Trial Updates – VJRegenMed

KarMMa-3 results: ide-cel vs standard regimens in triple-class exposed multiple myeloma

Anya Kerkache — Thu, 09 Mar 2023 11:31:45 +0000

In this video, Sergio Giralt, MD, Memorial Sloan Kettering Cancer Center, New York City, NY, gives an overview of the results of the KarMMa-3 study (NCT03651128), a Phase III trial comparing idecabtagene vicleucel (ide-cel) versus standard regimens in patients with triple class–exposed relapsed/refractory (R/R) multiple myeloma. The study reported a significant benefit for ide-cel in terms of complete remission (CR) rate and progression-free survival (PFS) across all risk categories. This interview took place at the 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR® held in Orlando, FL.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

Preliminary results from Phase I study of CD123 CAR-T therapy in pediatric R/R AML

Anya Kerkache — Thu, 09 Mar 2023 11:31:44 +0000

In this video, Swati Naik, MBBS, St. Jude Children’s Research Hospital, Memphis, TN, discusses the rationale and preliminary results from a Phase I study evaluating the safety and feasibility of CD123-targeting CAR-Ts in pediatric patients with relapsed/refractory (R/R) acute myeloid leukemia (AML; NCT04318678). Dr Naik first explains the challenges associated with designing effective CAR-Ts for AML, including the identification of a suitable target, patient heterogeneity, and the aggressive and immunosuppressive nature of the disease. Dr Naik then comments on the results of the study, which showed evidence of expansion and anti-leukemic activity at different dose levels, with no dose-limiting toxicities. This interview took place at the 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR® held in Orlando, FL.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

CARBON trial: investigating CTX110 allogeneic CRISPR-Cas9–engineered CAR-Ts in R/R LBCL

Anya Kerkache — Thu, 26 Jan 2023 12:11:30 +0000

Constantine Tam, MBBS (Hons), MD, FRACP, FRCPA, Peter MacCallum Cancer Centre, St. Vincent’s Hospital, University of Melbourne, Melbourne, Australia, reports results from the CARBON Phase I dose-escalation study (NCT04035434), testing the safety and efficacy of a novel allogeneic CRISPR-Cas9-engineered CAR-T product, CTX110, in heavily pre-treated patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). The study reported that the CAR-Ts showed clinical activity with the ability to expand in the body, and a low risk of graft-versus-host disease (GvHD). This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

The safety and efficacy of UCART123v1.2 in R/R AML: results from AMELI-01

Anya Kerkache — Thu, 26 Jan 2023 12:11:29 +0000

David Sallman, MD, Moffitt Cancer Center, Tampa, FL, discusses the results of the AMELI-01 Phase I trial of UCART123v1.2 in adult patients with relapsed refractory (R/R) CD123+ acute myeloid leukemia (AML; NCT04106076). UCART123v1.2 is an anti-CD123 allogeneic CAR-T product, that has been genetically modified to minimize the occurrence of graft-versus-host disease (GvHD). Overall, results suggest UCART123v1.2 is well tolerated, though severe cytokine release syndrome (CRS) did occur, and that three-drug lymphodepletion optimizes CAR-T expansion and patient responses. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

Phase I study of CC-98633 in R/R multiple myeloma

Anya Kerkache — Thu, 26 Jan 2023 12:11:26 +0000

In this video, Luciano Costa, MD, PhD, UAB School of Medicine, Birmingham, AL, shares some results from a Phase I study evaluating CC-98633, a next-generation BCMA-targeting CAR-T cell therapy, in patients with relapsed/refractory (R/R) multiple myeloma (NCT04394650). Dr Costa first highlights the NEX-T manufacturing process used to develop this agent, and then discusses the activity and safety of this agent as demonstrated in the study. This interview took place at the 64th ASH Annual Meeting and Exposition congress held in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

MOMENTUM: ARU-1801 in sickle cell disease

Simon Ng — Mon, 07 Feb 2022 17:06:07 +0000

Will Chou, MD, Aruvant, New York, NY, presents preliminary results from the Phase I/II MOMENTUM study (NCT02186418) of ARU-1801, a lentiviral gene therapy, in patients with sickle cell disease (SCD). 5 patients are currently enrolled and the investigational therapy has a promising efficacy and safety profile, with no vaso-occlusive episodes (VOE) or hospital stays reported in patients. Dr Chou additionally reports a durable response in patients, with therapeutic hemoglobin detected in patients 3 years after initial administration. This interview took place at Advanced Therapies Week 2022.

DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris

Simon Ng — Thu, 27 Jan 2022 18:00:59 +0000

Samik Basu, MD, Cabaletta Bio, Philadelphia, PA, talks on the differences between chimeric autoantibody receptor (CAAR) T-cells and chimeric antigen receptor (CAR) T-cells. Unlike CD19-targeted CAR T-cells which target and kill all CD19-positive B-cells, CAAR T-cells selectively eliminate memory B-cells that give rise to plasma cells generating autoantibodies. Whilst the intracellular portion of CAAR T-cells and CAR T-cells are similar, the extracellular portion of the CAAR T-cell contains a portion of the protein targeted by the autoantibody. Dr Basu additionally outlines the design and early results of the Phase I, dose-escalation DesCAARTes trial (NCT04422912) investigating the maximum tolerated dose of DSG3-CAART, an autologous desmoglein 3 (DSG3) CAAR T-cell, in patients with mucosal-dominant pemphigus vulgaris (mPV). DSG3-CAART appears safe and a dose-dependent increase in CAAR T-cell persistence was observed within the first 29 days after infusion. This interview took place at Advanced Therapies Week 2022.

jCell: allogeneic cell therapy for retinitis pigmentosa

admin_vjr_user — Tue, 04 Jan 2022 15:29:59 +0000

Gil Carrasquinho, MD, MBA, Santen Pharmaceutical Co. Ltd., discusses the mechanism of action of jCell Therapy, an investigational allogeneic cell therapy for retinitis pigmentosa (RP), a degenerative retinal disease. Human retinal progenitor cells (RPCs) are administered via intravitreal injection causing the release neurotropic factors, resulting in a reduction in photoreceptor cell death and restoration of surviving photoreceptor and retinal ganglion cell function. A Phase IIb trial (NCT04604899) evaluating jCell in patients with RP demonstrated promising efficacy and tolerability data. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.

RP-L301 lentiviral gene therapy for PKD

admin_vjr_user — Mon, 27 Dec 2021 12:22:59 +0000

Jose-Luis López-Lorenzo, MD, University Hospital Jimenez Diaz, Madrid, Spain, describes ongoing research into RP-L301, an investigational lentiviral gene therapy for pyruvate kinase deficiency (PKD). This approach involves autologous hematopoietic stem cells being transduced ex vivo with a lentiviral vector carrying a functional copy of the deficient PKLR gene. Based on preclinical studies in a murine model of PKD, a Phase I clinical trial (NCT04105166) evaluating RP-L301 is currently underway. While results obtained from two patients treated so far are promising, Dr López-Lorenzo notes that data from the treatment of more patients with a longer follow-up time is needed and highlights the need for a less complex and safer treatment protocol in the future. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

An update on the Phase I trial evaluating RP-L301 in PKD

admin_vjr_user — Mon, 27 Dec 2021 12:22:57 +0000

Jose-Luis López-Lorenzo, MD, University Hospital Jimenez Diaz, Madrid, Spain, provides an update on a Phase I clinical trial (NCT04105166) evaluating the safety of RP-L301, an investigational lentiviral gene therapy, in patients with pyruvate kinase deficiency (PKD). Dr López-Lorenzo outlines the study design, which involves the collection of peripheral blood hematopoietic stem cells (HSCs) which are transduced with a lentiviral vector carrying a functional copy of the deficient PKLR gene. Patients then receive a busulfan-containing conditioning regimen and then intravenous infusion of the RP-L301 HSC product. In the first two patients treated, hemoglobin levels were restored to normal levels, both report improved quality of life and no serious adverse events have been attributed to RP-L301. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.