New research into gene therapy in early dementia

Professor Christine van Broekhoven and her team, along with colleagues from the US Mayo Clinic and the University of British Columbia, discovered in 2006 that errors in the progranulin gene form the basis of what is called frontal lobe dementia. This type of dementia – after Alzheimer’s disease – is the second most important cause of dementia at a young age. In frontal lobe dementia, a familial form of dementia, neurons in the frontal and temporal lobes of the brain die prematurely. This gives different symptoms than Alzheimer’s disease. For example, people with frontal lobe dementia often show marked changes in behavior and personality, and they also have problems using language. The disease develops very quickly. Patients become increasingly dependent in a relatively short time and die an average of 8 years after the first symptoms appear. Until today, there has been no cure for this type of dementia.

genetic errors

The discovery that Progranulin Growth Factor deficiency causes this disease now forms the basis of new clinical research that offers hope for a treatment by Professor van Broekhoven and colleagues. After all, the results suggest that increasing your progranulin intake could be a promising strategy for treating this type of dementia. This is expected to slow or even stop the progression of the disease. To test this, VIB went looking for an industrial partner to develop the treatment. About two years ago, a licensing agreement was entered into with American Prevail Therapeutics to develop gene therapy for people with frontal lobe dementia. Currently, phase 1/2 of the clinical study has been released on this topic. Such a scientific breakthrough does not automatically mean that a treatment will follow soon. Unfortunately, this often takes years.

Professor Christine van Broekhoven and her team, along with colleagues from the US Mayo Clinic and the University of British Columbia, discovered in 2006 that errors in the progranulin gene form the basis of what is called frontal lobe dementia. This type of dementia – after Alzheimer’s disease – is the second most important cause of dementia at a young age. In frontal lobe dementia, a familial form of dementia, neurons in the frontal and temporal lobes of the brain die prematurely. This gives different symptoms than Alzheimer’s disease. For example, people with frontal lobe dementia often show marked changes in behavior and personality, and they also have problems using language. The disease develops very quickly. Patients become increasingly dependent in a relatively short time and die an average of 8 years after the first symptoms appear. Until today, there has been no cure for this type of dementia. Professor van Broekhoven and colleagues’ discovery that a deficiency of the progranulin growth factor causes this disease, now forms the basis of new clinical research that offers hope for a cure. After all, the results suggest that increasing your progranulin intake could be a promising strategy for treating this type of dementia. This is expected to slow or even stop the progression of the disease. To test this, VIB went looking for an industrial partner to develop the treatment. About two years ago, a licensing agreement was entered into with American Prevail Therapeutics to develop gene therapy for people with frontal lobe dementia. Currently, phase 1/2 of the clinical study has been released on this topic. Such a scientific breakthrough does not automatically mean that a treatment will follow soon. Unfortunately, this often takes years.