What's New in Clinical Trials

The CF community has seen the advent of disease altering treatments in the last few years and a spurred interest from researchers and companies for bringing even better therapies that will help the entire CF population.

This is incredibly exciting news for us as a community. Keeping up with the latest and understanding how these therapies could potentially help us will make us better prepared. We are eager to get these treatments through the clinical trial process and in our hands as fast as possible.

Why is clinical trial participation important?

Participating in a clinical trial is a way to play a more active role in your own health care. It also helps researchers and pharmaceutical companies determine whether a new potential therapy is going to be safe and effective for the community.

Believe it or not, many drugs in trials do not make it through to FDA approval. If the intended efficacy of the treatment was not met, the research of that particular drug can’t continue. It has to be reformulated or research has to start over completely. This takes a lot of money from biotechs and pharma companies who sometimes cannot get new investments for such research.

Contributing to the next generation

CF is a rare disease with a small population. Most new therapies come from small biotech companies that don’t have a surplus of funds.

Nonetheless, when you choose to participate in a clinical trial, even if they aren’t successful, you become a partner in scientific discovery! And, your contribution does help the next generation of people with CF to lead healthier lives.

New types of therapies on the horizon

In the last few years, gene therapy has rapidly gained interest and advancement. In CF, we can expect to see gene therapy available to us as a therapy option within the next few years.

Gene therapy

Gene therapy is different than gene editing. It is providing the corrected DNA fragment into the cells to make the CFTR work properly. This has to be continually dosed because the cells that are “fixed” with the corrected DNA die over the span of a few weeks and months. New cells that are made do not contain the corrected DNA.

Gene editing

Gene editing, on the other hand, is permanently changing the DNA in the stem cells to function properly. The new cells created from these permanently fixed cells will be properly functioning. Although this is ultimate goal to cure CF, this kind of research and treatments are still in the future. Currently there are a number of early research and phase 1 trials initiating in CF with gene therapy.

Fighting bacteria

In the infection research space, there are new treatments in trials examining methods. One is harnessing the immune system and antibody manipulation to fight off bacteria.Another is utilizing compounds such as nitric oxide and gallium to prevent bacterial replication.

Phage therapy

Phage therapy is also actively being researched as a viable alternative to antibiotics. It is the use of a specialized virus to kill bacteria in the lungs. This is a treatment I have taken part in myself.

In another article, I will share resources you can use to find out about trials and what trial participation involves!