This is an extremely promising avenue of research, and one that we are encouraging on this International Rare Disease Day. “What we are developing is as close as it gets to curing a form of early-onset dementia: we are targeting the genetic cause of the disease”, explains Prof. Dr. Patrik Verstreken who receives the Generet Prize for Rare Diseases 

Prof. Dr. Patrik Verstreken (Professor and researcher at the Center for Brain & Disease Research VIB – KU Leuven) is researching how a specific form of early-onset dementia can be treated with so-called ASOs (antisense oligonucleotides). This year, he receives the Generet Prize for Rare Diseases from the Generet Fund, managed by the King Baudouin Foundation and administered by the FNRS. This prize comes with a research budget of 1 million euros. 

Difficulty processing information and memory, mood swings, behavioural changes... the symptoms of dementia are all too familiar to many. For some, they come very early: several hundred thousand people worldwide carry a genetic mutation that inevitably causes them to develop dementia in their forties or early fifties. In Belgium, 300 to 400 people suffer from this disease. The disease strikes early and progresses rapidly. 

"It concerns familial versions of Alzheimer's," says laureate Prof. Dr. Patrik Verstreken (VIB – KU Leuven). Prof. Verstreken's team is particularly looking at a large Mexican family with the same mutation. In other families, it is a different mutation, but always on the presenilin gene, and always with the certainty that early-onset dementia will occur. 

"We are investigating how we can treat them with antisense oligonucleotides. ASOs are a type of medication that ensures certain parts of the DNA are expressed differently – 'differently' usually means 'less'," explains Prof. Verstreken. 

When asked when the drug will be available to patients, Prof. Verstreken understandably responds somewhat cautiously, "But I dare to hope within a foreseeable time, which in research terms means: within about ten years…Thanks to the funds from the Generet Fund, we can create more ASOs, conduct more preclinical tests, involve more families, learn about more mutations... In short, continue working towards a solution." 

See the press release of the King Baudoin Foundation...