The California Institute for Regenerative Medicine (CIRM) is investing $4 million in Ryne Bio’s late-stage preclinical project for idiopathic Parkinson’s disease (PD).
PD destroys dopamine-producing neurons and causes motor symptoms such as dyskinesia. Dyskinesia causes involuntary, erratic movements of the face, arms, legs, or trunk.
The disease can also cause dementia, depression, and sleep disorders.
PD ranks as the second most common neurodegenerative disease after Alzheimer’s disease. It affects about one million people in the United States.
California has an estimated 116,900 residents with PD, the highest number in the country.
Doctors often use medications such as levodopa to manage early PD symptoms. However, these medications become less effective as the disease progresses.
Ryne Bio’s proposed stem cell therapy could restore dopamine-producing neurons. These neurons support important functions, including movement and memory.
Ryne Bio researchers plan to deliver dopamine-producing cells to patients’ brains. The cells could replace lost neurons and improve motor function.
The grant will fund nonclinical safety studies required for an Investigational New Drug application.
The FDA requires these studies before researchers can begin clinical trials in people with PD.
CIRM has a strong interest in advancing this therapy. To date, CIRM has invested more than $59 million in Parkinson’s disease research.
That funding has helped move research from the discovery stage toward clinical trials.
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.