BioWire Weekly - 004

Monday Morning Biotech News

DALL·E 3 depiction of an antibody on top of a circuit board.

Happy Monday! Let’s be relentless this week.

I have a few interesting pieces of Biotech News to share with you all. These news briefs include applications of AI in medicine and drug development, a newly approved Parkinson’s treatment, the positive impacts of a ketogenic diet on mental illness, and a new antiviral CRISPR tool. Let me know what you think in the comments below.

AI Surpasses Pathologists in Pinpointing Origins of Metastatic Cancers

Schematic depicting the development of the AI tool by authors (Tian et al, 2024).

Scientists have developed an AI tool that surpasses human pathologists in identifying the origins of metastatic cancers (Tian et al, 2024). Often, secondary tumors are only detected after spreading to distant organs. This AI system, trained on 30,000 cell images from known tumor sources, can predict the primary cancer source with high accuracy, providing the top three likely origins in 99% of cases. The application of this technology could significantly reduce the need for invasive tests while enhancing diagnosis and treatment strategies for late-stage cancers. The study, Published in Nature Medicine, also showed that patients treated for the type of cancer predicted by the AI had higher survival rates, highlighting the tool's potential in clinical settings.

AI-Created Drug for Lung Disease Enters Phase II Trials in US and China

Drug design through generative AI (Baker Lab).

The world's first AI-created drug for treating Idiopathic Pulmonary Fibrosis (IPF) has progressed to Phase II clinical trials in both the U.S. and China. Developed by Insilico Medicine using generative AI, this drug aims to provide a new therapeutic option for a disease that impacts up to 20 individuals per 100,000 and incurs significant medical costs annually. The Phase II trials, involving 60 patients in each country, are focused on assessing the drug's efficacy and safety, building on favorable Phase I results that demonstrated good tolerance and safety profiles (Ren et al, 2024). This marks a significant advancement in AI-driven drug discovery, which I will be dedicating a full deep dive to in the near future.

New Parkinson’s treatment Tavapadon is a huge win for AbbVie following Cerevel Acquisition

Tavapadon is a promising new treatment for Parkinson's disease and has recently garnered significant attention following successful phase 3 trials. For readers unfamiliar with Parkinson’s, it is a neurological disorder that causes uncontrolled and involuntary movements. This once-daily oral medication acts as a dopamine D1/D5 receptor partial agonist, effectively reducing these involuntary movements. Those treated with Tavapadon reported fewer symptoms and better motor control for extended periods compared to their counterparts receiving a placebo. This breakthrough comes as part of AbbVie's $8.7 billion acquisition of Cerevel Therapeutics, a deal originally centered on Emraclidine, a mid-phase schizophrenia drug. While Emraclidine was the initial focus, Tavapadon's trial successes highlight its potential as a valuable addition to AbbVie's neuroscience portfolio.

Ketogenic diet improves severe mental illness

A pilot study by Stanford Medicine researchers shows that a ketogenic diet helped patients with schizophrenia or bipolar disorder. This study, published in Psychiatry Research focuses on those experiencing side effects from antipsychotic medications (Sethi et al, 2024). The keto diet (typically low in carbohydrates and high in fats) markedly improved metabolic health, eliminating metabolic syndrome in all participants. It also led to significant psychiatric improvements. Participants reported higher energy levels, better sleep quality, improved mood, and increased life satisfaction. These results suggest that dietary changes can effectively complement traditional medication, offering a new therapeutic avenue. Lead researcher Shebani Sethi, MD, calls this approach 'metabolic psychiatry.' It aims to use diet to affect brain metabolism and overall health, paving the way for future large-scale studies.

Innovative antiviral defense with new CRISPR tool

The illustration shows how Cas13d protein and crRNA are expressed and located in mammalian cells, with Cas13d-NES featuring a single NES motif (variant v5). RNA FISH staining highlights the nuclear presence of crRNA in cells using a nuclear-localized Cas13d-NLS version (3xNLS, variant v1), and a schematic depicts the nucleocytoplasmic shuttling mechanism of Cas13d, designed to transport crRNAs to the cytoplasm. (Gruber et al, 2024)

Researchers have developed a new version of the CRISPR/Cas13 system, named Cas13d-NCS, that enhances its effectiveness against RNA viruses by enabling the transport of CRISPR RNA (crRNA) from the nucleus to the cytoplasm (Gruber et al, 2024). This advancement allows Cas13d-NCS to effectively target and degrade RNA viruses like SARS-CoV-2, which replicate in the cytoplasm, overcoming previous limitations where Cas13d was only active in the nucleus. By fusing Cas13d with both nuclear localization and export sequences, this modified system can shuttle crRNAs to the cytoplasm where they can be effective. This development significantly improves the system's antiviral capabilities, offering potential as a highly precise antiviral therapy.

References:

Gruber, C., Krautner, L., Bergant, V., Grass, V., Ma, Z., Rheinemann, L., Krus, A., Reinhardt, F., Mazneykova, L., Rocha-Hasler, M. and Truong, D.J.J., 2024. Engineered, nucleocytoplasmic shuttling Cas13d enables highly efficient cytosolic RNA targeting. Cell Discovery, 10(1), p.42.

Ren, F., Aliper, A., Chen, J., Zhao, H., Rao, S., Kuppe, C., Ozerov, I.V., Zhang, M., Witte, K., Kruse, C. and Aladinskiy, V., 2024. A small-molecule TNIK inhibitor targets fibrosis in preclinical and clinical models. Nature Biotechnology, pp.1-13.

Sethi, S., Wakeham, D., Ketter, T., Hooshmand, F., Bjornstad, J., Richards, B., Westman, E., Krauss, R.M. and Saslow, L., 2024. Ketogenic Diet Intervention on Metabolic and Psychiatric Health in Bipolar and Schizophrenia: A Pilot Trial. Psychiatry research, 335, p.115866.

Tian, F., Liu, D., Wei, N., Fu, Q., Sun, L., Liu, W., Sui, X., Tian, K., Nemeth, G., Feng, J. and Xu, J., 2024. Prediction of tumor origin in cancers of unknown primary origin with cytology-based deep learning. Nature Medicine, pp.1-11.

https://www.fiercebiotech.com/biotech/abbvie-bound-cerevel-reports-phase-3-parkinsons-victory-early-win-87b-deal