Light chain amyloidosis (AL amyloidosis) is an incurable plasma cell neoplasm and the most common of a group of more than 30 rare conditions called amyloidoses. In this disease, amyloid, an abnormal protein, is deposited in the extracellular spaces of various organs, damaging them and preventing them from functioning properly. Lack of appropriate treatment leads to failure of the affected organs - usually the heart or kidneys - and death. Both the diagnosis and early implementation of treatment for AL amyloidosis is a major challenge for clinicians.
The goal of the STARLIGHT project, which has received funding, is to conduct a clinical trial in 20 patients with untreated AL amyloidosis to evaluate the experimental combination of granulocyte-macrophage colony-stimulating factor (GM-CSF) with the recently registered immunochemotherapy regimen D-VCD, but not yet reimbursed in Poland. Such treatment potentially offers hope for improving the prognosis of patients with AL amyloidosis who currently do not improve with current treatments.
In addition, the project will develop two advanced and previously unavailable in Poland methods of amyloid typing: using laser microdissection and mass spectrometry (MS) and immunoelectron microscopy (IEM). The development and implementation of these methods into routine clinical practice will introduce a modern standard for the diagnosis of amyloidosis in Poland.
The STARLIGHT study project was prepared by a team led by Prof. Krzysztof Jamroziak from the Department of Hematology, Transplantology and Internal Medicine UCC MUW and submitted to the competition as part of the Consortium formed by the Medical University of Warsaw and the National Oncology Institute of Maria Sklodowska-Curie - National Research Institute in Warsaw.