EPSRC Research and Partnership Hub in Microscale Science and Technology to Accelerate Therapeutic Innovation (MicroTex)

The problem. Proving the efficacy of new drugs is very costly and time-consuming and both grow exponentially with each stage of development – preclinical, first-in-human, then several phases of clinical trials. The pathway can take over ten years and cost more than US$1Bn. Many drugs fail and are discarded along the way, resulting in a huge waste of time, resource, and money. This does not deliver parity of access to populations across the globe nor across the strata of society and is severely constraining the rate of new drug development. This is especially true for drugs targeting infection and inflammation as the antibiotic pipeline is diminishing due to the huge costs for new drug development using outdated methods and trial methodologies.

A potential solution. One way forward lies in an approach called “Intra target Phase-0/Microdosing”. “Phase-0” occurs very early in the drug development cycle, before the longer and more expensive phases. “Intra target Microdosing (ITM)” involves administering a tiny amount of drug to a microscopically small region of the body and measuring any therapeutic benefit at that point. There is no wider systemic safety risk to the subject. When accomplished effectively, ITM can reject poor candidate drugs and raise confidence in the later success of promising drugs resulting in substantial savings of time, resource, and cost for drug developers and a more efficient pipeline of effective drugs becoming available to the population.

Our contribution. ITM, while promising, is at a relatively early stage of development. We have already, delivered multiple ITM studies , but have been limited by the underpinning measurement technology. One of the main factors limiting the rate of uptake is the capability of the techniques required to deliver a precise microdose to a specific location in the body then to measure the effect of that microdose later at the same location. This shortcoming must be addressed to unleash the potential of ITM. MicroTex will help bring this about.

First by leading the way – pursuing its own pioneering research to begin solving the problems by developing and optimising an initial set of scientific and engineering techniques to improve ITM in a small number of exemplar organs (we have selected the lung and the eye). We have the expertise and track record required to progress these technologies into human studies.

Second by making the case for ITM by actively partnering and networking to bring more players to the table – persuading members of the wider scientific and engineering community to participate in expanding the range of techniques available, and the number of conditions and organs addressed, alongside pharmaceutical companies and Phase-0 international networks.

Third by developing a people-centric research environment with embedded training capacity so that researchers and other professionals can access the knowledge and skills necessary to effectively implement the new multidisciplinary approaches thus allowing their use to spread rapidly.
This will all be enabled and augmented through deep engagement with patients and the wider population to explain the benefits, risks, and limitations, take on board their concerns, and work together with them to achieve the best solutions.

Our mission. Through development of a new toolset to increase the performance of ITM , we will bring about a positive transformation of the drug development pipeline, that will be of huge and long-term benefit to mankind.