20 June 2014

Biomedicine in the USA: Top ten

Top 10 American research institutes in the field of biomedicine

The material was prepared by the portal MEDFARMCONNECT

Today, research institutes occupy an important place in the development of medicines. In particular, their role is significant in conducting preclinical research and the discovery of new substances, as Big Pharma companies continue to reduce their spending on research activities at a rapid pace. Although most of the institutes represented in the rating are engaged in conducting fundamental research, more and more attention is being paid in these institutions to how existing drugs or those drugs that have not appeared on the market due to insufficient safety of their use can be used in a new way.

Experts of the specialized portal FierceBiotech Research in their rating (The top 10 biomedical research institutes) consider the most successful research institutes in the United States, the list of which is submitted in accordance with the amount of funding from the National Institutes of Health (National Institutes of Health).

The material presents the most significant research on the development of medicines implemented by these research organizations in 2013.

1. Johns Hopkins UniversityThe university's funding from the National Institutes of Health last year amounted to $574.85 million.

In 2013, he was awarded 1,281 specialized awards. One of the most significant works in the field of biomedicine, conducted by Johns Hopkins University and published in the scientific publication Cell on October 24, was a study in which a new barrier in the treatment of HIV/AIDS was discovered. It was found that the number of potentially active latent forms of HIV in infected immune T-cells can be 60 times higher than the previously established level. This so-called latent reserve of viable proviruses remains after the use of antiretroviral therapy stops the replication of the virus and stops the course of the disease. Although these research results indicate that it may be much more difficult to overcome HIV/AIDS than expected, they may prompt scientists to turn to new alternative approaches for the development of HIV drugs. Hereditary diseases are also being studied at Johns Hopkins University. So, in September last year, in the journal Science Translational Medicine, a group of scientists published data from a study describing a substance, the introduction of a single injection of which allowed to normalize the development of the cerebellum in a mouse model of Down syndrome. It is believed that abnormalities in the cerebellum may be associated with this disease. The dose of the drug administered at birth, which reboots the Sonic hedgehog signaling pathway responsible for growth and development, allowed to normalize the growth of the cerebellum and contributed to better learning and memory in mice. Now researchers are considering additional possibilities for how to safely subdue the potential of the Sonic hedgehog signaling pathway for the treatment of Down syndrome. University scientists are also working on new methods of treating malignant tumors. The Johns Hopkins Kimmel Cancer Center at Johns Hopkins University has developed a therapy that uses cell lines from the tumors of the patients themselves, which can help the attending physicians choose the most effective chemotherapy drugs for them.

2. University of California at San Francisco (University of California, San Francisco)Its funding from the National Institutes of Health last year amounted to $501.65 million.

In 2013, the University of California was awarded 1,174 specialized awards. The University of California in San Francisco not only receives high funding from the US federal budget, but also enjoys authority among representatives of Big Pharma as a leading medical research institute. In March 2013, the American pharmaceutical company Pfizer attracted the University of California as a partner to work with its Centers for Therapeutic Innovation on the development of low-molecular-weight candidate drugs. Under the partnership agreement, Pfizer funds the University of California to conduct preclinical and clinical research programs on such drugs. If certain goals are achieved, Pfizer will help with the organization of clinical trials, and the University of California at San Francisco will receive agreed payments and royalties when such a drug goes on sale. Last June, researchers at the University of California, San Francisco, discovered a mechanism that explains why some patients have multiple sclerosis progressing to a severe stage faster than others. In a mouse model of the disease, the researchers found that such an early development of the disease is associated with the absence of the Tob1 gene in CD4+ T cells. The results of the trial, published in the Journal of Experimental Medicine, can help develop an effective diagnosis of the course and severity of multiple sclerosis in different patients and, accordingly, choose a personalized treatment. In another landmark study last year, California scientists have established how, by targeting the surface of a number of cells, scarring of liver, kidney and lung tissues in mice can be stopped and reversed. In the future, this method can be used to treat fibrosis in humans.

3. University of Washington (University of Washington)Funding for the University of Washington from the National Institutes of Health last year amounted to $454.27 million.

In 2013, the university was awarded 932 specialized awards. Last year, scientists from the University of Washington, together with researchers from Johns Hopkins University and Yale University, formed the online database PhenoDB, which should help doctors in the study of unusual genetic diseases. The database will register such personal information of patients as medical history, symptoms, etc. Doctors will have the opportunity to use the database to document cases of unusual genetic diseases, after which researchers from Johns Hopkins University and Baylor College of Medicine in Houston will decide which diseases deserve further study. Also last year, scientists at the University of Washington identified 207 human genes whose expression differs from normal immediately after infection with the newest coronavirus that causes Middle East respiratory syndrome (MERS). In addition, the researchers found that certain classes of drugs, including inhibitors of specific kinases and one type of glucocorticoids, affect some of these genes. Scientists believe that these drugs can block the replication of the coronavirus and stop the progression of the disease. The University of Washington is also known as a developer of diagnostic materials. In 2013, General Electric applied to the university to jointly create a small paper test to determine infectious diseases using a nasal smear. Such a one-time test, in particular, allows you to diagnose methicillin-resistant Staphylococcus aureus.

4. University of PennsylvaniaThe university's funding from the National Institutes of Health last year amounted to $451.19 million.

In 2013, the University of Pennsylvania was awarded 1081 specialized awards. The famous oncologist Carl June works at the University of Pennsylvania. Last December, he presented the latest data on cancer-fighting T-cells, a technology that was licensed by Novartis. Thus, a total of 19 out of 22 children with lethal forms of acute lymphoblastic leukemia achieved complete remission after the use of CAR T therapy – CTL019, targeted therapy aimed at cancer cells that secrete the surface protein CD19. Among 32 adult patients with chronic lymphocytic leukemia, 7 had complete remission and 15 had a response to treatment. Carl Jun hopes that thanks to his development it will be possible to introduce a new method of treating different types of leukemia. The scientist has already planned to conduct multicenter tests. The June edition of the journal Cell Reports last year describes that researchers from the University of Pennsylvania have made progress in preclinical trials of new types of pancreatic cancer treatments. Currently, there are no non-invasive methods of early detection of this malignant tumor. Using stem cells, scientists from the Perelman School of Medicine at the University of Pennsylvania have created a cell line of a patient with advanced ductal adenocarcinoma of the pancreas. According to them, this is the first human model that demonstrates the earlier progression of the disease.

5. University of MichiganIts funding from the National Institutes of Health last year amounted to $412.02 million.

In 2013, the University of Michigan was awarded 1021 specialized awards. Last year, researchers at the University of Michigan found that the antidepressant tranylcypromine (TCP) can contribute to the regression of sickle cell anemia in mice. Tranylcypromine blocks LSD1 molecules in erythrocytes, thereby increasing the production of fetal hemoglobin and, thus, neutralizing the negative effect of hemoglobin S, which is produced by sickle cells. It is too early to talk about the widespread use of tranylcypromine, but scientists are already going to conduct a study among adults with this disease. Currently, new opportunities for the use of existing drugs are one of the topical topics of biomedical research and researchers at the University of Michigan managed to find another method of using the drug, which has been on the market for 15 years. Together with scientists from the University of California, San Diego, they determined that the drug amlexanox prevented weight gain in mice. In the USA, this drug in the form of a paste is used to treat mouth ulcers, and in Japan it is approved in another dosage form for the treatment of asthma. In their study, scientists from the University of Michigan fed two groups of mice a high-calorie diet. After the animals developed obesity, one group was given injectable amlexanox. In this group, the mice showed weight loss, they got rid of obesity and they had type 2 diabetes.

6. University of PittsburghFunding for the University of Pittsburgh from the National Institutes of Health last year amounted to $396.73 million.

In 2013, the University of Pittsburgh was awarded 963 specialized awards. At the end of 2013, researchers at the University of Pittsburgh School of Public Health launched a database called Project Tycho. The authors digitized all reports on morbidity from 1888 to 2011 and created a publicly accessible database. Scientists use this database to show how the introduction of vaccines has affected the spread of polio, measles, rubella and other currently controlled diseases. As noted by Dr. Donald Burke, this database has gathered together information about infectious diseases so that everyone has the opportunity to get acquainted and use these data. Last year, researchers from the Pittsburgh Children's Clinic and the University of Pittsburgh School of Public Health discovered a molecule that controls pulmonary infection in tuberculosis. They found that granulomas containing ectopic lymphoid structures similar to lymph nodes can effectively suppress tuberculosis. At the same time, granulomas that do not contain them are associated with active tuberculosis. Also in 2013, researchers from the University of Pittsburgh in collaboration with scientists from the Mount Sinai School of Medicine (Mount Sinai Medical Center) discovered a mechanism that regulates the replication of insulin-producing cells beta cells of the pancreas. This discovery can help in the treatment of type I and type II diabetes.

7. University of North Carolina at Chapel HillIts funding from the National Institutes of Health last year amounted to $383.75 million.

In 2013, the University of North Carolina was awarded 892 specialized awards. University researchers are using genetically modified mouse models of the disease in order to determine what response people will have to different combinations of chemotherapy drugs for the treatment of breast cancer. Last year, on genetically modified mice, they identified biomarkers of molecular subtypes of human breast cancer, in particular, those against which there are not enough available drugs. As Charles Perou, a leading researcher and representative of the Lineberg Cancer Center at the University of North Carolina, Charles Perou, noted, these studies are an excellent example of how well–matched mouse models can tell about the nature of human disease. In this case, scientists have spent years trying to find the appropriate models for specific human subtypes, and then treat animals with drugs identical to those used to treat cancer in humans. They have already managed to develop biomarkers of the response to treatment in mice, which will work in humans as well. In addition, the University of North Carolina, in collaboration with the Massachusetts Institute of Technology, is working on how to quickly create such nanoparticles for drug delivery that would be of various sizes and shapes, and their coating could perform several different functions at once.

8. University of California at San Diego (University of California, San Diego)Funding for the University of North Carolina from the National Institutes of Health last year amounted to $383.75 million.

In 2013, the University of North Carolina was awarded 892 specialized awards. University researchers are using genetically modified mouse models of the disease in order to determine what response people will have to different combinations of chemotherapy drugs for the treatment of breast cancer. Last year, on genetically modified mice, they identified biomarkers of molecular subtypes of human breast cancer, in particular, those against which there are not enough available drugs. As Charles Perou, a leading researcher and representative of the Lineberg Cancer Center at the University of North Carolina, Charles Perou, noted, these studies are an excellent example of how well–matched mouse models can tell about the nature of human disease. In this case, scientists have spent years trying to find the appropriate models for specific human subtypes, and then treat animals with drugs identical to those used to treat cancer in humans. They have already managed to develop biomarkers of the response to treatment in mice, which will work in humans as well. In addition, the University of North Carolina, in collaboration with the Massachusetts Institute of Technology, is working on how to quickly create such nanoparticles for drug delivery that would be of various sizes and shapes, and their coating could perform several different functions at once.

9. Stanford University (Stanford University)Stanford's funding from the National Institutes of Health last year amounted to $357.81 million.

In 2013, the university was awarded 828 specialized awards. Using computer prediction methods in drug development, researchers from Stanford University School of Medicine have discovered a new method of treating lung cancer. Thus, it was found that two little-used drugs imipramine (imipramine) and promethazine (promethazine), approved by the U.S. Food and Drug Administration (FDA) as antidepressants, have anti-cancer properties on cell culture and animal models of small cell lung cancer. Both drugs contributed to the death of cancer cells. Also in the study, these drugs were effective in human small cell lung cancer in mice that developed resistance to chemotherapy. In another study of a new use of medicines, scientists from Stanford University found that formoterol, an FDA–approved drug for the treatment of asthma, can improve cognitive functions in Down syndrome. This effect of formoterol is associated with its ability to strengthen neural connections in the hippocampus – the area of the brain responsible for learning. These conclusions were made during experiments with laboratory mice that were genetically implanted with Down syndrome.

10. Duke UniversityIts funding from the National Institutes of Health last year amounted to $350.25 million.

In 2013, Duke University was awarded 753 specialized awards. Last year, Duke University scientists found a way to prevent epilepsy in mice that have epileptic seizures for a long period of time. They found that inhibition of the TrkB receptor of the nervous system can prevent the manifestation of the disease. As noted by James McNamara, professor of neuroscience at Duke University, there are currently no effective means for the preventive treatment of disorders of the nervous system. The problem is that scientists have not yet been able to identify a molecular target for the treatment of epilepsy, which is why there are no drugs for its prevention now. It is assumed that the receptor isolated by a group of Duke University scientists may play a key role in the development of epilepsy. In 2013, Duke University received a $2 million grant to conduct research aimed at combating antibiotic resistance. Representatives of Duke University and the University of California at San Francisco will consider areas of work and create a research network that will explore ways to defeat bacteria resistant to modern antibiotics.

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