Small improvement seen in teenager with vCJDBMJ 2003; 327 doi: https://doi.org/10.1136/bmj.327.7418.765-a (Published 02 October 2003) Cite this as: BMJ 2003;327:765
Neurologists have expressed cautious optimism about small improvements and lack of toxicity in a UK teenager with variant Creutzfeldt-Jakob disease (vCJD) treated with an experimental drug, pentosan polysulphate.
The patient, 19 year old Jonathan Simms from Belfast, Northern Ireland, has been treated with pentosan polysulphate by injection into the brain since a decision by the High Court last year that the potential benefits outweighed the risks (BMJ 2003;326: 8).
Neurologists meeting in Belfast last week to review the early results of treatment considered that the drug seemed to be well tolerated and that it might have improved his condition.
After eight months' treatment, the patient had shown some improvement in neurological functions, including regaining the ability to swallow. His parents claimed that he had become more alert, responding to verbal instructions and attempting to speak. Monitoring of his heart rhythm showed less variation—thought to be associated with brain stem damage—than before starting treatment.
Nikolai Rainov, of the Walton Centre for Neurology and Neurosurgery, Liverpool, said: “The question of efficacy is still very much open. However, we believe that the brain stem now functions better than it did six to eight months ago.” He reported: “Some neurological functions have improved significantly over the course of treatment. The patient is even able to obey commands—something which was not the case six months ago.”
He considered that pentosan polysulphate might have a role in prolonging survival in patients with vCJD and in improving their quality of life. He added: “I believe that instigating treatment at an early stage could achieve significant extension of the life span of these patients.” See News Extra at bmj.com