Wednesday, 5 April 2017

The Role Of Medical Research - Scleroderma, Raynaud's, Autoimmune Rare Disease Patient, JSRD Vol 1 (2)

The Role of Medical Research 
Scleroderma, Raynaud's, Autoimmune Rare Disease. 

#SclerodermaFreeWorld  #RaynaudsFreeWorld 

 blog.raynaudsscleroderma.co.uk  






The role of medical research is of huge importance to the Raynuad’s and scleroderma patient, and to the rare disease patient, in general. 

There is currently no cure for scleroderma, as well as, the causative mechanisms still remain a mystery, albeit, the understanding of the cause and cure, is accelerating faster than ever before.

Investment in medical research provides immense HOPE to the scleroderma patient, which, when living with an incurable diagnosis, is the next best thing to the cause and cure being discovered.

Hope has been my constant companion of the last 19 years, along with the inspiration and comfort taken from being a patient of one of the world’s leading scleroderma pioneer medics, Prof Chris Denton.

At the 4th Systemic Sclerosis World Congress held in February 2016, in Lisbon- The Journal of Scleroderma and Related Disorders (JSRD) was launched. 

The JSRD is dedicated solely to scleroderma and its overlap disorders. 

The presence of this respected medical journal on the world stage provides hope to patients, by way of showing the current ongoing, actual medical research results and investment into such. 

Including, the approaches being utilised to endeavour to shine the desperately needed light on improving scleroderma patient treatments and care. 

I discussed more about The Journal and what this means to patients, here 

I also discussed ‘Why Global Collaboration is Important’, here   



Volume 1 Number 2, May - August 2016 JSRD includes the following contents:

Reviews
Systemic sclerosis trial design moving forward
Sindhu R. Johnson, Dinesh Khanna, Yannick Allanore, Marco Matucci-Cerinic, Daniel E. Furst
Extracorporeal shock wave therapy for digital ulcers associated with systemic sclerosis
Shinichiro Saito, Tomonori Ishii, Kenta Ito, Hiroaki Shimokawa
Emerging strategies for treatment of systemic sclerosis
Dinesh Khanna, Jorg H.W.Distler, Peter Sandner, Oliver Distler
Systemic sclerosis-related calcinosis
Ariane L. Herrick, Andrzej Gallas

Original Research Articles
Gender differences in systemic sclerosis: relationship to clinical features, serologic status and outcomes
Christine Peoples, Thomas A.Medsger Jr., Mary Lucas, bedda L. Rosario, Carol A. Feghali-Bostwick
Potential beneficial role for endothelin in scleroderma vasculopathy: inhibition of endothelial apoptosis by type B endothelin-receptor signalling
Yongqing Wang, Nezam Altorok, Bashar Kahaleh
Effect of endothelin-1 receptor antagonists on skin fibrosis in scleroderma patients from the EUSTAR database
Suzana Jordan, Jorg Distler, Britta Maurer, Ulrich A. Walker, Doerte Huscher, Yannick Allanore, Gabriela Riemekasten, Oliver Distler on behalf of EUSTAR
A randomised controlled trial of acupressure for the treatment of Raynaud’s phenomenon: the difficulty of conducting a trial in Raynaud’s phenomenon
Heather Gladue, Veronica Berrocal, Richard Harris, Pei-Suen Tsou, Gautam Edhayan, Ray Ohara, Dinesh Khanna
Mycophenolate mofetil following cyclophosphamide in worsening systemic sclerosis-associated interstitial lung disease
David Launay, Anne-Laure Buchdahl, Alice Berezne, Pierre-Yves Hatron, Eric Hachulla, Luc Mouthon

To read my blog post synopsis on ‘A randomised controlled trial of acupressure for the treatment of Raynaud’s phenomenon: the difficulty of conducting a trial in Raynaud’s phenomenon’, authors, Heather Gladue, Veronica Berrocal, Richard Harris, Pei-Suen Tsou, Gautam Edhayan, Ray Ohara, Dinesh Khanna, Click here

 
Dr Maureen Mayes, Professor at University of Texas McGovern School of Medicine, co-founder of the World Scleroderma Foundation, and co-author of ‘The Scleroderma Book’ (3 Aug 2015 publication date), states: 

‘A gene on the X chromosome has been identified as associated with scleroderma… 

To date there are 3 genes associated with scleroderma susceptibility that are located on the X chromosome - IRAK1, FOXP3, MECP2. IRAK1, FOXP3, are vital for T cell function and MECP2 is associated with DNA methylation’.  
 

This is most certainly a fantastic advancement in the understanding of the scleroderma process. 

And certainly, extremely thought provoking, as the statistics show the incidence of scleroderma to be higher in females than males.

During my 19 year patient experience, I have taken part in numerous clinical trials for medical research. 

I see this participatory opportunity as a duty, as well as a pleasure. 

A duty, as I hope to be able to help future scleroderma generations by utilising my body for medical research. 

I have written more about Taking Part in Clinical Trials, here
 
I have written about the importance of Expert Centres, here 

I am extremely grateful to all of those scleroderma patients who assisted in medical research which has enabled the long term outlook of living with a scleroderma diagnosis more favourable than ever before, in the event that an early diagnosis has prevented irreversible internal damage.

In my view, to take part in a clinical trial is a fantastic opportunity, to not only have the possibility of taking a potential improved treatment for scleroderma, which has been under years of scrutiny prior to even making it to the clinical trial arena for humans, but also, to have a thorough assessment and examination with novel techniques and experimental processes which may not be included in the normal course of events for purposes of a regular standard monitoring appointment.

There are currently numerous clinical trials taking place in scleroderma all over the world. A few examples include:


2. The Scleroderma Patient Centered Intervention Network (SPIN). 

I have participated in SPIN questionnaires online, for a few years now, for purposes of research. I think that the ongoing SPIN research is a fantastic tool for providing understanding into the holistic approach to treating and responding to scleroderma. 

If you would also like to participate in the SPIN study, please email me: nicola.whitehill@hotmail.co.uk 

3. The Royal Free Hospital in London is currently recruiting participants to look into the clotting factors in scleroderma patients, Click here

All in all, an investment in global medical research is essential for the scleroderma patient. 

The time taken for the newer biologic innovative medicines to reach the market place needs to be reduced, as does the access to these medicines, which have shown to have a remarkable improvement in disease progression and severity.  

Time is a luxury for the scleroderma patient and the rare disease patient alike. 

Medical research is making improvements, however, patients are quite literally ‘dying for a cure’. 

I am grateful that I am still around to be a scleroderma patient, in the hope that, investment and commitment to accelerate medical research will instigate the light bulb moment needed for a #SclerodermaFreeWorld’ and #RaynaudsFreeWorld' – imagine that!  
And, in the meantime I will continue to ‘Live the dream, Scleroderma Style’, with Hope, remaining as my best friend and companion.




An edited version of this article was published here, in my Column with Scleroderma News.  
September 2016.     


 

 



  

Scleroderma Family Day 2020

25th Anniversary Meeting

The Atrium, Royal Free Hospital, London, NW3 2QG

Chair: Professor Chris Denton

PROVISIONAL PROGRAMME


09.30 – 10.00           Registration and Coffee

10.00 – 10.20           Welcome                                                   Dame Carol Black
                                                                Prof Chris Denton & David Abraham

10.20 – 10.40           What is a Biopsy?                                    Dr Kristina Clark

10.40 – 11.05           Dental aspects of Scleroderma            Prof Stephen Porter

11.05 – 11.25           Gastrointestinal problems –                       Dr Fiza Ahmed
shedding new light on old problems 

11.25 – 11.50           Pulmonary hypertension and the heart Dr Gerry Coghlan

11.50 – 12.15           Scleroderma cohort studies –                  Dr Francesco 
                                 ‘Learning from our patients’                     del Galdo

12.15 – 14.15           LUNCH BREAK – see below  

14.15 – 14.45           25 years of progress –                          Prof Chris Denton
from ‘black box’ to ‘positive trials’

14.45 – 15.15           International speaker –                            Dr Madelon Vonk
                                 Scleroderma Management in Netherlands

15.30                        Raffle


Lunchtime Discussions Groups / Demonstrations include:

Clinical Trials                                                                  Rachel Ochiel and team
National Institute of Health Research                            Christine Menzies
Drug Information / monitoring                                        Pharmacy
Massage                                                                         Keith Hunt MBE
Pulmonary Hypertension Education / nursing            Sally Reddecliffe/Adele Dawson
Rheumatology Laboratory                                          Research Laboratory Staff
Scleroderma Education / Nursing                          Louise Parker/ Joseph Cainap
Thermography                                                                Dr Kevin Howell
SRUK                                                                             Ollie Scott
Sjogren’s syndrome                                                       British Sjogren’s Society 


To Read My Articles:  

Gift in My Will, Click here 

Planning for the Future, Click here  

 


Rare Disease Day:   

Rare Disease Day 2020:  

  

Listen to my interview with John Smeeton, (Silver Fox), from the Royal Free Radio, here  


 

Raise awareness and donate to medical research, order your #SclerodermaFreeWorld #RaynaudsFreeWorld tshirt  here   


 
Rare Disease Day 2020: NIHR 'Research gives hope to Rare Disease patients', Click here  

  


 
Rare Disease Day 2019: Leaving a Legacy Gift, Click here       



Rare Disease Day 2019: 11am 97.4RockFM headlines, NO CURE, Click here  

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here     

2017 Rare Disease Day Medical Research  


2016 Rare Disease Day Patient Voice   

2016 Rare Disease UK Parliamentary Reception       

  

Rare Disease Day is a fantastic opportunity for the entire rare disease community to shine a spotlight on their reality, combining as one unified voice. Where, at least one commonality presides –  
Medical Research
provides the brightest light,
for the illumination of 
the rare disease patients’ plight.


To read about medical research at the scleroderma unit, Click here   

Although rare disease patients are few in number, eg. 2.5 million scleroderma patients worldwide, (the World Scleroderma Foundation), the commonalities and golden hallmark for each rare disease patient are the same overall. 

For optimum patient care, 3 hallmarks preside:







supported by a medicaldream team’ 
(expert specialist as the clinical lead).

INVESTMENT in MEDICAL RESEARCH is CRUCIAL.   


To read my articles:  

Celebrating 20 years of being a patient at the Scleroderma Unit, Click here

2019 New Challenges, Click here
 
NIHR Video: 'My Experience of Clinical Trials', Click here 

If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here   

The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here  

2018 Scleroderma Awareness Raising and Medical Research, Click here    


SCLERODERMA:

Importance of an early diagnosis, Click here   
    
Taking Part in Clinical Research Trials, Click here     

The Importance of a Multi Disciplinary Medical ‘Dream Team’, Click here    

Expert Specialist Centres, Click here      
  
My Skin is Cured from Scleroderma, Click here   
    
UK Guidelines for Managing and Treating Scleroderma, Click here        

Fatigue, Click here         

Mobility, Click here      

Diet and nutrition, Click here      


Raynaud's- How to Diagnose, Click here        

'All you need to know', 2016 Awareness Video, Click here    

Global Patient Profiles 2018 Video, Click here    


Unmet Medical Needs, Click here     




Calcinosis Video, Click here    


The scleroderma tooth fairy, Click here     


Skin Cancer and scleroderma, Click here   

 

Sept 2017

Prof Chris Denton and I, Sept 2017

June 2019



This year, 2020, I am celebrating 22 years of being a patient at the Scleroderma Unit, The Royal Free Hospital - a world leading expert specialist, research centre.  

Read more, here.    

I am eternally grateful to the global scleroderma trail blazers Dame Prof Black and Prof Chris Denton, whose commitment and dedication to unlocking the scleroderma enigma, is nothing other than, superhuman. Along with the Raynaud's world trail blazer, Dr Kevin Howell.  

I am truly humbled and inspired by their work ethic and commitment to their patients.  

I am wholly appreciative for Prof Denton’s continued medical expertise and support, especially during my barrister qualifying years, 1997 - 2004. 
 
1st March 2004, I qualified as a self employed practising barrister. Further to having been told in 1997, by my original diagnosing doctor, that I was looking at a 15month prognosis. 
 


  

Chat Magazine May 2019

I very much hope to utilise my professional skills and qualifications along with my patient experience, to help achieve the #SclerodermaFreeWorld dream, hoping to improve understanding and best practice, in the meantime. Read more, here    


  

World Scleroderma Day 2018, Click here  

World Scleroderma Day 2017, 29th June, Click here    

World Scleroderma Day 2016, 29th June. Origins of the date, Click here   

World Scleroderma Day 2015, 29th June.  


James Carver, myself, Prof Chris Denton

I had the pleasure of being a presenter and part of the European co-hort who gave a presentation at the European Parliament, Brussels, in honour of World Scleroderma Day 2015.  

I am immensely grateful to James Carver, former MEP, for organizing this event in memory of his late wife Carmen, who sadly passed from Scleroderma.  

To view the presentation, Click here 

Raynaud's

October:  
Raynaud's, Click here  
 
To view the UK treatment guidelines for Raynaud’s Phenomenon, Click here   

To view the EULAR treatment guidelines, Click here   

To view Thermograph Video, Click here    

To view Thermograph image, Click here    

   
My Raynaud’s reality, Click here     


The global Raynaud's trailblazer - Dr Howell and I, Sept 2017

To view Nailfold Capillaroscopy equipment (used to identify possible blood vessel damage), Click here   

Invisible Disability Awareness Video, Click here

To read My ‘Invisible Disability’ experience, Click here   

50 Shades of Blue, Click here



 

For latest updates follow / subscribe:  

@SclerodermaRF 

@RaynaudsRf 

Twitter, Instagram, Blogger, YouTube, Facebook Page:

#SclerodermaFreeWorld           #RaynaudsFreeWorld    #Research  



Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London.  


Alternatively, to make a direct donation to fund medical research via the Royal Free Charity, Scleroderma Unit, Click here    
 


100% of your monies will be used for medical research purposes only. NO wages or admin costs. Thank You. 

  

  
Last Update: Feb 2020.       



The Role of Medical Research 
Scleroderma, Raynaud's, Autoimmune Rare Disease. 

#SclerodermaFreeWorld  #RaynaudsFreeWorld 

 blog.raynaudsscleroderma.co.uk  









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