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Category: biotech/medical – Page 825

The viral ALS Ice Bucket Challenge a few years ago raised major funding that resulted in the discovery of new genes connected to the disease. One of those genes is NEK1, in which mutations have been linked to as much as 2% of all ALS cases, making it one of the top-known causes of the disease.

But it wasn’t known how the mutated gene disrupts the function of the motor neuron and causes it to degenerate and die.

Northwestern Medicine scientists have discovered for the first time how this mutated gene leads to ALS (amyotrophic lateral sclerosis).

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We know that humans are an intelligent species. But this biologist breaks down the intelligence of each of our cells — and it will blow your mind.

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Michael Levin, a developmental biologist at Tufts University, challenges conventional notions of intelligence, arguing that it is inherently collective rather than individual.

Levin explains that we are collections of cells, with each cell possessing competencies developed from their evolution from unicellular organisms. This forms a multi-scale competency architecture, where each level, from cells to tissues to organs, is solving problems within their unique spaces.

Levin emphasizes that properly recognizing intelligence, which spans different scales of existence, is vital for understanding life’s complexities. And this perspective suggests a radical shift in understanding ourselves and the world around us, acknowledging the cognitive abilities present at every level of our existence.

Read the full video transcript: https://bigthink.com/the-well/intelligence-can-cells-think.

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The company achieved proof-of-concept with trastuzumab, an anti-HER2 antibody. Preclinical studies show Multilink increases cytotoxicity and improves tumor regression.

In mouse studies, tumor volume after 49 days was approximately half its original size with Trastuzumab Multilink (T-Multilink) with mertansine (DM1), but had grown four-fold when treated with the same compound without the linker. Comparable studies with T-Multilink-auristatin F showed complete tumor regression by about day 25, while treatment without the linker allowed the tumor to approximately triple in size.

“Multilink is a powerful technology to tackle cancers with low antigen expression,” Marx says. Debiopharm is using it to develop “novel, potent, stable, and safe ADCs for various antibodies.”

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Oral tyrosine kinase inhibitors (TKIs) are a class of cancer drugs that can be highly susceptible to issues with solubility in the gastrointestinal tract. Most crystalline TKI drugs have pH-dependent solubility that affects their bioavailability in an oral dosage form. Consequently, natural variations in gastric pH, or variations due to eating or taking antacids, can significantly impact drug absorption and, in turn, therapeutic efficacy.

Alternative formulation techniques such as amorphous solid dispersion (ASD) can still deliver the convenience of a pill while reducing TKIs’ sensitivity to physiological variation. This ensures more consistent—and higher—bioavailability. This whitepaper outlines the challenges associated with pH-dependent solubility for oral TKI drugs and reviews how leveraging ASD formulation can help create more effective, patient-friendly drug products.

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ElevenLabs, a year-old startup that is leveraging the power of machine learning for voice cloning and synthesis, today announced the expansion of its platform with a new text-to-speech model that supports 30 languages.

The expansion marks the platform’s official exit from the beta phase, making it ready to use for enterprises and individuals looking to customize their content for audiences worldwide. It comes more than a month after ElevenLabs’ $19 million series A round that valued the company at nearly $100M.

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A research team headed by investigators at Brigham and Women’s Hospital reported on the results of a study in which they used stem cells from Alzheimer’s disease (AD) patients to identify a potential mechanism by which a gene known as SORL1 may impact the risk for the neurodegenerative disorder. Their work found that loss of normal SORL1 function leads to a reduction in two key proteins, APOE and CLU, which are known to be involved in AD, and which play an essential role in the neurons of healthy individuals. The study findings suggest a potential new strategy for AD treatment, especially for patients not responsive to existing therapies.

“Understanding the subtypes of AD is relatively new in the field of neurology research,” said Tracy Young-Pearse, PhD, of the Ann Romney Center for Neurological Diseases. “This is getting at a precision neurology approach, with which we can better predict which patients may be responsive to Alzheimer’s treatment strategies that attack specific genes or target the problems they cause.” Young-Pearse is corresponding author of the team’s published paper in Cell Reports, which is titled, “ Cell-type-specific regulation of APOE and CLU levels in human neurons by the Alzheimer’s disease risk gene SORL1,” in which they concluded, “Taken together, we demonstrate that AD-relevant SORL1 loss of function results in neuron-specific reduction in APOE and CLU and dysregulated lipid homeostasis.”

AD varies widely in its age of onset, presentation, and severity. Key neurological features of AD, including the accumulation of amyloid-beta (Aβ) plaques in the brain, also vary across individuals. The anti-amyloid therapies, aducanumab and lecanemab, have received FDA accelerated and traditional approval, respectively, but not all patients respond to these drugs, warranting other treatment options.

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Waterborne illness is one of the leading causes of infectious disease outbreaks in refugee and internally displaced persons (IDP) settlements, but a team led by York University has developed a new technique to keep drinking water safe using machine learning, and it could be a game changer. The research is published in the journal PLOS Water.

As drinking water is not piped into homes in most settlements, residents instead collect it from public tap stands using storage containers.

“When water is stored in a container in a dwelling it is at high risk of being exposed to contaminants, so it’s imperative there is enough free residual chlorine to kill any pathogens,” says Lassonde School of Engineering Ph.D. student Michael De Santi, who is part of York’s Dahdaleh Institute for Global Health Research, and who led the research.

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Imagine walking on a treadmill and time is the speed at which the belt moves. As you age, the speed of the belt increases, representing time passing faster and faster as we age. However, what if we could speed up and run faster than the belt? This is widely known as the concept of Longevity Escape Velocity (LEV), which is essentially the idea of outrunning time itself.

Longevity Escape Velocity refers to a future scenario in which medical progress and innovation allows scientists to extend human lifespan at a rate greater than the passage of time. Imagine a world where each passing year not only adds another year to our lives but also enhances our health, vitality, and functional abilities. This concept, popularised by Biomedical gerontologist Dr. Aubrey de Grey, centres around the idea of developing rejuvenation therapies that repair age-related damage at the cellular and molecular levels, with the aim of effectively reversing the aging process.

Organisations such as Dr Aubrey de Grey’s LEV Foundation are playing a huge role in attempting to achieve this extraordinary milestone by striving to speed up the development of life extending therapies and endeavouring to make them accessible to all. It can be argued that they tend to focus on high risk, high reward research that other institutions might overlook due to its experimental nature, however without those that dare to think big, developments in longevity science will not make the strides needed to make a real impact in our future.

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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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