First Patient with Alzheimer’s Dosed in Bryostatin-1 Clinical Trial


Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1 for patients with Alzheimer’s disease.  The study, led in conjunction with the National Institutes of Health (“NIH”), will focus on patients with moderate to severe Alzheimer’s disease.


According to the AlzForum, Bryostatin-1:

is a natural product derived from the marine invertebrate Bugula neritina. It has potent and broad antitumor activity. Bryostatin 1 activates protein kinase C family members, with nanomolar potency for PKC1α and ε isotypes.

Basically, bryostatin-1 boosts protein kinase C (PKC), which has neutrophic effects. In other words, it helps improve neuronal health and growth, memory, and learning. In mice models of Alzheimer’s disease, fragile X syndrome, stroke, and traumatic brain injury (TBI), bryostatin-1 had anti-inflammatory benefits, and also improved neurological function. As of now, bryostatin-1 has received Orphan Drug Designation for the treatment of fragile X syndrome.

The recent Phase 2 trial will enroll up to 100 patients with Alzheimer’s disease. During this period, patients will not receive Nameda (memantine) for at least 6 months. In exchange, patients will undergo two 11-week (3~ month) bryostatin dosing cycles. Researchers will analyze patient outcomes through the Severe Impairment Battery score. In a prior trial, bryostatin-1 showed cognitive improvement and neuronal development.

Alzheimer’s Disease

Although doctors are not sure of the exact cause of Alzheimer’s disease, many believe the condition results from a combination of environmental factors, lifestyle choices, and genetics. Alzheimer’s disease occurs when brain neurons disconnect and die, causing brain degeneration. Generally, the condition occurs in patients over 65. Females are more likely to develop Alzheimer’s disease than males. Symptoms include:

  • Memory loss
  • Confusion
  • Aggression or agitation
  • Changes in mood, personality, and behavior
  • Jumbled speech
  • Loss of appetite
  • Changes in thinking, reasoning, decision-making, planning, and completing tasks
  • Aspiration
  • Pneumonia
  • Malnutrition and dehydration

Learn more about Alzheimer’s disease here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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