Promising Results from National Trial for Rare Blood Cancer Treatment

A recent clinical trial led by the South Australian Health and Medical Research Institute (SAHMRI) has shown encouraging early outcomes in treating chronic myelomonocytic leukemia (CMML), a rare and aggressive blood cancer. The trial explores a combination therapy involving the standard medication azacitidine and an investigational antibody known as Lenzilumab.

CMML poses significant treatment challenges due to its resistance to conventional therapies and its low survival rates. The current options often result in only short-term benefits for patients. The innovative approach investigates whether Lenzilumab, a drug developed in Australia, can improve these outcomes by targeting a key signaling molecule responsible for overproducing abnormal blood cells and inflammation.

Interim findings published in the journal "Blood" reveal that among 34 enrolled patients, 22 experienced notable improvements within a year of starting treatment. Blood tests demonstrated that harmful cells and inflammatory markers significantly decreased after three months, with continued improvements observed up to six months. Several patients have maintained their response for over a year, with some extending treatment beyond three years and at least one patient remaining in remission after four years.

Lead investigator Associate Professor Dan Thomas emphasized the significance of these results, stating, "Seeing this kind of response in such a tough-to-treat disease is extraordinary. Patients are not only improving initially but are maintaining their health over years, which is unprecedented."

Additionally, there is emerging evidence that this treatment strategy may benefit other related blood cancers such as acute myeloid leukemia (AML). In a notable case, a patient with resistant disease responded well when lenzilumab was added to their treatment, with cancerous cell levels dropping from 20% to about 7% in the bone marrow.

The early successes of this trial offer new hope for those affected by CMML and potentially other hematologic malignancies. Further research is underway to evaluate long-term outcomes and expand these promising results.

For more information, see the publication: Devendra Hiwase et al, "Durable Responses Observed in Chronic Myelomonocytic Leukemia Treated with Lenzilumab and Azacitidine," Blood (2024). Source: Medical Xpress.