Report of the international symposium 2010

International symposium October 7th, 2010: summary

Thursday, October 7th 2010, the ALS Liga welcomed about 200 participants at the scientific symposium concerning “SOS ALS”, the festival- and awareness week of our 15th anniversary. Among the numerous attendants were patients, families, care aides, care takers, doctors and scientists. The symposium had an international character, through the attendance of both foreign guest speakers as well as foreign audience.

During the morning program there was a possibility for the patients to get to know each other. We were all in need of catching up and getting to know people.

Prof. Wim Robberecht and rehabilitation specialist Hilde Beyens gave a presentation about the functioning of the UZLeuven Neuromuscular Reference Center (NMRC). As a typical example of one of the 6 recognized centers in our country, the NMRC UZ Leuven takes care of both a diagnostic as well as a therapeutic tailor-made care program. After a full characterization at the Neuromuscular consult the patients are referred to the multidisciplinary team of the NMRC. At the service of the PALS are administrative coordinators, specialized nurses, a rehabilitation expert, nutrition expert, psychologist, physiotherapist, remedial therapist, speech therapist and a pulmonologist. The medical and social framework offered is individually developed in close interaction with the home front. Next to that the NMRC participates in the research for ALS (clinical studies, heredity, stem cells, new treatments). An example of a technique currently under study was explained by the team, pneumology who are working together with the NMRC: via the stimulation of the diaphragm the respiratory muscles are trained and reduces their tiredness.

For lunch we enjoyed a meal advised by Piet Huysentruyt.

The kick-off of the afternoon program was a warm welcome to the audience by Evy Reviers, director of the ALS Liga. Moderator Jan Van Rompaey led the following international discussion by means of witty questions “why does this have to happen to me? – What can they do about it? – and the message of hope “they are working on it”.

Prof. Robberecht (UZLeuven en KU Leuven/VIB; BE) guided us through the biology of two types of nerve cells (peripheral moto-neuron, commanding neurons in the brain) which cause ALS. He also gave a transparent overview of the research done so far to find new treatments for ALS. After all already several ALS risk-genes have been determined from the genetic research concerning the hereditary form of ALS. Next to that scientists currently have a wide arsenal of suitable in vitro models (moto neurons grown on test scale) and animal models like the fruit fly, nematoden (roundworm), rodents (mouse, rat) and primates (monkey), so that in the end they would be able to do clinical trials with the human being.

Prof. Albert Ludoplph (University hospital Ulm; DUI) testified about his clinical experience with frontotemporal lobar degeneration (FTLD) with PALS.  He emphasized that this type of dementia is generally mild, and cannot be confused with the Alzheimer-type dementia. Current literature and numbers show (upto 40%) correlation between FTLD and ALS. Prof. Rudolph finds this numbers highly overrated based on his personal clinical follow ups of PALS. He talks about drastically lower numbers (about 5%), as stated also in historical documents.

Prof. Leonard Van den Berg (University Medical Centre Utrecht; NL) told us more about the functioning of ALS Centre Netherlands that was founded in 2003 as an answer to the needs of Dutch PALS. This clustering of specific ALS outpatients’ clinics with multidisciplinary teams, has significantly decreased the waiting period for the diagnosis, therapeutic consultation and obtaining medical devices. Next to that PALS get a greater say via meetings (patients talk, care takers listen on the second row). As a result the 21st of July has been named the Dutch ALS-day. The Center also collects samples (blood, urine, skin biopsy) of the more or less 600 patients they see per year. Because of that it was possible to start up a prospective ALS research Netherlands, which is now generating the first extensive lists with research results. This national Dutch study will serve as a model for a bigger European Research project about ALS which is now being introduced. Finally Prof. Van den Berg suggested to bundle the forces of the ALS Liga and the Dutch organizations so that in the future there could be a mutual ALS symposium for both stakeholders in Belgium and the Netherlands.

Prof. Peter Carmeliet (KU Leuven/VIB; BE) introduced doctor Annelies Quaegebeur who is preparing a PhD about the role of the metabolism in ALS, focusing on the therapeutic potential of VEGF and oxygen sensors. She gave us an overview of the most recent scientific results about the role of non-functioning mitochondria (buildup of harmful oxygen radicals) and VEGF (survival factor of moto neurons) in ALS, where rodent are the focal point (mouse with reduced buildup of VGEF shows ALS symptoms; administration of VGEF in the ALS rat model (SOD1 mutant) slows down the disease). Based on this animal models VGEF was considered to be safe and efficient as a potential cure for ALS. Clinical studies with humans, based on this research, are now in a stage where they are extensively defining the safety and effective dose of VEGF. The seconds part of the presentation focused on the importance of oxygen sensor PHD1 in ALS. The role of the oxygen sensor consists of measuring the oxygen level in the tissue, and is also responsible for the buildup of VEGF. In the past it has shown that the regulation of PHD1 protects against a heart attack, and recent research data also show a possible role in ALS.

Prof. Kathy Mitchell (University Ottawa Ontario; CAN) highlighted the work of the International Alliance for ALS/MND Associations, an international umbrella organization of patients of which the ALS Liga is also part. She delivered a message of hope and the conscious experience of every day. There are several appropriate channels for PALS so that they can have the appropriate medical care (multidisciplinary teams) and stay socially – (use of appropriate medical devices) and communicatively active (via social network sites such as Facebook). An area of attention is being able to choose the correct initiatives out of the abundant offer, such as scientifically based medical care (eg. Via

During the panel discussion the audience had the opportunity to interact with the speakers. Some questions that were raised:

- What is the current status of the clinical study with VEGF? The results of the safety study are expected at the end of March 2011. At this moment side effects of VEGF are favorable, but the potential effective dose of VGEF needs to be further refined. At this moment there has not yet been a comparable effectiveness study (VEGF versus placebo). The scientific/medical team is optimistic, but also careful about the switch from animal testing towards human testing.

- What is the current status of the clinical study with Talampanel? : The study has been ended negatively because of the fact that there were too many side effects. Because of these side effects there was a realistic possibility that it would not be possible to administer a high enough dose.

- What is the current status of the clinical study with Trophos ? : It is in phase 3. The results can be expected in about a year.

- What is the current status of the clinical study with a potential cure of Knopp Neurosciences / Biogen? : The start of phase 3 is expected at the end of 2010. Belgian patients are most likely to participate seen the fact that the inclusion criteria are broad.

- What is the difference between ALS en PLS? : In the case of PLS only the commanding nerve cells in the brain are affected, in contrast to ALS where also the peripheral moto neurons stop working. PLS is 10 times less common than ALS and knows a slower progress and harder diagnosis. Unfortunately Rilutek is not reimbursed for this indication. PLS with only a paralysis of the feet is possibly hereditary, PLS with difficulty of speech most likely is not.

- One of the participants asks the panel for attention about the fact that in France there is a possible correlation between higher incidence of ALS and the vaccination campaign against hepatitis B.

- Why is it that the pharmaceutical industry is performing research in ALS, despite the small number of patients? : ALS has the status “orphan disease”. Because of this a potential cure can be produced quicker and at a lower cost price. More insight into this “small” disease could have implications on the “bigger” markets in the pharmaceutical industry such as Parkinson, Alzheimer,… the implications of possible treatments of ALS are also more visible (such as decreasing the handicap) than of other diseases.

- Why is it that Rilutek has strong side effects? : Rilutek is normally well tolerated but in some cases there are strong side effects. In that case a decrease of the dose is highly recommended, after which the dose can be increased again. In case Rilutek is tolerated, we recommend to use it. At this moment it is the only, still insufficient, cure with proven neuro protective characteristics.

This scientific symposium ended with a festive reception on the occasion of 15 years ALS Liga. 


Translation: S. Janssens