Court authorises experimental treatment for CJD patient
25 February 2019
This case concerned a man, KG, who suffered from the human prion disease CJD. As was explained in the judgment, prion diseases are invariably fatal, neurodegenerative conditions.
They are involve the build-up in the brain and some other organs of a rogue form of a naturally-occurring protein known as the prion protein. The rogue protein results from a change in shape of the normal prion protein. Once formed in the body, these rogue proteins (or prions) recruit and convert more of the normal prion protein into the abnormal form, setting off a kind of chain reaction which leads to a progressive accumulation of the rogue protein.
As the disease progresses, the normal functions of the brain begin to be lost causing problems with memory, thinking, speech, balance and movement. Cumulatively, these changes lead to impairments in the ability to do everyday tasks and, when severe, patients enter a comatose state in which they lie still and mute in a bed, ultimately followed by death.
In this hearing the University College London Hospitals NHS Foundation Trust sought the court’s authority to administer a treatment known as PRN100 to the KG. KG, represented by the Official Solicitor, his family and the Trust all asked for the court’s approval.
The matter went before the court because PRN100 has never been tested on or administered to any person anywhere. It was thus a completely novel treatment. The administration of a new and untested treatment like this is dealt with by the Code of Practice to the Mental Capacity Act 2005. At paragraph 6.18, it says:
Some treatment decisions are so serious that the court has to make them
This includes “cases where there is a dispute about whether a particular treatment will be in a person’s best interests”
Because of the progressive effect of his condition, KG did not have the capacity to make an informed decision himself. He was not able to retain the details of the risks and benefits of the treatment and make a decision for himself with capacity. The drug PRN100 is a humanised monoclonal antibody that has been in development since 2007. However, the substantial cost of a formal clinical trial, together with the rarity of CJD and the high risk nature of drug development, has meant that is has not been possible to raise the necessary funding. It has only been tested on animals.
The Court listed factors against KG’s treatment thus: (1) there was no evidence as to whether it was safe or effective in humans because there have been no clinical trials; (2) there may be negative side effects or adverse reactions which may be serious or even life-threatening; (3) it was possible that KG’s death would be accelerated or that he would be maintained in a poor state for a longer period than otherwise would have been the case; (4) it was impossible to quantify the chances of a significant or material benefit to KG.
The factors in favour of treatment were that (1) he wished to have the treatment; (2) his wife and family wished him to have the treatment; (3) his quality of life, though diminishing, was still one that is clearly worth preserving; (4) there was some evidence from the animal studies that PRN100 could have a positive effect on the progression of sporadic CJD in humans; (5) there was a sufficient possibility of unquantifiable benefit to justify the risks involved when, without treatment, KG would certainly deteriorate further and die within a short period; and (6) there was a clear and thorough plan for protective monitoring and oversight.
The actual treatment in itself is not especially invasive. It involves the administration of medication into the vein and will be gradually titrated upwards. But in the case of KG, if it appeared not to be reaching the brain cells, it would have to be administered directly into the brain. This would involve a short operation to place a reservoir under the skin of the scalp connected to a tube inserted surgically through the skull and brain into the ventricles. Invasive testing after administration, such as lumber puncture, would be necessary.
In one of these very rare cases involving novel or experimental treatment, the wishes of the patient and their family are a very important factor. In an earlier case about CJD, in 2002, the President of the Family Division, then Dame Elizabeth Butler-Sloss, said
A patient who is not able to consent to pioneering treatment ought not to be deprived of the chance in circumstances where he would have been likely to consent if he had been competent.” (Simms v NHS Trust  EWHC 2734 (Fam))
Cohen J then turned to the question of publicity. Counsel for the Press Association pointed out that people around the world were interested in this case and that they would naturally want to know how the treatment and the patient were progressing and, of course, if the treatment did not work there would be a need to know if and when KG dies.
The family were concerned that there should be no publicity about their predicament. Both KG and his wife did agree that they did not mind updates being given provided that their identity was protected, as of course it has been.
What they do not want is for bad news to be put in the public domain as it would make a difficult situation even more intolerable
The Trust was therefore authorised to give such information from time to time as is requested and is approved by the family. In the event of KG’s death,
the press should be notified of that fact after 72 hours have passed, that is after a full three days. It is not a very long period, but I have to say I cannot imagine that in the awful circumstances of this illness, that the death will be anything other than predictable when it comes. That will make it no better for the family but, bearing in mind the confidentiality which surrounds this patient, I hope that they will be properly protected.