This past October, MyPART co-sponsored a workshop to bring rhabdomyosarcoma researchers, clinicians, patients, and advocates together to advance the science and care for rhabdomyosarcoma patients. The workshop was hosted by MyPART advocacy partner, the Summer’s Way Foundation. The November 2023 issue of The MyPART Minute mentioned some of the scientific talks presented at the meeting. In addition to moving research on rare cancers forward, MyPART is keenly aware that rare cancers have a devastating effect on patients and their families, whose stories provide a powerful reminder of why this work is so important. The video, compiled by Summer’s Way Foundation with participation from a coalition of advocacy groups, highlights rhabdomyosarcoma advocates and underscores the importance of collaboration to achieve progress in rare cancer research. The video also pays tribute to people who have lost their lives to rhabdomyosarcoma. Watch this inspiring and motivating video here:
https://drive.google.com/file/d/1FDcoQUGzaNRdSbydn0ZX4O0ty7EAasSJ/view
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On December 7-8th, MyPART co-sponsored a rare tumor research conference focused on SMARCB1 mutated cancers in collaboration with Texas Children’s Hospital, entitled “Bridging the Gap: SMARCB1 Mutated Tumors.” SMARCB1 is a protein important for DNA organization and regulation of gene expression. Tumors with mutations in the gene SMARCB1 tend to be aggressive and affect children and young adults. Patients with these tumors have a poor prognosis with few available treatment options. SMARCB1-mutated tumors include: malignant rhabdoid tumor, atypical teratoid rhabdoid tumor, renal medullary carcinoma, epithelioid sarcoma, chondrosarcoma, sinonasal carcinoma, myoepithelial carcinoma, and specific subtypes of chordoma.
Most SMARCB1-mutated tumor types are very rare, with incidence on the order of 1 in a million people per year, making clinical trials to test new therapies very difficult. With the approval of tazemetostat for epithelioid sarcoma in 2020, the conference focused on treatment advances in different SMARCB1-mutated tumors and whether they might be applied across different tumor types.
The meeting brought together researchers, clinicians, and advocates, and featured short research talks and an advocacy panel. Talks highlighted the latest translational and basic research efforts in SMARCB1 mutated cancers and current clinical trials. MyPART physician Dr. Mary Frances Wedekind presented two talks: the first on the chordoma cohort of MyPART’s Natural History Study of Rare Solid Tumors, and the second on an introduction to the PEPN2121 study: a clinical trial of Atezolizumab with Tiragolumab in SMARCB1/A4-Deficient Tumors.
MyPART co-leader, Dr. Karlyne Reilly, moderated an advocacy panel. The panel kicked off with personal stories from advocates about how their family members experienced SMARCB1 cancers. Advocates who participated in the roundtable were:
Advocates discussed several themes around the research and care of patients with SMARCB1-mutated tumors, including the importance of open dialogue between patients and physicians, the difficulty of collecting samples critical for research while patients are receiving a diagnosis and undergoing treatment, and the need to focus research efforts on preventing recurrence and metastasis in addition to identifying new treatments.
The conference was an important step in bringing together stakeholders in SMARCB1-mutated tumors to advance progress in the understanding and treatment of these tumors.
Learn more about MyPART research workshops here.
 Attendees of the SMARCB1-Mutated Cancers Workshop gather for a group photo. Photo is courtesy of Texas Children's Hospital.
Dr. Kimryn Rathmell officially began her role as the new NCI Director, following Dr. Monica Bertagnolli’s transition from NCI to NIH Director, on Monday December 18th. Dr. Rathmell is a world-renowned kidney cancer expert and has conducted translational research in addition to providing patient care as a medical oncologist. Before joining the NCI, Dr. Rathmell was the Hugh Jackson Morgan Chair in Medicine, chair of the Department of Medicine, and physician-in-chief at Vanderbilt University Medical Center in Nashville, Tennessee. Importantly, Dr. Rathmell is an advocate for rare cancer research, having an interest in rare kidney cancer, including renal medullary carcinoma. Dr. Rathmell was also involved in the creation of the rare kidney cancer advocacy organization, RMC Alliance. MyPART welcomes Dr. Rathmell and her shared interest in rare cancer research!
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