There is tremendous promise in scientific advances to address unmet medical needs. In the future, personalized medicine, biomarkers and other novel treatment modalities will change the course of diseases, saving and improving lives. Yet these treatments will be complex and iterative, requiring new levels of knowledge. In the past and also now, new treatments were approved based on novel mechanism of actions. In the future treatments will be approved based on dramatically different outcomes. One of those unmet medical needs is obviously severe incurable neurodegenerative disorders like Parkinson’s Disease. Let’s have a look into its future?
Induced Pluripotent Stem (IPS) cells were discovered in 2006, a feat honored by a Nobel Prize in 2012. These IPS cells can become nearly any kind of cell in the body, but they don’t raise the ethical concerns of using embryonic stem cells. Another potential advantage of IPS cells over embryonic stem cells is that they should be less prone to rejection by the patients’ immune systems, because the transplanted cells come from the individuals themselves.
The replacement brain cells are now being grown in a Lab at The Scripps Research Institute (San Diego, USA). Patches of skin the diameter of a pencil eraser were removed from the patients’ arms and turned into a new kind of stem cell that acts like embryonic stem cells that are then further manipulated into dopamine-producing neurons which are dying day by day in Parkinson patients for unknown reason causing lots of motor and non-motor symptoms.
This process is complete for first 10 patients suffering from Parkinson’s disease. The Scripps Research Institute is now conducting lab tests and animal studies to ensure that these cells are safe and efficacious. Lab is also working with a cell manufacturing facility and will begin transferring our patient’s stem cells to be cultured under clinical-grade conditions. This is very exciting as scientists are getting closer to being completely ready for clinical trial. “Clinical grade” cells for animal studies will be used in 2017, the results of which will be used for FDA application to begin the study in patients.
The patient will be treated with dopamine-producing neurons, made from his/her own cells! For Parkinson’s Disease it’s a big revolutionary step moving from the need of regular intake if multiple pills (just to live their lives somehow day-by-day and realizing unavoidable disease progression despite the treatment which is often associated with lots of side effects) towards to getting needed cells at right place which will be fully functioning and actually will “remove” disease symptoms. Miracle, isn’t it? Even if so, this miracle is made by scientists… people passionate about what they do and those dreaming to help millions of Parkinson patients in the world!
Would you like to see how clinical research will look in the future? Want to learn more about this research? Curious to meet the “star team”? Wish to know more about brave patients and read their stories? Looking forward to what happens next? You can follow the research’s exciting journey where this cutting edge science will graduate from the lab and into the Clinical Study following FDA approval, as well as read more and watch the video here.