ALS

AMYOTROPHIC LATERAL SCLEROSIS (ALS)

NEUROINFLAMMATORY PROCESSES IMPLICATED IN THE INITIATION AND PROGRESSION OF DISEASE

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, affects as many as 30,000 people in the U.S., with an estimated 5,000 new cases diagnosed each year.

It is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord resulting in loss of motor function and often paralysis and death.

Accumulating evidence suggests that neuroinflammatory processes are implicated in the initiation and progression of ALS. Pre-clinical research has also demonstrated an increase in microgliosis (the shifting of microglia from a neuroprotective to a chronic, pro-inflammatory state) before the onset of ALS symptoms.

In a pre-clinical ALS (SOD1) model, we demonstrated that our proprietary formulation of cromolyn sodium (AZT-101):

  • delayed disease onset and progress,
  • reduced motor deficits, and
  • significantly spared lumbar spinal cord motor neurons and reduced pro-inflammatory cytokine/chemokine levels in the spinal cord and plasma.

As a result, we are developing AZT-101 for the treatment of ALS and are planning to file for approval to begin clinical development. (See Pipeline for more on the clinical development of AZT-101.)

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