New drug combo targets an aggressive blood cancer at lower cost

Print 15 September 2016
Oliver Worsley / Fierce Biotech

Patients with chronic myeloid leukemia (CML) face not only a life-threatening cancer but also the high cost of the current therapy. A team at the University of Texas has now shown a drug combo that might stop the cancer--while offering the drug at a more reasonable price tag.  

Michael Andreeff and Bing Carter, who headed up the research with their team at the University of Texas, published their findings in the journal Science Translational Medicine.

The treatment combined two independently established tyrosine kinase inhibitors (TKIs), BCR-ABL and venetoclax. BCR-ABL inhibitors are the current standard of care for patients with CML. Despite their success in most patients, residual cancer stem cells can mount a second attack.

The cost of TKI treatment is skyward of $100,000 (USD), given the duration of the treatment to prevent relapse--a number that is unthinkable for the majority of patients. "Long-term treatment with TKIs comes at a high cost, both in terms of side effects and financially," said Carter in a release. "Worldwide, most CML patients cannot afford the extraordinary expenses associated with TKI-based therapy. And, unfortunately, for patients who progress to blast crisis, there are no meaningful treatments and survival is counted in weeks or months."

Combining the action of BCR-ABL and venetoclax--a drug that specifically targets BCL-2 involved in the “cell death” pathway of cells including cancer cells--the researchers were able to improve the outcome in a mouse model of CML, with an equivalent degree of CML as seen in patients with “end-stage” CML.

"It is believed that TKIs do not eliminate residual stem cells because they are not dependent on BCR-ABL signaling," said Carter. "Hence cures of CML with TKIs are rare."

Carter and her team believe this combination could be used in patients with CML, importantly targeting residual cancer stem cells, negating the need for long-term expensive TKI therapy.

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