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Health, Scientific progress

17 - 08 - 2017

Therapeutic advances against a deadly genetic disease

A substance validated in preclinical trials in a cat model, gave encouraging clinical results against Niemann-Pick disease.


Niemann-Pick disease is a rare neurological disease that affects children and adolescents. It causes a decline in neurological and cognitive functions. Death is inevitable and there is no treatment.


A 12- to 18-month clinical trial of patients aged 4-23 years tested the potential of VTS-270 to treat this disease.


VTS-270 aggravated the hearing loss often observed in patients but that could be compensated with hearing aids. However, activity, cognition and speech scores were improved. Blood tests showed an improvement in the biochemical disturbances associated with the disease. The authors conclude that VTS-270 stops or slows down the disease.


A new clinical study is in preparation to clarify the medical interest as well as the administration conditions (dose, frequency). The objective is to bring it to market.


The VTS-270 was evaluated in a preclinical trial in 2015 in a cat model of Niemann-Pick disease. Indeed, in 2003, the existence of a spontaneous mutation similar to that which causes Niemann-Pick disease in humans, was discovered to cause the same type of disease cats in breeding operation in 2003. Since then, the use of this model is a prerequisite for patient trials.