Published: Sep 14, 2014
By John Gever, Managing Editor, MedPage Today
BOSTON — A biologic drug targeting the granulocyte-macrophage colony stimulating factor (GM-CSF) was safe and showed hints of activity against brain lesions in multiple sclerosis (MS) patients in a phase I trial, researchers said here.
In patients receiving the highest dose of the monoclonal antibody drug, MOR103, declines in medians for both T1 hypointense and T2 lesion volumes on MRI were seen after 20 weeks in the double-blind, placebo-controlled study, reported Cris Constantinescu, MD, PhD, of the University of Nottingham in England, and colleagues.
Only one serious treatment-emergent adverse event was seen among the 25 patients receiving the investigational agent — an exacerbation of MS in a member of the middle-dosage group.
The trial results point toward a clinical impact from years of research into the role of GM-CSF in MS and other autoimmune conditions, he suggested to attendees at the European Committee for Treatment and Research in Multiple Sclerosis annual meeting, held jointly this year with its North American counterpart.
As the name suggests, GM-CSF is a growth factor for important immune system components. Although neither granulocytes nor macrophages are directly involved in MS pathology, the extensive cross-talk between different classes of immune cells means that modulation of one type can lead to wide-ranging effects elsewhere in the immune system.
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