New "Theranostic" Drugs Diagnose and Treat

Acute lymphoblastic leukemia may be treated with new class of drugs

(RxWiki News) Acute lymphoblastic leukemia (ALL) is the most common type of childhood cancer, diagnosed in some 5,000 children every year in the United States.

A new class of drugs being developed may provide a level of precision therapy never before available.

Researchers are working on a novel approach to treating ALL with a new platform of drugs that both diagnose and provides targeted therapy.

These so-called "theranostic" agents would be the first step in delivering truly personalized care to youngsters with this dangerous form of leukemia.

"Talk to your oncologist about the latest therapies available."

These new therapies are being developed at Case Western Reserve University School of Medicine, and the team leader is Anthony J. Berdis, PhD, assistant professor of pharmacology. 

The discovery involves several components. The team is working with an enzyme - terminal deoxynucleotidyl transferase  (TdT)  -  that is seen in unnaturally high levels in 90 percent of children with ALL. Researchers have designed an agent that targets this enzyme. 

This is the therapy side of the equation.

Next, the researchers are able to evaluate the activities of two anti-leukemia molecules by tagging them with a special dye. Observing the behavior of these molecules will allow clinicians to change the dosages as needed for each individual patient.

So the effect of the medication is being evaluated so that optimal dosing can be determined.

"The dosages can be raised or reduced to personalize the therapy to the exact needs of the individual child," Berdis told dailyRx in a telephone interview.

Currently, it can take weeks or months to learn how well a patient is responding to a targeted therapy. With this treatment, clinicians will know the response in two or three days, according to Berdis.

"This combines the best of both worlds and represents a leap to more personalized treatment," Berdis says. "This has the potential to do a lot of good for the kids," he adds.

He acknowledges a great deal of work remains before such treatment may become available. He hopes to get the necessary funding for safety testing in animal models in next 12-24 months. That will then begin the clinical trial process.

This study was published online March 6, 2012 in ACS Chemical Biology.

This research was supported by the Developmental Therapeutics Program at the Case Comprehensive Cancer Center, a National Cancer Institute (NCI) - designated Comprehensive Cancer Center.

No conflicts of interest were reported. 

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Review Date: 
March 12, 2012