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 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.

To read my articles: 


Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here   

March Autoimmune Disease Awareness Month 2018, Click here   

Global patient video, Click here      

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

Becoming a Patient Research Ambassador for the NIHR, Click here  


Scleroderma Awareness Month 2017 Patient Profiles Campaign, Click here     
 
Scleroderma Awareness Month 2017 Patient Profiles Campaign Patient Index, Click here        
The Pandora’s Box of the rare autoimmune disease Scleroderma, Raynaud's and Cancer, Click here



Key Event Dates 2018, Click here     

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       


Raynaud's, Click here    



World Scleroderma Day 29th June 2016, Click here

World Scleroderma Day 29th June 2017, Click here   


Why Global Collaboration is important to the Rare Disease Patient, Click here      



RARE DISEASE DAY:
The theme to this years Rare Disease Day is Research

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

2016 Rare Disease Day Patient Voice
2016 Rare Disease UK Parliamentary Reception 

2017 Rare Disease Day Flashback  

More unmet clinical needs  

Rare Disease Day 2016



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. 

I highlighted other areas of medical research interest within Week 3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017. 

VIDEO



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: 

Early Diagnosis
Expert Specialist Centres
Access to Innovative Medicines

Where MEDICAL RESEARCH investment is VITAL.

Research is the key. Abstracts from 2016 World Congress, Click here


Scleroderma Awareness Month Campaign 2016, Click here

Links

View video, here   

Preamble - here



The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19th May 2018. Browse the program, here

This year, I am celebrating 20 years of being a patient at this world leading expert specialist research centre. 

Sept 2017


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.
I am truly humbled and inspired by their work ethic. 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 diagnosing doctor, that I was looking at a 15month prognosis. 

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.
  
For latest updates follow: 

Facebook Page:

Twitter: @SclerodermaRF  @RaynaudsRf  


#SclerodermaFreeWorld           #RaynaudsFreeWorld
#ADAM #Scleroderma
#Raynauds
#RareDisease  

Living the dream, scleroderma style.  


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



100% of your monies will be used for medical research purposes only. Thank You. 
#HOPE 


 

Last Update: April 2018




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