The main purpose of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 to participants with Parkinson’s disease.
The General University Hospital of Catalonia is the first center in Spain that has been activated for a new and particular phase I trial in Parkinson’s (REASON), which seeks to cure the disease. The trial, which has already begun in the USA, involves the administration of an intrathecal injection – fluid-filled space between the thin layers of tissue covering the brain and spinal cord – with a dose of an antisense oligonucleotide, which avoids the signal that activates a gene called LRRK2, known as “the Basque mutation” or Parkina 8, with the idea of inhibiting the formation of the alpha-synuclein protein.
Parkinson’s disease, a neurodegenerative disorder of unknown cause, which causes resting tremor, slowness of movement and stiffness, is caused by a dopamine deficiency at the basal ganglia and is characterized by the presence of this anomalous protein that is the alpha-synuclein.
This is a brilliant experiment to try to modify the natural evolution of parkinson’s.
The main purpose of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 to participants with Parkinson’s disease. The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB094. The study is open to patients with Parkinson’s disease with verified presence or absence of mutations in the leucine-rich repeat kinase 2 gene (LRRK2), but also for patients without any verified genetic mutations related to the disease as it is postulated that this gene will also be over-expressed in all patients with Parkinson’s and not only the LRRK2 mutants. In the latter patients the progression of the disease would be avoided while in the former it could be avoided.
For Ernest Balaguer, director of research at the HUGC and head of the Clinical Research Unit (UDIC), this is a “brilliant experiment to try to modify the natural evolution of parkinson’s, both for patients who have this” basque mutation “and those who they do not have it, and it could prevent the progression of the disease. “The clinical trial appears on www.clinicaltrials.gov and will soon appear the General University Hospital of Catalonia, as well as on the Michel J Fox Foundation Trial Finder.
The Hospital develops several lines of research through the UDIC, led by Dr. Ernest Balaguer. It is noteworthy that of the last seven anti-Parkinson’s drugs that have been marketed in Spain, all of them have been evaluated at the General Hospital, because they have a great experience in these drugs