The Alpha-1 Project (TAP) today announced a commission to the University
of Massachusetts Medical School (UMMS) 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.”
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 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 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.
Copyright Business Wire 2014