Oxford BioMedica presented preclinical data for its Parkinson's disease product, ProSavin at the 31st Annual Meeting of the American Society of Neurosciences in November 2001
The data show that the administration of ProSavin in a preclinical disease model leads to the production of dopamine in brain cells that were previously unable to produce the neurotransmitter and that it corrects Parkinson's disease-like symptoms.
Parkinson's disease (PD) is a neurodegenerative disorder characterised by the death of brain cells that produce the neurotransmitter, dopamine.
Dopamine controls movement and its loss leads to the familiar symptoms of PD ie, rigidity, resting tremour and motor function impairment resulting in slow movements and poor balance.
The disease affects approximately 2.5 million people worldwide.
Current treatments include oral administration of L-dopa, the precursor of dopamine which can restore some degree of motor function.
However, the systemic administration of this drug soon becomes ineffective and has serious side effects.
As a result, the directors of BioMedica believe there is still an unmet medical need for an effective treatment for this disease.
ProSavin is a LentiVector which delivers three genes required for dopamine synthesis to brain cells that do not normally produce it.
This results in these cells becoming endogenous factories for dopamine, thereby replacing the dopamine-producing cells that die during the course of PD .
This achieves the goal of local and continuous production of dopamine without the systemic adverse effects of L-dopa administration.
Commenting on the preclinical results presented, chief executive professor Alan Kingsman said: "Through our research, we have shown that ProSavin is the first gene therapeutic product to deliver more than one gene into brain cells at precisely the site for treating PD.
It is also the first of its type to demonstrate effectiveness in reversing symptoms of the disease in well known model systems.
"Preclinical development of ProSavin is ongoing and we look forward to keeping shareholders updated on progress."
