JOBS workflow. JOBS consists of two steps. The preliminary step makes use of bulk-eQTL and sc-eQTL abstract statistics, (i.e., impact measurement and normal deviation), to estimate cell-type weights utilizing the non-negative least-square technique. Subsequently, leveraging these estimated cell-type weights, JOBS collectively fashions bulk- and sc-eQTLs and refines sc-eQTL impact estimates. The colours symbolize completely different cell varieties and the majority tissue. The darkness of the colour represents the energy of the eQTL results. Credit score: Cell Genomics (2025). DOI: 10.1016/j.xgen.2025.100820
People pay some huge cash for prescription medicines. In 2023, spending on prescribed drugs in the USA topped $722 billion, a pattern that is anticipated to proceed. But, almost 30% battle to pay for his or her prescriptions and greater than half of adults are apprehensive concerning the affordability of their household’s medicines, in line with polling by KFF.
One option to doubtlessly decrease the price of prescribed drugs? Discover new makes use of for current remedy, which is the concept behind drug repurposing.
“New drugs are expensive to develop. If we can identify new uses for off-patent drugs, we can accelerate the drug discovery process, saving both time and money,” stated Dajiang Liu, distinguished professor, vice chair for analysis and director of synthetic intelligence and biomedical informatics on the Penn State School of Drugs.
Liu and his colleagues just lately developed a drug repurposing pipeline to determine new potential therapeutics for autoimmune illnesses, similar to lupus and rheumatoid arthritis. They’ve printed their findings within the journal Cell Genomics. Within the following Q&A, Liu discusses drug repurposing, the way it works and its advantages.
What’s drug repurposing?
Drug repurposing is after we discover a new use for a drugs that is already available on the market. Present medication are authorised for a selected illness. Generally, these medication may additionally be efficient in treating different circumstances. Ailments can share the identical mechanism, or the pathway for a way a illness develops, although they could result in distinct circumstances.
What are the advantages of drug repurposing?
One main benefit of drug repurposing is that the medication are already available on the market and are protected. Usually, it takes a whole lot of effort and time to determine a drug’s security, however right here, we are able to transfer on to figuring out a drug’s effectiveness.
Value is one other subject, particularly if the repurposed drug is off patent, since generic medication are less expensive. If we are able to discover various makes use of for these medication, it may assist decrease the price of prescribed drugs.
What’s an instance of a drug that has been repurposed?
One well-known instance is hydroxychloroquine. It was initially developed as an anti-malarial drug, however individuals discovered proof, from experiments and statement, that it is fairly efficient at slowing down the development of lupus, an autoimmune illness, and in addition controlling flare-ups of the illness.
In my earlier analysis, we discovered that dextromethorphan, a cough drugs, was efficient for smoking cessation therapy.
What is the course of for screening potential candidates for drug repurposing?
Beforehand, drug repurposing was an advert hoc course of the place somebody, by luck, would possibly observe {that a} drug may very well be efficient for one more illness. There are tens and even tons of of 1000’s of authorised medication within the U.S. Meals and Drug Administration database, and somebody must dedicate their profession to screening them manually to determine one repurposed drug for one illness.
Now, we do not have to depend on the genius of a selected scientist or on luck. With an information science method, we are able to stream and automate the method to search for patterns in very complicated information utilizing machine studying, accelerating scientific discovery.
You and your collaborators have developed a novel method to discovering new makes use of for current medication. Are you able to inform me extra about it?
First, we determine therapeutics based mostly on how they affect the mechanisms concerned in a illness. In our work, we take a look at gene expression or how cells translate the knowledge contained inside a gene. In some illnesses, gene expression is elevated, whereas in different illnesses, it is decreased. If the gene expression is what’s inflicting the illness, by reversing it—lowering gene expression in instances the place it is elevated or elevating it in instances the place it is suppressed—we are able to doubtlessly reverse the illness as nicely.
We search for medication with these sorts of patterns of reversing gene expression. These are experiments with cells that occur within the lab.
Then, we validate the mechanism of the candidate drug. In our new mannequin, we do that by analyzing information from digital well being data. For instance, we take a look at the well being data of people that have taken the drug we’re focused on and take a look at biomarkers or illness incidence of the brand new illness that we need to deal with with the present drug.
What’s novel about your method?
This drug repurposing pipeline—going from mechanistic research to real-world information from digital well being data—is totally novel for autoimmune illnesses. Digital well being data have been used to have a look at the potential for brand spanking new lipid-lowering medicines as a result of you’ll be able to measure ldl cholesterol and see if it has decreased over time. For autoimmune illness, there is not one set of related biomarkers to have a look at.
We nonetheless have to do randomized managed trials and different research, however designing a mannequin for validating autoimmune disease-related medication this fashion is totally new. We additionally do not must give attention to just one autoimmune illness. We are able to take a look at all autoimmune illnesses collectively as a result of many autoimmune illnesses share related illness mechanisms, which accelerates the method and doubtlessly makes it extra environment friendly.
Lastly, going from mechanistic research of cells on to human information lets us soar over some labor-intensive and time-consuming steps. This method may doubtlessly enable us to validate medication extra shortly as a result of we are able to straight consider the efficacy of the drug in people.
Why is drug repurposing doubtlessly significant for individuals with autoimmune illness?
There aren’t a whole lot of good therapy choices and current remedies usually have unhealthy unwanted effects, which may restrict their use. The hope is that if yow will discover new medication, they may have fewer unwanted effects and is likely to be more practical.
You’ve got recognized some potential candidates for repurposing for autoimmune illnesses. What occurs subsequent?
Sure, we recognized six potential medication. For instance, hyoscyamine is a medicine usually prescribed for irritable bowel syndrome. It reverses illness gene expression the identical means as steroid medication, like cortisone, and we noticed that it reverses gene expression in genes related to rheumatoid arthritis. It may very well be efficient for treating rheumatoid arthritis and have fewer unwanted effects than the at present used anti-inflammatory medication.
The following step could be to take the recognized medication to medical trials and proceed to refine the framework to check different illnesses. With extra proof from human trials, we’re poised to rework well being care for a lot of People, making it extra inexpensive and efficient.
Extra info:
Lida Wang et al, An atlas of single-cell eQTLs dissects autoimmune illness genes and identifies novel drug courses for therapy, Cell Genomics (2025). DOI: 10.1016/j.xgen.2025.100820
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