Periodic Reporting for period 2 - REMEDi4ALL (BUILDING A SUSTAINABLE EUROPEAN INNOVATION PLATFORM TO ENHANCE THE REPURPOSING OF MEDICINES FOR ALL)
Okres sprawozdawczy: 2023-09-01 do 2025-02-28
The vast majority of the over 7000 known diseases lack effective treatment. Therefore, there is an urgent need to make better use of the medicines that we already have in hand. These include medicines that have already been approved for human use, as well as experimental medicines still in clinical trials already showing good pharmaceutical properties (e.g. body distribution and clearance, stability, bioavailability) and human safety. In fact, most approved drugs have the potential to treat many more and diverse diseases than they were originally approved for. The mission of REMEDi4ALL is thus to make it easier and more reliable to find these new medical uses for already-existing drugs. We also aim to show that many “repurposed” medicines can be taken into clinic faster and at the fraction of the cost of developing a completely new drug from scratch.
But with hundreds or even thousands of opportunities to repurpose approved and experimental medicines for the thousands of diseases that remain without approved treatment, REMEDi4ALL must also help to transform the drug repurposing field. This needs to involve not only scientists, doctors, and patients, but also regulators, policymakers, companies and investors, so that all researchers will face fewer barriers and be able to bring much-needed treatments to patients faster and more effectively.
But with hundreds or even thousands of opportunities to repurpose approved and experimental medicines for the thousands of diseases that remain without approved treatment, REMEDi4ALL must also help to transform the drug repurposing field. This needs to involve not only scientists, doctors, and patients, but also regulators, policymakers, companies and investors, so that all researchers will face fewer barriers and be able to bring much-needed treatments to patients faster and more effectively.
Launched in September 2022, REMEDi4ALL has already made great progress in creating an international platform for drug repurposing, spanning 26 sites in 11 different countries across Europe and UK. At our core we have two founding principles:
1. Researchers need to work closely with patients right from the very inception of a drug repurposing idea and continue that collaboration through every stage development: from approval by regulatory authorities to clinical practice to ensure that drugs are accessible and reimbursed for the patients who need them.
2. You can’t skip any steps in drug repurposing—even though a drug may already be approved, you still have to make sure that all the requirements for showing the safety and effectiveness for a new drug are also met for a repurposed drug. This is especially the case if the original drug must be modified (such as converting a topical cream to an oral tablet) or if the profile of the patients will be different (such as children if the drug had previously been approved only for adults). Taking shortcuts or not working closely with patients in the development process risks missing a crucial opportunity to use repurposing to address a critical unmet medical need.
We have applied our core principles to the four drug repurposing projects that launched together with the REMEDi4ALL platform. Two of these projects, in pancreatic cancer and “brittle bone” disease, are actively enrolling patients. A third project, in an ultrarare disease that affects multiple organ systems in the body and disrupts the normal development of infants and children, was given a huge boost thanks to the donation of key data from a major pharmaceutical company and has prepared its clinical trial application (CTA) through two rounds of formal scientific advice. Finally, our project in pandemic preparedness has screened hundreds of already-approved drugs for potential to be used as a first-line response for the next viral threat and will soon be selecting the most promising candidates for preclinical development.
1. Researchers need to work closely with patients right from the very inception of a drug repurposing idea and continue that collaboration through every stage development: from approval by regulatory authorities to clinical practice to ensure that drugs are accessible and reimbursed for the patients who need them.
2. You can’t skip any steps in drug repurposing—even though a drug may already be approved, you still have to make sure that all the requirements for showing the safety and effectiveness for a new drug are also met for a repurposed drug. This is especially the case if the original drug must be modified (such as converting a topical cream to an oral tablet) or if the profile of the patients will be different (such as children if the drug had previously been approved only for adults). Taking shortcuts or not working closely with patients in the development process risks missing a crucial opportunity to use repurposing to address a critical unmet medical need.
We have applied our core principles to the four drug repurposing projects that launched together with the REMEDi4ALL platform. Two of these projects, in pancreatic cancer and “brittle bone” disease, are actively enrolling patients. A third project, in an ultrarare disease that affects multiple organ systems in the body and disrupts the normal development of infants and children, was given a huge boost thanks to the donation of key data from a major pharmaceutical company and has prepared its clinical trial application (CTA) through two rounds of formal scientific advice. Finally, our project in pandemic preparedness has screened hundreds of already-approved drugs for potential to be used as a first-line response for the next viral threat and will soon be selecting the most promising candidates for preclinical development.
The broader and long-lasting impact of REMEDi4ALL relies in our goal to transform the drug repurposing landscape through wide dissemination of our advances and learnings, and in training the next generations of researchers and patient advocates. Our ambitious goal is to be able to make it faster, easier and cheaper to repurpose drugs for all projects that follow, even those not working directly with the REMEDi4ALL consortium.
We aim to demonstrate that common approaches can be applied across many drug repurposing projects, proving that there is no need to reinvent the wheel each time. As such, a key part of our success is bringing in a strong, patient-centric pipeline of new drug repurposing projects, where every project is designed to show that a common problem in drug repurposing can be overcome, and that the lessons learned can be directly used to support the next drug repurposing project facing a similar challenge.
Through this approach, our goal is to systematically tackle all the barriers that have burdened drug repurposing projects in the past, and to open the path to realizing the full potential of drug repurposing in addressing the hundreds and thousands of diseases that remain without approved treatments.
We aim to demonstrate that common approaches can be applied across many drug repurposing projects, proving that there is no need to reinvent the wheel each time. As such, a key part of our success is bringing in a strong, patient-centric pipeline of new drug repurposing projects, where every project is designed to show that a common problem in drug repurposing can be overcome, and that the lessons learned can be directly used to support the next drug repurposing project facing a similar challenge.
Through this approach, our goal is to systematically tackle all the barriers that have burdened drug repurposing projects in the past, and to open the path to realizing the full potential of drug repurposing in addressing the hundreds and thousands of diseases that remain without approved treatments.