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17/07/2018

VHIR participates in a trial in patients to test a drug for Huntington's disease

Huntington_gamez_884

17/07/2018

The Phase II study will determine the efficacy and safety of the compound in reducing choreic movements in 30 patients with Huntington's disease for a period of 6 months.

The biopharmaceutical company https://bit.ly/2NvFzI7 SOM Biotech -located in the Barcelona Science Park (PCB) and specialised in drug repositioning to treat minor diseases of the nervous system- has initiated the clinical phase of an innovative treatment for the choreic movements associated with Huntington's disease: the compound SOM3355, a drug currently marketed for high blood pressure. The new therapeutic activity of the drug was discovered by SOM Biotech through its computational platform based on artificial intelligence algorithms and, after the promising results of the preclinical trials, the company is initiating the study of Phase II of the proof of concept in humans in order to determine the efficacy and safety of the drug in patients with Huntington's disease.The trial is coordinated by neurologist Dr. Jaume Kulisevsky, director of the Biomedical Research Institute Sant Pau (http://www.recercasantpau.cat/en/ IIB Sant Pau) and of the Movement Disorders Unit at http://www.santpau.cat/web/public/presentacio Hospital de la Santa Creu i Sant Pau in Barcelona. It will be carried out in parallel with three more centres: https://www.vallhebron.com/en Vall d'Hebron University Hospital, under the leadership of Dr. Josep Gàmez, head of the Peripheral Nervous System research group at VHIR, https://www.hospitalclinic.org/en Hospital Clínic of Barcelona, and http://www.bellvitgehospital.cat/en_index.html Bellvitge University Hospital, under the leadership of Dr. Esteban Muñoz and Dr. Matilde Calopa, respectively.A rare and disabling disorderHuntington's disease is a neurodegenerative disorder triggered by a genetic alteration (the mutation of the gene that encodes the protein called huntingtin) that gradually destroys neurons in specific regions of the brain and involves severe motor, cognitive and behavioural dysfunction. It is an autosomal dominant hereditary disease: people with this mutation have a 50% chance of transmitting it to their offspring.According to internal studies of the company, the prevalence of this disease is between 6 and 8 per 10,000 people in Western countries, and most of those affected develop it between 35 and 55 years. Life expectancy is long, between 15 and 20 years, during which the disease progresses gradually and slowly, generating a large functional, social and family disability."Huntington's disease affects some 2,500 people in Spain and about 78,000 worldwide. There is no treatment that modifies the progression and the available medications are symptomatic with side effects that often make them unusable. SOM3355 is a safe drug that has been administered for many years to patients with hypertension and which has been highly valued by a market study conducted by Insights in Life Science (ilS)", states Dr. Raúl Insa, CEO of SOM Biotech.He adds: "We hope we can demonstrate the expected efficacy in this clinical trial since it would mean a considerable reduction of health expenditure as it is a well-known product and with a reduced cost with respect to other alternatives. If everything goes well, I could reach the market in a five-year period and achieve worldwide sales of around 600 million euros."SOM3355 is the second product of SOM Biotech that reaches the clinical phase. It is preceded by the successful case of compound SOM0226 for the treatment of transthyretin amyloidosis that the biopharmaceutical licensed to the North American Corino Therapeutics Inc., after obtaining very positive results in the Phase II study conducted at the Vall de Hebron Hospital in Barcelona.Efficacy and safety to combat choreic movimentsThe most visible and disabling symptoms associated with Huntington's disease are choreic movements (from the Greek khoreia, dance): abnormal, abrupt, irregular and uncontrolled movements that predominantly affect the extremities and facial muscles. Its intensity worsens in the final stages of this pathology, causing the motor function of patients to be severely affected during their last years of life.After an internal process of discovery and completion of preclinical trials, SOM Biotech verified that the drug SOM3355 offered great potential for the treatment of central nervous system movement disorders associated with Huntington's disease, and that its properties were susceptible to avoid the serious side effects of other drugs used for the treatment of the pathology, which has led them to obtain a restricted prescription. The application of SOM3355 could also be extended to tardive dyskinesia, Tourette's syndrome or hemiballismus, according to the company's own sources.Now this Phase II study will allow the pharmaceutical company to determine the efficacy and safety of the compound in the reduction of choreic movements in 30 patients with Huntington's disease during a period of 6 months."Currently, the treatment of movement disorder in Huntington's disease is limited by the side effects of the drugs used. SOM3355 offers a promising alternative to improve the tools available to treat this pathology. The clinical trial that we are now starting has a double-blind and randomized design that will allow us to objectively verify the drug's ability to improve abnormal movements. The participation of four centers in our environment will facilitate the recruitment of a sufficient number of patients and will guarantee the objectivity of the results. On the other hand, this study demonstrates the competitiveness and importance of the collaboration between bioscientific companies and hospitals and research centres in the country", states the coordinator of the trial, Dr. Jaume Kulisevsky.

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