Cystic Fibrosis

is a rare inherited disease that affects the chloride- and bicarbonate transporter CFTR.

CFTR-directed therapies

Small molecules to promote the processing, maturation and trafficking of CFTR have been sought for decades and two approved molecular therapeutics can be prescribed for CF patients in 2017. However, more than these two therapeutic molecules have been studied in the academic community, and the search for further therapeutic options to promote CFTR function is ongoing.

Our aim

We want to collect data on substances that have been tested as CFTR modulating agents and evaluate their relative potential. Because overarching structures are missing, no process on a harmonization of methods and read outs for the CFTR modulator research was initiated. Consequently, the research methods on CFTR modulating agents are heterogen and readout systems range from direct assessment of CFTR activity in excised cell membrane patches to indirect observations of salivary rates in animal models. Species studied range from human-derived cell cultures to various other organisms (mouse, rat, ape, hamster, ...). Merging of data will facilitate the interpretation of available data. Therefore we want to collect the published data and arrange it where applicable for an easier merging of the knowledge.
– this is our current collection: seed content


This is a community effort: You can join us and contribute (for instance, tell us if we have forgotten a drug that has already been shown to promote CFTR processing, function or activity). In the future, we will provide the opportunity to share data at this side, whereby we have a data sharing concept that will protect the intellectual property rights (under construction at the moment, currently, we welcome a registration of your contact details but cannot process any primary data).
In the future, when we can process primary data, you can decide whether other registered users can see your data at all, or you can decide on partial sharing if you want to keep the identity of the substance that you use private by using a non-chemical synonym.

If you decide to register so far unpublished data with us (even without naming the substance) we will provide in exchange:

Data sharing – a conflict of interest in itself

The project proposal resides in the area of conflict between the necessity to protect the intellectual property rights and the attractive opportunity to increase the value of the results through collaboration. The traditional means of communication within a scientific community are reflected within CandActCFTR: the data record within CandActCFTR can be compared with a poster display at a scientific conference and the virtual environment provided by CandActCFTR will promote an exchange of ideas within the scientific community in the same way as real meetings within a working group. Through data sharing, inactive compounds can be recognized earlier, and active compounds that work at several research sites will be recognized faster.

Sharing in the CFTR field has been successfully done before

If you are within the CF & CFTR community since the 1990s, you will remember that after the CFTR gene has been uncovered, newly identified mutations were shared prior to getting published: After the CF-causing gene CFTR has been found in the late 1980s, an initiative to collect data on novel CFTR mutations was launched by Lap-Chee Tsui of the Hospital for Sick Children in Toronto, Canada. Within the first year, a consortium of 130 members from 30 countries has been formed (Tsui LC, Dorfman R.: The cystic fibrosis gene: a molecular genetic perspective. Cold Spring Harb Perspect Med. 2013;3:a009472) and members have contributed their findings on mutations within the CFTR gene prior to publication in a scientific journal. The data was disseminated in the internet in as the Cystic Fibrosis Mutation Database in 1995 and currently attracts 500 users per day (Tsui LC, Dorfman R.: The cystic fibrosis gene: a molecular genetic perspective. Cold Spring Harb Perspect Med. 2013;3:a009472).

Who we are

CandActCFTR is maintained by three people and received advice from our SAB. We are funded by the DFG and strongly supported by the Mukoviszidose Institut gGmbH. We are not funded by the pharmaceutical industry, hence, we also welcome knowledge on substances that cannot be patented, have been developed for other purposes than CFTR directed therapies or are otherwise uninteresting from a commercial perspective. More info can befound here

Our projected related publications