A novel treatment for MDS patients in need of frequent blood transfusions

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Imetelstat has achieved meaningful and durable transfusion independence in clinical trials

The innovative drug  Imetelstat has been trialed on patients with lower-risk MDS who are RBC (Red Blood Cells) transfusion dependent and have experienced relapse or not been helped by erythropoiesis-stimulating agent (ESA).

This group of patients does not have many treatment options at the moment.

In this press briefing, recorded at the 24th Congress of the European Hematology Association (EHA) 2019, held in Amsterdam, Netherlands, Pierre Fenaux of St Louis Hospital, Paris, France, presents data on the use of Imetelstat in lower risk myelodysplastic syndrome (MDS) patients.

How does Imetelstat work? The science behind it

Scientists have observed that patients with MDS have a high telomerase activity in their stem cells. Telomerase is a naturally occurring enzyme, and its activity prevents cells from dying. Telomerase is repressed in most normal cells, preventing uncontrolled proliferation. In cancer cells, however, this enzyme is active.

Imetelstat, is a "telomerase inhibitor" and targets cells with active telomerase.

The study has reported that Imetelstat shows efficacy to control the enzyme and it's safe to use. It also reports biomarker data for patients with low-risk MDS who are red blood cells transfusion dependent and who were relapsed/refractory to erythropoiesis-stimulating agents.

About the study

The study is a two-part phase II/III study.

The primary endpoint (the main result that is measured at the end of a study to see if a given treatment worked) was 8-week red blood cells transfusion independence rate.

Key secondary end points were:

  • 24-week red blood cells transfusion independence rate
  • transfusion independence duration
  • haematologic improvement-erythroid

Of 57 patients enrolled and treated (overall population), 38 were non-del(5q) and hypomethylating agent and lenalidomide naïve (subset population).

Encouraging results from phase II of the study

A Phase II trial is still early days in terms of clinical trials – to read more about how this trial progresses – see this page at ClinicalTrials.gov

  1. The 8- and 24-week red blood cells transfusion independence rate in the overall population were 37% and 23%, respectively, with a median transfusion independence duration of 65 weeks.
  2. In the subset population, 8- and 24-week red blood cells transfusion independence rates were 42% and 29%, respectively, with a median transfusion independence duration of 86 weeks.
  3. Eight-week transfusion independence rate was observed across all subgroups evaluated.
  4. Cytogenetic and mutational data revealed a reduction of the malignant clones, suggesting disease modification activity.
  5. The most common adverse events were cytopenias, typically reversible within 4 weeks.

In conclusion, Imetelstat treatment results in a meaningful, durable transfusion independence rate across a broad range of heavily transfused patients with Low-Risk MDS who are ineligible for or relapsed/refractory to erythropoiesis-stimulating agents. Biomarker analyses indicated effects on the mutant malignant clone.


David P. Steensma, MD1Pierre Fenaux, MD, PhD2Koen Van Eygen, MD3Azra Raza, MD4Valeria Santini, MD5Ulrich Germing, MD, PhD6Patricia Font, MD7Maria Diez-Campelo, MD, PhD8Sylvain Thepot, MD9Edo Vellenga, MD, PhD10Mrinal M. Patnaik, MBBS11Jun Ho Jang, MD12Helen Varsos, MS, RPh13Jacqueline Bussolari, PhD13Esther Rose, MD13Laurie Sherman, RN14Libo Sun, PhD14Ying Wan, MD, PhD14Souria Dougherty, BS, MBA14Fei Huang, PhD14Faye Feller, MD14Aleksandra Rizo, MD, PhD14; and Uwe Platzbecker, MD15

1Dana-Farber Cancer Institute, Boston, MA
2Hôpital Saint-Louis, Université Paris Diderot, Paris, France
3Algemeen Ziekenhuis Groeninge, Kortrijk, Belgium
4Columbia University Medical Center, New York, NY
5MDS Unit, AOU Careggi-University of Florence, Florence, Italy
6Klinik für Hämatologie, Onkologie and Klinische lmmunologie, Universitätsklinik Düsseldorf, Heinrich-Heine-Universität, Düsseldorf, Germany
7Department of Hematology, Hospital General Universitario Gregorio Marañon, Madrid, Spain
8Hematology Department, The University Hospital of Salamanca, Salamanca, Spain
9CHU Angers, Angers, France
10Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands
11Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN
12Department of Hematology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea

13Janssen Research & Development, Raritan, NJ
14Geron Corporation, Menlo Park, CA
15Department of Hematology and Cell Therapy, University Clinic Leipzig, Leipzig, Germany

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