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Overview

The transplant program at WCMC is dedicated to pursuing clinical and translational research while providing superior patient care. Areas of research interest include:

1) Development of novel conditioning regimens for patients with advanced hematologic malignancies.

a. Leukemia

The concept of reduced intensity transplantation has not entirely fulfilled its promise. We now routinely offer transplantation to patients well into their 70s, but toxicity, chronic GVHD and recurrence remain major problems. WCMC transplant program is focusing on the development of new regimens with increased efficacy, but limited chronic GVHD. We have studied various modifications of current regimens as well as novel methods of GVHD prophylaxis. Examples of ongoing studies include the combination of CPX-351, a novel anti leukemic drug as a "bridge to transplant" (U.Gergis PI) or the use of clofarabine conditioning regimens. New studies under development will involve the incorporation of hypomethylating agents before transplant as a method of chemotherapy sensitization as well as post-transplant administration of hypomethylating agents. In the near future we hope to be able to assign treatment based on sophisticated detection of minimal residual disease.

b. Lymphoma

Thanks to the introduction of targeted treatment approaches, the outcome for patients with lymphoma has improved dramatically for most, but not all patients.We are particularly focused on improving outcomes for patients with lymphoma who failed initial treatments. We are devising novel treatment combinations and conditioning regimens particularly suited for such patients.

c. Myeloma

Outcomes for patients with myeloma have much improved. Patients live longer, healthier and more productive lives than ever before. This has in large part occurred because of the introduction of autologous transplant which remains a mainstay of the treatment for myeloma. In induces longer and better remissions than any other treatment and patients often can be without treatment (treatment holidays) for prolonged periods of time. But continued research is needed to reach the goal of cure. Two ongoing studies evaluate the incorporation of high dose lenalidomide and high dose Bendamustine in transplant conditioning regimens. The Principal Investigators for these studies are Dr. Mark and Dr. Shore respectively. Protocols for allogeneic transplantation are under development and may be suitable for patients with high risk disease.

Highlights

  1. 1
    Bone marrow transplantation · 2020
    PMID: 32518291DOISenior author6 citations
  2. 2
    Leukemia & lymphoma · 2024
    PMID: 39724014DOISenior author
  3. 3
    Leukemia & lymphoma · 2021
    PMID: 34586001DOIFirst author

Publications

357 publications
Completed grants (7)
  • MPI
    CAR T Cells for Advanced Thyroid Cancer
    National Cancer Institute · 2021–2023
    R01CA254035-01A1
  • MPI
    Hematopoietic Stem Cell Transplantation for Young Adults with Sickle Cell Disease
    National Heart, Lung, and Blood Institute · 2016–2021 · via Emory University
    U01HL128566-05
  • MPI
    IMPAACT P1107: Cord Blood Transplantation Using CCR5-Delta32 Donor Cells for the Treatment of HIV and Underlying Disease
    National Institute of Allergy and Infectious Diseases · 2014–2016 · via The Johns Hopkins University
    UM1AI068632-10
  • PI
    Immunologic and pharmacologic studies in allotransplant
    NCI · 2007–2014 · via NIH RePORTER
    K24CA116471-05
  • PI
    Busulfan conditioning: optimization, kinetics, genomics
    NCI · 2006–2009 · via NIH RePORTER
    R21CA115097-02
  • PI
    Minor Histocompatilibity Vaccination After Allo-Transpl*
    NCI · 2003–2006 · via NIH RePORTER
    R21CA101337-02
  • PI
    TCR-gene modified t-cells for adaptive immunotherapy
    NCI · 2003–2006 · via NIH RePORTER
    R21CA100240-02

Clinical research

3 studies
Completed & closed trials (3)
  • PI
    Compassionate Use of Out-of-Specification Axicabtagene ciloleucel for Treatment of Diffuse Large B-Cell Lymphoma in Patient A-B
    Weill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2022)
  • PI
    Long term follow up of subjects who received pbcar0101 in other clinical studies
    Precision BioSciences, Inc. · IRB STUDY CLOSURE (as of 2022)
  • PI
    A Phase 1 Open-Label, Multi-Center Trial of AB-110 in Adults With Hematologic Malignancies Undergoing Cord
    PHASE1 · Angiocrine Bioscience, Inc. · IRB STUDY CLOSURE (as of 2021)
    Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia in Remission; Myelodysplastic Syndromes; Other Acute Leukemias
    NCT03483324

News mentions

  • Patient Possibly Cured of HIV Infection by Special Stem-Cell Transplant
    February 18, 2022

    A patient living with HIV who received a blood stem cell transplant for high-risk acute myeloid leukemia has been free of the virus for 14 months after stopping HIV antiretroviral drug treatment, suggesting a cure, according to the Weill Cornell Medicine physician-scientists who performed the transplant and managed her care.

External relationships

Ownership

Hemogenyx LLC

Advisory/Scientific Board Member

ADC Therapeutics · Taylor & Francis Group, LLC

Professional Services

Autolus Limited

Other

CTI BioPharma · Gamida Cell, Inc. · Hemogenyx LLC · Intellia Therapeutics, Inc. · Pfizer, Inc.