There are literally thousands of websites on the internet that make claims about amazing alternative or off-label treatments (AOTs) and even cures for MND but with little or no scientific evidence to back these claims up. This presents a real problem for people living with the disease who may want to try them. Are they safe? Will they help? Or are they going to cause more problems than they solve?
In 2009 Dr Richard Bedlack, Professor of Neurology at Duke University in the USA, founded ALSUntangled to develop a system of review for some of these treatments using the available evidence, to make it easier for people with MND and their families to make more informed decisions about them.Read More »
Dr Richard Bedlack is based at the Duke ALS Clinic in America. Back in 2009 he founded ALSUntangled and to start off MND Awareness week he writes about the group and what they’ve achieved in the past five years.
ALSUntangled started in 2009 as a method for helping people living with MND (commonly known as ALS in America) make more informed decisions about alternative and off-label treatments (AOTs) they were interested in. I wasn’t sure how it would be received by people living with MND or by other clinicians.
Unproven treatments are treatments that have not undergone rigorous testing for their safety and effectiveness by means of a clinical trial. Also, they are often not supported by any reliable evidence.
People or organisations offering unproven or ‘alternative’ treatments may suggest that they will work better than riluzole. They often claim they can cure MND or significantly slow disease progression. These remarkable claims are not supported by any scientific evidence.
The MND Association co-funds an international group of scientists and clinicians, collectively known as ALS Untangled to investigate unproven or alternative treatments for MND. This group look for the evidence behind these unproven treatments for people living with MND concluding with their own recommendations.
These reviews are freely available and are published Open Access. You can download the most recent review on propofol here.
“Thank you for your support of ALSuntangled! Dr Bedlack et al. are doing wonderful work on behalf of people living with MND everywhere!” – Doug Hetzel, USA
A neurologist’s point of view
Association-funded researcher Prof Ammar Al-Chalabi explains his views on unproven treatments:
Many people with MND seek out unproven treatments because of the very difficult situation they and their families are in. My advice is that this is generally OK if the treatment is not harmful, is not expensive and is not obvious quackery. The difference of opinion between medical staff and people with MND mainly comes in the first part – whether the treatment is harmful. As doctors we are taught, “first do no harm”, whereas someone with MND will often say they are dying anyway so it makes no difference if the treatment is harmful because there might be a benefit. This is a powerful argument that can only be countered by the point that someone may have a slow form of MND and be shortening their life significantly or may greatly reduce their quality of life.
“I see people who are desperate, spending tens of thousands of pounds on treatments that are definitely pointless, like stem cell therapy consisting of giving an unknown substance intravenously at an unlicensed clinic. I see others who shun conventional medicine because they believe in alternative therapies, even though conventional treatments have a known side effect profile and alternative treatments do not. As medical staff we have an ethical obligation to provide clear advice in these situations, but I have not had a life threatening illness before and cannot say how I would react, so there is always a seed of doubt in my mind about whether I am doing the right thing.”
A person living with MND’s point of view
Charlie Fletcher is living with MND and explains her point of view on unproven treatments after reading the new Sense about Science booklet:
“Having been diagnosed with MND and confronted by the stark reality that there is no cure and incredibly limited medications available, was a hard pill to swallow. To then be bombarded by the media with advert after advert of extraordinary treatments and radical cures was overwhelming. Those with long-term illnesses tend to have a heightened sense of impending doom and a desperate need for hope so it is not surprising that many are swayed by these claims. I will admit that at times the little voice in the back of my mind suggests I might be overlooking the answer I’m after, but as a natural sceptic I am not likely to be tempted unless there is substantial evidence to support these theories.
“Whilst I can appreciate the draw of untested drugs and alternative therapies, I am not about to start licking trees on the off chance. However, medical research is an integral part of the search for possible treatments for diseases like MND and I am fully supportive of the incredible work being done. In my opinion, participating in clinical trials of drugs and methodologies is beneficial in the development of our knowledge of the disease and eventually a cure, and therefore, is something for which I would willingly offer my assistance.
“It is often difficult to separate the head from the heart, particularly for family and friends who are naturally driven by their need to make things better. The passing on of unlikely remedies and anecdotes for seemingly successful treatments, in my personal experience, is more of a hindrance than a help. Following all this advice can be exhausting, time-consuming and expensive, but more importantly it can create false hope, which can have a devastating effect especially when something doesn’t work.
“To my knowledge there is only one drug available and it is not even close to a cure; as far as I’m concerned, if the only drug that has passed its clinical trials and can be prescribed barely makes any difference, I am not likely to be convinced by the promises made by the latest, untested ‘miracle drug’.
“I find the information presented in this booklet an accurate and in-depth view of the realities of long-term, degenerative conditions and for this reason felt moved to add my contribution.”
We are aware that a number of MND organisations, forums and people living with MND have received spam messages regarding a gentleman who claims that he can stop the deterioration for MND through an ‘electro medicine’ treatment. He urges readers of the message to reply to his email so that he can provide his proof. We are concerned about this as the mailer, a Mr Oholiav, is selling an unproven treatment for MND that could either be harmful, or non-existent – either a ‘snake-oil’ salesman or a complete hoax.
Trust in evidence not hearsay
We are aware of countless internet sites and scams that offer unproven treatment regimes that ‘prove’ the effectiveness of treatments through anecdotal evidence: through quotes from people who have taken the treatment; through articles in the media and other publications that have not been peer reviewed; case study reports; and documents written by ‘Drs’ that look credible at a glance. However, these treatments have not been through clinical trials, and so there is little proof whether a treatment is safe or beneficial.
A treatment is proven to be effective through carefully controlled clinical trials that test both safety and effectiveness. Trials have to be very carefully designed and carried out to ensure that the treatment is tested in the most unbiased circumstances possible, while giving the treatment the best chance of showing that it is safe and effective. If a treatment successfully shows this through trials, then treatments can be licensed for use. Clinical trial results can then be published in reputable scientific journals – the cornerstone of knowledge sharing in terms of research.
Without researchers publishing their results, and their studies being reviewed by their peers for integrity, we simply wouldn’t know what to believe. Allowing your work and your claims to be subjected to peer review is the internationally recognised way of evaluating research work and medical treatments. And by researchers sharing their knowledge and experience in a controlled setting – through publishing peer reviewed scientific journal articles- we can trust in evidence, not hearsay.
As I’m writing this, we’re in the process of proof reading study overviews (abstracts) ready for the International Symposium on ALS/MND to be held in Sydney, Australia from 30 November to 2 December this year. It’s a real opportunity for researchers, clinicians and health and social care professionals to come together to share their recent study results – their evidence.
Flitting between reading the abstracts (which I must add have been peer reviewed), Mr Oholiav’s unproven treatment email, and our news that we’re now part of UK PubMed Central (read Belinda’s Blog entry published this morning for more information on this great news) is a real chalk and cheese affair. It’s a definite case of comparing how research should, and should not, be shared.
There are hundreds of researchers, clinicians, and health and social care professionals around the world dedicated to ending MND. These are the people who methodically set up their research projects; who place great care and attention in publishing their research papers in reputable journals; who attend the International Symposium on ALS/MND to learn about current avenues of MND research; and who share their knowledge with other researchers from around the globe through the appropriate channels. These are the people who have the right tools to unlock the secrets of MND.
We believe that exceptional claims require exceptional evidence. We believe too, that allowing treatments to be made available which have not been through the established clinical trial processes can encourage unscrupulous individuals to market ineffective treatments for financial gain and provide false hope to patients and their families.
We have already received feedback from a number of people affected by MND who have been emailed by Mr Oholiav who have informed us of their anger and upset over his correspondence. They have told us that they have deleted the email as they know it’s content is full of mis-information.
But we know that some people will want to explore unproven treatments as they see it, understandably, as a beacon of hope. This is why we urge anyone contemplating an unproven treatment to contact the research team here at the Association so that we can give them the latest information on unproven treatments to help them make an informed decision.
If you’ve received an email from Mr Oholiav, please let us know your thoughts by commenting below.
We have been made aware that there’s a new kid on the block in terms of unproven treatments, which is a new route of administration of stem cells at the X-Cell stem cell clinic based in Germany.
As a brief background of the story to date, last year, a group of international researchers collectively known as ALSUntangled investigated the claims of X-Cell.
ALSUntangled wrote, and published an article (in the journal ALS) on the X-Cell clinic which concluded that until they demonstrate the safety and effectiveness of their stem cell treatment through a rigorous clinical trial that they would not condone X-Cell centre’s protocol for people living with MND. We wrote about this in our ‘X-Cell Stem Cell Centre has been investigated by ALSUntangled’ blog article.
X-Cell has now adapted their strategy to use intravenous (IV) administration for stem cells, meaning that the cells are delivered into a vein in the arm rather than via surgery on the brain or spine. X-Cell claim that they are able to use this new, far less invasive route of administration because they also give patients IV mannitol to help the stem cells gain access to the central nervous system. Mannitol is a drug used to draw water out of the brain in cases of cerebral oedema (swelling of the brain). There has been research into mannitol use to ‘open’ the blood brain barrier for chemotherapy to improve delivery of drugs to tumors in the brain. However, there is a big difference in trying to get a reasonably small chemical through the blood brain barrier – which in real life terms is like a sieve from the blood through to the brain, and trying to get comparatively huge stem cells through the blood-brain barrier. Unless research is published to demonstrate that this is possible, then it is an unproven method.
We are aware that unproven treatments can seem attractive to people affected by MND given the lack of a treatment. However, they often come at a large cost and have not demonstrated their effectiveness in rigorous clinical trials. To find out more about what makes a good clinical trial, visit our website: ‘what makes a good clinical trial’, or ‘unproven treatments’.
If you are considering an unproven treatment and would like to know the facts about the information they provide, please contact us at email@example.com. We provide the facts so that people affected by MND can make up their own minds about whether it’s an option they would like to consider.
*Updated – the X-Cell centre has now been closed due to the German Government tightening the loophole that allowed the centre to offer unproven stem cell treatments.
Alternative treatments can be tempting if you have a disease like MND, for which conventional medicine can only offer one moderately effective drug. Unfortunately this temptation is easily preyed upon by clinics around the world that claim to be able to provide effective treatments or even a cure (at great expense of course), despite having no supporting evidence.
We often take enquiries from people with MND who are considering undertaking one of these ‘unproven’ treatments and want to know if we have more information about them. Often, all we can talk about is the lackof sound evidence that they work, so it can be helpful when a bit of hard evidence does come along – even if it’s suggesting that the treatment doesn’t work. This is the case with an article from the journal ‘Brian Pathology’, which crossed my desk recently.
Many people with MND have travelled to a clinic in Beijing to receive a type of stem cell treatment known as ‘olfactory ensheathing cell (OEC) transplantation’. This treatment is effectively experimental, having not undergone clinical trials, yet the Chinese clinic demands large sums of money from its patients and does not appear to follow their progress after treatment.
Independent neurologists who have tracked the progress of people with MND before and after treatment in China have failed to see any real improvement in their condition. Now, Italian scientists have presented in Brain Pathology some clues as to why this treatment isn’t working.
Two Italians with MND who had undergone the Chinese treatment generously donated their brains for use in research after they had passed away. This gave the scientists an opportunity to look at what had happened to the transplanted stem cells. They were able to see the tracks left by the needles used during the procedure and found that all of the tracks ended in different places, with none of them actually reaching the main motor neurone pathway from the brain to the spinal cord. The researchers did find some OECs, but they had been trapped within the needle tracks by the brain’s own defence mechanism – so the stem cells never got to where they were really needed. In addition there was no sign that the transplanted cells had produced any neurone-nourishing substances or turned into neurones or support cells – all mechanisms by which stem cells could potentially have some effect in MND. In light of all of this, it is not surprising that the researchers also found that brains showed all the typical hallmarks of MND degeneration and that their donors had experienced a typical progression of their disease.
This research may only have looked at the brains of two people with MND, but it all adds to the information we can give people who ask us about this treatment – and information means informed choice.