ClinicSpeak: thinking the impossible my new rose-tinted view of the world

Preventing end-organ damage: the new therapeutic target in MS. #ClinicSpeak #MSResearch #MSBlog

I attended a meeting today and spoke about treating-2-target beyond NEDA. I spent most of the talk discussing how we are beginnging to focus on preventing end-organ damage and daring to look at restoring lost function.


I am often asked why I went into medicine, neurology and MS. I am not sure, but as I was exposed to chronic disease at a young age I became interested in medicine. My father had an autoimmune disease of the kidney that caused his kidneys to fail. When I was 12 he started dialysis and he waited a further 10 years before agreeing to have a kidney transplant. The tragedy was that when he was a teenager he had a few episodes of blood in his urine, or haematuria, that was precipitated by exertion. Although it was brought to the attention of his GP it never taken seriously by his general practitioner (family doctor), nor himself, so the problem was never investigated. By the time he presented with headaches and nose bleeds due to severe secondary hypertension and problems with blood clotting he was in chronic renal failure. In other words the inflammation in his kidneys had caused irreparable end-organ damage.  His kidneys were small and scarred. The nephrologist who saw him said 'if only I had got to you sooner with immune therapies I would have had a chance of saving your kidneys'. If only; two small seemingly trivial words that mean so much and at a personal level resulted in so much some suffering, reduced quality of life and cost society so much money.

Fortunately my father lived in an era when salvage kidney therapy was possible; he had peritoneal dialysis, haemodialysis and subsequently a kidney transplant. All three of these innovations are modern miracles and are now part of routine clinical practice. In a previous era he would have died a young man. The lessons I learnt from father’s case were (1) get a good doctor who takes things seriously and (2) early diagnosis and treatment is better than salvage therapy. These lessons are as pertinent to MS today as they were in my father's case 40 years ago. Unfortunately for MSers we don’t have salvage therapy; we can’t repair or transplant the brain or spinal cord. Therefore, your best chance of avoiding end-organ damage is early effective therapy and getting yourself a good neurologist who takes treating your disease seriously. We have to start viewing DMTs as preventive therapies, i.e. to prevent irreparable end-organ damage.

In almost every MS clinic I do I see the difference between pwMS who have either had a delay in getting a diagnosis, or a delay in getting access to an effective therapy, compared to those pwMS with early access to optimal therapies with no evident disease activity (NEDA). A large number of the latter pwMS are leading near normal lives, whereas the former have to live with the consequences of end-organ damage. Bladder, bowel and sexual dysfunction, cognitive problems, anxiety, depression, fatigue, and the rest. I am stressing the hidden problems, because they are invisible to others but cause so many problems for individuals.  For those of who need reminding, the blue or symptomatic line in my MS tube map is the consequences of end-organ damage; may be I should change it to the end-organ damage line?


The good news is I spoke at to an audience of scientists working on an exciting pipeline of products that are neurorestorative. Yes, neuro-restorative. I have said before I thought neurorestoration was science fiction, not anymore. This class of compound stimulates axonal sprouting, synaptogenesis, remyelination and cortical plasticity. We are in the process of designing the phase 2 programme and the good news is that MS is one of the treatment targets. It is hard to believe how far the field has come in 25+ years; we have gone from no therapies to treatments that are promoting repair.

May be I should wear those pair of rose-tinted spectacles I bought recently more often? 


CoI: multiple

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