The California Institute for Regenerative Medicine (CIRM) is investing $4 million in a late-stage preclinical project by Ryne Bio aiming to improve treatment for Idiopathic Parkinson’s disease (PD).
PD is characterized by a loss of dopamine producing neurons that result in motor symptoms, such as dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk) and non-motor effects such as dementia, depression and sleep disorders.
PD is the second-most common neurodegenerative disease after Alzheimer’s disease affecting approximately 1 million people in the U.S. In California, it is estimated that 116,900 people live with PD, representing the highest number of people with the disease in the country.
At its early stages, PD can be treated with medication such as Levodopa to treat symptoms but these become less effective as the disease progresses.
The proposed stem cell therapy in this project offers the potential to restore dopamine neurons, which play a role in many important body functions, including movement and memory.
Investigators at Ryne Bio are aiming to deliver dopamine producing cells to replace the lost neurons to the brain of Parkinson’s disease patients to restore/improve motor function.
The current grant is being funded to conduct Investigational New Drug (IND) enabling, nonclinical safety studies per the US Food and Drug Administration (FDA) Guidance. The IND is the authorization needed to begin a clinical trial in Parkinson’s patients.
CIRM has a vested interest in seeing this therapy succeed. To date, CIRM has invested more than $59 million in helping research for Parkinson’s disease progress from a basic or Discovery level through clinical trials.
The Stem Cellar 
Why only idiopathic? Inherited Parkinson’s is still Parkinson’s. Most disease symptoms are either the same or similar. A misfolded protein can’t sneak into this research? A LRRK2 carrier wants to know.
Different types of cells express different types of receptor to interact with growth factors in their cellular environment. The interaction of each receptor type is specific to specific growth factor. The binding of receptor-ligand may trigger intracellular signaling for cell grow, differentiation and functioning. The deprivation of specific growth factor may cause the cells to become grow arrest, but cells require other growth factors to maintain their survival stage.
Stem cells require growth factors for growth and differentiation. The maturation of stem cells involve many stages of grow and differentiation. Each stage of progenitors requires specific growth factor for cells to response and turning on the new gene. The absence of specific growth factor or bypassed certain stages of maturation may produce premature cell with short lifetime. Therefore, stem cells therapy require growth factors treatment to produce mature and functioning dopamine producing neurons. Different ages of people have different abilities to produce growth factors to support stem cells development. Thus, growth factors research is important to determine growth factors requirement for maturation of stem cells to produce mature and functioning cells.