Novel therapy shows promise in improving corneal edema treatment

Cataracts are one of the most common ailments of an aging population, manifesting in the fifth or sixth decade of life. This condition, which leads to blurred or cloudy vision, is caused by proteins that break down in the lens of our eyes, forming an increasingly opaque mass. Although cataracts can cause blindness if untreated, they can be treated with a simple surgery to remove the aging lens. In the U.S. alone, more than 4.5 million people undergo the procedure each year with great success.

For a small population of those patients, however, surgery replaces one problem with another. The oxidative stress from the surgical procedure damages sensitive endothelial cells that line the back surface of the cornea. Even under normal conditions, these cells are slow to heal. But in people with pre-existing corneal disease or advanced cataracts, the insult to the cornea from surgery is larger than usual, leading to corneal edema that can defocus light coming into the eye. Th…

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Pfizer drops one weight loss drug, bets on another

Pfizer has made a strategic decision in the battle against obesity and diabetes. The pharmaceutical giant has chosen to discontinue its GLP-1 RA candidate, lotiglipron, to focus resources on the development of another – danuglipron. This Pfizer weight loss drug is now the lead candidate in late-stage trials for the treatment of obesity and diabetes. The decision comes after the discovery of elevated liver enzyme levels in some early trial patients for lotiglipron. However, similar concerns have not arisen with danuglipron.

The shift comes as the company aims to tap full receptor agonism for potential robust efficacy, capitalizing on its small molecule design expertise. With more than 1,400 patients already enrolled, the clinical trial results could potentially expand the treatment options for adults with obesity and Type 2 diabetes.

Already, GLP drugs semaglutide and tirzapatide are beginning to reshape the way we treat obesity.

Novo Nordisk’s sema…

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A deep dive into AWS’s strategy with generative AI and ML in life sciences

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The AI market is witnessing meteoric growth, with projections hinting at a potentially staggering increase over the next decade. Against this backdrop of rapid AI evolution, we recently spoke with Tehsin Syed, general manager of AWS Health. Syed shared that Amazon Web Services (AWS) is seeing growing interest from Big Pharma firms. “Nine of the top 10 pharma companies in the world have a large majority of the workloads running on AWS,” Syed said. The AWS cloud hosts more than 100,000 customers across industries.

AWS aims to provide a broad and deep range of AI services and takes an end-to-end approach to AI that includes infrastructure, software, hardware and services.In an increasingly competitive AI landscape, AWS recently launched a $100 million initiative, the AWS Generative AI Innovation Center, to accelerate enterprise generative AI adoption. As Syed put it, “AWS has a pe…

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FDA publishes draft guidance for psychedelic drug development

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In a sign of shifting attitudes, the FDA has released draft guidance to facilitate the development of psychedelic drugs. Historically, psychedelics have a long history of use in certain cultures, such as the indigenous tribes of the Amazon Basin and Native American communities, for millennia as sacraments.These substances were used not only recreationally but also experimentally. One example is MK-Ultra, a covert U.S. government research program that explored using LSD for mind control. The Manson Family, notorious for its cult-like crimes in the late 1960s, also contributed to psychedelics’ notoriety.

This complex history, along with social and political responses to the countercultural revolution of the 1960s, contributed to a backlash against these substances. Classic psychedelics such as LSD, psilocybin, mescaline, and DMT experienced a swift fall from mainstream favor after the 196…

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CGT demands vision, partnership and patient-centricity to transform healthcare

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As personalized medicine continues to progress, cell and gene therapy (CGT) development is poised to enable tremendous medical breakthroughs. Unlike traditional treatments which merely manage symptoms, CGT could treat the root biological cause of many diseases: the faulty genes.

“The cell and gene therapy space is truly exciting. What this ultimately means for patients and caregivers, and our ability to play an important part within this paradigm, is exhilarating,” said Dr. Panteli Theocharous, the global vice president and cell and gene therapy strategy lead at PPD.

With more than 20 years of experience in the pharma and biotech industries, Theocharous provides guidance on developing CGTs. He accentuates the importance of partnerships, data-driven approaches and patient-centricity in overcoming challenges.

Yet the path forward is filled with obstacles. In 2021, cell and gene …

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How 11 Big Pharma companies are using AI

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Pharma companies are ramping up their reliance on AI in drug discovery, clinical trials and manufacturing. Bloomberg recently frame the technology as providing a $50 billion opportunity to the sector. GlobalData noted that the pandemic played a role in stoking interest in AI. But given the hype often surrounding the technology, it can be difficult to gauge actual impacts and outcomes of such initiatives, which largely  remain undisclosed.

The pharma industry faces mounting pressures to discover and develop new drugs faster and at lower costs. AI has emerged as a promising tool to help overcome long-standing challenges like high failure rates, lengthy development timelines, and resource inefficiency.

While pharma companies are making big bets on AI, the actual impacts and outcomes of many initiatives remain largely behind the scenes. Still, there are some examples that provide…

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Guselkumab could offer hope for psoriatic arthritis patients resistant to TNF inhibitors

Janssen continues to strengthen the case that its interleukin-23 inhibitor TREMFYA (guselkumab) offers promise to many patients with psoriatic arthritis (PsA).

The company’s phase 3b COSMOS clinical trial involving 189 patients with active PsA and an inadequate response to one to two previous tumor necrosis factor (TNF) inhibitors, guselkumab showed sustained improvements in measures of minimal disease activity (MDA) over 48 weeks of treatment. TNF inhibitors are a class of medications that target and suppress the overproduction of the inflammatory protein TNF, making them a common treatment option for autoimmune diseases such as PsA.

The IL-23 inhibitor guselkumab provides a treatment alternative, recording sustained improvements in measures of minimal disease activity (MDA) over 48 weeks of treatment. In particular, 60 to 80% of patients achieved MDA across various measures.

Beyond guselkumab, another approved IL-23 inhibitor for the treatment of psori…

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8 considerations to boost clinical trial productivity with AI while dodging hallucination hurdles

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The development of new drugs is undeniably a data-intensive endeavor. Despite impressive advances in AI over the past years, researchers often continue to grapple with crushing data volumes. This hurdle is particularly apparent in clinical trials, where crucial data is often stored in machine-unfriendly formats such as PDFs, PowerPoint or HTML or other formats.

This article explores strategies to harness AI for data management in clinical trials while avoiding potential pitfalls such as data integrity issues and large language model hallucinations, which can lead to unreliable or distorted outputs.

1. Understand the complexity of clinical trial data

The complexity of clinical trial data can be difficult for someone outside the field to appreciate, according to Jeff Elton, CEO of Concert AI. “There can be 60 to 70 different levels of inclusion and exclusion criteria,̶…

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PsychoGenics’ SmartCube prompts a reevaluation of CNS drug discovery

SmartCube integrates behavioral neurobiology, robotics and computer vision to process and analyze large temporal and vector-based datasets. This platform uses proprietary bioinformatics and probabilistic causal inference algorithms to explore compounds’ potential to treat psychiatric disorders. [Image courtesy of PsychoGenics]

In an era of rapid AI progress, the quest to pioneer the first AI-developed drug candidates has led to an increasing number of these drug candidates entering clinical trials. One contender is ulotaront, an antipsychotic drug, that fared well in a phase 3 schizophrenia study published in NEJM in 2020. The trace amine-associated receptor 1 agonist has entered phase 2/3 studies to test its potential in generalized anxiety disorder and as an adjunctive therapy for major depressive disorder treatment.

“Not only is this the first drug discovered using machine learning that’s th…

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A current perspective on machine learning’s role in advancing clinical trials diversity

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The year 2020 was a watershed moment for many reasons, but notably, it cast a light on the pervasive health and social inequities that have long marred the U.S. The COVID-19 pandemic hit diverse populations disproportionately hard, as Deloitte and others have noted. Additionally, the tragic deaths of George Floyd, Breonna Taylor and others provoked an uproar over systemic racism that permeates society, including healthcare. This period of societal upheaval has also underscored the necessity of novel approaches involving techniques like the use of machine learning in clinical trials, to ensure that diverse populations are represented.

Such disparities in healthcare were further highlighted when Moderna, soon to become a critical player in the vaccine race, faced a glaring revelation in late 2020. Only 24% of participants in their phase 3 study were from communities of color, despit…

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A timeline of data integrity scandals and other controversies in biomedical research

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While scientific progress hinges on data integrity, plagiarism, data fabrication and image manipulation and other biomedical research scandals are ongoing concerns. An article in Science recently made waves by revealing startling conclusions from the research of the German neuropsychologist Bernhard Sabel, who developed a fake-paper detector. Publishing his findings in a preprint, Sabel concluded that there were likely high proportions of falsified or plagiarized papers in neuroscience and medicine in 2020 (34% and 24% respectively). That figure is substantially higher than the findings of a 2009 study in PLOS One reporting that 2% of scientists acknowledged having fabricated, falsified or modified data or results at least one time in their career. The findings seem to indicate a troubling rise in data integrity scandals in medical research.

To arrive at the conclusion tha…

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How to plan decentralized clinical trials

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Decentralized clinical trials offer a wide range of benefits for medical device makers and the patients they serve.

With that in mind, the FDA has released draft guidance for decentralized clinical trials to advance medical product development and research.

Decentralized clinical trials use locations other than traditional clinical trial sites for some or all of the trial-related activities. That means patients who might have previously needed to travel to research sites — their doctor’s office, for example, or a university hospital — can instead participate in trials at home or closer to home.

This allows a broader, more diverse representation of the population to take part in experimental medicine, helping to reduce gender, racial or socioeconomic inequality in medical care.

“The FDA has long considered the benefits of decentralized clinical trials. Advan…

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