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The biorevolution — the effective interaction of technology and medicine — is gaining momentum. We tell you what breakthroughs have already happened, and what are possible in the near future — and how they will change approaches to the treatment of diseases
Possibilities of biorevolution
Stefan Olrich, Member of the Board and Head of the Pharmaceuticals Division of Bayer, in his speech at the Nobel Vision forum, said: “We are facing a huge breakthrough, which we call the biorevolution. We combine modern science with digital technologies to provide patients with new medicines and treatments.” The use of some of the technologies that can be attributed to the biorevolution in medicine is not new, but in recent years it has yielded new results and made it possible to respond to current challenges. The most striking example is the development of anti-covid vaccines based on messenger RNA. To create them, it was necessary to decipher the genetic material of the virus, and modern technology made it possible to do this very quickly.
Another manifestation of the biorevolution is in the development of personalized medicine. Rapid progress in the field of genetics led to its appearance, including through the search for genetic markers associated with the development of various diseases. The main principle of such medical care is the selection of individual treatment regimens, taking into account the characteristics of each person.
“A personalized approach relies on the analysis of specific markers, such as gene fusions, and the subsequent selection of targeted drugs, which gives patients a chance to recover,” says Stefan Olrich. This approach may be more effective than current standardized therapies, and even help to fight diseases that are now considered incurable.
Gene therapy as a manifestation of the biorevolution
Thanks to research in the field of molecular biology, genetics, biotechnology and genetic engineering, the idea of gene therapy was born – the use of genetic material to treat diseases. For example, thanks to gene therapy, it is possible to introduce a “healthy” copy of a gene into cells, which will restore the function of an organ and cure a hereditary pathology caused by a mutation in DNA.
In 1984, a method was first developed for delivering “healthy” DNA to target cells, based on the use of a viral vector. The fact is that viruses can effectively penetrate cells and embed their genetic material there. When a viral particle is used as a vector, specialists make significant changes to it: dangerous infectious genes are removed, and “healthy” genes that can potentially stop the disease enter the cell instead. The technology of viral vectors is actively developing to this day.
Now, several more methods are being developed to change the “wrong” genes that lead to pathologies: for example, liposomes (special lipid nanoparticles) for delivering DNA directly into the cell. Particular attention is drawn to the famous CRISPR / Cas9 technology, for which its creators received the Nobel Prize – the ability to remove or replace incorrect sections of DNA due to a mechanism discovered by scientists in bacteria: when a virus attacks, a bacterial cell “remembers” its genes, and during the next attack it produces special Cas proteins, which, in combination with a special guide RNA, are able to “recognize” the virus and destroy its genetic material. Based on this mechanism, the CRISPR/Cas9 technology was created, which is a promising direction in modern genetic engineering. Scientists are widely studying approaches based on CRISPR / Cas9 systems – it is possible that in the future these approaches will find application in medicine for the treatment of cardiovascular, oncological and other diseases, the possibilities of therapy of which are still limited.
Gene therapy research is supported by companies such as Bayer. In 2020, Bayer acquired AskBio (Asklepios BioPharmaceutical), one of the leading companies in the development of gene therapy. AskBio technologies are based on the adeno-associated viral vector (AAV) and are already bearing fruit: a new treatment for Parkinson’s disease is now in the first phase of clinical trials. A potentially new therapy could help patients regain motor activity by delivering a gene encoding neurotrophic factor, a protein that promotes nerve cell regeneration, to neurons. In addition, clinical trials of LION-2022 are planned for the treatment of limb-girdle muscular dystrophy (LGMD) type 101I/R2 in the first half of 9. This is a rare genetic disease that manifests itself in childhood, which can lead to disability.
Cell therapy for the regeneration of damaged tissues and organs
The development of many chronic diseases leads to irreversible damage and loss of function of organs or tissues. In some pathologies of the heart, kidneys, lungs, liver, transplantation may be required – transplantation of donor material. But this method is not ideal – there are often not enough donors, and the transplant may not take root due to the patient’s immune response.
In addition, there is another serious problem: diseases for which only supportive treatment is possible. A potential solution to these problems is the use of cell therapy.
Cellular therapy is based on the enormous potential of stem cells – cells from which all cells of the body later develop. Potentially, they can be used to restore damaged structures: for example, the heart muscle, retina, or joints.
It is important to note that stem cells are already used in the treatment of oncological diseases – in the transplantation of bone marrow cells.
Once extracted, the stem cells are cultured in the laboratory and subjected to a number of modifications for specific needs – for administration to patients, they must develop into the required cell types. Pluripotent stem cells have the greatest potential – they can differentiate into almost any cell in the body and can potentially be used to regenerate almost any tissue.
There are many ethical issues associated with the use of embryonic material, and the possibility of reprogramming adult cells to give them the properties of pluripotent cells attracted the attention of researchers. In 2012, scientists Shinya Yamanaka and John Gurdon received the Nobel Prize in Medicine for the discovery of this technology.
There are many clinical trials being conducted all over the world on the use of pluripotent stem cells for the treatment of a wide variety of diseases: arthritis, damage to the heart muscle, ophthalmic and neurodegenerative pathologies. This is also one of the areas that Bayer focuses on. Since 2019, Bayer has owned BlueRock, whose researchers have used induced pluripotent stem cells to grow dopaminergic neurons and repair disease-damaged areas of the brain. The technology is currently in the first phase of clinical trials. “Patients will be able to receive dopamine on an ongoing basis and will not need additional medications. We hope to succeed in this study, as this approach can help improve the quality of life or save patients, achieve a full recovery,” says Stefan Olrich. In addition, BlueRock is actively exploring further applications of cell therapy in cardiology, immunology and neurology.
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