First use of pioneering phage virus therapy to treat patient with cystic fibrosis

A new treatment that uses a cocktail of naturally-occurring viruses to infect and destroy bacteria has been used for the first time to treat a fifteen year old patient at Great Ormond Street Hospital (GOSH).Isabelle aged 17, is recovering well and has had no significant side effects from the treatment, which was given at GOSH under Dr Helen Spencer, Consultant in Paediatric Respiratory Medicine. The GOSH team worked closely with experts on phage therapy from the University of Pittsburgh to develop this cutting edge treatment.

Isabelle was born with cystic fibrosis, a condition which causes mucus to build up in the lungs which can lead to infections of bacteria. After a double lung transplant and several courses of strong antibiotics, the bacterial infection was still present and had spread to infect her liver, and more than 20 other locations on her skin.

Doctors at GOSH worked with US scientists to develop a new therapy which uses bacteria-eating viruses called bacteriophages (phages) to fight bacterial infections. The team carried out tests in the lab to work out which combination of phages would have the best chance of clearing the infection. Two of the three viruses in the cocktail eventually used had been genetically modified to make them more effective.

In June 2018, Isabelle returned to GOSH and, after safety tests had been carried out, she was given the cocktail twice daily via a drip and applied to her skin. Six weeks later her liver scan revealed the infection had essentially disappeared.

Dr Spencer said: “To see her gradually improve over the last months has been incredible and we have watched her get back to a normal quality of life.”

The new treatment is particularly significant given the rise in antibiotic resistant bacteria. Phage therapy uses a different more targeted approach to fight specific infections so are of growing interest to doctors as an alternative therapy.

Dr Graham Hatfull who led the development of the phage therapy at the University of Pittsburgh said: “We didn’t think we’d ever get to a point of using these phages therapeutically. It’s a brilliant outcome.”

After six months of treatment, the infected areas on Isabelle’s skin had healed, and only one new infection had appeared. Now Isabelle still receives two infusions of the therapy a day but the infection is under control and she is now at home, studying for her A-levels and learning to drive.

So far this treatment has only been used on one patient so the team cannot be completely certain that Isabelle’s recovery was thanks to the new therapy. To test this the team are now planning clinical trials to explore whether similar therapies could be used to treat other patients.