CNS10-NPC-GDNF is an experimental stem cell-based therapy being developed by researchers at Cedars-Sinai Medical Center in Los Angeles for the treatment of amyotrophic lateral sclerosis (ALS). It is in the Phase 1 clinical trial stage.

How CNS10-NPC-GDNF works

ALS is caused by the death of motor neurons, or nerve cells, that control the movement of voluntary muscles. Another type of cell called an astrocyte also seems to play a role in this process. Astrocytes usually support the health and integrity of motor neurons by secreting a specific protein called glial-derived neurotrophic factor (GDNF), which is crucial for the survival of motor neurons. In ALS, astrocytes degenerate as the disease progresses.

CNS10-NPC-GDNF is a type of stem cell called a human neural progenitor cell (hNPC) that has been genetically engineered to produce GDNF. It is hoped that when transplanted into the spinal cord of the patients, the cells will grow into astrocytes and provide GDNF to motor neurons.

Researchers think this approach might increase the survival of motor neurons in ALS patients, improving motor function and delaying disease progression.

Studies with CNS10-NPC-GDNF

A preclinical study in rats assessed the safety and feasibility of transplanting GDNF-producing hNPCs. A total of 120,000 cells were injected at five distinct sites of the spinal cord of the animals.

The researchers confirmed that the transplanted cells produced human GDNF. The protein was also found in the neurons of the rat, suggesting that the host cells were taking up the GDNF released by the hNPCs. They also showed that the cells differentiated into astrocytes. After seven and a half months, the transplanted hNPCs were still found in the spinal cord, demonstrating that the cells survive for at least this amount of time.

In October 2016, the U.S. Food and Drug Administration approved a Phase 1 clinical trial (NCT02943850), which is currently ongoing. The trial includes 18 ALS patients whose disease symptoms started 36 months or less before the start of the study, and whose forced vital capacity (a measure of lung function) is greater than 60 percent.

The study is aimed at assessing the safety and effectiveness of transplanting CNS10-NPC-GDNF into the spinal cord of ALS patients.

Participants are divided into two groups. The first three people in each group receive sequential treatment with a minimum of a month between surgeries. For the remaining people in each group, the interval between surgeries is at least one week.

The safety of the treatment is evaluated by following the occurrence of adverse events for 12 months following the operation. Functional muscle tests and imaging are also being performed.

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