Posts Tagged ‘Josep M. Campistol’

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Wednesday July 25th, 2018

Hospital Clínic and Hospital Vall d’Hebron participate in a study that demonstrates the efficacy of a new drug to treat familial amyloidosis

Hospital Clínic of Barcelona and the Vall d’Hebron Barcelona Hospital Campus have participated in an international study that demonstrates the efficacy of a new pharmacological treatment for transthyretin amyloidosis, the most common form of familial amyloidosis. Josep M. Campistol, nephrologist and CEO at Hospital Clínic, and Josep Gámez, neurologist at the Vall d’Hebron Barcelona Hospital Campus, are co-authors of the study published in the New England Journal of Medicine (NEJM).

Hereditary transthyretin amyloidosis (TTR) is the most common form of familial amyloidosis. It is a rare systemic disease caused by mutations in the gene encoding for transthyretin (a protein) and affects about 50,000 people worldwide. The average life expectancy, untreated, from symptom onset is 3 to 15 years, and the presence of cardiomyopathies is associated with a worse prognosis.

The liver is the main source of the TTR protein and, in this type of amyloidosis, both normal and altered protein accumulate in the form of deposits of amyloid substance in peripheral nerves, heart, kidney and gastrointestinal tract. Thus, different forms of the disease appear, such as polyneuropathy (involvement of the brain) or cardiomyopathy (involvement of the myocardium). “Until now, current treatment options included liver transplantation or the use of drugs to stabilize the circulating protein and thus prevent the formation of deposits, but in many patients the disease continues to progress,” explains Josep. M. Campistol.

A new pharmacological treatment to reduce TTR in blood

In the study published in NEJM researchers evaluated the efficacy and safety of Inotersen, a second generation antisense oligonucleotide (ASO) inhibitor. It is a drug that inhibits the production of TTR in the liver by binding to the TTR microRNA and reducing the production of both normal and pathological TTR protein. It is easily to administer subcutaneously once a week. Due to the half-life of the drug, Inotersen provides a constant reduction of TTR over time.

A total of 172 patients with hereditary transthyretin amyloidosis with polyneuropathy, in the presence or absence of cardiac involvement, have participated in this international phase III study. The results show that treatment improves the neurological disease and the quality of life of the participants, regardless of the disease stage or the presence of cardiomyopathy. Thrombocytopenia (decrease in platelet count) and glomerulonephritis (inflammation the glomeruli, the small filters in the kidney), which appeared in 3% of patients in each case, were the most frequent adverse events and were managed with enhanced monitoring.

“Inotersen is a gene therapy that allows subcutaneous administration of oligonucleotides that favor the degradation of the TTR protein microRNA, thus reducing TTR liver production,” explains Josep Gàmez, who is also coordinator at Vall d’Hebron of the CSUR in Rare Neuromuscular Diseases and the European Reference Network on Neuromuscular Diseases (EURO ERN-NMD).

Gene therapy blocks the production of TTR protein

In a second article also published in the NEJM, the efficacy and safety profile of the Patisiran has been studied. It is an RNA interference (RNAi) directed to the liver to specifically block the production of TTR. This type of drugs is based on a natural process of the cells to silence genes and, in this specific case, the RNAi helps to eliminate the circulating protein and reduce the deposits of amyloid in peripheral tissues. Josep M. Campistol and Juan Buades, internal medicine specialist and coordinator of the multidisciplinary group on hereditary transthyretin amyloidosis at the University Hospital Son Llatzer in Mallorca, have participated in the study.

A total of 225 patients with transthyretin amyloidosis have participated in this phase III study and the results show that this treatment improves the diverse clinical manifestations of the disease, as well as the quality of life, the nutritional status and the autonomy of the patients.

Both pharmacological treatment and gene therapy slow the progression of the disease and improve the quality of life of patients, and these improvements are independent of the stage of the disease” says Josep M. Campistol. “Both studies show the potential of two new drugs for an orphan disease, for which until now there were no other treatment options. The available therapeutic arsenal is broadened now and it will change the clinical practice in this type of amyloidosis“, he concludes.

Article references:

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.

Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T.

N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.

Adams D, Gonzalez-Duarte A, O’Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB.

N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153.

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Wednesday February 28th, 2018

Hospital Clínic, Mobile World Capital Barcelona and AIS Channel present the first technological operating theatre with remote assistance as part of 5GBarcelona

Hospital Clínic de Barcelona and Mobile World Capital Barcelona have presented today, as part of the GSMA Mobile World Congress, a pilot project that will make it possible to provide remote assistance for surgical procedures in real time. Josep M. Campistol, managing director of  Hospital Clínic de Barcelona, Carlos Grau, managing director of MWCapital, and Antonio de Lacy, head of the Gastrointestinal Surgery Deaprtment at Hospital Clínic de Barcelona and the founder of AIS Channel, took part in the event. Read the rest of this entry »

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Thursday January 26th, 2017

Scientists at Salk Institute have made breakthroughs in the integration of human cells into embryos of a different species

Human iPS cells (green) contributed to a developing heart of 4-week-old pig embryo (Image: Salk Institute)Scientists at Salk Institute for Biological Studies in California have made breakthroughs on multiple fronts in the race to integrate pluripotent stem cells from one species into the early-stage development of another. This will allow to study the embryonic development of an organism, diseases, test new therapeutic drugs and the possibly grow transplantable. Dr. Juan Carlos Izpisúa, professor in Salk’s Gene Expression Laboratory, leads this study published in today’s issue of the journal Cell. Dr. Josep M. Campistol, CEO and nephrologist in Hospital Clínic and researcher in IDIBAPS, and scientists from the Catholic University of Murcia (UCAM), the Pedro Guillén Foundation, the CEMTRO Clinic in Madrid and the University of Murcia, have also participated.

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Thursday December 15th, 2016

New cellular reprogramming technique counters hallmarks of aging

Scientists from Salk Institute for Biological Studies showed that One clue to halting or reversing aging lies in the study of cellular reprogramming, a process in which the expression of four genes known as the Yamanaka factors allows scientists to convert any cell into induced pluripotent stem cells (iPSCs) Like embryonic stem calls, iPSCs are capable of dividing indefinitely and becoming any cell type present in our body. Dr. Juan Carlos Izpisúa Belmonte, professor in Salk’s Gene Expression Laboratory, is the senior author of the paper, published in Cell, in which also participated Dr. Josep Maria Campistol, CEO and nephrologist in Hospital Clínic, and researcher in IDIBAPS. Scientists from Universidad Católica de Murcia, among others, have also been involved. Read the rest of this entry »

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Thursday November 24th, 2016

Two Clinic professionals receive the Fundació Catalunya-La Pedrera “Talents” grants

Last Wednesday, November 23, was held in the Auditorium La Pedrera the 5th ceremony of delivery of the  Fundació Catalunya-La Pedrera “Talents” Program grants, in which Hospital Clínic participates for the first time with the granting of two Scholarships. The event was chaired by the Health Minister of the Generalitat de Catalunya, Antoni Comín, who was accompanied by Ms. Marta Lacambra, CEO of the Catalunya-La Pedrera Foundation; Dr. Jordi Ara, ICS North Metropolitan Area Manager; And Dr. Josep Mª Campistol, CEO of the Hospital Clínic, who was also chosen godfather of this year’s promotion.

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Wednesday November 23rd, 2016

Successful participation in the commemoration of the 20th anniversary of IDIBAPS

Last Friday, November 18th, the celebration of the 20th anniversary of IDIBAPS was held. About 300 people gathered in the inner courtyard Beatriu de Pinós to listen to the speeches of Ramon Gomis, director of IDIBAPS, Francesc Subirada, general director of Research, Josep M. Campistol, general director of Hospital Clínic, Jaume Reventós, responsible of the Operations and Institutional Relations Department of the General Directorate of Research and Innovation of the Department of Health and Elías Campo, research director of Hospital Clínic and director of the Clinic Foundation for Biomedical Research.

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