Plunge in clinical drug trials makes Britain an urgent case for treatment
Four-year-old Zunaira is watching the animated children’s series Love Monster on an overhead TV screen as she lies in a hospital bed with six electrodes stuck to her body.
Her 10-year-old brother Zain is busy on his iPad on a chair next to her in the clinical research facility at Great Ormond Street Hospital (Gosh) in central London, whose white walls have been brightened with reproductions of children’s drawings of leaves. They are here for a study trialling a new drug to treat a rare genetic kidney disorder that can be fatal.
Electrocardiograms (ECGs) to measure the heart’s activity and blood tests are performed before both children are injected with lumasiran to treat primary hyperoxaluria type 1 (PH1). The rare condition is caused by the buildup of oxalate, a substance normally filtered through the kidneys and expelled in urine.
“She gets very excited when she comes to the hospital because she gets treated like royalty,” says Zunaira’s mother, Hafsah.
Zunaira has been given the drug, initially every month and now every three months, since she was three months old, as part of a clinical trial that involves just five children. The results were so good that her brother Zain is getting the same drug on a compassionate basis, a rare treatment option that allows an unauthorised medicine to be used outside trials.
Her treatment is novel for another reason: Zunaira is a participant in the increasingly rare field of commercial clinical trials in the UK.
The dwindling nature of such pilots has prompted leading health figures to warn of the risk to the UK’s ability to develop cutting-edge treatments and bring them to patients.
At the Gosh research facility, which is funded by the National Institute for Health and Care Research,
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