Since its conception, biopharmaceutical company Geron Corporation (“Geron”) has worked to develop first-in-class therapies for hematologic myeloid malignancies. More recently, Geron began evaluating imetelstat for patients with lower risk myelodysplastic syndromes (MDS). In a press release from December 10, 2020, Geron announced a milestone in its Phase 3 IMerge study: 50% enrollment.
Imetelstat
Developed and exclusively owned by Geron, imetelstat is a telomerase inhibitor designed for patients with hematologic cancers. The therapy works by prompting apoptosis, or programmed cell death, in cancerous cells. Currently, imetelstat received Fast Track designation for its treatment of refractory or treatment-resistant non-del(5q) MDS and intermediate-risk to high-risk myelofibrosis (MF). Geron presented current results from the IMerge Phase 2 clinical trial at the American Society of Hematology (ASH) Annual meeting under Abstract #658.
Now, Geron will continue evaluating imetelstat for MDS through a Phase 3 IMerge trial, as well as for MF through the Phase 3 IMpactMF study. Altogether, 170 patients with low-risk MDS will enroll. To participate in the trial, patients must have previously received treatment with an erythropoiesis stimulating agent (ESA); however, for these same patients, this ESA did not provide symptom relief or halt disease progression. Instead, all patients are transfusion-dependent.
Within the study, researchers will evaluate the safety, efficacy, and tolerability of imetelstat. First, researchers will test how many patients are no longer transfusion dependent following an 8-week (2 month) period. Next, researchers will analyze this same level of independence following a 24-week (5.5 month) period. Overall, Geron hopes to achieve full trial enrollment by the middle of 2021.
Myelodysplastic Syndromes (MDS)
Overall, myelodysplastic syndromes (MDS) consist of a group of progressive conditions which prevent healthy blood cells from being formed in bone marrow. There are five subtypes of MDS: refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia. In each case, blood cells either never leave the bone marrow or die early. In approximately half of all cases, MDS progresses to acute myeloid leukemia (AML).
An estimated 60,000 Americans have MDS. Altogether, an estimated 16,000 new diagnoses are made each year. Typically, MDS is more common in males than females. While it can impact people of all ages, MDS is more prevalent in those over 60 years old. While doctors are not sure what exactly causes MDS, it is thought to be a mixture of genetics and external triggers such as chemical exposure or chemotherapy. Symptoms include:
- Thrombocytopenia (low platelet count)
- Easy bruising and bleeding
- Neutropenia (low white blood cell count)
- Frequent infections
- Anemia (low red blood cell count)
- Fatigue
- Heart palpitations
- Difficulty breathing / shortness of breath
- Pallor (pale skin)
- Chest pain
- Petechiae, or small, red spots under the skin
- Exercise intolerance
