Alnylam: Positive Topline Results in Phase 3 Study of Patisiran in ATTR Amyloidosis

  Patisiran Met the Primary Endpoint with a Statistically Significant Improvement in 6-Minute Walk Test Compared to Placebo at 12 Months –

– Patisiran Also Met the First Secondary Endpoint with a Statistically Significant Improvement in Quality of Life, as Measured by the Kansas City Cardiomyopathy Questionnaire, Compared to Placebo at 12 Months –

– Patisiran Demonstrated Encouraging Safety and Tolerability Profile in Patients with ATTR Amyloidosis with Cardiomyopathy –

– Company Plans to File a Supplemental New Drug Application in U.S. in Late 2022 –

– Full Data Will Be Presented at the 18th International Symposium on Amyloidosis –

– Alnylam to Host Conference Call Today at 8:00 am ET –

Management will discuss the APOLLO-B topline results via conference call on Wednesday, August 3, 2022, at 8:00 am ET. To access the call, please register online at https://register.vevent.com/register/BI6ac74b5406af42c5980ba67c6f9e419d. Participants are requested to register at a minimum 15 minutes before the start of the call. A replay of the call will be available two hours after the call and archived on the same web page for six months.

A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Company’s website approximately two hours after the event.

https://finance.yahoo.com/news/alnylam-reports-positive-topline-results-110000527.html

Applied DNA Sciences Shares Triples After Monkeypox Diagnostic Test Update

 

• Applied DNA Sciences Inc (NASDAQ: APDN) has initiated analytical validation of a company-developed, PCR-based monkeypox virus test specific to the genetic signature of the monkeypox virus.

• The test has been developed as an NYSDOH Laboratory Developed Test (LDT) type.

• If Applied DNA validates the test, a validation package will be submitted to the New York State Department of Health (NYSDOH) for approval.

• If approved, the test will be used to power ADCL's monkeypox testing services.

• Applied DNA's monkeypox test utilizes an A17L gene-target specific to monkeypox that enables the qualitative detection and differentiation of the monkeypox virus from other non-variola orthopoxviruses using real-time PCR.

• California Governor Gavin Newsom declared a state of emergency over the monkeypox.

• California confirmed 827 monkeypox cases, the second-largest state tally after the 1,390 infections documented in New York, according to the U.S. Centers for Disease Control and Prevention.

https://finance.yahoo.com/news/applied-dna-sciences-shares-triples-170210256.html

COVID Vaccine Injuries Quietly Being Compensated Around The World

 by Jennifer Margulis and Joe Wang via The Epoch Times,

Programs in countries around the world have begun quietly compensating people who have been injured by or died as a result of the COVID-19 vaccines.

Humans are biologically diverse, with respect to both genetic makeup and past environmental exposures. Because of this, explained neurologist Dr. Robert Lowry, people can react very differently to the same medication or vaccination. Whenever a new drug or biologic hits the market, some people will have bad reactions and others may even suffer serious adverse events as a result.

Even under the best testing conditions, rare reactions will be missed. This is especially true for any product which is fast-tracked or authorized for emergency use before all the phases of necessary testing are complete.

COVID-19 vaccines are no exception. Despite the fact that we are constantly and consistently assured that COVID-19 vaccines are safe, and that severe adverse reactions are “very rare,” the FDA and the CDC with its Advisory Committee on Immunization Practices, as well as the scientists and executives at each of the participating drug companies, know that some people will become permanently disabled or even die as a result of vaccination.

In fact, in 2011 the Supreme Court of the United States (pdf) reiterated the idea that vaccines, like other pharmaceutical products, are “unavoidably unsafe.”

In many countries around the world, consumers who are injured as a result of vaccines are covered by government compensation programs. In the United States there are two government-funded programs that are designed to compensate consumers for injuries, at the same time shielding vaccine manufacturers from liability for any serious injuries their products cause: The National Vaccine Injury Compensation Program (VICP) and the Countermeasures Injury Compensation Program (CICP).

Not a Single Claim Compensated in United States

As of July 1, 2022, not a single claim has been compensated by the CICP. However, 31 COVID-19 countermeasure claims have been denied, “because the standard of proof for causation was not met and/or a covered injury was not sustained,” according to the CICP website. “One COVID-19 countermeasure claim, a COVID-19 vaccine claim due to an anaphylactic reaction, has been determined eligible for compensation and is pending a review of eligible expenses.”

At the same time, countries around the world are quietly compensating families whose loved ones have been injured or have died as a result of COVID-19 vaccines.

Japan Pays Bereaved Family of 91-Year-Old

The Japan Times reported this week that a 91-year-old woman who died after suffering an allergic response and sudden heart attack is the first person in Japan to be compensated for a COVID vaccine injury.

A ministry of health panel ruled that a causal relationship “could not be denied” in her case. Her family will receive a lump sum worth approximately $325,000.

The panel reviewed another 11 cases ranging in age from 20s to 90s but did not reach any other ruling.

So far in Japan, according to the article, 3,680 people have applied for compensation, 820 have been approved and 62 denied, with decisions on another 16 being “postponed.”

Taiwan Compensates 10 Claimants

On June 24, 2022, Taiwan’s National Vaccine Injury Compensation Program held a meeting to review 65 cases, according to the Taipei Times. The Taiwanese program awarded compensation to 10 claimants. These awards included a lump sum worth $116,877 to the family of someone who died after receiving the AstraZeneca vaccine.

This patient was hospitalized 10 days after receiving the vaccine due to a headache and vomiting. Testing revealed thrombocytopenia, a sometimes-lethal blood disorder that is characterized by low platelets.

However, the patient was discharged the next day, only to return that evening after losing consciousness. The patient died of intracerebral hemorrhage, a common cause of stroke.

When the vaccination program first rolled out in the United States, in December of 2020, an otherwise healthy obstetrician-gynecologist, Dr. Gregory Michael, 56, of Miami Beach, Florida, also died of thrombocytopenia.

Although his death occurred approximately two weeks after he got Pfizer’s COVID-19 vaccine, and prompted an article exploring this side effect in the New York Times, the coroner deemed that there was no medical certainty that the complications from immune thrombocytopenia was vaccine-induced.

UK Pays Out for Vaccine Injuries

Vicki Spit was the first of a handful of people in the United Kingdom to be awarded compensation for injuries due to COVID vaccines, according to a June 24, 2022 article in the British Medical Journal.

Spit’s 48-year-old partner, Zion, became ill eight days after receiving the AstraZeneca vaccine and died. The victims were awarded the maximum: $150,000. As of May 2022, 1,681 claims for vaccine injury following COVID-19 vaccinations had been filed.

As quoted in the BMJ, Sarah Moore, a spokeswoman for the law firm representing the victims or their families, pointed out that though the awards will not do much to alleviate the financial hardships caused by the injuries, they constitute “the clearest statement yet, by the government, that in some rare instances the COVID-19 vaccines have caused very significant injury or death.”

Moore believes most of the compensation awards were for vaccine-induced thrombotic thrombocytopenia (VITT) or cerebral venous sinus thrombosis.

At the same time, Yahoo News has reported that 444 cases of VITT have been recorded in the United Kingdom from 49 million doses of the vaccine, with 81 deaths.

Canada Received More Than 700 Claims, Approved 8

According to Canada’s Vaccine Injury Support Program (VISP), from June 1, 2021 to June 1, 2022, 774 claims have been received. Eight of these claims were “approved by the Medical Review Board,” meaning “these claims represent cases where it has been determined by the Medical Review Board that there is a probable link between the injury and the vaccine and that the injury is serious and permanent.”

According to the VISP website, “eligible individuals may receive income replacement indemnities; injury indemnities; death benefits; coverage for funeral expenses; reimbursement of eligible costs such as otherwise uncovered medical expenses.”

Post Shot Blood Clots

Thrombocytopenia can lead to blood clots as well as hemorrhaging. Thrombosis is another word for blood clots. Since it is very unusual for an otherwise healthy younger person to suffer from blood clots, the connection between the vaccines and this injury are difficult to refute.

Scandinavian countries have paid more attention to COVID-19 vaccine injuries than other countries. Norway compensated its first three victims in July of last year, a woman in her 40s who died, as well as a man and a woman in their 30s who both survived their vaccine injuries.

All three were healthcare workers who received the AstraZeneca vaccine, which Norway stopped administering on March 11, 2021, due to reports of serious blood clots, low platelet counts, and abnormal bleeding.

In fact, Norwegian doctors were among the first to point out the connection between the vaccine and these injuries.

Denmark, too, has been quickly and quietly processing vaccine injury claims. The Danish government awarded compensation for their first case of VITT in May of 2021.

At the time, 158 people had filed claims for COVID-19 vaccine injuries. The director of the patient compensation board, Karen Inger-Bast, said, “Generally, we often see injuries from vaccination. We also see them from, for example, vaccination against influenza and children’s diseases. That’s also how it will be with COVID-19, with up to 5 million people being vaccinated.”

No Financial Assistance for Americans Harmed by Vaccines

Yet, while other countries are compensating people who have been injured by COVID-19 vaccines, America has yet to financially assist a single claimant. According to the Health Resources and Service Administration, as of July 1, 2022, the CICP had yet to award compensation to anyone for damages due to a COVID-19 vaccine.

https://www.zerohedge.com/medical/covid-vaccine-injuries-quietly-being-compensated-around-world

Flare-ups of gout are linked to heart attack and stroke

 Experts at the University of Nottingham, in collaboration with experts at Keele University, have found that the risk of heart attacks and strokes temporarily increases in the four months after a gout flare.

The research showed that gout patients who suffered from a heart attack or stroke were twice as likely to have had a gout flare in the 60 days prior to the event, and one and a half times more likely to have a gout flare in the 61-120 days prior.

The results of the study, led by Professor Abhishek in the School of Medicine at the University of Nottingham, are published in the journal JAMA.

Gout is a common form of arthritis that affects one in 40 adults in the UK. It is caused by high levels of uric acid, a chemical produced by breakdown of tissues in the body and present in certain foods and drinks.

At high levels, uric acid is deposited in and around joints as needle shaped urate crystals. Once released from their deposits, these crystals cause severe inflammation manifesting as joint pain, swelling, redness, and tenderness that often lasts for 1-2 weeks. These episodes, called gout flares, often recur. Inflammation is also a risk factor for heart attack and stroke.

People with gout tend to have more cardiovascular risk factors, although there have been no previous studies about whether gout flares are linked with an increased risk of heart attack and stroke. In this study, the experts examined whether there was a temporary increase in risk of heart attack or stroke after a gout flare.

The team used anonymized data from 62,574 patients with gout treated in the National Health Service in the UK. Of these, 10,475 experienced heart attack or stroke after the diagnosis of gout, while others of similar age, sex, and duration of gout, did not experience such events. They evaluated the association between heart attacks or strokes and recent gout flares and adjusted these results for comorbidities, socioeconomic deprivation, lifestyle factors and prescribed medications among other things. They found that gout patients who suffered a heart attack or stroke were twice as likely to have had a gout flare in the 60 days prior to the event, and one and a half times more likely to have a gout flare in the preceding 61-120 days.

They found a similar high rate of heart attack or stroke in the 0-60 and 61-120 days after gout flares compared with other time periods, when they used information from only patients who consulted for a gout flare and also experienced either heart attack or stroke. This further strengthened the finding that gout flares are associated with a transient increase in cardiovascular events following flares. The increased odds and rates persisted when people with pre-existing heart disease or stroke before their gout diagnosis were excluded, and when shorter exposure periods such as 0-15 and 16-30 days prior to heart attack or stroke, were considered.

Gout patients who died from a heart attack or stroke had over four times the odds of experiencing a gout flare in the preceding 0-60 days and over twice the odds of gout flare in the preceding 61-120 days.

Professor Abhishek, lead author on the study, said: "This is the first study of its kind to examine whether there is an association between recent gout flares and heart attacks and strokes.

"The results show that among patients with gout, patients who experienced a heart attack or stroke had significantly increased odds of a gout flare during the preceding 120-days compared with patients who did not experience such events. These findings suggest that gout flares are associated with a transient increase in cardiovascular events following flares.

"People with recurrent gout flares should be considered for long-term treatment with urate lowering treatments such as allopurinol. This is a reliable way of removing urate crystal deposits and providing freedom from gout flares. Patients should also be considered for concurrent treatment with anti-inflammatory medicines such as colchicine for the first few months because urate lowering treatments may trigger gout flares in the short term.

"People with gout should be encouraged to adopt a healthy lifestyle with appropriate treatment of conditions such as high blood pressure, high cholesterol, obesity and diabetes to minimise their background risk of heart attack and stroke."

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Story Source:

Materials provided by University of Nottingham. Note: Content may be edited for style and length.

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Journal Reference:

1. Mohammad Zeeshan, Ravish Rashpa, David J. P. Ferguson, Steven Abel, Zeinab Chahine, Declan Brady, Sue Vaughan, Carolyn A. Moores, Karine G. Le Roch, Mathieu Brochet, Anthony A. Holder, Rita Tewari. Genome-wide functional analysis reveals key roles for kinesins in the mammalian and mosquito stages of the malaria parasite life cycle. PLOS Biology, 2022; 20 (7): e3001704 DOI: 10.1371/journal.pbio.3001704

https://www.sciencedaily.com/releases/2022/08/220802121724.htm

Early physical therapy tied to less health care resource use for patients with acute lower back pain

 Early initiation of physical therapy (PT) for U.S. patients with acute lower back pain (LBP) was associated with less health care resource use in the first month and the first year after the initial onset of symptoms, according to a new study by Johns Hopkins Medicine. These health care resources included advanced imaging, specialty appointments, epidural steroid injections and emergency room (ER) visits. Patients who began PT early were half as likely to visit the ER within 30 days of symptom onset than those who did not receive early PT, which the study defined as starting within two weeks of symptom onset.

The study was published by BMC Health Services Research in July 2022.

Lower back pain affects a sizeable portion of the U.S. population, ranging from 1.4% to 20%, and it accounts for substantial health care expenditures.

"Our goal was to determine if early PT for patients with lower back pain had an impact on their overall health care resource utilization," says Richard Skolasky Jr., Sc.D., M.A., the study's senior author and the director of the Johns Hopkins Spine Outcomes Research Center. "We were especially curious about the 30 days after initial symptom onset, as this is when patients are most likely to seek care."

Using Truven MarketScan, a group of U.S.-based administrative health care insurance claims databases, the researchers selected nearly 980,000 U.S. claims cases. The de-identified patients in these claims had an average age of 47 and initially presented with acute LBP from 2010 through 2014. Approximately 11% of the patients received early PT.

From the cases reviewed, the team assessed the use of health care resources and the cost of the LBP-related services that were coded with a musculoskeletal system diagnosis. The team then compared these results at 30 days and at one year after presentation between patients who received early PT and those who did not.

Compared with those who did not receive early PT, the early PT group had a significantly lower incidence of using health care resources, such as ER visits, in the first month and the first year after presenting with acute LBP. The early PT group also spent less money on LBP-related services in the first 30 days after symptom onset.

"As the U.S. population ages, the prevalence of lower back pain is expected to increase, along with the associated costs of treating it," says Skolasky. "Furthermore, with advances in imaging and treatments, the cost of managing lower back pain has increased substantially. Our findings have important implications that may guide health care policy when examining downstream health care costs and resource utilization."

The research team believes that future research on the effects of early PT should incorporate measures of clinical outcomes to determine its impact on patient health.

tory Source:

Materials provided by Johns Hopkins Medicine. Note: Content may be edited for style and length.

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Journal Reference:

1. Majd Marrache, Niyathi Prasad, Adam Margalit, Suresh K. Nayar, Matthew J. Best, Julie M. Fritz, Richard L. Skolasky. Initial presentation for acute low back pain: is early physical therapy associated with healthcare utilization and spending? A retrospective review of a National Database. BMC Health Services Research, 2022; 22 (1) DOI: 10.1186/s12913-022-08255-0

https://www.sciencedaily.com/releases/2022/08/220802121732.htm

New national guidelines aim to prevent obesity in midlife women

 Women between 40 and 60 years old are the focus of new national guidelines aimed at preventing unhealthy weight gain that can lead to serious illness. The study review paper and clinical guidelines are published in the Annals of Internal Medicine.

"More than two-thirds of middle-aged women are overweight or obese. Given women's increased risk for weight gain in midlife, there is a critical need for intervention aimed at preventing obesity and the host of serious health outcomes associated with it," said Kimberly D. Gregory, MD, MPH, corresponding author of the clinical guidelines and vice chair, Women's Healthcare Quality and Performance Improvement in the Department of Obstetrics and Gynecology at Cedars-Sinai.

Gregory is part of the Women's Preventive Services Initiative (WPSI), which developed the recommendations based on the review of clinical trials involving nearly 52,000 middle-aged women. The initiative was launched in 2016 by the American College of Obstetricians and Gynecologists and operates in collaboration with the U.S. Department of Health and Human Services, Health Resources and Services Administration.

"In the past, most studies and recommendations have focused on investigating the benefits and harms of weight-loss tools used by women who were already overweight. But as a prevention strategy, these new guidelines strongly encourage healthcare providers to begin addressing the issue of weight gain and obesity risk with patients who are at normal weight," said Gregory.

Obesity has been declared epidemic in the United States, with 42% of adults having a body mass index (BMI) of over 30, according to the Centers for Disease Control and Prevention (CDC). A BMI of 18.5-25 is regarded as healthy, and one between 25 and 30 qualifies as overweight.

According to the study, women gain weight at an average of 1.5 pounds per year during midlife, which increases their risk for transitioning to an overweight or obese BMI. The new guidelines emphasize the need to help with weight management by counseling women while they are at a healthy weight and not waiting until they are overweight or have developed obesity.

"Women are at higher risk for severe obesity due to menopause and age-related physiological changes," said Amanda Velazquez, MD, director of Obesity Medicine in the Department of Surgery at Cedars-Sinai. "Significant weight gain is associated with a serious risk of developing cardiovascular disease, hypertension, Type 2 diabetes, fatty liver disease and numerous cancers. That is why advising middle-aged women with normal to overweight BMI on the need to limit weight gain is critical," said Velazquez, who did not participate in the study.

The WPSI review suggests that some behavioral counseling approaches to prevent future weight gain in midlife women could result in modest weight loss. Obesity and weight-loss expert Velazquez says it is important to remember that weight management is a lifelong journey and investment in good health and that there is plenty of help available.

"Do not give up. It is never too late to start making changes. There are quite a few weight management tools available, including individualized lifestyle plans, support groups that provide accountability and community, nutritional counseling, and new weight-loss medications. For people with severe obesity, a BMI over 40, bariatric surgery may be an option to consider," said Velazquez.

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Story Source:

Materials provided by Cedars-Sinai Medical Center. Note: Content may be edited for style and length.

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Journal Reference:

1. David Chelmow, Kimberly D. Gregory, Catherine Witkop, Susan Hoffstetter, Linda Humphrey, Carla Picardo, James J. Stevermer, Amy G. Cantor, Heidi D. Nelson, Sarah Son, Jeanne A. Conry, Francisco Garcia, Susan M. Kendig, Nancy O’Reilly, Amir Qaseem, Diana Ramos, Alina Salganicoff, Julie K. Wood, Christopher Zahn. Preventing Obesity in Midlife Women: A Recommendation From the Women's Preventive Services Initiative. Annals of Internal Medicine, 2022; DOI: 10.7326/M22-0252

https://www.sciencedaily.com/releases/2022/08/220802153229.htm

Using light to restore cell function

 New research from the University of Cincinnati shows early indications that light can be used as a treatment for certain diseases, including cancer.

Researchers from UC, the University of Illinois Urbana-Champaign and the University at Buffalo published the results of their study demonstrating light-activated proteins can help normalize dysfunction within cells in the journal Nature Communications July 25.

The research centers on the functions of mitochondria, organelles within a cell that act as the cell's "power plant" and source of energy. Organelles are tiny specialized structures that perform various jobs inside cells.

Jiajie Diao, PhD, one of the study's authors, said hundreds of mitochondria are constantly coming together (a process called fusion) and dividing into smaller parts (a process called fission) to stay balanced in healthy cells. But when mitochondria are not functioning properly, there is an imbalance of this process of fission and fusion.

This imbalance can lead to a number of mitochondrial diseases, including neurodegenerative diseases like dementia and certain cancers.

Diao said previous research found that another organelle within cells called a lysosome can play a role in mitochondria fission. When a mitochondria comes in contact with a lysosome, the lysosome can act like a pair of scissors and cut the mitochondria into smaller pieces.

The current research focused on jump-starting the fission process by bringing the lysosomes and mitochondria together within cells. This was accomplished using a technique known as optogenetics, which can precisely control specific cell functions using light.

"Many proteins in plants are light sensitive, informing plants whether it is day or night. Optogenetics borrows these light-sensitive proteins from plants and uses them in animal cells," said Kai Zhang, PhD, associate professor at the University of Illinois Urbana-Champaign and study co-author, who developed the optogenetic tools for controlling mitochondria and lysosomes with blue light. "By attaching such proteins to organelles, one can use light to control the interaction between them, such as mitochondria and lysosomes shown in this work," he said.

The researchers attached two separate proteins to mitochondria and lysosomes within stem cells. When stimulated by blue light, the proteins naturally bind to each other to form one new protein, which also brings the mitochondria and lysosome into contact. Once they are brought together, the lysosome can cut the mitochondria, achieving fission.

"We found that it can recover the mitochondrial function," said Diao, associate professor in the Department of Cancer Biology in UC's College of Medicine and a University of Cincinnati Cancer Center member. "Some of the cells can even go back to normal. This proves that by just using some simple light stimulation we can at least partially recover the mitochondrial function of the cell."

Diao said this technique could be especially useful for patients with dramatically oversized mitochondria that need to be divided into smaller pieces to achieve normal cell function. The technique could also be aimed at cancer cells, continually separating the mitochondria into smaller and smaller pieces until they can no longer function.

"Eventually the cancer cells will be killed because mitochondria is their energy," Diao said. "Without normal functional mitochondria, all of the cancer cells will be killed."

Since the proteins are activated by light, Diao said it allows for a more targeted approach to specific cells. Only cells exposed to the light are affected, meaning healthy cells nearby do not have their mitochondria unbalanced through the technique.

There are currently other processes that can be used to induce mitochondrial fission, but Diao said the optogenetic method is safer since it does not involve any chemicals or toxic agents.

"What we have is actually the natural process, we're just making it faster," Diao said. "So it's not like a chemical or a therapy or a radiotherapy where you need to reduce the side effects."

Next steps

Diao said his team is already at work on using the same technique to encourage fusion to address issues when mitochondria are unbalanced because they are too small and not coming together as they should within cells.

Further research from Zhang's lab will also include developing new optogenetic systems working with different colors of light, including green, red and infrared, since a longer wavelength will be needed to penetrate human tissue.

"We would like to further expand the toolbox by introducing multicolor optogenetic systems to give us multiple ways to control how organelles behave and interact," Zhang said. "For instance, one color makes organelles come together, while the other color forces them apart. This way, we can precisely control their interactions."

From the current research using human stem cells, the team hopes to progress to test its efficacy using animal models on the way to eventually test the technique in humans through clinical trials. At the same time, Diao said other research groups are studying the use of magnetic fields and acoustic vibrations instead of light to accomplish similar results.

This work was supported by the National Institutes of Health (NIH R35GM128837 and R21AG061600 to J.D.; R01GM132438 and R01MH124827 to K.Z.). The authors declare no competing interests.

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Story Source:

Materials provided by University of Cincinnati. Original written by Tim Tedeschi. Note: Content may be edited for style and length.

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Journal Reference:

1. Kangqiang Qiu, Weiwei Zou, Hongbao Fang, Mingang Hao, Kritika Mehta, Zhiqi Tian, Jun-Lin Guan, Kai Zhang, Taosheng Huang, Jiajie Diao. Light-activated mitochondrial fission through optogenetic control of mitochondria-lysosome contacts. Nature Communications, 2022; 13 (1) DOI: 10.1038/s41467-022-31970-5

https://www.sciencedaily.com/releases/2022/08/220802105038.htm