The EU has granted 6.7 M€ from the Horizon 2020 research program to a broad collaborative group of researchers, doctors, pharmaceutical companies and patient representatives.
 
The aim is to develop ‘personalized treatments’ for Cystic Fibrosis (CF) patients with uncommon genetic profiles throughout Europe. These patients with extremely rare gene mutations can otherwise never get access to possibly life-saving treatment. Once this concept has been proven, the methodology can extend to all patients with CF and other rare genetic diseases, so that HIT-CF will impact the entire field of orphan diseases.

Approach

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 and distributed to laboratories in the Netherlands (Utrecht), Belgium (Leuven) and Portugal (Lisboa). This requires taking a few millimeters of tissue via a tiny biopsy, a painless procedure.
CF drug candidates from Galapagos, Flatley Discovery Lab and possibly other companies will be tested in these organoids to check for their efficacy in the tissue of the individual patient.
 
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) for current and future “off-label” treatments of people with CF. 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.
 
See this short movie
 
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The Website www.HITCF.org will be launched soon for more information