Medical Breakthroughs, Research, and News

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Deaf Children Are First Humans to Have Hearing Pathway Restored in Dramatic Demonstration of New Gene Therapy

In China, a true medical breakthrough has been achieved in a human trial that had as many as 10 children born deaf have their hearing restored through a genetic therapy method.

MIT hailed it as China’s first domestic gene therapy breakthrough, as well as “the most dramatic restoration of a lost sense yet achieved.”

Much like other gene therapy treatments, children like Li Xincheng were injected with a reprogrammed virus that carried replacement DNA into the part of her body the scientists hoped to alter—in this case the location of the inner ear canal that detects vibrations and sends that information to the brain.

In less than a month, her mother, Qin Lixue, said she was hearing out of her treated ear for the first time in her 5 years of life, and repeating various rhymes and songs back to Lixue as she sang them with her hand over her mouth to prevent lip-reading.

It bears repeating that this, according to MIT Technological Review, has never happened before.

“We were careful, and a little bit nervous, because it was the first in the world,” says Yilai Shu, a surgeon and scientist at Fudan University in Shanghai, and part of the team who treated 5 children.

“Before the treatment, if you put them in a movie theater with the loudest sound, they wouldn’t hear it,” says Zheng-Yi Chen, an associate professor at Mass Eye and Ear, a Harvard-affiliated hospital in Boston, who helped design and plan the study. “Now they can hear close to normal speech, and one can hear a whisper.”

Of Shu’s five children, 4 recovered hearing, and one did not, which the team hypothesizes could be because the child already had a developed immune response to the virus that they used.

This new gene therapy is not a cure-all, as it was designed to correct a defect in a gene that produces a protein called otoferlin that is necessary to build the special hairs that vibrate to different frequencies in the inner ear and relay that information to the brain.

What a potential blessing to many, and who knows what other avenues open up to help others.

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This sounds like something that may be a life changer in many ways.

New 'Super Melanin' Cream Heals Skin From Sun Exposure And Even Chemical Burns

A new ‘super melanin’ skin cream developed by scientists at Northwestern University shows the ability to continuously heal sun damage and chemical burns.

The synthetic, biomimetic melanin cream demonstrated the potential to heal damage occurring throughout the day when skin is exposed to sunlight or environmental toxins.

It mimicks the natural melanin in human skin, and can be applied topically to injured skin, where it accelerates wound healing. These effects occur both in the skin itself and systemically in the body.

A study published this week in Nature Regenerative Medicine showed that when applied in a cream, the synthetic melanin can protect skin from sun exposure and heal skin injured by sun damage or chemical burns. The technology works by scavenging free radicals, which are produced by injured skin such as a sunburn. Left unchecked, free radical activity damages cells and ultimately may result in skin aging and skin cancer.

Melanin in humans and animals provides pigmentation to the skin, eyes and hair. The substance protects your cells from sun damage with increased pigmentation whenever the sun is ‘tanning’. That same pigment in your skin also naturally scavenges free radicals in response to damaging environmental pollution from industry smokestacks and car exhaust fumes.

“People don’t think of their everyday life as an injury to their skin,” said co-corresponding author Dr. Kurt Lu, who teaches dermatology and practices at Northwestern University School of Medicine. “If you walk barefaced every day in the sun, you suffer a low-grade, constant bombardment of ultraviolet light. This is worsened during peak mid-day hours and the summer season.”

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New Pacemaker Developed that Uses the Heartbeat to Recharge its Battery

By generating electrical energy from the heartbeat, a new pacemaker developed by scientists in Seattle was able to partially recharge itself.

Although the beat only generated 10% of the energy needed for the next heartbeat, the researchers hope that their breakthrough will become the standard, since changing a battery in a wireless pacemaker requires heart surgery, convincing most people to just implant a second one.

The new device is much smaller than a traditional pacemaker due to its wireless nature, measuring about one-third the size of a AAA battery and residing entirely in the heart’s right ventricle.

“We hope to prolong battery life further and expand access of this product to younger patients, who would hopefully require fewer implants over their lifetime,” said Dr. Babak Nazer of the University of Washington in Seattle, who led the paper demonstrating his team’s new invention.

“When we can improve upon our 10 percent harvesting efficiency, we hope to partner with one of the major pacemaker companies to incorporate our design and housing into an existing leadless pacemaker,” he added.

By converting mechanical energy into electrical energy, the experimental wireless pacemaker housing is able to partially recharge its battery—the same technology used in some experimental electricity-generating roads.

And if they can improve the recharge rate like they hope to, it could be an absolute game changer in the future.

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Eliminating peanut allergies?

Special Toothpaste Could End Severe Peanut Reactions for People With Allergies

For years, doctors have treated allergies by introducing small amounts of the dangerous allergen over a period of time, which desensitizes the patient to keep them safe.

Now, a special toothpaste may soon be saving people with peanut allergies from having severe reactions.

The proposed product would contain tiny amounts of the nut to build patients’ immunity over time.

Every participant in the small trial tolerated the highest dose of the peanut toothpaste without any moderate or severe systemic reactions. Some experienced a little itch in the mouth but it was a mild and transient reaction, similar to that which occurs at an injection sites when doctors give shots.

Speaking at the annual scientific meeting of the American College of Allergy, Asthma and Immunology (ACAAI) in California, allergy expert Dr. William Berger described the process called Oral Mucosal Immunotherapy (OMIT).

“OMIT uses a specially formulated toothpaste to deliver allergenic peanut proteins to areas of the oral cavity.

“OMIT as a delivery mechanism for peanut protein has great potential for food allergy desensitization,” he said.

Due to its targeted delivery and simple administration, it can desensitize patients to peanuts without requiring dozens of visits to a clinic over a period of years.

My nephew has developed such an allergy over the last couple of years. Poor kid misses his peanut butter and a number of other products that get cross-contaminated during production.

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First Advanced Parkinson's Patient to Walk Again with New Spinal Implant, 'It's Incredible'

A Frenchman who received a new spinal implant has regained significant motor functions including the ability to walk unaided for miles after losing all such faculties to advanced-stage Parkinson’s Disease.

In the latter stages of the moto-neuron disorder, patients lose the ability to correctly use their muscles, and at some points movement can shut off entirely and they can crumple to the floor.

63-year-old Marc from Bordeaux, France was diagnosed with the condition 20 years ago, and it got so bad that there had to be someone holding his arms at all times in case his walking gait just froze.

He was the subject of the experimental spinal implant technology developed through a collaboration between the Swiss Federal Institute of Technology in Lausanne and the Lausanne University Hospital that has also restored some mobility to a man with a spinal cord injury that left him paralyzed.

When you see images of people with spinal cord injuries walking again with the help of implants, there’s normally a computer attached to their back, or they’re in a laboratory setting.

But watching Marc walk with his new implants, it’s like the real thing. The implants still needs another 5 years of work, and the study published on Marc’s experience is seen as a major ‘stepping stone.’

“I practically could not walk anymore without falling frequently, several times a day. In some situations, such as entering a lift, I’d trample on the spot, as though I was frozen there, you might say,” Marc told the press. “Right now, I’m not even afraid of the stairs anymore. Every Sunday I go to the lake, and I walk around 6 kilometers [3.7 miles]. It’s incredible.”

There's video at the bottom of the article.

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This one will be interesting to follow as the potential is huge should it work out like one would hope.

World's First Whole-Eye Transplant Gives Arkansas Man New Hope

In a 21-hour procedure, 140 surgeons and attendants completed the first whole eyeball transplant on a military veteran who had suffered a near-fatal electrocution.

Replacing the better part of his entire face, and connecting a donor eyeball to the optic nerves, the surgery was declared a success, and though it isn’t clear whether the patient will be able to see out of his eye again, it has meant an enormous step forward for transplantation science and incredible peace of mind for his family.

Aaron James was working on a powerline in Mississippi when he accidentally touched a live wire and received a 7,200-volt shock in June 2021. His wife was called at their home in Arkansas and was told only that her husband wouldn’t be dead by the time she reached the hospital where he was admitted.

James remembers only getting up to go to work, and then waking up days later in a Dallas hospital burn ward where he was flown. He felt okay, he told CNN, so he knew he was going to be okay, but he was missing his face, and taking a selfie allowed him to see inside his own skull.

Dr. Eduardo Rodriguez, director of the Face Transplant Program at NYU Langone Health was briefed about James’ case, and after hearing that he was going to require his left eye amputated, told the surgeons to leave as much of the optic nerve intact for a possible transplant.

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I saw this story on one of the morning news stations. The guy is incredible. He's not going to see but his selflessness may allow someone else to eventually see.

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This could end up being insanely valuable.

An IV Bag for Disasters That Doesn't Need Gravity Scoops Up 2023 James Dyson Award for International Effort

A team of South Korean student inventors has been awarded the 2023 James Dyson Award for an air pressure-controlled IV bag that will allow rescuers in disaster situations much more flexibility when administering live-saving fluids and drugs.

Their inspiration was the Turkish-Syrian earthquakes in February 2023, which resulted in over 55,000 casualties, with a further 100,000 injured.

Throughout the evacuation process, medics had to move through harsh environments while holding up several IV packs in their hands for their patients.

The team from Hongik University in Seoul designed The Golden Capsule, a non-powered and hands-free IV device that uses elastic forces and air pressure differences rather than gravity. This means that medics in disaster zones do not have to hold up IV packs while transporting patients, and electricity is not required to control the infusion rate.

“The team has identified the limitations of existing IV injection methods, which rely on gravity and electricity, in disaster zones. Their Golden Capsule offers a much more practical, hands-free solution, using a pressurized bladder, which can be positioned anywhere, such as strapped to the patient’s side,” said Sir James Dyson, Founder and Chief Engineer at Dyson.

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This could end up being insanely valuable.

An IV Bag for Disasters That Doesn't Need Gravity Scoops Up 2023 James Dyson Award for International Effort

Here is the Dyson Award page with more details: https://www.jamesdysonaward.org/2023...olden-capsule/

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Immunotherapy for Hard to Treat Cancer Just Granted FDA Fast Track During Promising Clinical Trial

An Australian pharma company has been granted fast-track status by the United States FDA for cancer treatment trials of bile duct cancer.

This rare form of cancer has been found to be resistant to both conventional and immunotherapy treatments, but the firm’s new version of an existing treatment, developed as part of a collaboration with the US-based City of Hope, has shown exceptional promise.

In May of last year, City of Hope, a world-renowned cancer research and treatment organization in Los Angeles, dosed their first patient with a low amount of their oncolytic virus (i.e. a virus that infects oncolytic cells) in a phase 1 safety trial.

“Our previous research demonstrated that oncolytic viruses can stimulate the immune system to respond to and kill cancer, as well as stimulate the immune system to be more responsive to other immunotherapies, including checkpoint inhibitors,” said Daneng Li, M.D., principal investigator and assistant professor of City of Hope’s Department of Medical Oncology & Therapeutics Research.

The virus was engineered by Professor Yuman Fong, and it’s now been licensed by City of Hope to the Australian company Imugene for use in its Vaxinia treatment, known technically as “CF33-hNIS” for bile duct cancer, with CF33 being the virus, and hNIS being an additional chemical that allows researchers to image and track the virus in order to apply radiochemo treatment with extreme precision.

Bile duct cancer occurs inside and outside the liver and is rare in the US, with just 8,000 people on average receiving a diagnosis per year.

Fong’s original oncolytic virus has been extensively studied and played a role in wiping out smallpox around the world. It has a short and well-understood lifecycle that doesn’t involve the use of the human genome. It can both deliver tumor-destroying agents and reduce their size itself.

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Potential relief for MS

Exciting Clinical Trials of New Stem Cell Injection Treatment Shows Promise for Halting Multiple Sclerosis

A collaborative study involving experts in Europe and the US found the treatment of stem cells appears to protect the brains of MS patients from further damage.

In the first-ever clinical trials in humans, the researchers found patients injected with the stem cells exhibited no increase in disability or worsening of symptoms.

The promising study, published in the journal Cell Stem Cell, is hoped to lead to further clinical trials that could provide treatment for progressive MS.

More than two million people live with MS across the globe and, whilst some treatments currently available can reduce the severity and frequency of relapses, two-thirds of patients still transition into a debilitating secondary progressive phase of the disease within 25 to 30 years of diagnosis.

An autoimmune disorder like Lupus, ALS, and Crohn’s, MS is characterized by the body’s immune system attacking and damaging myelin—the protective sheath of tissue around nerve fibers, disrupting messages sent around the brain and spinal cord.

An immune cell called a microglial can attack the central nervous system in progressive forms of MS, causing chronic inflammation and damage to nerve cells.

Recent scientific advances involving the transplantation of stem cells have raised expectations that therapies could be developed to help ameliorate this damage.

Previous experiments in mice from the Cambridge University unit of the new study team have shown that skin cells reprogrammed to be brain stem cells and transplanted into the nervous system can help to reduce inflammation, and may even be able to help repair damage caused by MS.

The research team behind the latest study, incorporating experts from the UK, US, Switzerland, and Italy, completed a world-first early-stage clinical trial in which neural stem cells were injected into the brains of 15 patients with secondary MS recruited from two Italian hospitals.

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Electrical Brain Stimulation Improves Mental Processes in Alzheimer's Patients

Researchers found that twice-daily current stimulation sessions of around 20 minutes improved the memory and overall cognitive performance of elderly people living with Alzheimer’s disease.

The innovative technology, known as transcranial direct current stimulation (tDCS), could also be used as a treatment to improve brain plasticity in Alzheimer’s sufferers through the formation of new neural networks, collections of neurons that fire together.

The tDCS technology is delivered via a device with two electrodes placed over specific areas of a person’s head, which transmits a constant, low-intensity electrical current.

It’s already beginning to be used across many areas of medicine, including for the treatment of depression, and a similar method was even able to zap paralyzed limbs into motion again.

The researchers from China recruited 140 patients with mild to moderate Alzheimer’s and randomly allocated whether they would receive the two daily sessions of the active low-intensity current, or a sham version of the tDCS in a control or placebo group.

Each participant received their respective treatment five days a week, over a maximum period of six weeks.

All participants were over the age of 65 and had had the disease for more than six months, with all also having scored below 26 on the Mini-Mental State Exam (MMSE), in which a score of less than 24 is seen as abnormal and indicative of cognitive impairment.

The currents were applied to the prefrontal cortex—the region of the brain involved in higher-order activities such as planning, decision-making, working memory, moderating social behaviors, and controlling aspects of speech and language.

The researchers then used a different scale called the Alzheimer’s Disease Assessment Scale-Cognitive Test, which focuses on language and memory, to evaluate cognitive performance at the start of the trial, after two weeks, and again after six weeks.

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KSN - Wichita is testing ground for potentially breakthrough medical device

Biotech company Procyrion™ has developed a new device called Aortix™, and Wichita will be the first official testing ground for it. The first US randomized patient for the clinical trial was enrolled by Wichita cardiologist Bassem Chehab, MD, and cardiothoracic surgeon Brett Grizzell, MD, who heads St. Francis’ Structural Heart team.

The device is used to treat Acute Decompensated Heart Failure. Like in heart failure, the heart can no longer effectively pump blood. However, the medications that treat the symptoms, like edema, are not as effective and often require hospitalization to bring them under control.

“Today, there are no effective treatment options for patients with ADHF who remain congested despite being treated with high-dose intravenous diuretic therapy,” says Dr. Chehab in a news release from Ascension. “These patients have very poor outcomes in terms of death and rehospitalization, and therefore, new therapy options are critically needed.”

According to the manufacturer, Aortix™ works similarly to a ramjet engine. Doctors use a catheter to install the device in the aorta chamber.

As the heart beats and pulls blood into the aorta, some of that blood is pulled into the Aortix™ device. It speeds up and comes out the other end at a higher pressure into the bottom of the aorta. That change should allow the heart to perform better, which should also decrease fluid congestion in the body by allowing the kidneys to expel more fluid.

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Vaccine Targeting Triple-Negative Breast Cancer Shows Good Response in First Clinical Trial of Patients

A drug that targets the deadliest form of breast cancer has recently been found to elicit no side effects, and triggered an immune response in 75% of patients.

Conducted at the Cleveland Clinic with funding from the Pentagon, the vaccine was administered to 16 women in three separate doses. The form of the vaccine that went through trials is meant to stop the return of this aggressive form of cancer in those who have already been treated.

Further research will tool it to attack tumors in women who have yet to undergo treatment.

mRNA vaccines for cancer tumors are really where this technology comes into its own. Using a piece of the tumor to train immune cells like bloodhounds to search them out is far more effective than using it to train a single component of a constantly mutating virus, as was done to try and combat COVID-19.

It could be available in five years, estimates ABC’s medical correspondent Dr. Jennifer Ashton, who didn’t participate in the research.

According to Anixa Biosciences, the firm behind this vaccine, the drug will target a lactation protein, known as α-lactalbumin, that is present in the majority of triple-negative breast cancer patients.

“The data from our Phase 1 trial to date has exceeded our expectations, and we are pleased with our progress. This vaccine is designed to direct the immune system to destroy TNBC cancer cells through a mechanism that has never previously been utilized for cancer vaccine development,” stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences.

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FDA Approves Cure for Sickle Cell Disease, the First Treatment to Use Gene-Editing Tool CRISPR

The first FDA-approved treatment using CRISPR may see hundreds of African Americans cured of sickle cell disease.

Making headlines without end for years, but only approved for trials, a CRISPR product is now finally on the shelves, so to speak, and this iteration of the treatment is called Casgevy and manufactured by Vertex Pharmaceuticals.

CRISPR edits the genetic code of the body’s own stem cells to treat conditions that arise from defective or mutated genes. In the case of sickle cell, Homo sapiens evolved this genetic change to protect themselves against the malaria parasite of the African continent.

However the disease that sometimes results from the sickle cell gene, which affects over 100,000 Americans, can cause debilitating pain and shortened lifespan.

“It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product,” said Dr. Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex.

Until now, the only previous treatment for sickle cell disease was a bone marrow transplant from a donor which carried large risks such as rejection of the immune system. It’s also difficult to find a matching donor.