NEWS IN BRIEF
Biomedical briefing POLICY The UK government has launched a new program designed to give people with life-threatening illnesses the chance to receive new treatments before they are formally approved. Under the Early Access to Medicines Scheme unveiled on 14 March, drugs can receive a ‘promising innovative medicine’ designation if early clinical data suggest they are dramatically better than existing options. Pharma companies can then provide—but would have to pay for—any drug administrations. “The scheme offers a way by which unlicensed medicines can be available to patients before approval of a license to benefit public health,” Ian Hudson, chief executive of the UK Medicines and Healthcare Products Regulatory Agency, said in a statement. “It will also enable companies to gain additional knowledge and experience of these medicines in clinical use.”
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Sweet and low In order to fight obesity and tooth decay, the World Health Organization announced plans on 5 March to cut the recommended daily limit for sugar in half—from 10% of daily calories to 5%—setting up an expected clash with the processed food industry. The recommendations relate to what are called ‘free’ sugars—monosaccharides added to prepared foods, as well as disaccharides such as honey and syrup—and the suggested limits amount to just six teaspoons per day, or less than the total sugar content in a can of soda. Food manufacturers have opposed sugar limits in the past. Thus, “strong resistance from parts of the food industry is one prediction in which I have great confidence,” says Walter Willett, head of nutrition at the 326
Manfred Boehm, NHLBI
© 2014 Nature America, Inc. All rights reserved.
Early access
Researchers narrow in on the cause of bypass surgery failure This year, around 400,000 Americans will undergo coronary artery bypass surgery to restore blood flow in the heart. Yet, within a decade, half of the vein grafts involved in these surgeries will have narrowed to the point of failure, a process known as stenosis. A team led by Brian Cooley, a vein graft researcher now at the University of North Carolina School of Medicine in Chapel Hill, has now discovered a biological rationale for this narrowing. Working with mouse models and postmortem human tissue, the researchers showed that the endothelial cells (green) that
Harvard School of Public Health in Boston.
Global change After two years of planning, the Global Fund to Fight AIDS, Tuberculosis and Malaria put a new funding model into effect on 12 March. Under the new scheme, the Geneva-based organization—which raised $12 billion in commitments from donors in its most recent money-raising round late last year—will evaluate the disease burdens that different countries face and factor this into its decisions about which projects
line the inside of vein grafts shift to a more mesenchymal, or early-smooth-muscle–like, state (red) as they remodel to become more arterial. This cellular transition is mediated by the transforming growth factor-b (TGF-b) pathway and contributes to the thickening of the blood vessel wall seen in vein graft stenosis. “From TGF-b on down you could target any of the components of that pathway and see if you could knock it down,” Cooley says. His team reported the findings on 12 March in Science Translational Medicine (6, 227ra34, 2014).
to fund. Additionally, there is a new condition requiring countries to supplement the money they receive with domestic funds. “We have to encourage countries that are fighting these diseases to invest their money because long term it is not sustainable to get money from developed countries to fight these diseases,” says Seth Faison, a spokesperson for the Global Fund.
OTC overhaul The US Food and Drug Administration (FDA) held a public hearing on 25 and 26 March to explore possible changes
to the way it regulates most overthe-counter (OTC) drugs. Under the current 42-year-old system, manufacturers can sell OTC drugs without FDA premarket approval as long as the products conform to certain specifications about permissible ingredients and labeling claims. Because the FDA must go through a lengthy rulemaking process in order to modify these regulations—to require, for example, lower doses of an ingredient or new label warnings—it now often takes longer for the agency to address safety concerns about OTC drugs than it does for
VOLUME 20 | NUMBER 4 | APRIL 2014 NATURE MEDICINE
NEWS IN BRIEF Animal rights activists shift tactics toward individual scientists
Targets of illegal actions by animal rights activists in the US
1990–1999
2000–2012 Universities Individuals Companies Investors Business partners
says Darrell Kirch, president and chief executive of the Association of American Medical Colleges, where Skorton previously served as a board member. “He is a twenty-first century Renaissance man who will bring his combined passion for the arts, humanities, education, science and technology to this role.” Skorton (pictured) will take up his new position, succeeding outgoing secretary, civil engineer G. Wayne Clough, in July 2015.
prescription medications. “OTC drugs have a huge effect on the public health, and it’s important that the FDA be able to respond quickly to new safety information about these products,” says Patricia Zettler, a health policy researcher at the Stanford Law School Center for Law and the Biosciences in California.
PEOPLE Museum master On 10 March, the Smithsonian Institution named Cornell University president David Skorton to be its next leader. Skorton, a cardiologist by training, will be the first physician to head the venerable US institution—a $1.3 billion collection of 19 museums, nine research centers and a zoological park mostly in the Washington, DC area. “Dr. Skorton is a superb choice,”
BUSINESS Vaccine victors
Cornell University Photography
npg
© 2014 Nature America, Inc. All rights reserved.
Other
A German company called CureVac has won the EU’s first-ever innovation inducement prize. The European Commission offered the €2 million ($2.8 million) award to stimulate solutions for vaccine transportation and storage in parts of the developing world without reliable refrigeration. CureVac took the prize for its vaccines based on mRNA molecules, a platform that is relatively stable at ambient temperatures for long periods of time. “The recognition is really outstanding,” says chief executive officer Ingmar Hoerr. “This prize gives new validation to the whole technology that we’ve developed
NATURE MEDICINE VOLUME 20 | NUMBER 4 | APRIL 2014
Federation of American Societies for Experimental Biology, Foundation for Biomedical Research
Scientists beware: you and your associates could personally be at risk of attack from animal rights extremists. According to a new report from the Federation of American Societies for Experimental Biology (FASEB), illegal incidents in the US that involved animal rights activists took place largely at university facilities in the 1990s. Nowadays, however, individual researchers’ homes, business partners and investors are more often the target. “The universities have been structured in a way to better handle the challenge from extremist attacks,” says Kevin Kregel, a physiologist at the University of Iowa in Iowa City who co-chaired the FASEB committee that created the 12 March report. “That’s why it seems that these extremist groups and individuals are going toward the lowest hanging fruit.”
for 14 years and also to the company itself.” CureVac, which is headquartered in Tübingen, currently has vaccines for prostate cancer, lung cancer and rabies in early- or mid-stage clinical development.
Forest takeover The Irish generic drug giant Actavis announced on 18 February that it would buy New York–based Forest Laboratories for around $25 billion in cash and stock. With an ever-dwindling number of blockbuster drugs scheduled to lose patent protection in the next few years, the massive deal—the largest pharma acquisition in more than four years—should help Actavis shift away from gener-
ics by strengthening its US sales force and expanding its branded drug portfolio to include Forest’s top-selling Alzheimer’s drug Namenda (memantine) and other successful medications. “It gives the company more infrastructure to compete in this branded market,” says Michael Waterhouse, a healthcare analyst at Morningstar in Chicago.
DRUGS Metabolic medicine On 25 February, the FDA approved the first treatment for the metabolic abnormalities associated with inherited or acquired forms of generalized lipodystrophy. Patients with this rare condition have little fat tissue and often develop increased appetite, insulin resistance and high blood levels of triglycerides owing to a deficiency in the fat-derived hormone leptin, which regulates food intake and metabolism. Leptin replacement therapy with daily injections of Myalept (metreleptin), a synthetic form of the hormone made by San Diego–based Amylin Pharmaceuticals, “can get at the underlying problem and thereby improve the broad spectrum of metabolic abnormalities,” says Rebecca Brown, a pediatric endocrinologist at the US National Institute of Diabetes and Digestive and Kidney Diseases in Bethesda, Maryland, who was involved in open-label trials of the drug.
Corrections The March 2014 news article ‘Competition aims to revive languishing breast cancer inventions’ (Nat. Med. 20, 223, 2014) did not specify that the Center for Advancing Innovation coordinated the fundraising effort and inaccurately implied that NCI has 1,800 patented inventions in its portfolio. In fact, some of those inventions are not patent protected. The errors have been corrected in the HTML and PDF versions of the article. The February 2014 Opinion piece ‘Pediatric requirements in Europe stymie help for hemophilia’ (Nat. Med. 20, 117, 2014) did not specify the competing financial interests as a result of incomplete processing. The error has been corrected in the HTML and PDF versions of the article.
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Biomedical briefing POLICY The UK government has launched a new program designed to give people with life-threatening illnesses the chance to receive new treatments before they are formally approved. Under the Early Access to Medicines Scheme unveiled on 14 March, drugs can receive a ‘promising innovative medicine’ designation if early clinical data suggest they are dramatically better than existing options. Pharma companies can then provide—but would have to pay for—any drug administrations. “The scheme offers a way by which unlicensed medicines can be available to patients before approval of a license to benefit public health,” Ian Hudson, chief executive of the UK Medicines and Healthcare Products Regulatory Agency, said in a statement. “It will also enable companies to gain additional knowledge and experience of these medicines in clinical use.”
npg
Sweet and low In order to fight obesity and tooth decay, the World Health Organization announced plans on 5 March to cut the recommended daily limit for sugar in half—from 10% of daily calories to 5%—setting up an expected clash with the processed food industry. The recommendations relate to what are called ‘free’ sugars—monosaccharides added to prepared foods, as well as disaccharides such as honey and syrup—and the suggested limits amount to just six teaspoons per day, or less than the total sugar content in a can of soda. Food manufacturers have opposed sugar limits in the past. Thus, “strong resistance from parts of the food industry is one prediction in which I have great confidence,” says Walter Willett, head of nutrition at the 326
Manfred Boehm, NHLBI
© 2014 Nature America, Inc. All rights reserved.
Early access
Researchers narrow in on the cause of bypass surgery failure This year, around 400,000 Americans will undergo coronary artery bypass surgery to restore blood flow in the heart. Yet, within a decade, half of the vein grafts involved in these surgeries will have narrowed to the point of failure, a process known as stenosis. A team led by Brian Cooley, a vein graft researcher now at the University of North Carolina School of Medicine in Chapel Hill, has now discovered a biological rationale for this narrowing. Working with mouse models and postmortem human tissue, the researchers showed that the endothelial cells (green) that
Harvard School of Public Health in Boston.
Global change After two years of planning, the Global Fund to Fight AIDS, Tuberculosis and Malaria put a new funding model into effect on 12 March. Under the new scheme, the Geneva-based organization—which raised $12 billion in commitments from donors in its most recent money-raising round late last year—will evaluate the disease burdens that different countries face and factor this into its decisions about which projects
line the inside of vein grafts shift to a more mesenchymal, or early-smooth-muscle–like, state (red) as they remodel to become more arterial. This cellular transition is mediated by the transforming growth factor-b (TGF-b) pathway and contributes to the thickening of the blood vessel wall seen in vein graft stenosis. “From TGF-b on down you could target any of the components of that pathway and see if you could knock it down,” Cooley says. His team reported the findings on 12 March in Science Translational Medicine (6, 227ra34, 2014).
to fund. Additionally, there is a new condition requiring countries to supplement the money they receive with domestic funds. “We have to encourage countries that are fighting these diseases to invest their money because long term it is not sustainable to get money from developed countries to fight these diseases,” says Seth Faison, a spokesperson for the Global Fund.
OTC overhaul The US Food and Drug Administration (FDA) held a public hearing on 25 and 26 March to explore possible changes
to the way it regulates most overthe-counter (OTC) drugs. Under the current 42-year-old system, manufacturers can sell OTC drugs without FDA premarket approval as long as the products conform to certain specifications about permissible ingredients and labeling claims. Because the FDA must go through a lengthy rulemaking process in order to modify these regulations—to require, for example, lower doses of an ingredient or new label warnings—it now often takes longer for the agency to address safety concerns about OTC drugs than it does for
VOLUME 20 | NUMBER 4 | APRIL 2014 NATURE MEDICINE
NEWS IN BRIEF Animal rights activists shift tactics toward individual scientists
Targets of illegal actions by animal rights activists in the US
1990–1999
2000–2012 Universities Individuals Companies Investors Business partners
says Darrell Kirch, president and chief executive of the Association of American Medical Colleges, where Skorton previously served as a board member. “He is a twenty-first century Renaissance man who will bring his combined passion for the arts, humanities, education, science and technology to this role.” Skorton (pictured) will take up his new position, succeeding outgoing secretary, civil engineer G. Wayne Clough, in July 2015.
prescription medications. “OTC drugs have a huge effect on the public health, and it’s important that the FDA be able to respond quickly to new safety information about these products,” says Patricia Zettler, a health policy researcher at the Stanford Law School Center for Law and the Biosciences in California.
PEOPLE Museum master On 10 March, the Smithsonian Institution named Cornell University president David Skorton to be its next leader. Skorton, a cardiologist by training, will be the first physician to head the venerable US institution—a $1.3 billion collection of 19 museums, nine research centers and a zoological park mostly in the Washington, DC area. “Dr. Skorton is a superb choice,”
BUSINESS Vaccine victors
Cornell University Photography
npg
© 2014 Nature America, Inc. All rights reserved.
Other
A German company called CureVac has won the EU’s first-ever innovation inducement prize. The European Commission offered the €2 million ($2.8 million) award to stimulate solutions for vaccine transportation and storage in parts of the developing world without reliable refrigeration. CureVac took the prize for its vaccines based on mRNA molecules, a platform that is relatively stable at ambient temperatures for long periods of time. “The recognition is really outstanding,” says chief executive officer Ingmar Hoerr. “This prize gives new validation to the whole technology that we’ve developed
NATURE MEDICINE VOLUME 20 | NUMBER 4 | APRIL 2014
Federation of American Societies for Experimental Biology, Foundation for Biomedical Research
Scientists beware: you and your associates could personally be at risk of attack from animal rights extremists. According to a new report from the Federation of American Societies for Experimental Biology (FASEB), illegal incidents in the US that involved animal rights activists took place largely at university facilities in the 1990s. Nowadays, however, individual researchers’ homes, business partners and investors are more often the target. “The universities have been structured in a way to better handle the challenge from extremist attacks,” says Kevin Kregel, a physiologist at the University of Iowa in Iowa City who co-chaired the FASEB committee that created the 12 March report. “That’s why it seems that these extremist groups and individuals are going toward the lowest hanging fruit.”
for 14 years and also to the company itself.” CureVac, which is headquartered in Tübingen, currently has vaccines for prostate cancer, lung cancer and rabies in early- or mid-stage clinical development.
Forest takeover The Irish generic drug giant Actavis announced on 18 February that it would buy New York–based Forest Laboratories for around $25 billion in cash and stock. With an ever-dwindling number of blockbuster drugs scheduled to lose patent protection in the next few years, the massive deal—the largest pharma acquisition in more than four years—should help Actavis shift away from gener-
ics by strengthening its US sales force and expanding its branded drug portfolio to include Forest’s top-selling Alzheimer’s drug Namenda (memantine) and other successful medications. “It gives the company more infrastructure to compete in this branded market,” says Michael Waterhouse, a healthcare analyst at Morningstar in Chicago.
DRUGS Metabolic medicine On 25 February, the FDA approved the first treatment for the metabolic abnormalities associated with inherited or acquired forms of generalized lipodystrophy. Patients with this rare condition have little fat tissue and often develop increased appetite, insulin resistance and high blood levels of triglycerides owing to a deficiency in the fat-derived hormone leptin, which regulates food intake and metabolism. Leptin replacement therapy with daily injections of Myalept (metreleptin), a synthetic form of the hormone made by San Diego–based Amylin Pharmaceuticals, “can get at the underlying problem and thereby improve the broad spectrum of metabolic abnormalities,” says Rebecca Brown, a pediatric endocrinologist at the US National Institute of Diabetes and Digestive and Kidney Diseases in Bethesda, Maryland, who was involved in open-label trials of the drug.
Corrections The March 2014 news article ‘Competition aims to revive languishing breast cancer inventions’ (Nat. Med. 20, 223, 2014) did not specify that the Center for Advancing Innovation coordinated the fundraising effort and inaccurately implied that NCI has 1,800 patented inventions in its portfolio. In fact, some of those inventions are not patent protected. The errors have been corrected in the HTML and PDF versions of the article. The February 2014 Opinion piece ‘Pediatric requirements in Europe stymie help for hemophilia’ (Nat. Med. 20, 117, 2014) did not specify the competing financial interests as a result of incomplete processing. The error has been corrected in the HTML and PDF versions of the article.
327
