Cystic Fibrosis Trust

Inspire: Taking Gene Therapy from Lab to Life

To develop an effective treatment for Cystic Fibrosis (CF) in the lungs. Cystic Fibrosis is one of the UK’s most common life-threatening inherited diseases. Caused by a single faulty gene that results in the internal organs, especially the lungs and digestive system, becoming clogged with thick sticky mucus. This leads to chronic infections and inflammation in the lungs and 90% of deaths from CF are due to lung damage People with CF face a gruelling regime of daily treatment to try to keep their lungs and digestive system as healthy as possible. CF directly affects over 100,000 people globally and over 8,500 people in the UK. Life expectancy from the disease varies around the globe; in the UK around half of those with CF can expect to live over the age of 35. In other parts of the world life expectancy is much lower, as patients are not diagnosed early enough or do not have access to the necessary drugs and treatments to keep them well.

history Campaign has now closed

It ran from 6:21 PM, 27 June 2011 to 5:42 PM, 7 December 2011

Registered Charity in England and Wales (1079049)

Amount raised

£50

Donations

1

    Category

  • Health/WellbeingHealth/Wellbeing
  • Medical ResearchMedical Research

    Helping

  • Children (3-18)Children (3-18)
  • Women & GirlsWomen & Girls
  • Young People (18-30)Young People (18-30)
  • OtherOther

Location

Situation

The Cystic Fibrosis Trust is currently investing over £3.5 million a year on a programme of research which aims to add a healthy copy of the faulty CF gene to the lung. This method of treatment, known as gene therapy, offers the opportunity to fundamentally improve the quality of life and significantly reduce the number of lives lost to CF each year. This is because gene therapy aims to correct the underlying cause of CF instead of treating the symptoms of the disease. Also, unlike drug treatments, which are designed to treat a specific mutation of the gene, gene therapy will be able to treat all mutations and would therefore be of benefit to the majority of people with CF. In 2001, The Cystic Fibrosis Trust brought together the UK’s leading CF genetic scientists to drive this research forward through the UK Cystic Fibrosis Gene Therapy Consortium. This team of 80 dedicated scientists and clinicians are working to develop a clinically effective therapy to stop the lung damage that kills 90% of people with CF. This is the largest group studying CF gene therapy in the world and its work is peer reviewed by an independent Scientific Advisory Committee, appointed by the Trust. Since its formation the Consortium has developed a lead gene therapy product, which comprises the healthy copy of the gene and a carrier to get it into the right place in the cells lining the airways. This has taken many years of hard, long and expensive work. This research has now reached a key milestone on its journey to deliver gene therapy as an effective treatment for CF in the lungs. A pilot study involving 27 people with CF, which was started early in 2009, is already showing encouraging result in terms of both the safety and efficacy of the gene therapy product following the administration of a single dose. The patients taking part in the pilot study had a mild flu-like reaction to the dose but these symptoms were short lived and mostly gone within 48 hours. The dosing time, or the dose itself, will be altered to find an acceptable way of delivering the gene repeatedly. Many of the patients have also shown evidence of normal CFTR protein (which is absent or much reduced in people with CF) being produced in their nose and lungs following the gene therapy. In some cases, the amount of protein has been up to the level of that in people without CF. Whilst this only happened in a few of the patients these are the largest changes ever recorded using any type of therapy worldwide in CF. The encouraging results from the pilot study have given the CF Trust the confidence to proceed now with a multi-dose trial that will involve 100 people with CF. A preclinical toxicology programme has to precede the multi-dose trial but it should start around the middle of 2011 with the results being available in 2013. In 2010/2011 it will cost the Cystic Fibrosis Trust about £3.6 million to support the scientists and clinicians working on the Consortium. The nature of this research also means that the Trust has to commit to similar levels of annual funding up to 2013. In addition, the costs of the gene therapy product for the multi-dose trial are £9.9million, towards which we have already raised £6.4 million. This leaves a shortfall of £3.5 million, of which £700,000 needs to be paid by the end of March 2010; £390,000 in 2010/2011 and £2.4 million in 2011/2012.

Solution