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Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Huntington Potter, AD/PD 2022: Sargramostim, a potential role in the treatment of Alzheimer’s disease: Phase 2 Results

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Published Online: Mar 25th 2022

Sargramostim, an innate immune system modulator, has been FDA approved for treating leukopenia for many years. Sargramostim has been investigated in this phase 2 study to determine if it can safely halt or reduce cognitive decline and brain pathology in participants with mild-to-moderate Alzheimer’s disease.

Prof. Huntington Potter (University of Colorado, Denver, MS, USA) joins touchNEUROLOGY to discuss the phase 2 results (NCT01409915) to investigate whether  inflammation and the innate immune system in Alzheimer’s disease may provide therapeutic benefit, and what will be the next steps in the development of sargramostim in Alzheimer’s disease.

Questions:

  1. Could you tell us a little about sargramostim, its mechanism of action and clinical uses? (0:18)
  2. What is the rationale for investigating sargramostim in the setting of Alzheimer’s disease (AD)? (1:15)
  3. What were the aims, design and primary outcome measures of your study? (3:25)
  4. What will be the next step in the clinical development of sargramostim in AD? (5:52)
  5. What are the take home messages from your presentation? (7:58)

Disclosures: Huntington Potter has nothing to disclose in relation to this video interview.

Support: Interview and filming supported by Touch Medical Media. Interview conducted by Katey Gabrysch

Filmed as a highlight of AD/PD 2022.

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