Thursday, December 4, 2014

Introduction to Skin Cell Blog

We will tell you the story of how skin cells are turned into stem cells from a patient with young onset sporadic Parkinson’s disease. His name is Alex. Alex has volunteered to have a skin biopsy taken. As we progress, we will explain to him how his cells are becoming a model for Parkinson’s disease, to better understand the causes of neurodegeneration.

We will take you on the “journey” from a skin biopsy, how it grows, how it gets coaxed into becoming stem cells. We will also find out how the skin-derived stem cells are characterized and how the stem cells will become dopamine producing neurons.

What is Parkinson's disease? Parkinson’s disease is a multifactorial disease. Genetic and environmental causes have been reported and many pathways in the neurons can be compromised. There is no one type of Parkinson’s disease. Therefore, it is critical to understand the cause in every Parkinson’s patient when we want to find better therapies and potentially a cure.

Patient-derived stem cell models are powerful tools to understand disease and to test drugs. These cells models have a human genetic background, mimic the neuronal cell type that is most susceptible in Parkinson’s disease. These cells are derived from patients with Parkinson's disease.

We are very excited about this project. Alex’s motto is ‘keep moving’. Please keep moving with us on this journey. Stay tuned.

Lauren, Jessica, and Birgitt

Monday, December 1, 2014

About Lauren and Birgitt

Lauren Pijanowski got her Master’s in Biotechnology  and undergraduate degree at San Jose State University (SJSU) in Forensic Science. As a graduate student she joined the CIRM sponsored Stem Cell Internship in Laboratory based Learning program which prepared her with a broad understanding of stem cell biology through lectures, seminars, and in-depth laboratory work.
She also attended an intensive human stem cell workshop at Stanford University prior to beginning her internship at the Parkinson’s Institute. The goal of her work is to take skin cells, restore them to the stem cell state and use these stem cells to study Parkinson’s disease and the mechanisms of this disease with a more personalized approach to research.

Birgitt Schuele is an Associate Professor and Director of Gene Discovery and Stem Cell Modeling at the Parkinson’s Institute in Sunnyvale, California. She heads the neurogenetics program, which includes clinical genetic studies of families and sporadic cases with parkinsonism.

The Schuele team applies novel stem cell technologies to generate stem cells from patients with Parkinson’s disease. These skin-derived human stem cells build the foundation for further studies in the culture dish. The stem cells can be differentiated into neurons, specifically neurons that produce and release dopamine resembling the neurons that die in the brain of Parkinson’s patients. This unique human cellular model allows replicating conditions as in the human brain. These neurons are also electrically active and can ”fire” action potentials.

Parkinson’s research has been hampered for a long time because no pre-clinical model could faithfully replicate human disease. Also autopsy tissues from brain donors only have limited use as the disease process has already destroyed most of the dopamine neurons in the brain.

With these novel stem cell-derived neuronal model systems, living human dopamine neurons can be manipulated and studied for changes between patients and controls. This modeling approach has great promise for advancing science and discovery by having the tools to study early changes in the disease process as well as understanding environmental exposure which are the basis for drug screening.

For more information about The Schuele Team at The Parkinson's Institute, visit www.thepi.org

About Alex

Alex was diagnosed with Parkinson’s disease five years ago at age 36. He is a lawyer, ultra athlete, film producer, writer and father of three boys. It all started with a little shaking of his right hand. Parkinson’s disease has changed Alex’s life. He was on an uphill path in his successful career as a lawyer with an attitude that life has many more opportunities and challenges to come.

Parkinson’s disease is now his biggest challenge but it has also given him a new direction. He is raising awareness and funds for Parkinson’s disease that gives many of his fellow sufferers hope and inspiration to keep moving http://www.alexflynn.co.uk .

We don’t know why Alex got the disease so early and we currently don’t have a therapeutic strategy that allows us to treat the cause of his disease and not just his symptoms. The journey of Alex’s skin cells is a personalized approach to find the cause for Parkinson's disease.

Parkinson’s disease can have many different faces: some forms are slowly progressing and the disease has only a minor impact on daily activities for decades, other forms are more aggressive and within a few years patients are bedridden. Patients also respond very differently to the therapies and the type of drugs and the dosage needs to be carefully adjusted because of the changes in effectiveness of the medication over time.

We think that there is a significant need to develop approaches to realize a better understanding of what the underlying causes are in respect of each individual patient –a personalized approach.

Recent advancements in disease modeling allow us to now create patient derived dopamine producing neurons from skin cells; similar to the ones dying in a patient’s brain.

We will take you on a journey to develop a model of Parkinson's disease in a dish from a little piece of skin. Alex has donated a skin biopsy for our stem cell program. We have grown and have banked about 20 Million cells of his skin. Follow us and learn more about the disease and the novel individualized research that lays the foundation for causal treatments.