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ASCAT 2024 | Improving the accessibility of gene therapy: developing in vivo techniques
In this video, Annarita Miccio, PhD, Imagine Institute of Genetic Diseases, Paris, France, discusses the future role of gene therapy for treating patients with hematological conditions, highlighting that new techniques should be developed to widen the eligible patient population and to decrease the costs of this therapeutic approach. For example, Dr Miccio mentions the work of her team and other groups, which aims to develop an in vivo gene therapy technique that will allow patients to forgo stem cell collection and toxic conditioning regimens. This interview took place at the 19th Annual Scientific Conference of the Academy for Sickle Cell and Thalassaemia (ASCAT 2024) in London, UK.
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Transcript
Yes, I think this is the beauty of gene therapy is that it is a definitive option for the patient. So this is our goal for now. The current gene therapy strategies are very effective but still the patient may need to be followed up, they may receive other types of treatment, so for this reason we have to make them more efficacious, that’s for sure. But at least if we have an efficacious and safe therapy this will be definitive, which means that the patient comes once to the hospital and never anymore...
Yes, I think this is the beauty of gene therapy is that it is a definitive option for the patient. So this is our goal for now. The current gene therapy strategies are very effective but still the patient may need to be followed up, they may receive other types of treatment, so for this reason we have to make them more efficacious, that’s for sure. But at least if we have an efficacious and safe therapy this will be definitive, which means that the patient comes once to the hospital and never anymore. And so this is for sure a challenge. But in this case, if we offer a definitive therapy for patients, for sure there is a lot of room in the field of hematological disorders. But again, there are challenges, as we discussed before.
And the main challenge nowadays is really the cost. And so, indeed, that’s what we are working on. We are trying to develop strategies that are less costly. But also we have to think about other different systems that we could employ to widen the patient population that we could treat and reduce the cost and also reduce the cost of the technique that we are using. And if I can give you an example, for example, nowadays, most of the gene therapy for hematological disorders is based on the collection of the cells from the patient, the treatment in the lab, which is very costly, and they’re injected in the patient that receive a chemotherapy treatment. So this is a very complex, long, and heavy procedure, and also toxic because still you have a conditioning regimen that can lead to secondary malignancy.
And this is what we call the ex vivo gene therapy, but what we and many other groups are working on, and I will also discuss that, is an in vivo gene therapy in which we, for example, we just inject a viral vector or a nanoparticle in the patient without any conditioning. And this particle will be taught to go directly to the bone marrow where the stem cells are, so that in this case, the therapy will be just an injection and not a transplantation. And this for sure will reduce the complexity and the cost and the toxicity of the current gene therapy approach, so widening is use for many more patients.
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