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Amyloidosis Program Research

  • Giada Bianchi, MD
  • Often considered a rare disease, there is so much to be discovered about the biology and treatment of AL Amyloidosis. An important goal of the Amyloidosis Program at Dana-Farber/Brigham and Women's Cancer Center is to build a strong pipeline of innovative clinical trials predicated on laboratory discoveries. Our Amyloidosis Program team is fully engaged and excited about the basic and clinical research now underway. We have great confidence in the potential for our research to inform novel, well tolerated treatments that we can offer as alternatives or a bridge to autologous stem cell transplantation, and opportunities to design and manage individualized care for each patient.

    Current research efforts include:

    • The Targeted Secretion Inhibition (TSI) for plasma cell disorder research study, funded through Harvard Catalyst, is focused on preclinical proof of concept that blocking antibody secretion in AL amyloidosis and other plasma cell disorders is therapeutically effective. While proteasome inhibitors have proven to be effective in treating AL amyloidosis, patients almost universally develop resistance to this therapy, and therapeutic options in this patient population are limited with no FDA-approved medications.

      Therefore, there is an important unmet need for more effective therapies. Based on encouraging pre-clinical data, we are hopeful that this effort will result in early-phase clinical trials in patients with AL amyloidosis in the next three to five years.
    • A second laboratory study underway tests strategies to overcome resistance to proteasome inhibitors in AL amyloidosis by targeting stress response pathways that are unregulated during proteasome inhibition.
    • A Phase III study comparing standard of care cyclophosphamide, bortezomib, and dexamethasone (CyBoRD) with or without the anti CD38 antibody Daratumumab in newly diagnosed AL amyloidosis patients is currently open at the Dana-Farber Cancer Institute.

    We anticipate one or two other clinical trials to open at the Dana-Farber in 2019-2020.

    A useful resource for information on amyloidosis research is the Amyloidosis Research Consortium, a nonprofit organization dedicated to the development of and access to new and innovative treatments for amyloidosis.