Weill Cornell Medicine receives DoD grant to target triple-negative breast cancer

There are no targeted treatments approved for pancreas cancer, and we haven't had any significant progress for a long time. We've only come up with different chemotherapy combinations, and those are only moderately effective. This new treatment is staggeringly better than chemotherapy. Usually, when we think of an improvement in pancreatic cancer survival, we think of 25% improvement. This medication actually doubled survival in patients with advanced disease. We have patients who participated in the trial who are still alive, which is unheard of because the five-year survival rate for pancreatic cancer patients is only 13%-14%. If the drug is approved by the FDA, it will most likely become the new standard of care for advanced pancreatic cancer and could replace chemotherapy as a first-line treatment.

What we're trying to do here at Benevolent is develop systems and processes that allow us to tap into the huge amount of public information and some propriety databases that we have paid to access and through that, generate hypotheses which our scientists can then triage. We've got over ten times the amount of information in our database at present (and it’s growing) than we believe that companies such as Watson have. Bringing that and filtering the signals from the ‘noise’ to our experienced drug discovery chemists and biologists, means, firstly, we can reduce the amount of time we take to triage new hypotheses, and secondly, we can develop more predictive chemical tools to allow us to make better molecules and also to reposition existing molecules for new indication.

More specifically, the study showed that the female patient produced very few cathelicidins, which is a natural toxin found in the immune cells of the lungs needed to fight tuberculosis. In most people infected by tuberculosis, tuberculosis bacteria attack the immune cells of the lungs. The immune cells fight the bacteria by eating them. But the tuberculosis bacterium has developed various evasive mechanisms that reduce the immune cells’ ability to digest and thus to kill the Mtb. “You could say that the tuberculosis bacterium has developed a way to lull the immune cells to sleep. This enables the disease the hide inside the immune cells, making it invisible to other parts of the immune system,” Martin Kongsbak-Wismann explains. This is where vitamin D enters the picture. Because vitamin D is able to counteract the soporific effect of the tuberculosis bacteria by making the immune cells produce more of the cathelicidin toxin. “Cathelicidin is like a microscopic needle that is able to pierce the tuberculosis bacteria. And when it does, it weakens the bacteria’s soporific effect on the immune cells. This restores the immune cells’ ability to kill tuberculosis bacteria,” says Martin Kongsbak-Wismann and adds: “We were amazed by the effect of vitamin D. In immune cells from healthy control subjects, vitamin D improved the cells’ ability to fight Mtb, whereas in the female patient’s immune cells we saw no response to vitamin D. This shows that vitamin D is key to the immune system’s ability to fight Mtb and prevent tuberculosis.” Al-Jaberi, F.A.H., et al. (2022) Reduced vitamin D-induced cathelicidin production and killing of Mycobacterium tuberculosis in macrophages from a patient with a non-functional vitamin D receptor: A case report. Frontiers in Immunology. doi.org/10.3389/fimmu.2022.1038960.

The bulk of vitamins and supplements containing fish oil, biotin, and vitamins A, C, and E can potentially promote nail and hair growth. You can find dietary supplements with higher concentrations of vitamins and minerals that aid skin, hair, and nail health. Fish oil supplements make nails and hair shiny. Fish oil also serves as an agent that can decrease signs of aging. If sun exposure damages the skin, the omega-3 fatty acids in fish oil can help. Biotin, also known as B7, increases hair density and strengthens it. It is found naturally in bananas, eggs, and milk. Vitamins are vital for skin health as well. Vitamin A reduces acne flairs and helps with changes due to aging like wrinkling. A vitamin A deficiency may increase the risk of acne because it becomes difficult for dead skin to slough off hair follicles, blocking pores. Vitamin B lowers the risk of precancerous growths and nonmelanoma skin cancer. Vitamin C is an antioxidant. It leads the fight against free radicals to help stimulate collagen and brighten skin. It also helps to prevent the formation of unneeded melanin that causes uneven skin pigmentation.

There are several areas where AI could add value, but one of the most exciting is helping us translate complex experimental results into actionable insights for drug discovery teams. We see computational and experimental approaches as complementary rather than competing technologies. In vitro assays such as iSLAseizure generate highly relevant biological data, while AI and machine learning can help uncover patterns within that data that might otherwise be difficult to identify. One example is work I recently presented at the BioNow BioAI Symposium, in which we explored the use of structure-activity relationships (SAR) and machine learning models alongside iSLAseizure assay data. Supported by colleagues, including Dr. Louisa Zolkiewski and Dr. Kim Rockley, ApconiX has developed an in-house seizure liability dataset that we analyzed in collaboration with MedChemica. Using machine learning and chemical informatics approaches, we identified chemical substructures that show statistically significant relationships with seizure-related outcomes. The aim is not simply to predict risk, but to understand which molecular features may contribute to that risk and provide medicinal chemists with practical guidance they can apply much earlier in the discovery process. Looking ahead, I think this is where AI can have a particularly powerful impact. Rather than acting as a standalone prediction tool, it can work alongside human-relevant assays such as iSLAseizure to connect molecular structure, experimental biology, and safety outcomes. That combination has the potential to improve decision-making earlier in the discovery process, reduce late-stage safety surprises, and ultimately support the development of safer medicines for patients.