Pillar 3 of the FAST Roadmap to a Cure 2.0 consists of therapeutic programs which focus on “downstream targets to treat symptoms” by addressing different molecular pathways and effector proteins impacted by the missing UBE3A protein.
Currently, we have four programs in the pipeline under Pillar 3 and one active human clinical trial:
- Exogenous ketone supplements
- Purpose: Use of exogenous ketones to induce ketosis as a way to control seizures.
- Company: Disruptive Nutrition
- Clinical trial completed.
- GABAaɑ5 positive allosteric modulator (Alogabat)
- IGF-1, 2 ligands (NNZ-2591)
- BDNF
- Purpose: To use a downstream target of UBE3A to regulate learning and memory deficits.
- Institution: Brown University