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In 2025, medical science advanced in several areas that are already changing practice, not just adding new theoretical models. Instead of general formulations, concrete results from meta-analyses, randomized trials, and initial clinical cases that establish new therapeutic strategies where for decades it seemed everything was already understood.
In this collection, we've assembled precisely such works where fundamental discoveries directly translate to clinical decisions: from early but promising data on CAR-T therapy for solid tumors and the first Russian targeted molecules to meta-analysis of GLP-1 agonist effectiveness in type 1 diabetes, personalized CRISPR editing in infants, and gene therapy development for rare diseases.
These selections share one common denominator: they involve clinical trials and systematic reviews that either demonstrate fundamentally novel mechanisms of action or confirm significant clinical effects in well-characterized cohorts.
CAR-T therapy moves beyond hematology and shows initial results in solid tumors
Each year adds new chapters to the history of oncology discoveries. There's hope that one day this story will conclude with complete victory over cancer thanks to researchers worldwide seeking novel approaches to prevention and treatment. Until then, every breakthrough, every approved treatment, and every survival story becomes another page bringing us closer to this goal. In 2025, CAR-T therapy for solid tumors transitioned from "promising" to "developing" direction, and for the first time demonstrated encouraging results not only in hematologic but also solid tumors.
In the review Emerging advances in CAR-T therapy for solid tumors, authors summarize 12 phase I studies across different tumor sites presented at the American Society of Clinical Oncology (ASCO) Annual Meeting, noting "remarkable progress" in technology clinical development for various tumor locations.
In these small, predominantly non-randomized studies, partial and isolated complete responses were registered: in individual studies, overall response reached approximately 50%, and disease control up to 90%, while in one randomized phase II study of gastric and gastroesophageal junction cancer (the area where the esophagus transitions to the stomach), median survival increased from 5.5 to 7.9 months. Experts emphasize that these are only signals of effectiveness in heavily pretreated cohorts (patients who already underwent multiple lines of standard therapy—surgery, chemotherapy, targeted and other modalities, whose disease continues progressing), not a new treatment standard; however, such data form the foundation for subsequent research phases and potential commercialization of CAR-T approaches in solid oncology.
2025 was also landmark for fundamental tumor immunology: the Nobel Prize in Physiology or Medicine was awarded for elucidating the role of the FOXP3 gene and regulatory T-cells as a key immune system "brake." Understanding how peripheral immune tolerance is regulated enabled formulating the immune checkpoint concept and creating drugs that release these brakes—from CTLA-4 and PD-1/PD-L1 inhibitors to combination regimens with cancer vaccines and cellular therapy that today enhance cancer immunotherapy outcomes, including novel approaches with CAR-T and personalized anti-tumor vaccines.
Russia launches clinical trial of OM-RCA-01—world's first anticancer antibody of new class targeting FGFR1
Russia entered the global oncology development agenda in 2025: the country began clinical trials of the world's first new-class anticancer drug OM-RCA-01 targeting FGFR1 receptor. OM-RCA-01 is a targeted monoclonal antibody-based agent selectively blocking FGFR1, a protein involved in growth and metastasis of numerous tumors. According to the developer, ANO "Cancer Research Bureau," no registered analogs with this mechanism of action exist worldwide; patent protection is already secured in Russia, Europe, and the USA.
The drug is positioned as a potentially "platform" solution for different malignancies where FGFR1's role is established.
The project already received notable professional community recognition: development was awarded ASCO Merit Award by the American Society of Clinical Oncology and European Association of Urology award—for an early-stage Russian molecule, an important validation signal. Against the backdrop of global interest in novel targeted targets and FGFR inhibitors, launching the OM-RCA-01 clinical program became one of Russia's notable 2025 medical achievements, simultaneously strengthening its position on the international oncology research landscape.
Personalized mRNA vaccines against melanoma and NSCLC reduce recurrence risk nearly twofold in randomized treatment
In 2025, cancer vaccine topics ceased being exclusively academic and became part of the actual clinical agenda. In Russia, the N.F. Gamaleya Center completed production of initial test batches of personalized mRNA vaccine and is preparing to treat melanoma and non-small cell lung cancer patients.
Inclusion in the mandatory health insurance system is being discussed, which would make it available free of charge for patients. In parallel, FMBA is advancing two types of anti-tumor vaccines to clinical use—mRNA and peptide—where tumor neoantigen fragments are delivered using nanoparticles and viral vectors.
The international context establishes the expectation benchmark. Moderna's personalized mRNA vaccine mRNA-4157 (V940) combined with pembrolizumab in a randomized study of resected melanoma reduced recurrence or death risk nearly by half and decreased distant metastasis or death risk by 62%. In 2025, three-year data published confirmed effect durability. These results demonstrate personalized vaccination can substantially extend recurrence-free period, but remains technologically complex and expensive.
Today worldwide, over 120 clinical trials of RNA vaccines against cancer are ongoing, and including all cancer vaccine platforms, project and trial numbers are numbered in hundreds.
Why are people actively discussing the imminent availability of anti-cancer vaccines for patients right now? This results from at least three components: accessible sequencing technology revealing abnormal proteins with mutations (neoantigens) in tumors, artificial intelligence capabilities with algorithms for selecting appropriate neoantigens, and preceding years of research. Immunotherapy, which includes cancer vaccines, is the fourth tumor treatment modality after chemotherapy, radiation therapy, and surgery.
GLP-1 drugs: effectiveness confirmed in type 1 diabetes
One of 2025's notable achievements in studying antidiabetic drugs was the emergence of data on GLP-1 receptor agonist use in type 1 diabetes patients with obesity. At the American Diabetes Association's 85th Scientific Session (ADA 2025), a systematic review and meta-analysis of 13 studies was presented, demonstrating that adding GLP-1 drugs to insulin in adults with type 1 diabetes and body mass index ≥25 kg/m² leads to weight reduction averaging 4.5 kg, glycated hemoglobin (HbA1c) decrease of 0.25 percentage points, and daily insulin dose reduction of approximately 7 units.
Semaglutide (0.92-1 mg) and liraglutide (1.8-3 mg) demonstrated the most pronounced effect, with no serious adverse events registered in analyzed studies; principal adverse effects remained typical GLP-1 class gastrointestinal symptoms—nausea and vomiting. Authors conclude that GLP-1 agonists can be considered effective and relatively safe adjuvant therapy in selected type 1 diabetes patients with obesity, though longer and larger studies with more potent next-generation agents are needed. This represents an important step toward revising the traditional type 1 diabetes treatment paradigm based exclusively on insulin use.
Personalized CRISPR therapy first utilized in infants with rare metabolic diseases, avoiding liver transplantation
In 2025, Children's Hospital of Philadelphia and Penn Medicine first conducted personalized in vivo editing (DNA corrected directly inside the organism, not in ex vivo cells "in a test tube" with subsequent genome transplantation) in an infant with severe CPS1 enzyme deficiency disrupting the urea cycle.
Over six months, the team developed and manufactured individualized CRISPR-base-editing therapy packaged in lipid nanoparticles for targeted hepatic delivery, administering three infusions to a child under one year of age.
According to observation data, treatment was tolerated without serious adverse effects: the child better tolerated dietary protein, need for nitrogen-scavenging drugs decreased, and infection episodes no longer accompanied dangerous ammonia elevations—this provided an opportunity to postpone or potentially avoid liver transplantation.
The New England Journal of Medicine (NEJM) authors consider this case the first "proof-of-concept model" that CRISPR therapy can be rapidly adapted to a specific patient's unique mutation, opening the pathway to scalable personalized interventions for rare hereditary diseases.
Domestic Ataluren's emergence demonstrates that gene and targeted therapy for rare hereditary diseases is becoming reality in Russia as well. In parallel, other approaches to rare genetic pathologies are developing worldwide—from viral gene therapy for Duchenne muscular dystrophy to personalized CRISPR genome editing and stem cell technologies capable of not just slowing disease progression but fundamentally altering its trajectory.
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