A patient with Creutzfeldt-Jakob disease is to be the first to receive a new antibody treatment after winning court approval to circumvent drug approval laws.
The antibody has so far shown “encouraging” results in laboratory trials – but has not been subject to phase 1 clinical trials.
University College London Hospital has created a special oversight group, independent of its researchers and doctors, to steer the introduction of the treatment.
The antibody, PRN100, has been developed at the MRC Prion Unit at University College, London, UK, which is led by CJD expert Professor John Collinge.
Permission to administer the treatment has been given by the Court of Protection. The patient has sporadic disease.
Professor Collinge said: “The antibody has been designed to bind tightly to normal proteins in the brain. The aim is to prevent abnormal prions from being able to attach themselves to healthy proteins, meaning that they cannot grow and cause devastation throughout the brain.
“As this is the first time this treatment has been used in humans we cannot predict what the outcome will be but laboratory testing has shown the potential to treat prion infection. A key issue will be whether a sufficient quantity of the drug is able to cross the blood brain barrier to reach the brain tissue where it needs to work.
“We will proceed with extreme caution in very tightly controlled conditions. A team of experts from a range of disciplines will make collective decisions in the best interests of the patient.”
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