China has halted imports from European salmon suppliers amid fears
they might be linked to a coronavirus outbreak at a Beijing market,
although experts say the fish itself is unlikely to carry the disease.
State-run newspapers reported the virus was discovered on chopping
boards used for imported salmon at Beijing’s Xinfadi market, the source
of a cluster of infections that has sparked fears of a second wave of
the pandemic in China.
The reports prompted major supermarkets in Beijing to remove salmon from their shelves.
“We can’t send any salmon to China now, the market is closed,” Regin
Jacobsen, CEO of Oslo-listed salmon supplier Bakkafrost, told Reuters.
“We have stopped all sales to China and are waiting for the situation
to be clarified,” said Stein Martinsen, head of sales and marketing at
Norway Royal Salmon.
Genetic traces of the virus from the Beijing market outbreak suggested it could have come from Europe.
Keith Neal, an emeritus professor of the epidemiology of infectious
diseases at Britain’s University of Nottingham, said any link to salmon
was likely the result of cross contamination.
“Markets can be crowded places, so like in Wuhan, (they) help fuel
spread,” he said, referring to the Chinese city where the virus
originated.
Neal said finding a link to Europe was not surprising, given the global spread of the virus.
“China gave the world this virus and it was always very likely to
give it back to them. Finding a strain prevalent in Europe probably
reflects people returning to China after travelling to Europe,” he said.
Norway’s Food Safety Authority said there was no evidence fish could be infected.
Shares in Norway Royal Salmon, Faroe Islands-based Bakkafrost and Norway’s Mowi and Salmar were down 3-5% at 1225 GMT.
Both Bakkafrost and Norway Royal Salmon said employees had been tested for the virus, and none had tested positive.
China accounts for about 5% of global salmon demand, according to Bakkafrost.
Order cancellations were limited to China and had not affected other
markets, said the Norwegian Seafood Council, a marketing firm.
https://www.marketscreener.com/BAKKAFROST-6103708/news/China-halts-European-salmon-imports-over-suspected-link-to-virus-outbreak-30770936/?countview=0
Monday, June 15, 2020
Blood donors will get results of coronavirus antibody test, Red Cross says
The American Red Cross will test all blood, platelet and plasma
donations for COVID-19 antibodies so donors can learn whether they’ve
been exposed to the new coronavirus.
“We recognize that individuals and public health organizations desire more information about COVID-19, and as an organization dedicated to helping others, the Red Cross is fortunate to be able to help during this pandemic,” Chris Hrouda, president of Red Cross Biomedical Services, said in a news release.
The test—authorized by the U.S. Food and Drug Administration—may indicate if a person’s immune system has produced antibodies to the coronavirus, even if they didn’t have symptoms of COVID-19. A positive test result does not confirm immunity to the virus, and the test is not meant to diagnose illness, the Red Cross said.
For the next few months, blood, platelet and plasma donations will be tested using samples obtained at the time of donation. The samples will also undergo routine screening and infectious disease testing.
Now that surgeries and treatments put on hold by the pandemic have resumed, the Red Cross said there’s an urgent need for blood donations. At the same time, blood drives continue to be canceled as many businesses and community groups restrict the number of people at their locations.
The Red Cross noted that blood is perishable and can’t be stockpiled.
To ensure donor and staff safety during the pandemic, the organization has put several precautions into place.
They include checking temperatures to make sure staff and donors are healthy; providing hand sanitizer; social distancing; ensuring staff and donors wear face coverings; routinely disinfecting surfaces, equipment and donor-touched areas; wearing gloves and changing them often; and using sterile collection sets and an aseptic scrub for every donation.
If you’ve made an appointment to give blood, you should postpone it if you’re not feeling well, the Red Cross said.
https://medicalxpress.com/news/2020-06-blood-donors-results-coronavirus-antibody.html
“We recognize that individuals and public health organizations desire more information about COVID-19, and as an organization dedicated to helping others, the Red Cross is fortunate to be able to help during this pandemic,” Chris Hrouda, president of Red Cross Biomedical Services, said in a news release.
The test—authorized by the U.S. Food and Drug Administration—may indicate if a person’s immune system has produced antibodies to the coronavirus, even if they didn’t have symptoms of COVID-19. A positive test result does not confirm immunity to the virus, and the test is not meant to diagnose illness, the Red Cross said.
For the next few months, blood, platelet and plasma donations will be tested using samples obtained at the time of donation. The samples will also undergo routine screening and infectious disease testing.
Now that surgeries and treatments put on hold by the pandemic have resumed, the Red Cross said there’s an urgent need for blood donations. At the same time, blood drives continue to be canceled as many businesses and community groups restrict the number of people at their locations.
The Red Cross noted that blood is perishable and can’t be stockpiled.
To ensure donor and staff safety during the pandemic, the organization has put several precautions into place.
They include checking temperatures to make sure staff and donors are healthy; providing hand sanitizer; social distancing; ensuring staff and donors wear face coverings; routinely disinfecting surfaces, equipment and donor-touched areas; wearing gloves and changing them often; and using sterile collection sets and an aseptic scrub for every donation.
If you’ve made an appointment to give blood, you should postpone it if you’re not feeling well, the Red Cross said.
https://medicalxpress.com/news/2020-06-blood-donors-results-coronavirus-antibody.html
COVID-19 hospitalizations may mean big out-of-pocket medical costs for many
If past hospitalizations for pneumonia and other respiratory
illnesses are any guide, many Americans could face high out-of-pocket
medical costs for COVID-19 hospitalizations despite the fact that many
insurers have waived their cost-sharing requirements, a study from
researchers at Johns Hopkins Bloomberg School of Public Health suggests.
For their study, the researchers analyzed out-of-pocket costs for pneumonia and other upper respiratory illness hospitalizations from January 2016 through August 2019 as a potential indicator of likely COVID-19 costs. The researchers found that these out-of-pocket costs were particularly high for so-called consumer-directed health plans—which typically feature lower premiums, compared to standard plans, but higher deductibles that can be paid via tax-advantaged health savings accounts.
The findings were published online June 15 in the American Journal of Preventive Medicine.
Many big-name health insurers have voluntarily waived out-of-pocket cost sharing for COVID-19 treatment. However, employer-sponsored “self-insured” health insurance plans are not required to adhere to such waivers. Thus, tens of millions of Americans have high-deductible insurance plans that, in cases of COVID-19 hospitalization, may expose them to relatively high out-of-pocket costs.
“Congress is now debating whether or not to require all plans to waive cost sharing related to COVID-19 treatment. Our findings suggest that they might want to take action to broaden cost-sharing waivers to include these self-insured plans, because those out-of-pocket costs are otherwise going to be high for many people,” says study lead author Matthew Eisenberg, Ph.D., an assistant professor in the Department of Health Policy and Management at the Bloomberg School.
COVID-19, the disease that is caused by the novel coronavirus SARS-CoV-2, often features pneumonia in moderate and severe cases. It has struck more than 7 million people worldwide and killed more than 400,000 since its initial outbreak in China late last year. The U.S. has had the largest number of cases and deaths by far, with more than 250,000 hospitalizations for moderate to severe COVID-19, according to the Centers for Disease Control and Prevention.
To help reduce the cost burden of COVID-19 testing, Congress has banned insurance co-pays, out-of-pocket payments, and other cost sharing for COVID-19 testing, effectively requiring most insurers to cover the full cost. Many private insurance companies also have voluntarily extended such cost-sharing waivers for COVID-19 treatment. But about 60 percent of employer-sponsored health insurance plans are self-insurance plans. In such plans, the employer assumes the financial risk of medical care for their employees rather than the insurer. Such companies are not obliged to waive cost-sharing for COVID-19 treatment, and thus many of the millions of Americans insured under these plans could face high out-of-pocket costs, especially if their plans are high-deductible, consumer-directed plans.
To get a sense of the likely cost burden on patients hospitalized for COVID-19, Eisenberg and colleagues examined de-identified insurance claims for 34,395 unique hospitalizations from January 2016 through August 2019. They loked at out-of-pocket costs incurred by people who had been hospitalized during the 2016-2019 study period with pneumonia, acute bronchitis, lower respiratory infections, and acute respiratory distress syndrome. (Claims data on actual COVID-19 cases were not available in the database at the time of the study.) The cases examined did not include those for people ages 65 and over, who are normally covered by Medicare. The out-of-pocket costs included deductible payments, copayments, and coinsurance payments.
The researchers found that average out-of-pocket spending for the 2016-2019 study period for these respiratory hospitalizations was $1,961 for patients with consumer-directed plans versus $1,653 for patients in traditional, usually smaller-deductible plans.
The out-of-pocket cost gap was lowest for older patients age 56 to 64, and greatest—$2,237 vs. $1,685—for patients 21 and younger. The analysis was not designed to examine why the cost gap varied inversely with patient age, but one possible explanation proposed by the researchers was that, since younger patients are healthier on average, their hospitalizations may reflect more serious and thus more costly illness.
“For people already struggling with serious respiratory illness due to COVID-19, the added stress of managing large medical bills could be devastating at this moment when so many Americans are experiencing major financial strain and job loss,” says study co-author Alene Kennedy-Hendricks, Ph.D., assistant scientist in the Department of Health Policy and Management at the Bloomberg School. “This is a critical area to watch and to consider for policy action moving forward.”
Given that self-insured plans cannot be regulated at the state level, the researchers suggest that federal policymakers should consider waiving COVID-19 cost sharing for self-insured plan policyholders. They note too that while the likely out-of-pocket costs would be greatest for people enrolled in high-deductible, consumer-directed plans, the findings indicate that people in traditional plans also could face fairly large out-of-pocket costs.
“Financial Risk for COVID-19-like Respiratory Hospitalizations in Consumer-Directed Health Plans” was written by Matthew Eisenberg, Colleen Barry, Cameron Schilling, and Alene Kennedy-Hendricks.
Follow the latest news on the coronavirus (COVID-19) outbreak
https://medicalxpress.com/news/2020-06-covid-hospitalizations-significant-out-of-pocket-medical.html
For their study, the researchers analyzed out-of-pocket costs for pneumonia and other upper respiratory illness hospitalizations from January 2016 through August 2019 as a potential indicator of likely COVID-19 costs. The researchers found that these out-of-pocket costs were particularly high for so-called consumer-directed health plans—which typically feature lower premiums, compared to standard plans, but higher deductibles that can be paid via tax-advantaged health savings accounts.
The findings were published online June 15 in the American Journal of Preventive Medicine.
Many big-name health insurers have voluntarily waived out-of-pocket cost sharing for COVID-19 treatment. However, employer-sponsored “self-insured” health insurance plans are not required to adhere to such waivers. Thus, tens of millions of Americans have high-deductible insurance plans that, in cases of COVID-19 hospitalization, may expose them to relatively high out-of-pocket costs.
“Congress is now debating whether or not to require all plans to waive cost sharing related to COVID-19 treatment. Our findings suggest that they might want to take action to broaden cost-sharing waivers to include these self-insured plans, because those out-of-pocket costs are otherwise going to be high for many people,” says study lead author Matthew Eisenberg, Ph.D., an assistant professor in the Department of Health Policy and Management at the Bloomberg School.
COVID-19, the disease that is caused by the novel coronavirus SARS-CoV-2, often features pneumonia in moderate and severe cases. It has struck more than 7 million people worldwide and killed more than 400,000 since its initial outbreak in China late last year. The U.S. has had the largest number of cases and deaths by far, with more than 250,000 hospitalizations for moderate to severe COVID-19, according to the Centers for Disease Control and Prevention.
To help reduce the cost burden of COVID-19 testing, Congress has banned insurance co-pays, out-of-pocket payments, and other cost sharing for COVID-19 testing, effectively requiring most insurers to cover the full cost. Many private insurance companies also have voluntarily extended such cost-sharing waivers for COVID-19 treatment. But about 60 percent of employer-sponsored health insurance plans are self-insurance plans. In such plans, the employer assumes the financial risk of medical care for their employees rather than the insurer. Such companies are not obliged to waive cost-sharing for COVID-19 treatment, and thus many of the millions of Americans insured under these plans could face high out-of-pocket costs, especially if their plans are high-deductible, consumer-directed plans.
To get a sense of the likely cost burden on patients hospitalized for COVID-19, Eisenberg and colleagues examined de-identified insurance claims for 34,395 unique hospitalizations from January 2016 through August 2019. They loked at out-of-pocket costs incurred by people who had been hospitalized during the 2016-2019 study period with pneumonia, acute bronchitis, lower respiratory infections, and acute respiratory distress syndrome. (Claims data on actual COVID-19 cases were not available in the database at the time of the study.) The cases examined did not include those for people ages 65 and over, who are normally covered by Medicare. The out-of-pocket costs included deductible payments, copayments, and coinsurance payments.
The researchers found that average out-of-pocket spending for the 2016-2019 study period for these respiratory hospitalizations was $1,961 for patients with consumer-directed plans versus $1,653 for patients in traditional, usually smaller-deductible plans.
The out-of-pocket cost gap was lowest for older patients age 56 to 64, and greatest—$2,237 vs. $1,685—for patients 21 and younger. The analysis was not designed to examine why the cost gap varied inversely with patient age, but one possible explanation proposed by the researchers was that, since younger patients are healthier on average, their hospitalizations may reflect more serious and thus more costly illness.
“For people already struggling with serious respiratory illness due to COVID-19, the added stress of managing large medical bills could be devastating at this moment when so many Americans are experiencing major financial strain and job loss,” says study co-author Alene Kennedy-Hendricks, Ph.D., assistant scientist in the Department of Health Policy and Management at the Bloomberg School. “This is a critical area to watch and to consider for policy action moving forward.”
Given that self-insured plans cannot be regulated at the state level, the researchers suggest that federal policymakers should consider waiving COVID-19 cost sharing for self-insured plan policyholders. They note too that while the likely out-of-pocket costs would be greatest for people enrolled in high-deductible, consumer-directed plans, the findings indicate that people in traditional plans also could face fairly large out-of-pocket costs.
“Financial Risk for COVID-19-like Respiratory Hospitalizations in Consumer-Directed Health Plans” was written by Matthew Eisenberg, Colleen Barry, Cameron Schilling, and Alene Kennedy-Hendricks.
Explore further
More information: Matthew D. Eisenberg et al, Financial Risk for COVID-19-like Respiratory Hospitalizations in Consumer-Directed Health Plans, American Journal of Preventive Medicine (2020). DOI: 10.1016/j.amepre.2020.05.008
https://medicalxpress.com/news/2020-06-covid-hospitalizations-significant-out-of-pocket-medical.html
Super-potent human antibodies protect against COVID-19 in animal tests
A team led by Scripps Research has discovered antibodies in the blood
of recovered COVID-19 patients that provide powerful protection against
SARS-CoV-2, the coronavirus that causes the disease, when tested in
animals and human cell cultures.
The research, published today in Science, offers a paradigm of swift reaction to an emergent and deadly viral pandemic, and sets the stage for clinical trials and additional tests of the antibodies, which are now being produced as potential treatments and preventives for COVID-19.
“The discovery of these very potent antibodies represents an extremely rapid response to a totally new pathogen,” says study co-senior author Dennis Burton, Ph.D., the James and Jessie Minor Chair in Immunology in the Department of Immunology & Microbiology at Scripps Research.
In principle, injections of such antibodies could be given to patients in the early stage of COVID-19 to reduce the level of virus and protect against severe disease. The antibodies also may be used to provide temporary, vaccine-like protection against SARS-CoV-2 infection for healthcare workers, elderly people and others who respond poorly to traditional vaccines or are suspected of a recent exposure to the coronavirus.
The project was led by groups at Scripps Research; IAVI, a nonprofit scientific research organization dedicated to addressing urgent, unmet global health challenges; and University of California San Diego School of Medicine.
“It has been a tremendous collaborative effort, and we’re now focused on making large quantities of these promising antibodies for clinical trials,” says co-lead author Thomas Rogers, MD, Ph.D., an adjunct assistant professor in the Department of Immunology & Microbiology at Scripps Research, and assistant professor of Medicine at UC San Diego.
An approach that’s worked for other deadly viruses
Developing a treatment or vaccine for severe COVID-19 is currently the world’s top public health priority. Globally, almost 8 million people have tested positive for SARS-CoV-2 infection, and more than 400,000 have died of severe COVID-19. The daily toll of new infections is still rising.
One approach to new viral threats is to identify, in the blood of recovering patients, antibodies that neutralize the virus’s ability to infect cells.
These antibodies can then be mass-produced, using biotech methods, as a treatment that blocks severe disease and as a vaccine-like preventive that circulates in the blood for several weeks to protect against infection. This approach already has been demonstrated successfully against Ebola virus and the pneumonia-causing respiratory syncytial virus, commonly known as RSV.
Potent patient antibodies block the virus
For the new project, Rogers and his UC San Diego colleagues took blood samples from patients who had recovered from mild-to-severe COVID-19. In parallel, scientists at Scripps Research and IAVI developed test cells that express ACE2, the receptor that SARS-CoV-2 uses to get into human cells. In a set of initial experiments, the team tested whether antibody-containing blood from the patients could bind to the virus and strongly block it from infecting the test cells.
The scientists were able to isolate more than 1,000 distinct antibody-producing immune cells, called B cells, each of which produced a distinct anti-SARS-CoV-2 antibody. The team obtained the antibody gene sequences from these B cells so that they could produce the antibodies in the laboratory. By screening these antibodies individually, the team identified several that, even in tiny quantities, could block the virus in test cells, and one that could also protect hamsters against heavy viral exposure.
All of this work—including the development of the cell and animal infection models, and studies to discover where the antibodies of interest bind the virus—was completed in less than seven weeks.
“We leveraged our institution’s decades of expertise in antibody isolation and quickly pivoted our focus to SARS-CoV-2 to identify these highly potent antibodies,” says study co-author Elise Landais, Ph.D., an IAVI principal scientist.
If further safety tests in animals and clinical trials in people go well, then conceivably the antibodies could be used in clinical settings as early as next January, the researchers say.
“We intend to make them available to those who need them most, including people in low- and middle-income countries,” Landais says.
In the course of their attempts to isolate anti-SARS-CoV-2 antibodies from the COVID-19 patients, the researchers found one that can also neutralize SARS-CoV, the related coronavirus that caused the 2002-2004 outbreak of severe acute respiratory syndrome (SARS) in Asia.
“That discovery gives us hope that we will eventually find broadly neutralizing antibodies that provide at least partial protection against all or most SARS coronaviruses, which should be useful if another one jumps to humans,” Burton says.
Am I immune to COVID-19 if I have antibodies?
The research, published today in Science, offers a paradigm of swift reaction to an emergent and deadly viral pandemic, and sets the stage for clinical trials and additional tests of the antibodies, which are now being produced as potential treatments and preventives for COVID-19.
“The discovery of these very potent antibodies represents an extremely rapid response to a totally new pathogen,” says study co-senior author Dennis Burton, Ph.D., the James and Jessie Minor Chair in Immunology in the Department of Immunology & Microbiology at Scripps Research.
In principle, injections of such antibodies could be given to patients in the early stage of COVID-19 to reduce the level of virus and protect against severe disease. The antibodies also may be used to provide temporary, vaccine-like protection against SARS-CoV-2 infection for healthcare workers, elderly people and others who respond poorly to traditional vaccines or are suspected of a recent exposure to the coronavirus.
The project was led by groups at Scripps Research; IAVI, a nonprofit scientific research organization dedicated to addressing urgent, unmet global health challenges; and University of California San Diego School of Medicine.
“It has been a tremendous collaborative effort, and we’re now focused on making large quantities of these promising antibodies for clinical trials,” says co-lead author Thomas Rogers, MD, Ph.D., an adjunct assistant professor in the Department of Immunology & Microbiology at Scripps Research, and assistant professor of Medicine at UC San Diego.
An approach that’s worked for other deadly viruses
Developing a treatment or vaccine for severe COVID-19 is currently the world’s top public health priority. Globally, almost 8 million people have tested positive for SARS-CoV-2 infection, and more than 400,000 have died of severe COVID-19. The daily toll of new infections is still rising.
One approach to new viral threats is to identify, in the blood of recovering patients, antibodies that neutralize the virus’s ability to infect cells.
These antibodies can then be mass-produced, using biotech methods, as a treatment that blocks severe disease and as a vaccine-like preventive that circulates in the blood for several weeks to protect against infection. This approach already has been demonstrated successfully against Ebola virus and the pneumonia-causing respiratory syncytial virus, commonly known as RSV.
Potent patient antibodies block the virus
For the new project, Rogers and his UC San Diego colleagues took blood samples from patients who had recovered from mild-to-severe COVID-19. In parallel, scientists at Scripps Research and IAVI developed test cells that express ACE2, the receptor that SARS-CoV-2 uses to get into human cells. In a set of initial experiments, the team tested whether antibody-containing blood from the patients could bind to the virus and strongly block it from infecting the test cells.
The scientists were able to isolate more than 1,000 distinct antibody-producing immune cells, called B cells, each of which produced a distinct anti-SARS-CoV-2 antibody. The team obtained the antibody gene sequences from these B cells so that they could produce the antibodies in the laboratory. By screening these antibodies individually, the team identified several that, even in tiny quantities, could block the virus in test cells, and one that could also protect hamsters against heavy viral exposure.
All of this work—including the development of the cell and animal infection models, and studies to discover where the antibodies of interest bind the virus—was completed in less than seven weeks.
“We leveraged our institution’s decades of expertise in antibody isolation and quickly pivoted our focus to SARS-CoV-2 to identify these highly potent antibodies,” says study co-author Elise Landais, Ph.D., an IAVI principal scientist.
If further safety tests in animals and clinical trials in people go well, then conceivably the antibodies could be used in clinical settings as early as next January, the researchers say.
“We intend to make them available to those who need them most, including people in low- and middle-income countries,” Landais says.
In the course of their attempts to isolate anti-SARS-CoV-2 antibodies from the COVID-19 patients, the researchers found one that can also neutralize SARS-CoV, the related coronavirus that caused the 2002-2004 outbreak of severe acute respiratory syndrome (SARS) in Asia.
“That discovery gives us hope that we will eventually find broadly neutralizing antibodies that provide at least partial protection against all or most SARS coronaviruses, which should be useful if another one jumps to humans,” Burton says.
Explore further
More information:
Thomas F. Rogers et al. Isolation of potent SARS-CoV-2 neutralizing
antibodies and protection from disease in a small animal model. Science 15 Jun 2020, DOI: 10.1126/science.abc7520 , science.sciencemag.org/content … 6/15/science.abc7520
Systemic delivery of micro-dystrophin gene therapy in children with DMD
Researchers from Nationwide Children’s Hospital have published in JAMA Neurology
results from the first four patients treated in the first clinical
trial of systemic delivery of micro-dystrophin gene therapy in children
with Duchenne muscular dystrophy (DMD)—and initial findings suggest that
the therapy can provide functional improvement that is greater than
that observed under the standard of care.
“We are very pleased to report successful delivery of the micro-dystrophin transgene to the nuclei—corresponding to robust gene expression and proper localization of micro-dystrophin,” said Jerry Mendell, MD, the study’s co-author and principal investigator with the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital. “This coincides with improvements in functional measurements in study participants who received SRP-9001.”
DMD is a fatal neuromuscular disease that occurs in approximately one in every 5,000 males worldwide. It is caused by a mutation in the gene that encodes for dystrophin, and symptoms usually appear in infants and toddlers. The dystrophin gene itself is too large to fit in to the adeno-associated viral vector used in the gene therapy technology utilized by the study. Researchers have developed micro-dystrophin as a microgene that provides function while still fitting in the vector.
Four ambulatory participants, aged 4 to 7 years at time of infusion were treated with a single dose of 2.0 x 1014 vg/kg rAAVrh74.MHCK7. micro-dystrophin (SRP-9001 micro-dystrophin, Sarepta Therapeutics), which was infused through a peripheral limb vein. All treatment-related events were mild to moderate and there were no serious adverse events. The most common treatment-related adverse event was vomiting (9 of 18 events). Three patients had temporarily elevated levels of gamma-glutamyltransferase, which was resolved with corticosteroids.
At 12 weeks post-infusion, gastrocnemius muscle biopsy specimens showed a mean of 81.2% of muscle fibers expressing micro-dystrophin, with a mean intensity of 96% at the sarcolemma. Western blot showed a mean expression of 74.3% without fat or fibrosis adjustment and 95.8% with adjustment.
All of the participants had confirmed vector transduction and showed functional improvement of North Star Ambulatory Assessment (NSAA) scores. The NSAA is a 17-item measure of ambulatory functions with a score range from 0 to 34.
All participants also showed reductions in creatine kinase (CK) levels that were maintained for one year.
Other functional outcomes measured included time to rise, four-stair climb, 100-m timed test and handheld dynamometry for knee extensors and flexors and elbow extensors and flexors, but their results were more varied.
“NSAA score improvement and CK score reduction appear to be the most sensitive measures,” said Dr. Mendell. “Different magnitudes of improvement were observed in the other functional measurements, suggesting that a larger sample size and clinical trial are needed to validate improved motor function. Variation in clinical outcomes were associated with many factors, including age and disease severity.”
The study is intended to continue until March 2021.
“Duchenne muscular dystrophy is difficult to treat, and gene therapy offers a needed option having the potential to alter the course of the disease,” said Dr. Mendell. “Using currently available treatments, the condition is universally fatal, and patients usually succumb to the disease in their twenties.”
The safety results, together with biological and clinical markers of efficacy, provide proof-of-concept support for continuation of clinical trials for assessment of SRP-9001 using single-dose gene therapy in participants with Duchenne, he added.
“Following the 9-month update we shared last year, the publication of these results further supports the potential for SRP-9001 to provide clinically meaningful functional improvements in terms of speed and magnitude for DMD patients,” said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy at Sarepta Therapeutics. “We look forward to advancing our ultimate goal of profoundly improving the lives of as many patients living with DMD as possible.”
“We are very pleased to report successful delivery of the micro-dystrophin transgene to the nuclei—corresponding to robust gene expression and proper localization of micro-dystrophin,” said Jerry Mendell, MD, the study’s co-author and principal investigator with the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital. “This coincides with improvements in functional measurements in study participants who received SRP-9001.”
DMD is a fatal neuromuscular disease that occurs in approximately one in every 5,000 males worldwide. It is caused by a mutation in the gene that encodes for dystrophin, and symptoms usually appear in infants and toddlers. The dystrophin gene itself is too large to fit in to the adeno-associated viral vector used in the gene therapy technology utilized by the study. Researchers have developed micro-dystrophin as a microgene that provides function while still fitting in the vector.
Four ambulatory participants, aged 4 to 7 years at time of infusion were treated with a single dose of 2.0 x 1014 vg/kg rAAVrh74.MHCK7. micro-dystrophin (SRP-9001 micro-dystrophin, Sarepta Therapeutics), which was infused through a peripheral limb vein. All treatment-related events were mild to moderate and there were no serious adverse events. The most common treatment-related adverse event was vomiting (9 of 18 events). Three patients had temporarily elevated levels of gamma-glutamyltransferase, which was resolved with corticosteroids.
At 12 weeks post-infusion, gastrocnemius muscle biopsy specimens showed a mean of 81.2% of muscle fibers expressing micro-dystrophin, with a mean intensity of 96% at the sarcolemma. Western blot showed a mean expression of 74.3% without fat or fibrosis adjustment and 95.8% with adjustment.
All of the participants had confirmed vector transduction and showed functional improvement of North Star Ambulatory Assessment (NSAA) scores. The NSAA is a 17-item measure of ambulatory functions with a score range from 0 to 34.
All participants also showed reductions in creatine kinase (CK) levels that were maintained for one year.
Other functional outcomes measured included time to rise, four-stair climb, 100-m timed test and handheld dynamometry for knee extensors and flexors and elbow extensors and flexors, but their results were more varied.
“NSAA score improvement and CK score reduction appear to be the most sensitive measures,” said Dr. Mendell. “Different magnitudes of improvement were observed in the other functional measurements, suggesting that a larger sample size and clinical trial are needed to validate improved motor function. Variation in clinical outcomes were associated with many factors, including age and disease severity.”
The study is intended to continue until March 2021.
“Duchenne muscular dystrophy is difficult to treat, and gene therapy offers a needed option having the potential to alter the course of the disease,” said Dr. Mendell. “Using currently available treatments, the condition is universally fatal, and patients usually succumb to the disease in their twenties.”
The safety results, together with biological and clinical markers of efficacy, provide proof-of-concept support for continuation of clinical trials for assessment of SRP-9001 using single-dose gene therapy in participants with Duchenne, he added.
“Following the 9-month update we shared last year, the publication of these results further supports the potential for SRP-9001 to provide clinically meaningful functional improvements in terms of speed and magnitude for DMD patients,” said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy at Sarepta Therapeutics. “We look forward to advancing our ultimate goal of profoundly improving the lives of as many patients living with DMD as possible.”
More information:
Jerry R. Mendell et al, Assessment of Systemic Delivery of
rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular
Dystrophy, JAMA Neurology (2020). DOI: 10.1001/jamaneurol.2020.1484
FDA OKs Jazz Pharma’s lurbinectedin for lung cancer
Under accelerated review status, the FDA approves Jazz Pharmaceuticals’ (JAZZ +4.6%)
Zepzelca (lurbinectedin) for adult patients with metastatic small cell
lung cancer (SCLC) with disease progression on or after platinum-based
chemo.
The company in-licensed U.S. rights from Spanish drugmaker PharmaMar (OTCPK:PHMMF +2.8%) in December 2019.
Lurbinectedin is an alkylating agent that kills cancer cells by interrupting the DNA replication process.
Aetna nabs Medicaid contract in West Virginia
CVS (CVS -1.3%) unit Aetna Better Health of West Virginia has been awarded a Medicaid contract in the state through the Mountain Health Trust managed care program.
It begins on July 1, 2020 and runs through June 30, 2021.
The contract value is not disclosed.
https://seekingalpha.com/news/3583170-aetna-nabs-medicaid-contract-in-west-virginia
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