According to a story from ca.sports.yahoo.com, the biotechnology company Galecto, Inc. has announced encouraging findings from an intermediate assessment of its phase 2a clinical trial. This trial is evaluating the company’s investigational therapy GB2064 as a treatment for myelofibrosis, a rare type of bone marrow cancer. The data release indicated that the drug was able to reduce fibrosis, or scarring, that occurs in the bone marrow as part of the disease. Galecto is focused on developing innovative treatments for cancer and fibrosis.
About Myelofibrosis
Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.
A Promising New Treatment?
Fibrosis is a major component of the disease mechanism in the type of cancer and addressing this sign is critical for slowing or halting the progression of myelofibrosis. Four of the five patients that were deemed evaluable at the time of the intermediate assessment and received GB2064 for a minimum of six months saw a reduction of ≥1 grade in fibrosis in the bone marrow. This level of improvement suggests that the treatment could modify the course of the disease and impede its progression. Furthermore, these patients’ hemoglobin, white blood cell count, spleen volume, and other parameters were stable. None of them needed blood transfusion and two of the patients have decided to continue into the extension portion of the trial.
A total of sixteen patients have been dosed in the trial, with half of them having completed treatment or continuing to receive it; the other half halted treatment with GB2064 because of disease progression or adverse effects. However, none of the adverse events were considered serious.
Hopefully, this clinical trial will continue to proceed with positive results.
