Instead of bioreactors and injections – genetically modified capillaries
Regular injections of drugs based on recombinant proteins can be replaced with grafts – vessels that will independently secrete the drug into the blood. Researchers from the Children's Hospital in Boston (Children's Hospital Boston) have shown that genetically modified vessels can synthesize medicine "on demand".
A bioreactor by itselfThe technology described in the new issue of the journal Blood can be used to treat patients with hemophilia, hepatitis C, multiple sclerosis and other complex pathologies whose treatment requires protein drugs, the authors of the study explain.
So, the cause of hemophilia (hereditary disorder of blood clotting processes) is a deficiency in plasma of one of the clotting factors (factor VIII or IX in hemophilia A and B, respectively). Therefore, the treatment of such patients requires proteins – coagulation factors VIII and IX.
Multiple sclerosis (MS) is a disease associated with the attack of the immune system on the cells of the brain and spinal cord, which in a healthy person are separated from the body's defense system by a blood–brain barrier. And hepatitis C is a viral liver disease. Interferons – also proteins - are needed to treat patients with hepatitis or MS.
"Recombinant proteins for the treatment of patients with these and other diseases are obtained in bioreactors using cell culture," explains Professor Melero–Martin, one of the authors of the study. But scientists believe that the patient can be his own bioreactor: "Why not make the patient's cells synthesize these drugs on their own?"
Among the patients who need expensive protein preparations are people with cancer.
The vessels worked like kidneysIn an article published in Blood, scientists present experimental confirmation that vessels can independently "do intravenous injections" – inject medicine into the blood.
In the experiment, Professor Melero-Martin's team treated mice with anemia – anemia. Scientists have grown blood vessels in animals capable of synthesizing erythropoietin, a hormone that activates the production of red blood cells (erythrocytes).
To do this, they used human cells lining the cavity of blood and lymph vessels and the cavity of the heart – endothelial cells. Scientists have introduced a gene into endothelial cells that triggers the synthesis of erythropoietin. The improved vascular cells started working like kidneys or an embryonic liver: in response to hypoxia, they began to secrete erythropoietin.
However, scientists did not grow a vascular network. The course of the experiment was somewhat different: they mixed modified endothelial cells with gel and stem mesenchymal cells (from the bone marrow stroma). Such a cocktail was injected into mice under the skin. Within a week, new vessels capable of working "kidney-like" grew out of the suspension of cells and gel.
Experts have confirmed that the new vessels eliminate anemia caused by radiation and kidney pathologies. Now, the researchers hope, we can seriously think about how to replace regular injections with new vessels.
Details of the experiment can be found in the article Induction of erythropoiesis using human vascular networks genetically engineered for controlled erythropoietin releases.
Brief description of the work (Engineered, drug-secreting blood vessels reverse anemia in mice) published on the website of Children's Hospital Boston – VM.
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