Flutterby Fundraisers to Sponsor Consortium Scientist

Friday, November 3rd 2017

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Announcement from Flutterby Fundraisers.

Flutterby Fundraisers are delighted to announce our sponsorship of Dr Aarash Saleh, Clinical Research Fellow, Imperial College London.

Since coming together as a team in 2007 and obtaining charity status in 2014, we have had a close working relationship with Professor Eric Alton and the UK CF Gene Therapy Consortium.

Through your continued support we have been able to donate a quarter of a million pounds to the teams, assisting their research programmes based in 3 of the UK's leading Universities, Imperial College London, Oxford and Edinburgh.

This evening we are delighted to announce a new partnership, cementing that relationship as we take on the sponsorship of Dr. Saleh, who will be working on the new and exciting Wave 2 of research. We feel that being able to put a face to the project will give the charity and our supporters a much more personal touch and will allow us to give you more regular and informative updates on what is involved in the day to day processes of the research team.

We hope you will join us in welcoming Aarash to the Flutterby Family.

 

About Flutterby Fundraisers.

Flutterby Fundraisers for cystic fibrosis is a not for profit voluntary charity, funding research into a successful gene therapy treatment for one of the UK’s most commonly inherited life limiting conditions, cystic fibrosis.

1 in 25 people carry the gene that causes CF and around 11,000 babies, children and adults in the UK have the condition.

 

Dr Aarash Saleh, Clinical Research Fellow, National Heart and Lung Institute, Imperial College, London

Gene therapy involves using a molecular delivery vehicle (vector) to introduce healthy copies of genes which are missing or defective into the cells which need them. In 2015 a collaboration of scientists led by our group at Imperial showed for the first time ever that monthly gene therapy treatment nebulised to the lungs could stabilise lung function over a year. We are now planning to perform the first ever trial testing our next generation gene therapy. This treatment which is based on a virus (called a lentivirus) has been shown to be considerably more effective at delivering its healthy copy of the CFTR gene compared to the vector used in the 2015 trial. In our first trial the new treatment will be delivered to the noses of people with CF to look at whether it is effective at transferring the gene (efficacy) and to confirm that it does not cause harmful inflammation or immune responses (safety).

Thanks to the funds raised by Flutterby Fundraisers, I have been able make significant progress assessing a new technology called RNAscope that allows us to detect very low levels of specific genes present in cells with unprecedented accuracy so that we can determine the numbers of viral particles that have been transferred to cells. So far our data have confirmed earlier exciting results indicating that once the gene is transferred into cells it remains in place and is detectable for at least 6 months which could mean that a single dose of the new treatment would have a therapeutic effect lasting at least this long (see image below).

Mouse airway cells from animals that were treated with the gene therapy vector six months earlier. Red dots inside many of the cells indicate that they are continuing to produce the gene transferred by the vector

Funding from the Flutterby Fundraisers has also allowed me to perform experiments refining our safety measurements including how we detect shedding of virus particles in body fluids. By using a cutting-edge technology called digital PCR we hope to be able to detect low numbers of virus particles in various sample types. This will give us a more accurate way of confirming the safety of this product compared to previous trials and will potentially impact whether patients need to stay in hospital after a dose of viral gene therapy and if so for how long. 

 

Flutterby Fundraisers for cystic fibrosis


 

DNA fragments being cut from an agarose gel exposed to UV.

 

Pellets of DNA following precipitation.

 

Schematic diagram of the large human airways.

 

E.coli from a large scale industrial production of our clinical trial plasmid pGM169.

 

Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.

 

Human airway liquid interface cultures transduced with a lentivirus expressing Luciferase.

 

A CFTR Western blot, to confirm protein production in cell culture.