The difficulty in developing a treatment

 

The MRC Prion Unit at UCL and other researchers around the world have conducted long-term research to investigate potential antibody treatments for prion disease and to see if such treatments might work and what side effects or other safety issues might be anticipated. The immune system recognises foreign proteins and other parts of germs as being alien to the body and this leads to the body producing specific antibodies tailored to fight that infection. However, since prions are formed from one of the body’s own proteins they are not recognised in the same way by the immune system and lifesaving antibodies are not produced. This is one of the reasons why prions are so lethal.

 

 

How might PRN100 work?

 

PRN100 is an antibody specifically designed to bind tightly to the normal protein with the aim of preventing it from combining with the prions and in this way stop a chain reaction and formation of new prions.

 

Antibodies have also been found to have potent activity in laboratory test models of prion disease.

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The Challenges Ahead

 

Laboratory tests have provided a “proof of principle” that PRN100 works by preventing the chain reaction and the formation of new prions, but there are still many challenges for patient treatment.

 

Despite these unknowns, given the lack of any alternative treatment for a disease that is rapidly and invariably fatal, the research team at the MRC Prion Unit at UCL, patients, relatives and colleagues at UCLH feel that PRN100 should be offered to patients with rapidly progressing CJD at this time. The researchers are seeking Trust approval to do this.

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Patient Treatment Challenges 

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Will sufficient PRN100 get from the blood into the brain, across the blood-brain-barrier (a natural mechanism protecting the brain) if given by intravenous infusion (into a vein in the arm) or will it need to be given directly into the fluid (cerebrospinal fluid) around the brain?

 

Will there be immediate or long-term side effects of a severity that limits an effective dose being given?

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