The goal of this prospective, open, single-arm clinical trial was to evaluate the safety and potential efficacy of CAR T cell therapy in children with refractory/recurrent lupus nephritis. The persistence and cell phenotype of CAR-T cells in vivo and CAR-T treatment-related inflammatory factors were evaluated after treatment. To explore new therapeutic methods, in order to reduce the side effects of traditional therapeutic drugs, increase curative effect, and finally make patients obtain long-term survival and improve survival quality.

Start Date01 May 2025

Sponsor / Collaborator

Guangzhou Women & Childrens Medical Center

100 Clinical Results associated with Chimeric Antigen Receptors T Cells(Guangzhou Women & Childrens Medical Center)

100 Translational Medicine associated with Chimeric Antigen Receptors T Cells(Guangzhou Women & Childrens Medical Center)

100 Patents (Medical) associated with Chimeric Antigen Receptors T Cells(Guangzhou Women & Childrens Medical Center)

206

Literatures (Medical) associated with Chimeric Antigen Receptors T Cells(Guangzhou Women & Childrens Medical Center)

01 Apr 2025·JAMA Oncology

A Narrative Review

Review

Author: Longo, Dan L. ; Armitage, James O. ; Roschewski, Mark

ImportanceAchieving remission is the first step toward a cure in treating aggressive B-cell lymphomas. Radiographic imaging, such as fluorodeoxyglucose–positron emission tomography/computed tomography scans are the current standard to define remission at the end of therapy but lack specificity for lymphoma and cannot detect disease at the molecular level. Identifying measurable residual disease with ultrasensitive detection of circulating tumor DNA potentially offers the possibility of improving clinical outcomes.ObservationsEarly studies of circulating tumor DNA in aggressive B-cell lymphomas showed a strong association with overall tumor burden, and baseline quantitative levels are associated with clinical outcomes after frontline chemotherapy. Next-generation sequencing methods that detect lymphoma-relevant genetic aberrations in circulating tumor DNA can also be used for noninvasive genotyping that strongly mirror tissue biopsies. Rapid changes in circulating tumor DNA dynamics after 1 or 2 cycles of frontline chemotherapy or within weeks of treatment with chimeric antigen receptor T-cell salvage therapy are also highly prognostic. Although serial monitoring of circulating tumor DNA can detect molecular relapse 3 to 6 months before clinical relapse, improved analytical thresholds are required to detect measurable residual disease at a singular point at the end of therapy. Modern advances in circulating tumor DNA methods now allow for the reliable detection of measurable residual disease, with an analytical detection threshold of 1 in 1 million cell-free DNA molecules.Conclusions and RelevanceThe results of this review suggest that modern ultrasensitive methods of detecting circulating tumor DNA may improve the current definition of remission in aggressive B-cell lymphomas. Incorporating circulating tumor DNA at the end of therapy assessment identifies patients who do not require surveillance monitoring and introduces paradigms of treating measurable residual disease within clinical trials. Practical barriers, including standardization of collection, availability, turnaround times, and cost, remain hurdles preventing widespread implementation into clinical practice.

01 Apr 2025·Molecular Therapy

CAR T cell therapy for treatment of Acute Myeloid Leukemia, Advances and Outcomes.

Review

Author: Hopewell, Emily ; Salman, Huda ; Khalifeh, Malak

Despite recent U.S. FDA approval of multiple therapies for patients with acute myeloid leukemia (AML), clinical outcomes for those patients continue to remain poor. There are very few effective immunotherapeutic modalities such as allogeneic stem cell transplant, SCT, for AML, and this is, in part, due to a lack of known antigens which are unique to AML and not present on vital normal hematopoietic precursors. Additionally, AML is supported by a hostile marrow TME that has a notable role in dampening T cell effector function ^1-4. MDSCs and Tregs play a pivotal role in AML microenvironment immune hostility towards endogenous T cells as well as adoptively transferred T cells (ACT). There are many clinical trials that are designed to test the feasibility and efficacy of ACT including Chimeric Antigen Receptor T cell therapies (CAR T cell) in AML, yet none is FDA approved for this fatal disease. In this review, we dissect these trials, their contribution to this therapeutic direction and their success.

01 Mar 2025·Quantitative Imaging in Medicine and Surgery

18F-fluorodeoxyglucose positron emission tomography-compute tomography parameters for predicting the prognosis and toxicity in children and young adults with large B-cell lymphoma receiving chimeric antigen receptor T-cell therapy

Article

Author: Wang, Wei ; Wang, Hongrong ; Yao, Xilan ; Yao, Shuang ; Yang, Xu ; Yang, Jigang

BackgroundChimeric antigen receptor (CAR) T-cell therapy has been proven to be an effective choice for patients with relapsed or refractory large B-cell lymphoma (LBCL). Early identification of patients who may have a poor prognosis and develop severe side effects is necessary. In this study, we aimed to assess the value of ¹⁸F-fluorodeoxyglucose positron emission tomography-computed tomography (¹⁸F-FDG PET/CT) parameters in predicting the prognosis and toxicity of CAR T therapy for children and young adults with LBCL.MethodsThis retrospective cohort study included patients with LBCL under 30 years of age who underwent ¹⁸F-FDG PET/CT at Beijing Friendship Hospital of Capital Medical University and Beijing GoBroad Boren Hospital before CAR T-cell infusion within 1 month. ¹⁸F-FDG PET/CT metabolic parameters including maximum standardized uptake value (SUVmax), total metabolic tumor volume (TMTV), and total lesion glycolysis (TLG) were recorded. Clinical characteristics and laboratory indicators were also collected. The main endpoints were progression-free survival (PFS) and overall survival (OS) as estimated by the Kaplan-Meier method and log-rank test. We also assessed the relationship between these metabolic and clinical parameters and severe toxicities, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS).ResultsForty-five patients were recruited. The median duration of the follow-up period was 13.9 months. Patients with an age-adjusted international prognostic index (aaIPI) 2-3 (P=0.014) and TMTV >101.4 mL (P=0.026) had a shorter PFS. Patients with Eastern Cooperative Oncology Group (ECOG) performance status 2-3 (P=0.015) and TMTV >101.4 mL (P=0.011) had a shorter OS. Lactate dehydrogenase (LDH) > upper normal limit (UNL) (P=0.030) and TMTV >101.4 mL (P=0.042) were associated with grade 2-4 CRS, and C-reactive protein (CRP) > UNL (P=0.014) was associated with grade 2-4 ICANS.ConclusionsaaIPI and TMTV were independent risk factors for PFS, and ECOG score and TMTV had independent prognostic value for OS. Higher LDH and TMTV were associated with grade 2-4 CRS, and higher CRP was associated with more severe ICANS. Thus, integrating these metabolic parameters of ¹⁸F-FDG PET/CT and clinical-laboratory indicators can be valuable for managing children and young adults with B-cell lymphoma who have received CAR T-cell therapy.

100 Deals associated with Chimeric Antigen Receptors T Cells(Guangzhou Women & Childrens Medical Center)

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Indication	Highest Phase	Country/Location	Organization	Date
Lupus Nephritis	Phase 3	China	Guangzhou Women & Childrens Medical Center	01 May 2025

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