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Core Facilities

Cell Manipulation

Scientific Impact and Current Projects

Scientific Impact and Current Projects

The CMCF currently supports more than 40 diverse active clinical research protocols, with a focus on translational research in the immune therapy of cancer. Many of these clinical trials and therapies could not have been carried out without the cell manufacturing and regulatory support provided by the Core.

Notable examples of therapies and protocols CMCF currently supports are shown below:

Expansion of Hematopoietic Stem Cells (HSCs)

  • DF/HCC Protocol 08-274 (BB IND 13721): A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using Ex Vivo dmPGE2 Expanded Umbilical Cord Blood Units.  In this study, one of the two cords is treated with dmPGE2 prior to transplantation. The untreated UBC provides a backup source of HSC in the event that treatment with dmPGE2 results in unexpected toxicity to HSC in one product. Immunologic reconstitution is also monitored and compared to previous patients who received similar preparative regimens and untreated double UCB transplants. The CMCF assisted in the IND submission, developed standard operating procedures (SOP) and carried out validations of the manufacturing process. The CMCF has completed processing of dmPGE2 treated cord blood cells for nine patients and the study is ongoing.

Principal Investigators: C. CutlerDFCI, K. BallenMGH, and D. AviganBIDMC.
Cancer Center Programs: Leukemia and Cancer Immunology.
IND Sponsors: L. ZonCHB and Fate Therapeutics, San Diego, CA.
Funding Source: NIH-NHLBI.

Transduction of hematopoietic stem cell grafts for clinical use

  • Gene transfer for SCID-X1: A multi-institutional phase I/II clinical trial of somatic gene therapy for patients with SCID-X1 using bone marrow derived CD34+ cells transduced ex-vivo with a novel gibbon ape leukemia (GALV)-virus, pseudotyped gammaretroviral vector encoding the human common cytokine receptor gamma chain (gc). This clinical trial is performed in collaboration with two other sites in the United States and two sites in Europe.

Principal Investigators: Luigi Notarangelo, MD; David Williams, MD; Jerome Ritz, MD. 

  • Gene Therapy for patients with Wiskott Aldrich Syndrome (WAS): An open labelled, non-randomized, single center, pilot and feasibility, single arm cohort study involving a single infusion of autologous CD34+ cells transduced with the Lentiviral vector containing the human WAS protein gene (w1.6_hWASP_WPRE (VSVg)) in up to 5 patients with WAS. The CMCF prepared the cellular components for gene transfer using ex vivo culture and viral vector transduction.

Principal Investigator: Sung-Yun Pai, MD
Sponsor: David A. Williams, MD
Coordinating Center: Children’s Hospital Boston

Manufacturing of biochemically engineered implantable tumor vaccines

  • WDVAX melanoma protocol (IND pending): A Phase I Trial of a Dendritic Cell Activating Scaffold Vaccine (WDVAX) Incorporating Autologous Melanoma Cell Lysate in Metastatic Melanoma Patients. The CMCF prepares the tumor vaccine, conducts release testing, and manufactures the vaccine delivery scaffold (WDVAX) according to standards provided by the Wyss Institute for Biologically Inspired Engineering. The CMCF also provided regulatory support in preparing the IND application and clinical trial protocol.

Principal Investigator: F. Stephen Hodi, MD
IND Sponsor:  Glenn Dranoff, MD
Coordinating Center: Dana-Farber Cancer Institute

Generation of GM-CSF secreting autologous tumor cell vaccines for banking and clinical use

  • GVAX for MDS/AML:  DF/HCC 12-217-BB IND 7248 - A Randomized Placebo-controlled Phase II Trial of Irradiated, Adenovirus Vector Transfected GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) Versus Placebo Vaccination in Patients with Advanced MDS/AML After Allogeneic Hematopoietic Stem Cell Transplantation.

Principal Investigator: Vincent Ho, MD
IND Sponsor: Glenn Dranoff, MD
Coordinating Center: Dana-Farber Cancer Institute

  • K562 Cell Lines: Production of a Working Cell Bank (WCB) of GM-CSF Secreting K562 Cells and Production of a Master Cells Bank (MCB) and Working Cell Bank of K562-RGE Cells for use in Clinical Trials.