What do you expect from an effective DMT in progressive MS?

In clinic yesterday I was discussing the natalizumab SPMS trial with a SPMSer. It dawned on the MSer, after some discussion, that even if the trial was positive and they were randomised to active agent they would probably not notice any discernible effects at a personal or individual level. The reason for this is that we, the clinicians, don’t expect an effective drug for progressive MS to stop disease progression but to merely slow the rate at which SPMS (or PPMS) progresses. In other words if you were progressing a rate X before the drug you now progress at rate Y, which is simply a slower rate of progression than before. If this was the case you would have no indication the drug was necessarily working. Of course we would like the drug to stop disease progression all together, i.e. stabilise your disease, or hopefully improve your neurological functioning, but this seems unlikely based on observations from other progressive trials and in animal models of SPMS. I have already discussed this issue in some detail before on this blog (see previous survey), but would like to revisit it as I am not sure how to best communicate this information to potential trial participants in a clinical situation. I don't want to MSers volunteering for progressive trials to have unrealistic expectations.

Any ideas or suggestions would be helpful?


Other posts of interest on progressive disease


27 Apr 2012
Survey results: neuroprotection. "I am reassured that 55% of MSers expect and effective neuroprotective therapy to stabilise of improve their disability progression; this is a realistic expectation and something that may be ...

01 Apr 2012
A few weeks back one of the readers posted this comment: "Sick of conferences like MS Life. It's pure spin on the scientists' part. Firstly, how about you introduce truly efficacious therapies for progressive MS and then you'll ...
18 Mar 2012
I also spoke to them about the need for better trial designs for progressive MS and described the study design we are promoting using frequent lumbar punctures and neurofilament levels as an outcome measure. They were ...
International Collaborative for Progressive MS - Multiple Sclerosis ...
24 Aug 2012
Despite significant progress in the development of therapies for relapsing MS, progressive MS remains comparatively disappointing. Our objective, in this paper, is to review the current challenges in developing therapies for ...
17 Aug 2012
Development of protein biomarkers in cerebrospinal fluid for secondary progressive multiple sclerosis using selected reaction monitoring mass spectrometry (SRM-MS). Clin Proteomics. 2012;9(1):9. "The methods of this study ...
15 Aug 2012
Objective: The goal of this study is to assess the safety, therapeutic efficacy and mechanism of action of idebenone in primary-progressive multiple sclerosis (PP-MS) patients. Study Population: Adult, untreated patients with ...
19 Jul 2012
Giovannoni. Primary progressive MS. ACNR 2012;12(3):9-12. Although PPMS is relatively uncommon it remains a significant clinical problem both diagnostically and therapeutically. PPMS is almost certainly part of the MS ...

07 Jul 2012
Our strategic plan 2012-2016 identifies progressive MS as one of the three key priority areas for MSIF's international MS research going forward (along with paediatric MS and stem cells). This important research area has also ...
21 Jul 2012
But perhaps we should also introduce the Clinical Trials Network (CTN), which was set up specifically for Progressive MSers. This has been reported in some places but may not be common knowledge. As you know all too ...
28 Aug 2012
We compared OB+ versus OB- patients regarding progression to expanded disability status scale (EDSS) of 6.0 and to secondary progressive MS (SPMS). Cox proportional hazard models were used to compare the outcome ...
16 Jun 2012
BACKGROUND: Treatment options for MSers suffering from progressive forms of MS remain inadequate. Mast cells actively participate in the pathogenesis of MS, in part because they release large amounts of various ...
29 May 2012
Participants and treatments: 493 MSers with primary or secondary progressive MS were recruited to the study from 27 centres across UK between May 2006 and July 2008. It was a requirement for participants entering the trial ...
30 Jun 2012
Background:It is unclear if all patients with relapsing-remitting multiple sclerosis (RRMS) ultimately develop progressive MS. Onset of progressive disease course seems to be age- rather than disease duration-dependent.
13 Jul 2012
Thus, to develop safe therapeutic approaches for patients with progressive MS, it is essential to elucidate how mitoxantrone exerts it benefits. Accordingly, we initiated a prospective single-arm open-label study with 19 ...
16 Feb 2012
Challenging the dogma (1): progressive MS and remyelination. In response to a comment concerning dogma in the field of MS, I am going to publish a series of posts that are designed to "challenge the dogma". Dogma: ...

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