Sunday, 11 December 2016

What Companies have to go through

Saida T, Kira JI, Kishida S, Yamamura T, Ohtsuka N, Ling Y, Torii S, Lucas N, Kuesters G, Steiner D, Tibung JT; Natalizumab Trial Principal Investigators.Safety and Efficacy of Natalizumab in Japanese Patients with Relapsing-Remitting Multiple Sclerosis: Open-Label Extension Study of a Phase 2 Trial. Neurol Ther. 2016. [Epub ahead of print]

INTRODUCTION:The efficacy of natalizumab was evaluated in Japanese patients with relapsing-remitting multiple sclerosis (RRMS) in a 24-week, phase 2 bridging study. An open-label, 2-year extension study from this trial was conducted to assess the safety and efficacy of natalizumab treatment in Japanese patients.
METHODS: A total of 97 patients (43 previously on placebo; 54 previously on natalizumab) who had completed the bridging study were treated with 300 mg natalizumab every 4 weeks. Multiple sclerosis relapses, changes in Expanded Disability Status Scale (EDSS) scores, and adverse events were assessed at regular intervals. Anti-natalizumab and anti-JC virus (JCV) antibodies were measured.
RESULTS:After 2 years of natalizumab treatment, the mean adjusted annualized relapse rate was 0.30 (95% confidence interval [CI]: 0.18-0.52) among previously-on-placebo patients and 0.13 (95% CI: 0.05-0.29) among previously-on-natalizumab patients. The mean change in EDSS score from baseline to week 120 was -0.03 among previously-on-placebo patients and -0.18 among previously-on-natalizumab patients. In both groups, >90% of patients experienced ≥1 adverse event. Two previously-on-placebo patients developed persistently positive anti-natalizumab antibodies. Approximately 65% of all patients tested positive for anti-JCV antibodies at open-label treatment initiation. No deaths or progressive multifocal leukoencephalopathy cases were reported.
CONCLUSIONS:The efficacy and safety findings from this 2-year open-label extension study are comparable to and confirm the results of other clinical trials of natalizumab conducted in non-Asian patient populations, and provide longer-term evidence of efficacy and safety in Japanese patients.

This is nothing new and it is is an extension of phase II trial but why over ten years after natalizumab trial are companies still doing trials? In this case it is people from Japan because they are so different from the rest of the planet....

Advent Calendar 11

Saturday, 10 December 2016

#BeHuman: humbug

For all the Humbuggers out their we are starting a story board about living with MS at Christmas. #BeHuman

Somebody let us know yesterday what the true meaning of humbug is. 'Scrooge uses humbug to describe people who speak of love and charity but are being insincere. It is a pretence of the goodwill of the festive season and Scrooge does not want to be a part of the pretence'

At a work drinks party last night we were debating whether or not it was appropriate for the MS Society to use fictionalised stories about people with MS at Christmas to raise money. What do you think? I am sure you have enough of your own stories to fill a book. 

We are always touched by the personal stories you as a community are prepared to share with us; the following are three examples from yesterday.

...... "I've been indoors for 3 weeks because of a bad exacerbation. But I have a loving husband and son caring for me. I can walk somewhat, not drive yet. There are so many others who aren't as blessed as me, who need our love and care and, of course, money to help toward that."....

...... "My aunt was bedridden with MS by 50, and could only move her head by 55. She lived like that until she was 83. She didn't want to die though, she loved seeing her family and watching old films. I would love her strength and will."......

...... "My grandmother died from breast cancer in 2012 because she was too embarrassed to tell anyone. The doctors, who were limited in what they could after she presented to them finally, were very kind and caring, to both her and my dad.

Before my partner was formally diagnosed with breast cancer, her oncologist had told her that it was a sweat gland (atypical presentation and she was too young). When it became evident that it was cancer, his guilt and quick action were equally obvious and saved her life (the aggressive cancer hadn't spread during the delay and from the date on diagnosis, she was operated on in the public system literally the next day).
I can't imagine what life would have been like if the end to that story was like my grandmother's.

We are all shaped by our experiences. I agree, let's not forget humanity as we sing. Try and find some time away from MS too, for the good of yourself and MS.".....

Can I suggest starting a story board about how you experience Christmas? It will help make the community aware of the issues that arise from living with MS and more importantly it will help the MS Society with their STOP-MS fund raising campaign.

What if Santa... had MS?

Dr Mao Zhifeng, MD, PhD

Mao has just joined BartsMS as an ECTRIMS Clinical Training Fellow to DrK. He has undertaken research in the field of neuroimmunology since 2008 when he started his MSc at the Guangxi Medical University, China. After obtaining his MSc in 2010, he spent three years as a specialist registrar in Neurology.  He then continued his research interest in demyelinating diseases with a PhD (awarded in June 2016) at the Multiple Sclerosis Center of the 3rd affiliated Hospital of Sun Yat-Sen University. Mao is keen to learn how to undertake clinical trials of disease modifying drugs in MS, particularly in people with advanced MS, and will support our #ThinkHand campaign.  He also wants to learn more about service and pathway development for people with MS who are often marginalised simply because of the lower prevalence of MS in lower and middle income countries. Mao plans to stay with us until November 2017. 

This is his first BartsMS blog post.

"Imagine Dad explaining to his daughter that Father Christmas is no longer able to deliver toys on Christmas Eve as he sufferers from multiple sclerosis. That's the concept of a recent campaign by Alzheimer’sResearch UK where Santa is living with the effects of dementia.

Dementia is a syndrome with many potentially underlying diseases, of which cerebral "small vessel disease” (SVD) is one of the commonest. As we are – hopefully – contributing to the longevity of people with MS, vascular comorbidity becomes ever more relevant, and much of the MS Brain Health campaign is just as applicable to people with vascular risk factors.

This recent review is quite timely in that it highlights (i) pathological similarities between MS and SVD, (ii) the contribution of SVD to the overall outcome and well-being of pwMS, (iii) the chronic inflammatory milieu in MS that may render the brain more vulnerable for SVD, and (iv) drugs that target the microvasculature which may have effects beneficial on the overall outcome in MS."

Multiple sclerosis (MS) is a chronic inflammatory demyelinating disorder of the central nervous system wherein, after an initial phase of transient neurological defects, slow neurological deterioration due to progressive neuronal loss ensues. Age is a major determinant of MS progression onset and disability. Over the past years, several mechanisms have been proposed to explain the key drivers of neurodegeneration and disability accumulation in MS. However, the effect of commonly encountered age-related cerebral vessel disease, namely small vessel disease (SVD), has been largely neglected and constitutes the aim of this review. SVD shares some features with MS, i.e. white matter demyelination and brain atrophy, and has been shown to contribute to the neuronal damage seen in vascular cognitive impairment. Several lines of evidence suggest that an interaction between MS and SVD may influence MS-related neurodegeneration. SVD may contribute to hypoperfusion, reduced vascular reactivity and tissue hypoxia, features seen in MS. Venule and endothelium abnormalities have been documented in MS but the role of arterioles and of other neurovascular unit structures, such as the pericyte, have not been explored. Vascular risk factors (VRF) have recently been associated with faster progression in MS though the mechanisms are unclear since very few studies have addressed the impact of VRF and SVD on MS imaging and pathology outcomes. Therapeutic agents targeting the microvasculature and the neurovascular unit may impact both SVD and MS and may benefit patients with dual pathology.

Advent Calendar 10