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Dr. Amparo Solé talks about treatments for people with Cystic Fibrosis without the F508del mutation. Discover all the lines of research that currently exist in this new informative capsule.
Dr. Amparo Solé, a reference in Cystic Fibrosis
In this new informative capsule we once again have the company of Dr. Amparo Solé, rom the Transplant and Cystic Fibrosis Unit of La Fe Hospital in Valencia and to whom we awarded the 2022 Rose of the Sea award, to talk about the modulators for people with CF who do not have the F508del mutation.
Research with Kaftrio
Research is currently underway to find an effective treatment for people with Cystic Fibrosis who do not have the F508del mutation. According to Dr. Solé, Kaftrio is being tested against 18 different mutations, including some ending in X, and has been shown to be effective in in vitro tests. This study is already in the extension phase, which means that the results will be published shortly.
Messenger RNA research
Gene therapies, such as messenger RNA, are also going to begin, with three centers in Spain that will start the trial shortly, in order to have results in about four years. Dr. Solé also talks about scientific studies to provide genetic material to modify the mutation, although this is still in phase 1, that is, at a very early stage.
Application of Kaftrium in people without the F508del mutation in severe cases
Dr. Solé is in favor of applying Kaftrio in those people who do not have the F508del mutation and are in a critical situation, but who have mutations for which in vitro tests have shown that this drug has worked. It should be remembered that in Spain it is only indicated for people with the F508del mutation, so a special request must be made to be able to administer it in these cases.
Hopeful message
Finally, Dr. Solé wants to send a message of hope, because there is a lot of funding for research into treatments for people who do not have the F508del mutation and results of several studies may soon be published, not to mention messenger RNA therapy that will help patients with other severe mutations to be able to produce the CFTR protein.
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