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
- 1Bone marrow transplantation · 2020
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- 3
Publications
Funding
Completed grants (7)
- MPIU01HL128566-05Hematopoietic Stem Cell Transplantation for Young Adults with Sickle Cell DiseaseNational Heart, Lung, and Blood Institute · 2016–2021 · via Emory University
- MPIUM1AI068632-10IMPAACT P1107: Cord Blood Transplantation Using CCR5-Delta32 Donor Cells for the Treatment of HIV and Underlying DiseaseNational Institute of Allergy and Infectious Diseases · 2014–2016 · via The Johns Hopkins University
- PIK24CA116471-05Immunologic and pharmacologic studies in allotransplantNCI · 2007–2014 · via NIH RePORTER
- PIR21CA115097-02Busulfan conditioning: optimization, kinetics, genomicsNCI · 2006–2009 · via NIH RePORTER
- PIR21CA101337-02Minor Histocompatilibity Vaccination After Allo-Transpl*NCI · 2003–2006 · via NIH RePORTER
- PIR21CA100240-02TCR-gene modified t-cells for adaptive immunotherapyNCI · 2003–2006 · via NIH RePORTER
Clinical research
Completed & closed trials (3)
- PICompassionate Use of Out-of-Specification Axicabtagene ciloleucel for Treatment of Diffuse Large B-Cell Lymphoma in Patient A-BWeill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2022)
- PILong term follow up of subjects who received pbcar0101 in other clinical studiesPrecision BioSciences, Inc. · IRB STUDY CLOSURE (as of 2022)
- PINCT03483324A Phase 1 Open-Label, Multi-Center Trial of AB-110 in Adults With Hematologic Malignancies Undergoing CordPHASE1 · Angiocrine Bioscience, Inc. · IRB STUDY CLOSURE (as of 2021)Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia in Remission; Myelodysplastic Syndromes; Other Acute Leukemias
News mentions
- Patient Possibly Cured of HIV Infection by Special Stem-Cell TransplantFebruary 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.