Genoterapevtichesky medicines
Genoterapevtichesky medicines — rather new direction in the biotechnology industry. Advanced therapy medicinal products is translated into Russian as medicines of the advanced therapy, and represent the innovation medicines for medical application on the basis of gene therapy (gene therapy medicines), cellular therapy (somatic-cell therapy medicines) and tkaneinzhenerny products (tissue-engineered medicines). The main difference of genoterapevtichesky medicines consists that at single use perhaps absolute recovery of patients from the diseases which were earlier considered as incurable, including, and from oncological.
For 2020, gene therapy is one of the most expensive, however the weight of evidence suggests that over time treatment by genoterapevtichesky medicines will become available for a wide range of patients.
Genoterapevtichesky medicines for treatment of still incurable patients
For many incurable hereditary diseases by the beginning of 2020 it is not thought up effective treatment yet. For example, from hemophilia. Patients with this disease depending on weight of a status are forced to receive every week an injection of recombinant proteins which recover function of coagulability of blood. These are expensive and painful injections which, unfortunately, do not lead to treatment from a disease. And there are diseases from which in principle there is no therapy and genoterapevtchesky approach is the only way of therapy for 2020.
Many serious oncological illness has high resistance to standard chemotherapy and treatment by proteinaceous medicines. Development of genoterapevtichesky approaches will help to create effective medicines for treatment of such patients. And, of course, not only such socially important diseases as HIV infection, diabetes, but also a serious neurodegenerative illness against which there are no effective drugs yet are in focus of attention. Actually using genoterapevtichesky approaches it is possible to cure these diseases, but not just to offer the supporting therapy. The single injection is capable to replace with genoterapevtichesky medicine lifelong therapy.
As genoterapevtichesky medicines work
Essence of genoterapevtichesky medicines as this term it is defined in the right of EEU, comes down to delivering a piece of nucleinic acid, either DNA, or RNA in cages. These delivery systems should get to the necessary fabrics and provide reliable protection of nucleinic acid. The nature already gave us such tools, virus particles. Viruses are created by the nature to deliver nucleinic acid in cages. What is a virus? It is the proteinaceous capsule in which there is a fragment of genetic information. In normal lifecycle of a virus it gets to a cage, produces the DNA / RNA and in a cage new virus particles are acquired.
Respectively scientists used this mechanism of the nature and made the modified or recombinant virus particles which cannot be multiplied in a human body on the basis of different viruses and cause pathogenic statuses. But they can deliver a genome to target cages. Different virus particles are used depending on what effect and what type of gene therapy needs to be provided. For example, adenoassotsiirovanny viruses are the least immunogene virus particles and they can be applied to gene therapy of in Vivo – i.e. just to enter recombinant virus particles to the person for delivery of a therapeutic gene. It is absolutely safe method. Also there are virus particles which can be applied to approach ex vivo when cages are modified and then these modified cages are entered to the patient for achievement of therapeutic effect.
History
2019: Medicine for treatment of children with a spinal muscular atrophy is approved
In May, 2019 FDA approved the genoterapevtichesky medicine Zolgensma of Novartis company intended for treatment of children with the spinal muscular atrophy (SMA).
In the next years registration still of a number of genoterapevtichesky medicines for treatment of incurable diseases including in Russia is expected. Works on creation of the innovation genoterapevtichesky medicines are conducted today also by BIOCAD company, one of the largest international innovative biotechnology companies in Russia.
2017: Medicines for gene therapy of hereditary dystrophy of a retina and other diseases are approved
In 2017 the medicine LUXURNA, the first medicine on the basis of AAV for gene therapy of hereditary dystrophy of a retina was registered. The first genoterapevtichesky medicines on the basis of the modified T-cells of people (CAR-T therapy) for treatment of oncohematological diseases (a sharp limfoblastny leukosis, V-cellular lymphoma) Kymriah and Yeskarta were the same year approved.
2016: In Europe medicine for treatment of a severe form of the combined immunodeficiency is approved
In 2016 in Europe the genoterapevtichesky medicine STRIMVELIS for treatment of a severe form of the combined immunodeficiency connected with deficit of enzyme of an adenozindezaminaza (ADA-SCID) was approved.
2012: The first registered medicine
The way of development of gene therapy was long and difficult, complete of progress and defeats. One of early researches using adenoviruses led to loss of the patient, because of heavy ghost effects. This case slowed down development of gene therapy a little, but, fortunately, did not stop it. In 2012 there was a first registered genoterapevtichesky medicine on the basis of the recombinant adeno-associated virus (AAV) of GLYBERA for treatment of the rare incurable hereditary disease connected with deficit of a lipoproteinlipaza (LPL).
The 1990th: The first success of gene therapy - treatment of "the boy in a bubble"
The first success of gene therapy fell on the beginning of the 1990th when the group of researchers under the leadership of professor Andersen got permission to performing clinical trial for treatment of a severe form of the combined immunodeficiency connected with deficit of enzyme of an adenozindezaminaza (ADA-SCID). It is about so-called "bubble boy disease", or "the boy's syndrome in a bubble". This serious hereditary illness connected with severe violations in the immune system. Life of such patients is supported only in ideal sterile conditions, any infection can lead to death. Scientists took immune cells of such patient and using recombinant virus particles entered to them a missing gene which provided an expression of missing enzyme of an adenozindezaminaza, in cages of the immune system. Then these cages entered back to the patient, than provided his absolute recovery. This success urged on researchers to new researches behind which the boom of clinical trials of genoterapevtichesky medicines began.