Sarepta Therapeutics has been winning over investors with rising sales of its drug to treat Duchenne muscular dystrophy and a promising pipeline of drugs targeted at the fatal muscle-wasting disease, Andrew Bary writes in this week’s edition of Barron’s. Part of the optimism surrounding Sarepta is that it can bring to market two drugs similar to Exondys 51, which treats about 13% of DMD patients, he notes, adding that these drugs – casimersen and golodirsen – target mutations at other points on the dystrophin gene and could treat another 16% of DMD patients
Saturday, May 5, 2018
Phase 2 clinical study of universal influenza vaccine begins in US
A Phase 2 clinical trial of an investigational universal influenza vaccine intended to protect against multiple strains of the virus has begun in the United States. The study is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and is being conducted at four U.S. sites that are part of the NIAID-funded Vaccine and Treatment Evaluation Units (VTEUs). The trial is testing an experimental vaccine called M-001 for safety and its ability to produce potentially broad protective immune responses, both on its own and when followed by a standard, licensed seasonal influenza vaccine.
Influenza viruses mutate constantly, resulting in the emergence of viruses that may not always match those targeted by seasonal and pre-pandemic influenza vaccines. Seasonal influenza vaccines are made anew each year to match the strains predicted to circulate in the upcoming season. To receive the best protection against influenza, people must be vaccinated annually. However, if a particular influenza strain changes in an unanticipated way, or a different strain from that included in the vaccine spreads widely, the seasonal influenza vaccine may not be sufficiently protective. Each year, seasonal influenza sickens millions in the United States and results in 140,000 to 710,000 hospitalizations and between 12,000 and 56,000 deaths, according to the Centers for Disease Control and Prevention.
Influenza pandemics occur when a novel influenza strain for which people have little to no protection begins to spread among humans and present a greater public health threat than seasonal influenza. For example, the 1918 influenza pandemic killed at least 50 million people worldwide. An ideal universal influenza vaccine would provide durable protection for all age groups against multiple influenza strains, including those that might cause a pandemic.
“The 2017-2018 influenza season in the United States was among the worst of the last decade and serves as a reminder of the urgent need for a more effective and broadly protective influenza vaccine,” said NIAID Director Anthony S. Fauci, M.D. “An effective universal influenza vaccine would lessen the public health burden of influenza, alleviate suffering and save lives. There are numerous paths of inquiry that the scientific community is pursuing, with each new study yielding more critical information and bringing us closer to our shared goal.”
The new trial is being led by principal investigator Robert L. Atmar, M.D., of Baylor College of Medicine in Houston. The trial will test the M-001 vaccine candidate, developed and produced by BiondVax Pharmaceuticals based in Ness Ziona, Israel. The experimental M-001 vaccine contains antigenic peptide sequences shared among many different influenza viruses. Theoretically, it could protect against many current and emerging strains of influenza. Six previous clinical trials involving a total of 698 participants conducted by BiondVax in Israel and Europe indicated that the vaccine candidate was safe, well-tolerated and produced an immune response to a broad range of influenza strains.
The new study will enroll up to 120 healthy volunteers between the ages of 18 and 49 years. Participants will be assigned randomly to receive either two doses of the experimental vaccine or a placebo. They will be vaccinated twice, receiving one dose (1 mg; 0.4 milliliters) of M-001 or placebo via intramuscular injection on the first day and a second dose 22 days later. Approximately 172 days later, all participants will receive an approved seasonal influenza vaccine. During periodic additional clinic visits throughout the course of the study, blood will be drawn from study volunteers to evaluate their immune responses to both the experimental vaccine and to the seasonal vaccine. Each participant will be followed for approximately seven months.
The trial will take place at four NIAID-funded VTEUs. Patients will be enrolled at the Baylor College of Medicine; the University of Iowa in Iowa City; and Cincinnati Children’s Hospital Medical Center. Laboratory support will be provided by Saint Louis University. For more information about the study, go to ClinicalTrials.gov, using the identifier NCT03058692 .
NIAID conducts and supports research–at NIH, throughout the United States, and worldwide–to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.
ObamaCare insurers in Virginia propose major premium hikes for 2019
Two of Virginia’s ObamaCare insurers are requesting significant premium hikes for 2019, according to initial filings released Friday.
Both Cigna and CareFirst BlueCross BlueShield cited policies advocated by the Trump administration, including the repeal of ObamaCare’s individual mandate, as part of its justifications for the increases.
Cigna is proposing an average premium increase of 15 percent for its 103,264 customers in Virginia, with a range of increases from 6.4 percent to 40 percent.
CareFirst is proposing a 64 percent increase for its approximately 4,500 customers in the commonwealth, citing an increase in sicker people entering the marketplace.
The pro-ObamaCare group Protect Our Care Campaign was quick to attack the administration following the news.
“Until we stop Republicans’ war on health care, health care experts predict that rates will keep rising by double digits,” said the group’s director, Brad Woodhouse. “D.C. Republicans should start working on bipartisan solutions to make coverage more affordable, instead of helping their friends in the insurance industry make another buck on the backs of hardworking Virginians.”
There are seven insurers participating in ObamaCare exchanges in Virginia for 2019, and only three so far have posted their filing requests. The third, Optima, has requested a 5 percent decrease for next year.
The rate increases requested Friday by Cigna and CareFirst are not necessarily what state officials will approve.
Cigna said it anticipates sicker people will be entering the market this year because of the repeal of the individual mandate, which required people to buy insurance or face a tax penalty. The insurer also cited “anticipated changes to regulations” involving short-term plans and association health plans as the reason for the premium increases.
The Trump administration is pushing to allow people to buy short-term health insurance for up to 12 months, lifting Obama-era restrictions that limited the coverage to a maximum of three months. Under such plans, people with pre-existing conditions can be charged more.
The plans also do not have to comply with ObamaCare mandates for covering certain services, such as mental health treatment or prescription drugs.
The Trump administration argues the 12-month plans would create an alternative for people who can’t afford ObamaCare-compliant plans that cover a comprehensive list of services.
In January, the administration issued rules expanding “association health plans,” which allow small businesses or self-employed individuals to band together to buy coverage. Republicans argue the move will provide cheaper options outside ObamaCare.
Final regulations on both types of insurance are expected later this year.
Pharmacists can play role in identifying frailty
As the number of older people in the United States continues to increase, pharmacists and health care professionals need to recognize and address the health care challenges associated with age, including frailty, according to an article published in Drug Topics.
Noting that standardized and validated screenings to diagnose frailty are lacking, health care providers must educate themselves about frailty. Using their frequent patient contact and accessibility, community pharmacists can identify, screen, and refer patients at risk of frailty or who are frail.
There is currently no consensus on how to define or screen for frailty, which can include physical and psychosocial dimensions. Community pharmacists can identify certain risk factors for frailty, including gender, low socioeconomic status, chronic illnesses, neuroendocrine dysfunctions, and certain medications and medication regimens. If pharmacists suspect a patient is frail or at risk of becoming frail, they should identify medications within the medication records that may increase the risk of frailty, increase fall risk, or escalate cognitive decline. Patient interview may be warranted as patients sometimes use more than one pharmacy. A call to the patient’s physician might be necessary in certain cases where the pharmacist has concerns about frailty, declining health, and associated potential health risks.
“Pharmacists who screen, identify, and refer patients in preliminary stages of frailty will help decrease health care costs, improve the patient’s quality of life, and increase patient survival,” according to the report.
More information: Abstract/Full Text
Gynecologic exams not necessary for all adolescent girls
Q: At what age should a teenage girl have her first Pap smear and gynecologic exam? Should this be done by a pediatrician or another provider?
A: Pelvic exams and Pap smears are not necessary in healthy, adolescent girls who are not sexually active and who do not have gynecologic symptoms or other concerns. The recommended age for young women who have not previously needed a Pap smear to begin having the test is 21. Primary care providers, such as those in family medicine or internal medicine, can perform that exam, or a gynecologist can perform the exam.
In the past, many health care providers used to recommend that teens have a Pap smear at 16 or 18. Now, however, the American College of Physicians does not recommend any routine gynecologic exams for teens at low risk of having the HPV or other sexually transmitted infections.
The purpose of a Pap smear—a procedure that collects cells from the cervix—is to check for changes in those cells that could be a sign of cancer and look for the presence of high-risk types of HPV that are known to cause cervical cancer.
HPV is the most common sexually transmitted infection in the U.S. Although most cases of HPV do not cause symptoms or lead to problems, certain strains of the sexually transmitted HPV are considered high risk, and can lead to cancer and potentially be deadly if left untreated. Two strains of the virus cause 70 percent of all cervical cancer. You can greatly reduce your risk of HPV infection and cervical cancer by getting the HPV vaccine. This anti-cancer vaccine most effectively builds immunity when it’s given between ages 9 and 14.
If you are not sexually active as a teen, in most cases there is no need for a Pap smear because your risk of cervical cancer is low, and you have no risk of HPV or other sexually transmitted infections. Once you begin having sex, a Pap smear may be appropriate. But even in many of those situations, Pap testing still may not need to begin until 21.
Traditionally, some health care providers required a pelvic exam—an evaluation of the vulva, vagina, cervix, ovaries, uterus, rectum and pelvis for any abnormalities—before they would prescribe contraceptives. Again, that is no longer the case. A pelvic exam is not needed to obtain birth control. For most healthy women, the first pelvic exam also can wait until 21.
For young women whose medical history puts them in a higher risk category for infection or cancer, a Pap smear or pelvic exam may be recommended at an earlier age. Those risk factors include a weakened immune system due to chemotherapy, organ transplant or long-term corticosteroid use; exposure to the medication diethylstilbestrol, also known as DES, before birth; and HIV infection.
Some symptoms also may trigger a pelvic exam, such as heavy, painful periods; pelvic pain or pressure; abnormal bleeding or discharge; and itching, pain or lesions in the vulva or vagina. In those situations, a pelvic exam may be necessary, but a Pap smear typically is not required.
When it is time to get a pelvic exam and Pap smear, it’s best to work with a health care provider you know and trust. Before the exam, ask your health care provider about what will happen during the exam and what you can expect. Some providers have anatomical models they can use to describe the procedures beforehand, so you can clearly see what will happen. These procedures are important exams, but they need not be a source of anxiety or fear. If you have questions or concerns, talk to your health care provider.
Explore further: Study questions reasons for routine pelvic examsBuffett targets CEO for Berkshire-Amazon-JPMorgan health venture soon
Warren Buffett on Saturday said he hoped a chief executive would be found within a couple of months for the healthcare company being set up by Berkshire Hathaway Inc, Amazon.com Inc and JPMorgan Chase & Co. to lower patient costs.
Speaking at the annual shareholder meeting of his Berkshire Hathaway Inc conglomerate, Buffett maintained that healthcare is a “tapeworm” on American businesses, hurting their ability to compete with rivals in other countries.
While there is no guarantee the venture between Amazon.com, Berkshire and JPMorgan, which collectively employ more than 1 million people, will succeed, Buffett said they are well positioned to try.
“I think we’ll have a CEO within a couple of months,” Buffett said. “We want our employees to get better medical services at lower cost … The resistance will be unbelievable, and if we fail, at least we tried.”
Buffett, famed for his love of junk food, has said spiralling healthcare costs are responsible for 18 percent of U.S. gross domestic product, up from 5 percent in 1960, and he wants to slash a few percentage points off.
“We are attacking an industry moat,” Buffett said.
Amazon.com’s reputation as a disruptor prompted investors in January, when the venture was announced, to sell shares of companies that might be hurt, including drugmakers, health insurers and pharmacy benefit managers.
Buffett said the goal was to challenge the entire healthcare industry, not individual segments.
Yet Charlie Munger, a Berkshire vice chairman, suggested that Democrats could take over Congress after November’s elections and push for a national healthcare system financed by a single payer. Munger predicted that such a change would harm those drug benefit managers.
Todd Combs, one of Buffett’s investment deputies and a JPMorgan director, is working on the joint venture with Marvelle Sullivan Berchtold, a managing director of JPMorgan, and Beth Galetti, a senior vice president at Amazon.com.
“We want our employees to get better medical services at lower cost,” Buffett said. “We’re certainly going to give it a shot. We’ll see what happens.”
Finding the ‘brain switch’ for courage
In the animal kingdom, vision is vital for survival. This important sense informs the brain about predators and other threats, and in turn, the brain generates an appropriate reaction: courage or fear, fight or flight.
But how does this process take place? How do animals — humans included — integrate visual information with the appropriate brain circuits that control firstly our emotional states, and afterward, our behavior and actions?
New research brings us closer to an answer. Scientists led by Andrew Huberman, an associate professor of neurobiology and ophthalmology at Stanford University School of Medicine in California, have found the brain circuits “responsible” for the decision to either fight or flee in the face of danger.
Although the study was conducted in mice, the findings are relevant for humans. In fact, the results have important implications for understanding and managing post-traumatic stress disorder (PTSD), addiction, and phobias.
Lindsey Salay is the first author of the paper, which has now been published in the journal Nature.
The brain circuit of fear
To examine the rodents’ response to a threat, Salay and team simulated the approach of a bird of prey and used the c-Fos neuronal marker to track the activity of the mice’s neurons.
The researchers found increased activity in neurons that were grouped in a structure called the ventral midline thalamus (vMT).
Using brain mapping, the scientists were able to see what sensory information comes in and what information goes out of the vMT.
They revealed that the vMT receives information from a wide range of brain areas that process internal states, such as that of fear, but that it sends information out very selectively, to only two main areas: the basolateral amygdala and the medial prefrontal cortex.
The amygdala processes fear, aggression, and other emotions, while the medial prefrontal cortexuses its executive function to modulate emotional responses. The area is also deeply involved in anxiety.
Additional analysis shed yet more light on the trajectory of the brain circuit involved in the rodents’ response to the ominous predator.
Apparently, a nerve tract starts from the “xiphoid nucleus” — a cluster of neurons in the vMT — and continues to the basolateral amygdala.
Another tract follows an analogous path, this time from the so-called nucleus reuniens — another cluster of neurons built around the xiphoid nucleus — and leading up to the medial prefrontal cortex.
‘Turning on’ the courage switch
Having observed this trajectory in the brain, the researchers wondered whether or not selectively inhibiting certain neurons along these pathways produce specific fight-or-flight reactions.
To find out, Salay and team stimulated only the activity of the xiphoid nucleus while confronting the rodents with the image of the bird of prey. This made the mice freeze in front of the predator.
After that, they stimulated the activity of the tract that goes from the nucleus reuniens to the medial prefrontal cortex. This caused a surprising reaction: the mice became aggressive, getting ready to defend themselves.
Senior investigator Huberman describes the rodents’ behavior as one of undeniable courage. “You could hear their tails thumping against the side of the chamber,” he explains. “It’s the mouse equivalent of slapping and beating your chest and saying, ‘OK, let’s fight!'”
A second experiment confirmed the results: stimulating exclusively the nucleus reuniens for half a minute before showing the predator produced the same behavioral response: rather than hiding, the mice rattled their tails and exposed themselves in unprotected areas, ready to fight.
Huberman says that the findings are highly relevant to humans, given that human brains have a similar structure to the vMT.
He suggests that people living with phobias, anxiety, or PTSD might soon benefit from the findings, as reducing the activity in their vMT or in the adjacent neuronal clusters may help these people to overcome their fears.
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