"http://www.sombiotech.com/" SOM Biotech, located at the "http://www.pcb.ub.edu/homePCB/live/ct/p1.asp" Parc Científic de Barcelona, has initiated the clinical phase of an innovative oral therapy for transthyretin amyloidosis (ATTR): compound SOM0226, a reprofiled drug developed entirely by the biopharmaceutical company. The phase IIa proof-of-concept trial in humans is being conducted at the Vall d'Hebron University Hospital, coordinated by Dr. Josep Gámez from the "http://www.vhir.org/larecerca/grupsrecerca/ca_grups_equip.asp?Idioma=en&mv1=2&mv2=1&mh1=2&mh2=1&mh3=1&mh4=0&ms=0&area=4&grup=12&menu=3" Peripheral Nervous System research group, and the treatment has already been administered to the first patients. The hospital will assume, jointly with the biotech, the risks and return on investment of the development of the drug, with the recent support from the Spanish Ministry of Economy.Transthyretin amyloidosis is a rare disabling disease with a severe prognosis that primarily affects the peripheral nervous system and the heart and, in some cases, is inherited from parents to children. The disease develops when the liver and other parts of the body produce the mutated protein transthyretin (TTR), causing a number of aggregates that are deposited into multiple organs. This excessive accumulation of protein, known as amyloid deposits,causes local cell damage that presents with a variety of clinical symptoms. These symptoms include severe cardiac and neurological problems that lead to the death of the patient within a period of 5 to 15 years.“Currently there is no other drug undergoing marketing approval for transthyretin amyloidosis in Europe. The only treatment available is a drug discovered in the United States (with the exorbitant price of more than 140,000 € per patient and per year) and liver transplantation, suitable only for a particular population group and with very high social and health costs", says Dr. Raúl Insa, founder and CEO of SOM Biotech. SOM0226 is a potent inhibitor of the amyloidogenic process with an innovative dual mechanism of action: as stabilizer of the transthyretin protein and disruptor of the fibers once they are deposited into the tissues. To cut it short, it stops progression and reverts the disease. This drug may also be effective against the 4 variants of transthyretin amyloidosis - polyneuropathic, cardiac, senile systemic and the CNS selective - including the more advanced stages of the disease.As a consequence, "this new therapeutic alternative will not only be able to confront Transthyretin amyloidosis but will also be able to prevent its progression, avoiding the formation of amyloid fibers”, remarks Dr. Gámez.