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Category: biotech/medical – Page 25

A Naked Mole Rat Gene Was Put Into Mice, And It Made Them Live Longer

Naked mole rats are well known for living far longer lives than any rodent ought to have. It’s just one of their amazing talents for surviving in a challenging, even hostile underground environment.

A fascinating new study led by researchers from the University of Rochester in the US has shown a single gene could play a significant role in their longevity, one that could be transferred into other mammals to give their own life spans a nudge.

The gene – a version of what’s known as hyaluranon synthase 2 – produces an abundance of high-molecular-mass hyaluronic acid (HMM-HA), a compound already thought to mediate the risk of cancer in naked mole rats (Heterocephalus glaber).

Targeting the ‘undruggable’: New molecular degraders offer hope for aggressive breast cancer

In the battle against aggressive breast cancer, a once-elusive target is now within reach—thanks to a breakthrough from a team from the Faculty of Medicine at Hebrew University. Dr. Raphael Benhamou and M.Sc. student Liann Kassabri have developed innovative druglike molecules capable of degrading HuR, a key RNA-binding protein that stabilizes oncogenes and fuels cancer progression.

HuR (also known as ELAVL1) has long been labeled “undruggable” due to its structural flexibility and lack of a conventional active site. Overexpressed in many cancer types—particularly breast cancer—HuR fortifies by protecting mRNAs that drive and survival.

“We knew that simply blocking HuR wasn’t enough,” says Dr. Benhamou. “We needed to eliminate it altogether.” Strikingly, this elimination led to a three to four orders of magnitude improvement in anticancer properties compared to traditional HuR-binding molecules that do not induce degradation.

Skin swabs may help detect Parkinson’s years before symptoms appear

Remote, scalable cognitive behavioral therapy–based chronic pain programs are effective for treating individuals with high-impact chronic pain.

Importance Cognitive behavioral therapy (CBT) skills training interventions are recommended first-line nonpharmacologic treatment for chronic pain, yet they are not widely accessible.

Objective To examine effectiveness of remote, scalable CBT-based chronic pain (CBT-CP) treatments (telehealth and self-completed online) for individuals with high-impact chronic pain, compared with usual care.

Design, Setting, and Participants This comparative effectiveness, 3-group, phase 3 randomized clinical trial enrolled 2,331 eligible patients with high-impact chronic musculoskeletal pain from 4 geographically diverse health care systems in the US from January 2021 through February 2023. Follow-up concluded in April 2024.

Feature: Cough medicine shows promise in treating Parkinson’s disease

An over-the-counter cough medicine may be the key to slowing the progression of Parkinson’s disease.

Called Ambroxol, the drug is commonly used to break up phlegm, but early studies have shown it can also prevent the build-up of misfolded clumps of protein in the brain, known as Lewy bodies – a hallmark of Parkinson’s and other types of dementia.

Robarts scientist Dr. Stephen Pasternak is leading a phase 2 clinical trial to further study Ambroxol’s potential as a disease-modifying drug.

“Current treatments for Parkinson’s target the symptoms of the disease, such as movement, but don’t change the long-term progression of pathology in the brain,” he explained. “We hope Ambroxol will be a disease-changing drug.”

Dr. Stephen Pasternak is leading a phase 2 clinical trial to study Ambroxol, an over-the-counter cough medicine, with the goal of slowing or stopping the progression of Parkinson’s Disease Dementia.

Mum says new diabetes drug would be ‘kind of magic’ if it works

A world-first human trial of a drug designed to treat the underlying cause of type 1 diabetes has begun in Australia.

Five patients with the condition have already been dosed as part of the trial, including mum-of-two Caecilie Wickstroem Giralde, who was diagnosed last year.

University of Queensland researcher Ranjeny Thomas has spent more than a quarter of a century developing the drug, designed to rebalance the body’s immune response in people with type 1 diabetes, which affects more than 130,000 Australians.

The immune system starts to recognise insulin-producing cells in the pancreas as something it needs to attack and destroy in people with type 1 diabetes — one of dozens of auto-immune diseases in which the body starts to attack itself.

Professor Thomas, who is based at UQ’s Frazer Institute, said the experimental drug — dubbed ASITI-201 — was designed to retrain the immune system so it no longer attacks the insulin-producing pancreatic cells, known as beta cells.

The drug, given as an injection under the skin, combines fragments of a protein found in the beta cells of people with type 1 diabetes and vitamin D to calm the immune response.

Continue reading “Mum says new diabetes drug would be ‘kind of magic’ if it works” | >

Will implantable brain-computer interfaces soon benefit people with motor impairments?

A review published in Advanced Science highlights the evolution of research related to implantable brain-computer interfaces (iBCIs), which decode brain signals that are then translated into commands for external devices to potentially benefit individuals with impairments such as loss of limb function or speech.

A comprehensive systematic review identified 112 studies, nearly half of which have been published since 2020. Eighty iBCI participants were identified, mostly participating in studies concentrated in the United States, but with growing numbers of studies from Europe, China, and Australia.

The analysis revealed that iBCI technologies are being used to control devices such as robotic prosthetic limbs and consumer .

FDA grants orphan drug designation to UAB-developed gene therapy for ALS

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS) developed at the Universitat Autònoma de Barcelona and licensed to the U.S. company Klotho Neurosciences, Inc.

The drug uses a viral vector of the AAV (adeno-associated virus) type that expresses the secreted isoform of Klotho (s-KL) protein, with neuroregenerating, antioxidant and anti-inflammatory properties. In order to reach the neuromuscular junctions affected by the ALS disease, the vector acts under the control of a DNA sequence that regulates the expression of the protein specifically in the muscle (a muscle-specific promoter), so that therapeutic activity is directed towards the neuromuscular junctions. This innovative approach has shown very promising results in the most widely used mouse model for the preclinical study of ALS, delaying the onset of the disease, preserving neuromuscular function and extending survival.

The technological development was led by UAB researchers, with the involvement of the CIBER, ICREA and Vall d’Hebron Research Institute, co-owners of the intellectual property relating to the use of the Klotho protein and licensed to Klotho Neurosciences –a start-up company based on knowledge generated at UAB and listed on Nasdaq in 2023 (NASDAQ: KLTO)-. The technology was developed by the research groups of Assumpció Bosch and Miquel Chillón, both from the UAB Department of Biochemistry and Molecular Biology and the UAB Institut de Neurociències (INc-UAB). The research project also included the collaboration of the group led by Professor Xavier Navarro, researcher at the Institut de Neurociències and the UAB Department of Cellular Biology, Physiology and Immunology, and expert in neuroregeneration and motor neuron diseases.

“The orphan drug designation for the therapy we have developed acknowledges the relevance of treatments targeting muscle and neuromuscular junction as a strategy for ALS”, says Assumpció Bosch, principal investigator of the study. “To date, we have been able to demonstrate efficacy in a leading animal model for this pathology. We are now testing it in other ALS models to confirm that this therapeutic solution can be applied to the widest possible number of patients”, adds Sergi Verdés, postdoctoral researcher on the research team.

Receiving the orphan drug designation by the FDA underscores the potential of the treatment for the rare and severely disabling disease ALS, which affects around 65,000 people in Europe and for which there is no effective treatment. This recognition offers advantages such as seven years of exclusivity for the drug in the U.S. market, fee waivers and tax incentives for clinical trials.

This Common Blood Pressure Drug Extends Lifespan, Slows Aging in Animals

The hypertension drug rilmenidine has been shown to slow down aging in worms, an effect that in humans could hypothetically help us live longer and keep us healthier in our latter years.

Previous research has shown rilmenidine mimics the effects of caloric restriction on a cellular level. Reducing available energy while maintaining nutrition within the body has been shown to extend lifespans in several animal models.

Whether this translates to human biology, or is a potential risk to our health, is a topic of ongoing debate. Finding ways to achieve the same benefits without the costs of extreme calorie cutting could lead to new ways to improve health in old age.

Stem cell transplant without toxic preparation successfully treats genetic disease

An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a Phase I clinical trial has shown.

While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem cell transplant extremely risky, they expect it may also work for patients with other genetic diseases that require stem cell transplants.

“We were able to treat these really fragile patients with a new, innovative regimen that allowed us to reduce the toxicity of the stem cell transplant protocol,” said the study’s co-senior author, Agnieszka Czechowicz, MD, Ph.D., assistant professor of pediatrics.

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