CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS

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Grant Award Details

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Human Stem Cell Use:
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Grant Application Details

Application Title:

CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS

Public Abstract:
Therapeutic Candidate or Device

CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF


Amyotrophic Lateral Sclerosis (ALS)

Therapeutic Mechanism

This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor cortex.

Unmet Medical Need

There are currently only two FDA-approved therapies with minimal benefits, but there is no cure for ALS. Thus there is a huge unmet medical need to find additional therapies with longer-lasting benefits.

Project Objective

Phase 1/2a completed

Major Proposed Activities

  • Enrollment of 16 patients to a Phase 1/2a trial to demonstrate safety and preliminary efficacy of the cellular product
  • GMP manufacturing of a new CNS10-NPC-GDNF working cell lot for future clinical trials in ALS.
Statement of Benefit to California:
ALS is a devastating disease and there are over 6,000 cases in CA. If this treatment works it will provide one of the only ways to slow down motor neuron disease progression. This illness costs the state of California millions of dollars in healthcare costs and immense suffering to those Californians affected by the disease. Additionally, because over 90% of the funds will be used in California, it will help stimulate the economy.