Clinical trials are key to advancing medicine, including COVID-19 vaccine

MIAMI – Registered clinical trials reached record levels in the past year, increasing the demand for people willing to participate, and Miami resident Marilyn Strauss Diaz was eager to join a COVID-19 vaccine trial underway at the University of Miami Health System.

“We can’t depend on a lab rat. We have to have human trials for there to be a successful vaccine, so we have to have humans come forward and be a part of trials,” she said.

“Before COVID, we usually would have a couple of hundred clinical trials here going at the same time at the University of Miami that are focused on a lot of different diseases. With COVID, there was a slowdown and now we’re ramping up again and starting a lot of these studies,” said Dr. Olveen Carassquillo, who is part of the research team.

“COVID has the most urgency. That’s a public health crisis so a lot of research is focused on that, but we have a lot of landmark cancer studies going on with different treatment regimens and prevention studies. We also have a lot of cardiovascular disease, a lot of other studies focused on stroke, on preventing dementia,” Carassquillo added.

And South Florida’s diverse population makes it a major focus of research efforts, something Strauss Diaz is proud to be a part of.

“At the end, it will be beneficial to so many,” she said.

To learn more about clinical trials at the University of Miami Health System go to: https://umiamihealth.org/clinical-trials

Copyright 2020 by WPLG Local10.com – All rights reserved.

Source Article

Read more

How Parents Feel About Their Kids in COVID-19 Vaccine Trials

Katelyn Evans, 16, has never met Randy Kerr—and there’s no reason she should have. It was 66 years ago that Kerr, then 6, became briefly famous, receiving the first injection of Jonas Salk’s experimental polio vaccine during the massive field trial of hundreds of thousands of children in the spring of 1954. History notes that the vaccine worked, and the children who stepped forward to receive either the actual shot or a placebo were heroically dubbed the Polio Pioneers.

Evans is a pioneer of the modern age, one of an eventual group of 600 children in the 16-to-17 year-old age group (along with 2,000 more between 12 and 15) to volunteer to be part of a Phase 3 trial to test an experimental COVID-19 vaccine made by the multinational pharmaceutical giant Pfizer. The company had already enrolled 42,113 adult volunteers in its Phase 2 and 3 trials, but only recently did the U.S. Food and Drug Administration (FDA) give approval to include children. And Evans, a high school junior in Cincinnati, was among the earliest, receiving her first of two injections on Oct. 14, at Cincinnati Children’s Hospital.

“She was the youngest one to receive the vaccine at that point in time,” says her mother, Laurie Evans, an elementary school teacher. In the spring, the family saw a news report that Pfizer was looking for volunteers and Evans and both of her children signed up. “Katelyn was the only one who got the call,” Laurie says. “I know from the response we’ve gotten that there are some people out there who don’t think this is the smartest thing for us to have done. But I’m more afraid of COVID than the vaccine.”

With good reason. The 8.8 million Americans who have contracted the disease include about 800,000 children, with the American Academy of Pediatrics (AAP) reporting a 13% increase in total pediatric cases in just the first two weeks of October. Children with COVID-19 may typically fare better than adults who catch the virus, but they can still become severely ill: some 3.6% of total U.S. COVID-19 patients who have had to be hospitalized have been children, according to the AAP. That reality makes volunteering for the Pfizer field trial more than an act of public-service heroism; it is also a potential act of preventive medicine.

Certainly, that’s the way Sharat Chandra saw things. Sharat was already part of the Pfizer adult trial and when word first went around that children would soon be included too, he and his wife discussed the possibility of enrolling their 12-year-old son Abhinav, and then posed the question to him.

“I raised it to my son and we felt that it might be a good thing for him because if he got the vaccine, it could protect him from getting the virus himself,” Sharat says. “Because he was attending school in person, we felt that it would be good to minimize his risk for infection, if we can.”

Abhinav Chandra participating in Pfizer's COVID-19 vaccine trial.

Abhinav Chandra participating in Pfizer’s

Read more

Israel begins human trials for coronavirus vaccine

Israel began human trials for its own coronavirus vaccine as the Jewish state’s second lockdown comes to a gradual end.

Two volunteers in separate hospitals – one in Tel Aviv and another in Jerusalem – have started the trials. If the two, Segev Harel and Aner Ottolenghi, feel in good health in the next two days, another 80 people will join the first phase of the trial.

According to officials, each volunteer will get either a dose of the vaccine or a placebo and will be sent home, where they will be monitored for three weeks for any possible side effects or whether they develop antibodies.

Harel said he is very proud to be part of such an important national mission and said he is not nervous.

Segev Harel at Sheba Medical Center in Tel Aviv.

Segev Harel at Sheba Medical Center in Tel Aviv.
(Israeli Defense Ministry)

MODERNA WILL HAVE 20M CORONAVIRUS VACCINES READY BY YEAR’S END

If the first phase is successful, the second phase is scheduled for December and will include nearly 1,000 volunteers. A final phase will be open to 30,000 volunteers and will begin in April 2021.

Israeli Prime Minister Benjamin Netanyahu and Defense Minister Benny Gantz visited the Sheba Medical Center in Tel Aviv, where the first vaccine was administered.

Segev Harel meets with Prime Minister Benjamin Netanyahu and Defense Minister Benny Gantz at Sheba Medical Center in Tel Aviv.

Segev Harel meets with Prime Minister Benjamin Netanyahu and Defense Minister Benny Gantz at Sheba Medical Center in Tel Aviv.
(Israeli Defense Ministry)

“There is a cause for a measure of cautious optimism today,” Gantz said. “Optimism, because I think excellent work is being done by our top scientists across the board, all of whom are giving their very best, and this is cause for optimism.”

He said it will take time to track and process the data and draw conclusions. He asked for patience.

“I see the light at the end of the tunnel,” Netanyahu said. “I see the vaccines in the state of Israel. In this means or another, a vaccine developed here or abroad we will bring enough vaccines to the Israeli citizens, and we will break free from this pandemic.”

The commercial name of the vaccine is “BriLife.” The first part of the name, “Bri”, refers to the Hebrew word for health, “briut,” the second part, “il” alludes to Israel, and “life” speaks to the importance of the vaccine.

Israeli scientists have begun the first coronavirus vaccine trial.

Israeli scientists have begun the first coronavirus vaccine trial.
(Israeli Defense Ministry)

CLICK HERE FOR COMPLETE CORONAVIRUS COVERAGE

According to the Israel Institute for Biological Research, large-scale production of the vaccine has been implemented. To date, the institute has produced more than 25,000 vaccine doses.

“The work of all the hidden heroes in the institute and the hospitals has been poured into a little bottle which has the potential to bring a life-saving vaccine to the citizens of the state of Israel,” Dr. Shmuel Yitzhaki, head of the Biology Division at the IIBR told Fox News.

Officials said that, if all goes well, the vaccine could be ready for the general population as early as summer 2021.

CLICK

Read more

Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology

Largest studies in POMC and LEPR deficiency obesities demonstrate that treatment with setmelanotide reduced body weight and hunger

BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

“Results from Rhythm’s pivotal Phase 3 studies, which are the largest studies to date in POMC and LEPR deficiency obesities, provide evidence regarding the safety and efficacy of setmelanotide and we believe they validate its potential long-term use as a novel treatment for severe obesity and hyperphagia,” said co-author Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany. “It is important to recognize the signs of these rare genetic disorders because we may soon have a targeted treatment option available for the first time for obesity disorders caused by impairments of the MC4R pathway.”

Rhythm initially reported positive topline data from the Phase 3 studies in August 2019 and subsequently presented updated data in a late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019.

Eight of 10 participants with POMC deficiency obesity (80%; P<0.0001 compared with historical data) and five of 11 participants with LEPR deficiency obesity (45%; P=0.0001 compared with historical data) achieved at least 10 percent weight loss at approximately one year. The mean percent change in “most hunger” score in participants aged 12 years and older was -27.1 percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7 percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea.

“These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment,” said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. “These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity.”

In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug

Read more

Genprex Unveils New Branding for Upcoming Combination Clinical Trials in Non-Small Cell Lung Cancer

New branding will support upcoming combination clinical trials, including the Company’s trial combining REQORSA™ immunogene therapy drug with AstraZeneca’s Tagrisso®, which received FDA Fast Track Designation earlier in 2020

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the launch of new branding for its upcoming oncology clinical trials combining its lead drug candidate, REQORSA(quaratusugene ozeplasmid), with AstraZeneca’s Tagrisso® (osimertinib), which received U.S. Food and Drug Administration (FDA) Fast Track Designation earlier this year, and for the combination of REQORSA with Merck’s Keytruda® (pembrolizumab), for the treatment of non-small cell lung cancer (NSCLC).

These trials will use the trial brand “Acclaim,” which the Company believes evokes its enthusiasm and the hope these trials represent for NSCLC patients and the oncology community. Acclaim-1 will be used to identify the REQORSA and Tagrisso combination clinical trial, and Acclaim-2 will be used to identify the REQORSA and Keytruda combination clinical trial.

“We are enthusiastically preparing for our upcoming clinical trials and are excited to launch the adoption of this branding,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We believe the Acclaim brand communicates our passion for providing hope to NSCLC patients for important new treatment options in the fight against this devastating disease and aligns us with the clinical, medical and patient communities.”

The trial brand was developed in order to encourage early exposure of the Company’s clinical programs to the broad audience that Genprex’s business addresses, including patients, healthcare practitioners, clinical investigators, investors, employees and others. Genprex plans to initiate the Acclaim-1 clinical trial and the Acclaim 2 clinical trial in the first-half of 2021. Acclaim-1 is a Phase 1/2 clinical trial using a combination of REQORSA with Tagrisso in patients with late stage NSCLC with mutated epidermal growth factor receptors (“EGFRs”) whose disease progressed after treatment with Tagrisso. Acclaim-2 is a Phase 1/2 clinical trial using a combination of REQORSA with Keytruda in NSCLC patients who are low expressors (1 to 49%) of the protein, programmed death-ligand 1 (PD-L1).

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing potentially life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also

Read more

Address Medical Mistrust to Recruit Minorities to COVID-19 Trials

To end HIV and control COVID-19, medicine must earn back the trust of people in Black, Latinx, and Native American communities, said Jonathan Mermin, MD, MPH, director of the National Center for HIV/AIDS, Viral Hepatitis, STD, and TB Prevention at the CDC.

During the closing plenary of the United States Conference on HIV/AIDS October 21, Mermin laid out four principles behind those actions to the audience of USCHA:

  • Actively work with communities most affected by health inequities

  • Make plans to address them transparent

  • Work with community members on those plans and listen to their feedback

  • Do a good job

“Trust will come with proof of action,” he told the audience, watching virtually from their homes and offices.

But clinicians can take action in the exam room and during clinical trial recruitment to help the process along, said Russell Campbell, MA, deputy director of the Office of HIV/AIDS Network Coordination at the Fred Hutchinson Cancer Research Center, Seattle, Washington.

“Distrust in the medical and research establishment has deep roots,” he said during a presentation earlier in the week. To address this, clinicians must learn “culturally appropriate and meaningful engagement to influence the participation of historically underrepresented communities in healthcare and research.”

From Slavery to HeLa to Clinical Practice

Medical mistrust doesn’t stem only from the denial of treatment to the men who were part of the Tuskegee Syphilis Study. It also includes reckonings with the racist abuse of study participants by some of science’s most acclaimed leaders. Take, for instance, J. Marion Sims, MD, the so-called father of gynecology, who gained much of that information by experimenting without anesthesia on enslaved women; or Cornelius Rhoads, MD, whose name was removed from an award at the American Association for Cancer Research when objections grew concerning racist remarks Rhoads made about Puerto Rican patients in the 1930s. Or consider the story of Henrietta Lacks, the originator of the HeLa genes that have guided oncology research. Her genetic material was being mined for oncological insights without her permission and without compensation.

“The groups that have been systematically and intentionally denied treatment and known cures for diseases on behalf of research have just really taken the brunt,” Campbell said during a session role-playing best practices for addressing medical mistrust among potential clinical trial participants, conducted earlier in the conference.

“African Americans, American Indians, Puerto Ricans, Guatemalans, and others really still are heavily impacted by these abuses of research.”

And it shows in clinical outcomes. Research into HIV antiretroviral treatment adherence has found that medical mistrust was associated with a 76% likelihood that Black Americans living with HIV would be nonadherent to their treatments. But race-based medical mistrust drew into question the necessity of treatment at all.

A 2016 study in the journal AIDS Care found that although White gay men in Boston and primarily Black gay men in Jackson, Mississippi both reported concerns about side effects and lack of culturally appropriate care, it was the Black gay men in Jackson who expressed stronger medical mistrust

Read more

These teens, tweens are some of the Covid-19 vaccine trial’s youngest volunteers

When Abhinav, an Ohio seventh grader, learned that a Covid-19 vaccine trial near his hometown was enrolling children, he wanted to participate. But there was one aspect of the study that had him worried.

“I was mostly a bit nervous about the blood draws, because I had one about five years ago, and it wasn’t so comfortable,” Abhinav, 12, said.

Nonetheless, he decided to enlist in the trial, which is run by Pfizer Inc.

“I think that it could really benefit the world, and I think it could also help scientists know more about the coronavirus,” said Abhinav, whose family asked that their last name not be used to protect their privacy.

Abhinav received his first injection at Cincinnati Children’s Hospital Medical Center last Thursday, making him among the youngest participants in the world to take part in a Covid-19 vaccine trial. His father, Sharat, a bone marrow transplant physician, had already been in the trial himself, and encouraged Abhinav to sign up after experiencing no ill effects other than a fever that lasted for a day or two.

“I’m happy that he’s doing his bit for science,” Sharat said of his son. “With the Pfizer study, no major side effects have been reported so far, so that made me comfortable with enrolling Abhinav as well.”

Earlier this month, Pfizer became the first pharmaceutical company in the United States to receive approval from the Food and Drug Administration to test its vaccine on children as young as 12. The kids who have signed up say they are proud to be participating — and say they feel safe doing so.

“They were talking about symptoms, and they were just fatigue, low-grade fever, headache. I was thinking, ‘I hope I don’t have anything like that because I don’t want it to mess with school or work,’” Katelyn Evans, 16, of Green Township, Ohio, said. “But I wasn’t thinking about my permanent health for a short-term inconvenience.”

Katelyn Evans, 16, receives a Covid-19 vaccine during a trial at Cincinnati Children's Hospital Medical Center on Oct. 14. (Cincinnati Children's Hospital Medical Center)
Katelyn Evans, 16, receives a Covid-19 vaccine during a trial at Cincinnati Children’s Hospital Medical Center on Oct. 14. (Cincinnati Children’s Hospital Medical Center)

Like most of the other coronavirus vaccine trials, Pfizer’s involves getting two shots. Participants are given the shots three weeks apart and then have their health monitored for two years by researchers. The participants do not know whether they are receiving a placebo injection or an actual Covid-19 vaccine.

Either way, the youngest trial volunteers say they have confidence in the vaccines that are being developed — putting them at odds with many adults across the country.

Manufactured during a highly politicized pandemic in a matter of months versus the years or decades that a vaccine typically takes, the Covid-19 vaccines have prompted growing skepticism. A survey in October from Stat News and the Harris Poll found that only 58 percent of the American public would get a vaccine when it becomes available, down from 69 percent of those polled in August.

But researchers say the vaccines they are testing

Read more

After pausing for safety concerns, AstraZeneca and Johnson & Johnson have resumed their U.S. vaccine trials.

Late-stage coronavirus vaccine trials run by AstraZeneca and Johnson & Johnson have resumed in the United States after the companies said Friday that serious illnesses in a few volunteers appeared not to be related to the vaccines.

Federal health regulators gave AstraZeneca the green light after a six-week pause, concluding there was no evidence the experimental vaccine had directly caused neurological side effects reported in two participants. The AstraZeneca news was first reported by The Wall Street Journal.

Johnson & Johnson said that its trial, which had been on pause for 11 days, would restart after a company investigation determined that a “serious medical event” in one study volunteer had “no clear cause.” To maintain the integrity of the trial, the company said, it did not check whether the volunteer received the vaccine or the placebo.

Dr. Luciana Borio, a former acting chief scientist at the Food and Drug Administration, welcomed the announcements, citing the urgent need for multiple vaccines to remain in the race for a product that could protect the global population from the coronavirus, which has already killed more than a million people worldwide.

“The demand for safe and effective Covid vaccines exceeds any single manufacturer’s production capacity,” Dr. Borio said. “We really need several in the field.”

An F.D.A. spokesperson declined to comment on Friday afternoon.

AstraZeneca and Johnson & Johnson are two of the four companies now in late-stage clinical trials in the U.S. for experimental coronavirus vaccines. Both companies are using adenoviruses, which typically cause harmless colds. The adenovirus is engineered so that it can chauffeur a coronavirus gene into human cells.

Their two high-profile competitors, Moderna and Pfizer, also in advanced trials, are instead using a technology based on genetic material known as mRNA. Delivered into human cells, the mRNA prompts the production of coronavirus proteins, triggering an immune response.

AstraZeneca moved swiftly into clinical trials, enrolling thousands of volunteers for its vaccine trials around the world in countries including Brazil, India, South Africa and Britain. A large, late-stage trial kicked off in the United States at the end of August. But all the trials were halted days later, on Sept. 6. A volunteer who had received the vaccine in the United Kingdom reportedly experienced symptoms of transverse myelitis, or inflammation of the spinal cord, triggering a global pause to the company’s efforts.

The incident sparked some concern among experts, who noted that a similar adverse neurological event, reported months ago, had occurred in another vaccinated volunteer. While this earlier event prompted its own pause in AstraZeneca’s trials, an independent safety board ultimately determined it was unrelated to the vaccine, allowing studies to resume.

Following the second AstraZeneca halt in September, trials abroad rapidly resumed in most countries. But the American hiatus persisted, with few details released as to why.

According to two vaccine experts familiar with the situation who were not authorized to discuss it publicly, the F.D.A. did not directly tie the vaccine to the two neurological illnesses, although it could

Read more

J&J, AstraZeneca resuming trials of vaccines [Video]

Two trials involving coronavirus vaccine candidates are resuming. AstraZeneca has restarted the U.S. trial of its experimental COVID-19 vaccine, and Johnson & Johnson is set to resume its trial on Monday or Tuesday.

AstraZeneca said Friday the U.S. Food and Drug Administration has deemed it safe to continue testing the vaccine. The company had to pause its U.S. trial last month after a report of a serious neurological illness in a participant in its trial in the UK.It is developing its vaccine with researchers at Oxford University.

Johnson & Johnson said Friday that the independent safety panel, the Data and Safety Monitoring Board, has recommended that it resume trial recruitment after finding no evidence that the vaccine caused a volunteer to fall ill. The company says it’s on track to produce trial data of the vaccines’ effectiveness by the end of this year or early 2021.

Both companies have contracts to supply vaccines to the U.S. and other governments if they are cleared by regulators.

Video Transcript

Two trials involving coronavirus vaccine candidates are resuming. AstraZeneca has restarted the US trial of its experimental COVID-19 vaccine, and Johnson & Johnson is set to resume its trial on Monday or Tuesday.

AstraZeneca said Friday, “The US Food and Drug Administration has deemed it safe to continue testing the vaccine.” The company had to pause its US trial last month after a report of a serious neurological illness in a participant in its trial in the UK. It is developing its vaccine with researchers at Oxford University.

Johnson & Johnson said Friday [? that, ?] “The independent safety panel, the Data and Safety Monitoring Board, has recommended that it resume trial recruitment after finding no evidence that the vaccine caused a volunteer to fall ill.” The company says it’s on track to produce trial data of the vaccine’s effectiveness by the end of this year or early 2021.

Both companies have contracts to supply vaccines to the US and other governments if they are cleared by regulators.

Source Article

Read more

AstraZeneca and Johnson & Johnson to resume COVID-19 vaccine trials as U.S. daily cases top 71,000

On Friday, both AstraZeneca and Johnson & Johnson announced that their coronavirus vaccine trials are set to resume in the U.S. after hitting pause when volunteers became sick. The Food and Drug Administration gave the green light after investigations found no link between those cases and the vaccine.

The announcement comes as the U.S. recorded more than 71,000 coronavirus cases on Thursday, which is the highest single-day increase since July, according to figures from Johns Hopkins University. A total of 41 states are reporting an increase in average new cases, and 15 states have reported record hospitalizations in the last week.

“This really does become a moment where all Americans have to recognize that each of us individually has a responsibility if we want to turn this around,” said Dr. Francis Collins, director of the National Institutes of Health.

In hot spot Wisconsin, new cases are up nearly 40% compared to two weeks ago. Wisconsin resident Ava Pennicuik, 15, is still suffering from hot flashes and extreme fatigue three months after recovering from COVID-19.

“I still feel sick sometimes. Some days are good and then some days aren’t,” said Pennicuik. “Like one day I’ll have a really bad day and also super dizzy and tired, and I’ll have to take a nap, and the other day, like, I can just do stuff, like, normally.”

The latest data from researchers at the University of Washington says wearing face masks in public could save nearly 130,000 lives in the U.S. this fall and winter.

Katelyn Evans, 16, became the first teen to get an injection as part Pfizer’s COVID-19 vaccine study at Cincinnati Children’s Hospital, the Associated Press reported. It’s part of a push to safely inoculate school-age children.  

“I figured, you know, the more people they test, the more information they get and the sooner they can put out a vaccine to keep everyone safe,” said Evans.

Since the pandemic began, more than half a million American children have been infected with COVID-19. While vaccine trials continue to move forward, the first shots are unlikely to be recommended for kids. Vaccines can’t be given to children unless they’ve been tested in their age group, the AP reported.

Source Article

Read more