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Genetic therapies: what you need to know
Why are we talking about genetic therapies?
The first UK genetic therapy trials for CF will open later this year. This could mark a whole new era for CF treatment. We have already seen dramatic changes in CF treatments over the last few years with the development of CFTR modulator treatments like Kaftrio. However, nearly 10% of people with CF are not able to take these treatments.
Genetic therapies work very differently to current treatments for CF. They aim to produce functional copies of CFTR protein. While there are no guarantees with any new trial, genetic therapies could offer significant benefits for anyone with CF, including those not eligible for modulators.
Hope for newly diagnosed families
Respiratory paediatrician Dr Maya Desai works with children and young people in the Midlands. She is particularly conscious of the fact that revolutionary treatments like Kaftrio are not available to all.
She hopes these genetic therapy trials will give hope to people in her clinic who are not able to take modulators.
"Before the advent of modulators, we were able to speak positively to families of newly diagnosed babies and children about future advances. We did not know when this would happen but that time has come. In the same way, we can now provide hope to those families whose children are not already on a highly effective treatment" she said.
A life without limits for my daughter
Rachel’s 14-year-old daughter has CF and is unable to benefit from the CFTR modulators. She explains how this research gives her hope there could be a future treatment that can give her daughter a life without limitations. For her, clinical trials means that this is one step closer.
"Genetic therapy research gives us hope that she won't be left behind and that there could be something coming in the hopefully not too distant future that could benefit all people with CF regardless of their genetic makeup" Rachel said.
How can we prepare for genetic therapies?
Genetic therapies are a very new and different type of treatment. As genetic therapy clinical trials become a reality, it’s important that we are all as informed as possible. That’s why we have created a range of accessible resources to explain what genetic therapies are and what these trials will involve.
Our new resources include:
- FAQs
- Q&A video with genetic therapy experts
- A genetic therapy glossary
- Training resources for CF professionals
We hope these resources will be useful whether you’re deciding to take part in a trial, supporting others who might be, or hoping to stay up to date with the latest developments.
The importance of accessible information
Michelle Shore is a Trustee of Cystic Fibrosis Trust. She also has cystic fibrosis and is unable to benefit from modulators.
She is interested in the prospect of genetic therapies as for her it offers a route to better health and a less stressful future for her family.
"I cannot stress enough how important accessible information is for me", Michelle said. "I read every article about medical developments that I can get my hands on. I watch every video, and I bombard my CF team with questions. When you know what’s happening in the background, the feeling of helplessness reduces, slightly."
How we’re supporting trials
The Trust will be supporting the delivery of genetic therapy trials through the Clinical Trials Accelerator Platform (CTAP) and by continuing to fund early research into genetic therapies.
Jess Longmate, Head of CTAP, explains: “We’ve recently extended our CTAP network to ensure we have the expertise and resources to deliver this type of trial safely. We are also developing our Business Development Strategy to make sure we continue to have a strong pipeline of genetic therapy trials in the UK.”
Our Trials Tracker lists all CTAP trials before they open. You don’t necessarily need to be registered with a specific clinic to enroll. If you see a clinical trial of interest on the Trials Tracker, click the ‘Get in Touch’ button within the trial page to ask the study team about taking part.
CF LIVE event – ask a genetic therapies expert!
Join our CF LIVE online event on Tuesday 13 June at 7pm to have your questions on genetic therapies answered. The event will include a Q&A discussion where our panel of genetic therapies experts will answer your questions.
Register for the event today to submit your questions in advance:
Register for CF LIVE on genetic therapies
Find out more about cystic fibrosis clinical trials and the Clinical Trials Accelerator Platform. You can also find a list of current trials on the Trials Tracker.
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