Drug repurposing is the process of identifying new therapeutic uses for approved or investigational drugs that were originally developed for other medical conditions. Since drug repurposing involves screening drugs that might already be approved or have a known safety profile, treatments can be developed more quickly and at a lower cost compared to the traditional drug development process.

The types and number of compounds in a screen will depend on the research model and screening approach being used. Perlara has access to many drug-repurposing libraries, ranging from 1,500 to as many as 8,500 compound libraries. The composition of each library is different, but will typically include a combination of approved drugs, drugs in various stages of clinical development, nutraceuticals, supplements, bioactives or tool compounds. Based on your specific requirements and budget, we'll recommend the most suitable compound library for your screen.

In most cases, if a compound is available for purchase and research purposes, it can be included in a screening service. We will work with you to determine the best way to incorporate this compound into the screening process to obtain the most meaningful results.

The duration of the drug repurposing process can vary widely based on several factors such as the research model being used, the screening approach, and the drug repurposing candidates identified. From our experience, this process can span anywhere from 3 months to several years.

There are a series of conditions that need to be met in order to launch an initial clinical trial of a newly-identified repurposing medicine. The major components include the identification of (a) medicine(s) that are safe (or at least that have a favorable balance of risk and potential reward), a reasonable understanding of the biological mechanism by which the medicine might improve the patient’s condition, availability of the medicine(s) in a pharmaceutical-grade stock, and a supportive clinician or clinical team to prescribe the medicine (if necessary) and monitor the patient’s response. There are also sometimes project-specific challenges that your Cure Guide can help you navigate.

The results from drug repurposing screens provide information on how different compounds affect the phenotype of the disease model (e.g. growth in yeast), which helps us identify the most promising repurposing candidates for a disease. Specifically, they reveal which compounds improve the disease phenotype (referred to as rescuers) and which worsen it (referred to as sensitizers).

Both categories of hits provide valuable insights into the underlying disease mechanisms and potential disease-modifying targets. Understanding the mechanisms of action of screen hits and considering the attributes of a drug (such as its approval status, route of administration and safety profile), help identify the most clinically actionable repurposing candidates for a disease.

Once hits have been identified from the screen, our goal is to advance safe and clinically actionable drug-repurposing candidates into the clinic as fast as possible. However, the specific pathway from a hit to the clinic can vary depending on the repurposing candidates discovered in the screen. For instance, if readily available nutraceuticals or supplements are identified in a screen, treatment may begin within days of receiving the screening results. Whereas if a repurposing candidate is an existing approved drug or is still in clinical development, additional follow-up experiments or patient studies may be required to understand how the drug is working and to assess its efficacy and safety in patients.

With our extensive experience in translating discoveries from the lab to the clinic, we'll help devise and implement the best strategy for developing new treatments, guiding you every step of the way.

The applicability of drug repurposing data from one mutation to other mutations of the disease-causing gene may vary. Different mutations can alter the structure and function of the protein encoded by the disease-causing gene, potentially influencing how drugs interact with it, and each mutation can affect the disease's biological mechanisms differently. Therefore, while certain drugs may show promise in targeting one mutation, further validation across research models of different mutations may be necessary to fully understand their efficacy and applicability across various mutations causing the disease.

While there is no guarantee that a drug that can improve the disease state of a particular mutation will translate to others, in our experience common rescue mechanisms often exist, and there are factors that make this more likely. If different mutations have a similar pathological mechanism (i.e. loss-of-function, haploinsufficiency, dominant negative, gain-of-function, etc.), the likelihood that a particular drug candidate will translate across these mutations is higher. Your Cure Guide can help you understand the pathological mechanism of your mutation and ways to experimentally validate which hits from your screen will translate more broadly across different mutations.

Any data generated by research conducted or contracted by Perlara fully belongs to the client.

Perlara believes in the principles of open science and transparency. We frequently publish the latest discoveries from our partnerships with rare disease families and foundations on our Substack. We prioritize the sharing of de-identified results, to protect our clients and collaborators' intellectual property.