Medical Breakthroughs, Research, and News

[WstateU]

This one is big for a lot of people.

Baldness May Be Treated by Sugar That Naturally Occurs in the Human Body

I’m definitely not Fabio, but they put me on ‘Finasteride’ about 20 years ago when I had a bought with Prostatitis; the difference is amazing… the young ‘flappers’ still look my way.

[SubGod22]

Potential New Source for Drugs to Fight Antibiotic-Resistant Bacteria Found Deep in Arctic Ocean

“Promising” antibiotic candidates were found by Finnish scientists in microbes under the seafloor in the Arctic Ocean.

70% of all currently licensed antibiotics have been derived from actinobacteria in the soil, but most environments on Earth have not yet been searched for them.

Scientists say that focusing the search on actinobacteria in other habitats is a good strategy—especially if it were to yield new molecules that neither kill bacteria outright nor stop them from growing, but only reduce their “virulence” or capacity for causing disease.

They explained it’s hard for targeted pathogenic strains to evolve resistance under such conditions, while such anti-virulence compounds are also less likely to cause unwanted side effects.

The rate of discovery of fundamentally new antibiotics has been much slower than in previous decades, and opening research objectives to include antivirulent agents could greatly increase the number of potential candidates available for testing.

“We discovered a compound that inhibits enteropathogenic E. coli (EPEC) virulence without affecting its growth, and a growth-inhibiting compound—both in actinobacteria from the Arctic Ocean,” said Professor Päivi Tammela, of the University of Helsinki.

Tammela and his team were part of the crew onboard Kroprins Haakon, a research vessel that made a trip around the seas off the Norwegian archipelago of Svalbard in 2020. They developed a new suite of methods that can test for the anti-virulence and antibacterial effects of hundreds of unknown compounds simultaneously.

They targeted an EPEC strain that causes severe and sometimes deadly diarrhea in children under five, especially in developing countries. EPEC causes disease by adhering to cells in the human gut.

Once it adheres to those cells, EPEC injects so-called ‘virulence factors’ into the host cell to hijack its molecular machinery, ultimately killing it.

The tested compounds were derived from four species of actinobacteria, isolated from invertebrates sampled in the Arctic Sea off Svalbard.

[pinstripers]

"In medical news, a new test can now detect prostate trouble months earlier than any previous test.
The only downside? It involves shoving some huge device up your ass.
So... it's not all gravy, you know?"

------------Norm Macdonald

[SubGod22]

Targeted Sound Waves Treat Pain and Depression in as Little as One 40-minute Session

A crown-like device has been relieving patients of pain and depression in clinical trials, and has the authors excited.

Called the Diadem, named for a crown-like adornment worn by sovereigns across time, it sends therapeutic sound waves to targeted regions of the brain with millimeter precision.

These sorts of non-pharma treatments can raise eyebrows with some, as for the last generation, the prescription of SSRIs for depression has been widespread and nearly universal among American clinicians.

But the paper’s lead author Tom Riis, a postdoctoral researcher in the Department of Biomedical Engineering at the University of Utah, reports rave reviews among his team.

“We’ve been blown away by the positive results so far,” Rise told New Atlas. “After just a single 40-minute stimulation session, patients are showing immediate, clinically substantial improvements in symptoms.”

“While it should be kept in mind that not every participant saw drastic improvement, in the ones that did the change could be quite remarkable,” he added. “For several, you could just see it in their eyes—coming out of the session, their mood and behavior were a total 180 from when they had walked in. They were noticeably at ease, less burdened, more present.”

The paper on the device was conducted during phase 2 clinical trials, and the authors, blown away by the results as they admitted, are quickly looking to advance to phase 3.

In the phase 2 results, among 20 people who were treated with the Diadem, 60% of the patients reported a 33% reduction in pain immediately following treatment. When tested on those with clinically significant depression, 10 of 14 reported remission one week later after just one session with the device.

The Diadem works by sending ultrasonic frequencies to specific areas of the brain like the anterior cingulate cortex which is partly the center of pain and emotional regulation in our minds.

[C0|dB|00ded]

Targeted Sound Waves Treat Pain and Depression in as Little as One 40-minute Session

I like my targeted sound waves in the form of a concert.

[WstateU]

I sleep to the sound of the ocean every night...

"I sleep like a baby..."

[SubGod22]

I actually pull up thunderstorms often times when I go to bed.

[SubGod22]

This is awesome.

100 Times Improvement in Sight Seen After Gene Therapy Trial for Disease That Deteriorates Vision in Childhood

Patients with an inherited disease that caused them to lose much of their sight early in childhood experienced a quick return of vision after they received gene therapy.

The new treatment addressed the genetic mutation that caused their vision’s deterioration, letting them see 100 times better than before.

Some patients even experienced a 10,000-fold improvement in their vision after receiving the highest dose of the therapy, according to researchers from the University of Pennsylvania’s School of Medicine.

“That 10,000-fold improvement is the same as a patient being able to see their surroundings on a moonlit night outdoors as opposed to requiring bright indoor lighting before treatment,” said the study’s lead author, Artur Cideciyan, PhD, a professor of Ophthalmology and co-director of the Center for Hereditary Retinal Degenerations.

“One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire.”

A total of 15 people participated in the Phase 1/2 trial, including three pediatric patients. Each patient suffered from Leber congenital amaurosis (LCA1) as a result of mutations in the GUCY2D gene, which is essential to producing proteins critical for vision. The rare condition, which affects 100,000 people worldwide, causes significant amount of vision loss as early as infancy.

All subjects had severe vision loss with their best measure of vision being equal or worse than 20/80—meaning if a typically-sighted person could see an object clearly at 80 feet, these patients would have to move up to at least 20 feet to see it. Glasses provide limited benefit to these patients because they correct abnormalities in the optical focusing ability of the eye, and are unable to address medical causes of vision loss, such as genetic retinal diseases like LCA1.

The clinical trial published in The Lancet tested different dosage levels of the gene therapy, ATSN-101, which was adapted from the AAV5 microorganism and was surgically injected under the retina.

Improvements were noticed quickly, often within the first month, after the therapy was applied and lasted for at least 12 months.

[SubGod22]

Science is fascinating.

Researchers Bend DNA Strands with Light, Revealing a New Way to Study the Genome

With the flick of a light, researchers have found a way to rearrange life’s basic tapestry, bending DNA strands back on themselves to reveal the material nature of the genome.

Scientists have long debated the physics of chromosomes—structures at the deepest interior of a cell that are made of long DNA strands tightly coiled around millions of proteins.

Do they behave more like a liquid, a solid, or something in between? Much progress in understanding and treating diseases like cancer depends on the answer.

A Princeton team has now developed a way to probe chromosomes and quantify their mechanical properties: how much force is required to move parts of a chromosome around and how well it snaps back to its original position.

According to their findings, the answer to the material question is that the chromosome acts in some ways like fluid, and in others with elasticity. By leveraging that insight in exacting detail, the team was able to physically manipulate DNA in new and precisely controlled ways.

“What’s happening here is truly incredible,” said Cliff Brangwynne, the director of Princeton’s Omenn-Darling Bioengineering Institute and principal investigator of the study. “We haven’t been able to have this precise control over nuclear organization on such quick timescales before.”

The key to the new method lies in the researchers’ ability to generate tiny liquid-like droplets within a cell’s nucleus. The droplets form like oil in water and grow larger when exposed to a specific wavelength of blue light. Because the droplets are initiated at a programmable protein—a modified version of the protein used in the gene-editing tool known as CRISPR—they can also attach the droplet to DNA in precise locations, targeting genes of interest.

With their ability to control this process using light, the team found a way to grow two droplets stuck to different sequences, merge the two droplets together, and finally shrink the resulting droplet, pulling the genes together as the droplet recedes. The entire process takes about 10 minutes.

Physically repositioning DNA in this way represents a completely new direction for engineering cells to improve health and could lead to new treatments for disease, according to the researchers. For example, they showed they could pull two distant genes toward each other until the genes touch. Established theory predicts this could lead to greater control over gene expression or gene regulation—life’s most fundamental processes.

In order to fit the human genome into each cell’s nucleus, DNA and the chromosomes they contain need to be tightly coiled. However, since DNA is both a carrier of information and a physical molecule, the cell needs to unfurl the tightly coiled parts of the DNA to copy its information and make proteins.

[SubGod22]

This could be huge.

Lab-Grown Blood Stem Cells Could Replace Donor Bone Marrow Transplants

A team of Australian researchers has developed a method to morph personalized stem cells into hematopoietic stem cells, something that would promise risk-free bone marrow transplants

For a myriad of blood and bone marrow-based diseases including leukemia, a bone marrow transplant is the best standard treatment option available.

However, risks abound with the procedure such as mismatched donor cells prompting attacks on the host’s own tissues, leading to inflammation and even death.

Conducted at the Murdoc Children’s Research Institute in Australia (MCRI), the team first performed the common procedure of taking human cells from the hair, skin, and nails, and using a process to reprogram them to morph back into ‘pluripotent’ or ‘multi-power’ stem cells.

Pluripotent cells are richly found in human embryos and infants and have the ability to take the form of any cell in the body. It’s been a decade since Nobel Prize winner Shinya Yamanaka found out how to change any cell in the body back into pluripotent stem cells.

The authors of the new study from MCRI explain that the next step—of turning pluripotent stem cells into hematopoietic stem cells—which can take any form of blood cell, has been difficult to discover, but if it could be standardized, then bone marrow transplants for sensitive individuals like childhood leukemia patients would have much better success rates.

“The ability to take any cell from a patient, reprogram it into a stem cell, and then turn these into specifically matched blood cells for transplantation will have a massive impact on these vulnerable patients’ lives,” says Elizabeth Ng, lead author of the study and Group Leader of the Blood Development Laboratory at MCRI.

“Prior to this study, developing human blood stem cells in the lab that were capable of being transplanted into an animal model of bone marrow failure to make healthy blood cells had not been achievable. We have developed a workflow that has created transplantable blood stem cells that closely mirror those in the human embryo.”

[SubGod22]

Moderate Coffee and Caffeine Consumption Is Associated with Preventing Onset of #1 Killer

Regular coffee or caffeine consumption may offer a protective effect against developing multiple cardiometabolic diseases like coronary heart disease and stroke, the most common killers in human society today.

Detailed in new research published in the Endocrine Society, three cups of coffee per day were associated with those in the study cohort who had a lower profile for a novel risk marker called “new-onset cardiometabolic multimorbidity.”

Cardiometabolic multimorbidity (CM) refers to the coexistence of at least two cardiometabolic diseases, and the prevalence of individuals with CM is becoming an increasing public health concern as populations age around the world, notes the study.

The COVID-19 pandemic showed that the burden of co-morbidities in high-income countries means that swaths of the population are at all times especially vulnerable to novel infections, particularly upper respiratory tract infections.

Coffee and caffeine consumption could play an important protective role in almost all phases of CM development, the researchers from China and Sweden found.

“Consuming three cups of coffee, or 200-300 mg caffeine, per day might help to reduce the risk of developing cardiometabolic multimorbidity in individuals without any cardiometabolic disease,” said the study’s lead author Chaofu Ke, M.D., Ph.D. at Suzhou Medical College of Soochow University, in Suzhou, China.

The study found that compared with non-consumers or consumers of less than 100mg caffeine per day, consumers of a moderate amount of coffee (3 drinks per day) or caffeine (200-300 mg per day) had a 48.1% or 40.7% reduced risk for new-onset CM.

Dr. Ke and his colleagues based their findings on data from the UK Biobank, a large and detailed longitudinal dietary study with over 500,000 participants aged 37-73. The study excluded individuals who had ambiguous information on caffeine intake.

The resulting pool of participants included a total of 172,315 individuals who were free of any cardiometabolic diseases at baseline for the analyses of caffeine, and a corresponding 188,091 individuals for the analyses of coffee and tea consumption.

Drink up!

[Kung Wu]

Since 3 cups per day give me a 48.1% reduced risk, I am going to treat this like Covid vaxxers and take 9 cups to get a 144.3% reduced risk!

[SubGod22]

In World First, Stem Cells Reverse Woman's Type-1 Diabetes

“I can eat sugar now,” said a woman from Tianjing, China, who recently became the first human to have their type-1 diabetes cured through a stem cell procedure.

Using the patient’s own stem cells, the results offer hope of limitless treatment options for type-1 diabetes, where special insulin-producing cells were previously needed from a donor.

Unlike type-2 diabetes which can be developed through poor diet and lifestyle choices, type-1 diabetes develops on its own in certain humans.

Type-1 diabetes is classified as an autoimmune disorder, as the immune system attacks islet cells in the pancreas responsible for producing insulin, a vital metabolic signaling hormone that instructs muscle tissues to absorb excess glucose and other sugars out of the bloodstream.

Treated with exogenous insulin and a mixture of immunosuppressants, the only thing like a cure is an islet-cell transplant, for which there are not anywhere near enough donors to meet the demand.

Instead, Chinese researchers reverse-engineered the patient’s own tissues to produce pluripotent stem cells which they then chemically reprogrammed to form islet cells. Once transplanted, the researchers, whose first subject was a 59-year-old man, observed he was producing his own insulin within 3 months, and after 4 months had a glycemic range that was 98% similar to that of a non-diabetic.

James Shapiro, a transplant surgeon and researcher at the University of Alberta in Edmonton, Canada, told Nature press that the results of the surgery are stunning. “They’ve completely reversed diabetes in the patient, who was requiring substantial amounts of insulin beforehand.”

In this more recent case of the young woman, the cultivated stem cells were injected during the surgery into the abdominal muscles—a new introduction site for this procedure. Previously, donor islets were transplanted into the liver, where they couldn’t be observed.

By contrast, in the abdominal tissue, the islet cells’ activity could be monitored with an MRI machine.

Sounds promising.

[SubGod22]

Gene Therapy at Duke Improves NC Dad's Failing Vision Just in Time for His Baby's Birth

A touching story comes now from North Carolina of a man who saw a reversal in his progressive blindness just in time to see the face of his newborn son.

The new father was born with retinitis pigmentosa (RP) and knew even from a young age that it would mean a total loss of vision.

Tyler Wilfong never had near-sight, and by 23 he had his driver’s license revoked for the loss of peripheral vision. Gradually he had to rely more and more on help from others to get around.

“It was inevitable, but I kept my faith in God and I just had a feeling that one day something would change,” he recalled to CBS 17. “And you know, 30-some years later, here comes this opportunity.”

Many different faulty gene copies can lead to RP, and after applying for candidacy in a medical trial at Duke University, he found that the gene therapy being tested was for the ones at fault for his vision loss.

Sounds like a promising development that has a long way to go to impact a greater number of people with RP.

There's a three minute news clip at the bottom of the article.

[SubGod22]

Single-Dose Gene Therapy is Potentially Life-Changing for Adults with Hemophilia

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial by researchers from the University of Pennsylvania Perelman School of Medicine.

Hemophilia is a genetic disorder that limits the blood’s ability to clot and affects around 30,000 people in the United States, mostly males. Left untreated, it can cause spontaneous bleeding, particularly internal bleeding into the joints, which, over time, can cause painful joint damage and mobility issues.

Hemophilia B is caused by a lack of clotting factor IX. The gene therapy enables the liver to create factor IX, which allows the blood to clot and protects patients from frequent bleeds.

“What we saw from patients in this study was that within a few days of receiving the gene therapy infusion, it took root, and their bodies started making factor IX for the first time in their lives,” said study investigator and lead author Adam Cuker, MD, MS, section chief for Hematology, and clinical director of the Penn Blood Disorders Center and the Penn Comprehensive Hemophilia Program.

“We always want to be careful about using the word ‘cure’ especially until we have longer follow-up data, but for many of these patients, it’s been life changing.”

After at least one year of follow-up, participants in the study had an average 71 percent reduction in bleed rate after receiving the gene therapy, compared to the year prior, when they were treated with prophylactic infusions of factor IX, the standard treatment for the disease.

In fact, more than half of the 45 patients in the study did not have any bleeds after receiving gene therapy.

Based on the results of this study, the FDA approved the gene therapy (fidanacogene elaparvovec) in April 2024. Cuker was the site lead for the clinical trial at Penn Medicine, which was one of the top-enrolling sites for the study. It represents the second form of gene therapy approved to treat hemophilia B. The first such therapy (etranacogene dezaparvovec-drlb) was approved in November 2022, and Penn Medicine is one of several medical centers in the United States where this treatment is available to patients.

Gene therapies have very specific guidelines that determine eligibility and require specialized knowledge to carry out patient screening and selection, education about treatment risks and benefits, and post-therapy monitoring. Penn Medicine offers access to numerous clinical trials for gene therapy and expertise in administering FDA-approved gene therapies.