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Lidia’s story – Stem cell therapy for cerebellar spinocerebellar ataxia

Lidia was born prematurely at 29 weeks gestation. In her first year of life, the girl was diagnosed with spinal-cerebellar ataxia and Hirschsprung's disease. Spinal-cerebellar ataxia is a rare neurological disorder characterised, among other things, by difficulties in motor coordination. Hirschsprung's disease is a rare, congenital disorder of intestinal innervation that is genetically determined.

Umbilical cord blood saves girl with severe pulmonary hypertension

Stem cells derived from umbilical cord blood are used as standard treatment for 80 diseases. In recent years, there have been new reports about the use of these cells for additional diseases. Doctors in Germany successfully used them to treat pulmonary hypertension in a 3-year-old girl. Pulmonary hypertension - what is it? Pulmonary arterial hypertension (PAH) is currently an incurable disease. It leads to death within about five to seven years after diagnosis. The disease is named for the increased pressure in the pulmonary vessels and can lead to overload and failure of the right side of the heart. The causes of pulmonary hypertension can vary, but all patients struggle with shortness of breath or severely limited mobility, among other symptoms. For this reason, researchers are constantly looking for new ways and therapies to extend the lives of people with pulmonary hypertension and improve their comfort. Stem cells and pulmonary hypertension Doctors in Germany have announced the first success in treating pulmonary hypertension with cells from umbilical cord blood (HUCMSC - human umbilical cord mesenchymal stem cell). The patient was a three-year-old girl suffering from PAH and Rendu-Osler-Weber disease who was intravenously injected with allogeneic stem cells from her baby brother's umbilical cord blood. The injections were performed five times over a six-month period. The girl's clinical condition was assessed after two and six months. Not only were there no side effects, but there was also an increase in exercise tolerance and an improvement in cardiovascular parameters. Before the stem cell infusion, the girl suffered from growth disorders and weight gain. After the first infusion she started to grow - in three months she grew 10 cm. Moreover, the girl is now six years old and feels well. According to the researchers, the regeneration of the vascular system damaged by the disease, as well as the significant alleviation of cellular damage, are responsible for the improvement due to cord blood stem cell therapy. The study reports, among other things, the role of the prostaglandin PGE2, which may play a key role in the regenerative and immunomodulatory abilities of cord blood stem cells. The researchers unanimously emphasize that further research should be conducted and that additional opportunities and uses for cord blood-derived stem cells should be actively sought. Bibliography: https://www.aabb.org/news-resources/news/article/2022/07/13/cord-blood-derived-stem-cells-may-help-treat-pulmonary-hypertension https://www.sciencedirect.com/science/article/abs/pii/S1053249822018472 https://www.nature.com/articles/s44161-022-00083-z

Stem cell treatment for infertility

Infertility is a problem that affects about 15% of couples worldwide. For this reason, specialists from all over the world are constantly looking for new treatment options. One possibility seems to be the use of stem cells in therapy. Infertility problem The term "infertility" refers to the inability to become pregnant with regular sexual intercourse for at least one year (with some exceptions) without the use of contraceptives. The problem of infertility is very real today. Interestingly, the percentage of each type of infertility, i.e. male, female and idiopathic, is almost identical and is about 30% in each case. Although the clear causes for the increasingly frequent problems in childbearing are not known, many experts see air pollution and the high level of food processing as the cause of this phenomenon. Drugs used in chemotherapy for cancer treatment, for example, are also sometimes the cause of infertility. Despite the methods available to treat infertility and assisted reproductive techniques, many couples cannot wait to have children. Use of stem cells in the treatment of infertility In the context of infertility studies, scientists have wondered why females of certain species remain fertile throughout their lives, even though every woman reaches menopause at some point in her life. According to previous knowledge, each woman is born with a limited number of eggs that are no longer produced during her lifetime, but only "mature" into a fertilisable egg. Studies on mice have shown that the ovaries of females of this species contain stem cells from which eggs are constantly produced. Since these are constantly produced by the mouse body, the mouse remains fertile throughout its life. It was therefore suspected that similar cells (so-called germ cells) could also be found in the ovaries of women. With the help of very specific markers that bind to proteins found only on the surface of stem cells, it was possible to prove that they are also present in the ovaries of women. This discovery has led to new attempts to treat female infertility and to "stimulate" the production of new, fertilisable eggs with the help of stem cells. In addition, stem cells can also help women who suffer from scarring (atrophy) of the lining of the uterus, which prevents proper implantation of the embryo and contributes to infertility.Research into the use of stem cells in the treatment of infertility is being conducted by Dr Jaroslaw Kaczynski, a gynaecologist and endocrinologist, among others. As part of a research grant, he injected stem cells from the umbilical cord into the ovaries of 4 women struggling with infertility. Two of them became pregnant after a single injection of the cells. Although this research still needs to be studied in depth, it is very promising and could soon set a new trend in infertility treatment. Stem cells are also being researched in the treatment of male infertility. Here, too, experts have achieved considerable success in converting stem cells into male germ cells. Where else are stem cells used? By preserving your cord blood and cord, you have the opportunity to treat diseases that may affect your child or his or her siblings. For some diseases (e.g. leukaemia), preserving cord blood can significantly speed up treatment; for others, it may be the only chance of improving health. This is because umbilical cord blood and cord are rich in stem cells that build up the body and repair damaged cells and tissues. Stem cells derived from umbilical cord blood are currently used to treat cerebral palsy, amyotrophic lateral sclerosis, leukaemia and lymphoma. In addition, the cord blood can also be used to treat other family members if it is compatible. The material, the umbilical cord blood of the child, would be disposed of without the collection, so it is a kind of safeguard for the whole family. Stem cell treatment is still relatively new. For this reason, the list of diseases that can potentially be treated with stem cells in the future is growing.

Stem cells from umbilical cord blood cured HIV-infected patient

The news that a woman from the United States has become the third person in the world to be cured of HIV infection recently made the rounds. A disease that was previously considered incurable can be successfully treated with stem cells from umbilical cord blood. This is a great hope for both patients and researchers. . Current treatment of patients with HIV When HIV infection is detected, antiretroviral drugs are administered to the patient. HAART therapy is necessary for life, as it prevents viral replication and significantly slows the progression of the disease to full acquired immunodeficiency syndrome (AIDS). It also allows the immune system to rebuild and reduces the number of complications associated with the development of the disease. Unfortunately, the drugs used carry the risk of side effects and drug resistance. They also do not guarantee a cure. For this reason, researchers are still looking for a way to completely rid the person's body of the HIV virus and stop taking antiretroviral drugs. Why were stem cells transplanted in HIV patients? Stem cells were transplanted in both the earlier patients who were cured of HIV infection and the last patient because all three patients had cancer. In the last case, the patient was battling acute myeloid leukemia. Since it was difficult to find a bone marrow donor, it was decided to transplant stem cells from umbilical cord blood. After the transplant, immune antibodies began to be produced, and the HIV virus could no longer attack them and reproduce. This stopped the progression of the disease and eliminated the need for antiretroviral drugs. Stem cells from umbilical cord blood as hope for HIV patients The target cells in the human body for HIV include those that contain the CD4 receptors necessary for infection and the CCR5 coreceptors. The cure in all three cases is due to a mutation in the CCR5 gene, which "protects" against HIV infection. Both the cells transplanted into the American woman and the earlier patients contained this mutation, which results in a non-functional receptor. This prevents the HIV virus from attacking cells of the immune system, inhibiting its replication. Interestingly, this mutation is thought to be present in about 3% of the population, which means that this group of people is immune to HIV infection. What is the advantage of cells from umbilical cord blood over bone marrow? Two patients who have so far been cured of HIV infection have received stem cells from bone marrow. An American woman is the first person to be cured with cells from umbilical cord blood. Stem cells are present in bone marrow and umbilical cord blood, among other places. However, donors derived from cord blood have a lower risk of rejection and transplant complications due to their lower immunological maturity. In addition, their regenerative capacity is 10 times greater than that of bone marrow-derived cells. It also shortens the search for a donor, which, as in the case of the American woman described, can be very difficult because of race or origin. Because stem cells from umbilical cord blood are stored at very low temperatures, the risk of damage and aging is much lower than for cells from bone marrow. Whether stem cells from umbilical cord blood will become a widely used therapy for HIV-infected patients remains to be seen for the time being. What is certain is that the list of diseases for which stem cells can be used is growing. This, in turn, should dispel any doubts parents may have about collecting umbilical cord blood and storing their child's stem cells. Birth is a once-in-a-lifetime opportunity.

Vermehrung von Nabelschnurblut-Stammzellen als neue Hoffnung für Krebspatienten

Umbilical cord blood stem cells have been used in medicine for over 30 years to treat more than 80 different diseases such as leukemias, lymphomas and sickle cell anemia. In the field of regenerative medicine, the use of umbilical cord blood stem cells has also been shown to provide clinical benefits to patients. Because of its limitations, cord blood has been used more frequently in pediatric patients than in adults. However, the results of recent clinical trials show that the increased cord blood stem cells can also be successfully transplanted in adult patients. Umbilical cord blood stem cells are the hope of 21st century medicine. Currently, more than 3,000 studies are underway worldwide in various fields to explore the potential of stem cells. The results of a study on the use of augmented cord blood stem cells in the treatment of patients with blood cancers, published last week, show that cord blood stem cells grown in the laboratory and transplanted into patients are of significant clinical benefit. Omidubicel is the name of a preparation of hematopoietic cells from one unit of umbilical cord blood that have been propagated under laboratory conditions. A clinical trial evaluated its effectiveness compared with standard cord blood stem cell transplantation. The study lasted from January 2015 to January 2020 and included 125 patients aged 13 to 65 with blood cancers. Patients who received omidubicel achieved adequate white blood cell recovery 10 days earlier than patients who underwent standard therapy. They also had faster platelet recovery [up to 42 days vs. 90 days], a lower incidence of bacterial or invasive fungal infections (37% vs. 57%), and spent more time out of the hospital in the first 100 days after transplantation than patients in the control group. The proliferation of hematopoietic cells was the Holy Grail of hematology - an idea that, despite many attempts over decades, no one was able to realize. The results of this study show that the point has been reached where the number of cells no longer matters. Even more - the increased cells from cord blood can regenerate the hematopoietic system faster after transplantation. This could mean a wider introduction of this type of methods into clinical practice - says Emilian Snarski, MD, Medical Director of FamiCord Group. This is another study that shows the possibility of propagation of hematopoietic cells from cord blood and their effective use in the treatment of blood cancers. Thanks to such methods of proliferation of hematopoietic cells from cord blood, cord blood can be effectively used even if the number of collected cells was relatively small. Link to the abstract