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BPDCN Center Research

  • Our team is focused on basic and clinical research to better understand BPDCN and improve outcomes. Because there is no standard of care for BPDCN, identifying novel therapies is especially important.

    Recent research done at Dana-Farber and collaborating institutions has helped identify new molecular targets for new therapies for BPDCN. Findings include:

    • All BPDCN cells overexpress CD123 (interleukin-3 receptor), a cell-surface protein. The BPDCN Center is one of eight sites nationally that offers a clinical trial of targeted immunotoxin SL-401, a novel agent that targets the IL-3 receptor (CD123).

    Studying the Genomics of BPDCN

    The research lab of Andrew Lane, MD, PhD is also studying the genomics of BPDCN and modeling how specific somatic alterations (such as mutations in the DNA of BPDCN cells) contribute to dendritic cell transformation. Researchers recently discovered that BPDCN is highly and uniquely dependent on the BCL-2 protein for survival. BCL-2 is a "pro-survival" protein that enables cancer cells to escape the "pro-death" signals that normally command abnormal cells to self-destruct through apoptosis, a natural quality-control process in the body. Further, researchers confirmed that BPDCN is very sensitive to venetoclax, a BCL2 inhibitor developed at Dana-Farber. Two patients in a pilot study with relapsed disease responded to venetoclax. An upcoming clinical trial will evaluate venetoclax in newly diagnosed BPDCN patients. This research was reported in Cancer Discovery.

    Lane’s lab research also led to the discovery that the reason why BPDCN is more common in males than females (~3:1 ratio) may be related to specific mutations on the X chromosome associated with the disease.

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  • Andrew Lane, MD, PhD, Shares Updates on Treating BPDCN
    Dr. Lane recaps the promising results presented at the ASH 2017 Annual Meeting from a phase 2 trial of SL-401 for blastic plasmacytoid dendritic cell neoplasm (BPDCN), as well as results from his lab exploring why some patients respond to SL-401 and others do not.