Medical Breakthroughs, Research, and News

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Single-Dose of Gene Therapy Hailed as 'Magic Wand' for Patients with Deadly Condition, Transforming Lives

CRISPR is at it again: this time providing a single-dose option to cure a debilitating genetic disorder called hereditary angioedema.

Patients who took part in the first human trial have reported dramatic improvements in their health and quality of life, easing or completely removing the painful and potentially fatal swelling that arises from the condition.

Hereditary angioedema (HAE) is a rare genetic disorder that affects about 1 in 50,000 people, and causes bouts of painful swelling arising from leaky blood vessels. The swelling can occur at any time, as often as twice a week, and can last for hours or even days.

In some cases the swelling will occur in the throat, and hospitalization is required.

HAE is caused by a mutation in the C1 inhibitor gene, which codes for the regulation of the protein kallikrein, which itself helps regulate another protein called bradykinin. The dysfunction of this three-part interaction leads to the leaky blood vessels inherent in the disease.

The phase-one human trial was conducted in the UK, Netherlands, and New Zealand, and saw 10 patients receive a dose of nanolipids delivered via the Nobel Prize-winning CRISPR gene-editing technology that corrects the C1 kallikrein gene.

“I’ve had a radical improvement in my physical and mental wellbeing,” said 54-year-old Cleveland, from Suffolk, UK. “The randomness, unpredictability, and potential severity of the attacks have made trying to live my life almost impossible. I spent my life constantly wondering if my next attack would be severe.”

Another patient described it acting as sure and thorough as a “medical magic wand,” and described herself as having a “whole new life.”

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Tomato Juice Can Kill Salmonella and Other Bacteria with Its Super Anti-Microbial Properties

Scientists have shown that tomato juice kills a particular bacteria responsible for Typhoid fever, a debilitating tropical disease, in addition to other bacteria that can harm people’s digestive and urinary tract health.

Salmonella Typhi is a human-specific pathogen often transmitted in food that not only causes all the symptoms of food poisoning but the potentially deadly Typhoid fever, which even after decades of medical advancement is still a major worldwide public health concern.

The team behind the discovery—from Cornell University, New York, set out to discover which antimicrobial peptides in tomato juice made it so effective against Salmonella.

First they checked to see if tomato juice really does kill Salmonella Typhi and once they had confirmed it did, the team looked at the tomato genome to find the antimicrobial peptides that were involved.

The most significant discovery is that tomato juice is effective in eliminating Salmonella Typhi, its hypervirulent variants, and other bacteria that can harm people’s digestive and urinary tract health.

In particular, the team identified 2 antimicrobial peptides that can eliminate these pathogens by impairing the bacterial membrane, a protective layer that surrounds the pathogen and keeps it together.

[WstateU]

Tomato Juice Can Kill Salmonella and Other Bacteria with Its Super Anti-Microbial Properties

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Buck Institute Scientists Discover a Potential Way to Repair Synapses Damaged in Alzheimer's Disease

A new study proposes a strategy for reversing the memory problems that accompany Alzheimer’s disease and related dementias, exploring an alternative to the laser-focused approach of big pharma to target toxic tau proteins known to go hand in glove with these diseases.

Rather than remove the damaging compound, scientists at the Buck Institute for Research on Aging aimed to reverse the damage the compound caused using an innate protein found in the kidneys and the brain.

“While newly approved drugs for Alzheimer’s show some promise for slowing the memory-robbing disease, the current treatments fall far short of being effective at regaining memory. What is needed are more treatment options targeted to restore memory,” said Buck Assistant Professor Tara Tracy, PhD, the senior author of the study.

The protein in question is called KIDRA, and along with being produced in the kidney, Tracy and her team identified its presence throughout brain synapses, which are the connections between neurons that allow memories to be formed and recalled.

They also found that KIDRA is deficient in the brains of people with Alzheimer’s and dementia.

“We wondered how the lower levels of KIBRA affected signaling at the synapse, and whether understanding that mechanism better could yield some insight into how to repair the synapses damaged during the course of Alzheimer’s disease,” said Buck Staff Scientist Grant Kauwe, Ph.D., co-first author of the study.

“What we identified is a mechanism that could be targeted to repair synaptic function, and we are now trying to develop a therapy based on this work.”

To figure out how KIBRA affects synapses, the team created a shortened functional version of the KIBRA protein. In laboratory mice that have a condition mimicking human Alzheimer’s disease, they found that this protein can reverse the memory impairment associated with this type of dementia. They found that KIBRA rescues mechanisms that promote the resilience of synapses.

Definitely sounds like an avenue worth pursuing and if this pans out, it's an absolute game changer for those who have, will have, or may eventually have a form of dementia.

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Kinder than Chemo Cancer Drug Cured This Young Man of Leukemia - Available in the US

Picture a very unhappy situation: you have leukemia; what are your options? Everyone knows about the side effects of chemo, and most people will have some idea about the Nobel Prize-winning CAR-T cell therapy—but there’s also a third option.

It seems startling to be so overlooked because it’s so straightforward. Blinatumomab cured the young fellow above of his B-cell acute lymphoblastic leukemia.

The BBC reports that 20 medical centers in the UK are already using off-brand stocks of blinatumomab to treat this cancer, and the country has already approved the drug for adult use.

‘Blina’ as it’s referred to for short, is also an immunotherapy drug; it seeks out and kills cancer cells that typically disguise themselves from the body’s innate immune system. However, unlike chimeric antigen receptor T cells (CAR-T cell), blina is cheaper.

Blina is a kind of targeted therapy drug called a bispecific T-cell engager (BiTE). It’s administered via a pump and plastic bag through a tube inserted into the patient’s arm.

The bag is carried around in a slim backpack, and the supply lasts a few days. CAR-T cell therapy requires a patient’s cells to be taken out and modified to fight off whichever cancer is present—which takes time.

Like CAR-T, the healthy normal cells are not destroyed as in the case of chemotherapy, allowing the patient to continue leading a mostly normal life.

“Chemotherapies are poisons that kill the leukemic cells but also kill and damage normal cells—and that is what causes their side effects,” said chief investigator and consultant pediatric hematologist, Professor Ajay Vora to the BBC. “Blinatumomab is a gentler, kinder treatment.”

Gentler on the body—not on the cancer. Arthur, now 11, was one of the first kids to receive the treatment at Great Ormond Street Hospital in London. His family got the word that his blood cancer was cured on New Year’s Eve.

If I'm reading everything correctly, Blina is only used for this type of cancer, but it seems like it would be the preferred way to attack it. Chemo is no joke and sometimes does more harm than good. Having something that doesn't destroy the body while destroying the cancer is definitely a positive. Would love to see this expanded into other treatments as well.

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New Prosthetic Hand Allows Man to Sense Temperature: 'I could feel the warmth of another person'

The first-ever prosthetic limb that allows the wearer to sense temperature and feel the warmth of another person has been created.

The MiniTouch device allows amputees to perceive and respond to temperature, an ability that developers hope will improve their human connections.

It works by transmitting thermal information from the fingertip of the prosthetic hand to the wearer’s residual arm.

Using the device, a man in Italy who’s been an amputee for three decades was able to differentiate between hot and cold objects with 100 percent accuracy.

The scientists in Italy and Switzerland hope it could soon restore a full range of sensations through prosthetics.

The MiniTouch prosthetic hand provided realistic and real-time thermal sensory feedback to a 57-year-old man from Pistoia—the first amputee to try the device.

Fabrizio was overcome with emotions after feeling the warmth of another person again, 37 years after his hand was amputated from the wrist.

“It was a very strong emotion for me. It was like reactivating a connection with someone.”

Using the MiniTouch, Fabrizio was able to discriminate between, and manually sort, objects of different temperatures or materials. (See the video below from Reuters…)

“When one of the researchers placed the sensor on his own body, I could feel the warmth of another person with my phantom hand,” said Fabrizio.

The team says the new technology, presented in a study published in the journal Med, marks the first time natural temperature sensations have been incorporated into a functional artificial limb—one of the last frontiers for restoring sensation to robotic hands.

This really is a big deal for amputees.

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New Spiral-Shaped Lens is Massive Improvement for Eyewear: 'Potentially Revolutionizing Ophthalmology'

Ophthalmologists have developed a spiral-shaped contact lens that maintains clear focus at different distances and in varying light conditions.

The new lens works much like progressive lenses used for vision correction but without the distortions typically seen with those lenses. It could help advance contact lens technologies, intraocular implants for cataracts, and miniaturized imaging systems.

The inspiration for the design came when the paper’s first author, Laurent Galinier, was analyzing the optical properties of severe corneal deformations in patients. This led him to conceptualize a lens with a unique spiral design that causes light to spin, like water going down a drain.

This phenomenon, known as an ‘optical vortex,’ creates multiple clear focus points, which allow the lens to provide clear focus at different distances.

“Creating an optical vortex usually requires multiple optical components,” Galinier told Optica. “Our lens, however, incorporates the elements necessary to make an optical vortex directly into its surface. Creating optical vortices is a thriving field of research, but our method simplifies the process, marking a significant advancement in the field of optics.”

In Optica, Optica Publishing Group’s journal for high-impact research, the researchers describe the new lens, which they call the spiral diopter, and Bertrand Simon from another optics laboratory said their invention could revolutionize ophthalmology.

“Unlike existing multifocal lenses, our lens performs well under a wide range of light conditions and maintains multifocality regardless of the size of the pupil,” said Simon from the Photonics, Numerical and Nanosciences Laboratory in France. “For potential implant users or people with age-related farsightedness, it could provide consistently clear vision, potentially revolutionizing ophthalmology.”

Pretty cool

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New Zinc Treatment 'Can Help to Restore Lost Hearing' in Mice - University Research Shows

Anyone who has ever been to a loud concert knows the feeling of ringing ears. Some people experience temporary or even permanent hearing loss or drastic changes in their perception of sound after the loud noises stop.

Scientists have discovered the biological mechanism of hearing loss caused by loud noise, which helped them find a way to prevent it.

When exposed to loud noises some people experience temporary or even permanent hearing loss or drastic changes in their perception of sound after the loud noises stop.

Researchers from the University of Pittsburgh in the US have now discovered that this noise-induced hearing loss stems from cellular damage in the inner ear that is associated with the excess of free-floating zinc, a mineral that is essential for proper cellular function and hearing.

Their experiments showed drugs that work as molecular sponges trapping excess zinc can help restore lost hearing, or if administered before an expected loud sound exposure, can protect from hearing loss.

“Noise-induced hearing loss can be debilitating. Some people start hearing sounds that aren’t there, developing a condition called tinnitus, which severely affects a person’s quality of life,” said Professor Thanos Tzounopoulos from the Pittsburgh Hearing Research Center.

“Noise-induced hearing loss impairs millions of lives but, because the biology of hearing loss is not fully understood, preventing hearing loss has been an ongoing challenge.”

To get their results, published in the journal Proceedings of the National Academy of Sciences, the team studied the inner ear cells of mice.

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This is crazy and could be insanely valuable in detecting breast cancer and catching it early.

Hand-held Test for Breast Cancer Uses Your Saliva and Gives Accurate Readings in 5 Seconds

Do women prefer a mammogram to test for breast cancer? Or would they rather place a tiny sample of saliva on a test strip and get the results in under five seconds?

A new hand-held portable device is not only extremely quick and easy to use but very cost effective, say scientists from the University of Florida and National Yang Ming Chiao Tung University in Taiwan.

The device itself, about the size of your hand, uses common components that cost just five dollars and uses widely available glucose testing strips costing just a few cents each.

The biosensor works by using paper test strips treated with specific antibodies that interact with the targeted cancer biomarkers.

When a drop of saliva is placed on the strip, pulses of electricity are sent to electrical contact points on the biosensor device.

Compared to the costly alternatives of Mammograms, which expose women to radiation—or MRIs and ultrasounds which require expensive equipment—researchers called the device revolutionary.

The team believes their device, which uses the open-source hardware-software platform Arduino, can help people in remote areas to detect breast cancer early on.

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New Synthetic Compound Can Kill Superbugs' Resistant to Antibiotics

Harvard researchers have created an antibiotic that can overcome many drug-resistant infections, which have become a deadly global health menace, killing a million people every year.

The new synthetic compound called cresomycin proved to be “highly effective” at killing deadly superbugs that are resistant to antibiotics, including Staphylococcus aureus and Pseudomonas aeruginosa.

Cresomycin is one of several promising compounds the team from Harvard University has developed to win the war against superbugs.

The team led by Professor of Chemistry and Chemical Biology Andrew Myers reported in Science how the new molecule demonstrates an improved ability to bind to bacterial ribosomes—biomolecular machines that control protein synthesis.

Disrupting ribosomal function is a hallmark of many existing antibiotics, but some superbug bacteria have evolved shielding mechanisms that prevent drugs from working.

The new molecule draws inspiration from the chemical structures of ‘lincosamides’, a class of antibiotics that includes the commonly prescribed clindamycin.

[SubGod22]

Belgian Boy is the First Child in the World to Have Been Cured of Brain Stem Glioma, a Brutal Cancer

Advances in medicine over the decades have seen the 5-year survival rate of children diagnosed with cancer increase to 85%, but there are outliers to that average—with few more sinister than brain stem glioma.

When French doctor Jacques Grill first saw these tumors in a young Belgian boy, it was a straightforward, agonizing conversation he knew he had to have with the family: 6-year-old Lucas was going to die.

But it was not to be, and following the results of an experimental treatment randomly assigned to the young boy, the unbelievable happened.

“Over a series of MRI scans, I watched as the tumor completely disappeared,” Dr. Grill told AFP. “I don’t know of any other case like him in the world.”

Brain stem glioma, officially called diffuse intrinsic pontine glioma (DIPG), is rare and lethal, diagnosed in just 300 children annually in the United States, and 100 in France, where Dr. Grill practices.

The two-year survival rate is 10%, and no drug is widely available for its treatment beyond radiotherapy.

After Lucas’ diagnosis, the family traveled to France to take part in a randomized controlled trial called the Biomede trial, looking for medications to combat DIPG. Lucas was randomly assigned the drug everolimus, which he took for more than 5 years with remarkable success.

Sounds like this kid may end up being exactly what was needed to find a cure for this cancer down the road and scientists are optimistic.

[SubGod22]

FDA Approves New Drug for A Timeless Illness - Frostbite - to Save Fingers and Toes from Amputation

It’s not a cure for ALS or cancer, but the pharmacists who just developed a “game-changer” treatment for frostbite deserve plenty of congratulations nonetheless.

On February 14th, the FDA approved Aurlumyn (iloprost) injection to treat severe frostbite in adults to reduce the risk of finger or toe amputation.

Frostbite can occur in several stages, and severe frostbite occurs when both the skin and underlying tissue are frozen and blood flow is stopped, sometimes requiring amputation. Iloprost, the active ingredient in Aurlumyn, is a vasodilator (a drug that opens blood vessels) and prevents blood from clotting.

“This approval provides patients with the first-ever treatment option for severe frostbite,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research. “Having this new option provides physicians with a tool that will help prevent the life-changing amputation of one’s frostbitten fingers or toes.”

Cold is often described as being like a “vice” or as having a “grip” in so small part because once a human’s hands or feet are frostbitten, it’s a bit like a hostage situation. One can’t simply immerse them in nice hot water, because if the ice crystals forming in the blood and tissues are warmed too fast, they will burst, causing intense pain, swelling, tissue damage, and internal bleeding.

However, warm blood from the body’s core can’t arrive at the frostbitten extremities due to the presence of frozen blood and tissue, and the only option is to slowly rewarm the frostbitten area with body heat, but even this may not be able to reverse the damage.

This has been used for years in Europe and Canada and is apparently effective up to three days after frostbite has set it.

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Scientists Find Potential Universal Antivenom to Treat Snakebites, from Kraits to King Cobras

By screening billions of human antibodies, scientists may have taken the first steps towards developing a universal antivenom for snakebites. Their screen identified one which counteracts a protein in venom found in a variety of snakes including king cobras and black mambas.

Researchers at Scripps Research Institute then found that the antibody protected mice against this variety of snake venom, and the scientists published their work in the journal Science Translational Medicine.

Everyone knows that hospitals in snake-prone areas carry antivenom which reverses the toxicity of snake bites, but it’s not a very developed or sophisticated field of medicine.

Existing antivenoms involve extracting antibodies from animals which, through exposure therapy, have developed an immunity to the venom of a particular snake.

Obviously though, this provides protection against one single snake species, but in rural communities across the tropics in low and middle-income countries, there may be several snake species that humans come in contact with.

“This antibody works against one of the major toxins found across numerous snake species that contribute to tens of thousands of deaths every year,” says senior author Joseph Jardine, PhD, assistant professor of immunology and microbiology at Scripps Research. “This could be incredibly valuable for people in low and middle-income countries that have the largest burden of deaths and injuries from snakebites.”

Scripps has been ranked several times as first in the United States, and second in the world, for biomedical discoveries, and their faculty includes multiple Nobel laureates.

Like almost all animals, snakes attack humans when they are cornered, surprised, or trying to escape. Nearly all snakes eat rodents, and rural communities often practice agriculture, an environment where rodents thrive. This, unfortunately, puts humans in close proximity to snakes on the hunt for rodents, and leads to death and amputation across Asia, South America, Africa, and Australia.

In the new work, the researchers isolated and compared venom proteins from a variety of elapids—a major group of venomous snakes including mambas, cobras, and kraits. They found that a type of protein called three-finger toxins (3FTx), present in all elapid snakes, contained small sections that looked similar across different species.

In addition, 3FTx proteins are considered highly toxic and are responsible for whole-body paralysis, making them an ideal therapeutic target.

[Kung Wu]

Scientists Find Potential Universal Antivenom to Treat Snakebites, from Kraits to King Cobras

How the in heck did you stumble across this?

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Magic