Experimental approaches to treat challenging diseases: mathematical model simulating Alzheimer's progression, CRISPR gene editing 'shredding' tumours; Potential therapeutic advances in Parkinson's Disease and Multiple Sclerosis; Revolutionizing drug development with cyclic proteins and peptides; Mathematical modeling for personalized Alzheimer's treatments; Using CRISPR to target recurrent glioblastoma and hyper-mutated tumors.
USP30 inhibition could be a potential disease-modifying therapy for Parkinson's disease.
Stem cell therapy shows promising results in treating multiple sclerosis, preventing further brain damage.
CRISPR gene editing holds potential for targeting and destroying glioblastoma cells.
Deep dives
Experimental drug shows potential as a therapy for Parkinson's disease
An experimental drug called MTX325 has shown potential as a disease modifying therapy for Parkinson's disease. By inhibiting the enzyme USP30, the drug promotes mitophagy, the removal of dysfunctional mitochondria. This is important because Parkinson's disease is associated with mitochondrial dysfunction. Inhibition of USP30 can improve mitophagy, reduce dysfunctional mitochondria, and protect dopaminergic neurons, which are crucial for dopamine production. These findings suggest that USP30 inhibition could be a potential disease-modifying therapy for Parkinson's disease.
Stem cell therapy shows promise for multiple sclerosis
A stem cell therapy for multiple sclerosis (MS) has shown promising results in an early stage clinical trial. The therapy, developed by researchers at the University of Colorado, the University of Cambridge, and the University of Milano Bikocha, prevents further damage to the brain caused by the body's immune system attacking and damaging myelin. The treatment was well tolerated and showed no increase in disability or worsening of symptoms in the patients. Additionally, the therapy's dose of injected stem cells correlated with a reduction in brain volume reduction over time. While further clinical trials are needed, this study is an encouraging step towards a new way of treating MS.
Researchers develop a new method for creating stable proteins for drug development
Scientists at the University of Bath have developed a method for joining the head and tail of proteins, making them more stable and easier to get into cells. This technique could aid in the development of new pharmaceuticals in a simpler, greener, and less expensive way. The researchers used an enzyme called OAAEP1 from a tropical plant and transferred it into bacterial cells, allowing for mass production of proteins while joining together the ends. This method improves heat and chemical stability of proteins and peptides, making them more robust and suitable for drug discovery. The researchers are excited about its potential applications in various industries, including pharmaceuticals, food, and biotechnology.
Mathematical model predicts Alzheimer's disease progression
Researchers from Penn State University and Duke University Medical Center have created a mathematical model that accurately predicts Alzheimer's disease progression in individual patients. The model uses clinical biomarkers such as fluid markers and MRI brain images to map disease progression and provide personalized treatment recommendations. The model can also be used by pharmaceutical companies to test multiple drug therapies and patient scenarios, speeding up the drug development process. The researchers anticipate that the model will be ready for clinical use within five years and could also be applied to other inflammatory diseases.
CRISPR gene editing shows promise for treating glioblastoma
Scientists at Gladstone Institutes have used CRISPR gene editing to target and destroy glioblastoma cells. Referred to as 'cancer shredding,' the technique involves using CRISPR to zero in on recurring tumor cells and eliminate them. The researchers successfully destroyed tumor cells while sparing healthy cells in an early-stage study conducted on cell lines from a glioblastoma patient. This approach could have potential applications in treating other highly mutated cancers. The researchers see CRISPR as a gateway to a new therapeutic approach that avoids the possibility of tumor cell escape.
Conclusion
This week's round-up of industry news highlights significant progress in various areas of drug discovery and therapy development. Promising findings include an experimental drug showing potential for Parkinson's disease, a stem cell therapy showing promise for multiple sclerosis, a new method for creating stable proteins for drug development, a mathematical model for predicting Alzheimer's disease progression, and the use of CRISPR gene editing to target glioblastoma cells. These advancements bring hope for the development of effective treatments for these debilitating diseases.
The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates.
The key drug discovery news this week focuses on experimental approaches to the treatment of some of the most challenging diseases, including a mathematical model that can simulate Alzheimer’s progression and using CRISPR gene editing to ‘shred’ tumours.
