Medical Breakthroughs, Research, and News

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Experimental Eyedrops Bring Back Boy's Eyesight

A teen born with a rare condition that causes blisters in his eyes can now see again, thanks to gene therapy eyedrops.

Antonio Vento Carvajal was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. He's been legally blind for much of his 14 years.

But his skin improved when he joined a clinical trial to test the world's first topical gene therapy. That gave Dr. Alfonso Sabater an idea.

What if it could be adapted for Antonio's eyes?

Carvajal had multiple surgeries to remove scar tissue from his eyes, but it always grew back. His vision kept getting worse, eventually deteriorating so much that he didn't feel safe walking around.

Sabater had no answers then, and tried to reassure the boy: "I'll find a solution. I just need some time. I'm working on it."

"'Yeah, I know you're going to do it'," Sabater recalled Antonio saying. "That gave me the energy to continue."

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Groundbreaking Immunization Against Lyme Disease Takes Aim at the Microbiomes of Ticks and Mosquitos

The glory days for some of the most debilitating pathogenic diseases may be numbered thanks to a new class of immunizations that change the gut microbiome of ticks and mosquitoes.

Rather than targeting the pathogen that causes a disease, the vaccine targets the microbes that the pathogen requires to survive, and a new study investigating a vaccine that did just that demonstrated it was remarkably successful in mice.

While human use for a vaccine typically takes 8-10 years of tests and development, a similar vaccine could be prepared for animals such as dogs, cats, and livestock much sooner which would have the potential to save pet owners hundreds in veterinary bills, and ranchers tens of thousands.

Lyme disease needs little introduction: it’s a debilitating condition caused by the Borrelia genus that can leave a human or animal weak and bedridden for a decade. It’s been previously discovered that Borrelia pathogens can alter a tick’s gut microbiome

A new study found that by immunizing mice with a harmless E. coli bacteria as a Trojan horse, it elicited an immune response within the tick’s gut that greatly reduced the amount of Escherichia-Shigella, a common bacterial taxa found within the guts of mice and humans.

The authors report that this greatly decreased the ability of Borrelia afzelii, one of the pathogens that cause Lyme disease, to survive within its tick host, and protected the mice as well.

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Molecule that Kills Most Solid Cancer Tumor Cells Leaving Others Unaffected Shows Promise After 20 Years' Hard Work

City of Hope today announced that the first patient to receive its novel, promising cancer medicine AOH1996 is doing well.

The Phase 1 clinical trial testing the safety of a potentially cancer-stopping therapy in people with reoccurring solid tumors is expected to continue for the next two years. The investigational pill has been effective in preclinical research treating cells derived from breast, prostate, brain, ovarian, cervical, skin, and lung cancers.

Linda Malkas Ph.D., pioneer of AOH1996 at City of Hope’s Department of Molecular Diagnostics & Experimental Therapeutics, began her research 20 years ago, believing that proliferating cell nuclear antigen (PCNA), which plays an essential role in the replication and repair of cells, would be a less toxic cancer therapy that targets mutated cancer cells while leaving normal cells alone.

The new treatment has been shown in preclinical research to target PCNA and inhibit the growth and spread of a broad range of human cancer cells.

The research protocol notes that AOH1996 is not toxic to healthy cells and that treatment with this medicine both pauses cell DNA synthesis and inhibits DNA repair, leading to a type of cell death known as apoptosis in the cancer cells.

So many different avenues showing promise. I like it.

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Interesting medical news out of the UK that has a history as far back as at least Rome.

Hospitals Could Use Honey and Vinegar as Antibiotic for 'Low-Cost' Wound Care

In a win for homeopathic medicine, the nation that discovered penicillin is now opting to use food ingredients as a wound cleaner in hospitals.

The UK is trialing, and seeing major success, with using a combination of medicinal-grade honey and food-grade vinegar to clean wounds rather than antibiotics or antiseptics.

Known as oxymel, the mixture has been written about in the medical literature of at least Classical Rome, and likely older civilizations as well.

“In our survey of pre-modern recipes we noticed a pattern of combining honey and vinegar to wash or dress wounds and swellings, and this inspired us to focus on that combination in our analysis,” said Dr. Erin Connelly, a researcher on the study.

Today at Warwick University, Connelly and her team of researchers at the British National Health Service were looking to see if bacterial concentrations could be reduced when ensconced within the protections of a biofilm.

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AI Can Accurately Detect 20% More Breast Cancers than Traditional Screening by Radiologists

A trained radiologist assisted by artificial intelligence was able to spot 20% more breast cancers in mammogram screenings without any increase in false positives, a new study showed.

Pitting the man-machine team against two trained radiologists, the authors believe it’s the first randomized controlled trial demonstrating the effectiveness of AI-assisted breast cancer screening.

The study found that out of 80,000 mammogram screenings done in Sweden, the human-AI pairing was able to identify breast cancer in 6 out of 1,000 women, compared to a rate of 5 in 1,000 for the two radiologists, corresponding to a 20% increase.

Yet the scientist felt the AI was not overly sensitive and didn’t contribute to any unusually high rate of false positives.

While this won’t mean that you’ll be screened by robots any time soon, the authors believe the study represents that AI detection could be a safe and effective way to increase the speed, efficiency, and accuracy of breast cancer screenings.

“The greatest potential of AI right now is that it could allow radiologists to be less burdened by the excessive amount of reading,” study co-author Dr. Kristina Lång, an associate professor of radiology diagnostics from Lund University in Sweden, told CNN.

One of the reasons that Dr. Lång mentions the burden of reading is that the human within the human-AI pairing had their workload reduced by 44% over the course of the study.

Early detection of breast cancer, as in the case with most cancers, can increase the survival rate significantly. Routine mammograms are recommended as women age, and currently the rate of this cancer is increasing. This would suggest that the work per radiologist will only increase over the years as average age of the Western population continues to rise.

Interesting use of AI that could be a long-term benefit.

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How Lizards Regenerate their Tails Could Lead to Arthritis Treatments: Key Cartilage Cells Identified

Lizards have the ability to shed their tails to escape predators, after which they will regrow in around 60 days’ time.

Although it looks like a normal tail, the original bone is actually replaced by softer cartilage.

Now researchers at the Keck School of Medicine in the University of Southern California have identified key cells that facilitate that cartilage regeneration.

They believe their discovery could unlock ways to rebuild cartilage damaged by osteoarthritis, a crippling degenerative disease for which at present there is no cure, and which affects 10% of all Americans.

Lizards are among the only higher vertebrates capable of regenerating cartilage that does not harden and are the closest relatives to mammals that can regenerate.

Could eventually be a game changer for millions.

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Using the Body's 'Invisible Scalpel' to Remove Brain Cancer With Immunotherapy at Salk Institute

A team of scientists at the Salk Institute have found that a particular kind of immunotherapy was able to train the brain’s ‘invisible scalpel’ on an incurable form of brain cancer in mice.

Glioblastoma is the most common form of brain tumor, and while it’s also the most deadly, our brains come equipped with the immune cells needed to destroy them.

The problem is, cancer tumors have mechanisms that allow them to evade the detection of the immune system. That’s where immunotherapy comes in. While not universally effective, many tumors can be treated or even cured by immunotherapy, which helps the immune system see through these evasive mechanisms and destroy the cancer.

Professor Susan Kaech, director of the NOMIS Center for Immunobiology and Microbial Pathogenesis, led a team to investigate whether immunotherapy could be used on glioblastomas by experimenting with an immunotherapy drug called anti-CTLA-4 and two specialized immune cells called CD4+ T cells and microglia respectively.

“There are currently no effective treatments for glioblastoma—a diagnosis today is basically a death sentence,” says Professor Susan Kaech, senior author of the paper published in the journal Immunity.

“We’re extremely excited to find an immunotherapy regimen that uses the mouse’s own immune cells to fight the brain cancer and leads to considerable shrinkage, and in some cases elimination, of the tumor.”

Sure would be nice to give people with this cancer a better chance at survival.

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Newly-Identified Molecule Could Be the Key to Targeting Obesity and Osteoporosis

Scientists in Australia have identified a molecule that could hold the key to developing peptide-based drugs targeting obesity, osteoporosis, and inflammatory diseases.

Researchers at the Victor Chang Cardiac Institute are shedding light on how the small molecule regulates the sensors that are central to many processes in the body—including how nerve cells in the skin can sense when we are being touched.

They believe it will now be possible to design new therapeutics that could either ramp down or dial down the activity of the sensors, also known as PIEZO ion channels.

The first targets would be obesity and bone diseases such as osteoporosis.

“These are really key molecules that constantly provide information to the brain such as where our bodies are in space, sensing touch and even pain,” said lead author Dr. Charles Cox.

“This interacting molecule we have identified represents a switch enabling us to regulate these channels, widely expressed throughout the body, which is why it could be useful for a whole range of diseases in the future.”

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Scientists Regrow Retina Cells to Tackle Leading Cause of Blindness Using Nanotechnology

Macular degeneration is the leading cause of blindness in developed countries, but regrowing the human cells lost to this condition was the feature of a new successful treatment that took advantage of advances in nanotechnology.

Regrowing the cells of the human retina on a scaffold of synthetic, tissue-like material showed substantial improvements over previously used materials such as cellulose, and the scientists hope they can move on to testing their method in the already blind.

Macular degeneration is increasing in prevalence in the developed world. It’s the leading cause of blindness and is caused by the loss of cells in a key part of the eye called the retina.

Humans have no ability to regrow retinal pigment cells, but scientists have determined how to do it in vitro using pluripotent stem cells. However as the study authors describe, previous examples of this procedure saw scientists growing the cells on flat surfaces rather than one resembling the retinal membrane.

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A Powerful Antibiotic That Can Tackle Superbugs Has Been Discovered in the 'Dark Matter' of Bacteria

A new drug, called Clovibactin, has been created out of “dark matter bacteria” which rather than being a research term, was created by the press to suit the mysterious origins of Clovibactin and potential future drugs like it, which could represent an urgently needed new font of antibiotics.

Scientists in the US managed to grow the bacteria out of a morass of life cleverly coined ‘bacterial dark matter,” due to the fact that it was based on a lifeform that can’t be cultured in a petri dish.

99% of all bacteria are similarly unculturable and could not be grown in laboratories previously, hence the moniker dark matter, because 95% of the universe is made up of dark matter and dark energy.

The new drug was discovered from bacterial dark matter found in sandy soil from North Carolina, and developed by an international team of researchers alongside the company NovoBiotic in Boston.

“We urgently need new antibiotics to combat bacteria that become increasingly resistant to most clinically used antibiotics,” said Dr. Markus Weingarth, a researcher from the chemistry department of Utrecht University.

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New Blood Test Could Diagnose Parkinson's Before it Begins Damaging the Nervous System

American scientists developed a test that focuses on damage to mitochondrial DNA in the blood linked with Parkinson’s disease.

Current diagnoses of Parkinson’s are largely based on clinical symptoms after significant neurological damage has already occurred, and researchers hope this new test could be used to diagnose patients prior to such damage occurring.

Parkinson’s is the second most common neurological disease behind Alzheimer’s. The progressive disorder affects the nervous system and parts of the body controlled by nerves and afflicts around ten million people across the globe.

The study team, led by neuroscientists at the Duke University School of Medicine focused their work on DNA damage in mitochondria.

Mitochondria contain their own DNA, which can undergo damage separately from the nuclear DNA that encodes most of an organism’s genome, and previous studies have shown the association between mitochondrial DNA damage and an increased risk of Parkinson’s disease.

Sounds like this could lead to better treatment and a better life as it would be caught before Parkinson's takes its toll.

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Ultrasound Scanners Inside of a Bra Could Detect Breast Cancer Earlier, and Between Screenings

A miniature ultrasound device, fitted to a flexible polymer strap, can be slipped into a bra and detect breast cancer tumors among those most at risk of developing one.

The invention is significant for several reasons, and if perfected and developed, could save hundreds of lives.

The first reason it could be a major medical advancement is that developing breast cancer tumors in their earliest stages leads to a nearly 100% survival rate. The second is that developing a tumor even in the space between mammograms does happen, and not at a small rate. Depending on the country, 20-30% of all tumors are found in people who were in between routine screenings.

The device was developed at MIT, and once worn, it allows the user to move an ultrasound tracker across the bra and check for tumors.

“We changed the form factor of the ultrasound technology so that it can be used in your home. It’s portable and easy to use, and provides real-time, user-friendly monitoring of breast tissue,” says Canan Dagdeviren, an associate professor in MIT’s Media Lab, senior author of the study demonstrating its functionality, and inventor of the device.

Has anyone not been impacted in some way by breast cancer? I know I have a cousin who fought and won. I've had friends who've lost mothers to it. I also know some people are genetically more susceptible to it and something like this could go a long way towards saving lives.

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Here's another one that could be massive!

Implanted Artificial Kidney That Frees Patients From Dialysis On Horizon After Successful Trial

Sufferers living with kidney failure can soon look forward to a future without dialysis or a long waits for an organ transplant, thanks to efforts from the University of California in San Francisco that produced an implantable device that mimics the organ’s daily functions.

Their first clinical trial showed that kidney cells, housed within the device called a bioreactor, can work quietly in the background, like a pacemaker, and does not trigger the recipient’s immune system to go on the attack—a major stumbling block for patients who need to take harsh immunity-suppressing drugs after receive donor transplants.

More than 500,000 people in the U.S. require dialysis several times a week, whereby their blood is filtered by a machine. Many seek kidney transplants, but there are not enough donors, and only about 20,000 people receive them each year. An implantable artificial kidney would be a boon.

The device, developed by the university’s Kidney Project, has been successfully implanted and tested in pigs for seven days. The next step will be a month-long trial—first in animals and then humans.

The scientists plan to fill the bioreactor with additional kidney cells that can mimic all the functions, such as balancing the body’s fluids and releasing hormones to regulate blood pressure—then, a full artificial kidney will be born.

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Experts Find Out Why Exercise Prevents Alzheimer's Disease - Which Could Lead to Cure

The reason exercise can prevent Alzheimer’s disease has been discovered and it could lead to new treatments for the currently incurable condition.

Experts have found that a hormone called irisin released during a work-out is associated with the plaques and tangles in the brain thought to cause Alzheimer’s.

Physical exercise has been shown to reduce amyloid beta deposits in various mouse models but the mechanisms involved have remained a mystery.

Now the team from Massachusetts General Hospital have published the results in the journal Neuron, which appear to solve the puzzle.

Previously the researchers had developed the first 3D human cell culture models of Alzheimer’s. Their studies documented two major hallmarks of the condition, the generation of amyloid beta deposits followed by tau tangles in the brain.

Alzheimer's is one of the worst diseases out there in my opinion. Hopefully this discovery can lead to a true treatment, or pave the way for something else that does.

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Psychedelic Drugs May Be Able to Treat Brain Injuries, Stimulating New Neurons to Replace Impaired Ones

Most brain injury research so far—conducted in test tubes and lab animals, with only a few small studies in people—points to the psychedelics’ potential to limit brain damage after injury, stimulate the birth of new neurons to replace impaired ones, and open the learning windows that let the brain acquire new skills.

One recent study, published in the journal Nature, found lab animals taking psychedelics adopted skills as adults that were previously limited to childhood.

This has been shown in psychedelic studies before when Johns Hopkins found that MDMA, also known as ‘ecstasy’ reopened “critical windows” of brain development typical of singular periods in life when the brain is more sensitive to environmental stimuli.

These critical windows are key to post-dose therapy sessions in states where psychedelic-assisted therapy is a legal medical practice. The brain is now open to new ideas and a skilled psychoanalyst can help patients recover from serious trauma in those key moments.

Much of this has been known for years, but now, Johns Hopkins is further opening these doors of discovery by identifying this critical window reopening can be induced by other compounds, such as psilocybin, the psychoactive ingredient in psychedelic mushrooms and LSD.

Furthermore, scientists at the famous institution have found that these critical window periods form naturally in humans following a traumatic brain injury or stroke, and psychedelics can keep that critical open for longer than would normally be the case.

The idea is that psychedelic therapy could keep the critical windows open long enough to recover far more function than would otherwise be the case.