Bio

Community Engagement Liaison serving the Stanford University Postdoctoral Association (SURPAS)
Co-chair of JEDI-SURPAS
https://surpas.stanford.edu/about/the-surpas-leadership-team/

Stanford Advisors

All Publications

  • Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial. Nature medicine Agarwal, R., Bertaina, A., Soco, C., Long-Boyle, J. R., Saini, G., Kunte, N., Hiroshima, L., Chan, Y. Y., Willner, H., Krampf, M. R., Nofal, R., Barbarito, G., Sen, S., Van Hentenryck, M., Walck, E., Scheck, A., Perriman, R. J., Bouge, A., Istomina, E., Din, H. N., Klinger, E. F., Cheng, J. C., Wlodarski, M. W., Boelens, J. J., Shizuru, J. A., Pang, W. W., Weinberg, K., Parkman, R., Roncarolo, M. G., Porteus, M., Czechowicz, A. 2025

    Abstract

    Current hematopoietic stem cell transplantation (HSCT) conditioning strategies cause widespread tissue damage and systemic toxicities, especially in patients with DNA-repair deficiencies such as Fanconi anemia (FA). We have developed an alternative conditioning approach that incorporates the anti-CD117 antibody, briquilimab, which targets host hematopoietic stem and progenitor cells in place of genotoxic irradiation- and busulfan-based chemotherapy. Here we report a phase 1b clinical trial in patients with FA and bone marrow failure, evaluating safety and efficacy of briquilimab-based conditioning in combination with rabbit anti-thymocyte globulin, cyclophosphamide, fludarabine and rituximab immunosuppression and T cell receptor (TCR)αβ+ T cell-depleted and CD19+ B cell-depleted haploidentical HSCT. Primary endpoints of the trial included safety and engraftment, and secondary endpoints included pharmacokinetic measures and hematological and immunological recovery. All three patients have each undergone 2 years of follow-up to complete the phase 1b analysis. No treatment-emergent adverse events or acute graft-versus-host disease was observed. Patients experienced minimal toxicities, with typical mucositis and no veno-occlusive disease. Median neutrophil engraftment was 11 days (range 11-13 days) with robust donor chimerism up to 2 years post-HSCT (99-100%), meeting the primary endpoints of the study. Briquilimab cleared in each patient before HSCT without the need for adjustment. Red blood cell, platelet and lymphocyte recovery was comparable to previous reports with TCRαβ+ T cell-depleted and CD19+ B cell-depleted grafts. All patients are alive and well with resolution of earlier chromosomal breakage abnormalities in peripheral blood lymphocytes post treatment. These data demonstrate the broad potential of this protocol in maintaining HSCT efficacy while reducing toxicity. The phase 2 trial is ongoing (ClinicalTrials.gov identifier: NCT04784052 ).

    View details for DOI 10.1038/s41591-025-03817-1

    View details for PubMedID 40696207

    View details for PubMedCentralID 6509544

  • Evaluation of Bone Marrow in Fanconi Anemia Patients Treated with Briquilimab Antibody-Based Conditioning and TCRαβ<SUP>+</SUP> T-Cell/CD19<SUP>+</SUP> B-Cell Depleted Haploidentical Grafts Soco, C., Krampf, M. R., Chan, Y., Hoang, H., Kunte, N., Saini, G., Weinberg, K. I., Parkman, R., Bertaina, A., Agarwal, R., Porteus, M., Czechowicz, A. D. ELSEVIER. 2024: 2015-2016

    View details for DOI 10.1182/blood-2024-206381

    View details for Web of Science ID 001412856700039

  • Radiation and Busulfan-Free Hematopoietic Stem Cell Transplantation Using Briquilimab (JSP191) Anti-CD117 Antibody-Conditioning, Transient Immunosuppression and TCR α β + T-Cell/CD19+B-Cell Depleted Haploidentical Grafts in Patients with Fanconi Anemia Agarwal, R., Bertaina, A., Soco, C., Saini, G., Kunte, N., Hiroshima, L., Chan, Y., Willner, H., Krampf, M. L., Nofal, R., Barbarito, G., Sen, S., Felber, M., Van Hentenryck, M., Walck, E., Scheck, A., Thongthip, S., Logan, A. C., Dougall, K., Bouge, A., Boelens, J., Long-Boyle, J. R., Weissman, I. L., Shizuru, J., Pang, W. W., Weinberg, K. I., Parkman, R., Roncarolo, M., Porteus, M., Czechowicz, A. AMER SOC HEMATOLOGY. 2023

    View details for DOI 10.1182/blood-2023-178400

    View details for Web of Science ID 001159306700230