Isosafrole, a stiripentol analog, is a potent LDH inhibitor. Stiripentol is a new-generation antiepileptic drug, and its chemical structure is unrelated to other antiepileptic drugs. Seizures and epileptiform activity are reduced by inhibition of the metabolic pathway via lactate dehydrogenase (LDH), which is a component of the astrocyte-neuron lactate shuttle. Isosafrole is a substructure of stiripentol that lacks the hydroxyl group and tertiary-butyl group of stiripentol. Isosafrole strongly inhibited the pyruvate-to-lactate conversion by both LDH1 and LDH5, suggesting that it inhibits lactate production itself. Isosafrole suppresses seizures in vivo. Going by the lactate reduction by ketogenic diets, LDH inhibitors for the pyruvate-to-lactate conversion, such as isosafrole, would be more effective for antiepileptic actions. Isosafrole is a known inducer of some of the liver enzymes of the cytochrome P-450 group in rodents, especially CYP1A2.

3.beta-3-[2-(Diethylamino)ethoxy]androst-5-en-17-one hydrochloride or U18666A, an amphipathic cationic amine, is a cell permeable research tool drug that inhibits cholesterol synthesis and trafficking, and also a weak inhibitor of hedgehog (Hh) signaling. U18666A had been used in several models both in vivo and in vitro to mimic Niemann-Pick type C disease (NPC) and for assessing the importance of molecular trafficking through the lysosomal pathway in other conditions such as atherosclerosis, Alzheimer's disease, and prion infections. U18666A also provided animal models for such disorders as petite mal (absence) epilepsy and cataracts. U18666A inhibits the enzyme desmosterol reductase responsible for reducing the desmosterol in cholesterol biosynthesis, and also blocks LDL-(low density lipoprotein) cholesterol transport from the lysosomes into the endoplasmic reticulum thereby increasing the level of caveolina-1 located within the plasma membrane caveolae. U18666A was shown to be a potent antagonist of alpha4beta2 neuronal nicotinic acetylcholine receptors and may be useful as a tool in the functional characterization and pharmacological profiling of nAChRs.

Pifithrin-alpha is a small molecule p53 functional inhibitor reported to behave like an antiapoptotic agent in neurodegenerative models. Pifithrin-alpha is a prodrug that under physiological conditions spontaneously undergoes ring closure to yield pifithrin-beta. Pifithrin-beta demonstrated antiproliferative and neuroprotective effects in vitro. Pifithrin-beta is able to activate the aryl hydrocarbon receptor (AhR) in a complete independent way of the p53 inhibition.