They search the medicine cabinet for new tools against rare diseases

2023-03-17

Taking a promising substance the full distance from laboratory to treatment requires large resources. Consequently, rare diseases are seldom first in line when new ideas are tried. Now the Faculty of Pharmacy is joining several European consortia on the outlook for new therapies among well-known drugs.

(Image removed) Alina Meyer and Ola Spjuth, The Faculty of Pharmacy

Finding new therapeutic areas for existing drugs, drug repurposing, is rapidly increasing in medical importance. A recent study shows in an analysis of a number of drugs that these can be used, on average, against eleven times more diseases than intended, and with the aim to streamline the path to healthcare, the European Union investing 25 million euros in REMEDI4ALL, an infrastructure with the capacity to pilot promising candidates to clinical trial.

(Image removed) Ola Spjuth, Professor of Pharmaceutical Bioinformatics

“Drug repurposing is providing fantastic opportunities, and with REMEDI4ALL we are uniting a selection of universities and companies that with joint expertise can provide the support needed to take a project to the next level. This is a consortium our team enters with a very positive feeling,” says Ola Spjuth, Professor of Pharmaceutical Bioinformatics, whose robotic laboratory for cell profiling is one of 24 European environments recruited to the consortium.

A common denominator of the diseases that are in focus of drug repurposing is that healthcare still lacks curative treatment. One example is ADPKD, Kidney cysts, a hereditary disease in which fluid-filled cysts form in the kidneys. Recently, the University of Leiden presented a line of drug molecules that are assessed to have potential effect also against ADPKD. With funding from Horizon 2020, the research environments in DRUGtrain will now try out the most suitable candidate to proceed with.

(Image removed) Alina Meyer, PhD student, DRUGtrain

“During the pandemic, this scientific field experienced a public breakthrough, and now the prospect of combining its close-to-patient perspective and the opportunity to make valuable contributions to healthcare has really caught my interest. In parallel with our operational activities, DRUGtrain is also an investment in strengthening the overall conditions for drug repurposing and the development of strategies of compounds, which adds another dimension to our work, says Alina Meyer, PhD student at the Department of Pharmacy.”

The possibilities of repurposing a drug for other treatments than the primarily intended is defined by its ability to target more than one protein, but even the most promising prospect face a complex journey. Among the scientific thresholds is that new therapeutic areas often require both changed dosage and additional toxic studies. Once past these, legal trials await in the form of patent aspects prior to publication and introduction to the market.

“This is a challenging terrain, and until now the skills required to force it have been spread across Europe. Now we have five years to establish REMEDI4ALL as a platform that research groups with interesting drug candidates can turn to for support to evaluate their projects, plan and execute necessary experiments, as well as get assistance with regulatory and legal issues. We have just left the starting blocks, but the fact that this consortium gathers several frontline environments in our field makes me convinced that we have what it takes to reach our high set goals, says Ola Spjuth.

FACTS REMEDi4ALL & DRUGtrain

Repurposing of Medicines 4 All (REMEDi4ALL) is a consortium that gather 24 companies and industries from all over Europe which, with EU funding, will establish a platform for Drug repurposing.
The Swedish work within REMEDi4ALL has its hub at SciLifeLab under the leadership of Ola Spjuth and Jordi Carreras Puigvert, Uppsala University, and Annika Jenmalm-Jensen and Päivi Östling, KI.
DRUGtrain is a European consortium and a Marie Curie-Skldowska doctoral network, receiving principal funding from Horizon 2020 (EU's research and innovation funding programme)
In the DRUGtrain consortium, nine PhD students, among them Alina Meyer at Uppsala University, are analyzing the potential of selected drug molecules for future treatment of Autosomal dominant polycystic kidney disease (ADPKD).

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CONTACT

(Image removed) Ola Spjuth, Professor
Faculty of Pharmacy
ola.spjuth@farmbio.uu.se

(Image removed) Alina Meyer, PhD Student
Faculty of Pharmacy
Alina.Meyer@farmaci.uu.se

Text: Magnus Alsne, photo: Mikael Wallerstedt a o