The Sunday Times
Full article:
After chemotherapy failed to cure Emily Whitehead’s severe form of leukaemia, Oxford BioMedica’s new treatment has given her five cancer-free years
At just six years old, cancer patient Emily Whitehead had run out of options. Three rounds of chemotherapy had failed to cure a severe form of leukaemia. Her doctors could do no more; she was sent into hospice care.
Her desperate parents were left with only one option. They put Emily into an experimental programme, making her the first patient in the world to receive a new kind of gene therapy. The revolutionary treatment uses a stripped-down version of the HIV virus that reprograms cells to hunt down and kill cancer.
Within weeks of a single injection, Emily was at home, playing football again and spending time with her dog Lucy, who was born around the time she was diagnosed.
Five years later, Emily is a fit, healthy 12-year-old who aspires to be an artist and enjoys making YouTube videos of her craft projects. And the British university spinout behind the technology that saved her life is on the cusp of finding out whether the treatment is likely to get full regulatory approval.
In two weeks, America’s Food and Drug Administration will put the treatment devised by Oxford BioMedica, in league with Swiss pharmaceutical giant Novartis, before a panel of independent experts. If they like the findings from the human trials, it could pave the way for a full approval in October. A European Medicines Agency filing could follow later this year.
Last week, the market started to get excited; shares in Oxford BioMedica surged 40% amid expectations that it will be given the green light. Analysts predict the treatment could become worth in excess of $1bn (£770m).
Oxford BioMedica developed the technique of modifying the HIV virus to carry the treatment; Novartis is behind the treatment itself, after working with the University of Pennsylvania.
John Dawson, chief executive of Oxford BioMedica, is investing in his laboratories in Oxford to ensure they are ready for an upcoming — and vital — FDA inspection. “Parents are scrambling to get to this drug in the US,” he said. “We are the best in the world at doing this. It is hugely significant for us as a company.”
Analysts at Jefferies say the collaboration with Novartis is a significant endorsement of Oxford BioMedica’s technology, cementing its position as a leader in what is known as the lentiviral vectors field.
Jefferies estimates the treatment could cost up to $400,000 (£308,000) per patient in the US, with Oxford BioMedica receiving a royalty on each treatment. New York investment bank Rodman & Renshaw reckons that this royalty would be about 3% of overall treatment cost. On that assumption, it predicts Oxford’s share price could double to 14p.
By 2022, it forecasts $127.3m in annual revenue to Oxford BioMedica from sales of CTL019, the scientific name for the treatment. The company’s overall revenues were £30.8m last year.
For 20 years, Oxford BioMedica has sought to develop medicines for serious diseases such as Parkinson’s, blindness and cancer. It was founded in 1995 by professors Alan and Susan Kingsman, a couple who worked in Oxford University’s biochemistry department. They left to set up Oxford BioMedica, initially in search of a cancer vaccine.
The company floated on AIM in December 1996 with a market capitalisation of £50m — it now stands at £229.7m. Its partners include Glaxo Smith Kline, Sanofi and Orchard Therapeutics. However, it is the leukaemia treatment that excites investors and offers hope to millions of families. For Emily’s parents, from Philipsburg, Pennsylvania, it simply saved their only child’s life. “We were fighting every day so she could survive to the next,” said her father, Tom, 46.
Emily was the first of only 10 children to receive the treatment. In 2010, she was diagnosed with acute lymphoblastic leukaemia, a blood cancer that originates in young white blood cells in the bone marrow. It accounts for about 25% of cancers diagnosed in children under 15, and affected 760 children in the UK in 2014, the most recent figures show.
Initially, doctors were hopeful that Emily would recover. The first signs were good. However, 16 months after she went into remission, Emily suffered a relapse.
Her parents sought the help of the children’s hospital in Philadelphia, where Emily had been treated. Here, the couple learnt of the trial using the Oxford BioMedica and Novartis gene therapy. It works by removing the patient’s T-cells, a type of white blood cell, and reprogramming them in a lab. The genetically modified cells are injected back into the patient, where they multiply into an army of cancer-killing cells.
Kyriacos Mitrophanous, Oxford BioMedica’s chief scientific officer, said: “We take the HIV virus and strip out all the bits that cause disease, but retain the capabilities of getting genes and DNA into cells.” The body’s own attack cells are trained to recognise leukaemia. A key feature of the treatment is that it requires just one injection.
OXFORD BIOMEDICA IN NUMBERS
40% Increase in the share price last week
10 The number of days until a key US meeting to assess the treatment for approval
£229m The company’s current market capitalisation
In the first global trial of 63 relapsed children and young people, 83% of patients reached complete remission within three months of treatment, according to Novartis.
Emily received the treatment in April 2012 when she received the genetically modified cells in the clinical trials. She spent several weeks in intensive care before her parents were told the treatment had worked. Only two months after returning home on June 1, 2012, Emily was back on the sports field.
Her remarkable story is not unique; other children have enjoyed similar recoveries. Among them is Austin Schuetz, from Wisconsin, who was just three when diagnosed with the same kind of leukaemia. After multiple relapses, he was enrolled in a trial. Five years on, he is celebrating 3½ years cancer-free.
Another is Tori Lee of New Jersey, diagnosed with the disease in 2008, aged five. Years of chemotherapy had failed and she was offered the gene therapy. After five days in hospital, Tori went home and was back at school a month later.
The Whiteheads established a foundation in their daughter’s name to spread awareness of the therapy. Tom will speak at the FDA advisory hearing on July 12, backing approval for the treatment. The FDA’s decision is due in October.
“They need to know how much this has changed our lives, changed the world and given families hope,” said the power plant worker.
“The doctors never tell you what’s going to happen long term. But when she gets up in the morning everything is normal. That’s what a cure looks like as a parent.”
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