Innovative Foam Technology Enhances Gene Therapy Delivery
Introduction
Gene-based therapies can potentially treat a wide range of genetic diseases by introducing specific cell function-altering genetic material into a patient. Using gene editing technology to make genetic changes in cells can correct disease-causing mutations or lower the risk of disease. However, in order to be effective, it is essential that gene delivery vectors deliver the genetic material to target cells.
Only a handful of gene-based therapies have been approved by the FDA for rare genetic diseases. The current FDA-approved in situ gene therapies expose the patient to the vector systemically or inject the vector into a confined space. In the small spaces, the vector has limited opportunity to leak therapeutic material meaning it is able to unload its DNA cargo into a maximum number of target cells.
Although this delivery approach appears promising, it is not feasible for most organs which are located within large body cavities where the vector would not be retained at the application site. Therefore, improving existing delivery technology and developing new delivery methods is a key step in paving the way for the next generation of gene-based therapies.
Medicated Foam
Foam has specific properties that make it a suitable candidate as a drug-delivery vector. Its tightly packed air bubbles are separated by layers of continuous liquid called lamellae. Therapeutic payloads become highly concentrated in the lamellae allowing foam to deliver highly concentrated doses of medicine to large areas even if the initial dose is small.
Furthermore, the foam is versatile and provides a controlled release of gene therapy agents at desired locations since it can be applied locally or injected to fill body cavities. This property ensures that the vector is evenly dispersed over a large surface area.
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The unique physiochemical properties of foam give it the advantages of high stability, sustained delivery at the target site, and very little to no leakage of the therapeutic agent to non-target tissue.
Application of Foams in Drug Delivery
A group of bioengineers at the Fred Hutch Cancer Center are leveraging the advantageous characteristics of foam to address the challenges associated with drug delivery. They reported that a foaming liquid is more effective than a standard liquid formulation at transferring gene therapy components to cells in lab studies.
Their study used a COVID-19 mRNA vaccine as a model to test the foam's efficacy. The team mixed mRNA with foaming agents such as methylcellulose to create the foam. Results of a mouse study demonstrated that when the foam was injected into mice it successfully delivered the gene therapy to targeted cells without notable side effects.
Matthias Stephan, Professor of Translational Science & Therapeutics Division, Fred Hutch Cancer Center, summarised the potential applicability of foam: "Our gene therapy foam shows for the first time that by taking a small amount of an expensive gene therapy drug, increasing its volume by embedding it in a solution that is mostly made of densely packed air bubbles and then applying it to cells, we can achieve a strong and safe transfer of gene therapy agents to cells."
Stephan anticipates that this gene therapy foam could treat cancers in confined spaces such as ovarian, pancreatic, and gastrointestinal cancers. This could be a critical stride towards treating gruelling diseases with unmet needs.
Conclusion
Although this study is still in the proof-of-concept stage, the researchers predict that this gene therapy strategy could soon be quickly adopted into clinical practice to tackle challenges that hinder the application of gene therapy.
Given that gene therapies are the most expensive treatments, the use of foam could improve patient accessibility and reduce the costs of gene therapies. Since the first batch of gene-correcting drugs is already displaying limitations, this successful project shows that the application of foams in the drug delivery space deserves more attention and exploration.
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