Patient power transforming the treatment landscape

July 26, 2017

Traditionally patients were passive recipients of treatments for their ailments. With the advent of social media this landscape is slowly transforming as patients demand more and better treatments. A prime example of this is in cystic fibrosis, where the Cystic Fibrosis Trust are funding ground breaking research including a phase II patient clinical trials through its Venture and Innovation Award to Verona Pharma. Previously the Trust had funded Verona’s pre-clinical programme for this compound.


‘The Venture Innovation Awards are designed to ensure that the Trust’s funds go as far as possible to supporting ground-breaking treatments and innovative research by bringing in vital external funding. The RPL554 trial has the potential to make a difference to the lives of people with Cystic Fibrosis, and we are thrilled that the drug is moving into this important trial stage,” said Paula Sommer, head of research at the UK Cystic Fibrosis Trust. This means that patients are becoming more involved in directing and directly funding vital research into their conditions that have traditionally been under researched and are poorly understood.


Another example is in the area of ALS or motor neuron disease, a devastating neurological disease that often leads to death within 4 years. A diagnosis on par with cancer but one that has received a fraction of the research historically. No one knows what causes it or how to stop it. In 2014 the ice bucket challenge was harnessed by patients with ALS to highlight this situation. The ice bucket challenge went viral on social media during July – August 2014. 


Within weeks of the challenge going viral, The New York Times reported that the ALS Association had received $41.8 million in donations from more than 739,000 new donors from July 29 until August 21. This is translated to more than double the $19.4 million the association received during the year that ended January 31, 2013. On August 29, the ALS Association announced that their total donations since July 29 had exceeded $100 million.


This funding allowed the ALS Association to fully fund a number of research programs. One of these was Project MinE, a large data-driven initiative. The project’s researchers announced in July 2016 that they have identified a new gene associated with the disease, which experts say could lead to new treatment possibilities. Project MinE has been working to sequence the genomes of 15,000 people with the disease, and the discovery, which was described in a paper published in the journal Nature Genetics, involved more than 80 researchers in 11 countries.


The discovery was significant, “because it helps us understand what’s triggering this and can help us better find a treatment.” Said Brian Frederick, executive vice-president of communications and development at the ALS Association.


In this digital era, healthcare landscape changes rapidly, placing the patients at the centre of attention and shaping the clinical trial development according to their needs. This simply means that the days of patients sitting passively as their conditions get worse seem to be over and that can only be a good thing.

[Sources: The New York Times: ‘Ice bucket challenge donations for A.L.S. research top $41 million’


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