iPSC Learning

Research

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         As you may recall from all the science classes, research starts with a question, or a topic of interest. Scientists generate a hypothesis regarding to the question, design experiments to test the hypothesis, generate results and draw conclusions from the findings. With more information, scientists cycle through the process again.

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         For the development of treatments, scientists start with a general research on the physiology and mechanisms behind a disease. For example, in order to develop a iPSC treatment for macular degeneration, researcher might start with generating retinal tissue for the study of ocular morphogenesis or disease modeling. At the same time, they might also design and improve protocols for iPSC reprogramming and differentiation.

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         After the general research, scientists start to use the findings from disease modeling to develop potential treatment methods by testing in animal models. For example, they might test iPSC-derived retinal tissue graft in a murine model to study its efficacy.

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Clinical Trials

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         Once research shows promising results, researchers may take one step forward and bring the treatment to clinical trials, or human testing. Clinical trials are monitored closely by institutional groups and government agencies to ensure that the experiments are safe and ethical. There are two levels of regulation:

• Institutional

  • Clinical trials need to be approved by Institutional Review Boards (IRBs) before any experiment takes place. IRB consists of scholars with expertise in research and regulations who are usually from the institution where the clinical trials take place. Approval from IRB on the procedure and organization of the clinical trials is required before anything else can happen.

• National

  • National agencies such as the U.S. Food and Drug Administration (FDA) also oversee the clinical trials. They also devise the regulation behind clinical trials and ensure its enforcement at an institutional level.

 

Once the clinical trials are approved, it enters a 4-stage process.

 

Phase 1: Test for safety in a small group

Phase 2: If Phase 1 approved, test for safety in a slightly larger group

Phase 3: Test for efficacy in a large group and FDA approval

Phase 4: More trials for long-term studies

 

          The following video gives a detailed overview of how the clinical trials work, what the 4 stages are and what regulations provide protections for patients.