Turcot Syndrome

New England Journal of Medicine Study Led by Memorial Sloan Kettering Cancer Center Demonstrates That Immunotherapy May Replace Surgery for Certain Solid Tumors LOS ANGELES, April 28, 2025 /PRNewswire/ -- A clinical trial funded by Stand Up To Cancer® (SU2C) and led by Memorial Sloan Kettering Cancer Center (MSK) researchers has revealed that immunotherapy may eliminate the need for standard treatments such as surgery, chemotherapy and radiation in patients with certain types of solid tumors. Continue Reading Maureen Sideris, cancer survivor Published in the New England Journal of Medicine (NEJM) and presented simultaneously at the 2025 American Association for Cancer Research Annual Meeting, the results demonstrate that 80% of trial participants did not require standard treatments after six months of treatment with immunotherapy alone. The trial included people with non-rectal mismatch repair-deficient (MMRd) cancers, including gastroesophageal, hepatobiliary, colon, genitourinary, and gynecologic; these cancers have a genetic defect that makes them harder to repair but also more likely to respond to immunotherapy. This trial is the first time that immunotherapy, which can help the immune system recognize and attack cancer cells, has been shown to replace surgery for a variety of solid tumors. "This study represents a significant shift in treatment for a number of solid tumor cancers," said Julian Adams, Ph.D., president and CEO of Stand Up To Cancer. "Eliminating invasive surgeries that often carry significant risks and lifelong challenges not only saves lives—it sustains the quality of those lives. Stand Up To Cancer is proud to support research that delivers real hope to people impacted by cancer." The trial is led by Luis Diaz Jr., M.D. and Andrea Cercek, M.D.; both are gastrointestinal oncologists at MSK. Diaz is the leader of the SU2C Colorectal Cancer Dream Team and Cercek is a team member. The clinical trial expands on a MSK study, partially funded by SU2C, which utilized an immunotherapy drug and showed that rectal cancer tumors completely disappeared for every trial participant – without the need for standard treatments. From 2017 to 2024, Stand Up To Cancer supported the SU2C Colorectal Cancer Dream Team in creating new strategies to detect and treat colorectal cancer earlier, using insights from genetics, biology, and the immune system – as well as funding more than 10 clinical trials led by the Dream Team. The team's research has focused on unlocking the immune system to fight colorectal cancer, developing targeted therapies for key genetic mutations, and advancing precision prevention strategies to stop cancer before it starts. With SU2C support, the team previously led a pivotal clinical trial that contributed to the first FDA approval of immunotherapy as a front-line treatment for certain metastatic colorectal cancers—helping patients avoid chemotherapy. They later made headlines with their rectal cancer trial—now expanded in the new NEJM study. "Utilizing immunotherapy alone could change how we treat some cancers, helping patients avoid significant side effects and other challenges associated with current surgical approaches, radiation and chemotherapy treatments," said Diaz. "Without Stand Up To Cancer, this research and these results would not have been possible. It's incredibly exciting to think we may be on the verge of a new standard of care for MMRd cancers—one that is less invasive, less toxic, and just as effective." One Survivor's Success Story Maureen Sideris, 71, was diagnosed with gastroesophageal junction cancer in 2022. "My husband Tom and I were preparing for the worst," she recalls. But after joining the trial at Memorial Sloan Kettering and being treated with immunotherapy alone—without surgery, chemotherapy, or radiation—she says, "I felt like I won the lottery!" The experience not only saved Maureen's life—it preserved her quality of life. "I was afraid that if I got surgery on my esophagus, I wouldn't be able to talk for a while, which would be awful. To have immunotherapy alone was just amazing." Maureen is also a colorectal cancer survivor, which she was diagnosed with in 2008. She remembers becoming familiar with SU2C around that time and has supported SU2C's mission as a donor for many years. "It can't be a more noble cause," she says. "So many people are benefitting from cancer research thanks to funding from Stand Up To Cancer and other organizations like it; we couldn't donate our money to a better cause because it's something that is going to help everyone impacted by cancer, now and in the future." About Stand Up To Cancer Stand Up To Cancer® (SU2C) raises awareness and funds research to detect and treat cancers with the aspiration to cure all patients. SU2C is a 501(c)(3) charitable organization and was initially launched as a division of the Entertainment Industry Foundation. Established in 2008 by media and entertainment leaders, SU2C utilizes these communities' resources to engage the public in supporting a new, collaborative model of cancer research, to increase awareness about cancer prevention, and to highlight progress being made in the fight against the disease. As of April 2024, more than 3,100 scientists representing more than 210 institutions are involved in SU2C-funded research projects. As SU2C's scientific partner, the American Association for Cancer Research (AACR) and a Scientific Advisory Committee, led by William G. Nelson, M.D., Ph.D., conduct rigorous competitive review processes to identify the best research proposals to recommend for funding, oversee grants administration, and provide expert review of research progress. Current members of the SU2C Founders and Advisors Committee (FAC) include Katie Couric, Sherry Lansing, Kathleen Lobb, Lisa Paulsen, Rusty Robertson, Sue Schwartz, Pamela Oas Williams, and Ellen Ziffren. The late Laura Ziskin and the late Noreen Fraser are also co-founders. Julian Adams, Ph.D., serves as SU2C's president and CEO. For more information visit StandUpToCancer.org, Instagram, TikTok, Twitter, Facebook, and YouTube. SOURCE Stand Up To Cancer WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

View of Lake Michigan shore and Chicago skyline / iStock, Sean Pavone With the American Association for Cancer Research’s annual conference now underway, oncology researchers can expect potentially practice-changing data in head and neck and lung cancer and key updates on hot modalities. The American Association for Cancer Research’s annual conference is in full swing, with Merck, GSK and Memorial Sloan Kettering Cancer Center presenting new data for oncology cornerstones Keytruda and Jemperli. Meanwhile, AACR CEO Margaret Foti closed the event’s opening ceremony with a rare condemnation of the Trump administration’s science and health policy moves since January, STAT News reported. Stay tuned to BioSpace as we keep you updated on all of the biggest data and news as the conference continues this week. Updated: April 28, 8:00 CST/9:00 a.m. EST GSK’s Jemperli Eliminates Need for Surgery in dMMR Locally Advanced Cancers Phase II results presented Sunday showed that GSK’s PD-1 inhibitor Jemperli can trigger complete tumor clearance in patients with locally advanced, mismatch repair-deficient (dMMR) cancers, regardless of tumor type. These data, published simultaneously in the New England Journal of Medicine , suggest that Jemperli could eliminate the need for surgery in these patients. Sunday’s findings come from 103 patients with stage II-III resectable dMMR cancers: 49 with rectal cancer and 54 with nonrectal solid tumors such as gastroesophageal and genitourinary cancers. After treatment with Jemperli, all 49 patients with rectal cancer showed a clinical complete response and decided to forego surgical operation. Of these, 37 sustained their response levels through 12 months of follow-up. Meanwhile, 35 patients with other cancer types reached clinical complete response, of whom 33 chose non-surgical management. Overall, 84 patients across both cohorts achieved complete response, 82 of whom decided to skip surgery. Boehringer Ingelheim Touts Over 70% NSCLC Response Rate for Zongertinib Boehringer Ingelheim’s investigational HER2 tyrosine kinase blocker zongertinib elicited a 71% objective response rate in certain patients with non-small cell lung cancer (NSCLC) in the Phase III Beamion LUNG-1 trial, according to a Monday readout. The study focused on patients with advanced disease harboring HER2 mutations. Results showed that 7% of patients achieved complete treatment response, indicating no signs of disease. Partial response was 64%, while disease control rate was 96%. Median progression-free survival was 12.4 months. Of note, zongertinib also demonstrated intracranial activity in 27 previously-treated patients with brain metastasis. In this subgroup, the candidate hit a response rate of 41% and a disease control rate of 81%. Zongertinib was also safe overall in Beamion LUNG-1, with no drug-related instances of interstitial lung disease, cardiotoxicity or deaths. Adverse events grade 3 or higher were infrequent, and the most common side effect was diarrhea. Boehringer Ingelheim filed its drug application for zongertinib in February, winning the FDA’s Priority Review designation. A decision is expected in the third quarter.

Based on the Phase 3 CheckMate-8HW trial, Opdivo plus Yervoy demonstrated reduction in the risk of disease progression or death by 79% vs. chemotherapy in the first-line setting and by 38% vs. Opdivo monotherapy across all lines of therapy 1 The approval was granted more than two months ahead of the Prescription Drug User Fee Act goal date PRINCETON, NJ, USA I April 08, 2025 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo ® (nivolumab) plus Yervoy ® (ipilimumab) as a first-line treatment of adult and pediatric patients (12 years and older) with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC). 1 This approval is based on the CheckMate-8HW trial, which is the largest Phase 3 trial (n=839) of immunotherapy in patients with MSI-H/dMMR mCRC, evaluating Opdivo plus Yervoy (n=354) vs. Opdivo monotherapy (n=353) in the all-lines setting and Opdivo plus Yervoy (n=202) vs. investigator’s choice chemotherapy (n=101) (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in the first-line setting. 1 Opdivo plus Yervoy met the dual primary endpoints of progression free survival (PFS) when compared to Opdivo monotherapy across all lines of therapy and when compared to chemotherapy in the first-line setting, as assessed by Blinded Independent Central Review (BICR). 1 This approval, granted more than two months ahead of the June 23, 2025 Prescription Drug User Fee Act goal date, follows the FDA’s prior decision to grant the application Breakthrough Therapy Designation and Priority Review status. “There is an unmet need for additional treatment options such as a dual immunotherapy approach for patients with previously untreated MSI-H/dMMR unresectable or metastatic CRC, which is an aggressive form of cancer and can be particularly difficult to treat,” said Heinz-Josef Lenz, MD, CheckMate-8HW investigator and Deputy Director for Research Programs and Head of the Gastrointestinal Cancers Program at the USC Norris Comprehensive Cancer Center. 2,3,4,5,6 “The meaningful outcomes in CheckMate-8HW underscore how initiating treatment with the dual immunotherapy combination of nivolumab plus ipilimumab may result in a notable survival benefit. 1,5 This approval has the potential to redefine traditional approaches of care for patients with this form of CRC.” In the CheckMate-8HW trial, Opdivo plus Yervoy demonstrated a 38% reduction in the risk of disease progression or death vs. Opdivo monotherapy in immunotherapy-naïve patients across all lines of therapy (Hazard Ratio [HR] 0.62; 95% Confidence Interval [CI] 0.48–0.81; P =0.0003). 1 Assessing the dual primary endpoint of PFS, the trial demonstrated that median PFS was not reached with Opdivo plus Yervoy (95% CI: 53.8-Not Estimable [NE]) and was 39.3 months with Opdivo monotherapy (95% CI: 22.1-NE). 1 PFS rates at 12-, 24-, and 36-months were also numerically higher compared to Opdivo monotherapy (76% vs. 63%, 71% vs. 56%, and 68% vs. 51%, respectively). 1 In Kaplan-Meier (KM) curves showing PFS rates with Opdivo plus Yervoy compared to Opdivo monotherapy, an early separation was observed at two months and sustained at three years. 1 Opdivo plus Yervoy also met a key secondary endpoint, demonstrating superior overall response rate (ORR) by BICR compared to Opdivo monotherapy (n=296, 71% vs. n=286, 58%; P =0.0011). 1 Of the most common all-cause adverse reactions (ARs) occurring in ≥10% of patients, similar rates of grade 3-4 ARs were observed between Opdivo plus Yervoy and Opdivo monotherapy. 1 The safety profile for the dual immunotherapy combination remained consistent with previously reported data and the ARs observed were manageable with established protocols, with no new safety signals identified. 1,5 Additional safety information can be found in the U.S. Full Prescribing Information for Opdivo . The Opdivo plus Yervoy vs. chemotherapy arm of the CheckMate-8HW trial showed that the combination regimen reduced the risk of cancer progression or death by 79% compared to chemotherapy in first-line patients (HR 0.21; 95% CI: 0.14-0.32; P <0.0001). 1 This arm also assessed the other dual primary endpoint of PFS, where median PFS was not reached with Opdivo plus Yervoy (95% CI: 38.4-NE) compared to 5.8 months with chemotherapy (95% CI: 4.4-7.8). PFS rates were numerically higher with Opdivo plus Yervoy vs. chemotherapy at 12- and 24-months (79% vs. 21% and 72% vs. 14%, respectively). 1 KM curves comparing PFS with Opdivo plus Yervoy vs. chemotherapy showed an early separation at three months, which was sustained through two years. 1 The regimen of Opdivo plus Yervoy represents the first-ever dual immune checkpoint inhibitor combination to demonstrate significant efficacy benefit compared to Opdivo monotherapy and chemotherapy in MSI-H/dMMR mCRC patients. 1,5 Opdivo and Yervoy are associated with the following Warnings and Precautions: severe and fatal immune-mediated adverse reactions including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, nephritis with renal dysfunction, dermatologic adverse reactions, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials. 1 Please see the Important Safety Information section below. “This approval marks our ninth indication for an Opdivo -based treatment in the gastrointestinal space. 1 We are witnessing the transformative potential of dual immunotherapy in treating GI cancers,” said Wendy Short Bartie, senior vice president of Oncology Commercialization at Bristol Myers Squibb. 2,3,4,5 “People with MSI-H/dMMR metastatic colorectal cancer face high unmet need, and Opdivo plus Yervoy is an important new approach in the first-line setting. 2,3,4,5 This milestone can offer hope, and it underscores our commitment to continue reaching more patients with new treatment options.” 1 “Colorectal cancer is the third most commonly diagnosed cancer and the second most common cause of cancer-related death for men and women combined in the U.S., and concerning trends show that incidence is increasing in people younger than 50,” said Nicole Sheahan, President of the Global Colon Cancer Association. 6,7 “Despite the prevalence of CRC, there remains a high unmet need, highlighting the urgency for additional treatment options. 2,3,5,6,7 We are thrilled with this FDA approval as Opdivo plus Yervoy offers an exciting new first-line approach for patients with MSI-H/dMMR metastatic colorectal cancer.” 1 Opdivo as a single agent, or in combination with Yervoy , was previously granted accelerated approval in MSI-H/dMMR CRC adult and pediatric patients (12 years and older) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. 1 Today’s FDA decision converts this second-line indication to full approval for Opdivo monotherapy and expands the indication for Opdivo plus Yervoy into the first-line setting based on the CheckMate-8HW trial. 1 About CheckMate-8HW CheckMate-8HW is a Phase 3, randomized, multicenter, open-label trial evaluating Opdivo plus Yervoy compared to Opdivo alone or chemotherapy (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in patients with unresectable MSI-H/dMMR mCRC. 8 In the CheckMate-8HW study, 839 patients were randomized to receive either Opdivo monotherapy ( Opdivo 240 mg Q2W for six doses, followed by Opdivo 480 mg Q4W), Opdivo plus Yervoy ( Opdivo 240 mg plus Yervoy 1 mg/kg Q3W for four doses, followed by Opdivo 480 mg Q4W), or investigator’s choice of chemotherapy. 8 The dual primary endpoints of the trial were PFS for Opdivo plus Yervoy compared to Opdivo alone across all lines of therapy and PFS for Opdivo plus Yervoy compared to chemotherapy in the first-line setting, as assessed by Blinded Independent Central Review (BICR). 8 The study is ongoing to assess various secondary endpoints, including overall survival (OS), and BMS will continue to work with the study investigators to present these data and longer-term follow-up in the future. 8 Select Safety Profile from CheckMate-8HW The safety analysis in CheckMate-8HW included 288 patients, of whom 200 received Opdivo plus Yervoy. 1 Serious adverse reactions occurred in 46% of patients receiving Opdivo plus Yervoy . 1 The most frequent serious adverse reactions reported in ≥1% of patients who received Opdivo plus Yervoy were adrenal insufficiency (2.8%), hypophysitis (2.8%), diarrhea (2.0%), abdominal pain (2.0%), small intestinal obstruction (2.0%), pneumonia (1.7%), acute kidney injury (1.4%), immune mediated enterocolitis (1.4%), pneumonitis (1.4%), colitis (1.1%), large intestinal obstruction (1.1%), and urinary tract infection (1.1%). 1 The most common adverse reactions reported in ≥ 20% of patients treated with Opdivo plus Yervoy were fatigue, diarrhea, pruritis, abdominal pain, musculoskeletal pain, and nausea. 1 Fatal adverse reactions occurred in 2 (0.6%) patients who received Opdivo plus Yervoy ; these included myocarditis and pneumonitis, 1 each. 1 Opdivo and/or Yervoy were discontinued in 19% of patients and were delayed in 48% of patients for an adverse reaction. 1 About Colorectal Cancer Colorectal cancer (CRC) is cancer that develops in the colon or the rectum, which are part of the body’s digestive or gastrointestinal system. 9 With more than 154,000 new cases estimated to be diagnosed in the United States in 2025, CRC is the third most commonly diagnosed cancer. 7,10 Trends show that incidence is increasing in people younger than 50, and mortality rates have increased in people younger than 55 since the mid-2000s. 7 Mismatch repair deficiency (dMMR) occurs when the proteins that repair mismatch errors in DNA replication are missing or non-functional, leading to microsatellite instability-high (MSI-H) tumors. 11,12 Up to 7% of people with mCRC have MSI-H/dMMR tumors and may often have poor outcomes with standard chemotherapy. 5 INDICATIONS OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC). OPDIVO ® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy. OPDIVO ® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT). OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC). OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC). OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. Please see U.S. Full Prescribing Information for OPDIVO and YERVOY . Clinical Trials and Patient Populations CheckMate-649–previously untreated advanced or metastatic gastric cancer, gastroesophageal junction and esophageal adenocarcinoma; CheckMate-577–adjuvant treatment of esophageal or gastroesophageal junction cancer; 8HW: Previously CheckMate-142–MSI-H or dMMR metastatic colorectal cancer in combination with YERVOY; 8HW: Previously CheckMate-142–MSI-H or dMMR metastatic colorectal cancer, as a single agent; Attraction-3–esophageal squamous cell carcinoma; CheckMate-648—previously untreated, unresectable advanced recurrent or metastatic esophageal squamous cell carcinoma in combination with chemotherapy; CheckMate-648—previously untreated, unresectable advanced recurrent or metastatic esophageal squamous cell carcinoma combination with YERVOY; CheckMate-040–hepatocellular carcinoma, in combination with YERVOY. Bristol Myers Squibb: Creating a Better Future for People with Cancer Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine and, through innovative digital platforms, are turning data into insights that sharpen their focus. Deep understanding of causal human biology, cutting-edge capabilities and differentiated research platforms uniquely position the company to approach cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future. About Bristol Myers Squibb’s Patient Access Support Bristol Myers Squibb remains committed to providing assistance so that cancer patients who need our medicines can access them and expedite time to therapy. BMS Access Support ® , the Bristol Myers Squibb patient access and reimbursement program, is designed to help appropriate patients initiate and maintain access to BMS medicines during their treatment journey. BMS Access Support offers benefit investigation, prior authorization assistance, as well as co-pay assistance for eligible, commercially insured patients. More information about our access and reimbursement support can be obtained by calling BMS Access Support at 1-800-861-0048 or by visiting www.bmsaccesssupport.com . About the Bristol Myers Squibb and Ono Pharmaceutical Collaboration In 2011, through a collaboration agreement with Ono Pharmaceutical Co., Bristol Myers Squibb expanded its territorial rights to develop and commercialize Opdivo globally, except in Japan, South Korea and Taiwan, where Ono had retained all rights to the compound at the time. On July 23, 2014, Ono and Bristol Myers Squibb further expanded the companies’ strategic collaboration agreement to jointly develop and commercialize multiple immunotherapies – as single agents and combination regimens – for patients with cancer in Japan, South Korea and Taiwan. About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , X , YouTube , Facebook and Instagram . References SOURCE: Bristol Myers Squibb