Scientific title: The beta-2 score and beyond: new composite outcomes measures of islet cell function for use in clinical trials
Principal investigator: Professor Colin Dayan
Institution: Cardiff University
Duration of award: 01 October 2019 – 30 September 2022
This project aims to overcome two major roadblocks to developing and licensing immunotherapies for people newly diagnosed with type 1 diabetes:
- The length of clinical trials in type 1 (which can be three times longer than in other autoimmune conditions, such as psoriasis).
- The unclear path to getting a new therapy approved by regulators (since no immunotherapies for type 1 have been licensed before).
To do this, the researchers will use mathematical modelling to improve the way clinical trials are analysed for success.
What will Professor Dayan do?
In type 1 diabetes, the current way to tell how well a new immunotherapy treatment is working is to measure the amount of a molecule called C-peptide in the person’s blood after a meal.
C-peptide is made by the body when it creates insulin, so if there is more of it in the blood, then the person trying the treatment is making more insulin.
But this doesn’t take into account all the other outcomes that matter to people with type 1 – like the number of hypos they experience, or how difficult their blood glucose is to manage.
So, with this project, Professor Dayan aims to create a better way to test how well an immunotherapy is working.
To do this, he will adapt a system that already exists, called the BETA-2 score. BETA-2 is a combination of four measurements, and is used when people with type 1 are given a transplant of beta cells, to give doctors an idea of how effective the transplant has been.
By re-analysing data from more than 2,400 participants on 25 previous immunotherapy clinical trials, Professor Dayan will see if the BETA-2 score would have worked for these trials and, if so, how the score should be calculated.
He will also explore whether any other measurements could be added to the score to make it more accurate.
This should tell him the best set of measurements to take to accurately and quickly assess how well a new treatment is working.
How will this research help people with type 1?
This research, if successful, could make it possible to run trials more quickly and cheaply, making it easier to develop new treatments for type 1.
It would also make life easier for clinical trial volunteers, as the BETA-2 score can be calculated from one blood test, so does not require a full hospital visit.
The team will also work with medicine regulators in Europe and the US to ensure that this new test is rigorous enough to pass their standards for licensing new treatments – another vital part of speeding up the process of bringing a new treatment to people with type 1.
Is JDRF funding any other research like this?
JDRF is also co-funding Professor Dayan to lead the Type 1 Diabetes UK Immunotherapy Consortium. The consortium promotes immunotherapy research in type 1 diabetes, by running more clinical trials, and improving the way they are carried out.