Public Release:  Alpha-1 project commissions UMass Medical School to develop Alpha-1 protein antibody

PiZ antibody to speed testing of potential new therapies

University of Massachusetts Medical School

MIAMI, FL and WORCESTER, MA, (January 8, 2014) - Researchers at the University of Massachusetts Medical School (UMMS) have been commissioned by the Alpha-1 Project (TAP) to develop a PiZ antibody. The antibody will be used to track the presence of mutant alpha-1 PiZ protein in human blood serum, an essential tool in testing potential therapies for Alpha-1 Antitrypsin Deficiency (Alpha-1).

Alpha-1 is a genetic condition characterized by low or absent levels of alpha-1 protein in the blood. Normal alpha-1 protein protects the lungs against damage caused by neutrophil elastase. In Alpha-1, the mutant PiZ protein is misfolded and cannot be transported into the blood. This can lead to emphysema due to the loss of the alpha-1 protein's protective effects in the lung, and liver disease caused by the abnormal buildup of alpha-1 protein in the liver cells.

UMass Medical School scientists plan to optimize the antibody to track the PiZ protein in human macrophages (white blood cells) and liver tissue. The antibody could be used, along with a currently available antibody that tracks normal (PiM) protein, to test a dual-function viral strategy to both reduce the body's production of abnormal PiZ protein and increase production of the normal PiM protein. The contract also calls for the PiZ antibody to be made available to other researchers and industry who request it.

"The production and dissemination of the PiZ antibody is another example of our commitment to provide tools to researchers and industry in finding a cure for Alpha-1," said Jean-Marc Quach, executive director of TAP.

"Tremendous progress has been made over the last several years in the search for a breakthrough treatment for Alpha-1," said Terence R. Flotte, MD, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost, dean of the School of Medicine and professor of pediatrics and microbiology & physiological systems at UMMS. "While tools have been available to assess total amounts of alpha-1 and PiM protein, there has not been a specific assay to pick up only the mutant PiZ protein in human serum and liver tissue samples. As more therapeutic options aimed at down regulating or degrading PiZ become available, it is essential we have a way to easily and efficiently track its release and evaluate new potential treatments."

"This is an exciting step forward in seeking new therapies for Alpha-1," said John Walsh, president and CEO of the Alpha-1 Foundation and member of TAP's board of directors. "UMMS researchers are doing cutting-edge research on both reducing the amount of defective PiZ protein and increasing the amount of healthy PiM protein in the body. The PiZ antibody will speed their progress."

Christian Mueller, PhD, assistant professor of pediatrics and the Gene Therapy Center at UMMS said, "Recently we characterized an antibody clone that was able to differentiate between human PiZ and PiM protein in mice sera. By further characterizing this antibody specifically for human serum we can more readily detect the presence of the disease-causing PiZ protein circulating in the blood using standard diagnostic tools."

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About The Alpha-1 Project

Mission statement: The Alpha-1 Project will work with patients, academia, pharmaceutical and biotech companies in the relentless pursuit of cures and therapies for COPD and liver disease caused by Alpha-1 Antitrypsin Deficiency. For more information, visit http://www.thealpha-1project.com.

The Alpha-1 Project is a wholly-owned for-profit subsidiary of the Alpha-1 Foundation. For more information on the Foundation, visit http://www.alpha-1foundation.org.

About the University of Massachusetts Medical School

The University of Massachusetts Medical School (UMMS), one of five campuses of the University system, is comprised of the School of Medicine, the Graduate School of Biomedical Sciences, the Graduate School of Nursing, a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. Its mission is to advance the health of the people of the Commonwealth through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education. The Medical School attracts more than $240 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2006, UMMS's Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator and the Blais University Chair in Molecular Medicine, was awarded the Nobel Prize in Physiology or Medicine, along with colleague Andrew Z. Fire, PhD, of Stanford University, for their discoveries related to RNA interference (RNAi). The 2013 opening of the Albert Sherman Center ushered in a new era of biomedical research and education on campus. Designed to maximize collaboration across fields, the Sherman Center is home to scientists pursuing novel research in emerging scientific fields with the goal of translating new discoveries into innovative therapies for human diseases.

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