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Orphan Disease




One of the institute's priorities is to develop diseased and patient specific stem cells for drug discovery initiatives to find treatments for orphan (rare) diseases. The institute is collaborating with Cellular Engineering Technologies to develop non-viral, cMyc-free and feeder-free induced pluripotent stem (IPS) cells. For a list of orphan diseases, go to the Orphan Disease List page.


What are IPS cells?


IPS cells are pluripotent stem cells that have the same properties as embryonic stem cells, but do not have the same ethical controversy because embryos are not used. The below figure describes the process. Adult cells derived from a patient are genetically reprogrammed by the introduction of multiple genetic elements. Transformed IPS cells then can convert into all of the same 200 specialized cells as embryonic stem cells. The advantage of IPS cells is that it avoids using embryos and the stem cells are specific to a patient.

Utility of IPS cells:


These cells can then be used as test models to screen drugs with research tools to find treatments for patients (See below figure).

Despite the increased financial expenditures in medical research, the number of FDA-approved drugs over time has been decreasing. The Institute’s goal is to serve as a catalyst, with a novel model that incorporates the private medical practice sector as an equal partner to increase the drug discovery output and productivity for academia, government and industry.


The Institute is recruiting doctors to participate in our research activities to find treatments and cures for patients with rare orphan diseases. A rare orphan disease is defined as a disease with less than 200,000 suffering from a specific ailment. The Institute and CET have created a research program to accelerate drug development by creating stem cell models from patients with rare diseases, which can be utilize to screen drugs that potentially can be investigated in clinical trials. These stem cell will be utilized to screen drugs that potentially could be used as therapies for patients. The Institute is actively recruiting patients with the following disorders to develop stem cell lines for screening drugs: (1) Lysosomal Storage Diseases; (2) Genetic Diseases; (3) Neurological Diseases with Strong Family History –e.g. Alzheimer and Parkinson Disease, ADHD, dyslexia, Autism and ALS.


The Institute has a federally registered Institutional Review Board (IRB). This structure allows the Institute to recruit patients with rare diseases and to procure their tissue samples for producing stem cell models for screening drugs that may lead to clinical trials. The Institute can recruit patients anywhere within the United States to obtain the necessary tissue, but we need the support of private physicians around the country to realize this program. Patients interested in participating in such research should enter their contact information on our Patient Data Registry page.


The Institute is also asking private practice doctors to consider assisting our research program. Interested physician should enter their contact information on our Physician Registry page.

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