Targeted drug for leukemia tested at Penn Medicine helps patients live longer

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The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the USFDA based on interim results of the clinical trial ADMIRAL according to an article in EurekAlert for Science News.


A total of 371 adult patients with relapsed or refractory (resistant to treatment) AML, and presenting with a mutation in the FLT3 gene, were included in the ADMIRAL trial. One arm of the trial, 247 patients, received the drug, gilteritinib, and 124 patients in the second arm were treated with chemotherapy.

It has been noted that patients diagnosed with FLT3 mutations who have relapsed and were then treated with chemotherapy exhibited a low response and poor survival. 

In order to reduce the risk of relapse, oncologists would often use aggressive chemotherapy and marrow transplant. Gilteritinib has been designed for this group that until now had no other recourse.

About the Fms-like Tyrosine Kinase 3 (FLT3) Gene

FLT3 is a gene that creates fms-like tyrosine kinase 3 (FLT3) found in a group of receptor tyrosine kinases. These receptors send signals into the cells that are responsible for major cellular events. Additional information about FLT3 is available here.

Mutated FLT3 genes have been discovered in approximately thirty percent of leukemia cells. They are generally found in non-cancerous bone marrow where they regulate blood cells. If however, the gene becomes mutated cancer will metastasize unless a drug such as gilteritinib is administered to halt FLT3’s progress.

There are two prevalent FLT3 mutations. The first is the aggressive FLT3 internal tandem duplication (ITD) which is known to cause frequent relapses. Even when treated with current drugs it has an overall survival of about four months.

The second mutation is found in the tyrosine kinase domain (TKD) that is spread out in 300 residues. This mutation has shown resistance to many of the FLT3 inhibitors. Information about FLT3 mutations is available here.

About Gilteritinib XOSPATA® Trial Results

Gilteritinib has been proven to inhibit activity in both ITD and TKD. Astellas Pharma, Inc. is the manufacturer of the drug which is registered under  the trade name of XOSPATA®.

A followup of overall survival found that 37% of FLT3 patients who were treated with gilteritinib were still alive at the one-year mark versus 16.7% of subjects who were treated with chemotherapy.

Results were similar when combining the rates of complete remission (no signs of cancer) or partial remission (incomplete blood count recovery). The gilteritinib cohort recorded 34% while the chemotherapy group recorded 15%.

Side effects were similar in both arms. They included febrile neutropenia (white blood count-related fever) 44%; anemia 34% and fever from various causes 39%.

Severe adverse events that were possibly caused by gilteritinib were diminished platelet count (24%), anemia (20%), and febrile neutropenia (15%). In addition, 11% discontinued as a result of various side effects.

Early Treatment May Increase Cure Rates


The side effects of gilteritinib were favorable, allowing the subjects to receive the drug as outpatients. Gilteritinib’s safety factor means that it can stabilize the disease, enable patients to undergo bone marrow transplant and also continue treatment post transplant.  This would reduce chances of a relapse.

The consensus is that using these well-tolerated agents at the onset of the disease could bring about cure rates for this very high-risk leukemia.



1-Apr-2019 Targeted drug for leukemia tested at Penn Medicine helps patients live longer Data presented at AACR Annual Meeting on recently approved drug that targets common mutation in AML University of Pennsylvania School of Medicine Print E-Mail
ATLANTA – An inhibitor drug that targets a specific mutation in relapsed or refractory acute myeloid leukemia (AML) helps patients live almost twice as long as those who receive chemotherapy.

Read the source article at EurekAlert! Science News

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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