Research

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Portfolio

Clinical trial support

As we are broadening the scope of our services, here are some examples of the type of support for phase I/II trials we are currently providing.

  • Molecular monitoring of patients enrolled in clinical trials
  • Biomarker profiling by ELISA
  • Phenotypic and functional cellular assays to evaluate alloreactivity in a haplo transplant setting
  • Immunomonitoring studies

Process and Product Development for Cell Therapy

The TransLab has deep experience working on a diverse pipeline of projects covering the fields of cancer therapy, regenerative medicine and gene therapy. As of today, the TransLab has enabled 11 clinical trials. Some notable examples include:

  • Cancer therapy

    • Allogeneic unrelated umbilical cord blood, expanded with PGE2.
    • Epstein-Barr virus specific T cell therapy for nasopharyngeal carcinoma.
    • In vitro expanded autologous invariant natural killer T cells in cancer.
    • MART1-specific CTLs for melanoma.
    • Use of Regulatory T cells and IL-2 to treat steroid-refactory chronic graft verus host disease
  • Regenerative medicine: Cultivated Autologous Limbal Epithelial Cells (CALEC) use of lung derived mesenchymal progenitor cells for the treatment of advanced emphysema.
  • Gene Therapy: There are exciting new developments in the field of autologous transplant of genetically modified hematopoietic stem/progenitor CD34+ cells. The TransLab has played a key role in the development of clinical trials for several genetic deficiencies:
    • X-linked Severe Combined ImmunoDeficiency (X-SCID), Wiskott-Aldrich Syndrome (WAS), X-linked Chronic Granulomatous Disease (X-CGD), Childhood Cerebral Adrenoleukodystrophy (CCALD).

Executive committee

  • David Williams, MD, Director, Translational Research, Boston Children's Hospital
  • Jerome Ritz, MD, Executive Director, Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute
  • Leslie Silberstein, MD, Director, Transfusion Medicine, Boston Children's Hospital
  • Jon Aster, MD, PhD, Brigham and Women Hospital
  • Scott Pomeroy, MD, PhD, Neurologist-in-Chief of Boston Children’s Hospital


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