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Yahoo
24-06-2025
- Business
- Yahoo
Priothera Secures €1.7 million i-Nov Funding by Bpifrance for Rare Blood Cancer Clinical Program
Priothera Secures €1.7 million i-Nov Funding by Bpifrance for Rare Blood Cancer Clinical Program Funding to support MOCART, a clinical programme evaluating mocravimod added to standard CAR-T cell therapy – Priothera, a late-stage biopharma company pioneering the development of mocravimod, a novel oral sphingosine 1 phosphate (S1P) receptor modulator, to treat hematologic malignancies, today announced that it has been awarded nearly €1.7 million in non-dilutive funding through the i-Nov innovation competition. Part of the France 2030 initiative, i-Nov is a flagship French government program operated by Bpifrance to support breakthrough innovation from high-potential French companies across strategic sectors. The funding will support Priothera's clinical programme to evaluate whether adding mocravimod to commercial CAR-T cell therapies could improve patient outcomes. CAR-T cell therapies represent a novel and promising modality for the treatment of hematological malignancies. They have demonstrated the potential for remarkable clinical responses and durable disease control in patients with acute lymphoblastic leukemia (ALL), non-Hodgkin lymphoma and multiple myeloma. However, their use is still associated with significant challenges, as 40-60% of patients treated with CAR-T cells experience high-grade toxicities, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), a form of severe neurological toxicity. Mocravimod is a novel, oral S1P receptor modulator with a unique dual mechanism of action that has the potential to enhance the effectiveness of CAR-T cell therapy by: Reducing the incidence and severity of CRS and ICANS, and Improving response rates and durability of treatment 'We are honoured to receive this i-Nov funding from Bpifrance, which underscores the innovation and therapeutic potential of mocravimod beyond allo-HCT,' said Florent Gros, Co-Founder and CEO of Priothera. 'With its unique immunomodulatory properties, mocravimod is well-positioned to become a key component in the next generation of cell therapy regimens. The MOCART trial represents an exciting expansion of our clinical development into CAR-T therapy, building on our deep expertise in allo-HCT and momentum from our ongoing global Phase 3 MO-TRANS trial in acute myeloid leukemia.' Priothera continues to advance mocravimod in the MO-TRANS global Phase 3 study for patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic cell transplantation (allo-HCT). The company remains focused on unlocking the full therapeutic potential of S1P receptor modulation across multiple settings in blood cancers. *** About mocravimodMocravimod (KRP203) is a synthetic S1P receptor modulator being developed for the adjunctive and maintenance treatment of AML to enhance the curative potential of allo-HCT. Mocravimod's dual mechanism of action preserves the graft-versus-leukemia (GvL) effect, critical for eliminating cancer cells while reducing the risk of graft-versus-host disease (GvHD), a major complication following allo-HCT. This novel treatment approach – mocravimod being the only S1P receptor modulator in development to treat blood cancers – tackles a high unmet medical need and aims to improve treatment outcomes and patients' quality of life. About PriotheraPriothera is a late-stage biopharma company pioneering the development of mocravimod, a potential new standard of care in hematologic cancers, in addition to cellular therapies such as hematopoietic cell transplantation and CAR-T cell therapies. Mocravimod is being developed as an adjunctive and maintenance therapy for hematological malignancies requiring allogeneic hematopoietic cell transplant (allo-HCT), focusing initially on acute myeloid leukemia (AML). Mocravimod is currently the only treatment with the potential to reduce transplant side effects of graft-versus-host disease (GvHD) without compromising the graft's anticancer effect against leukemia (Graft-versus-Leukemia, or GvL), thereby enhancing the curative potential of allo-HCT. Founded in 2020, Priothera operates in France, with headquarters in Dublin. The company is led by a highly experienced management team with deep expertise in hematology, oncology, immunology and cell-based therapies. Priothera is backed by leading international life sciences investors, including Fountain Healthcare Partners, abrdn, EarlyBird Venture Capital, BEI and Bpifrance Grand Est. For more information please visit or follow Priothera on LinkedIn Contacts PriotheraFlorent Gros, CEOE: info@ MEDiSTRAVA ConsultingSylvie Berrebi, Frazer HallE: priothera@ +44 (0) 203 928 6900
The Hindu
24-06-2025
- Health
- The Hindu
CAR T-cell therapy and its promise of new hope for cancer treatment
In recent years, cancer treatment has taken a big leap forward with a new, advancement known as CAR T-cell therapy. This may may sound complex, but the idea behind it is fairly simple: using the power of a patient's own immune system to fight and destroy cancer cells. What is CAR T-cell therapy? Our body's immune system has special cells called T-cells that help fight viral infections and cancer. In CAR T-cell therapy, doctors take these T-cells from a person's blood (a process known as leukapheresis) and modify them in a lab so they can recognise and destroy cancer cells more effectively. These modified cells are called CAR T-cells, where 'CAR' stands for Chimeric Antigen Receptor, a special protein added to help the T-cells find cancer cells. Once these smart T-cells are ready, they are put back into the patient's body through an intravenous (IV) drip. From there, they search for cancer cells and attack them. How does this therapy help? CAR T-cell therapy has shown promising results so far, especially in blood cancers like leukaemia and lymphoma. For many patients who do not respond to other treatments like chemotherapy or radiation, CAR T-cell therapy has helped achieve remission, which means their cancer signs stopped growing or even disappeared. This therapy is not just another medicine—it is personalised for each patient, using their own cells. That is what makes it powerful and unique. Where does CAR-T help? CAR T-cell therapy is approved for use in haematological malignancies like leukemia, non-Hodgkin lymphoma and multiple myeloma. It is indicated in patients who have either relapsed (cancer has returned after treatment) or who have a refractory (cancer has not responded to prior treatment) disease. Are there any side effects? Like any powerful treatment, CAR T-cell therapy can have side effects. The most common side effect is cytokine release syndrome (CRS), which is like a strong immune reaction. It can cause fever, low blood pressure, or trouble with breathing. However, doctors are trained to handle these symptoms, and many patients recover from them within a few days. Another possible side effect is a condition known as immune effector cell-associated neurotoxicity syndrome (ICANS) where patients have symptoms like headache, changes in consciousness, confusion, and loss of balance. These side effects are mostly temporary and can be managed medically. The path ahead At present, CAR T-cell therapy is used mainly for certain types of blood cancers, but scientists are working to expand this therapy for use in solid tumours like breast or lung cancer. With more research and support, it could become more widely available and affordable. CAR T-cell therapy is akin to training your own immune system to be a smart soldier that can find and destroy cancer. It gives new hope to people who have very few options left. While it may not be the answer for every cancer yet, it is a big step toward a future where cancer can be fought more naturally, with fewer side effects and better results. (Dr. Arshad Raja is a consultant in haematology & haemato oncology at Kauvery Hospital Alwarpet. arshadraj1989@
Yahoo
16-06-2025
- Business
- Yahoo
J&J reports results from antibody combo trial for MM patients
Johnson & Johnson (J&J) has reported new outcomes from the Phase II RedirecTT-1 trial of bispecific antibodies, Talvey (talquetamab-tgvs) and Tecvayli (teclistamab-cqyv) for relapsed/refractory multiple myeloma (r/r MM). The data showed a high overall response rate (ORR) with durability in those who have triple-class-exposed (TCE) RRMM with true extramedullary disease (EMD). In the trial, which enrolled 90 subjects, the investigational combo resulted in an ORR of 78.9%, with over half of the subjects achieving a complete response or better, representing a significant improvement over the average ORR of less than 40% for this patient group. Notably, responses were also found to be high among those previously treated with B-cell maturation antigen (BCMA) CAR-T or anti-FcRH5 bispecific antibodies. According to the company, subjects in the trial showed deep and durable responses, with 66.2% remaining in response at the data cutoff and a median follow-up of 13.4 months. At one year, 61% of subjects were progression-free and alive, and 74.5% were alive, with median overall survival not yet reached. The combo was found to be consistent with prior reports of them as single agents. Subjects had the option to switch to once-a-month dosing, which might have contributed to better tolerability. The reports of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were found to be mostly low grade. The study's findings were featured at the 2025 European Hematology Association Congress. EMD represents a severe form of MM, where myeloma cells form tumours in soft tissues and organs. Johnson & Johnson innovative medicine multiple myeloma disease area leader and vice-president Jordan Schecter said: 'Patients with extramedullary myeloma, especially those who have exhausted prior therapies, need more effective treatment options. 'Our first-in-class bispecific antibodies, Talvey and Tecvayli, have transformed treatment for relapsed or refractory multiple myeloma.' Recently, J&J reported that Tremfya decreased the symptoms and signs of active psoriatic arthritis (PsA) at 24 weeks in individuals against a placebo in the Phase IIIb APEX trial. "J&J reports results from antibody combo trial for MM patients" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Medscape
08-05-2025
- Health
- Medscape
Lymphoma and CAR T–Related Renal Injury: Assessing the Risks
In the treatment of non-Hodgkin lymphoma with CD19 chimeric antigen receptor (CAR) T-cell therapy, baseline kidney function did not appear to be associated with an increased risk of developing acute kidney injury (AKI) from the treatment. However, other key factors, including low albumin, showed a link. 'Our study is the largest to date examining renal toxicity in CD19 CAR T–treated lymphoma patients,' senior author Roni Shouval, MD, PhD, director of the Precision Cellular Therapy Laboratory at Memorial Sloan Kettering (MSK) Cancer Center, New York City, said in an interview. While the findings show no link between baseline kidney function and the development of AKI, ultimately, 'hypoalbuminemia may be a better flag for AKI risk and a prompt for closer monitoring or proactive interventions such as fluid management and nephrotoxin avoidance,' Shouval said. The study was published in March in Haematologica . CAR T-cell therapy is known to provide durable disease remission in relapsed or refractory non-Hodgkin lymphoma, including in the disease subsets of large B-cell lymphoma (LBCL) and mantle cell lymphoma (MCL). While the common treatment-related toxicities of cytokine release syndrome (CRS) and immune effector cell–associated neurologic syndrome (ICANS) are well documented, less has been reported regarding AKI treatment–related effects and outcomes. The US Food and Drug Administration Adverse Event Reporting System (FAERS) AKI Reports To investigate, Shouval, first author Alexander P. Boardman, MD, also of MSK, and colleagues first screened data from the FAERS database, identifying 5912 patients who had adverse events related to CD19 CAR T-cell therapy reported between January 2017 and September 2022. The patients had a median age of 62 years, and the most common CAR T-cell therapies utilized were axi-cel (n = 3526 patients), followed by tisa-cel (n = 1780), liso-cel (n = 178), and brexu-cel (n = 428). The main indications for CAR T-cell treatment were non-Hodgkin lymphoma (88%) and acute lymphoblastic leukemia (11%). Overall, 211 (3.6%) patients developed AKI that was determined to have a primary etiology of CD19 CAR T-cell therapy, with no significant differences based on age, sex, event year, type of CD19 CAR T-cell therapy, or indication. Of note, AKI was more common among patients treated with CD19 CAR T-cell therapy than among all other patients with cancer in the FAERS database (age- and sex-adjusted reporting odds ratio [ROR], 1.72). AKI developed at a median of 2.5 days post-infusion, and patients with AKI were more likely to have had concurrent CRS than those with other CAR T-cell reports not involving AKI (64% vs 50%; P < .001). The rates of life-threatening illness (22.75% vs 8.00%) and mortality (49% vs 23%; P < .001 for all) were higher among patients with AKI than among those with other CD19 CAR T-cell reports not involving AKI. 'In the FAERS dataset, we observed a disproportionately high reporting of AKI among CAR T recipients compared to other oncology patients, reinforcing that renal toxicity is a consistent safety signal on a population level,' Shouval said. A Closer Look In a subsequent single-center analysis, the authors identified 399 patients with relapsed or refractory non-Hodgkin lymphoma treated with CD19 CAR T-cell therapy at MSK Cancer Center between April 2016 and December 2023. Of the patients, 84% had LBCL, 11% had MCL, and 5% had non-LBCL. Their median age was 66 years, and the most common treatments included were axi-cel (46%), tisa-cel (20%), liso-cel (29%), and brexu-cel (5%). Of note, patients' pre-lymphodepletion estimated glomerular filtration rate (eGFR) was not associated with overall or progression-free survival, CRS grade ≥ 2, ICANS grade ≥ 2, or neutropenia. 'Thus, baseline renal function was not significantly associated with efficacy or toxicity endpoints,' the authors reported. AKI, Kidney Outcomes From the time of CAR T-cell infusion to day 100, AKI of any grade was reported among 39 (10%) of the MSK patients, with only a small percentage (5%) having grade ≥ 2 AKI. The majority of AKI cases (71.8%) were attributed to prerenal factors; 14 cases occurred concurrently with CRS. For survival outcomes, those developing AKI within 100 days of treatment had significantly lower progression-free survival (hazard ratio [HR], 2.63; P < .001) and overall survival (HR, 3.36; P < .001) than those without AKI. And among those with AKI grade ≥ 2, progression-free survival was significantly lower (HR, 3.14; P < .001), as was overall survival (HR, 4.18; P < .001). Low Albumin, Inflammatory Markers Linked to Increased AKI Risk Notably, after a multivariate adjustment, serum albumin level prior to lymphodepletion was a significant risk factor for AKI (HR, 0.15; P < .001). In addition, higher levels of inflammatory markers, specifically serum interleukin 6 (HR, 1.74; P = .008) and tumor necrosis factor alpha (HR, 2.23; P < .001), on day 0, just prior to CAR T-cell infusion, were significantly associated with the risk for AKI 'Intriguingly, we are the first to determine that hypoalbuminemia is strongly associated with risk of AKI after CAR T-cell therapy,' the authors reported. 'Hypoalbuminemia emerged as an even more robust independent predictor of AKI than baseline eGFR,' Shouval added. 'In clinical terms, we believe hypoalbuminemia is surrogate for frailty and possibly systemic inflammation,' he said. 'Therefore, patients with low albumin should be recognized as high risk for renal complications.' 'Overall, we find that most AKI events after CAR T-cell infusion are of grade 1 and that most patients recover from these events within 3 months,' the authors wrote. However, in looking at the eGFR and AKI outcomes together, Shouval concluded that 'these data position AKI as both a clinically meaningful and prognostically significant complication, deserving greater attention in the CAR T risk-benefit assessment.' Findings Point to a 'Cytokine-Driven Phenomenon' Commenting on the research, Michael D. Jain, MD, PhD, medical director of the Immune and Cellular Therapy Program at the H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, noted that the findings underscore that CAR T-cell therapy 'is really a cytokine-driven phenomenon rather than related to comorbidities that cause baseline chronic kidney disease [CKD].' He added that low albumin, often a marker of inflammation/cytokines, 'might contribute to AKI because it affects intravascular volume and thus blood flow to the kidney.' Jain agreed that the risk for AKI and other complications from CAR T is 'related more to tumor burden and inflammation than to baseline renal dysfunction.' 'In our center, we have protocols to give CAR T cells to patients who are on dialysis and believe we can safely deliver their therapy,' he noted. A Word of Caution Re CKD In a smaller study of 155 patients treated with CAR T-cell therapy for relapsed/refractory LBCL, a history of CKD was, contrarily, found to indeed be associated with AKI on a multivariate analysis (RR, 2.7; P = .04). However, while in Shouval's study, most patients who had low eGFR (< 60 mL/min/1.73 m2) received either liso-cel or tisa-cel, in this study, the majority of patients received axi-cel. Based on that, 'I would offer caution [using CAR T-cell therapy] in those with CKD, especially in those receiving axi-cel,' senior author Narendranath Epperla, MD, of the James Cancer Hospital, The Ohio State University, Columbus, Ohio, said in an interview. He added, however, that Shouval's research importantly 'highlights a clinically relevant yet understudied complication of CAR-T that would aid clinicians in decision-making and counseling peri-CAR T.'
Yahoo
14-02-2025
- Health
- Yahoo
Press Release Biocartis NV: Biocartis announces new data on early CAR-T vector load assessment with Idylla™ presented at ASTCT: A Potential Breakthrough in LBCL Treatment Monitoring
PRESS RELEASE - 14/02/2025, 09:30 CET Biocartis announces new data on early CAR-T vector load assessment with Idylla™ presented at ASTCT: A Potential Breakthrough in LBCL Treatment Monitoring Mechelen, Belgium, 14 February 2025 – Biocartis NV ('Biocartis'), an innovative molecular diagnostics company, is pleased to announce that new research on the prototype Idylla™ CD19 CAR-T vector load Assay was presented as a poster at ASTCT in Honolulu, HI, US, on 13 February 2025. The new study1 (Bharadwaj et al, 20252) demonstrated that early CD19 CAR-T vector load quantification in peripheral blood may improve the prediction of clinical outcomes and toxicity management for patients receiving axicabtagene ciloleucel (axi-cel) for Large B-cell Lymphoma (LBCL). The study enrolled 100 patients undergoing axi-cel therapy. Blood samples were analyzed using both droplet digital PCR (ddPCR) centrally, and local testing in the 3 centers with the prototype Idylla™ CAR-T Assay on the Idylla™ Platform. The study positioned the Idylla™ Platform as an automated, local hospital testing system that can provide results in approximately 90 minutes with only 2 minutes of hands-on time, straight from 0.5 ml of blood. The results revealed a 98.6% concordance between the prototype Idylla™ CAR-T Assay and ddPCR, confirming its reliability as a potential early alternative to standard methods. Key Findings: Early CAR-T Vector load increase predicted severe ICANS: Patients experiencing a steep rise in CD19 CAR-T vector load within the first 5 days post-infusion were more likely to develop severe immune effector cell-associated neurotoxicity syndrome (ICANS). Day 3 Idylla™ measurements predicted ICANS severity: Predictive modeling identified day 3 vector load as a significant predictor of ICANS severity using Idylla™. Correlation with Progression-Free Survival (PFS): Patients were stratified into high and low vector load slope groups obtained during the first 5 days after infusion, with significant differences in PFS observed for both Idylla™ and ddPCR measurement methods. Kaplan-Meier curves revealed PFS rates separate by day 50 (100% vs. 70%), and persisted until the end of follow-up beyond day 300 (90% vs. 50%) for high vs. low vector load increases. These findings suggest that, once approved, local hospital testing with Idylla™ could revolutionize toxicity management and treatment monitoring in CAR-T therapy. By providing real-time, accessible vector load measurements, clinicians may better predict toxicity risk, personalize treatment strategies, and improve patient outcomes. W. Michael Korn, M.D., Chief Medical and Scientific Officer of Biocartis, commented: 'These findings underscore the power of early, local hospital testing in CAR-T therapy. By providing real-time insights into CAR-T expansion dynamics, Idylla™ may enable clinicians to better predict toxicity risks and optimize treatment strategy - ultimately improving patient outcomes.' ----- END ----- More information: info@ Biocartis NV. Generaal De Wittelaan 11B, 2800 Mechelen, Belgium About Biocartis With its revolutionary and proprietary Idylla™ Platform, Biocartis aspires to enable personalized medicine for patients around the world through universal access to molecular testing, by making molecular testing actionable, easy, fast and suitable for any lab. The Idylla™ Platform is a fully automated sample-to-result, real-time PCR (Polymerase Chain Reaction) based system designed to offer in-house molecular biomarker testing in only 3 hours, allowing fast and optimal treatment selection. Idylla™'s continuously expanding menu of molecular diagnostic tests and research assays addresses key unmet clinical needs. Today, Biocartis offers tests supporting melanoma, colorectal, lung, breast, thyroid, brain and blood cancer. More information: Follow us on X (Twitter): @Biocartis. The Idylla™ CAR-T Assay is a prototype and is not commercially available. The Biocartis and Idylla™ trademark and logo are used trademarks owned by Biocartis. © February 2025, Biocartis NV. All rights reserved. 1 This study was conducted with direct support from Biocartis.2 Bharadwaj, S. et al. (2025). Rapid increase in Blood CD19 CAR-T Vector Load during the First 5 Days Post Infusion is Associated with Severe ICANS. in to access your portfolio
