A Pre-Sub is a formal mechanism through which companies can request a meeting, written feedback or a teleconference directly with FDA prior to submitting a formal application. It’s a chance to have productive dialogue with the agency before you've committed to a regulatory pathway, before you’ve locked your design, and, hopefully, before you've spent years heading in the wrong direction. Used well, it's essentially a pre-game conversation with the referee. 

Adoptive cell therapy and immune cell engagers represent innovative immunotherapeutic approaches that redirect immune function against cancer cells, extending beyond traditional checkpoint inhibitors. While demonstrating substantial success in hematologic malignancies, their application to solid tumors faces significant challenges including antigen heterogeneity, limited immune cell trafficking, immunosuppressive microenvironments, and potential toxicity. Recent regulatory approvals validate the clinical feasibility and safety of these engineered immunotherapies in solid tumors. This comprehensive review examines antigen selection principles, mechanisms of action, current translation barriers, and strategies for overcoming limitations in solid tumor treatment, providing clinicians with an essential overview of these evolving therapeutic platforms and their future development prospects.

States are advancing significant pharmacy benefit manager and drug pricing legislation in 2026, with Virginia and Ohio leading reform efforts. Virginia's legislature passed the Affordable Medicine Act, establishing a Prescription Drug Affordability Advisory Panel to adopt maximum fair prices for Virginia-regulated health plans, building on federal Inflation Reduction Act standards. The panel will require PBMs to report financial information and mandate state-regulated health plans disclose cost-saving benefits to enrollees. Virginia's governor is expected to sign the legislation, which marks a notable shift following previous gubernatorial vetoes of similar affordability measures. Additionally, Ohio has advanced complementary PBM oversight legislation. These state-level initiatives reflect growing legislative momentum to address prescription drug affordability and enhance transparency in pharmacy benefit management outside the federal regulatory framework.

Tumor organoids represent a transformative advancement in immunotherapy research, offering three-dimensional in vitro models that more accurately recapitulate the complexity of human malignancies compared to traditional two-dimensional cell cultures. These sophisticated systems enable researchers to better understand tumor microenvironment dynamics, immune cell interactions, and patient-specific treatment responses. By preserving tissue architecture and cellular heterogeneity, tumor organoids facilitate personalized medicine approaches and accelerate drug discovery processes. This innovative platform allows investigators to evaluate immunotherapeutic efficacy, predict clinical outcomes, and identify potential resistance mechanisms in a controlled laboratory setting. Consequently, tumor organoids hold significant promise for optimizing cancer immunotherapy strategies and improving patient therapeutic outcomes.

Autologous chimeric antigen receptor (CAR) T-cell therapy has revolutionized treatment for advanced blood cancers, enabling remission in patients who failed previous therapies, with some achieving long-term cancer-free status. Despite their clinical success, CAR T-cell immunotherapies face significant limitations that require further investigation. The article explores the next generation of developments in this field, addressing current challenges and investigating strategies to enhance therapeutic efficacy and overcome existing disadvantages. These advancements are critical for expanding CAR T-cell applications and improving outcomes in precision oncology.

In vivo CAR T-cell engineering represents a transformative shift in cellular immunotherapy, moving beyond traditional ex vivo manufacturing constraints. While CAR T-cell therapy has successfully reshaped treatment for hematologic malignancies, current approaches face significant limitations including extended production timelines, logistical complexity, and variable product quality across patients. Emerging in vivo strategies address these challenges by engineering immune cells directly within patients through targeted genetic delivery, eliminating the need for external cell extraction and modification. This paradigm reframes cellular therapy from a static manufactured product into a programmable biological system, potentially improving scalability, accessibility, and therapeutic consistency while accelerating treatment delivery for aggressive malignancies.

Des chercheurs de l’université du Mississippi ont mis au point de minuscules capsules contenant un agent anticancéreux, intégrées à un petit implant fabriqué par impression 3D. Le principe : placer cet implant directement à côté de la tumeur, pour que le médicament agisse là où il le faut, sans se disperser dans tout le corps. Pour l’instant, les tests se limitent à des cultures de cellules cancéreuses du sein, en laboratoire.

Introduits par voie naturelle, ces bâtonnets permettent une irradiation ciblée au plus près des cellules cancéreuses. "L’avantage de l’endoscopie, c’est qu’on est très près de la tumeur. Dans le tube digestif, on est contre le pancréas. Et donc, de façon très précise, on peut mettre ces sources qu’il faut rapprocher les unes des autres à peu près tous les 3 à 4 millimètres, de façon à traiter l’ensemble de la tumeur", explique Pierre-Yves Eyraud, médecin en hépato-gastroentérologie et oncologie digestive au CHU de Grenoble Alpes.

A woman with three autoimmune conditions improved dramatically after personalized CAR T-cell therapy. Misryoum explains how the approach resets harmful antibody production—and what challenges remain.A single treatment can sound like a miracle in medicine, but for one woman with multiple autoimmune diseases, the turnaround has been stark—and carefully engineered.

A l'ouverture du groupe des assises du médicament consacré au dispositif médical, j'osais évoquer la possibilité que la prochaine affaire Mediator puisse impliquer un dispositif médical (DM). Je ne m'attarderai pas sur les fermes dénégations qui m'avaient alors été opposées tant par les représentants des industriels que par ceux de l'Afssaps. Le marquage CE allié à la matério-vigilance étaient là pour protéger nos patients… nous pouvions être tranquilles…

Companies and researchers often fail to disclose negative trial results, resulting in significant gaps in the public record and a publication bias that obscures the true landscape of drug development outcomes—overrepresenting successes and underrepresenting failures. This gap can also create a distorted perception of the safety and efficacy of medical products.

Intervertebral disc degeneration (IDD) is a major cause of low back pain (LBP), significantly affecting on global disability and healthcare costs. Traditional treatments primarily focus on symptom management rather than addressing the underlying causes, such as the decline in nucleus pulposus (NP) cells and reduced extracellular matrix (ECM) synthesis. Cell therapy shows promise by replenishing NP cells, activating resident cells, and enhancing ECM deposition. However, the harsh microenvironment of IDD can hinder the survival of transplanted cells. Biocompatible hydrogels, with their ECM-like properties, provide controlled delivery of therapeutics and biologics, creating a supportive environment for cell therapy and demonstrating strong regenerative potential.

Désormais, l’accès aux soins virtuels se transforme avec le lancement par Amazon de son assistant intelligent, baptisé Health AI. Disponible sur le site et l’application du géant du e-commerce, cet agent conversationnel s’adresse en priorité aux membres Prime américains qui bénéficient d’un accès gratuit et permanent à des services de santé en ligne. Un projet nourri à la fois par les frustrations courantes du système médical, délais interminables, manque de personnalisation, paperasse et par la volonté d’allier expertise clinique (One Medical) et technologies de pointe. L’objectif affiché est clair : simplifier un parcours devenu trop complexe pour des millions d’Américains.

GLP-1 receptor agonists — sold under brand names like Ozempic and Mounjaro — were initially developed to treat diabetes. But instead of addressing biomarkers linked to certain disease outcomes, these drugs influence the central cardio-kidney metabolic process, Vaduganathan said. This overarching approach has made GLP-1 drugs the most effective and tolerable choice for most patients treating diabetes and obesity. It’s also what makes it likely that they influence a number of closely related diseases.

New findings on cancer survival rates offer hope for the more than 2 million Americans diagnosed each year. Seven out of 10 Americans diagnosed with cancer now survive five years or more, according to the American Cancer Society, a 7 percent increase since the mid-1990s, when the rate stood at 63 percent.The survival rate data — from patients diagnosed with cancer between 2015 and 2021 — showed, significantly, that those with high-mortality cancers and advanced diagnoses had the largest gains. Myeloma survival rates, for example, rose from 32 percent to 62 percent, and liver cancer survival rates from 7 percent to 22 percent.

Agentic artificial intelligence (AI) gains popularity swiftly. This technology refers to AI systems designed to act autonomously towards specific goals. Agentic AI can plan, decide, take actions, and use tools without constant human prompting. Its goal-oriented behavior and decision-making capabilities make it valuable in different industries, involving healthcare. In this blog, we explore agentic AI in healthcare by focusing on real-world use cases. 

The A H3N2 flu virus, which historically caused the most hospitalizations and deaths in older people, is the most common strain in the US so far. Over 90% of analyzed H3N2 infections are a new subclade K variant, which differs from the strain in current flu vaccines. Flu seasons often don’t peak until January or February so health officials say it’s too early to know how big a problem the mismatch will be. The current flu season has already seen nine pediatric deaths, and emergency department visits for flu in children have exceeded last season's peak. The US Centers for Disease Control and Prevention estimates at least 11 million illnesses, 120,000 hospitalizations, and 5,000 deaths from flu so far this season, recommending vaccination for everyone aged six months and older.

En 2023, le prix net médian des cellules CAR-T industrielles en France a été estimé entre 212 550 et 220 110 €. Cette estimation, fondée sur les données publiques disponibles, permet de produire des hypothèses et ainsi mieux cerner le prix réel effectivement supporté par le système de santé tout en révélant l’opacité des mécanismes de tarification. La fixation des prix des cellules CAR-T repose sur un équilibre permanent entre valorisation de l’innovation, soutenabilité budgétaire et équité d’accès aux soins. À mesure que de nouvelles thérapies innovantes apparaissent et que leurs indications s’étendent, il devient indispensable de développer des évaluations médicoéconomiques complètes et transparentes ainsi que des outils de suivi performants en vie réelle tels que le registre DESCAR-T, afin d’orienter les décisions publiques et de garantir un accès équitable à ces innovations thérapeutiques majeures.

One of the prevalent theories regarding the development of ICANS postulates that the rapid proliferation of CAR T cells leads to a robust inflammatory response, releasing a cascade of cytokines that may impact the central nervous system. High levels of these cytokines can penetrate the blood-brain barrier, leading to neuroinflammation and subsequent neurological symptoms. This cytokine release syndrome (CRS) often accompanies ICANS, further complicating the clinical picture.

Current “ex vivo” CAR-T therapies are bespoke and labor-intensive. They require a personalized manufacturing slot for every single patient, leading to bottlenecks that leave desperate patients waiting weeks or months.Vyriad’s approach, known as in vivo CAR-T, uses a modified lentivirus (LV-169) to act as a delivery truck. The Mechanism: The virus is engineered to seek out and bind specifically to T-cells inside the body The Payload: Once attached, it delivers a genetic instruction manual that teaches the T-cell to spot B-cell maturation antigen (BCMA), a protein found on multiple myeloma cancer cells The Result: The patient’s body becomes its own manufacturing facility, generating killer cells within days rather than weeks.