Stem Cell Therapy Research

Those investigating the use of stem cells to treat diseases will often make use of pre-clinical data to identify potential treatment possibilities. What his means is that the researchers look at laboratory studies showing the behaviour of stem cells in different circumstances and consider these behaviours in light of the underlying pathology of a particular condition. For example, in the autoimmune condition of Multiple Sclerosis a patient’s symptoms are thought largely to do with a self-reactivity against the myelin sheaths surrounding nerve cells throughout the body. Although other aetiologies have been proposed for MS, such as focal axonal degeneration, and chronic cerebrospinal venous insufficiency, myelin destruction does seem to play a major part in the disease. Investigating the growth of new myelin in the laboratory would then be a potential treatment to reverse the symptoms of MS, and this type of therapy combined with a way of preventing the immune system from attacking the myelin could, theoretically, be a cure for multiple sclerosis.

Laborious Stem Cell Testing

However, before such claims can be made, a number of processes must be gone through which can take years to fund, conduct, and analyze at each stage. Laboratory tests looking at the behaviour of stem cells and their ability to differentiate into neural cells and myelin need to be conducted. Researchers will then need to observe any potential improvements or risks associated with the stem cells prior to implementing the therapy in living tissue. Animal models are often used to work out the best site, dosage, and conditions for a stem cell transplant (although this is not always representative of the reaction in a human patient). Finally, if the evidence looks good for clinical significance, a treatment protocol may be formed and a Phase I trial carried out to test dosage safety in human patients.

Phase I, II, and III Trials – What Are They?

Phase I trials do not constitute a basis for treatment, they only aim to ensure safety of a particular therapy and note any potential side-effects or necessary alterations in dosages. If a clinical trial passes Phase I (which usually takes about a year) and goes onto Phase II then the researchers begin to look at how effective the treatment actually is for the condition. Phase II can take anything from one to three years and researchers may discover that modifications are necessary which could then send them right back to Phase I trials for a different approach. If the treatment appears to be significantly effective then a Phase III trial will usually commence in order to compare the new therapy with an existing conventional treatment in order to assess clinical significance and, often, cost effectiveness.

Stem Cell Research Time and Costs

Research is costly, time-consuming, and can be terribly frustrating for the researchers themselves, and the patients they hope to help in the long-run. Some promising stem cell research may never make it through these three stages to be considered for approval as a therapy as funding may not be available, the science may lead the researchers in a different direction, or serious complications may arise forcing the termination or hiatus of the study. This was the case with stem cell research being carried out by Geron for spinal cord injury which was put on hold by the FDA after Geron reported finding a higher frequency of small cysts within the injury site in animals treated with their GRNOPC1 stem cell intervention. Further research, in animals, by Geron has led to the hold being lifted and the trial using human embryonic stem cells for spinal cord injury being back under way. During the time of the hold Geron developed several other research trials using their patented therapy for diseases such as Alzheimer’s and Multiple Sclerosis.

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