Dozens of clinical trials are testing mRNA treatment vaccines in people with various types of cancer, including pancreatic cancer, colorectal cancer, and melanoma. Some vaccines are being evaluated in combination with drugs that enhance the body’s immune response to tumors. But no mRNA cancer vaccine has been approved by the US Food and Drug Administration for use either alone or with other cancer treatments.

“mRNA vaccine technology is extremely promising for infectious diseases and may lead to new kinds of vaccines,” said Elad Sharon, M.D., M.P.H., of NCI‘s Division of Cancer Treatment and Diagnosis. “For other applications, such as the treatment of cancer, research on mRNA vaccines also appears promising, but these approaches have not yet proven themselves.”

With findings starting to emerge from ongoing clinical trials of mRNA cancer vaccines, researchers could soon learn more about the safety and effectiveness of these treatments, Dr. Sharon added. Over the past 30 years, researchers have learned how to engineer stable forms of mRNA and deliver these molecules to the body through vaccines. Once in the body, the mRNA instructs cells that take up the vaccine to produce proteins that may stimulate an immune response against these same proteins when they are present in intact viruses or tumor cells. Among the cells likely to take up mRNA from a vaccine are dendritic cells, which are the sentinels of the immune system. After taking up and translating the mRNA, dendritic cells present the resulting proteins, or antigens, to immune cells such as T cells, starting the immune response.

“Dendritic cells act as teachers, educating T cells so that they can search for and kill cancer cells or virus-infected cells,” depending on the antigen, said Karine Breckpot, Ph.D., of the Vrije Universiteit Brussel in Belgium, who studies mRNA vaccines. The mRNA included in the Pfizer-BioNTech and the Moderna coronavirus vaccines instructs cells to produce a version of the “spike” protein that studs the surface of SARS-CoV-2. The immune system sees the spike protein presented by the dendritic cells as foreign and mobilizes some immune cells to produce antibodies and other immune cells to fight off the apparent infection. Having been exposed to the spike protein free of the virus, the immune system is now prepared, or primed, to react strongly to a subsequent infection with the actual SARS-CoV-2 virus.

Source: https://www.cancer.gov/

Allergan, an AbbVie (NYSE: ABBV) company, today announced that VUITY^™ (pilocarpine HCl ophthalmic solution) 1.25%, the first and only eye drop approved by the U.S. Food and Drug Administration (FDA) to treat presbyopia, is now available by prescription in pharmacies nationwide. Presbyopia, or age-related blurry near vision, can be diagnosed through a basic eye exam by an eye doctor (optometrist or ophthalmologist) and is a common and progressive eye condition that affects 128 million Americans, or nearly half of the U.S. adult population.

“We are pleased to be able to bring this first-of-its-kind treatment to market sooner than expected for the millions of Americans with presbyopia who may benefit from it,” said Jag Dosanjh, senior vice president medical therapeutics, Allergan, an AbbVie company. “This significant innovation in age-related eye health reflects our commitment to advance vision care and expands our leading portfolio of treatments for eye care providers and their patients.”

“Many Americans deal with presbyopia, which typically begins around age 40, by relying on reading glasses or resorting to work-arounds like zooming in on their digital devices to see up close. As an optometrist who also has presbyopia, I’m personally and professionally excited to try VUITY for myself, as well as offer it to my patients with age-related blurry near vision,” said optometrist Dr. Selina McGee, Fellow of the American Academy of Optometry. “With VUITY now available, it is a good time for those who experience age-related blurry near vision to visit their eye doctor for an exam and to discuss their options to manage this common condition.”

Source: https://news.abbvie.com/

New data from an ongoing Phase 1/2 clinical trial has revealed an experimental immunotherapy led to successful response rates in 73% of patients suffering from multiple myeloma, a deadly form of blood cancer. Based on this promising data, an application to the US Food and Drug Administration (FDA) has been filed to bring the drug to market.

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Rice University and Baylor College of Medicine researchers have shown they can seradicate advanced-stage mesothelioma tumor in mice in just a few days with a treatment combining Rice’s cytokine “drug factory” implants and a checkpoint inhibitor drug.

The researchers administered the drug-producing beads, which are no larger than the head of a pin, next to tumors where they could produce continuous, high doses of interleukin-2 (IL-2), a natural compound that activates white blood cells to fight cancer. The study, published online today in Clinical Cancer Research, is the latest in a string of successes for the drug-factory technology invented in the lab of Rice bioengineer Omid Veiseh, including Food and Drug Administration (FDA) approval to begin clinical trials of the technology this fall in ovarian cancer patients.

“From the beginning, our objective was to develop a platform therapy that can be used for multiple different types of immune system disorders or different types of cancers,” said Rice graduate student Amanda Nash, who spent several years developing the implant technology with study co-lead author Samira Aghlara-Fotovat, a fellow student in Veiseh’s lab.

The cytokine factories consist of alginate beads loaded with tens of thousands of cells that are genetically engineered to produce natural IL-2, one of two cytokines the FDA has approved for treatment of cancer. The factories are just 1.5 millimeters wide and can be implanted with minimally invasive surgery to deliver high doses of IL-2 directly to tumors. In the mesothelioma study, the beads were placed beside tumors and inside the thin layer of tissue known as the pleura, which covers the lungs and lines the interior wall of the chest.

“I take care of patients who have malignant pleural mesothelioma,” said Dr. Bryan Burt, professor and chief of Baylor’s Division of Thoracic Surgery in the Michael E. DeBakey Department of Surgery. “This is a very aggressive malignancy of the lining of the lungs. And it’s very hard to treat completely by surgical resection. In other words, there is often residual disease that is left behind. The treatment of this residual disease with local immunotherapy — the local delivery of relatively high doses of immunotherapy to that pleural space — is a very attractive way to treat this disease.”

Veiseh said the mesothelioma study began when Burt and Baylor surgeon and associate professor Dr. Ravi Ghanta heard about the early results of ovarian cancer animal tests Veiseh’s team was conducting with collaborators at the University of Texas MD Anderson Cancer Center. In March, Veiseh and MD Anderson collaborators published a study showing IL-2-producing beads could eradicate advanced-stage ovarian and colorectal tumors in mice in less than a week.

“They were really impressed by the preclinical data we had in ovarian cancer,” Veiseh said of Burt and Ghanta. “And they asked the question, ‘Could we actually leverage the same system for mesothelioma?’”

Source: https://blogs.bcm.edu/

New eye drops can limit the use for reading glasses. Vuity has just been approved by the Food and Drug Administration (FDA), and local ophthalmologists say it can be a life-changer. The drops are meant for people dealing with Presbyopia, an age-related eye issue that causes blurry vision. 

“We all know the reading glasses are annoying,” said Dr. Ella Faktorovich, an ophthalmologist with Pacific Vision Institute. “Within 15 minutes you can see your computer, you can see your phone so you can really improve the range of vision. I think it is huge.” She says the drops target the focusing mechanism in the eye. The drops shrink the pupils and increase focus on theeye.

“There are many kinds of this medicine in trials, but this is the first to be approved,” she said. “It is pretty remarkable.” It can help people like Lovester Law, who is currently writing a book. He says he spends hours looking at a screen to write. “After I read too much or write to long, I just have to close my eyes and relax,” he explained.

“If we live long enough our eyes are going to age, they are not going to be like they used to be.” People who want the drops will have to consult an eye doctor, because they are only available through a prescription. Doctors at UCSF say this breakthrough can be a catalyst for future eye treatment. “The data we have shows that it really really works,” stated Julie Schallhorn, Associate Professor of ophthalmology at UCSF. “It is an exciting time to be in this field, and an exciting time for our patients.“

The FDA approval of VUITY was based on data from two pivotal phase 3 clinical studies, GEMINI 1 and GEMINI 2, which evaluated the efficacy, safety and tolerability of VUITY for the treatment of presbyopia.

Source: https://news.abbvie.com/

The Food and Drug Administration (FDA) on August 16 approved the first online visual acuity test made by the telehealth company Visibly. This clearance will allow adults ages 22 to 40 to evaluate their vision from the convenience of their homes. The FDA approval may increase access for people who are in need of a renewed prescription for glasses or contacts but cannot travel to an appointment. However, it’s not a replacement for an in-person eye exam.

“During the pandemic, a lot of people delayed elective health care that was really important,” said Yuna Rapoport, MD, MPH, a board-certified ophthalmologist at Manhattan Eye. “Overall, this remote vision test is helpful. If you really want an accurate prescription, and there’s a way to get to a doctor’s office, I would still say that that’s a better option.”

According to Visibly, the online visual test is best for “people whose vision has not changed, have recently completed a comprehensive eye exam,” and are looking to renew an expired prescription. The test is not a substitute for, nor does it provide screening or diagnosis for eye health or eye diseases, which should be performed by a licensed provider, according to the FDA.

Paul Foley, Visibly’s chief operating officer, said in a press release that the online vision test will increase at-home use and complement in-person eye care. The test takes about six minutes to complete and 90% of the prescriptions are issued within 24 hours, according to the company.

Source: https://www.govisibly.com/
AND
https://www.verywellhealth.com/

People with obesity lost 24 kilograms on average when they were treated with the highest dose of a new hunger-blocking drug in a large clinical trial. “It’s really exciting. The weight loss they’re showing is dramatic – it’s as much as you get with successful bariatric surgery,” says Michael Cowley at Monash University in Melbourne, Australia, who wasn’t involved in the research.

The drug used, called tirzepatide, combines synthetic mimics of two hormones known as GLP-1 and GIP that our guts naturally release after we eat to make us feel full. In a late-stage clinical trial, more than 2500 people in nine countries, who weighed 105 kilograms on average at baseline, were asked to give themselves weekly injections of tirzepatide at low, medium or high doses or a placebo for 72 weeks, without knowing which one they were taking.

The highest dose of tirzepatide was most effective, resulting in 24 kilograms of weight loss on average, equivalent to a 22.5 per cent reduction in body weight. In comparison, participants taking the placebo lost just 2 kilograms on average. The results were announced on 28 April by US pharmaceutical giant Lilly, which is developing the drug.

In June 2021, the US Food and Drug Administration approved another obesity drug called semaglutide, which contains a GLP-1 mimic on its own, without the addition of GIP. Semaglutide also promotes weight loss, but by about 15 per cent on average, suggesting that the added GIP component in tirzepatide gives an extra boost, says Cowley. Like semaglutide, tirzepatide can trigger side effects such as nausea, vomiting, diarrhoea and constipation that seem worse at higher doses. However, doctors’ experience withsemaglutide has revealed that starting patients on low doses and gradually increasing them can avoid these side effects, and the same may be true for tirzepatide, says Joseph Proietto at the University of Melbourne in Australia. One advantage of obesity drugs is that they can be discontinued if necessary, says Proietto. “The downside of bariatric surgery is that you can never ever have a normal meal again, not even for a special occasion,” he says. “With medication, you can still do this.”

Source: https://www.monash.edu/
AND
https://www.newscientist.com/

In a first-of-its-kind surgery, a 57-year-old patient with terminal heart disease received a successful transplant of a genetically-modified pig heart and is still doing well three days later. It was the only currently available option for the patient. The historic surgery was conducted by University of Maryland School of Medicine (UMSOM) faculty at the University of Maryland Medical Center (UMMC), together known as the University of Maryland Medicine.

This organ transplant demonstrated for the first time that a genetically-modified animal heart can function like a human heart without immediate rejection by the body. The patient, David Bennett, a Maryland resident, is being carefully monitored over the next days and weeks to determine whether the transplant provides lifesaving benefits. He had been deemed ineligible for a conventional heart transplant at UMMC as well as at several other leading transplant centers that reviewed his medical records.

 “It was either die or do this transplant. I want to live. I know it’s a shot in the dark, but it’s my last choice,” said Mr. Bennett, the patient, a day before the surgery was conducted. He had been hospitalized and bedridden for the past few months.  “I look forward to getting out of bed after I recover.”

The U.S. Food and Drug Administration granted emergency authorization for the surgery on New Year’s Eve through its expanded access (compassionate use) provision. It is used when an experimental medical product, in this case the genetically-modified pig’s heart, is the only option available for a patient faced with a serious or life-threatening medical condition. The authorization to proceed was granted in the hope of saving the patient’s life.

“This was a breakthrough surgery and brings us one step closer to solving the organ shortage crisis. There are simply not enough donor human hearts available to meet the long list of potential recipients,” said Bartley P. Griffith, MD, who surgically transplanted the pig heart into the patient. Dr. Griffith is the Thomas E. and Alice Marie Hales Distinguished Professor in Transplant Surgery at UMSOM. “We are proceeding cautiously, but we are also optimistic that this first-in-the-world surgery will provide an important new option for patients in the future.”

Considered one of the world’s foremost experts on transplanting animal organs, known as xenotransplantation, Muhammad M. Mohiuddin, MD, Professor of Surgery at UMSOM, joined the UMSOM faculty five years ago and established the Cardiac Xenotransplantation Program with Dr. Griffith. Dr. Mohiuddin serves as the program’s Scientific/Program Director and Dr. Griffith as its Clinical Director.

“This is the culmination of years of highly complicated research to hone this technique in animals with survival times that have reached beyond nine months. The FDA used our data and data on the experimental pig to authorize the transplant in an end-stage heart disease patient who had no other treatment options,” said Dr. Mohiuddin. “The successful procedure provided valuable information to help the medical community improve this potentially life-saving method in future patients.”

Source: https://www.medschool.umaryland.edu/

Viagra could be a useful treatment against Alzheimer’s disease, according to a US study. Alzheimer’s disease, the most common form of age-related dementia, affects hundreds of millions of people worldwide. Despite mounting numbers of cases, however, there is currently no effective treatment.

Using a large gene-mapping network, researchers at the Cleveland Clinic integrated genetic and other data to determine which of more than 1,600 Food and Drug Administration-approved drugs could be an effective treatment for Alzheimer’s disease. They gave higher scores to drugs that target both amyloid and tau – two hallmarks of Alzheimer’s – compared with drugs that targeted just one or the other.

US scientists say users of sildenafil – the generic name for Viagra – are 69% less likely to develop the form of dementia than non-users

“Sildenafil, which has been shown to significantly improve cognition and memory in preclinical models, presented as the best drug candidate,” said Dr Feixiong Cheng, the study lead.

Researchers then used a database of claims from more than 7 million people in the US to examine the relationship between sildenafil and Alzheimer’s disease outcomes by comparing sildenafil users to non-users.

They found sildenafil users were 69% less likely to develop Alzheimer’s disease than non-sildenafil users after six years of follow-up. To further explore the drug’s potential effect on Alzheimer’s disease, researchers developed a lab model that showed that sildenafil increased brain cell growth and targeted tau proteins, offering insights into how it might influence disease-related brain changes. Cheng cautioned that the study does not demonstrate a causal relationship between sildenafil and Alzhemer’s disease. Randomised clinical trials involving both sexes with a placebo control were needed to determine sildenafil’s efficacy, he said.

Dr Ivan Koychev, a senior clinical researcher at the University of Oxford, who was not involved in the study, said it was “an exciting development” because “it points to a specific drug which may offer a new approach to treating the condition”.

Prof Tara Spires-Jones, deputy director of the Centre for Discovery Brain Sciences at the University of Edinburgh, said there were several important limitations to consider. “While these data are interesting scientifically, based on this study, I would not rush out to start taking sildenafil as a prevention for Alzheimer’s disease.”

Dr Susan Kohlhaas, director of research at Alzheimer’s Research UK, said: “Being able to repurpose a drug already licensed for other health conditions could help speed up the drug discovery process and bring about life-changing dementia treatments sooner. “Importantly, this research doesn’t prove that sildenafil is responsible for reducing dementia risk, or that it slows or stops the disease. The only way to test this would be in a large-scale clinical trial measuring sildenafil effect against the usual standard of care.”

The findings were published in Nature Aging.

Source: https://www.theguardian.com/

Pfizer CEO Albert Bourla said he expects the company’s Covid-19 treatment pill to be effective against the omicron variant of the virus causes Covid-19.

“The good news when it comes to our treatment, it was designed with that in mind, it was designed with the fact that most mutations are coming in the spikes,” Bourla explained.  “So that gives me very high level of confidence that the treatment will not be affected, our oral treatment will not be affected by this virus.”

Pfizer submitted its application earlier this month to the Food and Drug Administration (FDA) to authorize the pill, Paxlovid, for emergency use. In a clinical trial of people age 18 and over, Pfizer found the pill reduces hospitalization and death by 89% when taken with a widely used HIV drug within three days of the start of symptoms. The pill blocks an enzyme the virus needs to replicate. It is used in combination with HIV drug ritonavir, which slows the human metabolism to allow the Paxlovid to remain active in the body longer at a higher concentration to combat the virus.

https://www.cnbc.com/