ALS awareness month

What did we get up to, and what's next?

ALS awareness month has finally come to a close, and what a month it was. The purpose was to raise awareness and funds for research for a cure for ALS (amyotrophic lateral sclerosis) – a complex, progressive neurodegenerative disorder that attacks certain cells in the brain and spinal cord necessary for muscle movement.

The ALS Therapy Development Institute(ALS TDI) chose the theme ‘In three words’ – encouraging people to share their thoughts, hopes and feelings about ALS with the online community. Our team also took part (see below).

Busy bees

Things really took off when for the first time in 22 years, the US’ FDA (Federal Food and Drug Administration agency) approved a drug called Radicava (edaravone)for the treatment of  ALS (also known as Lou Gehrig’s disease). We walked into our office on a Monday morning to find things in a state of (very positive) flurry; emails and phones were flooded with messages from patients around the world asking whether we could help them to import edaravone (sold under the name of Radicu tin Japan). The news had made headlines; now everyone was aware of this ALS treatment, which had already been approved by Japan’s regulatory agency in 2015, and which we’d already been assisting patients and their specialists in accessing for months.

Based on requests that we’ve received lately, the FDA’s stamp of approval seems to have boosted the confidence of GPs and neurologists worldwide to prescribe edaravone to their patients – some of whom have been trying to access it for a while. (All patients require a doctor’s prescription to ordera drug through us.)

Buzzing all around

We’ve been absolutely delighted to be able to deliver Radicutto a record amount of patients in these recent days. The excitement of patients we’ve been in contact with when they realised that they could access this drug straight away (and at a much lower cost than the current projected US price) was palpable:

“It seems too good to believe that we could have this drug in two weeks...I hope you know how needed you are…[helping us in] obtaining needed drugs in a timely way. Navigating the treacherous waters of red tape and trials seems so incredibly cruel when the possibility of slowing the progression of this awful disease hangs in the balance.”

It was an absolute joy to see patients finally accessing this medicine after so long:

“My father took the first dose yesterday and we are all very pleased with that...I’m ready to spread the word [to all] patients in need [of this] medicine that cannot be easily reached.”

It’s messages like these that keep us going, because that’s why we started everyone.org – to help patients navigate the often complex and confusing sea of licensing paperwork – getting the latest approved medicines to them in the comfort of their home country as soon as they’ve been approved by a well reputed regulatory body somewhere in the world – fast, pronto, rápidamente! It’s all about patients, and giving them direct access to the best treatment options out there.

It doesn’t stop here

ALS awareness month is over for another year, but our work has just begun. It’s been estimated that edaravone will be directly available in US pharmacies by August 2017; but even after then, there will still be thousands of ALS patients in Europe, Asia, Australia and beyond without direct access to this treatment.

Additionally, as a global community, building awareness about ALS and further research never stops. There are exciting developments in ALS research, from stem-based treatments to explorations of the genetic underpinnings of some forms of ALS; more and more companies are moving drugs from early stage studies to Phase 2 and 3 trials.

How long until we find a cure for ALS? We wish we knew. But one thing’s for sure – as soon as a cure is discovered and gets approved somewhere in the world, we’ll be working hard to ensure that every patient has a pathway to access it – as quickly as possible.

“I chose these three words because I believe in the power of togetherness, shared knowledge and collective intelligence. That’s what we need if we’re going to fight ALS globally. An open mind and open innovation.” – Sjaak, Co-founder.

“ALS in an awful, unpredictable disease that affects people physically and psychologically. We need more funding for research...let’s find a cure for ALS!” – Katrin, COO

“ALS awareness month was such a success but it doesn’t stop there. We need to ensure that we continue to spread the word and raise awareness about ALS.” – Deirdre, Communications Manager

View more of our #inthreewords on our Instagram account).