Summary

Acute myeloid leukemia is a challenging condition in elderly patients because conventional chemotherapy often causes severe side effects. A new drug called sapacitabine, which can be given orally, was tolerable and efficacious in a clinical trial involving a small group of patients over 70.

The way is now clear for a larger trial which could result in the drug reaching the clinic in the next few years.

Introduction

Acute myeloid leukemia (AML) and myelodysplastic syndrome are blood cancers that affect older people. Treatment can be a challenge because age, in itself, or co-existing illness seem to lead to unacceptable side effects and even increased mortality with existing chemotherapy agents. Sapacitabine, an oral drug, acts by interfering with the cancer cell DNA and also its cell cycle. It is being developed as a treatment for AML and has reached the stage of Phase II clinical trials.

What was done

A total of 60 patients aged 70 years or more with untreated or first relapse AML went into one of three dosing schedules with sapacitabine. They were joined by 31 patients with a related condition called myelodysplastic syndrome (MDS). The investigating team was based at the prestigious Texas M.D. Anderson Cancer Center.

What was found

In a three day dosing schedule, the overall response rate in AML was 35% and 25% of this group experienced a complete remission. In the MDS group, seven patients experienced a complete remission.

What this study means

It’s important to know that it is still early days for sapacitabine. Drugs that get through Phase II often fail the next hurdle of the Phase III trial. But the results from this study look good and it is interesting that elderly patients were involved – unusual in a clinical trial. An oral drug offers huge benefit for patients with cancer in terms of convenience and quality of life and sapacitabine has the added advantage of having two different modes of action.

Source

Garcia-Manero G et al A randomized Phase II study of sapacitabine, an oral nucleoside analogue, in elderly patients with AML previously untreated or previously treated MDS Journal of Clinical Oncology 27:15s 2009 (suppl; abstr 7021)