Rare Disease Report

NT-proBNP Response a Valid Biomarker for AL Amyloidosis Studies

JULY 20, 2016
James Radke, PhD
The Amyloidosis Research Consortium has concluded that NT-proBNP response is a valid outcome measure for predicting survival in AL amyloidosis patients.
That conclusion is posted in the pivotal paper published in the journal Leukemia and can be accessed here.
AL amyloidosis is an ultra-rare, progressive and fatal disease characterized by the accumulation of abnormal, misfolded protein (amyloid) in various tissues and organs.
This amyloid protein builds up in the heart, kidneys, liver, soft tissue, and nervous system, resulting in multiorgan failure and death. Up to 70% AL amyloidosis patinets have accumulations of amyloid in cardiac tissue that will lead to cardiomyopathy and ultimately to death.
No therapies have been approved for the treatment of patients with AL amyloidosis.

Gold standard biomarker

In a press release, Giampaolo Merlini, MD, of the Amyloidosis Research and Treatment Center at the University of Pavia and IRCCS Policlinico San Mateo in Italy, and senior author of the paper said, “NT-proBNP has emerged as an analytically validated, gold standard biomarker for the determination of cardiovascular risk and disease in patients with AL amyloidosis."
Dr Merlini added, "In five independent studies in almost 1500 patients, NT-proBNP responses consistently reflect changes in cardiac function and predict survival in patients with AL amyloidosis after interventional treatment. Importantly, NT-proBNP predicts clinical outcome and survival independent of therapy type, treatment class or regimen."
The consortium is hopeful establishing NT-proBNP response as a valid biomarker will lead to more clinical trials and ultimately, a treatment for AL amyloidosis.

Raymond L. Comenzo, MD, a study author and Founding Director of the John Conant Davis Myeloma and Amyloid Program and Director of the Blood Bank and Stem Cell Processing Laboratory at Tufts Medical Center said, "A significant barrier to therapeutic development is posed by all diseases that are serious but rare, as traditional clinical trials with clinical endpoints such as survival and progression free survival are difficult, if not impossible to conduct."

Dr Comenzo added, "The amyloidosis expert community is in agreement that NT-proBNP should be used as a surrogate end point for survival in patients with AL amyloidosis with cardiac involvement."

Patients agree

Similar sentiments were expressed by patients. Isabelle Lousada, founder CEO of the Amyloidosis Research Consortium and an amyloidosis patient commented, "We believe that using NT-proBNP as a surrogate endpoint for survival represents a concrete, positive movement toward the rapid assessment of new therapeutics for AL amyloidosis and we are working with the FDA to validate NT-proBNP through the biomarker qualification program, to potentially save many lives."

For more information, visit www.arci.org

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