Potential role of stem cells in the therapeutics for major diseases - Printable Version
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Potential role of stem cells in the therapeutics for major diseases - Kamat2010 - 11-01-2012
Stem cells have shown great promise in the treatment of a number of major diseases. Their unique properties of differentiation have made their studies a widely researched area. Although, there are some ethical issues surrounding the subject, the development in this area can offer solutions and therapeutics for many fatal diseases with minimal complications. However, in-depth research and scientific testing on human trials is essential before it can have wide application for treatment throughout the world.
Heart diseases are one of the major diseases affecting majority of the population all over the world. The inability of the heart cells to regenerate and repair themselves after the occurrence of a heart attack prevents the application of further treatments on such patients. In such cases, stem cells have helped in regenerating damaged heart muscle cells by the introduction of adult stem cells from non-heart tissues into the damaged heart. Hence, they play an important role in the treatment of heart diseases.
Neuronal diseases and spinal cord injuries have always been a cause of fear for people as the treatment of these diseases are quite complicated and their complete cure is quite difficult to attain. In this area also, stem cells have made a major mark with proof of successful studies in rodent models. The condition of paralysis or impairment of motor function due to spinal cord injury was found to be reversible to perfectly normal condition with the introduction of embryonic stem cells. In a motor neuron degenerative disease called Lou Gehrig’s disease also, it was found that the introduction of embryonic stem cells helped in the restoration of the function of motor nerve cells. However, all these studies were found to be successful in the rodent models; hence, they remain to be translated into the human studies. Stem cells have proved successful in the treatment of Parkinson’s disease, in which they were able to restore the secretion of neurotransmitter, dopamine, with the transplantation of the stem cells. However, there were possibilities of hypersensitivity to increased dopamine levels. Hence, detailed study is necessary before it can be translated into potential treatment.
Stem cells have provided a path for the treatment of the degenerative retinal diseases. The successful differentiation of the stem cells into photoreceptor cells and the introduction of these photoreceptor cells into the retina of the patients helped in restoring the vision of the patients, thereby preventing complete blindness. The differentiation of the stem cells into specialised pancreatic cells with the ability to secrete insulin has provided a gateway to the treatment of diabetes associated with impaired insulin secretion due to the destruction of the pancreatic β-cells by the immune system.
The stem cells have played a major role in the treatment of cancer like leukaemia and this is one of the successful applications of stem cell based therapies. The disease of Leukaemia affects the white blood cells, which become cancerous and lose their normal functioning. The ability of the normal white blood cells to fight against different infections and protect the body is lost. This results in the vulnerability of the body to develop infections as the immune system is compromised and the functionality of the different vital organs within the body is lost. The bone marrow transplant along with chemotherapy helps in replacing the damaged blood cells with fresh blood cells. The patient’s damaged cells are removed by chemotherapy and the new stem cells introduced by transplantation give rise to new blood cells, thereby restoring the functions of the blood cells. The introduction of new refinements in the technique has removed the necessity of a match between the donor and the patient’s bone marrow. The multiplication of the stem cells in the donor blood to high concentrations has removed the need for matching the donor and patient, thereby improving the patient outcome, and helping in the eradication of cancer in future.
New research studies have shown the reattachment of the lost teeth and the growth of new teeth in place of lost ones with the help of stem cells within the patient’s body, thus opening a new area of research in the field of dentistry. The ability of the stem cells to repair bones and also regenerate lost bones within the body has demonstrated the remarkable progress in the stem cell research. Thus, stem cells research has become the future for different therapeutics and can be the ultimate solution for decreasing the suffering and increasing the mortality of the mankind.
RE: Potential role of stem cells in the therapeutics for major diseases - BojanaL - 01-25-2013
Stem cells could accelerate drug development for Parkinson’s disease
Parkinson’s disease is progressive neuromuscular disorder that (usually) affects people over the age of 50. Most prominent signs of the disease are tremors, trembling of legs, arms, jaws, bradykinesia and impaired movement coordination. Since disease is progressive, most patients eventually loss ability to complete their usual daily activities and require special care. Parkinson’s disease affects both sexes, but men have slightly higher chance to develop disease. People all around the world suffer from Parkinson’s disease. It is estimated that 7 to 10 million people throughout the world live with the disease. Just in USA over million people is diagnosed with Parkinson’s disease, and this number overcome total number of all USA patients diagnosed with Lou Gehrig’s disease, multiple sclerosis and muscular dystrophy. Parkinson’s disease is expensive, both for the patients and for the economy of the country that supports the patients. Estimated individual cost are around 2500 dollars per year, but surgical interventions can rise the price to 100 000 dollars per person. Special care, medications and inability of patient to work, cost economy of USA 25 million dollars per year. At the moment, there is no cure for Parkinson’s disease; few medications are used to alleviate the symptoms of the disease. Main goal of various scientists around the world is to find a drug that could stop disease progression.
Parkinson’s disease develops sporadically or as consequence of mutated genes. Around 15% of Parkinson’s cases are inherited (already recorded cases of disease in family), and the rest of the cases are result of still unclear combination of environmental and genetic factors. Part of the brain affected by the disease is substantia nigra, where dopaminergic neurons are located. These neurons release dopamine and induce smooth muscle movements. When 50-80% of these neurons die, loss of muscle control and impaired movement in general will become obvious.
Latest experiments with stem cells could accelerate development of efficient treatments for Parkinson’s disease. Those cells allow scientists to track disease development and its progression directly in a dish. Skin cells isolated from patients will be transformed into induced pluripotent stem cells or iPS, using several (in normal circumstances silent) transcription factors that will reverse adult cell to the pluripotent stage. When this phase is accomplished, stem cells will be guided to mature into neuronal cells. Healthy cells will be turned into typical Parkison's neurons so that impaired cellular processes and potential beneficial effect of new or already known drugs could be studied. Neuronal cells in Parkinson’s disease show impaired function of the mitochondrial system: altered energy production, sensitivity toward oxidative stress and increased sensitive to various toxins which normal mitochondria would survive. Although several genetic mutation located at 17 different location within the genome are associated with Parkinson’s disease, only couple of genes are closely examined. Two well examined mutated genes are LRRK2 and PINK1 and they are usually found in the patients with already noted cases of disease in family. Mitochondrial function is closely associated with LRRK2 and PINK1 genes; it was noted that oxygen consumption rate (turnover of glucose to energy) is lower in patients with mutated LRRK2 gene and higher in patients with mutation in the PINK1 gene. Mitochondria shows higher vulnerability toward oxidative stress in PINK1 mutated cells compared to LRRK2 cells. Both type of cells showed sensitivity toward toxins. Cells exposed to toxin were treated with few drugs to assess their potential to reverse toxic effect. Oxidative stress in the cells is normally prevented by antioxidants. Coenzyme Q10 is used in treatment of Parkinson’s disease and when tested in the dish, neurons carrying both types of mutations showed beneficial response. Rapamycin is immunosuppressant that could delay onset of neurodegenerative disorders such as Alzheimer’s disease. After exposing neuronal cells to rapamycin, only cells carrying LRRK2 mutation showed beneficial effect; rapamycin didn’t show any effect on PINK1 cells. This type of experiments showed that different types of mutations result in specific types (subgroups) of disease and that drug efficiency depends on genetic background of each patient.
Analysis of genetic background provides more information on disease type and could lead to personalized treatment of Parkinson’s disease. Although ideal treatment is still far away, stem cells in the research of Parkinson’s disease could improve future treatment options.