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A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Inhibiting steroid production with metyrapone, a common therapy used in Cushing’s disease, may not only normalize cortisol levels but also lead to the spontaneous remission of ectopic tumors producing adrenocorticotropic hormone (ACTH), according to a case study. Researchers presented the case of a 71-year old women whose ACTH-producing lung tumor regressed spontaneously…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to fluasterone (ST-002) for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis, and high blood sugar in Cushing’s syndrome patients, SteroTherapeutics announced. Cushing’s syndrome occurs when the body is exposed to high levels of the hormone cortisol over a long period.

Using magnetic resonance imaging (MRI) with superior magnetic strength enabled determination of precise tumor location in a patient with Cushing’s disease, a new case report describes. The research, “Value of pituitary gland MRI at 7 T in Cushing’s disease and relationship to inferior petrosal sinus sampling: case…

Strongbridge Biopharma has begun dosing patients in another Phase 3 clinical trial of its Cushing’s syndrome therapy Recorlev (levoketoconazole). The company will enroll roughly 35 patients in the LOGICS (NCT03277690). Those participating in another Phase 3 trial, SONICS (NCT01838551), can enter the LOGICS study as well. LOGICS “marks an…

A very rare case of Cushing’s syndrome developing as a result of a large and also rare cancer of the nasal sinuses gives insights into how to screen and treat such an anomaly, of which fewer than 25 cases have been reported in literature. Paraneoplastic esthesioneuroblastoma (ENB), a very rare type…

Assessment of late-night salivary cortisol (LNSC) levels is a poor diagnostic tool for detecting Cushing’s disease in obese patients, a new study from Germany shows. The test demonstrated a particularly poor sensitivity in obese people, meaning it will often suggest a patient has Cushing’s disease when that is not the case…

Tumors located outside of the pituitary gland — most commonly small-cell lung cancer — can lead to ectopic Cushing’s syndrome, a new case report shows. The study with that finding, “Case-series of paraneoplastic Cushing syndrome in small-cell lung cancer,” was published in the journal Endocrinology, Diabetes…

Detailed imaging analysis of patients with Cushing’s disease (CD) and other disorders caused by pituitary adenomas (tumors that arise from the pituitary, a small gland in the brain) provides correlation with clinical characteristics and treatment response, a new Turkish study reports. The research, “Clinicopathological…

Strongbridge Biopharma recently received a second patent from the U.S. Patent and Trademark Office covering the use of Recorlev (levoketoconazole) for the treatment of Cushing’s syndrome. The patent (No. 9,918,984) is titled “Methods and Compositions for Treating Diabetes, Metabolic Syndrome and Other Conditions” and will be active until Jan. 10,…