On Tuesday 7th November, the SciDF discussed about new biomedical technologies to better understand their potential and how to manage these new therapies.
Gene therapy is the scientific and medical approach on modifying genetic material in order to provide a therapy. Current diseases that are being addressed range from immune or metabolite deficiencies to retinopathy, haemophilia or cancer, even though complex diseases are more difficult to target. In fact, the main goal is to target cells using external genetic material to transform their genomic content.
Since the 1950s, with the discovery of DNA and knowing the fact that some bacteria exchanges genetic material through the so called bacteriophage viruses, the gene therapy concept came to the scientific minds. The first cure treatment was developed for the β-thalassemia disease, by inserting β-globin gene that patients lacked. However, it was not exempt of criticism in society as the technique was still on its first stages. With more expertise in the current days, lentiviruses (retroviruses, like HIV, attenuated) and adenoviruses are the main vectors used to transform targeted cells.
To consider recent cases, take the example of lipoprotein lipase deficiency in pancreas. The patients follow a very strict diet since they cannot digest some lipids. But there was a clinic assay to treat this and cure was provided for free in exchange of taking part in the trial. Later, this gene therapy was approved and made public under the name Glybera. However, the costly price of Glybera, 1 million € , caused a drastic decrease in the number of patients being treated. Imlygic and Strimvelis are yet other examples of this socioeconomic challenge that an approved treatment faces, while Kymriah or Luxturna have been better introduced to society.
Gene therapy so far can be used to cure but only to palliate in polygenetic-caused diseases. It could also become a tool to adjust personal treats like predisposition to obesity or to boost some genetic features, leading to an ethical debate.
A challenge that remains for most treatments is being able to target with the highest specificity. While some procedures add a gene to the cell, more complex ones could potentially substitute them. With this substitution, there would not be genotoxic or secondary effects due to a rude technique, but only the specific gene modification.
Spain is the 10th country in the world ranking on easiness to perform clinical gene therapy assays. Dr. Ramón Alemany expressed his concern on balancing risks and benefits on treatments, which should be achieved by a thorough patient follow-up. Prof. Itziar de Lecuona agreed on that and explained the role of Bioethics in Action, which mainly seeks to evaluate treatment studies and cases on the basis of current professional perspective and far from old, not appropriate laws.
The talk followed by highlighting the role of ethics committees. Those who decide what is suitable to be legal or not should have a strong background on their field, like the most up-to-date gene therapy or international laws, so committees are formed by a multidisciplinary group. At the same time, they must be economically and politically independent, since they decision on funding affects equilibrium on public and private initiatives.
Taking a look on ethics laws, the Oviedo Agreement (1997) is a reference to follow. The human genome must not be edited nor the germinal line (which affects the progeny), while the somatic line (which doesn’t affect the progeny) is allowed to be experimented on. However, this biomedicine agreements should be international and, currently, there are some parts of the world where this basic ethic rule is misrepresented or questioned. China is an example where some CRISPR-Cas treatments have been applied to humans, something impossible in the UE because of regulations.
While provoking debate on public assistants, the speaker told again that a sense of solidarity has to be always active in gene therapy issues without forgetting that we live in a market society. The event finished with a discussion on the ethics committees, the interests and resources of the private and public sectors for promoting therapies and the risk-benefits balance.