Midostaurin is a multi-target protein kinase inhibitor

Protein kinase inhibitor

A protein kinase inhibitor is a type of enzyme inhibitor that specifically blocks the action of one or more protein kinases. Hence, they can be subdivided or characterised by the amino acids whose phosphorylation is inhibited:...

 being investigated for the treatment of acute myeloid leukemia

Acute myeloid leukemia

Acute myeloid leukemia , also known as acute myelogenous leukemia, is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. AML is the most common acute...

 (AML) and myelodysplastic syndrome

Myelodysplastic syndrome

The myelodysplastic syndromes are a diverse collection of hematological medical conditions that involve ineffective production of the myeloid class of blood cells....

 (MDS). It is a semi-synthetic derivative of staurosporine

Staurosporine

Staurosporine is a natural product originally isolated in 1977 from the bacterium Streptomyces staurosporeus.It was the first of over 50 alkaloids to be isolated with this type of bis-indole chemical structure...

, an alkaloid

Alkaloid

Alkaloids are a group of naturally occurring chemical compounds that contain mostly basic nitrogen atoms. This group also includes some related compounds with neutral and even weakly acidic properties. Also some synthetic compounds of similar structure are attributed to alkaloids...

 from the bacterium Streptomyces

Streptomyces

Streptomyces is the largest genus of Actinobacteria and the type genus of the family Streptomycetaceae. Over 500 species of Streptomyces bacteria have been described. As with the other Actinobacteria, streptomycetes are gram-positive, and have genomes with high guanine and cytosine content...

 staurosporeus, and is active in patients with mutations of CD135

CD135

Cluster of differentiation antigen 135 also known as Fms-like tyrosine kinase 3 or receptor-type tyrosine-protein kinase FLT3 is a protein that in humans is encoded by the FLT3 gene...

 (FMS-like tyrosine kinase 3 receptor).

After successful Phase II clinical trial

Clinical trial

Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...

s, a Phase III trial for AML has started in 2008. It is testing midostaurin in combination with daunorubicin

Daunorubicin

Daunorubicin or daunomycin is chemotherapeutic of the anthracycline family that is given as a treatment for some types of cancer. It is most commonly used to treat specific types of leukaemia...

 and cytarabine

Cytarabine

Cytarabine, or cytosine arabinoside, is a chemotherapy agent used mainly in the treatment of cancers of white blood cells such as acute myeloid leukemia and non-Hodgkin lymphoma. It is also known as Ara-C...

. In another trial, the substance has proven ineffective in metastatic melanoma

Melanoma

Melanoma is a malignant tumor of melanocytes. Melanocytes are cells that produce the dark pigment, melanin, which is responsible for the color of skin. They predominantly occur in skin, but are also found in other parts of the body, including the bowel and the eye...

.