Dana-Farber Cancer Institute

A new study published in Science led by Dana-Farber/Boston Children's Cancer and Blood Disorders Center (Boston Children's Hospital) researchers introduces Perturb-multiome, a novel genomic screening tool that systematically reveals how transcription factors shape blood cell development. By combining CRISPR-based knockouts with single-cell multiomic profiling, this approach pinpoints which DNA elements drive blood cell production and influence disease risk. The study found that DNA regions occupying just 0.3% of the genome account for a disproportionately large share of genetic influence on blood cell traits. “In our early work, we used genetic association studies to identify BCL11A as a principal switch regulating fetal hemoglobin, a discovery that laid the groundwork for the development of Casgevy as a gene therapy for sickle cell disease and thalassemia. Now, with our new Perturb-multiome approach, we can systematically unearth how thousands of genetic variants influence blood cell production and change disease risk, creating fresh opportunities for improved targeted treatments,” said Vijay Sankaran, MD, PhD. Read more: https://bit.ly/4hYLHH7

Emilie Center, RN, was just days into her nursing career here when she helped save a life — off the clock and outside the hospital. Center was walking with her mother in a 5K walk-run in her hometown when she noticed a group of people off to one side of the course. She rushed over and found a man in cardiac arrest and a bystander performing CPR. Identifying herself as a nurse, Center took over and continued performing CPR even after emergency services personnel arrived. Once the man was breathing on his own, she comforted his wife and two adult daughters as he was readied for transport. "It was my first time having to use CPR on somebody," recalls Center, a clinical research nurse in the Immune Effector Cell (IEC) Therapies Program. "Even though we have very realistic training mannequins, it’s nothing like the rush of adrenaline you get when it’s real." A few weeks later, the runner’s wife called to thank Center and tell her he was doing well. The incident turned Center into a local hero, but what meant the most was hearing that she had inspired others to sign up for CPR classes. Growing up in a large family that included several nurses, Center was first drawn to the profession while helping care for her grandmother. She worked as a clinical nursing assistant in an assisted living facility while earning her nursing degree and gained a special affinity for oncology patients and their resiliency. She began her career at Brigham and Women’s Hospital, eventually joining us to work with cellular therapy patients. Her role involves coordinating care, educating patients about the process and potential side-effects, and monitoring them when they receive their infusions of manipulated cells. "I love it because we can help such a wide and growing range of patient populations as more cancers get approved for cellular therapy," says Center. "It’s an exciting group to be a part of." She didn’t plan on telling her new colleagues about what happened at the 5K, but word got out when someone noticed a newspaper highlighting Center’s heroics. "I was not surprised when I heard that Emilie saw someone in distress and jumped in to help without hesitation," says Maura Dacey, MSN, RN, director of Research Nursing. "Emilie has a keen eye for the details that embody a clinical research nurse, and along with her calm and steady demeanor she was able to put all those assessments skills immediately to use when she saw this man in trouble." Center says it was especially impactful because of what happened a few months earlier – her father had a massive heart attack. She recognized the seriousness of his symptoms and called an ambulance, leading to his successful recovery. Her father is now set to walk her down the aisle in April. After her honeymoon, Center will return to work, hoping for no more off-the-job surprises.

Ellie McLaughlin and Suzy Amor, Patient and Family Advisory Council (PFAC) members joined Yaminah Romulus, Manager of Government Affairs, at the Alliance for Childhood Cancer Action Days in Washington, DC. This two-day event provided parents, children, healthcare professionals, and others from across the country an opportunity to advocate for important childhood cancer issues before Congress. The team met with staff from various offices of the Massachusetts congressional delegation to urge Congress to: 💙 Prioritize the “Childhood Cancer Package” by cosponsoring The Accelerating Kids’ Access to Care Act (H.R. 1509/S. 752), which would help children with cancer as they often require specialized care that may not be available in their home state, and The Give Kids a Chance Act (H.R. 1262), which would address some of the most pressing research needs of children and families with cancer 💙 Protect federal funding for childhood cancer research by opposing any changes to NIH that will harm children with cancer and by fully funding the Childhood Cancer STAR Act and the Childhood Cancer Data Initiative (CCDI) 💙 Oppose any changes to Medicaid that will cut funding, restrict access, or reduce the quality of services for children and their families

Researchers presented new research studies during the 2025 Society of Gynecologic Oncology (SGO) Annual Meeting, held March 14-17. The Dana-Farber led studies exemplified innovative clinical research for the improvement of treatment for gynecologic cancers. Panagiotis Konstantinopoulos, MD, PhD, the Velma Eisenson Chair for Clinical and Translational Research in Gynecologic Oncology, presented findings from the RESOLVE study of a phase 2 clinical trial showing that treatment with metformin, letrozole, and abemaciclib for recurrent estrogen receptor-positive endometrial cancer is safe and appears to induce deeper and more durable responses than letrozole and abemaciclib alone. The team initiated the trial based on preclinical research suggesting synergy between the three drugs, which together inhibit the estrogen receptor, CDK4/6, and PI3K pathways. All 25 patients in the trial received the three medicines. At a median of 17 months of follow-up, three patients had a complete response, five a partial response, and sixteen had stable disease. Based on next-generation sequencing of tumors, the team found that all the complete and partial responses were observed in patients with no specific molecular profile (NSMP) endometrial cancers without RB1 or CCNE1 mutations, suggesting this patient group will derive the most benefit from this combination. "Addition of metformin to hormonal therapy and CDK4/6 inhibition with abemaciclib demonstrated encouraging and durable evidence of activity in NSMP endometrial cancers providing support for simultaneous inhibition of ER, CDK4/6 and PI3K pathways in this setting," said Konstantinopoulos. Elizabeth K. Lee, MD presented results from a phase 1/2 clinical trial reporting that Rinatabart sesutecan (Rina-S), an investigational, novel antibody-drug conjugate directed at folate receptor alpha (FRα), showed encouraging preliminary antitumor activity as a single agent in a dose expansion cohort of patients with advanced ovarian cancer. The open-label, multicenter phase 1/2 study tested Rina-S in a cohort of 42 patients with heavily treated advanced ovarian cancer. Patients received one of two doses of Rina-S (100 mg/m2 or 120 mg/m2) every three weeks. After a median of 24 weeks of follow-up, 22.7% of patients taking the lower dose and 55.6% of patients taking the higher dose had confirmed objective responses, with 2 complete responses in the higher dose group. Side effects included low blood counts and gastrointestinal distress. The results support further testing of single-agent Rina-S at 120mg/m2; enrollment of patients with platinum resistant ovarian cancer is ongoing in a phase 2 study (RAINFOL-OV1) and in a randomized phase 3 study (RAINFOL-OV2/GOG-3107, NCT06619236). "Rina-S, a novel FRa-directed antibody-drug conjugate, demonstrated encouraging activity in patients with ovarian cancer, across FRa expression levels," said Lee. "These findings support the further study of Rina-S in ovarian cancer."

Meet Yuxin Liu, MD, who treats patients with multiple myeloma and other plasma cell disorders. Her clinical research focuses on novel therapies in newly diagnosed and relapsed multiple myeloma. https://bit.ly/41VUyUW

Did you know PFAS, also called "forever chemicals," are linked to cancer? Tim Rebbeck, PhD, explains how PFAS exposure can cause DNA damage and immune system changes, increasing the risk of kidney and testicular cancers. While PFAS exposure often comes from drinking water, the best way to reduce overall cancer risk is by avoiding or quitting smoking, limiting processed foods and alcohol, and maintaining an overall healthy lifestyle where possible.

Last week, we had the pleasure of hosting legislators from the Health Care Financing Committee for a tour of the Yawkey Building on our Longwood campus. Highlights of the tour included Friends’ Place, our pharmacy, clinical labs, treatment rooms, and the healing garden. Following the tour, they met with our President and CEO, Benjamin L. Ebert, MD, PhD, to discuss the evolving healthcare landscape in Massachusetts.   We appreciate the opportunity to connect with many legislators, including Committee’s Cochairs, Senator Cindy Friedman, and Representative John Lawn. Their support and advocacy are essential in driving our mission of advancing expertise and innovation, expanding access to care, and reducing the impact of cancer in the Commonwealth and around the world.

Jorge Castillo, MD, and Shayna Sarosiek, MD, highlight the impact the Bing Center has in advancing novel agents for Waldenström’s Macroglobulinemia. https://bit.ly/3DzTXiq

This weekend, thanks to the support of the Boston Red Sox, Dana-Farber and The Jimmy Fund supporters gathered at Fenway Park to celebrate the impact of the Dana-Farber Campaign. The event highlighted the power of community and the critical work that our donors make possible as we continue to defy cancer together. Attendees engaged with The Message of Hope wall, an interactive display showcasing caring and inspiring messages that will bring hope to patients and their families. Performers and speakers represented the diversity, dedication, and commitment of our community. President and CEO Benjamin L. Ebert, MD, PhD, shared his vision for revolutionizing cancer care and research. Our patients, supporters, and faculty shared stories of impact and hope. Thank you to everyone who attended and all those who continue to partner with us to change the future of cancer care.

Save the date for our 12th Annual Cancer Disparities Research Symposium with keynote speaker Rachel Issaka, MD, MAS. Register here: https://bit.ly/4hBo9IQ