All is not well in the relationship between the world’s leading MND Association and MND advocates and sufferers in the US. MND is known as Amyotrophic Lateral Sclerosis (ALS) in the US. The ALS Association represents approximately 30,000 people with ALS (pALS). The ALS Association raised $115 million in 2014 from the Ice Bucket Challenge (IBC) that went viral worldwide and has continued to raise substantial funds for the ALS Association compared with the period before 2014.
Enter a startup biotech company, Brainstorm Cell Therapeutics, that owns rights to a treatment, known as NurOwn, demonstrated to be safe and has shown signs of efficacy. NurOwn is currently in its final phase of clinical trials in the US. The clinical trials process and bringing a treatment to market is an expensive and risky business. It is unsurprising that Brainstorm approached the ALS Association for funding to advance its clinical trials. Unfortunately not only was funding, worth noting, not forthcoming despite the $115 million raised from the IBC but Brainstorm’s clinical trials were held up for a lengthy period. NurOwn may have already been available today.
Unsurprisingly, when it comes to MS, the National Multiple Sclerosis Society hasn’t made the same management or PR mistakes as the ALS Association. The National Multiple Sclerosis Society has made a grant to advance Brainstorm’s phase 2 clinical trial in MS. To date there is no real support for either Brainstorm or NurOwn from the ALS Association or its management. There is the occasional statement by the ALS Association aimed at reputation management to limit the damage done from its failure to meaningfully support NurOwn, which may be seen by some as ‘NurOwn-washing’. This inaction is interpreted by some pALS as a failure by the ALS Association to take decisive action to aid the effort to cure the disease.
The void created has allowed organisations such as I AM ALS [www.iamals.org / @iamalsorg] to emerge as the voice of pALS driving forward advocacy for treatments and legislation to ensure access to new drugs from the increasing pipeline going through FDA approved clinical trials in the US.
How could the above potentially affect MND sufferers in the UK?
The ALS Association is at the head of the international ALS MND Alliance. The ALS MND Alliance mission statement is listed below.
‘Our two main focuses are Community and Capability and we can do this as we build our Capacity.'
The ALS MND Alliance board members are listed on the below link and include MND Association.
https://www.alsmndalliance.org/about-us/board-of-directors/
The MND Association provides tremendous work in fulfilling its mission statement listed below.
‘We improve care and support for people with MND, their families and carers.’
While not listed in the above remit, in 2020 it would be even better if the MND Association could advocate for a review and the introduction of treatments that may allow many MND sufferers to live until NurOwn and other effective drugs are approved. The MND Association could perhaps do more in this respect, if not held back by its association with the international ALS MND Alliance and the ALS Association’s past failures to get behind NurOwn, a lifesaving treatment that may be the first to be approved by the FDA for treatment of MND.
It is unlikely that the MND Association would oppose or hinder attempts to introduce treatment with Bone Marrow Mesenchymal Stem Cells (BMMSCs) or NurOwn in the UK, simply as the basic BMMSCs procedure is already available in many countries and the list of countries providing it is growing. Sooner or later it may become apparent that there has been a failure by not ensuring that such treatments were reviewed or made available earlier in the UK. However, what many MND sufferers in the UK would like the MND Association to do is actively advocate for such treatments and coordinate with MHRA/NICE, NHS and treatment centres to ensure early access to such treatments. Early, taking into account the time MND sufferers have left to live.
The message to the MND Association is to actively work without further delay for the introduction of such treatments this year. If the MND Association can take up this challenge, its management will be said to truly have 2020 vision.
Happy New Year.
Please see the below links for the letter from Brainstorm Cell Therapeutics to the ALS Association.