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Associated Press
2 days ago
- Business
- Associated Press
IMFINZI® (durvalumab) regimen reduced risk of progression, recurrence or death by 29% in early-stage gastric cancer vs. chemotherapy alone in MATTERHORN Phase III trial
WILMINGTON, Del.--(BUSINESS WIRE)--Jun 1, 2025-- Positive results from the MATTERHORN Phase III trial showed perioperative treatment with AstraZeneca's IMFINZI ® (durvalumab) in combination with standard-of-care FLOT (fluorouracil, leucovorin, oxaliplatin, and docetaxel) chemotherapy demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of event-free survival (EFS) versus chemotherapy alone. Patients were treated with neoadjuvant IMFINZI in combination with chemotherapy before surgery, followed by adjuvant IMFINZI in combination with chemotherapy, then IMFINZI monotherapy. The trial evaluated this regimen versus perioperative chemotherapy alone for patients with resectable, early-stage and locally advanced (Stages II, III, IVA) gastric and gastroesophageal junction (GEJ) cancers. These results will be presented today during the Plenary Session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL (abstract #LBA5) and simultaneously published in The New England Journal of Medicine. In a planned interim analysis, patients treated with the IMFINZI-based perioperative regimen showed a 29% reduction in the risk of disease progression, recurrence or death versus chemotherapy alone (based on an EFS hazard ratio [HR] of 0.71; 95% confidence interval [CI] 0.58-0.86; p<0.001). Estimated median EFS was not yet reached for the IMFINZI arm versus 32.8 months for the comparator arm. An estimated 78.2% of patients treated with the IMFINZI-based perioperative regimen were event-free at one year compared to 74.0% in the comparator arm; the estimated 24-month EFS rate was 67.4% versus 58.5%, respectively, signaling a greater magnitude of benefit over time for the IMFINZI-based regimen. For the secondary endpoint of overall survival (OS), a strong trend was observed in favor of the IMFINZI-based perioperative regimen (HR=0.78; 95% CI 0.62-0.97; p=0.025). The trial will continue to follow OS, which will be formally assessed at the final analysis. Yelena Y. Janjigian, MD, Chief Attending Physician of the Gastrointestinal Medical Oncology Service, Memorial Sloan Kettering Cancer Center, New York, and principal investigator for the trial, said: 'Despite receiving curative-intent surgery and chemotherapy, patients with gastric and gastroesophageal cancers frequently develop recurrent disease. Results from the MATTERHORN trial showed that more than two-thirds of patients treated with a durvalumab-based perioperative regimen had not experienced a recurrence or were progression-free after two years. This new treatment approach should become the new standard of care in this setting based on these results.' Cristian Massacesi, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca, said: 'This immunotherapy-based perioperative regimen has the potential to change the clinical paradigm in early gastric and gastroesophageal junction cancers based on the reduction in risk of progression, recurrence or death by nearly a third and the strong trend towards improved survival. As the third positive trial of perioperative treatment with IMFINZI across multiple tumor types, the MATTERHORN trial further validates this approach and highlights our commitment to bringing novel therapies to early stages of disease where there is the greatest chance for cure.' Summary of results: MATTERHORN The safety profile for IMFINZI and FLOT chemotherapy was consistent with the known profiles of each medicine, and the percentage of patients that completed surgery was similar compared to chemotherapy alone. Grade 3 or higher adverse events due to any cause were similar between the two arms. In a previously reported interim analysis for the key secondary endpoint of pathologic complete response (pCR), the IMFINZI - based regimen more than doubled the pCR rate compared to neoadjuvant chemotherapy alone (19% versus 7%, odds ratio 3.08; p<0.001). IMPORTANT SAFETY INFORMATION There are no contraindications for IMFINZI ® (durvalumab) or IMJUDO ® (tremelimumab-actl). Severe and Fatal Immune-Mediated Adverse Reactions Important immune-mediated adverse reactions listed under Warnings and Precautions may not include all possible severe and fatal immune-mediated reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. Immune-mediated adverse reactions can occur at any time after starting treatment or after discontinuation. Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, adrenocorticotropic hormone (ACTH) level, and thyroid function at baseline and before each dose. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate. Withhold or permanently discontinue IMFINZI and IMJUDO depending on severity. See USPI Dosing and Administration for specific details. In general, if IMFINZI and IMJUDO requires interruption or discontinuation, administer systemic corticosteroid therapy (1 mg to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Immune-Mediated Pneumonitis IMFINZI and IMJUDO can cause immune-mediated pneumonitis, which may be fatal. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. Immune-Mediated Colitis IMFINZI with IMJUDO and platinum-based chemotherapy can cause immune-mediated colitis, which may be fatal. IMFINZI and IMJUDO can cause immune-mediated colitis that is frequently associated with diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-Mediated Hepatitis IMFINZI and IMJUDO can cause immune-mediated hepatitis, which may be fatal. Immune-Mediated Endocrinopathies Immune-Mediated Nephritis with Renal Dysfunction IMFINZI and IMJUDO can cause immune-mediated nephritis. Immune-Mediated Dermatology Reactions IMFINZI and IMJUDO can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson Syndrome (SJS), drug rash with eosinophilia and systemic symptoms (DRESS), and toxic epidermal necrolysis (TEN), has occurred with PD-1/L-1 and CTLA-4 blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-exfoliative rashes. Immune-Mediated Pancreatitis IMFINZI in combination with IMJUDO can cause immune-mediated pancreatitis. Immune-mediated pancreatitis occurred in 2.3% (9/388) of patients receiving IMFINZI and IMJUDO, including Grade 4 (0.3%) and Grade 3 (1.5%) adverse reactions. Other Immune-Mediated Adverse Reactions The following clinically significant, immune-mediated adverse reactions occurred at an incidence of less than 1% each in patients who received IMFINZI and IMJUDO or were reported with the use of other immune-checkpoint inhibitors. Infusion-Related Reactions IMFINZI and IMJUDO can cause severe or life-threatening infusion-related reactions. Monitor for signs and symptoms of infusion-related reactions. Interrupt, slow the rate of, or permanently discontinue IMFINZI and IMJUDO based on the severity. See USPI Dosing and Administration for specific details. For Grade 1 or 2 infusion-related reactions, consider using pre-medications with subsequent doses. Complications of Allogeneic HSCT after IMFINZI Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/L-1 blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/L-1 blockade and allogeneic HSCT. Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1/L-1 blocking antibody prior to or after an allogeneic HSCT. Embryo-Fetal Toxicity Based on their mechanism of action and data from animal studies, IMFINZI and IMJUDO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. In females of reproductive potential, verify pregnancy status prior to initiating IMFINZI and IMJUDO and advise them to use effective contraception during treatment with IMFINZI and IMJUDO and for 3 months after the last dose of IMFINZI and IMJUDO. Lactation There is no information regarding the presence of IMFINZI and IMJUDO in human milk; however, because of the potential for serious adverse reactions in breastfed infants from IMFINZI and IMJUDO, advise women not to breastfeed during treatment and for 3 months after the last dose. Adverse Reactions Unresectable Stage III NSCLC Resectable NSCLC Metastatic NSCLC Limited-stage Small Cell Lung Cancer Extensive-stage Small Cell Lung Cancer Locally Advanced or Metastatic Biliary Tract Cancers Unresectable Hepatocellular Carcinoma Primary advanced or Recurrent dMMR Endometrial Cancer Muscle-Invasive Bladder Cancer (MIBC) The safety and effectiveness of IMFINZI and IMJUDO have not been established in pediatric patients. Indications: IMFINZI, as a single agent, is indicated for the treatment of adult patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (cCRT). IMFINZI in combination with platinum-containing chemotherapy as neoadjuvant treatment, followed by IMFINZI continued as a single agent as adjuvant treatment after surgery, is indicated for the treatment of adult patients with resectable (tumors ≥4 cm and/or node positive) NSCLC and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. IMFINZI, in combination with IMJUDO and platinum-based chemotherapy, is indicated for the treatment of adult patients with metastatic NSCLC with no sensitizing EGFR mutations or ALK genomic tumor aberrations. IMFINZI, as a single agent, is indicated for the treatment of adult patients with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (cCRT). IMFINZI, in combination with etoposide and either carboplatin or cisplatin, is indicated for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC). IMFINZI, in combination with gemcitabine and cisplatin, is indicated for the treatment of adult patients with locally advanced or metastatic biliary tract cancer (BTC). IMFINZI in combination with IMJUDO is indicated for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC). IMFINZI in combination with carboplatin and paclitaxel followed by IMFINZI as a single agent is indicated for the treatment of adult patients with primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR) as determined by an FDA-approved test. IMFINZI in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by single-agent IMFINZI as adjuvant treatment following radical cystectomy, is indicated for the treatment of adult patients with muscle-invasive bladder cancer (MIBC). Please see additional Important Safety Information throughout and Full Prescribing Information including Medication Guide for IMFINZI and IMJUDO. You may report side effects related to AstraZeneca products. Notes Gastric and gastroesophageal junction cancers Gastric (stomach) cancer is the fifth most common cancer worldwide and the fifth-highest leading cause of cancer mortality. 1 Nearly one million new patients were diagnosed with gastric cancer in 2022, with approximately 660,000 deaths reported globally. 1 In many regions, its incidence has been increasing in patients younger than 50 years old, along with other gastrointestinal (GI) malignancies. 2 In 2024, there were approximately 43,000 drug-treated patients in the US, European Union (EU) and Japan with early-stage and locally advanced gastric or GEJ cancer. 3 Approximately 62,000 patients in these regions are expected to be newly diagnosed in this setting by 2030. 4 GEJ cancer is a type of gastric cancer that arises from and spans the area where the esophagus connects to the stomach. 5 Disease recurrence is common in patients with resectable gastric cancer despite undergoing surgery with curative intent and treatment with neoadjuvant/adjuvant chemotherapy. Approximately one in four patients with gastric cancer who undergo surgery develop recurrent disease within one year, and one in four patients do not survive beyond two years, reflecting high unmet medical need. 6-7 Additionally, the five-year survival rate remains poor, with less than half of patients alive at five years. 8 MATTERHORN MATTERHORN is a randomized, double-blind, placebo-controlled, multi-center, global Phase III trial evaluating IMFINZI as perioperative treatment for patients with resectable Stage II-IVA gastric and GEJ cancers. Perioperative therapy includes treatment before and after surgery, also known as neoadjuvant/adjuvant therapy. In the trial, 948 patients were randomized to receive a 1500mg fixed dose of IMFINZI plus FLOT chemotherapy or placebo plus FLOT chemotherapy every four weeks for two cycles prior to surgery. This was followed by IMFINZI or placebo every four weeks for up to 12 cycles after surgery (including two cycles of IMFINZI or placebo plus FLOT chemotherapy and 10 additional cycles of IMFINZI or placebo monotherapy). In the MATTERHORN trial, the primary endpoint is EFS, defined as time from randomization until the date of one of the following events (whichever occurred first): RECIST (version 1.1, per blinded independent central review assessment) progression that precludes surgery or requires non-protocol therapy during the neoadjuvant period; RECIST progression/recurrence during the adjuvant period; non-RECIST progression that precludes surgery or requires non-protocol therapy during the neoadjuvant period or discovered during surgery; progression/recurrence confirmed by biopsy post-surgery; or death due to any cause. Key secondary endpoints include pCR rate, defined as the proportion of patients who have no detectable cancer cells in resected tumor tissue following neoadjuvant therapy, and OS. The trial enrolled participants in 176 centers in 20 countries, including in the US, Canada, Europe, South America and Asia. IMFINZI IMFINZI ® (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumor's immune-evading tactics and releasing the inhibition of immune responses. IMFINZI is also approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with IMJUDO ® (tremelimumab-actl) in unresectable hepatocellular carcinoma (HCC). IMFINZI is also approved as a monotherapy in unresectable HCC in Japan and the EU. In addition to its indications in GI cancers, IMFINZI is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, IMFINZI is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of IMJUDO and chemotherapy for the treatment of metastatic NSCLC. IMFINZI is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy for the treatment of extensive-stage SCLC. IMFINZI is approved in the US and other countries as a perioperative treatment in combination with neoadjuvant chemotherapy for muscle-invasive bladder cancer based on the NIAGARA Phase III trial. Additionally, IMFINZI plus standard-of-care Bacillus Calmette-Guérin induction and maintenance therapy demonstrated a statistically significant and clinically meaningful improvement in disease-free survival for patients with high-risk non-muscle-invasive bladder cancer in the POTOMAC Phase III trial. IMFINZI in combination with chemotherapy followed by IMFINZI monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in US and EU). IMFINZI in combination with chemotherapy followed by olaparib and IMFINZI is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in EU and Japan. Since the first approval in May 2017, more than 374,000 patients have been treated with IMFINZI. As part of a broad development program, IMFINZI is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several gastrointestinal cancers. AstraZeneca in GI cancers AstraZeneca has a broad development program for the treatment of GI cancers across several medicines and a variety of tumor types and stages of disease. In 2022, GI cancers collectively represented approximately 5 million new cancer cases leading to approximately 3.3 million deaths. 9 Within this program, the Company is committed to improving outcomes in gastric, liver, biliary tract, esophageal, pancreatic, and colorectal cancers. In addition to its indications in BTC and HCC, IMFINZI is being assessed in combinations, including with IMJUDO, in liver, esophageal and gastric cancers in an extensive development program spanning early to late-stage disease across settings. Fam-trastuzumab deruxtecan-nxki, a HER2-directed antibody drug conjugate, is approved in the US and several other countries for HER2-positive advanced gastric cancer. Fam-trastuzumab deruxtecan-nxki is jointly developed and commercialized by AstraZeneca and Daiichi Sankyo. Olaparib, a first-in-class PARP inhibitor, is approved the US and several other countries for the treatment of BRCA- mutated metastatic pancreatic cancer. Olaparib is developed and commercialized by AstraZeneca and Merck & Co., Inc., known as MSD outside the US and Canada. The Company is also assessing rilvegostomig (AZD2936), a PD-1/TIGIT bispecific antibody, in combination with chemotherapy as an adjuvant therapy in BTC, in combination with bevacizumab with or without IMJUDO as a 1st-line treatment in patients with advanced HCC, and as a 1st-line treatment in patients with HER2-negative, locally advanced unresectable or metastatic gastric and GEJ cancers. Rilvegostomig is also being evaluated in combination with fam-trastuzumab deruxtecan-nxki in previously untreated, HER2-expressing, locally advanced or metastatic BTC. AstraZeneca is advancing multiple modalities that provide complementary mechanisms for targeting Claudin 18.2, a promising therapeutic target in gastric cancer. These include sonesitatug vedotin, a potential first-in-class antibody drug conjugate licensed from KYM Biosciences Inc., currently in Phase III development; AZD5863, a novel Claudin 18.2/CD3 T-cell engager bispecific antibody licensed from Harbour Biomed in Phase I development; and AZD6422, an armored autologous chimeric antigen receptor T-cell (CAR T) therapy, currently being evaluated in an investigator-initiated trial (IIT) in collaboration with AbelZeta in China. In early development, AstraZeneca is developing two Glypican 3 (GPC3) armored CAR Ts in HCC. AZD5851, currently in Phase I development, is being developed globally, and C-CAR031 / AZD7003 is being co-developed with AbelZeta in China where it is under evaluation in an IIT. AstraZeneca in immuno-oncology (IO) AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumor immune response and stimulate the body's immune system to attack tumors. AstraZeneca strives to redefine cancer care and help transform outcomes for patients with IMFINZI as a monotherapy and in combination with IMJUDO as well as other novel immunotherapies and modalities. The Company is also investigating next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer, including cell therapy and T-cell engagers. AstraZeneca is pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. The Company is focused on exploring novel combination approaches to help prevent treatment resistance and drive longer immune responses. With an extensive clinical program, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure. AstraZeneca in oncology AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients. The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyze changes in the practice of medicine and transform the patient experience. AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death. AstraZeneca AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca's innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit and follow the Company on social media @AstraZeneca. References Dr. Janjigian provides consulting and advisory services to AstraZeneca. US-102068 Last Updated 6/25 View source version on CONTACT: Media Inquiries Fiona Cookson +1 212 814 3923 Jillian Gonzales +1 302 885 2677 US Media Mailbox:[email protected] KEYWORD: ILLINOIS DELAWARE UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: RESEARCH FDA CLINICAL TRIALS BIOTECHNOLOGY HEALTH PHARMACEUTICAL GENERAL HEALTH SCIENCE ONCOLOGY SOURCE: AstraZeneca Copyright Business Wire 2025. PUB: 06/01/2025 08:05 AM/DISC: 06/01/2025 08:05 AM
Medical News Today
4 days ago
- Health
- Medical News Today
COVID-19: Who do the new vaccine guideline changes affect?
Two experts answer key questions about the recent COVID-19 vaccine guideline changes. Image credit: MR.. Health Secretary Robert F. Kennedy Jr. recently announced more key changes to the Centers for Disease Control and Prevention (CDC) recommendations for COVID-19 vaccination. Per these changes, the CDC no longer recommends COVID-19 vaccines to pregnant people or healthy children. The only populations for whom the shots are still recommended are older adults and people at high risk of developing severe COVID-19 following infection with SARS-CoV-2, the virus that causes this illness. These changes have caused some concern as a new, more transmissible variant of SARS-CoV-2 has emerged in the United States. On May 27, 2025, the United States Health Secretary Robert F. Kennedy Jr. announced in a social media post that the Centers for Disease Control and Prevention (CDC) would no longer be recommending COVID-19 vaccination to pregnant individuals or healthy children. The announcement came shortly after another statement published by Food and Drug Administration (FDA) officials in The New England Journal of Medicine (NEJM), which suggested that COVID-19 immunization programmes should, going forward, focus on older adults and those at high risk of developing severe disease, should they become infected with SARS-CoV-2, the virus that causes COVID-19. These changes to the official immunization schedule have spurred questions and caused some concern, particularly as a new, more transmissible variant of SARS-CoV-2, called NB.1.8.1, has emerged in the U.S. Medical News Today has spoken to Daniel Ganjian, MD, FAAP, board certified pediatrician at Providence Saint John's Health Center in Santa Monica, CA, and Monica Gandhi, MD, MPH, an infectious disease specialist at the University of California, San Francisco, to answer the most pressing questions. One concern when it comes to the potential impact on pregnant people is that a lack of access to COVID-19 vaccination might actually increase the risk of pregnancy complications. 'If COVID-19 vaccines are no longer recommended for pregnant individuals, pregnant individuals would lose a key intervention that reduces their risk of severe COVID-19, ICU [intensive care unit] admission, preterm birth, and perinatal death,' Ganjian told MNT . In a similar vein, Gandhi emphasized: 'Although I completely agree that the people most in need of booster COVID-19 shots ever year are those who are older (65 years or older) and those [who are] immunocompromise[d] or [have] multiple medical conditions, pregnant women are actually relatively immunocompromised and so are at risk of severe COVID-19.' 'So, I would have preferred that the guidance did not name healthy pregnant women as not needing the shot,' she told us.' Insofar as children are concerned, while kids who are healthy overall may run a lower risk of developing severe COVID-19, infection with the coronavirus that causes it could nevertheless impact their health in unexpected ways. It could, for instance, trigger long-term conditions that may lead to other complications. 'For children, removal of COVID-19 vaccine recommendations would increase the risk of severe COVID-19, hospitalization, and complications such as multisystem inflammatory syndrome in children (MIS-C), particularly among those with underlying medical conditions,' Ganjian said. 'The American Academy of Pediatrics recommends COVID-19 vaccination for all children 6 months of age [and older], as vaccine effectiveness against severe outcomes is well established,' he added. Still, he did note that, even with the current changes to the vaccination recommendations, 'children with immunocompromising conditions or other high-risk comorbidities may still have access to vaccines under specific clinical indications,' although, he cautioned, 'this would depend on regulatory and public health policy decisions.' Gandhi noted that while 'healthy children with previous immunity — either from vaccination or previous infection — do not need COVID-19 vaccinations,' those who have not had a chance to acquire that immunity could now be left exposed to health risks. Thus, 'I would prefer children without previous immunity to get the COVID-19 vaccine,' she told us. 'From a public health perspective, rescinding broad recommendations would likely signal a shift in risk assessment, possibly due to changes in epidemiology, variant severity, or vaccine effectiveness, but would also risk increased morbidity [disease susceptibility] in vulnerable populations,' Ganjian cautioned. Gandhi emphasized that a more discerning approach might prioritise the health and safety of pregnant people by offering them vaccine boosters, while reiterating that children who have already developed some immunity should be O.K. without further vaccination. 'I think pregnant women should get booster shots because they [are] relative[ly] immunocompromise[d]. Healthy children with prior immunity (either from natural infection or the vaccine) do not need COVID-19 boosters if we take a risk-based approach to vaccination.' — Monica Gandhi, MD, MPH On a related note, Gandhi also expressed some concern about the FDA's recent recommendation, also mentioning the NEJM statement from May 2025, to conduct plac ebo-controlled trials for new COVID-19 vaccines. This is because, at this point, participants receiving a placebo instead of a vaccine might be left unnecessarily exposed to severe disease. 'The original COVID-19 vaccine trials (such as with the Moderna or Pfizer vaccines) used a placebo-controlled design because there was no prior COVID-19 vaccine, so using a placebo as a comparison was ethical,' she explained. 'However, we now have COVID-19 vaccines, and it would not be ethical to compare a new COVID-19 vaccine with a placebo in a vulnerable individual (e.g. an older person) when their control could be a prior COVID-19 vaccine formulation,' Gandhi pointed out. 'For instance, I would never allow my 90-year-old father to be in a vaccine trial where he was either getting a new COVID-19 vaccine or a placebo, as being in the placebo arm would leave him unprotected, and he is in the category of individuals who need a yearly booster COVID-19 shot,' she admitted candidly. One question that remains is whether, given the changes to recommended COVID-19 vaccination schedules, pregnant people and parents who may still wish to vaccinate their children might still be able to access the shots privately. 'Based on current knowledge, pregnant individuals or parents who wish to vaccinate their children may still be able to access COVID-19 vaccines through private purchase or off-label use if available, but this would depend on regulatory status and local healthcare provider policies.' 'However, insurance coverage and public health supply may be limited if recommendations are withdrawn,' he also cautioned. Immune System / Vaccines Infectious Diseases / Bacteria / Viruses COVID-19
India Today
16-05-2025
- Health
- India Today
In world's first, doctors customise DNA to treat baby with rare liver disorder
Doctors have successfully used a customised form of gene editing to ease the symptoms of a rare and life-threatening genetic liver disorder in a baby is the first time this type of CRISPR-based technology has been used in a living human with a specific baby, identified as "KJ" by his family, was just 7 months old when he received the experimental treatment in February He was born with a severe condition called carbamoyl phosphate synthetase 1 (CPS1) deficiency, a disorder so rare it affects only one in a million disease is caused by a faulty gene in the liver, leading to dangerous build-ups of ammonia in the blood, which can cause brain damage, coma, or even death. if not managed boy's case was reported in The New England Journal of Medicine (NEJM) and at a gene therapy GENE EDITINGKJ was treated using base editing, a more precise and safer version of the better-known CRISPR gene editing standard CRISPR, which cuts both strands of DNA, base editing changes just a single letter in the DNA sequence, which minimises the risk of any and doctors from the University of Pennsylvania and Children's Hospital of Philadelphia (CHOP) custom-designed the base editor specifically to fix the mutation in KJ's CPS1 this, they developed the entire treatment, from concept to delivery, in just six editing tool was delivered through tiny fat particles called lipid nanoparticles, which were injected into KJ's bloodstream so they could reach his liver AFTER THREE DOSESThough KJ still requires a special diet and medications to help control his condition, early signs suggest gene editing is receiving three doses of the base editor, he can now tolerate more protein in his diet and needs less medicines to control his ammonia more promising, KJ recently recovered from two viral infections without the usual dangerous spikes in ammonia that typically follow in such to the report, doctors did not perform a liver biopsy to confirm the gene correction directly, as it was considered too risky for the his improved condition is "strong indirect evidence" that the treatment has worked at least in father, Kyle Muldoon, said during a press conference, "We're very, very happy with the results."KJ is expected to go home soon, doctors A CURE, BUT A MAJOR STEP FORWARDWhile KJ isn't cured, he may need additional doses in the his cause proves that personalised gene editing for rare genetic diseases is not only possible but can be done in a matter of Kiran Musunuru of Penn Medicine, one of the lead researchers of the case, said, 'Our hope is that this will be the start of something that many others around the world will pick up on.'TAILOR-MADE GENOMEThe success of this personalised base editor adds to a growing list of gene therapy approaches for rare methods include using CRISPR-like tools to insert entire healthy genes into the genome. One such treatment recently helped another baby with a similar urea cycle disorder, allowing that child to stop medication and eat a normal these treatments are still experimental and can carry risks, such as unwanted immune responses or unintended gene edits, the scientists believe they represent the future of medicine for patients with rare and deadly genetic conditions.'This seems to be safe. And there are early signs that it's going to benefit him,' said Dr. Rebecca Ahrens-Nicklas, who helped treat historic step in gene editing could soon change how doctors treat, not just manage, genetic diseases, one patient at a time. advertisement
South China Morning Post
11-05-2025
- Health
- South China Morning Post
Drug trial offers hope for patients with hard-to-treat lung cancer in China, US
A new medication has shown promise as a safe and effective therapy for a hard-to-treat type of lung cancer , according to an international clinical trial led by Chinese and US scientists. Advertisement The findings were published in the New England Journal of Medicine (NEJM) on April 28 and presented at the American Association for Cancer Research Annual Meeting in Chicago on the same day. The researchers found that the oral medication zongertinib outperformed the standard treatment option when treating HER2-mutated non-small cell lung cancer (NSCLC). It works by targeting the HER2 protein and blocking the activity of the protein's tyrosine kinase, which is responsible for signalling cell growth. This type of lung cancer is particularly difficult to treat. Unlike other NSCLC variants, it only has one treatment option that has been approved by the US Food and Drug Administration: intravenous antibody-drug conjugate (ADC) therapy. And ADC-class medications carry risks of adverse effects, including diarrhoea, nausea, fatigue and skin rashes. Wang Xin, professor at the Chinese Academy of Medical Sciences Cancer Hospital, said in an April 29 review of the NEJM study that zongertinib had the 'potential to establish a new benchmark for targeted therapy in HER2-mutant NSCLC'. 10:51 New oral treatment for blood cancer offers 97% cure rate, Hong Kong researchers say New oral treatment for blood cancer offers 97% cure rate, Hong Kong researchers say The multi-cohort study was conducted at 82 sites around the world, including 18 US institutions, 17 in China and three in Japan. The analysis published by the NEJM included 188 NSCLC patients, with Asian populations accounting for a majority.
Forbes
07-05-2025
- Health
- Forbes
Medical Journals Under Scrutiny From Justice Department
Prior Editor of New England Journal of Medicine Dr. Marcia Angell holding papers while standing ... More outside office; journal's logo on glass door in back. (Photo by) Getty Images The interim U.S. attorney for the District of Columbia has been sending letters to a handful of influential medical journals asking questions concerning influence from funders, competing viewpoints and misinformation. Among the journals targeted include the New England Journal of Medicine, CHEST and JAMA. Dr. Eric Rubin, the editor-in-chief of The New England Journal of Medicine, said , 'we were concerned because there were questions that suggested that we may be biased in the research we report.' These threatening letters to multiple medical journals set a dangerous precedent to science and journalism because free speech in medical journals should be protected under the First Amendment. Medical journals, particularly the ones that have been targeted by the federal government, engage in a rigorous peer-review process before articles are accepted for publication. When an article is submitted to a journal, most medical journals utilize double-blinded peer review, meaning the article is sent out typically to two to four medical experts to assess the validity of the manuscript and if publication is warranted. The reviewers have no knowledge of who the authors are, and the authors similarly do not know who their reviewers are. In addition, authors are always asked to acknowledge any funding they have received for the research they are presenting and writing about. In this way, medical journals are constantly and meticulously reducing and eliminating bias in the publication process. Medical journals are critical for the general public because they serve as the foundation for evidence-based medicine. Medical journals play an integral role in shaping healthcare guidelines, vaccine recommendations as well as influencing preventive screening guidelines for important diseases like cancer. They ensure this important information is accessible for the general public to understand and support informed decision-making to empower doctors, patients and news outlets to make informed decisions about health. Journals are responsible for disseminating cutting-edge research that leads to improved diagnostics and potential cures for diseases that could be life-threatening. Finally, the rigorous peer-review from scientific journals ensures that all the medical information reaching the public is accurate and credible. Make no mistake about this- the letters sent to some of the most influential medical journals by the federal government are a threat to science, free speech and public health. These letters are an attempt to influence scientists and potentially silence them, particularly if what they are publishing is contradictory to what the current administration believes about vaccines, diseases and health. The Trump administration as already purged important health information from websites, halted flu vaccination campaigns and curtailed studies about misinformation. Medical journals should be free and safe from any sort of political censorship. These journals do not spread propaganda, they are carefully peer-reviewed and are designed to educate Americans and the public with evidence-based information that help save lives. It is through medical journals we know that vaccines are safe and effective in combating measles, as opposed to other therapies like steroids or cod liver oil. When evidence-based information, particularly with respect to health gets censored, it impacts the health of all Americans. It provides a medium for misinformation to go unchecked and spread instantaneously, faster than any virus given how fast information can spread on social media. Americans are already paying the price on misinformation, given the measles outbreaks throughout America with parents giving their children vitamin A as opposed to vaccines in an attempt to treat and prevent measles. Guidance for health should never be shaped by a particularly ideology, but rather by data and evidence, and this is precisely what medical journals provide. The right to scientific expression is fundamental to democracy, the first amendment and any free society. If the federal government interferes with the content of medical journals, they are not just silencing scientists, they are silencing all Americans. Americans lose access to high quality information that can be life-saving, they lose the ability to make informed decisions on what medications to take and they lose the ability to decide if they need to vaccinate themselves and their families against an infectious disease. If medical journals are silenced; democracy, free speech, health, well-being and our very lives are all on the line.
