Top 10 Drug Discovery News Stories of 2017
2017 has been an exciting year for the drug discovery field. This list details the top 10 most-read news stories we published from the field this year.
Parkinson's Risk Halved by Asthma Drug
In August this year, researchers at the Department of Public Health and Primary Care, University of Bergen reported that an existing asthma medication could potentially be repurposed as a treatment for Parkinson’s disease, according to data from their large-scale study. More than 100 million Norwegian patient’s prescriptions were analysed to determine whether there was a link between the treatment of Parkinson’s disease and the prescription of asthma, and blood pressure medication. They found evidence that the use of asthma medication halved the risk of developing Parkinson’s disease. Whereas a specific type of medication for hypertension doubled the risk.
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First Successful Gene Therapy Trial Reported for People with Haemophilia A
Data from an investigational gene therapy trial for patients with haemophilia A, presented in December, indicated that those whom received a single infusion of the drug showed improved levels of FVIII, a blood clotting factor. A functional copy of the FVIII gene was transferred in to the body via a viral vector. All patients eventually began producing FVIII, with FVIII levels adjusting to, and remaining within the normal range. This is the first gene therapy that has demonstrated potential long-lasting effects after just a single infusion. In addition, no patients receiving the therapy showed evidence of an adverse immune response.
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World’s First ‘Molecular Robot’ Capable of Building Molecules
A world-first! Scientists at the University of Manchester have created tiny molecular robots capable of manipulating and building molecules. The scientists can programme the robots to carry out chemical reactions, enabling them to perform basic tasks such as building other molecules. In the future these robots could be used in numerous medical applications, and to assemble molecules and materials in a molecular factory setting. This miniaturized manufacturing approach also reduces material demand meaning its adoption could cut overall costs.
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Novel Drug Targeting Method Could Improve Immune Disease Therapies
A team from the University of Edinburgh, have developed a new therapy delivery system able to target drugs to specific cells within the body. In the future, this could lead to more effective immune disease therapies, with fewer side effects. A drug compound was coupled to a fluorescently tagged carrier molecule, which only becomes active once inside the acidic macrophage cell environment. The researchers observed that the treatment was able to discriminate closely related sub-populations of macrophages, exclusively affecting pro-inflammatory M1 cells and leaving anti-inflammatory ones untouched.
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Immunotherapies Transfer into the Brain
For immunotherapies to be effective as a treatment for brain disease, antibodies need to get past the blood-brain barrier. Now they can, thanks to researchers from Uppsala University. The team have designed a novel antibody. This new design increases the antibody uptake to the brain, by almost 100-fold. Transferrin is one of the select few large molecules able to naturally pass the blood-brain barrier. The researchers were able to manipulate the antibodies using components that bind to the transferrin receptor, ‘tricking’ the receptor to transport the antibody through the barrier to the brain. This approach enabled them to transfer high levels of a modified antibody, that specifically targets a protein involved in Parkinson’s disease, in to the brain of mice.
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Body Clocks Cause Wounds Sustained at Night to Heal More Slowly
A study published in November, revealed that wounds heal approximately 60% faster if the wound is sustained during the day, as opposed to at night. These new findings may have implications for the timing of surgical procedures and presents potential new drug targets that could improve wound healing. The team from the University of Manchester are the first to show how our body clock regulates cellular activity They discovered that actin, a cytoskeletal protein involved in cell migration within fibroblasts, involved in the wound-healing response, is regulated by the circadian rhythm.
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Potential for Anti-Chagas Therapies Using Structure-based Drug Design
Scientists from Tokyo Tech and Nagasaki University have developed potential new anti-Chagas therapies by merging the principles of structure-based drug design, with in vitro methods, and X-ray crystallography. By collectively applying these techniques, they were able to ‘drill down’ the number of potential lead compounds more efficiently, allowing them to take forward only the most promising drug candidates. Through virtual screening they identified a protein required for the survival of a Trypanosoma species, they homed in on this target protein; spermidine synthase (SpdSyn) and were able to identify potential drug candidate inhibitors using a screening search. In the future the team hope to demonstrate the feasibility of applying their approach to the discovery of treatments for other diseases.
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Viagra Could Now be Available Without Prescription
The Medicines and Healthcare products Regulatory Agency (MHRA), issued a press release on November 28, announcing the reclassification of Viagra Connect (sildenafil 50mg) from a prescription only medicine (POM) to a pharmacy medicine. Viagra could now be available without prescription, for men >18 years with erectile dysfunction. Judgement as to the suitability of the medication and the decision to dispense will therefore rest with the pharmacist. The MHRA hope that the change in classification will help raise awareness of erectile dysfunction and encourage more men to seek medical advice and help.
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Multi-Organ-on-a-Chip Could Change Approaches to Drug Development
Researchers have created a novel multi-organ-on-a-chip (MOC) in vitro toxicity model. This 3D system allows scientist to reproduce the complex microenvironments of both the liver and lung. Testing has shown that the lung and liver tissues can be held in a stable state for at least 4 weeks and the researchers were able to confirm the metabolic capacity of the liver tissues. The main aim of the 3D ‘vasculature-on-a-chip’ model is to improve the prediction of the effects of intoxicants and therapeutics on the body, while reducing the need for animal models.
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Potential New Antidepressant Drug
University of Bath scientists have developed a novel drug for depression and anxiety. The compound, BU10119, works via a unique mechanism of action, and is unlike traditional antidepressant drugs; selective serotonin reuptake inhibitors (SSRIs). This could be a promising alternative for patients that do not respond to SSRIs. The compound was investigated in preclinical mouse studies. The team noted that mice administered BU10119 displayed antidepressant-like behaviours. Development of this compound was stimulated by previous research in to the use of buprenorphine and naltrexone, two existing drugs that, in combination, also exhibited potential as an antidepressant.
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