Posts with «disease & medical conditions» label

Moderna begins early-stage trials of mRNA-based HIV vaccine

Moderna has begun early-stage clinical trials of an HIV mRNA vaccine, the company announced this week. On Thursday, it administered the first doses of a shot it co-developed with the International AIDS Vaccine Initiative to volunteers at the George Washington University School of Medicine and Health Sciences.

Like the company’s COVID-19 vaccine, the new treatment uses messenger RNA to “trick” the human body into producing proteins that will trigger an immune response. Moderna hopes the shot will induce a specific class of white blood cells known as B-cells, which can then turn into broadly neutralizing antibodies. Those proteins are “widely considered to be the goal of HIV vaccination, and this is the first step in that process,” according to the company.

As part of the trial, Moderna plans to test both a primary vaccine and a booster shot. The Phase 1 trial will involve 56 healthy, HIV-negative adult participants. The company will give 48 of those individuals the mRNA vaccine. Thirty-two of that group will also receive the booster shot. To the final eight involved in the first trial, the company will only administer the booster shot. Moderna says it will then monitor the entire group for six months to gauge the safety of the vaccine. It also plans to examine the immune response the vaccine triggers at the molecular level to determine if it’s effective.

Messenger RNA technology could lead to treatments for a host of deadly diseases, including malaria, but a breakthrough against HIV would be particularly noteworthy. According to statistics from the US government, approximately 1.2 million Americans have the virus, which can lead to the deadly AIDS disease. While outcomes for HIV patients have improved significantly since the ‘90s thanks to the development of new treatments and medication, no HIV vaccine has successfully passed early clinical trials.

FDA authorizes Pfizer's Covid antiviral pill for people 12 and older

The FDA has issued an emergency authorization Pfizer's antiviral pill Paxlovid, making it the first oral method for treating mild to moderate cases of COVID-19. The treatment is meant for high-risk people 12 and older who could progress to a more serious COVID infection. The best part? The FDA says it could be available to use within a few days, making it another tool as we face the Omicron variant wave.

Paxlovid is available by prescription only, and it's meant to be taken within five days of first noticing COVID symptoms. According to Pfizer's tests, it can prevent hospitalization or death by 88 percent in high-risk patients. The treatment, which can be prescribed to both vaccinated and unvaccinated people, consists of 30 pills taken over five days. It includes the protein inhibitor nirmatrelvir and rotinavir, which keeps that inhibitor from breaking down in your body. Side effects include an impaired sense of taste, high blood pressure, diarrhea and muscle aches. 

“This authorization provides a new tool to combat COVID-19 at a crucial time in the pandemic as new variants emerge and promises to make antiviral treatment more accessible to patients who are at high risk for progression to severe COVID-19," Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in a statement.

So far, the US has ordered enough pills to treat 10 million people, the New York Times reports. The company plans to deliver enough pills to cover 65,000 Americans within a week. And after that, production is expect to ramp up, with 150,000 courses delivered in January and 150,000 in February. It also won't be the only antiviral pill around: Merck's competing treatment is expected to be approved soon, and it'll likely be more readily available than Pfizer's. Merck's option is far less effective, though—tests show it can only prevent hospitalization or death by 30 percent. (Still, that's better than having no treatment.) 

Hitting the Books: An ode to the joy of antibiotics

The stress and uncertainty surrounding the COVID pandemic, along with misinformation about the life-saving vaccines developed in response have broken many a weak mind over the past two years, leading people to try everything from injecting themselves with bleach and inhaling nebulized hydrogen peroxide, to slugging down horse dewormer in misguided attempts to outwit modern medicine. Surprise, none of it actually works. What's worse is that this sort of behavior is nothing new. Quack homeopathic remedies have existed for centuries — curing the bubonic plague through blood letting, self-flagellation, or sitting in hot sewers to drive off the fever, for example — and supported by little more than anecdotal evidence.

In their latest book, Patient Zero: A Curious History of the World's Worst Diseases, Dr. Lydia Kang and Nate Pedersen delve into the fascinating histories of some of humanity's deadliest diseases and the society-preserving works of the scientists who the developed cures, vaccines and treatments to counter them. In the excerpt below, we take a look at the deployment of antibiotics and antitoxins in the fight against diphtheria, anthrax, and other deadly diseases.  

Workman Publishing

Excerpted from Patient Zero:A Curious History of the World's Worst Diseases by Lydia Kang, MD, and Nate Pedersen. Workman Publishing © 2021


Aside from putting barriers between us and plagues, the next primary approach to defeating them was to attack them directly, thanks to breakthroughs in science that created and discovered antibiotics and antitoxins. Some of these medicines aren’t simply employed against microorganisms like bacteria, but act as antifungals, antivirals, and antiparasitics as well. Today, there are more than a hundred types of medicines in this group. The World Health Organization (WHO) maintains a list of medicines deemed essential for a country’s healthcare system to best care for its citizenry, and a large chunk of those essential meds battle infectious diseases.

Some might assume that penicillin was the first definitive weapon discovered in our fight against pathogens, but there were several that preceded it and broke significant ground when they were discovered.

The Prussian-born Emil von Behring was a doctor and an assistant to the famed Robert Koch at the Institute for Hygiene in Berlin. In 1888, he developed a way to treat those suffering from diphtheria and tetanus. Not a disease familiar to many these days, diphtheria is prevented by a vaccine that is usually coupled with your routine tetanus shot. In the 1800s, diphtheria was a terrible killer that inflamed a victim’s heart, inflicted paralysis, and caused a suffocating membrane to cover the throat. In Spain, the disease was so rampant in 1613 it was nicknamed El Año de los Garrotillos, or “The Year of Strangulations.”

Much of the disease caused by diphtheria is driven by the toxin created by Corynebacterium diphtheriae. Von Behring infected rats, rabbits, and guinea pigs with weakened (attenuated) forms of it, then gathered their serum—the liquid fraction of their blood, minus the red and white blood cells. That light, honey-colored liquid, which contained antibodies to the diphtheria toxin, was then injected into another set of animals that were sickened with fully virulent diphtheria bacteria.

The newly infected animals given the serum didn’t die because they gained a passive form of protection against the toxin with the donated serum. In 1891, a child’s life was saved using this new method for the first time. The serum was produced in large quantities using animals like sheep and horses. At a time when 50,000 children died annually from diphtheria, it was a miraculous treatment.

Tetanus serum was created soon after, becoming a workable treatment by 1915. Today, antitoxins are used to treat botulism, diphtheria, and anthrax. The same principles of antitoxin treatment are utilized for antivenom therapy to remedy poisonous animal bites, including those from black widow spiders, scorpions, box jellyfish, and cobras. A treatment called passive antibody therapy, in which the serum of patients recovered from an infection is given to other sick patients (also called convalescent plasma therapy), may have been helpful during the COVID-19 pandemic, though data is still forthcoming. Antibodies against infections can not only treat diseases like toxic shock syndrome, but prevent infections during exposures, such as those for hepatitis A and B and botulism. But the antibodies themselves have been employed to treat more than just bites, stings, and infections. Intravenous immunoglobulins from pooled donors treat a variety of disorders, such as ITP (immune thrombocytopenia) and severe immune deficiency diseases.

Another antibody therapy—monoclonal antibodies—has been a game-changer in treatments over the last decade or so, the first one approved by the FDA in 1986. These specially designed antibodies are used to treat several types of cancers (melanoma, breast, and stomach, among many others) and autoimmune diseases (including Crohn’s disease, rheumatoid arthritis, and psoriasis). The antibodies themselves are Y-shaped proteins that bind to a specific protein. In doing so, they can elicit a whole range of effects: switching on or off immune system cascades, destroying cells, blocking or engaging cell activities. The antibodies only bind to a single antigen, hence “mono,” and are produced by clones of cells that churn out the antibodies in large amounts. Sometimes they can also be bound to radioactive particles, delivering radioactivity directly to a cancer cell. Others can be bound to a chemotherapy agent. Often, they work alone.

In the realm of cancer therapy, most of us have some understanding of chemotherapy. But the origin of the term chemotherapy itself actually came from the fight to treat infections, not cancer. At the turn of the twentieth century, antibiotics had yet to establish themselves as a cure for infections. That changed with a physician and scientist named Paul Ehrlich. He was born in 1854 in East Prussia (now Poland) where his father ran a lottery office. During his career, he took advantage of the burgeoning German dye industry to experiment on how cells looked stained with different chemicals. His love of color led to some notable idiosyncrasies, like carrying colored pencil stubs in his pockets. But Ehrlich’s work led to what would become the famous Ziehl-Neelsen acid-fast stain for tuberculosis. (Unfortunately, he also stained his very own TB bacteria from his sputum, though luckily survived the illness.) Later he collaborated with the aforementioned Emil von Behring, a Nobel Prize–winning physiologist, on serum therapy for tetanus and diphtheria.

But perhaps Ehrlich’s most notable discovery happened by accident as he sought a chemical cure to treat a specific disease—a “chemotherapy.” Specifically, he hoped to cure sleeping sickness, a disease caused by a microscopic parasite called Trypanosoma brucei. He had been working with a chemical called atoxyl (meaning “nontoxic”), ironically an arsenic compound. Ehrlich coined the term “magic bullet” related to his hope of finding that perfect chemical that would hopefully kill a very specific pathogen, the Trypanosoma parasite, and not the patient. He ended up testing nine hundred variations of the arsenic compounds on mice. None were particularly effective, but he revisited #606 because it seemed to have an effect on a newly discovered bacterium believed to cause syphilis. In 1910, the medicine called Salvarsan (sometimes simply called “606”) was proven to be effective—it killed the syphilis spirochete and left the guinea pigs, rabbits, and mice alive.

In the next few decades, new research would be applied to battle not just the pandemics of old, but daily infections that could upend people’s lives. A scratch or bite could kill if those Staphylococcus or Streptococcus infections spiraled out of control. A German scientist named Gerhard Domagk began working with a group of chemicals called azo dyes that had a characteristic double nitrogen bond. Azo dyes can color textiles, leather, and foods various shades of brilliant orange, red, and yellow. When an azo compound had a sulfonamide group attached (a nitrogen and sulfur link with two oxygen atoms double-bonded to the sulfur, should you need to impress friends at a party), they knew they’d found something special. The sulfonamide group inhibits a bacteria’s ability to make folate, a necessary B vitamin. Humans, on the other hand, can obtain folate through their diet. And so another magic bullet was born. The new compound seemed to work in mice infected with Streptococcus, otherwise known as strep.

Domagk used the new medicine, called KL 730 and later patented as Prontosil, on his own daughter Hildegard. Suffering from a severe strep infection, she received a shot of Prontosil and recovered, though the drug left a telltale dyed, reddish discoloration at the injection site.

“Sulfa” drugs would go on to be used in a variety of medicines, including antibiotics (trimethoprim and sulfamethoxazole, aka Bactrim), diabetes medicines (glyburide, a sulfonylurea), diuretics (furosemide, or Lasix), pain meds (celecoxib, or Celebrex), and are also used today to treat pneumonia, skin and soft tissue infections, and urinary tract infections, among others.

Domagk’s work won him the Nobel Prize in 1935. However, the Nazis, who disapproved of how the Nobel committee tried to help German pacifist Carl von Ossietzky, had their Gestapo arrest Domagk for accepting the prize and forced him to give it back. He was able to receive it later in 1947.

FDA proposes rule for over-the-counter hearing aids

The Food and Drug Administration is moving closer to making more affordable over-the-counter (OTC) hearing aids a reality for millions of Americans with mild or moderate hearing loss. The agency issued a proposal to create a category of approved devices that people would be able to buy without a prescription, hearing exam or having to arrange a fitting with an audiologist.

"The proposed rule is designed to help increase competition in the market while also ensuring the safety and effectiveness of OTC and prescription hearing aids," the FDA said. Around 15 percent of adult Americans (some 37.5 million) have hearing difficulties, according to the agency.

The FDA's goal is to make it easier for those who could benefit from hearing aids to actually get one — it says only a fifth of people who fall into that category use such a device. The agency is hoping to tackle some of the barriers people might encounter, including cost, ease of access, social stigma and state and federal regulations.

In 2017, the federal government passed the Over-the-Counter Hearing Aid Act with the aim of improving access to more affordable hearing aids. Hearing aids have only available with a prescription as the FDA classed them as Class I or II medical devices. President Joe Biden signed an executive order in July that, in part, instructed the Secretary of Health and Human Services to publish a proposed rule for OTC hearing aids within 120 days.

The proposal is now open to a 90-day public comment period. If and when the rule is finalized, it will come into effect 60 days after it's published in the federal register.

Several companies have already made moves to gain a foothold in the OTC hearing aid market. Earlier this year, Bose started selling its SoundControl hearing aids after gaining approval from the FDA, while Jabra unveiled its Enhance Plus earbuds a couple of months ago. Other companies are blending hardware and tech in hearing aids, including Bragi and Olive Union.

Apple, meanwhile, recently updated AirPods Pro with a feature that amplifies the volume of other people's voices in conversation while reducing ambient noise. The company is also said to be looking into ways of using AirPods as health devices.

Jabra's latest earbuds are for people with mild-to-moderate hearing loss

A number of companies make "hearing enhancement" devices that look more like a set of earbuds than a piece of clinical tech. Nuheara has been a staple at CES with its IQbuds line and companies like Bose, Bragi, Olive and others have offered a mix of tech and hardware to assist with hearing loss. Even Apple plans to introduce a "Converstaion Boost" for its AirPods Pro. Since the FDA allows companies to sell directly to consumers with mild-to-moderate hearing loss without the need for a prescription, the list of options is constantly growing. Another company that's specifically equipped to blur the line between hearing aid and true wireless earbuds is Jabra, thanks to the auditory assistance expertise of its parent company GN. 

With the Jabra Enhance Plus, the company offers a more approachable device for people who may not need what all-day hearing aids offer just yet. Jabra describes these earbuds as "a miniaturized true wireless form factor" that's 50 percent smaller than its stellar Elite 75t model. Those are already some of the smallest buds I've tested, so reducing the size even further makes the Enhance Plus more comfortable and more discreet. Plus, a design that resembles earbuds rather than a traditional hearing aid helps reduce the stigma around wearing something that helps your hear better. 

Inside, four separate sound processing features work to improve audio quality. The Enhance Plus analyzes sound to keep things as natural as possible while also reducing noise for speech clarity. The earbuds also ensure feedback doesn't hinder amplification and they isolate sounds coming from in front of you. 

Jabra

In addition to providing hearing enhancement, the Jabra Enhance Plus can work just like a set of true wireless earbuds to play music and take calls. Similar to other earbuds, the Enhance Plus comes with multiple sizes of ear tips to help you find the best fit, on-board controls and water/dust resistance (IP52). Jabra says they'll last 10 hours on a charge with 30 hours total when you factor in the charging case. An app assists with setup and offers a degree of customization. 

Jabra plans to launch the Enhance Plus at "select hearing care clinics" in the US "towards the end of the year." A licensed professional will conduct a hearing test to make sure these earbuds are appropriate. There's no word on pricing just yet, but the company says it's applying for approval under the FDA's self-fitting category. If you're looking for more of a true hearing aid rather than these "enhancers," Jabra also offers the Enhance Pro. It carries the more traditional behind-the-ear hearing aid design along with a charging case. It's also pricey, starting at $1,800. 

If that's what you're after, the Bose SoundControl hearing aids went on sale in May in a handful of states. That device puts the company's audio expertise to use to help you hear better, and Bose said it was the first FDA-cleared hearing aids that could be sold directly to consumers. What's more, they're more affordable at $850, but they run on the typical zinc-air batteries for hearing aids rather than being rechargeable. 

IBM's AI can predict how Parkinson's disease may progress in individuals

In the past, we’ve seen doctors use AI software to detect brain tumors, kidney illness and various cancers. Now, researchers from IBM and Michael J. Fox Foundation (MJFF) say they’ve developed a program that can predict how the symptoms of a Parkinson’s disease patient will progress in terms of both timing and severity. In The Lancet Digital Health journal, they claim the software could transform how doctors help patients manage their symptoms by allowing them to better predict how the disease will progress.

“Our aim is to use AI to help with patient management and clinical trial design. These goals are important because, despite Parkinson’s prevalence, patients experience a unique variety of motor and non-motor symptoms,” IBM said.

The breakthrough wouldn’t have been possible without the Parkinson’s Progression Markers Initiative, a study the Michael J. Fox Foundation sponsored. IBM describes the dataset, which includes information on more than 1,400 individuals, as the “largest and most robust volume of longitudinal Parkinson’s patient data to date” and says it allowed its AI model to map out complex symptom and progression patterns.

It’s estimated Parkinson’s disease affects more than 6 million people globally, and there’s currently no known cure for it. IBM Research and MJFF plan to continue work on the AI model. In the future, they hope to make it better at providing more granular characterizations of the various stages of the disease.

A magnetic helmet shrunk a deadly tumor in world-first test

We've seen helmets and AI that can spot brain tumors, but a new hard hat can actually treat them, too. As part of the latest neurological breakthrough, researchers used a helmet that generates a magnetic field to shrink a deadly tumor by a third. The 53-year-old patient who underwent the treatment ultimately passed away due to an unrelated injury. But, an autopsy of his brain showed that the procedure had removed 31 percent of the tumor mass in a short time. The test marked the first noninvasive therapy for a deadly form of brain cancer known as glioblastoma.

The helmet features three rotating magnets connected to a microprocessor-based electronic controller operated by a rechargeable battery. As part of the therapy, the patient wore the device for five weeks at a clinic and then at home with the help of his wife. The resulting magnetic field therapy created by the helmet was administered for two hours initially and then ramped up to a maximum of six hours per day. During the period, the patient's tumor mass and volume shrunk by nearly a third, with shrinkage appearing to correlate with the treatment dose.

The inventors of the device — which received FDA approval for compassionate use treatment — claim it could one day help treat brain cancer without radiation or chemotherapy. “Our results...open a new world of non-invasive and nontoxic therapy...with many exciting possibilities for the future,” said David S. Baskin, corresponding author and director of the Kenneth R. Peak Center for Brain and Pituitary Tumor Treatment in the Department of Neurosurgery at Houston Methodist Neurological Institute. Details of the procedure have been published in the peer-reviewed journal Frontiers in Oncology.

Moderna enters clinical trials for its mRNA-based flu vaccine

Moderna has injected its mRNA-derived vaccine for the seasonal flu into a human volunteer for the first time as part of a Phase 1/2 clinical study, the company announced on Wednesday. 

This is a very early test for the new vaccine technology, geared primarily towards building a baseline understanding of the treatment's "safety, reactogenicity and immunogenicity," according to a Moderna release. mRNA-1010, as the vaccine has been dubbed, is designed to be effective against the four most common strains of the virus including, A H1N1, H3N2, influenza B Yamagata and influenza B Victoria. According to the World Health Organization, these strains cause between 3 and 5 million severe cases of flu every year, resulting in as many as 650,000 flu-related respiratory deaths annually. In the US alone, roughly 8 percent of the population comes down with the flu every winter. The company hopes this vaccine will prove more potent than the current 40 - 60 percent efficacy rate of conventional flu vaccines.  

“We are pleased to have begun this Phase 1/2 study of mRNA-1010, our first mRNA seasonal flu vaccine candidate to enter the clinic. We expect that our seasonal influenza vaccine candidates will be an important component of our future combination respiratory vaccines,” Stéphane Bancel, Moderna's CEO said. “Respiratory combination vaccines are an important pillar of our overall mRNA vaccine strategy. We believe that the advantages of mRNA vaccines include the ability to combine different antigens to protect against multiple viruses and the ability to rapidly respond to the evolution of respiratory viruses, such as influenza, SARS-CoV-2 and RSV. Our vision is to develop an mRNA combination vaccine so that people can get one shot each fall for high efficacy protection against the most problematic respiratory viruses."

This vaccine has been generated using the same genomic techniques the company utilized to develop its COVID-19 treatment in 2020. The technique works by exploiting the human body's own cells to reproduce snippets of viral DNA to instigate an immune response and prime the body against future infection. Since this method doesn't require the entire virus (either weakened or dead) but rather just a birt of its genetic code, mRNA vaccines could be applied to any number of deadly modern diseases including malaria, TB — even cancer.