AgeX and UCI to Research Huntington Disease Therapies
New Hope for Huntington's Disease
Updated: February 19, 2020
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AgeX Therapeutics will collaborate with neurology researchers at the University of California, Irvine, (UCI) to research cell-based therapies for neural Huntington’s disease.
The game plan is to first develop therapies for Huntington’s disease and then later apply relevant findings to other neurological diseases like Alzheimer’s and Parkinson’s disease.
UCI Beall Applied Innovation, which connects the University of California, Irvine, with the Orange County business community to create jobs and foster local economic growth, is supporting the research.
Research Promises New Hope for Huntington’s Disease
Although there are treatments for those with Huntington’s disease, they only mitigate the symptoms. The collaborative venture between AgeX and UCI hopes to provide therapeutic options beyond palliative care.
Huntington’s disease is a rare genetic disorder with fewer than 200,000 cases in the United States. This inherited disease slowly destroys brain neurons over the span of decades.
Unlike other genetic diseases whose symptoms appear in childhood, this disorder only becomes noticeable when people reach their thirties or forties. Because of this long delay in the progression of symptoms, doctors regard it as an adult-onset genetic disease.
Adult patients may show psychiatric symptoms, cognitive decline, emotional problems, and uncontrolled physical movements.
Research Lead
UCI Professor of Psychiatry and Human Behavior Leslie Thompson will lead the preliminary research, and organizers expect the project to take a year to complete. She is an ideal choice for the collaborative venture because her laboratory has gathered a significant amount of animal data that will expedite an investigational new drug (IND) application with the FDA by 2021 to start clinical trials to treat Huntington’s disease.
Research Goals
The goal of the research project is to develop an efficient method to extract neural stem cells directly from human pluripotent stem cells. These are self-replicating cells from human fetal tissue or human embryos that grow into cells and tissues. The focus will be to get enough pure pluripotent cells to design a cell-based therapy.
If the researchers can improve neural stem cell production within the scheduled year, then cell-based therapies can move straight into clinical development.
Purpose of Collaboration
Collaboration between UCI and AgeX Therapeutics offers a range of mutual benefits and will allow the UCI research team to use AgeX’s PureStem technology. This unique innovation has refined the intricate process of deriving stem cells for manufacturing purposes.
Working with PureStem technology offers researchers three large advantages: first, manufacturing can be scaled for mass production; second, the cost of cell-based therapies will be more affordable; and third, it will be possible to isolate clonal cells with particularly high purity and identity.
The end results of the collaboration will not only clinically validate an effective therapy but it will also open the doors for a commercial opportunity. If the project unfolds according to optimistic expectations, then Professor Thompson and her team of researchers and AgeX Therapeutics will jointly own a company that commercializes cell therapies based on licensed inventions.
About AgeX Therapeutics
AgeX Therapeutics, Inc. is an American company that develops and commercializes therapeutic products to increase human longevity. The company has numerous innovative cell-based technologies on the market.
Some core products in its pipeline to expand human life and rejuvenate age-related tissues include PureStem®, a stem cell manufacturing technology; UniverCyte™, which uses stem cells to boost immunotolerance; AGEX-VASC1, which uses vascular progenitor cells to help patients with tissue ischemia, a condition that restricts the blood supply to tissues; AGEX-BAT1, which uses brown fat cells to help patients with Type II diabetes; Tissue Regeneration (iTR™) which regenerates tissue to reverse signs of aging; and HyStem®, which engrafts PureStem cell therapies in the human body.
AgeX Therapeutics shares its proprietary technology platforms with recognized scientists at the world’s leading universities to establish licensing and collaboration agreements. However, regardless of the type of therapeutic solution under investigation, scientists involved in these collaborative projects share the common goal of designing allogeneic, highly defined, off-the-shelf pluripotent stem cell products to find solutions for age-related diseases that have little to no effective medical treatments.
In summary, if clinical trials are successful in finding a cell-based therapy for adult-onset neural Huntington’s disease, this will bring about huge advances in medicine, particularly for those with late-onset genetic-based diseases that become increasingly apparent in an aging population.
At present, all current therapies for Huntington’s disease are only palliative. Current drug treatments can only provide relief from the stress and symptoms of the disorder. Any discovered therapeutic treatments for Huntington’s disease will also generate bold, new therapies for other neurological diseases that currently only have palliative treatment plans, such as Alzheimer’s disease or Parkinson’s disease.
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