Researchers explore drug repurposing strategy for Parkinson's disease

This is a win for the field. Until now, we have been focused on immune therapies that might make tumors more vulnerable to the body's immune system, and on finding new chemotherapy combinations that kill cancer cells. This new treatment has given us a new focus, and I think it will spur a lot of scientific discovery over the next few years. There have only been a handful of KRAS researchers and their relevance to therapy was always questioned. That is about to change. The most important next step for the field is to better understand the biology of cancer. We know that many pancreatic tumors will eventually become resistant to daraxonrasib, and we need to understand how this happens. We also need to identify additional genetic pathways and treatments that can target them. That's how we will turn pancreas cancer from a deadly, deadly cancer into something we can manage-and one day, even cure.

Picking the right target is absolutely fundamental. I mentioned that over half of failures of new medicines in development are due to us picking the wrong target. Even if we just doubled our success rate at picking the right target, it would have a huge impact on the development of new medicines for people. One of the reasons, and why I think artificial intelligence could be really important in helping us do that, is that, quite often, there may be some data buried in the literature that tells us a target is really good. Equally valuable is the fact that there may be something buried in the literature that tells you a target is not good. It's much harder to publish negative data than positive data. That target invalidation can be just as valuable as a target validation. It's really about being able to tap into all that knowledge and look up all the facts. That would mean that our systems and other people's AI systems can really be much more effective at picking the right target.

The tools we are building and applying at ApconiX are currently internal-facing, supporting our scientists and collaborators in delivering thoroughly researched, evidence-based, and authoritative toxicology information in a timely manner. Even though I did not directly interact with ApconiX while I was at Redx, my experience in biopharma strongly shapes how I think about usability. At ApconiX, we constantly consider how others will interpret and use the data and insights we generate. For example, our early SAR work around seizure liability has identified preliminary relationships between chemical substructural patterns and seizure risk. Although this work is in its infancy, these are the kinds of insights that discovery chemists could interpret directly and apply from day one to help mitigate risk. For target safety assessments, the ApconiX data science team have collated a large data sets including our Acuity expression atlas for expression data, enabling our data scientists and bioinformaticians to generate highly informative, data-driven insights and visualizations. By leveraging AI tools, such as LLM workflows and agents, we can efficiently support consistent and thorough analyses, making safety information more accessible and actionable.

Daily, if you eat a balanced diet that includes healthy foods, you technically should not need a vitamin supplement. A healthy diet incorporates lean proteins, healthy fats, grains, fruits, and vegetables. But if you do not have a balanced diet, you may be considering hair, skin, and nail vitamins. Some people prefer supplements, sometimes choosing a multivitamin that can supply all of your essential minerals and vitamins. But taking too much of vitamins or unnecessary supplements is wasteful because the body gets rid of excess vitamins and minerals or, worse, it can be dangerous. The following is a guide to choosing which vitamins you may want to consider supplementing and when. Deficiencies in the nutrients that keep the skin, hair, and nails healthy can cause changes over time. For example, not enough intake of vitamins A and E, along with not enough biotin, can cause scaly and rough skin patches, eczema, and hair loss. If there is a deficiency, vitamins will help. However, if there is no deficiency, there is no clear evidence that supplements will make a difference. No research studies concluded that supplements treat or prevent age-related, natural hair damage or loss or lead to healthier skin. Two studies in the early 1990s did suggest that a biotin supplement may cause the hair to become stronger and strengthen weak nails. However, the studies were small and not reproduced.

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.