Human Ebola vaccine trial to start

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A highly anticipated test of an experimental Ebola vaccine will begin this week at the National Institutes of Health, amid mounting anxiety about the spread of the deadly virus in West Africa.

After an expedited review by the U.S. Food and Drug Administration, researchers were given the green light to begin what's called a human safety trial, said Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases (NIAID).

It will be the first test of this type of Ebola vaccine in humans.

The experimental vaccine, developed by the pharmaceutical company GlaxoSmithKline and the NIAID, will first be given to three healthy human volunteers to see if they suffer any adverse effects. If deemed safe, it will then be given to another small group of volunteers, aged 18 to 50, to see if it produces a strong immune response to the virus. All will be monitored closely for side effects.

The vaccine will be administered to volunteers by an injection in the deltoid muscle of their arm, first in a lower dose, then later in a higher dose after the safety of the vaccine has been determined.

Some of the preclinical studies that are normally done on these types of vaccines were waived by the FDA during the expedited review, Fauci said, so "we want to take extra special care that we go slowly with the dosing."

The vaccine did extremely well in earlier trials with chimpanzees, Fauci said. He noted that the method being used to prompt an immune response to Ebola cannot cause a healthy individual to become infected with the virus.

Still, he said, "I have been fooled enough in my many years of experience... you really can't predict what you will see (in humans)."

According to the NIH, the vaccine will also be tested on healthy volunteers in the United Kingdom, Gambia and Mali, once details are finalized with health officials in those countries.

Trials cannot currently be done in the four countries affected by the recent outbreak -- Guinea, Sierra Leone, Liberia and Nigeria -- because the existing health care infrastructure wouldn't support them, Fauci said. Gambia and Mali were selected because the NIH has "long-standing collaborative relationships" with researchers in those countries.

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Human Ebola vaccine trial to start

Human trial of experimental Ebola vaccine begins this week

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NEWS

Posted YESTERDAY, 5:41 AM Updated YESTERDAY, 5:42 AM

A highly anticipated test of an experimental Ebola vaccine will begin this week at the National Institutes of Health, amid mounting anxiety about the spread of the deadly virus in West Africa.

After an expedited review by the U.S. Food and Drug Administration, researchers were given the green light to begin what's called a human safety trial, said Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases.

It will be the first test of this type of Ebola vaccine in humans.

The experimental vaccine, developed by the pharmaceutical company GlaxoSmithKline and the NIAID, will first be given to three healthy human volunteers to see if they suffer any adverse effects. If deemed safe, it will then be given to another small group of volunteers, aged 18 to 50, to see if it produces a strong immune response to the virus. All will be monitored closely for side effects.

The vaccine will be administered to volunteers by an injection in the deltoid muscle of their arm, first in a lower dose, then later in a higher dose after the safety of the vaccine has been determined.

Some of the preclinical studies that are normally done on these types of vaccines were waived by the FDA during the expedited review, Fauci said, so "we want to take extra special care that we go slowly with the dosing."

The vaccine did extremely well in earlier trials with chimpanzees, Fauci said. He noted that the method being used to prompt an immune response to Ebola cannot cause a healthy individual to become infected with the virus.

Still, he said, "I have been fooled enough in my many years of experience... you really can't predict what you will see (in humans)."

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Human trial of experimental Ebola vaccine begins this week

HD Video Feedback Hannah Lagerwey in Peru Cusco Health care and Children Programs – Video

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HD Video Feedback Hannah Lagerwey in Peru Cusco Health care and Children Programs
HD Video Feedback Hannah Lagerwey in Peru Cusco Health care and Children Programs 8 Weeks July 1st to August 26, 2014 https://www.abroaderview.org.

By: A Broader View Volunteers

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HD Video Feedback Hannah Lagerwey in Peru Cusco Health care and Children Programs - Video

Health care spending growth is slow but rising

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Health care costs have been growing at a historically low rate but are poised to increase again starting this year, according to a Centers for Medicare and Medicaid Services report out today.

Though growth over the next nine years is likely to be lower than it was from the 1990s through most of the 2000s, it will average 6% from 2015 to 2023, up from the 5.8% CMS predicted last year.

That's below the 7.2% increase in health care spending from 1990 through 2008.

The growth in health spending for 2013 is estimated to have slowed to 3.6%, but the rate will rise to 5.6% for 2014, the report says.

The leading contributors to the increased growth are more people being covered through the Affordable Care Act, expected economic growth and the aging population, CMS says. Some of the impact on the older populations is tempered by the fact that Baby Boomers are healthier than many other new enrollees in Medicare as most are coming from private insurance, the study says.

Since the 2007-08 recession, economic recovery has been weaker than anticipated, which has tamped down the rate of health care spending.

Every year, CMS releases an analysis of how Americans are likely to spend their health care dollars in the coming decade.

Sean Keehan, a study co-author, says increasingly high health care cost-sharing by consumers helps to slow growth. When people, rather than their insurers and employers, pay more out of pocket for their health care, they simply choose to have fewer procedures.

CMS Administrator Marilyn Tavenner says the report shows "health care costs are increasing at a slower rate thanks to the Affordable Care Act."

The number of uninsured is likely to decline by nearly half from 45 million in 2012 to 23 million by 2023 as a result of the coverage expansions associated with the Affordable Care Act, the CMS report says.

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Health care spending growth is slow but rising

Health Care Costs About to Rise Again

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WASHINGTON (AP) The nation's respite from troublesome health care inflation is ending, the government said Wednesday in a report that renews a crucial budget challenge for lawmakers, taxpayers, businesses and patients.

Economic recovery, an aging society, and more people insured under the new health care law are driving the long-term trend.

Projections by nonpartisan experts with the Health and Human Services department indicate the pace of health care spending will pick up starting this year and beyond. The introduction of expensive new drugs for the liver-wasting disease hepatitis C also contributes to the speed-up in the short run.

The report from the Office of the Actuary projects that spending will grow by an average of 6 percent a year from 2015-2023. That's a notable acceleration after five consecutive years, through 2013, of annual growth below 4 percent.

Although the coming bout of health-cost inflation is not expected to be as aggressive as in the 1980s and 1990s, it will still pose a dilemma for President Barack Obama's successor. Long term, much of the growth comes from Medicare and Medicaid, two giant government programs now covering more than 100 million people.

The United States is expected to spend more than $3 trillion on health care this year, far above any other economically advanced country. Yet Americans are not appreciably healthier, and much what they spend appears to go for tests and treatments of questionable value. Fraud also siphons off tens of billions of dollars a year.

Because health care spending is so high, shifts of a couple of percentage points have significant economic consequences. Health care inflation has recently been in line with overall economic growth, keeping things manageable.

As spending rebounds, health care again will start consuming a growing share of the economic pie, crowding out other worthy priorities. From 17.2 percent of the economy in 2012, health care is expected to grow to a 19.3 percent share by 2023, the report said.

"The period in which health care has accounted for a stable share of economic output is expected to end in 2014, primarily because of the (health care law's) coverage expansions," it concluded.

Yet if Obama's Affordable Care Act is an immediate trigger for rising costs, the analysts who produced the report said it is not the only factor. It's probably not the most important one when placed next to a recovering economy and an aging population. Traditionally, the state of the economy has been the strongest driver of health care spending.

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Health Care Costs About to Rise Again

Government report forecasts rising health care inflation

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FILE: June 27, 2012: A patient talks with a doctor at Camillus Health Concern, in Miami.AP

WASHINGTON The nation's respite from troublesome health care inflation is ending, the government said Wednesday in a report that renews a crucial budget challenge for lawmakers, taxpayers, businesses and patients.

Economic recovery, an aging society, and more people insured under the new health care law are driving the long-term trend.

Projections by nonpartisan experts with the Health and Human Services department indicate the pace of health care spending will pick up starting this year and beyond. The introduction of expensive new drugs for the liver-wasting disease hepatitis C also contributes to the speed-up in the short run.

The report from the Office of the Actuary projects that spending will grow by an average of 6 percent a year from 2015-2023. That's a notable acceleration after five consecutive years, through 2013, of annual growth below 4 percent.

Although the coming bout of health-cost inflation is not expected to be as aggressive as in the 1980s and 1990s, it will still pose a dilemma for President Barack Obama's successor. Long term, much of the growth comes from Medicare and Medicaid, two giant government programs now covering more than 100 million people.

The United States is expected to spend more than $3 trillion on health care this year, far above any other economically advanced country. Yet Americans are not appreciably healthier, and much what they spend appears to go for tests and treatments of questionable value. Fraud also siphons off tens of billions of dollars a year.

Because health care spending is so high, shifts of a couple of percentage points have significant economic consequences. Health care inflation has recently been in line with overall economic growth, keeping things manageable.

As spending rebounds, health care again will start consuming a growing share of the economic pie, crowding out other worthy priorities. From 17.2 percent of the economy in 2012, health care is expected to grow to a 19.3 percent share by 2023, the report said.

"The period in which health care has accounted for a stable share of economic output is expected to end in 2014, primarily because of the (health care law's) coverage expansions," it concluded.

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Government report forecasts rising health care inflation

GOP seizes on 2008 statement by Mark Udall on health care reform

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Republican operatives believe they have found a smoking gun against Democratic U.S. Sen. Mark Udall, who said during a 2008 debate he was against a "government-sponsored" solution for health care.

The then-congressman, who was running for an open seat in the U.S. Senate, echoed arguments made by conservatives.

"I'm not for a government-sponsored solution," Udall said. "I'm for enhancing and improving the employer-based system that we have."

In a debate overshadowed by other issues rising energy prices and the war on terror Udall's answer that July barely created a ripple. But in the context of Sen. Udall's vote for the Affordable Care Act in 2010 and his tough re-election bid against Republican Congressman Cory Gardner in November, the statement takes on new meaning.

Udall's vote for the health care law has provided fodder for a number of campaign attack ads.

"Mark Udall was elected on a lie," Gardner said. "Mark Udall promised he would not support a government-sponsored solution and he broke that promise and voted for Obamacare."

Udall's campaign says if Republicans believe they have found a smoking gun, they're simply shooting themselves in the foot because Obamacare is not government-run health care.

"They are grasping at straws," Udall spokeswoman Kristin Lynch said.

The influential PolitiFact bolsters Udall's argument. The Pulitzer Prize winning independent fact-checking website awarded its 2010 Lie of the Year to the claim that the Democratic law amounted to a "government takeover of health care."

" 'Government takeover' conjures a European approach where the government owns the hospitals and the doctors are public employees. But the law Congress passed ... relies largely on the free market," PolitiFact concluded.

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GOP seizes on 2008 statement by Mark Udall on health care reform

Health care spending to grow more slowly, government report suggests

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Washington

Health care spending to grow slower

National spending on health care will continue to surge in coming years, but at a slower rate than the previous two decades, according to new government analysis of the nation's $3 trillion health care tab. The Department of Health and Human Services report suggests that changes under way in medical care and insurance coverage may help rein in the notoriously high-cost system, even as millions of Americans gain insurance through the federal health law. But the slowing still may not make health care affordable, as medical spending is projected to outpace economic growth in the next decade, the report suggests.

Reno, Nev.

13 hurt in chemical mishap at museum

Authorities said 13 people, most of them children, were injured Wednesday when a demonstration about the science of tornadoes went awry at a museum in downtown Reno. Officials said eight children and one adult were treated at a hospital for minor burns and minor smoke inhalation. Four others were treated at the scene. Officials say a chemical flash, "similar to if someone threw gasoline on a fire," apparently occurred during a routine demonstration at the Terry Lee Wells Discovery Museum to create a whirling tornado effect.

Washington

VA rules may open door to benefits

Thousands of Vietnam-era veterans barred from receiving benefits because of less-than-honorable discharges may be eligible for upgrades under a new set of guidelines released by the Defense Department on Wednesday. The new rules offer the first guidance to military discharge review boards on how to address post-traumatic stress disorder. Many experts and veterans' advocates assert that the disorder may have contributed to misconduct by veterans who were later kicked out of the military and stripped of benefits.

Vienna

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Health care spending to grow more slowly, government report suggests

Genetic 'hotspot' linked to endometrial cancer aggressiveness

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PUBLIC RELEASE DATE:

3-Sep-2014

Contact: Ron Gilmore rlgilmore1@mdanderson.org 713-745-1898 University of Texas M. D. Anderson Cancer Center

Parents of twins often tell them apart through subtle differences such as facial expression, moles, voice tone and gait. Similarly, physicians treating women with endometrial cancer must be able to distinguish between different versions of this disease form that, on the surface, appear the same.

With endometrial cancer, the most common gynecological cancer in the western world and the fourth most prevalent in the U.S., it can literally be a matter of life and death. Mortality rates from this cancer have nearly tripled in the last 25 years and are thought to be attributed to the rising incidence of obesity.

Scientists at The University of Texas MD Anderson Cancer Center in Houston have identified genetic mutations in endometrioid endometrial carcinoma (EEC), the most common form of this cancer of the uterine lining. The mutations revealed a more lethal version of an EEC subtype previously thought to respond well to treatment. It's possible that by identifying these patients early on, oncologists can try more aggressive treatment approaches to increase the likelihood for a positive outcome.

"EEC is categorized into subtypes that help determine risk of recurrence and guide treatment," said Wei Zhang, Ph.D., professor of pathology at MD Anderson. "Most patients have Type I, which can be diagnosed early and generally has a good outcome with treatment."

Type I accounts for 70 to 80 percent of all EECs. Type II is more troublesome and is usually diagnosed late in the cancer's progression resulting in a poor prognosis. Zhang's team, however, identified a cluster of patients within Type I that appears to have a more virulent form of it previously not recognized. Zhang labeled this patient group as Cluster II.

"The patients were mostly younger and obese that's typical for Type I. What's unusual is for patients in this disease category to have decreased survival rates," said Zhang. "Molecular subtyping of EEC may help oncologists with diagnosis and prognosis within this unique subset."

Zhang believes that by being able to identify molecular "attributes," physicians can identify EEC patients at risk for this more lethal form of the disease.

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Genetic 'hotspot' linked to endometrial cancer aggressiveness

ARCA Biopharma Announces Health Canada Acceptance Of Genetic-AF Clinical Trial Application

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WESTMINSTER, COLO.--(BUSINESS WIRE)--

ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company developing genetically targeted therapies for cardiovascular diseases, today announced that the Companys Clinical Trial Application (CTA) for the GENETIC-AF clinical trial evaluating GencaroTM as a potential treatment for atrial fibrillation (AF) has been accepted by Health Canada. ARCA anticipates that clinical trial sites in Canada will be active in the fourth quarter of 2014.

Dr. Michael R. Bristow, President and CEO, ARCA biopharma, Inc. (Photo: Business Wire)

ARCA is evaluating Gencaro, a pharmacologically unique beta-blocker and mild vasodilator, as a potential treatment for AF in the Phase 2B/3 GENETIC-AF clinical trial, which is currently enrolling patients in the United States. ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it potential to be the first genetically targeted therapy for the prevention of atrial fibrillation.

Dr. Michael R. Bristow, Founder and CEO of ARCA, commented, At ARCA, we believe a personalized medicine approach to drug development, tailoring medical treatment to the individual genetic characteristics of each patient, can enable more effective therapies, improve patient outcomes and reduce healthcare costs. If the GENETIC-AF trial successfully confirms the atrial fibrillation data analysis from a prior Phase 3 clinical trial, Gencaro has the potential to be the first genetically targeted treatment for the prevention of this important cardiovascular disorder and provide a much needed treatment option for patients in an area of high unmet medical need.

About Atrial Fibrillation (AF)

Atrial fibrillation, the most common sustained cardiac arrhythmia, is considered an epidemic cardiovascular disease and a major public health burden. The estimated number of individuals with AF globally in 2010 was 33.5 million. According to the 2014 American Heart Association report on Heart Disease and Stroke Statistics, the estimated number of individuals with AF in the U.S. in 2010 ranged from 2.7million to 6.1million people. Hospitalization rates for AF increased by 23% among US adults from 2000 to 2010 and hospitalizations account for the majority of the economic cost burden associated with AF.

AF is a disorder in which the normally regular and coordinated contraction pattern of the hearts two small upper chambers (the atria) becomes irregular and uncoordinated. The irregular contraction pattern associated with AF causes blood to pool in the atria, predisposing the formation of clots potentially resulting in stroke. AF increases the risk of mortality and morbidity due to stroke, congestive heart failure and impaired quality of life. The approved therapies for the treatment or prevention AF have certain disadvantages in patients with heart failure and/or reduced left ventricular ejection fraction (HFREF) patients. These include toxic or cardiovascular adverse effects, and most of the approved drugs for AF are contra indicated or have warnings in their prescribing information for such patients. The Company believes there is an unmet medical need for new AF treatments that have fewer side effects than currently available therapies and are more effective, particularly in HFREF patients.

GENETIC-AF Clinical Trial

GENETIC-AF is a Phase 2B/3, multi-center, randomized, double-blind clinical trial comparing the safety and efficacy of Gencaro to Toprol-XL for prevention of symptomatic AF/atrial flutter in HFREF patients. ARCA plans to enroll only patients with the genetic variant of the beta-1 cardiac receptor which the Company believes responds most favorably to Gencaro. GENETIC-AF has an adaptive design, under which the Company initiated the trial as a Phase 2B trial in approximately 200 patients. The GENETIC-AF Data Safety Monitoring Board (DSMB) will analyze certain data from the Phase 2B portion of the trial and recommend, based on a comparison to the pre-trial statistical assumptions, whether the trial should proceed to Phase 3 and seek to enroll an additional 420 patients.

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ARCA Biopharma Announces Health Canada Acceptance Of Genetic-AF Clinical Trial Application

Is a gluten-free diet enough to control the complications of celiac disease?

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PUBLIC RELEASE DATE:

3-Sep-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 2, 2014A lifelong gluten-free diet (GFD) is the conventional approach to managing celiac disease, a chronic inflammatory disorder affecting the small intestine that can result in malnutrition. However, recent evidence shows that a GFD may not be sufficient to prevent serious complications related to celiac disease. A detailed discussion of the metabolic disorders and functional abnormalities that can develop, and nutritional treatments for these is presented in a Review article published in Journal of Medicinal Food, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Medicinal Food website until October 2, 2014.

Sara Farnetti and coauthors, Catholic University of the Sacred Heart (Rome, Italy), cover a broad scope of digestive and nutrient absorptive processes in individuals with celiac disease that may be compromised due to increased inflammation. In the article "Functional and Metabolic Disorders in Celiac Disease: New Implications for Nutritional Treatment", the authors discuss how diminished gallbladder and pancreatic function, and increased gut permeability may contribute to the development of overweight and obesity, and impaired glucose and lipid metabolism and insulin secretion in these patients.

"This article reviews the extensive literature on lifelong gluten-free diet supplementation to celiac disease patients and makes outstanding recommendations," says Journal of Medicinal Food Editor-in-Chief Sampath Parthasarathy, MBA, PhD, Florida Hospital Chair in Cardiovascular Sciences, University of Central Florida, Orlando. "The authors conclude that plant oils and products are able to stimulate the gall bladder to promote the absorption process and provide better nutrition to these patients. The conclusion that a lifelong gluten-free diet provision must be accompanied by proper nutrient supplementation is a sound one; however, caution must be exercised in using fried oil as a gall bladder stimulant."

About the Journal

Journal of Medicinal Food is an authoritative, peer-reviewed, multidisciplinary journal published monthly in print and online. Led by Editors-in-Chief Sampath Parthasarathy, MBA, PhD, and Young-Eun Lee, PhD, Wonkwang University, Jeonbuk, Korea, this scientific journal publishes original research on the bioactive substances of functional and medicinal foods, nutraceuticals, herbal substances, and other natural products. The Journal explores the chemistry and biochemistry of these substances, as well as the methods for their extraction and analysis, the use of biomarkers and other methods to assay their biological roles, and the development of bioactive substances for commercial use. Tables of content and a sample issue may be viewed on the Journal of Medicinal Food website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including The Journal of Alternative and Complementary Medicine. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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Is a gluten-free diet enough to control the complications of celiac disease?

Are rising health care costs inevitable?

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PUBLIC RELEASE DATE:

3-Sep-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 3, 2014If continuing increases in health care costs are inevitable, as some economists predict, is it possible for health care delivery reform to succeed in reducing the overall burden of health care expenditures on the U.S. economy? According to the results of a new study, the focus should shift from cost control to improving utilization rates and quality outcomes, as described in detail in an article in Population Health Management, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Population Health Management website until October 3, 2014.

In the article "Evaluating Health Care Delivery Reform Initiatives in the Face of 'Cost Disease'," Steven Thompson, PhD, University of Richmond, VA, Rajiv Kohli, PhD, College of William and Mary (Williamsburg, VA), Craig Jones, MD and Nick Lovejoy, Vermont Blueprint for Health (Williston, VT), and Katharine McGraves-Lloyd and Karl Finison, Onpoint Health Data (Portland, ME), analyzed claims by patients in Vermont covered by Medicaid and commercial insurance for the 5-year period 2007-2011. The authors evaluated utilization rates and cost of care for inpatient services for individuals treated in patient-centered medical homes, which are part of a novel delivery system model in Vermont that focuses on enhancing preventive health services. Through disease prevention and improved disease management, a goal of this delivery model is reduced inpatient care, with a corresponding decrease in per patient cost of care.

"Research like this is very important in building the evidentiary basis for the Patient Centered Medical Home," says Editor-in-Chief David B. Nash, MD, MBA, Dean and Dr. Raymond C. and Doris N. Grandon Professor, Jefferson School of Population Health, Philadelphia, PA. "It is especially important when it comes to helping persons in the Medicaid program."

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About the Journal

Population Health Management is an authoritative peer-reviewed journal published bimonthly in print and online that reflects the expanding scope of health care management and quality. The journal delivers a comprehensive, integrated approach to the field of population health and provides information designed to improve the systems and policies that affect health care quality, access, and outcomes. Comprised of peer-reviewed original research papers, clinical research, and case studies, the content encompasses a broad range of chronic diseases (such as cardiovascular disease, cancer, chronic pain, diabetes, depression, and obesity) in addition to focusing on various aspects of prevention and wellness. Tables of Contents and a sample issue may be viewed on the Population Health Management website. Population Health Management is the official journal of the Population Health Alliance.

About the Publisher

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Are rising health care costs inevitable?

'Biopharming' Offers A Powerful New Approach To Ebola And Other Diseases

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Due to the largest-ever Ebola virus outbreak in Africa and the treatment of a handful of patients with an experimental drug, there has been a resurgence of interest in therapeutics to treat the disease. One aspect that has been largely ignored is that the drug, called ZMapp, a mixture of three varieties of humanized monoclonal antibodieswhich bind, in vivo, to proteins on the surface of the Ebola virusrepresents an exquisite tour de force of genetic engineering.

The drug is obtained from genetically engineered tobacco plants that have been infected with genetically engineered plant viruses. During infection of the tobacco plants over the course of a week, the viruses, which are completely harmless to animals and humans, produce huge amounts of the antibodies. The plants are then harvested and homogenized and the antibodies are purified and formulated for administration. They bind to proteins of the Ebola virus in patients and elicit a humoral (antibody) and cellular (lymphocyte) response to the virus.

A seminal study of ZMapp in monkeys experimentally infected with Ebola virus was reported last week in the journal Nature. All 18 rhesus macaques treated with the drug recovered, even when it was administered beginning up to five days after infection. According to the journals press release, Three doses of ZMapp, administered at three-day intervals starting on day three, four or five after rhesus macaques were infected with Ebola virus, resulted in the survival of all 18 animals, while the three rhesus macaques that did not receive ZMapp all succumbed to Ebola virus infection by day eight. The drug reversed severe Ebola virus disease symptoms such as excessive bleeding, rashes and elevated liver enzymes. These findings are particularly encouraging because they provide precisely the kind of evidence of efficacy needed for regulatory approval of ZMapp, which will be evaluated by FDA under the animal rule. This applies to the development of drugs when human efficacy studies are not ethical or feasible.

Obtaining medicines from plants is not new. Many common medicines, such as morphine, codeine, cocaine and the laxative Metamucil are all purified from plants. But the promise of a relatively new approach called biopharming lies in using genetic engineering techniques to induce crops such as corn, tomatoes and tobacco to produce high concentrations of high-value pharmaceuticals.

Biopharming emerged with great promise about 15 years ago, with clinical trials of vaccines and drugs made in bananas, tomatoes and tobacco. Unfortunately, the field confronted the zeal and risk-aversion of regulators. In 2002, a company called Ventria purified two human proteins from genetically engineered rice and found that when added to oral rehydration solution given to children with diarrhea, they markedly shortened the duration of symptoms and reduced the incidence of recurrence. This potential public health breakthrough has been effectively blocked by the FDA: In 2010 the company approached the Food and Drug Administration for recognition that these proteins, which are found in human tears and breast milk, are generally recognized as safe (a regulatory term of art), but received no response. Ventria was unwilling to market the product without the FDAs endorsement, so it isnt available, depriving children in developing countries of a life-saving therapy.

More than a decade ago, scientists at Arizona State University created a biopharmed vaccine against Norwalk virus, the bug that annually causes millions of cases of diarrhea on cruise ships and in nursing homes. This vaccine, initially produced in tomato fruit and more recently in tobacco leaves, is still being studied to find an optimal formulation for administration.

The field testing of biopharmed plants has proved particularly problematic. In 2003 the U.S. Department of Agricultures Animal and Plant Health Inspection Service announced onerous new rules for the field testing of biopharmed crops. That ended most entrepreneurial interest in biopharming. Mapp Biopharmaceutical, the privately-owned company that makes the experimental Ebola drug ZMapp, boasts a workforce of nine people and has been completely financed by government grants and contracts.

USDAs rules on the cultivation of the biopharmed plants in the field impose highly prescriptive, one-size-fits-all design standards, as contrasted with performance standards, which would specify an end-pointsuch as gene-transfer below a certain levelthat must be achieved by whatever means. USDAs regulation fails to take into account the actual risks of a given situation.

The ostensible objective of the regulation is to avoid biopharmed drugs contaminating food, if crop plants are used in the drug production. The food industry, including groups such as the Grocery Manufacturers of America and the U.S. Rice Producers Association, has raised NIMBYnot in my backyardobjections. They claimed that biopharmed plants could contaminate their food-grade crops, but that fear is overblown and can be avoided in several ways. Production in a non-food crop is an obvious one, and that has affected manufacturing decisions for many new biopharmed vaccines and drugs. For example, the developers of the Norwalk virus vaccine switched from tomato to tobacco both to improve drug yields and to avoid becoming embroiled in disputes with NGOs and regulators about the supposed risks of genetic engineering and possible contamination of food.

The risk of plant-made drugs getting into food products is now virtually nonexistent because the companies involved have switched to production in facilities with rigidly controlled environments, mainly using tobacco. This approach was greatly advanced by the investment in 2010 of more than $80 million in facilities by the federal Defense Advanced Research Projects Agency (DARPA) to expand the tobacco-growing capacity at several companies. The investment was driven by DODs desire to expand the nations ability to respond with new drugs and vaccines to emerging diseases or attacks with biological agents. These sorts of facilities, which have a high degree of control over growth conditions, are essential for the reproducible production of high-quality drugs. This constructive public-private collaboration set the stage for ZMapp, the Ebola drug, to be produced by one of the companies Kentucky Bioprocessing.

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'Biopharming' Offers A Powerful New Approach To Ebola And Other Diseases

Pioneer Award recipients Robin Ali, Ph.D., Jean Bennett, M.D., Ph.D., and William Hauswirth, Ph.D.

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PUBLIC RELEASE DATE:

3-Sep-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 3, 2014Robin Ali, PhD, University College London, Jean Bennett, MD, PhD, Perelman School of Medicine, University of Pennsylvania, and William Hauswirth, PhD, University of Florida College of Medicine, are co-recipients of the Pioneer Award, recognized for their leadership and contributions to the field of gene therapy to treat retinal degeneration leading to blindness. Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers, is commemorating its 25th anniversary by bestowing this honor on the leading Pioneers in the field of cell and gene therapy selected by a blue ribbon panel* and publishing a Pioneer Perspective by the award recipients.

Dr. Ali, Professor of Human Molecular Genetics, led proof-of-concept studies demonstrating the feasibility of using gene therapy to repair photoreceptor defects in the eye and of using cell transplantation for retinal repair. He also had a pioneering role in the first clinical trial for inherited retinal degeneration.

Dr. Bennett, Professor of Ophthalmology, Cell and Developmental Biology, recalls her first experiences with molecular biology and gene transfer technology, acquired in the lab of Dr. W. French Anderson, known as "the father of gene therapy." She describes her developing career, including the decision to go to medical school and to focus her research on developing adeno-associated virus (AAV) gene therapy techniques for restoring vision to patients affected by retinal degeneration in her Pioneer Perspective article entitled "My Career Path for Developing Gene Therapy for Blinding Diseases: The Importance of Mentors, Collaborators, and Opportunities," available on the Human Gene Therapy website.

Dr. Hauswirth, Rybaczki-Bullard Professor of Ophthalmology, traces his involvement in the field of retinal gene therapy to his early interest in studying the interaction between light and biological molecules. He provides a historical perspective on the discovery of the gene mutations responsible for several of the most common inherited eye diseases and the advances in AAV gene therapy technology being developed and applied to deliver replacement genes. His Pioneer Perspective, entitled "Retinal Gene Therapy Using Adeno-Associated Viral Vectors: Multiple Applications for a Small Virus," is available on the Human Gene Therapy website.

"These groups brought forward the first convincing clinical results of in vivo gene therapy, which paved the way for the current renaissance we are seeing in the field," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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*The blue ribbon panel of leaders in cell and gene therapy, led by Chair Mary Collins, PhD, MRC Centre for Medical Molecular Virology, University College London selected the Pioneer Award recipients. The Award Selection Committee selected scientists that had devoted much of their careers to cell and gene therapy research and had made a seminal contribution to the field--defined as a basic science or clinical advance that greatly influenced progress in translational research.

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Pioneer Award recipients Robin Ali, Ph.D., Jean Bennett, M.D., Ph.D., and William Hauswirth, Ph.D.

On the Horns of the GMO Dilemma

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Can genome-editing technology revive the idea of genetically modified livestock?

Four years ago, Scott Fahrenkrug saw an ABC News segment about the dehorning of dairy cows, a painful procedure that makes the animals safer to handle. The shaky undercover video showed a black-and-white Holstein heifer moaning and bucking as a farmhand burned off its horns with a hot iron.

Fahrenkrug, a molecular geneticist then at the University of Minnesota, thought he had a way to solve the problem. He could create cows without horns. He could save farmers money. And by eliminating the dairy industrys most unpleasant secret, he might even score a public relations success for genetic engineering.

The technology Fahrenkrug believes could do all this is called genome editing (see Genome Surgery and Genome Editing). A fast, precise new way of altering DNA, its been sweeping through biotechnology labs. Researchers have used it to change the genes of mice, zebrafish, and monkeys, and it is being tested as way to treat human diseases like HIV (see Can Gene Therapy Cure HIV?).

With livestock, gene editing offers some extraordinary possibilities. At his startup, Recombinetics, located in St. Paul, Minnesota, Fahrenkrug thinks he can create blue-ribbon dairy bulls possessing traits not normally found in those breeds but present in other cattle, such as lack of horns or resistance to particular diseases. Such molecular breeding, he says, would achieve the same effects as nature might, only much faster. In short, an animal could be edited to have the very best genes its species can offer.

That could upend the global livestock industry. Companies could patent these animals just as they do genetically modified soybeans or corn. Entrepreneurs are also ready to challenge the U.S. Food and Drug Administration, which has never approved a GMO food animal. They say gene editing shouldnt be regulated if its used to merely swap around traits within a species. Were talking about genes that already exist in a species we already eat, says Fahrenkrug.

The use of the technology remains experimental and far from the food chain. But some large breeding companies are starting to invest. There may be an opportunity for a different public acceptance dialogue and different regulations, says Jonathan Lightner, R&D chief of the U.K. company Genus, which is the worlds largest breeder of pigs and cattle and has paid for some of Recombinetics laboratory research. This isnt a glowing fish. Its a cow that doesnt have to have its horns cut off.

GMO Bust

To date, GMO food animals have been a complete bust. After the first mice genetically engineered with viral DNA appeared in the 1970s, a parade of other modified animals followed, including sheep that grow extra wool thanks to a mouse gene, goats whose udders made spider silk, and salmon that mature twice as quickly as normal. But such transgenicsanimals incorporating genes from other speciesmostly never made it off experimental farms.

Opponents of genetically modified organisms (GMOs) gathered millions of signatures to stop frankenfoods, and the FDA has held off approving such animals as food. AquaBounty Technologies, the company that made the fast-growing transgenic salmon, has spent 18 years and $70 million trying to get the fish cleared. Two years ago, the University of Guelph, in Ontario, euthanized its herd of enviropigs, engineered with an E. coli gene so they pooped less phosphorus, after giving up hope of convincing regulators.

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On the Horns of the GMO Dilemma