This Drug Could be the First Approved Treatment for a Rare Blood Cancer

According to a story from OncLive, the results of a Phase II trial for an experimental drug called SL-401 showed that the product could be a potentially effective treatment for a rare type of blood cancer called blastic plasmacytoid dendritic cell neoplasm. This form of cancer has limited treatment options, and most patients do not survive for very long.


Blastic plasmacytoid dendritic neoplasms (BPDCN) is a cancerous malignancy that affects plasmacytoid dendritic cells, a relatively rare type of immune cell that plays a role in fighting viral infections. What causes this disease is not known. It can occur in people of all ages but most commonly affects the elderly. It does not respond well to most treatments, and frequently recurs. Symptoms of BPDCN include swollen lymph nodes, skin lesions on the head, face, and chest, and an abnormal proliferation of diseased cells throughout the body. Unfortunately, there are no universal treatment regimens for BPDCN. Cytotoxic chemotherapy is a common choice, as are approaches for other blood cancers, but even if remission is achieved, BPDCN often relapses quickly and resists treatment. There are multiple experimental drugs being tested as therapies for the cancer. To learn more about BPDCN, click here.

Trial Results

The results of the SL-401 trial were encouraging. The drug was considered safe and achieved a 90 percent overall response rate. There are currently no treatments that specifically target BPDCN. SL-401 has a unique mechanism that targets CD123, a marker that is heavily expressed in almost all patients with this cancer. This allows the drug to target plasmacytoid dendritic cells specifically. Many of the patients in the study had already experienced disease relapse. 45 percent of relapsed/refractory patients went on to get stem cell transplants, another procedure which can prolong survival even further.

Hidden Capability

So far, SL-401 has only been tested as a single-agent therapy, and the drug could have even greater potential if used in combination with something else. Hopefully the drug will continue to show its effectiveness in future trials, and more research could reveal its capability as part of a combination treatment. BPDCN patients should have more hope than ever before that a new treatment could be just around the corner.

Check out the original trial study here.


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