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

11 thoughts on “ClinicSpeak: thinking the impossible my new rose-tinted view of the world”

  1. 1. rose-colored – reflecting optimism; "a rosy future"; "looked at the world through rose-colored glasses"

  2. Questions: 1. Brain atrophy: what’s the point until you actually figure out how to measure it accurately? How can you treat to something you can’t even measure? Until you can tell me in a clinical setting with accuracy and conviction that I’m losing brain atrophy, all I hear is blah blah blah. My brain atrophy MRI showed I had normal atrophy levels for people my age. Then my neuro told me it’s all a bit useless and some of it depends on how much water I drank and I ended up rolling my eyes. 2. I had an induction treatment 12 months ago. If I am brave enough and have a LP to measure my spinal fluid neurofilments and they are found to still be there (as I suspect they would): what would your recommendation to me be in terms of future treatment?How is the Brexit “depression” going (speaking of viewing the world through rose tinted glasses)?An Australian person with MS killed herself this week, she was well known on the various MS support forums. She was in her 50s. So sad.

    1. Yes sad.If you are brave and have neurofilaments then you need another course of the induction therapy if you are not depleting then you need to think about switching

  3. My humble opinion, very humble, is that we should focus on early detection. We can do so much for most people. If we catch people earlier then the outcome is better. That will free up resources to fight PPMS. Some ideas, may be mad and rambling:1 thickness of optic near and delta between two eyes2 focus time like the automated eye test 3 target acquisition for a displayed image All done with a pair of goggles. Record performance over a period of time between visits to obgyn

  4. Hello ProfG, thank you for sharing this very personal story. In spite of all the challenges your father still managed to raise you well. Do you have any parenting suggestions for pwMS? How can we make sure our children are not burdened by this disease and instead learn valuable life lessons from the experience?

    1. We have a project called Digesting Science that aims to teach children about the science behind MS so they can then understand some of the symptoms they might see in their parents: We run it as regular educational events in London but now have kits that we can send out to other centres. If you want to get in touch with us bookings (at) then I can let you know if there's an event being arranged in your area for 2017

  5. Thank you for the personal, emotional and inspiring post profG. Better days will come and science will always progress. Sometimes the progression will be slower, sometimes faster.Hope and optimism are essential elements for any improvement.

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