RxPG News Feed for RxPG News

Medical Research Health Special Topics World
 Asian Health
 Food & Nutrition
 Men's Health
 Mental Health
 Occupational Health
 Public Health
 Sleep Hygiene
 Women's Health
 Canada Healthcare
 China Healthcare
 India Healthcare
 New Zealand
 South Africa
 World Healthcare
 Latest Research
 Alternative Medicine
 Clinical Trials
 Infectious Diseases
 Sports Medicine
   Medical News
 Awards & Prizes
   Special Topics
 Odd Medical News

Last Updated: Feb 19, 2013 - 1:22:36 AM
Research Article
Latest Research Channel

subscribe to Latest Research newsletter
Latest Research

   EMAIL   |   PRINT
More than a third of high-risk leukemia patients respond to an experimental new drug

Dec 9, 2012 - 5:00:00 AM
Long-term survival from the therapy is still unknown, Levis says, but of the group of 137 patients, 47 (34 percent) were able to receive a transplant after responding to quizartinib. Some of these patients have survived two years after treatment with no disease recurrence.

[RxPG] A new drug for patients with acute myeloid leukemia (AML) marked by a specific type of genetic mutation has shown surprising promise in a Phase II clinical trial. In more than a third of participants, the leukemia was completely cleared from the bone marrow, and as a result, many of these patients were able to undergo potentially curative bone marrow transplants, according to investigators at the Johns Hopkins Kimmel Cancer Center and nine other academic medical centers around the world. Many of the participants who did well with the new drug, quizartinib or AC220, had failed to respond to prior therapies.

We can put two-thirds to three-quarters of adults with AML into remission with chemotherapy, but there's a 50 percent chance of the disease coming back, which usually ends up being fatal, says Mark Levis, M.D., Ph.D., lead investigator on the study and associate professor of oncology and medicine at Johns Hopkins. Many patients in this trial were able to go on to receive a potentially life-saving bone marrow transplant. It caught us by surprise how well it works, he adds.

A report on the study is expected to be presented December 9 during a press briefing at the American Society of Hematology's annual meeting in Atlanta.

For the clinical trial, researchers enrolled 137 AML patients, the majority of whom carried a mutation in a gene called FLT3-ITD within their leukemia cells. The FLT3 gene produces an enzyme that signals bone marrow stem cells to divide and replenish. In about a quarter of patients with AML, the disease mutates FLT3 so that the enzyme stays on permanently, causing rapid growth of leukemia cells and making the condition harder to treat.

A FLT3-ITD mutation tells us that, typically, patients will need very intensive chemotherapy just to achieve a remission, and then the disease will regrow quickly, Levis says. So, we have learned to try to perform a bone marrow transplant soon after we get the patient into remission, before the cancer relapses.Quizartinib, which blocks the FLT3 enzyme, and is available in liquid oral form, is so potent that it typically starts working in just two days, Levis says, though it may take up to 60 days to completely eliminate AML cells from the bone marrow.

At Johns Hopkins and nine other centers, the 137 AML recruited patients received quizartinib at a starting dose of 90 mg/day for women and 135 mg/day for men, and were treated continuously during 28-day cycles. Study participants either had relapsed, did not respond to second-line chemotherapy or had relapsed following hematopoietic stem cell transplantation.

Forty-four percent (44) of the 99 participants with a FLT3-ITD mutation experienced some form of complete remission, typically one in which the leukemia was cleared from the bone marrow, but the patient still needed blood and platelet transfusions. Thirty-four percent (13) of the 38 participants in whom the FLT3-ITD mutation was not detectable experienced this type of response.

The most common side effects with quizartinib were nausea (38 percent), anemia (29 percent), QT prolongation (an abnormality found on an EKG; 26 percent), vomiting (26 percent), febrile neutropenia (development of fever in someone with low white blood cell count; 25 percent), diarrhea (20 percent), and fatigue (20 percent). Fourteen patients (10 percent) experienced side effects severe enough to discontinue taking the drug. Investigators have been testing lower doses of the medication since the trial to reduce side effects, Levis says.

Long-term survival from the therapy is still unknown, Levis says, but of the group of 137 patients, 47 (34 percent) were able to receive a transplant after responding to quizartinib. Some of these patients have survived two years after treatment with no disease recurrence.

Based on results, the company that makes the drug, Ambit Biosciences, is planning larger Phase III trials, in which patients who have the FLT3 mutation will receive either quizartinib or chemotherapy after random grouping. Meanwhile, Levis and other physicians are continuing to study the drug in a clinical trial testing lower doses.

Related Latest Research News
Bone loss associated with increased production of ROS
Sound preconditioning prevents ototoxic drug-induced hearing loss in mice
Crystal methamphetamine use by street youth increases risk of injecting drugs
Johns Hopkins-led study shows increased life expectancy among family caregivers
Moderate to severe psoriasis linked to chronic kidney disease, say experts
Licensing deal marks coming of age for University of Washington, University of Alabama-Birmingham
Simple blood or urine test to identify blinding disease
Physician job satisfaction driven by quality of patient care
Book explores undiscovered economics of everyday life
Gene and stem cell therapy combination could aid wound healing

Subscribe to Latest Research Newsletter

Enter your email address:

For any corrections of factual information, to contact the editors or to send any medical news or health news press releases, use feedback form

Top of Page

Contact us

RxPG Online



    Full Text RSS

© All rights reserved by RxPG Medical Solutions Private Limited (India)