Friday, 16 June 2017

Progress In Medical Research. Week 2, Scleroderma Awareness Month 2017. Raynaud's, Autoimmune Rare Disease. Patient Profiles.

Progress In Medical Research

Week 2: Scleroderma Awareness Month 2017

Raynaud's, Autoimmune Rare Disease. Patient Profiles 

Progress In Medical Research

Week 2: Scleroderma Awareness Month 2017

Raynaud's, Autoimmune Rare Disease. Patient Profiles 

Progress In Medical Research

This year, further to the theme of Rare Disease Day 2017 being ‘Research, where possibilities are limitless’, I made the focus of my Scleroderma Awareness month campaign, ‘Research’, combining with the current unmet patient clinical needs, for the scleroderma and Raynaud’s rare disease patient.   

By giving the campaign a global approach, a best practice guide for treatment and management can be devised.   

To read more about why I chose ‘Patient Profiling and Research’ as the focus of my 2017 campaign, Click here      

As well as, 'Why Research and Patient Profiles? Scleroderma Awareness Month 2017, Raynaud's, Rare Autoimmune Disease, Click here     

In this article, I intend to summise some of the common symptoms experienced by the patients featured within the profile campaign so far, as well as, provide a summary as to the current research approach taken, towards improving the scientific understanding as to the biological processes involved in the disease process.    

I am extremely grateful and very much humbled by the stories of the patients who have taken part in the 2017 Scleroderma Awareness campaign. I did not envisage the trip down memory / nightmare lane, back to my first 7 years after having been told those three words, ‘You have scleroderma’, which this experience revoked. 

As, at this time, my symptoms were out of control, attacking my body with a vengeance.     

Read more about my initial diagnosis, here    

I thought that I was all cried out from the scleroderma ‘not so fun bus’ years ago, but this experience reopened the floodgates and proved that my Sjogren’s overlap can be overcome on occasions!   

This year marks my 20th anniversary from first being told those three life changing words, followed with ‘a 15 month prognosis’.   

Every day, I remain in a state of awe that I have defied my diagnosing doctor’s expectations.   

I am convinced that I am still here today, to be the scleroderma parrot which I have become, due to the expertise of the specialised Scleroderma Unit at the Royal Free Hospital. 

In particular, the superhuman beings, Dame Prof Carol Black and Prof Chris Denton. I attended here, December 1998, having been first diagnosed in September 1997.   

I am now 13 years chemo / immunosuppressant free, with my skin having reversed to normal healthy skin. 

The only medication which I take is bosentan 125mg twice a day, in an attempt to reduce my digital ulcers. I have seen a marked improvement in my hands since taking this medication. 

I was honoured to be a member of the medical team who devised the NHS England Guidelines for treating digital ulcers in scleroderma, published in 2015. 



Every patient who has been featured in the campaign so far, have all referred to digital ulcers and Raynaud’s. 

As well as, all patients have included their current reality in relation to treatment and management. 

Most patients take a cocktail of numerous cytotoxic medications, and have to attend so many medical appointments, it is their full time job.  

I have written about the full time job of managing the symptoms, Click here    

In relation to the cause of scleroderma, due to the disease belonging to the autoimmune family, research understanding is focussed around the immune response with various cytokines (chemical substances) acting as biomarkers. 

So, in essence, the higher the level of the biomarker, the more aggressive / active the disease. 

Further studies are required to confirm this understanding more, as well as, to identify all bio markers involved.   

This approach is very encouraging for determining a speedy timely diagnosis, in the hope to prevent any life-threatening damage, whilst keeping treatments options still available.   

Clearly, there is much room for improvement in relation to the treatments currently used for managing the disease. The treatments are only acting on symptom suppression, at best.  

Last year, the paper ‘Emerging strategies for treatment of systemic sclerosis’ was published in the Journal of Scleroderma and Related Disorders, Vol. 1 Issue 2, May – Aug 2016, pp177 – 240. 

This paper includes all of the current differing approaches being investigated, in trying to understand the immune response involved in the scleroderma process. 

Access to this paper is currently free, Click here

Read my synopsis of the paper, Click here    

Infographic of pathways involved, Click here


At the annual Scleroderma Family day held last month, at the Scleroderma Unit, The Royal Free hospital, London, Dr. Corrado Campochiaro, Honorary Clinical Fellow in Rheumatology, gave a presentation, ‘Personalised medicine approaches in Scleroderma’, where he detailed current understanding as to the biological markers which are prevalent for a diagnosis, along with gene expression for such.

The 5th Systemic Sclerosis World Congress, is due to take place Feb 15 – 17 2018, in Bordeaux, France next year. 

This bi-annual event is the hub of the most up to date understanding of the entire scleroderma disease process.   

Overall, an improvement in the medical understanding as to the nature of the disease has improved immensely over the course of the last decade. 

However, due to the multi complex body systems involved in the disease process, research still falls way behind with providing an understanding as to the cause.  

To read more about why I chose ‘Patient Profiling and Research’ as the focus of my 2017 campaign, Click here    

Why Research and Patient Profiles? Scleroderma Awareness Month 2017, Raynaud's, Rare Autoimmune Disease, Click here   

2017 INDEX Scleroderma Awareness Campaign Patient Profiles, Click here    


INDEX to Scleroderma Awareness 2016 Campaign, Click here     




An edited version of this article was published here, in my Column with Scleroderma News. 
June 2017. 

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   

Becoming a Patient Research Ambassador for the NIHR, Click here  

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


Diet and nutrition, 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  
  


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. No wages or admin costs. Thank You.
#HOPE 


 

Last Update: April 2018


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