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17th of October 2018

Health



Patient with Creutzfeldt-Jakob disease to receive experimental treatment

Oct. 9 (UPI) -- For the first time, a patient with the rare, fatal brain disease Creutzfeldt-Jakob will be given an antibody compound developed in London.

Researchers at University College London have created PRN100 for treating the disease, which currently has no cure, according to an announcement Tuesday.

The disease affects 1 in every million people per year worldwide, including about 300 cases in the United States, according to the National Institute of Neurological Disorders and Stroke.

Symptoms of CJD, including failing memory, behavioral changes, lack of coordination and visual disturbances, occur around age 60 -- and about 90 percent of individuals die within 1 year. Patients on average die within six weeks of being diagnosed.

Researchers report that lab testing of the antibody has been encouraging, and that UCL received the go-ahead from a judge in the Court of Protection, who determined it is lawful and in the patient's best interests to receive the unlicensed treatment.

"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," said Dr. John Collinge, director of the Medical Research Council Prion Unit at UCL. "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."

Creutzfeldt-Jakob is the most common of prion diseases that affect humans and animals.

About 5 percent of all CJD cases are inherited as a result of a faulty gene, while the rest develop from a sporadic form of the disease.

Sporadic CJD may develop because some normal prions spontaneously change into the infectious form of the protein and then alter the prions in other cells in a chain reaction.

The immune system normally produces antibodies to fight infections but abnormal prions are made of one of the body's own proteins and immune system does not make antibodies to fight them.

"Caring for patients with CJD involves trying to use medicines to alleviate symptoms only but sadly, the disease always results in the rapid death of the patient," UCLH's Chief Executive Professor Marcel Lev said. "The court's confirmation today is an important step forward in tackling this devastating illness."

In the study, a patient with sporadic CJD will initially receive the treatment intravenously at UCLH.

The researchers don't expect the treatment to reverse any previous brain damage.

UCLH created an oversight group, independent of the MRC Prion Unit and treating clinicians, that considered the clinical, safety, legal and ethical issues arising from the potential use of this unlicensed treatment for CJD.

After treatment ends with the patient, UCLH will decide whether to treat a second one.

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