Neurodegenerative diseases like Parkinson's, ALS, and retinitis pigmentosa are caused by neuron deterioration.
Neurodegenerative illnesses encompass a wide range of serious conditions, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and retinitis pigmentosa, all of which are underpinned by the progressive degeneration of neurons. A recent study published in Stem Cell Reports proposes protecting existing neurons to avoid their degeneration is a viable therapy option until technologies for regenerating or replacing damaged neurons from stem cells are developed.
U.S. researchers at Cedars-Sinai Medical Center genetically engineered neural progenitor cells (NPCs), a type of cell which makes neurons and supporting brain cells called astrocytes, to release a specific neuroprotective protein after a recent proof of principle clinical trial suggested that this approach is safe and possibly effective in patients with ALS.
Clive Svendsen and others have gone this a step further by creating a designed NPC supply from reprogrammed adult human blood cells, which might be tailored to the individual patient. The modified NPCs were examined in a rat model of amyotrophic lateral sclerosis (ALS) and a rat model of retinal degeneration, where they effectively reduced the loss of neurons in both species.
Remarkably, modified NPCs prevented the rapid loss of vision seen in untreated eyes of rats with retinal degeneration. Cells transplanted into the spinal cord of rats lived there for up to nine months, where they differentiated into astrocytes that provide support for neurons. The researchers think this mixture is to credit for the therapeutic effects seen.
Clinical trials on the safety and efficacy of designed; long-lasting, patient-specific NPCs for the treatment of neurodegenerative disease will have a solid foundation in this work.