaluate the safety and anti-tumor activity of two antibodies, tiragolumab and atezolizumab, in the treatment of SMARCB1-deficient tumors and SMARCA4-deficient tumors.
In addition to the clinical trial effort, MyPART and collaborators, including Dr. Rajkumar Venkatramani, will host a SMARCB1-deficient cancer research workshop at Baylor College of Medicine, December 6-8, 2023. MyPART shares the mission of finding treatments for SMARCB1-deficient cancers with SMARCB1 Hope and is excited to partner together towards improving the lives of patients with these rare tumors. Learn more about SMARCB1 Hope here.
|
Allison Dockman joined the MyPART team as a postbaccalaureate fellow after graduating from Haverford College in May 2023. Allison joined MyPART because of her interest in pediatrics and her desire to get more clinical experience before applying to medical school. Her role in MyPART will focus on examining the connection between potential biomarkers and clinical status. She explains, “With the help of other MyPART researchers, I plan to explore whether specific biomarkers are indicative of tumor presence or treatment response in rare pediatric tumor patients.”
When asked about her favorite part of MyPART, Allison says “While I was initially drawn to MyPART due to their incredible work, I knew it was where I wanted to work as soon as I started meeting the MyPART team. I’m very grateful to be working in an environment that is supportive and encouraging, both in terms of my work at NCI and my future goals.” MyPART welcomes Allison to the team!
Learn more about the members of the MyPART team here.
|
Several members of the MyPART team and NCI Center for Cancer Research (CCR) colleagues attended the first TFE/Translocation Renal Cell Carcinoma (TRCC) Symposium, hosted by Dr. Jim Geller from Cincinnati Children's Hospital on September 21-22 in Covington, Kentucky, right across the river from Cincinnati, Ohio! Translocation renal cell carcinoma is a form of rare kidney cancer that is more common in children and young adults and is characterized by changes to the TFE gene. Drs. Brigitte Widemann and Abby Sandler presented on MyPART’s Natural History Study of Rare Solid Tumors and the future directions of a national protocol studying rare tumors through the Childhood Cancer Data Initiative. Kathy Liu, President and Founder of Joey’s Wings, a co-sponsor of the meeting and a MyPART advocacy partner, was in attendance and kicked off the meeting by sharing her family’s experience with TRCC and addressing the needs of the TRCC community. Meeting co-organizers, Dr. Marston Linehan, Chief of CCR’s Urologic Oncology Branch (UOB) and Dr. Ramaprasad Srinivasan, UOB Senior Investigator, both presented talks on their decades of experience studying kidney cancers. This 2-day symposium represented the first time the TRCC research community came together in one room to discuss the latest in TRCC research and standard of care. Clinicians, translational researchers, basic researchers, advocates, and families from the US and Europe gathered to discuss the priorities for advancing TRCC research and patient care.
Earlier in the month, MyPART co-leaders, Drs. Brigitte Widemann and Karlyne Reilly, presented the latest updates from the MyPART team and the Natural History of Rare Solid Tumors to the National Cancer Advisory Board (NCAB) on September 6th. NCAB meetings, which are held 5-6 times throughout the year, convene Presidentially-appointed stakeholders in cancer research and care from industry, academia, and advocacy to provide advice on NCI programs. Learn more about the National Cancer Advisory Board here.
 Researchers, clinicians, advocates, and families gather for the first TFE/Translocation Renal Cell Carcinoma Symposium on September 21-22, 2023 in Covington, KY.
- The North American Neuroendocrine Society (NANETS) held a neuroendocrine tumor (NET) education meeting on the NIH campus in Bethesda, Maryland on September 8th. On September 9th, the advocacy organizations LACNETS and The Pheo Para Alliance co-hosted a patient education meeting in collaboration with NCI. MyPART physician, Jaydira Del Rivero helped to organize both meetings and presented current NCI research on NETs.
- POB Senior Investigator, Dr. Naomi Taylor, along with MyPART’s Drs. John Glod and Abby Sandler participated in Cancer Kids First (Illinois Chapter) Cancer Specialist virtual panel on September 12th. Drs. Taylor, Glod, and Sandler discussed their unique career paths in the field of cancer research with students interested in potentially pursuing a career in cancer research.
- The annual Pediatric and Wildtype GIST Clinic, held in collaboration with MyPART advocacy partner, The Life Raft Group, was held September 20-22nd at the NIH Clinical Center in Bethesda, Maryland. The GIST Clinic is one of MyPART’s rare tumor clinics, which bring patients and families together with expert physicians and researchers from around the country to learn more about rare solid tumors and provide treatment recommendations.
|
