Eflornithine
M. Lindsay Grayson, Sara E. Cosgrove, Suzanne M. Crowe, M. Lindsay Grayson, William Hope, James S. McCarthy, John Mills, Johan W. Mouton, David L. Paterson in Kucers’ The Use of Antibiotics, 2017
Eflornithine is generally less toxic than the other drugs commonly used for sleeping sickness, including pentamidine, suramin, and melarsoprol. However, side effects are frequent, potentially severe, and need to be managed appropriately. In some respects, the toxicity profile of eflornithine is similar to that of cytotoxic drugs used for the treatment of cancer (Burri and Brun, 2003). In general, reactions increase in frequency and severity with dose and duration of therapy, and are related to the underlying general condition of the patient (Milord et al., 1992; Burri and Brun, 2003). However, they are generally reversible after treatment is ceased. The common adverse reactions observed in clinical studies are bone marrow toxicity, seizures, and alopecia; gastrointestinal side effects are seen especially when the drug is orally administered.
The practical management of hormonal treatment in adults with gender dysphoria
James Barrett in Transsexual and Other Disorders of Gender Identity, 2017
Hirsuitism and menstrual irregularity can be controlled using the oral contraceptive pill. These medication induce SHBG production whilst suppressing ovarian androgen production and effectively reducing hirsuitism. Dianette is especially effective as it contains a combination of the ethinylestradiol with the anti-androgenic progestin cyproterone acetate, which helps to control the effects of androgens on the hair follicle. An alternative approach is to use a topical preparation eflornithine (Vaniqa 11.5%), which inhibits the enzyme ornithine decarboxylase in the hair follicle and so reduces hair growth. Local measures such as waxing, sugaring, laser and electrolysis are also extremely effective in controlling excess body hair.
Endocrinology and infertility
Marwan Habiba, Andrea Akkad, Justin Konje in MRCOG Part 2, 2017
F. This patient appears to have a clearly defined problem with increased hair growth in one particular, relatively small, area of her body. As there are no other symptoms to address and the affected surface area is small, a topical agent might be most appropriate. Eflornithine is the first licensed topical hair retardant in the UK. The mode of action is decreasing androgen sensitivity of hair follicles. You may wish to consider that cyproterone acetate is the only licensed systemic agent for the treatment of hirsutism. Interestingly (and perhaps more importantly on the more global scale of health), eflornithine, marketed as Ornidyl1 rather than Vaniqua1, is effective in combating sleeping sickness.
Laser-assisted hair removal for facial hirsutism in women: A review of evidence
Published in Journal of Cosmetic and Laser Therapy, 2018
Eflornithine, an antiprotozoal drug, is an FDA-approved prescription cream (Vaniqa®, Allergan, Irvine, CA.) licensed for facial hirsutism in adult women (28). It has been studied as an adjunct to a long-pulsed alexandrite laser in a randomized, placebo-controlled trial in which statistically significant superiority was evident in comparison with laser treatment alone (29). Clinical effect was assessed on the upper lip in women aged 18 years or older with unwanted facial hair. Whilst individuals with clinical stigmata of hyperandrogenism were excluded from the study it could be relevant to laser hair removal in PCOS related facial hirsutism, because of the need of a higher treatment efficacy. This single-center study received public funding and with the follow-up at 6 months the outcomes did not mirror long-term efficacy. In addition, like many of the contemporary studies of laser hair removal, assessment was subjective. Safety of the treatments in combination was assured so any future long-term follow up of a trial of this kind with may prove worth-while.
Emerging compounds and therapeutic strategies to treat infections from Trypanosoma brucei: an overhaul of the last 5-years patents
Published in Expert Opinion on Therapeutic Patents, 2023
Francesco Melfi, Simone Carradori, Cristina Campestre, Entela Haloci, Alessandra Ammazzalorso, Rossella Grande, Ilaria D’Agostino
Eflornithine (Figure 1) is recognized to be only efficacious against Tbg and can be safely associated with nifurtimox as a combination therapy. Eflornithine was first designed as an anticancer drug, and then it was repurposed for the treatment of late-stage HAT, because of its BBB permeability [11]. The drug correlates with racemic α-difluoromethyl-ornithine (DFMO) displaying a high structural analogy with ornithine. Indeed, it takes advantage of the amino acid transporter AAT6 to cross cell membrane easily [12]. The substrate ornithine is generally involved in the parasite polyamine biosynthetic pathway to produce spermidine, a component of trypanothione, the protozoan corresponding compound of mammalian glutathione. By hampering ODC, eflornithine can limit the crucial polyamine biosynthesis [13] and allow the accumulation of ornithine, S-adenosylmethionine, and decarboxylated S-adenosylmethionine, which finally block some methylation reactions of cellular macromolecules [14,15]. Surprisingly, eflornithine was found to counteract hirsutism in women, which was exploited as a repurposed therapeutic approach [16].
Challenges and opportunities with drug repurposing: finding strategies to find alternative uses of therapeutics
Published in Expert Opinion on Drug Discovery, 2020
Alan Talevi, Carolina L. Bellera
Systems medicine/network pharmacology offers an integrative perspective on previous (and seemingly colliding) paradigms in drug discovery: phenotypic-oriented and target-oriented, ‘rational’ drug discovery. Network and metabolic control analysis can be useful tools to design multi-target therapeutics or, alternatively, choose a synergistic drug combination. For instance, nifurtimox–eflornithine combination therapy has been included in World Health Organization’s Model List of Essential Medicines to manage advanced stages of the Gambiense form of sleeping sickness. The combination is easier to administer, it has a shorter treatment duration than the eflornithine monotherapy, and it is potentially protective against the emergence of resistant parasites. Interestingly, both drugs in the exemplified combination are repurposed cases: eflornithine was initially developed for cancer treatment in the late 1970s, and nifurtimox was originally approved for the treatment of American trypanosomiasis.
Related Knowledge Centers
- African Trypanosomiasis
- Bone Marrow Suppression
- Hirsutism
- Trypanosoma Brucei
- Medication
- Nifurtimox
- Intravenous Therapy
- Topical Medication
- Oral Administration
- Seizure