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What We Know

Therapeutic advancement in higher-risk MDS has been limited in the last 15 years, leaving patients and physicians ill-equipped for far too long1,2

  • Hematopoietic stem cell transplantation (HSCT) is the only potentially curative option for patients with higher-risk MDS, but advanced age at diagnosis and comorbidities prevent a majority of patients with higher-risk MDS from receiving HSCT2

Hypomethylating agents (HMAs) are the current standard of care, but they fall short of generating complete responses in a majority of higher-risk patients2-4

  • Response rates to HMAs are limited. In a randomized, open-label, phase III study, ~30% of patients with higher-risk MDS achieved either a complete response or partial response, and ~50% of patients with higher-risk MDS achieved hematologic improvement. The primary endpoint, median overall survival, was ~2 years4
  • Real-world data suggest that median overall survival with HMAs in higher-risk MDS is shorter than the best clinical trial overall survival outcome reported, which is ~2 years4,5
  • A lack of durable responses with current HMAs for higher-risk MDS makes relapse, progression to AML, and high transfusion burden likely1,2,6

What We’re focused on

Across certain tumor types, immunotherapy has helped transform treatment for patients with cancer

  • T-cell checkpoint inhibitors have paved the way in immunotherapy, working in the adaptive immune system to effectively treat a number of solid tumors7
  • However, immunotherapies targeting the adaptive immune system have only created meaningful outcomes in limited types of liquid tumors so far7
  • A new class of immunotherapy that looks beyond traditional immune checkpoint inhibitors is needed

The innate immune system, including the activity of macrophages and phagocytic signals, may offer important clues in the investigation into higher-risk MDS7,8

Is there more of the immune system left to unlock? Gilead is committed to following the thread.

Be sure to get updates from Gilead as the case evolves

Get Updates
  1. Steensma DP. Blood Cancer J. 2018;8(5):47. doi:10.1038/s41408-018-0085-4
  2. Platzbecker U. Blood. 2019;133(10):1096-1107. doi:10.1182/blood-2018-10-844696
  3. Silverman LR, McKenzie DR, Peterson BL, et al. J Clin Oncol. 2006;24(24):3895-3903. doi:10.1200/JCO.2005.05.4346
  4. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Lancet Oncol. 2009;10(3):223-232. doi:10.1016/S1470-2045(09)70003-8
  5. Zeidan AM, Stahl M, Sekeres MA, et al. Cancer. 2017;123(19):3662-3672. doi:10.1002/cncr.30903
  6. Bell JA, Galaznik A, Blazer M, et al. Leuk Lymphoma. 2019;60(1):49-59. doi:10.1080/10428194.2018.1464155
  7. Salik B, Smyth MJ, Nakamura K. J Hematol Oncol. 2020;13(1):111. doi:10.1186/s13045-020-00947-6
  8. Chao MP, Takimoto CH, Feng DD, et al. Front Oncol. 2020;9:1380. doi:10.3389/fonc.2019.01380