Eloxx Pharmaceuticals Announces Participation in the HIT-CF Project on Cystic Fibrosis
The goal of the European HIT-CF project is to investigate whether a positive response to therapies in a patient derived organoid can be predictive of clinical response in a controlled trial. The project represents a new era in CF treatment and personalized medicine, as it has the potential to shift therapeutic trials from patients to the laboratory. The organoid model could be extended to all patients with CF and other rare genetic diseases to identify appropriate therapeutic options. The EU has granted
“We are extremely excited to be participating in the HIT-CF research project which has the potential to lead to treatment options for the many CF patients based on their functional responses in the lab,” said Dr.
About HIT-CF (www.hitcf.org)
HIT-CF is a European research project (H2020 - No 755021) which aims to provide better treatment and better lives for people with CF and rare mutations. The project is coordinated by Prof Dr Kors van der Ent of the University Medical Center
Adults with CF and a rare CF genotype reported to the European Cystic Fibrosis Society Patient Registry (ECFSPR), will be approached by their doctors and encouraged by the patient organizations (united in CF-Europe) to participate in the studies within the scope of this project. Firstly, stem cells from the patients’ own intestine will be grown into “mini guts” (termed organoids) by the foundation Hubrecht Organoid Technology (HUB) and distributed to laboratories in
Secondly, based on the effects in organoids, selected patients will be invited to participate in drug trials (organized within one of 43 CF-centers of the ECFS Clinical Trial Network and assisted by Julius Clinical) to evaluate the real life benefit of these compounds for these patients. HIT-CF aims to enable access to the most relevant drugs in development, and each trial group will test a drug candidate from one of the pharmaceutical consortium partners.
In parallel with this project, the pharmaceutical partners will work towards market approval of their drug candidates for the larger group of patients with more common genetic profiles.
The ultimate goal of this project is to develop a path for access to therapies for patient groups or individuals who show positive response to the therapy in an organoid test. One of the major impacts of this project will be the innovative methodologies used to acquire approval (and reimbursement. This will represent a new era in CF treatment as it implements a new type of personalized medicine based on organoids, by shifting therapeutic trials from patients to the laboratory.
Organoids are cell cultures that grow in a culture dish, and look similar to the organ from which they are derived. Intestinal organoids can therefore also be called mini-intestines. To make intestinal organoids for the HIT-CF project, rectal tissue samples (biopsies) will be obtained. This procedure is not painful and takes 5-10 minutes. Because organoids are made from stem cells, they contain the same mutations as the person from whom the biopsies are derived. The drug candidates target the basic defect of CF, and the organoids will be used to test on which mutations the drugs have a positive effect.
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Source: Eloxx Pharmaceuticals