World ALS Day 2017: the fight against an unseen killer

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disorder that leads to progressive muscle weakness and paralysis. About half of the patients die within three years after the diagnosis. No treatment currently exists. On the occasion of World ALS Day 2017 we present an overview of the latest in the fight against an unseen killer.

what is als?

If you want to solve a problem, you first need to understand the underlying causes. That’s no easy task in the case of ALS. It’s a neurodegenerative disease that leads to progressive muscle weakness, making it increasingly difficult for the patient to move, talk, swallow, and breathe.

The exact causes are not yet entirely clear. Ninety percent of the patients have sporadic ALS, which is probably caused by a combination of genetic errors and environmental factors. In recent years, researchers have gained a lot of insight into these genetic errors thanks to intensive research into genetic ALS, which affects ten percent of the patients.

Neurologist Philip Van Damme has already dedicated seventeen years of his life to ALS research. "All things considered, I see a lot of progress compared with seventeen years ago, when I first started doing research into ALS as a doctoral student. We may not have found the ultimate cure, but we’re definitely getting ahead. The outside world assesses our work on the basis of this final step - a treatment - but our research has really gained momentum. This gives us reason to be hopeful."

More information

raising awareness

A lot has changed in recent years, says ALS researcher and Vice Rector for the Biomedical Sciences Group Wim Robberecht. "We’ve come an incredibly long way in the past ten years. Several initiatives have contributed to these changes. One of them is the global Ice Bucket Challenge (whereby people raised awareness of ALS by pouring a bucket of ice over their heads, ed.), which gave an incredible boost to public awareness of the disease and led to a tsunami of research projects."

In the wake of the Ice Bucket Challenge the Flemish Government supported ALS research, and donations to the Belgian ALS League as well as KU Leuven funds increased. And patient organizations such as Een Hart voor ALS raise money for research as well. These extra resources are very welcome because there is still no cure, no drug to beat ALS. 

  • Researchers know that patients’ SOD1 gene contains an error and harms the neurons. That’s where abnormal protein clots develop that probably play a major role in the development of the disease. A specific type of gene therapy may slow down the production of these proteins and have an immediate effect on the error that triggers the entire disease process. This type of gene therapy has already shown promising results for a different motor neuron disease, so researchers believe it may also help ALS patients. The studies are still in progress. They only apply to patients with genetic ALS,  but the new insights will also help advance research into sporadic ALS.
  • A clinical study was recently published about Edaravone, a drug with a positive effect on patient’s decline, including those who don’t have the genetic form of the disease. Up to now, this positive effect has only been described for a limited group of people who met specific conditions, were selected in an early stage, and for whom the doctors expected a quick deterioration. Whether the drug will be registered in Europe is still unclear, but the American Food and Drug Administration (FDA) has recently approved Edaravone.
  • At the same time, we’re learning more about the glial cells that provide support for neurons and surround them. Recent findings indicate that these cells probably contribute to neurodegeneration.  
  • Scientists around the world are also looking for biomarkers to measure the progress of the disease more quickly and more accurately. These pieces of the puzzle are still missing. You can closely monitor the size of the tumour in cancer patients and check virus concentrations in the blood of HIV patients, but you cannot predict where ALS will start, and the progress of the disease is different in every patient. It’s very important that we keep looking for more accurate measuring methods so that when we start clinical studies we can be more specific.
  • In its early stages ALS is difficult to diagnose. The average time between the first symptoms and the diagnosis is a year. To make a quicker diagnosis possible we need better tests for ALS. Researchers have discovered that the lumbar fluid of ALS patients contains a higher concentration of the structural proteins in the cytoskeleton known as neurofilaments , perhaps because these are released by affected motor neurons. Therefore, a lumbar puncture may soon make an accurate diagnosis possible.
  • VIDEO: Researcher Steven Boeynaems about ALS research

    VIDEO: Researcher Philip Van Damme about ALS research

    A patient’s story: "It’s ALS, there’s no medication to stop it"

    André Laevers cannot talk about his son without tears glistening in his eyes. After all, Lorenz was a special person, and he was barely 34 when he lost his life to ALS. "A week earlier he was still playing poker with his friends. They stood by him until his dying day and now they organize a festival in his honour every year."

    "But dad, it’ll go away. That’s what our Lo said after waking up with a stiff tongue one morning. He was young and not one to worry. But then he helped a customer in our insurance office and this man told me that Lo seemed drunk. I sent my son to the GP right away."

    "The doctor didn’t find anything. This was back in 2006. ALS was not nearly as known as it is today. In any case, I’d never heard of it. Only six months and several specialists later did we get the verdict. Lo had gone into KU Leuven neurologist Wim Robberecht’s office by himself but he was back in the hallway a minute later to ask me to come with him after all. The professor came straight to the point. He said: It’s ALS, the symptoms are only going to get worse and I have no medication to stop that."

    Lo’s speech deteriorated very quickly. "After a couple of months he had already lost the ability to speak. We communicated with an alphabet: we pointed to the letters and he blinked his eyes if it was the letter he meant."

    "It really moved us that Lo’s friends didn’t turn their backs on him when he was sick. Each Thursday they came to our house to play poker. They put the cards in Lo’s hand and he blinked his eyes to indicate if he wanted to fold, call, or bet. Lo and his friends also celebrated New Year together. When he was already in a wheelchair they went on holiday to Southern France together. In his final week Lo went to Mechelen, the city where one of his friends had opened a restaurant. He didn’t want to miss out."

    "Lo was sturdy and athletic, so we thought that he’d still be with us for a long time. But none of that makes a difference when it’s ALS. Lo became completely paralyzed. He needed constant help at home and he woke me up every night to move his legs. That was something he could no longer do himself."

    "One hot day in the beginning of July, six years after the diagnosis, he started gasping for breath and we had to take him to the ER. Lo was put on a ventilator. Those first days, we thought that the doctors would be able to patch him up as usual, until Professor Robberecht made it clear to us that it wouldn’t the case this time. What do you say to your child at a moment like that? Are you in pain? He couldn’t even answer."

    Lo was sturdy and athletic, so we thought that he’d still be with us for a long time. But none of that makes a difference when it’s ALS.

    At his funeral his friends came up with the idea to organize a festival to keep Lo’s memory alive and to raise money for the Laevers Fund. "I had established this Fund three years earlier together with Leuven University Fund. I had asked Professor Robberecht if I could offer any financial help for his research into ALS. Thanks to the Laevers Fund, he was able to hire an extra researcher."

    "Ever since Lo died the Fund always receives the proceeds from the annual Lolands Festival, of which the sixth edition will take place in September. On average, 800 people come to the barbecue and in the evening thousands of people come to the performance in the big festival tent. Last year Daan gave a top performance. The sponsors enjoyed the performance in a separate VIP lounge. It’s such a great party that I always think: Lo would have loved this."

    how can you help?

    The help of donors is one of the reasons why KU Leuven doctoral student Steven Boeynaems will soon be able to start working at Stanford University. "For a long time, Stephen Hawking was the only ALS patient I knew existed. When I started working on my PhD four years ago, this changed. At the time, I was studying a specific type of DNA mutations that turned out to be relevant for ALS. I started looking into it in the lab of professors Wim Robberecht, Philip Van Damme, and Ludo Van Den Bosch."

    "Some of my most crucial experiments at KU Leuven were funded with donations made to KU Leuven for ALS research. The mutation I used to study was only discovered in 2011 but thanks to the flexible resources from the fund I could get to work almost immediately. This would never have been possible if I’d have had to apply for funding from the government, for instance, because this type of funding requires a project to be mapped out long beforehand."

    "Another advantage of resources acquired through donations is that they allow us to test preliminary ideas. Once you’ve done that, you have a better idea of which direction you have to head into and you can more easily convince the government and larger funding organizations. So I can only thank the donors, and I hope that one day I’ll be able to reward them through my research. I’ve seen too many ALS patients pass away. This has motivated me even more to spend the rest of my career working on the disease. This has become very personal."

    support als research at KU Leuven


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