What diseases can be cured at the cellular level
Checkered recipe
Ekaterina Pichugina, Moskovsky Komsomolets, 11.08.2012
The possibilities of using cellular technologies in medicine today pay great attention all over the world. And while the official use of the same stem cells in Russia is limited mainly to the treatment of malignant blood diseases and burns, the times are about to come when many diseases will be treated with cells.
For example, recently in our country they approved the method of using fat stem cells ("waste" liposuction) for a facelift and breast augmentation, whereas in America they are ready to use it even for the treatment of diabetic foot and heart attacks.
Well-known world scientists told the MK columnist about new achievements of cell therapy during the international symposium "Topical issues of gene and cell technologies" held in the capital.
Will stem cells defeat HIV?A case that occurred a few years ago at the Berlin Charite clinic gave scientists hope that a deadly disease can be defeated with the help of cellular technologies.
It was discovered by accident. A patient diagnosed with HIV was treated for another terrible disease that he also had – leukemia (blood cancer). In such cases, doctors often do a bone marrow transplant. The man was transplanted hematopoietic (hematopoietic) stem cells, in which the CCR5 gene was mutated (5% of the world's population has this). It was found that cells with such a mutation do not have a receptor that ensures the penetration of the immunodeficiency virus into lymphocytes. Bottom line: in addition to the fact that the patient's hematopoiesis was restored (which was supposed to happen), he unexpectedly... got rid of HIV. As doctors say, "the virus has lost a substrate for parasitizing, which was manifested by clinical recovery."
And now serious research is underway in the field of the possibility of treating the immunodeficiency virus using stem cells with a mutation in the CCR-5 gene. However, there are very few people with a natural mutation that prevents HIV infection, and their donor potential is very limited. What to do?
German professor Axel Zander came up with the idea of genetic modification of hematopoietic stem cells of bone marrow and umbilical cord blood. A mutant gene can be inserted into donor cells, and the result is the same: HIV loses the ability to penetrate into these cells. This approach opens up fantastic prospects in the treatment of AIDS. Clinical studies of this method have been going on since 2009 at the German Eppendorf Clinic under the guidance of Professor Zander.
– Professor Zander is a very serious doctor and researcher, and the work he is currently engaged in is extremely interesting, – says corresponding member of the Russian Academy of Medical Sciences, Professor Alexander Rumyantsev. – If this approach shows its effectiveness, it can become very promising for modern treatment of patients with HIV.
The body is in business Previously, stories about how fat pumped out of the butt can fight wrinkles on the face or increase breasts seemed anecdotal.
Now they have become an ordinary reality. Mark Hedrick, one of the developers of the technology, a well-known doctor, the head of the American company Cytori Therapeutics, told the observer of "MK" about how the adipose tissue stem cells remaining after liposuction can be used for the benefit of mankind. From 1998 to 2005, he directed the Regenerative Bioengineering Laboratory of the Department of Surgery in Los Angeles. The genius invention of him, a plastic surgeon, was prompted by a passion for waste-free production: "It was just a shame to throw away the fat remaining after liposuction, and we started thinking about how to use it," says Professor Hedrick. – A detailed study of adipose tissue stem cells turned my idea of fat upside down: after all, adipose tissue has unique properties that can be in demand in various fields of medicine! In addition, the stem cells obtained from it do not need to be grown – they can be used immediately while they are fresh." So, from 120 ml of fat, 5 ml of processed valuable stem cells are obtained; this is 2.5 times more than from the same amount of bone marrow.
The result of 10 years of research by the professor was the development of special equipment that allows to obtain cell populations directly in the process of liposuction, which can be used immediately. Today they are used for so–called lipofilling - correction of cosmetic defects of the face and body, rejuvenation and creation of new contours with the help of injections of their own adipose tissue enriched with stem cells. In addition, the technology is used to increase or correct the breast – and note that no silicone implants are required for this, while the "new breast" from fat takes root much better. In addition, the patient can simultaneously perform liposuction of fat from the hips and increase the breast. Today, such devices are available in 250 clinics around the world, and they have recently appeared in Russia (including in the Burnazyan clinic).
As the director of the Institute of Human Stem Cells Artur Isaev notes, "the development of methods for hardware cell isolation is one of the trends in the development of modern cellular technologies. This approach eliminates the problems with the delivery of the material to a remote laboratory, its sterility and reduces the number of patient visits to the clinic. Within 2-3 hours in the operating room, the patient can undergo liposuction, isolate fat cells and adipose tissue stem cells and immediately use a fat graft for reconstructive operations. It is for such technologies that the future lies."
Professor Hedrick is currently conducting clinical trials of the use of adipose tissue stem cells in the treatment of cardiovascular diseases, stress urinary incontinence, diseases of the gastrointestinal tract, therapy of degenerative processes of intervertebral discs, as well as for wound healing. After all, these cells have regenerating – healing and tissue–restoring properties. "This technique is already being used in reconstructive surgery and the treatment of various types of soft and hard tissue injuries; it is beginning to be used for vascular repair and treatment of diabetic foot," Mr. Hedrick shared with MK.
Three parents will save from genetic diseasesShukhrat Mitalipov, our compatriot, now living in the USA, has become one of the leading embryologists of our planet today.
In 2007, he woke up famous after performing the world's first therapeutic cloning of a primate (male macaque). Two years later, he announced the development of a new technique for the treatment of genetic diseases by replacing "corrupted" genes in eggs. So far, it has been studied only on primates, but just a few years – and people can get a new chance to escape from deadly genetic diseases. Mitalipov now heads the Oregon Laboratories Division of Reproductive Sciences.
The technology he invented provides for the treatment of genetic diseases even before birth, at the cellular level. "Defective genes containing the disease are transmitted to the child through the germ cells of the parents – sperm and egg. Our idea is to correct the "corrupted" genes and, with artificial insemination, get an absolutely healthy child who himself will bring healthy offspring in the future. The idea seems to be simple, but until now scientists had no idea how it could be implemented and how to "fix" a gene with a disease in a single cell," Mitalipov told MK. According to the scientist, part of genetic diseases (more precisely, 250 syndromes) are stored in the genes of cellular mitochondria. Basically, we are talking about hereditary neuromuscular pathologies. If you replace a defective gene in an egg with a healthy donor one, the child will be spared from genetic diseases. According to Mitalipov's calculations, the first child who will be able to "fix" the defective gene in this way will be born in three years. "Once I was told that what we are doing is simply impossible and unworkable. I proved the opposite. Science is developing by leaps and bounds, so I think our technology will soon allow mothers who have a high risk of inherited mitochondrial mutations to have healthy children," the scientist says.
...The use of cellular technologies for the treatment of hereditary diseases is also of concern to the famous Japanese scientist Mitsuo Oshimura. The Director of the Japanese Chromosome Design Center presented to the attention of Russian scientists the technology of creating and using artificial human chromosomes and stem cells for the treatment of rare diseases. However, so far only experiments on mice speak about her achievements, but this is encouraging for the scientist.
Last year, Professor Oshimura published the results of a paper that for the first time described the possibility of using artificial chromosomes for the treatment of Duchenne myodystrophy (a disease in which muscle tissue atrophies). The disease is caused by a mutation of the gene responsible for the synthesis of the protein dystrophin, necessary for the normal functioning of the muscular apparatus. Almost always, the victims of this disease are boys who do not manage to live to the age of 25.
Oshimura's studies on mice have shown that gene therapy using artificial chromosomes can normalize the work of animal muscles. The bottom line: an artificial chromosome with a DNA fragment without mutation is created in the laboratory. It is placed in a stem cell, which acts as a vehicle for the correcting gene. In the process of subsequent cultivation, cells are obtained, the transplantation of which can restore organs and tissues whose work is disrupted by hereditary diseases. Experts consider the technology very promising, because with its help, a large amount of DNA can be injected into cells without destroying the existing genome.
– Artificial human chromosomes can be of great practical importance for the treatment of a wide range of diseases, including hereditary ones. The main advantages of artificial chromosomes are practically unlimited capacity, as well as the possibility of controlled removal of these chromosomes from cells. The most convenient means of transferring artificial chromosomes into the patient's body are stem cells," says Corresponding member of the Russian Academy of Sciences, Head of the Laboratory of Molecular Biology of Stem Cells of the Institute of Cytology of the Russian Academy of Sciences Alexey Tomilin.
Oshimura himself told the MK columnist that his invention would not be widely used until 10 years later – first it was necessary to prove its complete safety.
Growing donor organs is just around the corner Italian Professor Paolo Macchiarini, head of the Department of Regenerative Surgery and Biotransplantation at the University Clinic of Florence, Professor of regenerative Surgery at the Karolinska Institute in Stockholm, four years ago for the first time in the world transplanted a tissue-engineered trachea to a patient diagnosed with tracheal cancer.
The organ was manufactured in a special bioreactor based on an artificial frame, into which the patient's own stem cells (bone marrow and nasal mucosa) were transplanted. In 2009, the same trachea was "grown" already inside the patient's body, without the use of a bioreactor. By 2010, 9 similar operations had been performed in Western Europe. In the same year, a tracheal transplant operation using regenerative medicine methods was also performed for the first time in Russia at the Academician B. V. Petrovsky Scientific Center (it was performed by Paolo Macchiarini himself).
Now Professor Macchiarini dreams of creating a technology for growing organs based on regenerative medicine. He is already working on the creation of bioengineered esophagus, liver, lungs, heart, pancreas. "This opens up the widest opportunities for transplantologists, since such operations do not require donor organs, many ethical issues are removed, the operation becomes more accessible," says the Italian professor.
This year, Paolo Macchiarini became one of the 39 winners of the Russian Government's mega grant to attract leading scientists to Russian universities. He led the creation on the basis of the Kuban State Medical University and the Regional Clinical Hospital of Russia's first research, educational and clinical center for regenerative medicine, which will conduct research on the regeneration of the respiratory tract and lung. In addition, in 2012, for the first time, two tracheal transplantations were performed in Russia, created on the basis of a nanocomposite framework using the patient's own cells.
... Regarding the possibility of growing a human brain from stem cells, Professor Macchiarini is not sure what he said in an interview with MK: "We should not expect that tomorrow we will create a full–fledged brain - this is neither technically nor ethically impossible. However, we will be able to grow some segments of the brain in order to restore its function, which will help in the fight against Parkinson's disease, some neurological diseases."
Portal "Eternal youth" http://vechnayamolodost.ru13.08.2012