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

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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.

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

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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 KIBRA, named because it is found in the kidney and the brain. 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 KIBRA 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.

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

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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.

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Test That Can Spot 18 Early-Stage Cancer Signals Shows 84% Sensitivity in First Human Trial

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An experimental cancer test already being studied in humans shows that by examining blood proteins instead of tumor DNA, it may be possible to detect up to 18 early-stage cancers with exceptional accuracy.

Cancer tests don’t often use the same methods of detection, and having one or two unified testing options would likely save thousands of lives.

A US biotech firm called Novelna recently presented their findings of a trial of 440 humans with a total of 18 different cancers. Blood plasma samples were taken from each patient, along with 44 healthy blood donors.

By analyzing trace proteins in the blood, the Novelna team were able to achieve a high “sensitivity,” or the detection rate of early-stage tumors, and a high “specificity” or the control for false-positives. Furthermore, the proteins controlled for in the test are sex-specific.

At stage I (the earliest cancer stage) and at the specificity of 99%, the panels were able to identify 93% of cancers among males and 84% of cancers among females.

“This finding is the foundation for a multi-cancer screening test for the early detection of 18 solid tumors that cover all major human organs of origin for such cancers at the earliest stage of their development with high accuracy,” the authors wrote in the journal BMJ Oncology. “These findings pave the way for a cost-effective, highly accurate, multi-cancer screening test that can be implemented on a population-wide scale.”

The team acknowledged the small trial size and admitted that larger trials would be needed to confirm the accuracy already established, but they also highlighted that almost all of the proteins for almost all of the cancers were present in the blood samples at very low levels, indicating the importance of such tests for catching tumors before they form.

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Baby That Had Spinal Surgery While in the Womb Can Now Walk and Run

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Piper Kelly, a healthy 5 year old captured on the fter undergoing spinal surgery while in the womb of her mother. November 12 2023. See SWNS story SWNAspine. A little girl whose parents feared she might be wheelchair-bound for life can now walk and run - thanks to £9k womb surgery. Georgia Axford and Tyler Kelly, then just 19 and 21, were told their unborn baby had spina bifida after the 20-week scan.The condition causes weakness or paralysis in the lower limbs - and the parents were told it was likely their child wouldn't walk.The couple decided to travel to Germany for treatment, which saw the unborn baby operated on in the womb - at a cost of £9,000.

A little girl whose parents were told she would never walk is now running around like other 5-year-olds after receiving emergency spinal surgery while still in the womb.

This miracle of modern medicine was available to first-time parents Georgia Axford and Tyler Kelly, then just 19 and 21, who were told their unborn baby had spina bifida after an ultrasound at 20 weeks.

The condition causes weakness or paralysis in the lower limbs, and the parents were told it was likely their child wouldn’t walk.

The couple decided to travel to Germany for treatment, which saw the unborn baby operated on in the womb at a cost of just £9,000, or around $11,500.

Piper-Kohl Kelly was then born healthy in July 2018 and named after surgeon Dr. Thomas Kohl. Now five, she can walk and run and recently took part in her school’s sports day.

“Seeing her run on the tracks was amazing. I never thought she’d be able to do something like that,” said Georgia. “Sometimes it doesn’t feel real. I think back to what we were told, and they were really negative about Piper’s diagnosis.”

Spina bifida can leave sufferers dependent on supports or crutches, and in severe cases, they can be wheelchair-bound. Doctors were confident that this would be the case for Piper-Kohl.

Georgia hails from South Yorkshire, in the Northeast of England. For all the stress and negativity of the diagnosis, and even though Piper was induced at 32 weeks and sat in the neonatal intensive care unit for 52 days, they ended up getting the chance to live a normal life from the same moment as other babies.

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New 10 Minute Treatment Restores Sense of Smell and Taste in Patients with COVID Parosmia

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Using an image-guided, minimally invasive procedure, scientists may be able to cure the loss of smell, known as parosmia, occasionally found in people who were infected with COVID-19.

While most COVID patients did recover their sense of smell over time, some patients however continue to have these symptoms for months, or even years, after infection.

Lead author professor Adam Zoga said that post-COVID parosmia is increasingly being recognized, and that patients can develop distaste for foods or drinks they used to enjoy.

“Parosmia has previously been reported as a rare disorder occurring after brain trauma, brain surgery, stroke, viral syndromes, and with some head and neck tumors,” said Zoga. “We were not entirely confident that the procedure would work for parosmia.”

The treatment involves injecting anesthetic directly into the stellate ganglion on one side of the neck to stimulate the autonomic nervous system, which is accurately achieved with CT guidance.

The minimally invasive procedure takes less than 10 minutes, and no sedation is necessary. It’s been used to treat several other conditions including cluster headaches, phantom limb pain, Raynaud’s and Meniere’s syndromes, angina, and cardiac arrhythmia.

For the study, 54 patients were referred by an ear, nose, and throat specialist after at least six months of post-COVID parosmia that was resistant to pharmaceutical and topical therapies.

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New Medicines May Lie Under the Sea–This Device Sniffs Them Out

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Marine organisms are constantly releasing invisible molecules under the ocean’s surface, and much like a million anonymous plants in the Amazon, scientists believe that some of these compounds could be the medicines of the future.

A study team at the Mediterranean Institute of Marine and Continental Biodiversity and Ecology has developed a proof-of-concept device that sniffs, in a way, seawater, trapping dissolved compounds for analysis.

They have shown that it could “easily” concentrate molecules that are present in underwater caves and that it holds promise for drug discovery in fragile ecosystems, such as coral reefs.

“A drop of seawater is like a spoonful of dilute soup: it’s a complex broth of dissolved molecules from ocean-dwelling organisms,” said study co-author Doctor Thierry Pérez.

To identify what’s in the mixture, scientists need to be able to observe concentrations of those molecules in isolation.

Dr. Pérez and his colleagues wanted to develop an underwater instrument that captures and enriches dissolved compounds produced by sponges and other marine organisms without harming their ecosystem.

They created a waterproof device that could be easily handled by an underwater diver and that could pump seawater through disks, which have a similar feel and thickness as make-up remover pads.

The team tested the instrument—called the In Situ Marine moleculE Logger, or “I-SMEL” – in 65-feet-deep Mediterranean sea caves that contained a variety of massive sponges.

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After Taking Vitamin B2 Baby Becomes Solitary Case of Recovery from Rare Genetic Disease

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Vitamin B2, or riboflavin, is a key compound in energy metabolism, cellular respiration, antibody production, and in the case of a 1-year-old baby from California, perhaps the reason he was able to recover from Mitchell Syndrome.

If that disease sounds unfamiliar to you, that’s because it’s one of the rarest diseases known to medicine. There have been just 20 recorded cases of this genetic disease, and it was only named back in 2019.

Augustine was born a perfectly healthy boy on May 27th, 2022, but at three-months he had to be hospitalized with hypoglycemia, and his health afterwards began to deteriorate.

He began to lose his hearing and he also had difficulty moving. At six months, the tot stopped eating altogether. At first doctors said it was just down to teethin, but an MRI scan showed deterioration of the protective covering of nerve fibres in his brain known as demyelination.

Augustine’s mother Kristen and his father Moses, “begged” for further genetic testing, which revealed a genetic mutation of a gene called ACOX1, leading to the diagnoses of Mitchell syndrome aged seven months.

The newly diagnosed neurological illness is caused by a rare genetic mutation which attacks the nerves which control hearing, movement and vision.

“At the time, the hospital were only aware of three patients with the disorder, who had all passed away, that was incredibly hard to hear,” said Kristen. “It wasn’t until weeks later that I started asking more questions.”

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17-Year-old Wins $150,000 in Science Talent Search for Remarkable Way to Diagnose Pediatric Heart Disease

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In the oldest and most prestigious young adult science competition in the nation, 17-year-old Ellen Xu used a kind of AI to design the first diagnosis test for a rare disease that struck her sister years ago.

With a personal story driving her on, she managed an 85% rate of positive diagnoses with only a smartphone image, winning her $150,000 grand for a third-place finish.

Kawasaki disease has no existing test method, and relies on a physician’s years of training, ability to do research, and a bit of luck.

Symptoms tend to be fever-like and therefore generalized across many different conditions. Eventually if undiagnosed, children can develop long-term heart complications, such as the kind that Ellen’s sister was thankfully spared from due to quick diagnosis.

Xu decided to see if there were a way to design a diagnostic test using deep learning for her Regeneron Science Talent Search medicine and health project. Organized since 1942, every year 1,900 kids contribute adventures.

She designed what is known as a convolutional neural network, which is a form of deep-learning algorithm that mimics how our eyes work, and programmed it to analyze smartphone images for potential Kawasaki disease.

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Sisters With Rare Disorder Make Dramatic Leaps With New Therapy After Doctors Said They’d Never Walk or Talk

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What parent could imagine anything worse than hearing their newborn has a genetic mutation that impedes neuromotor development that’s so rare, it affects just 300 infants worldwide?

Yet even for something as rare as a CASK Disorder, the miracles of modern medicine can lend a healing hand—as they did for Anna and Bella Burkhart.

Bella had a noticeably small head that announced what tests would later confirm: Bella was born with a rare genetic disorder. Mother Emily Burkhart immediately suspected a genetic problem because she’d seen this before—in Bella’s older sister, Anna.

A decade earlier, doctors predicted Anna would never walk, talk, or have a fulfilling life. Today, she rides the bus to school, interacts with her friends on TikTok, has plans to dye her hair, and even made her middle school cheerleading squad.

When Anna, and Bella for that matter, were born, few signs of the coming disorder were present, but both soon began to miss developmental milestones such as the inability to grasp, roll around, and crawl. She didn’t walk until she was nearly 3 years old.

Her parents sought answers for Anna’s delays. After years of testing and hospitalizations, Anna was finally diagnosed with a rare mutation of a gene common in many species, called CASK. Doctors knew little about the condition, said Burkhart.

“Basically, she was just going to struggle, and she was probably going to be wheelchair-bound and non-verbal,” she said. “I just wasn’t going to accept that.”

Soon, the family was visiting Roanoke, Virginia so Anna could receive intensive therapy through an experimental research protocol. While customary occupational and physical therapy is delivered for only one hour a week, therapists at the Neuromotor Research Clinic at the Fralin Biomedical Research Institute at Virginia Tech, work with children 3 to 6 hours a day, 5 days a week, for up to 4 weeks.

Turning a corner

The research clinic’s team had worked with many children with disabilities that had affected their brain development and function, including those with cerebral palsy. But the team never worked with a child with Anna’s diagnosis before and wasn’t sure what to expect.

“What Anna needed was different than previous kids we’d worked with,” said Dory Wallace, a senior occupational therapist. “It was about helping her learn how to pay better attention, how to use her words, and how to interact and engage with the people in her environment. Once we met her, we completely changed what we thought we were going to do to meet her needs.”

Anna responded right away.

“I love seeing that light switch go on when a child realizes she’s learned something new,” Wallace said. Two weeks later, the change was already dramatic.

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