In the House of Representatives of the United States panel today released a long awaited proposal to accelerate development new medical treatments. The massive paper, 393 pages, called the Act 21st Century Cures, has been under development by Fred Upton (R-MI) and Diana DeGette (D-CO) of the Committee on Energy and Commerce House since last April . He was born of a series of hearings and roundtable discussions last year on ways to stimulate innovation in biomedical research and streamline clinical trials and drug approval process. The result is a long list of ideas summarized in this document.
The document is a "discussion paper" the committee said in a statement. Upton invited comments through Twitter handle # Cures2015. "Some things can be deposited, certain elements may be added, but everything is on the table as we hope to initiate a thoughtful discussion towards a more brilliant product," he said in a statement.
DeGette, in a statement, said she appreciated the effort of Upton "to share publicly as possible the legislative language on a number of proposals that could be included in a future bill. ... Although I do not endorse the draft document, I know that with continued commitment, we can achieve a bipartisan consensus to support research and biomedical remedies advance. "
many parts of the project are to convince developers of drugs to focus on diseases for which there are few treatments. a section, called the Act Dormant therapies, takes aim medicines for diseases complex, such as Alzheimer's disease, which are particularly long to develop and test. by setting a fixed period of patent exclusivity after such treatments gain approval (and not when the drug is first identified), the bill would give drug developers more time to recover costs and make profits. other provisions extend the exclusivity period for patents on medicines to treat rare diseases, new antibiotics, and for improving existing drugs such as a new formulation which reduces side effects.
The draft proposal also aims to speed clinical trials and integrate new types of data in the US assessment process Food and Drug Administration. The agency would be required to set standards for how it will evaluate "placeholders" for -measure biological response of a patient to a drug that may indicate the benefits faster than measures such as survival and disease progression. It should also be the agency to develop a new method for incorporating patient feedback in the evaluation of a new treatment.
The project also offers a 21 century Cures Consortium, involving federal agencies, pharmaceutical companies, patient groups and other stakeholders who work together on ways to get drugs to patients more quickly. National Institutes of Health (NIH) beneficiaries would be required to share data, and data from health care facilities would be made available for research.
Certain provisions to NIH, which funds most basic biomedical research in the nation, have been proposed by the agency. For example, the NIH drug development branch, the National Centre for the Advancement of Science Translational, would no longer be forbidden to fund clinical trials beyond the stage known as phase IIA name.
Other representative proposals Andy Harris (R-MD) may be more controversial. For example, the project calls for setting aside a part of the NIH Common Fund to finance the proposals of scientists early in their careers. Although this is a smaller version of a back plane Harris earlier that would have required NIH to lower the average age of a member for the first time, he could still draw concerns about such a setting fallow. Harris also wants NIH to develop a comprehensive strategic plan. This idea flopped when last attempted agency, there are more than 2 decades.
Other sections remain to be fulfilled, including one on "precision medicine," the subject of an initiative announced by President Barack Obama in his State of the Union address last week .
Biomedical research groups offer praise. the project "represents a promising opportunity," said Carrie Wolinetz, president of the United coordination group for medical research, in a statement. "Funding for medical research has stalled over the past decade. ... The initiative of the 21st century Cures will help revive its engine. "
" The initiative could be a game changer for the ecosystem of medical innovation, "said Mary Woolley, president and CEO of the advocacy group Research! America, a report. "Among the many provisions beneficial research! America fought for a measure to reduce the administrative burden on researchers, "she noted. (Research Universities have long complained about the regulatory burden.)
The approach is also attracting interest the Senate of the United States. a statement by senators Lamar Alexander (R-TN) and Patty Murray (D-WA), the top Democrat and Republican, respectively, the health Committee of the Senate, noted that one of the priorities of their committee is also "the modernization of the Food and drug administration, helping to get treatment and medication to patients as quickly and as safely as possible."
committee now plans to gather feedback on the project, with an eye to push the legislation before the end of this year.
* updated January 27 5:31 p.m. :. this article has been updated to clarify that the document does not constitute a formal legislative proposal
In the House of Representatives of the United States panel today released a long awaited proposal to accelerate development new medical treatments. The massive paper, 393 pages, called the Act 21st Century Cures, has been under development by Fred Upton (R-MI) and Diana DeGette (D-CO) of the Committee on Energy and Commerce House since last April . He was born of a series of hearings and roundtable discussions last year on ways to stimulate innovation in biomedical research and streamline clinical trials and drug approval process. The result is a long list of ideas summarized in this document.
The document is a "discussion paper" the committee said in a statement. Upton invited comments through Twitter handle # Cures2015. "Some things can be deposited, certain elements may be added, but everything is on the table as we hope to initiate a thoughtful discussion towards a more brilliant product," he said in a statement.
DeGette, in a statement, said she appreciated the effort of Upton "to share publicly as possible the legislative language on a number of proposals that could be included in a future bill. ... Although I do not endorse the draft document, I know that with continued commitment, we can achieve a bipartisan consensus to support research and biomedical remedies advance. "
many parts of the project are to convince developers of drugs to focus on diseases for which there are few treatments. a section, called the Act Dormant therapies, takes aim medicines for diseases complex, such as Alzheimer's disease, which are particularly long to develop and test. by setting a fixed period of patent exclusivity after such treatments gain approval (and not when the drug is first identified), the bill would give drug developers more time to recover costs and make profits. other provisions extend the exclusivity period for patents on medicines to treat rare diseases, new antibiotics, and for improving existing drugs such as a new formulation which reduces side effects.
The draft proposal also aims to speed clinical trials and integrate new types of data in the US assessment process Food and Drug Administration. The agency would be required to set standards for how it will evaluate "placeholders" for -measure biological response of a patient to a drug that may indicate the benefits faster than measures such as survival and disease progression. It should also be the agency to develop a new method for incorporating patient feedback in the evaluation of a new treatment.
The project also offers a 21 century Cures Consortium, involving federal agencies, pharmaceutical companies, patient groups and other stakeholders who work together on ways to get drugs to patients more quickly. National Institutes of Health (NIH) beneficiaries would be required to share data, and data from health care facilities would be made available for research.
Certain provisions to NIH, which funds most basic biomedical research in the nation, have been proposed by the agency. For example, the NIH drug development branch, the National Centre for the Advancement of Science Translational, would no longer be forbidden to fund clinical trials beyond the stage known as phase IIA name.
Other representative proposals Andy Harris (R-MD) may be more controversial. For example, the project calls for setting aside a part of the NIH Common Fund to finance the proposals of scientists early in their careers. Although this is a smaller version of a back plane Harris earlier that would have required NIH to lower the average age of a member for the first time, he could still draw concerns about such a setting fallow. Harris also wants NIH to develop a comprehensive strategic plan. This idea flopped when last attempted agency, there are more than 2 decades.
Other sections remain to be fulfilled, including one on "precision medicine," the subject of an initiative announced by President Barack Obama in his State of the Union address last week .
Biomedical research groups offer praise. the project "represents a promising opportunity," said Carrie Wolinetz, president of the United coordination group for medical research, in a statement. "Funding for medical research has stalled over the past decade. ... The initiative of the 21st century Cures will help revive its engine. "
" The initiative could be a game changer for the ecosystem of medical innovation, "said Mary Woolley, president and CEO of the advocacy group Research! America, a report. "Among the many provisions beneficial research! America fought for a measure to reduce the administrative burden on researchers, "she noted. (Research Universities have long complained about the regulatory burden.)
The approach is also attracting interest the Senate of the United States. a statement by senators Lamar Alexander (R-TN) and Patty Murray (D-WA), the top Democrat and Republican, respectively, the health Committee of the Senate, noted that one of the priorities of their committee is also "the modernization of the Food and drug administration, helping to get treatment and medication to patients as quickly and as safely as possible."
committee now plans to gather feedback on the project, with an eye to push the legislation before the end of this year.
* updated January 27 5:31 p.m. :. this article has been updated to clarify that the document does not constitute a formal legislative proposal
The United States is currently experiencing what promises to be one of the worst measles outbreaks since the virus was declared eliminated from the country in 00. He began to early January at Disneyland Resort in Anaheim, California, and has since expanded to 14 states and infected 84 people, according to the Centers for Disease Control and Prevention (CDC). Measles is caused by a paramyxovirus of the genus Morbillivirus , is one of the most contagious diseases in the world, infecting more than 0% of susceptible hosts that come into contact with an afflicted individual. In the absence of generalized vaccination, the average person with measles will infect on average from 12 to 18 others; however, Ebola virus is usually transmitted at 1.5 to 2.5 people. Children in particular are more likely to suffer complications from an infection of measles. Although the overall mortality rate for children who get measles is only between 0.1% and 0.2%, as much as one in 20 children will also develop pneumonia. Symptoms of the disease can be managed with common anti-inflammatory medications, hydration, and the rest, but like many other viral diseases, there is no cure and antibiotics are ineffective. Mortality rates are much higher in developing countries.
What is measles are for the immune system?
measles virus is transmitted from person to person through the air in aerosol coughed- on droplets are inhaled. The virus is usually the first contact with the lung tissue of the host, where it infects immune cells called macrophages and dendritic cells, which serve as a system of defense and early warning. Hence, infected cells migrate to the lymph nodes where they transmit the virus to the B and T cell surface protein A on these white blood cells, known as CD150, is used as the virus entry point during this critical stage. Infected cells B and T then migrate through the body of viral particle release in the blood. The spleen, lymph nodes, liver, thymus, skin and lung are potential destinations of the virus. In rare cases (about one in 1000 cases), the virus can cross the blood-brain barrier and cause a dangerous swelling of the brain; infection of the lung cells causes coughing that keeps the viruses circulating in the population.
Why measles cause rash?
One of the classic presentations against measles infection is a rash characterized by red flat spots that starts on the face and the body moves down all the way on foot in a few days . The rash is a symptom of inflammation occurring in the skin. As the virus circulates in the blood, it infects the skin capillaries. immune cells detect infection and react by releasing chemicals such as nitric oxide and histamine, which destroy viral invaders and call other immune cells into action. These chemicals, however, cause swelling and damage to host cells, which causes the often rash itching, which usually occurs at the same time with a fever that can reach up to 40 ° C.
Why return of measles?
Measles has been back. Last year was one of the worst years in recent history of the United States: CDC reported 644 cases of 23 separate outbreaks during 2014; between 01 and 2013, not a single year has seen more than 250 cases. (Because measles was declared "eliminated" in the United States in 00, outbreaks were triggered by the virus "imported" from other countries, who then finds an unvaccinated person.) With 84 people already infected in 2015 , things are not off the best start.
the number of measles infections each year from 01 to 2014. CDC 
part of the reason for the resurgence is an increase in the number of parents who refuse to vaccinate their children against the virus. Despite a huge amount of scientific and medical evidence demonstrating both the safety and efficacy of the vaccine against measles, some parents refuse to vaccinate their children for reasons of personal conviction. The problem is compounded because many parents like mind are geographically grouped. For all of the state of California, the vaccine refusal rate is only 2.6%, indicating that most citizens are aware of the benefits of vaccination. However, a recent study of pediatric health records revealed that in some areas the vaccine refusal rate is as high as 13.5%. Other factors such as lack of time, education, or money can cause parents to fall behind inadvertently vaccinating their children.
Disneyland provides an ideal starting point for an epidemic. In such a world of atmosphere, a single infected person could come into contact with hundreds or thousands of people in a day. Although 0% of people are immunized, high infectivity of the virus will allow it to spread in the population by jumping between sensitive individuals.
How can I protect my children get measles?
Get vaccinated.
A drug the US government, once under the "extremely dangerous and unfit for human consumption "deserves a second chance, a study on rats suggests. Researchers report that a slow-release version of the compound reverses diabetes and diseases of nonalcoholic fatty liver disease (NAFLD), an untreatable condition that can lead to cirrhosis and liver cancer.
Diabetes has already become an epidemic. And up to 30% of people worldwide may have a metabolic disease less known but related NAFLD, in which the lipid-family of molecules that includes fats-accumulate in the liver. Although extra fat often causes some problems, about 10% to 20% of people develop non-alcoholic steatohepatitis (NASH), a serious disease in which inflammation and scarring can cause cancer and liver failure. So far, there are no drugs approved to treat either condition. "This is one of the largest unmet needs in medicine today," said Rohit Loomba hepatologist at the University of California, San Diego.
To meet this need, endocrinologist Gerald Shulman School of medicine and colleagues at Yale University suggested resurrecting a drug with a dark history. 2,4-dinitrophenol (DNP) Originally used as an industrial chemical and explosive, the compound has attracted attention researchers after the french munitions workers were exposed to high levels of it during World War one frequent consequence of this exhibition was losing weight even another consequence was sometimes death. after d other research has suggested that the compound induced obese people to lose weight, drug companies in the 1930s included DNP in diet pills that were available without a prescription. the Food and drug Administration (FDA) has banned compound at the end of the decade, however, as it caused side effects such as cataracts and was responsible for a death grip.
Despite its bad reputation, DNP has some virtues. By modifying the activity of mitochondria power plants that provide cells with energy, it forces the body to burn fat. It provides other metabolic benefits as well. For example, people with NAFLD or diabetes usually have insulin resistance, which means that their cells do not respond normally to the hormone that controls sugar levels in the blood. However, when Shulman and colleagues DNP fed to rats, they found that the drug has stimulated the insulin sensitivity of the animals.
The researchers decided to design a safer version of DNP that would retain its advantages. First, they tried to limit the effects of the drug by creating a version that is active mainly in the liver. In a study published in 2013, researchers have shown that this version of the drug was about one-tenth as toxic as the DNP standard. In addition, the targeted drug reduced fat accumulation in the liver of rats that had NAFLD and improve the insulin sensitivity of the animals.
But the researchers thought they could do better. In their new study, they packed the original form of DNP in a pill that dissolves and releases the drug for 12 to 24 hours slowly. This strategy reduces the amount of drug in the bloodstream. When fed to rats that eat a diet low in fat and loaded develop their own version of NAFLD, the slow release drug reduced their liver lipid levels of about 0%, Shulman and his colleagues report online today in Science . Rodents who have used drugs have also seen improvements in their amounts of insulin sensitivity and glucose in the blood. In rats with NASH, the drug reduced fibrosis, scarring can cause cirrhosis and liver failure. The team also showed that reversed diabetes in rats. Comparing the doses that provide these benefits with the doses that cause side effects, the researchers determined that the slow-release version is safer than the drug targeted liver.
The study suggests that this softer version of DNP could be useful for treating diabetes and NAFLD, says Shulman. It reduces fat accumulation and corrects defective liver metabolism of glucose, so "it is the cause of these diseases." He and his colleagues plan further studies on the drug animals and hope to pass safety tests in people.
Sean Koppe hepatologist at the University of Illinois Hospital & Health Sciences System in Chicago said the results warrant testing the drug in humans. "They show that it has a wide window between therapeutic and toxic levels," he said. Loomba said the drug's ability to slow fibrosis, a characteristic of NASH, is encouraging, and it also supports load safety testing in people. "These preclinical data is very exciting," he said.
Koppe Loomba and agree that if DNP proves to be safe and effective in other trials, it could gain FDA approval, despite its history. prohibited drugs have a prior return. The first example is thalidomide, which was banned in 1960 because it caused birth defects, but has now found a niche in the treatment of cancer and leprosy.
Tokyo- Investing in the world's ability to respond epidemic "is really invest in the resilience of the global economic system," said Group President of the World Bank Jim Yong Kim today in advance of an international meeting in Japan.
Kim met with reporters in Tokyo before a conference on the reduction of the UN disaster risk which begins tomorrow in Sendai. Last October, after what he called a global response "late, insufficient and slow" to the Ebola epidemic, Kim launched the idea of an emergency installation pandemic. Today, he expanded the idea, including why the World Bank should be involved.
He said natural disasters and epidemics have a devastating impact on economic growth, with countries and least able to afford it bear the greatest burden citizens. The World Bank estimated that the Ebola epidemic takes a bite of $ 1.6 billion on the economies of Guinea, Liberia and Sierra Leone. "Before the beginning of the epidemic, the three countries had some of the highest [economic] The growth rates in the world, and now their rates should be near or below zero," he said
the World Bank has committed over $ 500 million to deal with the crisis, but the money began to flow 8 months after the start of epidemic, he said. "We need to respond more quickly to the next outbreak," he said. And that requires better planning.
The emergency department pandemic proposed to build on lessons Ebola from the crisis. "the World health Organization [WHO] must be the center of the response," said Kim, a physician and anthropologist who once headed the HIV / AIDS department of wHO. It said the WHO response capacity has deteriorated in recent years and needs to be restored. "If we had enough money to WHO, I think we could have put the Ebola outbreak much earlier, "he said.
Kim said the response to larger epidemics, he considered inevitable, require coordination between a number of UN agencies. For example, the World Food Programme could provide expertise in moving supplies, while the United Nations Children's Fund could apply his knowledge of working with basic health networks. But these roles must be defined in advance, he said. He would also see the creation of a large reserve corps of doctors and nurses for emergency duty and periodic exercises "to be ready for us when major events occur," he said.
Planning a major health emergency should involve financial and business stakeholders in the public and private sectors, he noted, that epidemics can wreak havoc on the economy. Bring the table to insurance companies will help focus attention on the economic risks of disease outbreaks, he argued, and pharmaceutical, transportation and communications companies all have a vital role to play in response to a epidemic disease.
Kim said he was struggling to attract the attention of finance ministers and bank officials when he worked in public health. But now that he is president of the World Bank, he said, "they should listen."
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See the full textthe Ebola epidemic in West Africa has caused enormous suffering, but scientists also see it as a chance to test experimental therapies that could save lives in the future. With the decreasing number of cases, however, it becomes less likely that all tests will reach a conclusion. Now scientists are wondering whether some tests must be stopped so that the most promising therapies tests that only now available have a better chance to reach a conclusion. A group of experts from the World Health Organization gave zmapp TKM-Ebola and the highest priority, but in a recent meeting did not call for ongoing studies of favipiravir and convalescent blood arrest. The experts convince a group of Italian doctors to test zmapp instead of amiodarone heart drugs and criticized an interferon test has now begun in Guinea.
Mice and rats communicate in the ultrasonic frequency range, and it is thought that cats have evolved the ability to hear these acute squeals better hunt their prey. Now, a new study suggests that sensitivity to higher frequency sounds can cause seizures in some older cats. After receiving reports of the problem, dubbed the "syndrome of Tom and Jerry" because of the way the cartoon cat is often surprised by the sounds, the researchers surveyed cat owners and reviewed the medical records of their pets, looking for an overview of the types and times of crises and sounds that provoked them. in 96 cats, they found evidence of the syndrome they call cats audiogenic reflex seizures. the most common types crumpling of triggering input sounds included tinfoil, rattling a metal spoon on a ceramic feeding bowl, and tinkling glass. the severity of the seizure ranged from brief twitching to more severe episodes where the cat has lost consciousness and stiff and jerky for several minutes, the researchers report online today in the Journal of Feline Medicine and Surgery both pedigree and nonpedigree cats were susceptible, even if race was common. Thirty of 96 cats were Burmese (photo). Because seizures coincided with old age, the average age of onset was 15 years, veterinarians might miss the mess while dealing with other feline health problems, researchers say. Minimize exposure to problematic sounds, preliminary clinical trials with levetiracetam-a drug anticonvulsant used to control epilepsy in a small sample of the cats seemed to help limit the occurrence of crises.
