The Hospital Universitari General de Catalunya, which was the first center in Spain activated for the phase I trial in Parkinson’s (REASON) with which it is intended to modify the natural progression of the disease, continues to include new patients, after having been the one that included to the first patient in Spain.
The research consists of administering by injection intrathecally – a fluid-filled space between the thin layers of tissues that cover the brain and spinal cord – of a dose of an antisense oligonucleotide, which avoids the signal that activates a gene called LRRK2, known as “the Basque mutation” or Parkin 8, with the idea of inhibiting the formation of the alpha-synuclein protein.
Parkinson’s disease, a neurodegenerative disorder of unknown cause, which produces tremor at rest, slowness of movement and rigidity, is caused by a deficiency of dopamine at the level of the basal ganglia and is characterized by the presence of this abnormal protein that is the alpha-synuclein.
The main objective 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 for patients with Parkinson’s disease with verified presence or absence of mutations in the leucine-rich repeat kinase 2 (LRRK2) gene, but also for patients without any verified disease-related gene mutation since it is postulated that this gene will also be overexpressed in all Parkinson’s patients and not just the LRRK2 mutants. In the latter patients the progression of the disease would be prevented 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), it is a very innovative approach to try to modify the natural history of Parkinson’s, both for patients who have this “Basque mutation” and for those who they do not have it, and it could prevent the progression of the disease. ”
At the Hospital Universitari General de Catalunya, several lines of research are developed through the UDIC, led by Dr. Ernest Balaguer. It should be noted that all anti-Parkinson drugs authorized in the last 20 years have been evaluated.