Drug discovery trends to watch: Highlights from the CAS Insights webinar
From microbiome science to AI-powered modeling, the drug discovery field is evolving rapidly in fascinating ways. In our recent CAS Insights webinar, experts from Bayer Consumer Health and CAS explored emerging breakthroughs that are reshaping pharmaceutical research. Our panelists discussed advances in personalized medicine, early diagnostics, and new therapeutic strategies. Their conversation offers us a quick, intriguing glimpse into the future of drug development.
Microbiome science moves into systemic health
Dr. Rami Ammar, Director of Science Innovation for Global R&D, Front-End Innovation at Bayer Consumer Health, kicked off the discussion by highlighting the expanding role of microbiome research in drug discovery. While probiotics have traditionally been associated with digestive health, Ammar explained that the field is now exploring broader applications—including mental health, metabolic disorders, and dermatology. He described the microbiome as a “hidden organ” and noted its genetic dominance:
“One of the most fascinating trends in drug discovery... is the expansion of probiotic use beyond gut health, even broader than probiotics… biotics in general, including probiotics, prebiotics, and postbiotics, as well.”
Dr. Ammar also pointed out that emerging fields like pharmacomicrobiomics and toxicomicrobiomics are:
“… increasingly important in understanding how our gut microbiome or microbiome in general interacts with drugs and toxins, and can lead to personalized and effective treatment strategies.”
He also cited examples of approved probiotic therapies for conditions like C. difficile and antibiotic-associated diarrhea, underscoring the clinical relevance of microbiome-based interventions. He closed with the hopeful observation:
“As we continue to explore the complex relationship between the microbiome and systemic health, I believe we are on the brink of groundbreaking discoveries that could really help in transforming our understanding of medicine and pave the way for innovative therapeutic solutions.”
Early diagnosis for neurodegenerative diseases
Our second panelist, Dr. Angela Zhao, Manager of Scientific Analysis and Insights at CAS, focused on the importance of biomarkers for early neurogenerative disease diagnosis, particularly for Alzheimer’s and Parkinson’s. She explained that while biomarkers are routinely used to monitor physical health—such as cholesterol and glucose levels—there is a gap when it comes to assessing brain health.
“We don’t have a good collection of biomarkers to tell how healthy our brain is. You don’t want to do a spinal cord fluid sampling or PET scan in your regular doctor’s visit. But imagine if we could also have a collection of those biomarkers from blood or urine samples for your brain health. That would be amazing.”
Dr. Zhou also pointed to recent regulatory approvals for Alzheimer’s diagnostics as a sign that the field is moving toward more accessible and preventive approaches.
PROTACs gain traction in targeted therapies
Dr. Gary Gustafson, Senior Customer Success Specialist at CAS, came to our discussion with over 25 years of experience as a Medicinal Chemist in the pharmaceutical industry, having been part of the team that produced the recently FDA-approved drug, Olutasidenib. Dr Gustafson discussed the recent rise of PROTACs (proteolysis-targeting chimeras), which offer a novel approach to eliminating disease-causing proteins. Unlike traditional small molecules that inhibit protein function, PROTACs are designed to degrade the proteins by recruiting the cell’s natural machinery. As Dr. Gustafson said:
“A single PROTAC can actually degrade multiple proteins. They are less hampered by protein mutations or overexpression of the proteins that sometimes can hinder small molecules... “
He pointed out that more than 80 drugs are in clinical development or some kind of clinical evaluation, and that more than one hundred organizations are working in this area.
AI and digital twins: Toward more ethical and translatable research
The panel also explored how artificial intelligence is helping researchers simulate human biology more accurately, reducing reliance on animal models. Dr. Ammar described Bayer’s work with organ-on-chip technologies and physiologically based pharmacokinetic (PBPK) modeling to create digital twins of human systems:
“Reducing the animal use is not only for ethical purposes, but also for transformative and more human translatable results.”
Dr. Zhou added that AI enables more complex biomarker profiling, potentially involving dozens or even hundreds of indicators. Dr. Gustafson emphasized the importance of high-quality data in training AI algorithms and predictive analytics, noting that CAS tools like BioFinder are helping researchers prioritize compounds and explore drug-repurposing opportunities.
Final thoughts: Collaboration, curiosity, and convergence
As the discussion wrapped up, Dr. Zhou encouraged the audience to stay curious and explore adjacent fields to maintain a broad perspective, noting that many great discoveries involve researchers from different fields. It was clear from the wide-ranging, lively discussion that the future of drug discovery will be shaped by the convergence of biology, data science, and engineering—whether it's leveraging the microbiome for systemic health, identifying early biomarkers for neurodegenerative diseases, or designing therapies that eliminate proteins. While tools like AI simulations and organ-on-chip technologies are gaining traction, researchers who work together, with data connected across many parameters, will be better equipped to translate their shared insights into impact.
Watch the full webinar
To hear directly from the experts and explore these trends in more depth, watch the full webinar. The session includes detailed examples, data visualizations, and additional commentary on the future of pharmaceutical research.
Questions from the webinar
We didn’t have time to get to all our audience questions during the hour, so we asked our panelists to provide some thoughts.
Question: Can PROTACS target proteins that were previously considered “undruggable?”
Yes—this is one of the most exciting aspects of PROTAC technology. Unlike traditional small molecules that inhibit protein function by binding to active sites, PROTACs work by tagging proteins for degradation via the ubiquitin–proteasome system. This allows them to target proteins that lack accessible binding pockets or are involved in complex interactions, such as transcription factors and scaffold proteins.
Question: Do you have any insights into radiopharmaceutical conjugates? Which radionuclides do you believe are going to be hot topics going forward?
Radiopharmaceutical conjugates (RDCs) are gaining momentum, especially in oncology, where they combine targeted delivery with potent radiation. Lutetium-177 (^177Lu) remains a leading radionuclide due to its favorable half-life and beta-emission profile, but actinium-225 (^225Ac), an alpha-emitter, is emerging because of its ability to deliver highly localized cytotoxicity. Clinical trials are increasingly exploring ^225Ac-labeled drugs for prostate and neuroendocrine cancers, and major pharma investments suggest alpha therapies will be a key focus going forward. You may be interested in our recent CAS Insights article, “Theranostics: How nuclear medicine is changing precision oncology.”
Question: How can AI and quantum computing help with high-throughput screening of some of the molecular PROTAC targets?
AI is transforming high-throughput screening (HTS) by enabling virtual screening of vast chemical libraries, reducing cost and time while improving hit accuracy. Machine learning models can predict PROTAC activity and optimize compound selection without physical assays. Quantum computing adds another layer by simulating molecular interactions at atomic precision—especially useful for modeling complex PROTAC functions.
Question: CAR-T has been huge for blood cancers, but solid tumors are tougher. What’s the most promising innovation you’ve seen that could finally crack that challenge?
We wrote about recent CAR-T advances last October. Since then, one of the most promising innovations we’ve seen is a new CAR-T cell therapy developed at the Mayo Clinic that targets PD-L1, a protein commonly overexpressed not only on tumor cells but also within the immunosuppressive tumor microenvironment. By designing CAR-T cells to recognize PD-L1, researchers were able to simultaneously attack both the tumor and its protective surroundings, overcoming a major barrier to treatment. In preclinical models of breast cancer, lung cancer, melanoma, and glioblastoma, this approach led to significant tumor reduction.
