The programmes of £750,000 each are due to start in September this year and will run for three and four years respectively. Both programmes of research will build on the work achieved in two of the Trust’s first SRCs: Professor Davies’ ‘Pseudomonal infection in CF: better detection, better understanding, better treatment’ SRC and Dr Gray’s ‘INOVCF: Innovative non-CFTR Approaches for Cystic Fibrosis Therapy’ SRC. Find out more about this research here.
What is personalised medicine?
Personalised medicine means finding the most appropriate drug for each patient, by taking into account the many different factors that contribute to cystic fibrosis (CF) symptoms in a particular individual, and by tailoring their treatment and care accordingly. For example, people taking genotype-specific medicines (also known as precision medicines, for example Orkambi and Kalydeco) will have started taking the drugs at different ages, with different levels of lung function and different CF-related complications. This means that for each person their care and treatment will be slightly different. For those not lucky enough to be able to take precision medicines, there are still lots of ways that your CF care and treatment can be tailored to you – for example knowing in much greater detail how to treat the bugs growing in your lungs.
Last year the Trust specifically requested research proposals to increase our knowledge of how personalised medicine should work in CF, one of the key themes in the Trust’s new research strategy. Find out more about each of these programmes below.
From Newcastle to the Netherlands: restoring airway function using alternative chloride channels
One of the functions of CFTR, the gene that is damaged in CF, is to allow the flow of chloride and bicarbonate into the cells that line the surface of the lungs. In people with CF, chloride and bicarbonate cannot get through the CFTR channel, leading to thick, sticky, acidic mucus (find out more here) that bacteria thrive in and which is harder to clear. However, there are other channels that allow the passage of chloride and bicarbonate. The focus of this research programme will be to find new ways to increase the activity of two alternative chloride channels (ACCs), so that they can ‘compensate’ for the faulty CFTR.
The research will be conducted in labs in Newcastle, Utrecht in the Netherlands, Lisbon in Portugal and Regensburg in Germany. The team, led by Dr Mike Gray, will be studying the effects of drugs that 1) increase the amount of ACCs found on the surface of the cells and 2) increase their activity. This approach has the potential to develop a treatment that people with any genotype can take. An important question that will also be answered is whether combining the ACC drugs with existing CFTR drug modulators could have any additional benefit.
“We are very excited about the new SRC, which builds on previous work supported by the Trust. Our ultimate goal here is to develop new drug therapy approaches that will help restore lung function in a patient-specific, personalised manner for all people with cystic fibrosis” commented Dr Mike Gray, Principal Investigator of the SRC.
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Personalised approach to Pseudomonas
In her previous SRC, Professor Jane Davies and her team found a large variation in how different strains of Pseudomonas aeruginosa bacteria live in the lungs of people with cystic fibrosis. These different strains can make a big difference to whether Pseudomonas becomes established in the lungs and how easy it is to treat.
The ‘Personalised approach to Pseudomonas’ SRC will investigate better ways to detect Pseudomonas, understand how it settles in the lungs on a strain-by-strain basis, and develop more effective drugs for treating it.
For someone living with CF the first step is detecting the presence of an infection. The researchers will be looking at methods for rapid and easy detection of Pseudomonas, looking for its presence in the most easy-to-collect way possible. Working with industrial and academic collaborators, Professor Davies and her colleagues at Imperial and Amsterdam University will be looking at ways of detecting Pseudomonas in breath – a method which sounds simple, but despite a number of research attempts, is not available as a clinical tool.
Pseudomonas acts like a carefully coordinated war machine: from building defensive layers, to poisoning its attackers with toxins, to preparing for battle behind the scenes, the infection has a number of strategies that allow it to carefully adapt itself to living in the lungs of people with cystic fibrosis. An important aspect of the new SRC is to understand these strategies better, and spot any differences in strategies between different strains. Ultimately, this detailed understanding will lead to the development of more effective treatments.
“We are delighted to have been granted Personalised Medicine SRC funding to build on the achievements of the collaboration established from our first award”, commented Professor Jane Davies, Principal Investigator of the SRC.
“Just as no two patients are identical, the bacterial strains harboured by patients are also different, either in their genes or their behaviours. Understanding these differences, and their implications for treatments, could substantially reduce the detrimental effect Pseudomonas has on someone’s lung health.”
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Find out more about personalised medicine
Personalised medicine is an important aspect of the Trust’s new research strategy. If you’d like to hear more about personalised medicines from the experts then you may be interested in this year’s UK CF Conference in Birmingham from 9-10 September. Book your tickets today!