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

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PUBLIC RELEASE DATE:

9-Jan-2014

Contact: Jim Fessenden james.fessenden@umassmed.edu 508-856-2000 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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Alpha-1 project commissions UMass Medical School to develop Alpha-1 protein antibody

Another Voice: Medical schools must also instruct on health care policy

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By Rahul Rekhi

Since its inception more than a century ago, modern medical education has undergone a series of quiet revolutions. Yet this comprehensive expansion in one critical area masks a relative neglect of another: Medical curricula today largely omit training on health policy.

The result? Even as todays medical students graduate with a deep scientific fluency, they leave all but illiterate when it comes to the health care system.

I can bear witness to this firsthand. The curriculum of Stanford Medical School, where I am a deferred first-year student, does not incorporate a single required course on health policy or the health care system across four years and 249 credits of training.

And this oversight comes with consequences. To illustrate, recent research in JAMA Internal Medicine found that fewer than half of medical students nationwide understand even the basic components of the Affordable Care Act.

On a systemic level, this illiteracy directly impedes our ability to institute meaningful health policy reforms that tackle such thorny issues as quality-based physician payments, comparative effectiveness guidelines or end-of-life care. Without willing and capable physician leaders to guide, implement and sustain such major shifts for the decades to come, reform efforts almost certainly will founder.

Consequently, efforts to rein in health care costs and improve patient outcomes must begin by modernizing medical curricula to incorporate health policy training.

For example, a national mandate that fundamental knowledge of health systems be a prerequisite for medical licensing would encourage medical schools to incorporate course work on basic principles of health policy and economics. This teaching, moreover, should be nonpartisan and nonideological, focusing instead on the nuts and bolts of health systems akin to what law or business school students learn about policymaking and institutional governance.

Furthermore, the advent of so-called massive open online courses, or MOOCs, means that financial concerns the costs of expanding medical curricula to encompass health care policy may be unwarranted. Online health policy courses, such as the one taught by physician/policymaker Ezekiel Emanuel at the University of Pennsylvania, could serve as a functional stand-in when a university lacks a department or set of instructors dedicated to health policy.

Whatever the medium, it is imperative that we install health policy as an integral part of the national medical curricula, lest we continue to churn out a generation of students who are ill-equipped to make sense of the challenges and changes to come.

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Another Voice: Medical schools must also instruct on health care policy

Medical students take mini-med school to SCSDB students

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Students from Edward Via College of Osteopathic Medicine (VCOM) present a "mini med school" training workshop Wednesday at the South Carolina School for the Deaf and the Blind in Spartanburg, Aryeona Freeman, center, practices CPR on a manikin with medical students Rosmery Victoria, left, and Lauren Hildebran, right, during the workshop.

A "mini-medical school" in Spartanburg Wednesday was a learning experience for students of both S.C. School for the Deaf and the Blind and Edward Via College of Osteopathic Medicine (VCOM).

Thirteen second-year medical students from VCOM visited the SCSDB campus, teaching hands-on presentations about the heart and other organs, bones, muscles and CPR.

About 60 middle and high school students attended the extracurricular program. The mini-med school was coordinated by VCOM student Matt Fenlason.

"You're going to get to touch a real lung, a real heart and brain," Fenlason told a group of students before the hands-on activities began, using sign language as he talked.

"Eeeewww," several students responded, but curiosity won out. The students donned rubber gloves and handled the organs (covered in a preservative plastic). They also took a pulse, listened to heartbeats with a stethoscope, pumped the chest of a CPR simulator and performed jumping jacks to learn how the heart rate increases during physical activity. Students crowded around the presentation tables and spoke or signed enthusiastically as they participated.

The presentations stressed the importance of a healthy lifestyle and even impressed some of the staff members when they saw the difference between a healthy lung and the lung of a smoker.

"This benefits any teenager," said SCSDB President Maggie Park, who stopped by to check out the presentation. "While other students may be able to hear this on the radio or watch TV, our students may not. Having an interpreter here helping them access the information makes a big difference."

Fenlason, of Colorado, grew up with sign language because his mother is a professional interpreter, and he is now a sign language interpreter at First Baptist Church in Spartanburg. He chose VCOM partly because it was near the location of the SCSDB, where he volunteers as a wrestling coach.

The VCOM students have conducted mini-med schools at Dorman and Spartanburg high schools, but this was the first time they visited SCSDB. Most of the medical students had to learn some sign language in order to be able to conduct the presentation. The VCOM students arrived at the campus two hours early to practice signing, said Cora Richardson, after-school program coordinator and resident life services director for SCSDB.

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Medical students take mini-med school to SCSDB students

Rafael Campo’s student physicians embrace poetry to hone art of healing

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JUDY WOODRUFF: Now, Jeffrey Brown continues his series with U.S. poet laureate Natasha Trethewey called "Where Poetry Lives," taking us to places where poetry and literature connect to everyday life.

In past stories, they visited a program for Alzheimer's patients in New York, and one in Detroit that encourages young students to write about themselves and their city.

Tonight, a different kind of connection, through the practice of medicine and healing.

JEFFREY BROWN: Outside Boston's Beth Israel Deaconess Hospital on a recent frigid morning, Natasha Trethewey met up with a former poetry student of hers from Emory University.

Do you remember her as a teacher?

SAMYUKTA MULLANGI, student at Harvard Medical School: Of course I do.

RELATED INFORMATION

Doctor/Poet Rafael Campo finds rhythm through a stethoscope

(LAUGHTER)

JEFFREY BROWN: And Natasha remembers Sam, Samyukta Mullangi, fondly as well.

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Rafael Campo's student physicians embrace poetry to hone art of healing

U of M med school warns of doc shortage

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Shortage on the way (UMN) Listen Medical school warns of residency crunch

Jan 10, 2014

A national training bottleneck threatens Minnesotas ability to fill increased demand for doctors in coming years, University of Minnesota medical school officials warn.

Stagnant federal and state funding has limited the number of residency positions where they can train. Unless the number of residencies increases Minnesota will be short a projected 2,000 physicians a decade from now, they say.

If you want to be able to control your workforce and have workforce available in the future, youve got to build your own, said Troy Taubenheim, director of the Metro Minnesota Council on Graduate Medical Education.

Tens of thousands of new patients are expected to flood hospitals in the state over the next decade many of them aging Baby Boomers and those newly insured under the Affordable Care Act, according to numbers provided by Taubenheim.

Meanwhile, close to half of the states physicians will be old enough to retire within a decade. If they do, Taubenheims figures indicate, under current conditions the state will be able to replace fewer than half of them.

Simply expanding medical school enrollment wont produce enough doctors to fill such gaps because of a training bottleneck, health officials say.

After students graduate from medical school, they cant practice until they receive several years of training in hospitals and clinics training known as a residency.

The problem is that the number of residencies is effectively capped by government funding.

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U of M med school warns of doc shortage

Mark Rienzi, of The Becket Fund for Religious Liberty, on "The Kelly File" 1/3/2014 – Video

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Mark Rienzi, of The Becket Fund for Religious Liberty, on "The Kelly File" 1/3/2014
Mark Rienzi, Senior Counsel for The Becket Fund for Religious Liberty, and Lead Counsel for the Little Sisters of the Poor, discusses HHS mandate challenge o...

By: SBPublicAffairs

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Mark Rienzi, of The Becket Fund for Religious Liberty, on "The Kelly File" 1/3/2014 - Video