At the sleep research meeting in San Antonio this year, there were signs of an impending pharmaceutical arms race in the realm of narcolepsy.
The big fish in a small pond, Jazz Pharmaceuticals, was preparing to market its recently FDA-approved medication: Sunosi/solriamfetol. Startup Harmony Biosciences was close behind with pitolisant, already approved in Europe. On the horizon are experimental drugs designed to more precisely target the neuropeptide deficiency in people with classic narcolepsy type 1 Read more
HIV researchers are becoming increasingly bold about using the “cure” word in reference to HIV/AIDS, even though nobody has been cured besides the “Berlin patient,” Timothy Brown, who had a fortuitous combination of hematopoetic stem cell transplant from a genetically HIV-resistant donor. Sometimes researchers use the term “functional cure,” meaning under control without drugs, to be distinct from “sterilizing cure” or “eradication,” meaning the virus is gone from the body. A substantial obstacle is that HIV integrates into the DNA of some white blood cells.
HIV cure research is part of the $35.6 million, five-year grant recently awarded by the National Institutes of Health to Yerkes/Emory Vaccine Center/Emory Center for AIDS Research. Using the “shock and kill” approach during antiviral drug therapy, researchers will force HIV (or its stand-in in non-human primate research, SIV) to come out of hiding from its reservoirs in the body. The team plans to test novel “latency reversing agents” and then combine the best one with immunotherapeutic drugs, such as PD-1 blockers, and therapeutic vaccines.
The NIH also recently announced a cluster of six HIV cure-oriented grants, named for activist Martin Delaney, to teams led from George Washington University, University of California, San Francisco, Fred Hutchinson Cancer Research Center, Wistar Institute, Philadelphia, Beth Israel Deaconess Medical Center and University of North Carolina. Skimming through the other teams’ research plans, it’s interesting to see the varying degrees of emphasis on “shock and kill”/HIV latency, enhancing the immune response, hematopoetic stem cell transplant/adoptive transfer and gene editing weaponry vs HIV itself.
Rep. Tom Price (R-GA) expressed support for strong federal funding of scientific and biomedical research in a town-hall-type meeting Wednesday with Emory faculty and students, organized by the graduate student group Emory Science Advocacy Network.
Price tied a major expansion of federal funding for scientific research to reform of entitlements such as Medicare and Social Security (like this). Asked whether he could envision a large increase in the National Institutes of Health budget, comparable to the doubling in funding that occurred in the 1990s, Price replied: “In the near term, I don’t see it.”
Price with Emory Science Advocacy Network officers/members
Price also advocated streamlining the Food and Drug Administration’s approval processes for new antibiotics and medical devices, and giving scientists more discretion in how federal research dollars are allocated.
In a question-and-answer session, Emory ethnobotanist Cassandra Quave urged Price to have Congress give increased attention to the problem of antibiotic resistance, in which some bacterial infections are becoming difficult to treat.
“Yes, we need more resources going into this,” Price said, going on to support a “dual track” approval process for new antibiotics.
Price expressed concerns that the United States’ role as a leader in medical innovation was waning, because of regulatory constraints that drive devices such as heart valves to be tested elsewhere first.
“We’re already losing bright minds,” he said, citing how colleagues from other surgical specialties were visiting other countries to learn new techniques.
Price, who represents parts of Cobb, Dekalb and Fulton counties, was appointed chairman of the House Budget Committee at the end of 2014, replacing Rep. Paul Ryan (R-WI).
Before his election to Congress in 2004, Price was an orthopedic surgeon. He grew up and went to medical school in Michigan, and came to Georgia for his orthopedic surgery residency at Emory. He was an assistant professor at Emory and medical director of the Orthopedic Clinic at Grady Memorial Hospital, while he was a member of the State Senate. Read more
Biomarkers circulating in the bloodstream may serve as a predictive window for recurrent stroke risk and also help doctors accurately assess what is happening in the brains of patients with acute traumatic brain injury (TBI).
Michael Frankel, MD
Researchers at Emory University School of Medicine, led by principal investigator Michael Frankel, MD, Emory professor of neurology and director of Grady Memorial Hospitalâ€™s Marcus Stroke & Neuroscience Center, are studying biomarkers as part of two ancillary studies of blood samples using two grants from the National Institutes of Health.
In the $1.47 million, four-year grant called â€œBiomarkers of Ischemic Outcomes in Intracranial Stenosisâ€ (BIOSIS), Emory researchers are analyzing blood samples from 451 patients from around the country who were enrolled in a study known as SAMMPRIS (Stenting and Aggressive Medical Management for Preventing Recurrent stroke in Intracranial Stenosis), the first randomized, multicenter clinical trial designed to test whether stenting intracranial arteries would prevent recurrent stroke.
Researchers in the SAMMPRIS study recently published their results in the New England Journal of Medicine, showing that medical management was more effective than stenting in preventing recurrent strokes in these patients. Frankel’s BIOSIS research team is using blood samples from these same patients to continue learning more about the molecular biology of stroke to predict risk of a stroke occurring in the future.
â€œOur goal is to learn more about stroke by studying proteins and cells in the blood that reflect the severity of disease in arteries that leads to stroke. If we can test blood samples for proteins and cells that put patients at high risk for stroke, we can better tailor treatment for those patients,â€ says Frankel.
Patients with narrowed brain arteries, known as intracranial stenosis, have a particularly high risk of disease leading to stroke. At least one in four of the 795,000 Americans who have a stroke each year will have another stroke within their lifetime. Within five years ofÂ a firstÂ stroke,Â the risk for another stroke can increase more than 40 percent. Recurrent strokes often have a higher rate of death and disability because parts of the brain already injured by the original stroke may not be as resilient.
The other study, â€œBiomarkers of Injury and Outcome in ProTECT IIIâ€ (BIO-ProTECT)” is a $2.6 million, five-year NIH grant in which Frankelâ€™s team will use blood to determine what is happening in the brain of patients with acute TBI.Â The blood samples are from patients enrolled in the multicenter clinical trial ProTECT III (Progesterone for Traumatic brain injury, Experimental Clinical Treatment), led by Emory Emergency Medicine Professor, David Wright, MD, to assesses the use of progesterone to treat TBI in 1,140 patients at 17 centers nationwide.
In the BIO-ProTECT study, Emory is collaborating with the Medical University of South Carolina, the University of Pittsburgh, the University of Michigan and Banyan Biomarkers.
TBI is the leading cause of death and disability among young adults in the US and worldwide. According to the Centers for Disease Control and Prevention, approximately 1.4 million Americans sustain a traumatic brain injury each year, leading to 275,000 hospitalizations, 80,000 disabilities, and 52,000 deaths.
Acute TBI leads to a cascade of cellular events set in motion by the initial injury that ultimately lead to cerebral edema (swelling of the brain), cellular disruption and sometimes death. Tissue breakdown leads to the release of proteins into the bloodstream. These proteins may serve as useful biomarkers of the severity of the injury and perhaps provide useful information about response to treatment.
Using the large patient group in the ProTECT III trial, the researchers hope to validate promising TBI biomarkers as predictors of clinical outcome and also evaluate the relationship between progesterone treatment, biomarker levels and outcome.
â€œIf we can better determine the amount of brain injury with blood samples, we can use blood to help doctors better assess prognosis for recovery, and, hopefully whether a patient will respond to treatment with progesterone,â€ says Frankel.
Researchers at Emory University and the University of Georgia have received funding from the National Institutes of Health to study the neurobiological mechanisms for how regular aerobic exercise may prevent drug abuse relapse. The grant is for $1.9 million over the next five years.
â€œThis research will provide new insight into how regular exercise may attenuate drug abuse in humans,â€ Weinshenker says â€œMore importantly, it may reveal a neural mechanism through which exercise may prevent the relapse into drug-seeking behavior.â€
During the study, Weinshenker and UGA co-investigator Philip Holmes, professor of psychology in the Franklin College of Arts and Sciences, will measure exercise-induced increases of the galanin gene activity in the rat brain.
Vivian Pinn, MD, associate director for research on womenâ€™s health, and director of the Office of Research on Womenâ€™s Health at NIH, opened the meeting with Emoryâ€™s conference chair, Nanette Wenger, MD, professor of medicine (cardiology), Emory School of Medicine, and chief of cardiology at Grady Memorial Hospital.
Nanette K. Wenger, MD
In a career that spans more than 50 years, Wengerâ€™s dedication to reducing womenâ€™s disability and death from cardiovascular disease has made her one of the countryâ€™s most-respected experts on coronary heart disease in women. In 2009, Wenger received the Lifetime Achievement Award from the American College of Cardiology.
Although Wenger has earned dozens of awards in her celebrated career, she says her greatest professional achievement has been to help change a major paradigm in cardiology: the assumption that heart disease affects only men. A half a century ago heart disease was thought of as a “man’s disease.”