Investigational drug shows promising results in Phase II study of aggressive, often fatal blood disorder with no approved therapies

November 14, 2018

A Phase I/II study, led by investigators at The University of Texas MD Anderson Cancer Center, reports an investigational drug called tagraxofusp has demonstrated high response rates in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare but highly aggressive - and often fatal bone marrow and blood disorder - for which there are no existing approved therapies.

Findings from the study are being presented Dec. 3 at the 60th American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego by Naveen Pemmaraju, M.D., associate professor of Leukemia.

"This was the largest prospectively designed multi-center, multi-cycle clinical trial specifically dedicated to patients with BPDCN," said Pemmaraju. "We observed high response rates including an overall response rate of 90 percent among frontline-treated patients. "These findings give us hope for patients who have had no treatments specific to this disorder."

BPDCN patients historically have experienced poor outcomes and low response rates with a variety of cytotoxic chemotherapy approaches following diagnosis. While there are no approved therapies or standard of care, patients quite often receive chemotherapy approved for other blood cancers, and/or stem cell transplants, however the majority of patients are older or do not qualify for standard chemotherapy approaches.

Forty-five patients with a median age of 70 years were enrolled in the seven-site study which provided tagraxofusp as a first-line or relapsed/refractory treatment. Median follow up for the study was 13.8 months.

Patients are continuing to be enrolled for the next stage of the clinical trial which will ensure ongoing access to the drug. Tagraxofusp is a novel targeted therapy that targets CD-123, a cell surface receptor, expressed in BPDCN and other hematologic malignancies.

The drug is also being investigated in other clinical trials, including for patients with chronic myelomonocytic leukemia (CMML) and myelofibrosis. Tagraxofusp received a Breakthrough U.S. Food and Drug Administration therapy designation with a rolling biological license application submitted to the FDA.
-end-
Other MD Anderson researchers participating in the study included Marina Konopleva, M.D., Ph.D., and Hagop Kantarjian, M.D., both of the Department of Leukemia. Other participating institutions included Dana-Farber Cancer Institute, Boston; Stemline Therapeutics, New York; Moffitt Cancer Center, Tampa, Fla.; City of Hope National Medical Center, Duarte, Calif.; Ohio State University, Columbus; Winship Cancer Institute of Emory University, Atlanta; Duke University Medical Center, Durham, N.C.; Roswell Park Cancer Institute, Buffalo, N.Y.; S.C. Spence Consulting, Westwood, Mass.; and Boston University School of Medicine. The study was funded by Stemline Therapeutics.

University of Texas M. D. Anderson Cancer Center

Related Clinical Trial Articles from Brightsurf:

Clinical trial investigates gabapentin for alcohol use disorder
This randomized clinical trial investigated if gabapentin, a drug often used to treat nerve pain, would be useful in the treatment of patients with alcohol use disorder (problem drinking that becomes severe) and a history of alcohol withdrawal symptoms.

Electrical stimulation helps treat constipation in clinical trial
Electrical stimulation benefited women with constipation in a recent clinical trial published in Alimentary Pharmacology & Therapeutics.

Treatment of migraine pain in randomized clinical trial
Adults experiencing a migraine of moderate or severe severity took the drug ubrogepant or placebo and reported if after two hours they were free of pain and of their most bothersome migraine-associated symptom in this randomized clinical trial.

First entirely digital clinical trial encourages physical activity
As little as a daily ping on your phone can boost physical activity, researchers from the Stanford University School of Medicine and their collaborators report in a new study.

HIV vaccine nears clinical trial following new findings
A promising vaccine that clears an HIV-like virus from monkeys is closer to human testing after a new, weakened version of the vaccine has been shown to provide similar protection as its original version.

Sickle cell drug showing promise in clinical trial
An investigational drug for the treatment of sickle cell disease is showing early promise in clinical trials for impacting biomarkers of the disease in patients, reported UConn School of Medicine researchers.

Meditation goes digital in new clinical trial
Scientists at UC San Francisco have developed a personalized digital meditation training program that significantly improved attention and memory in healthy young adults -- a group already at the peak of brain health -- in just six weeks.

Could blockchain ensure integrity of clinical trial data?
UC San Francisco researchers have created a proof-of-concept method for ensuring the integrity of clinical trials data with blockchain.

Eating crickets can be good for your gut, according to new clinical trial
A new clinical trial shows that consuming crickets can help support the growth of beneficial gut bacteria and that eating crickets is not only safe at high doses but may also reduce inflammation in the body.

Idera Pharmaceuticals presents clinical data from the ILLUMINATE-204 trial of the combination of tilsotolimod and ipilimumab for anti-PD-1 refractory metastatic melanoma at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting
Idera Pharmaceuticals Inc., a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with rare cancers and rare diseases, announced results from the ongoing ILLUMINATE-204 trial investigating tilsotolimod, Idera's intratumorally-delivered Toll-like Receptor 9 agonist, in combination with ipilimumab (Yervoy®).

Read More: Clinical Trial News and Clinical Trial Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.