Part of the National Institutes of Health (NIH), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and SteroTherapeutics have entered into a Cooperative Research and Development Agreement (CRADA) in an effort to test a DHEA. This non-androgenic analogue of dehydroepiandrosterone is meant to treat various manifestations of Cushing syndrome.
About Cushing Syndrome
Cushing syndrome is an endocrine disorder that is characterized by high levels of cortisol in the blood. Symptoms include weight gain, acne, fatty deposits, thin skin that is easily bruised, purple stretch marks, muscle weakness, glucose intolerance, fatigue, depression, cognitive dysfunction, bone loss, anxiety, high blood pressure, and irritability. All of these symptoms may be the result of various causes, with the two major causes being an overproduction of cortisol by the body and oral corticosteroids. Other possible causes include a tumor or abnormality of the adrenal gland, ectopic ACTH syndrome, or Cushing disease. In terms of treatment, it depends on the cause. An issue with the adrenal gland or tumor can be remedied with surgery. Other treatment options include radio therapy and adrenal-directed medications that control cortisol levels.
About the Agreement
Both SteroTherapeutics and the NICHD entered into this agreement with the intention of running a Phase 2 trial of a potential treatment for Cushing syndrome. This study will evaluate the dosage regimen, safety, and efficacy of the DHEA, which is meant to treat various manifestations of the syndrome, such as fatty liver disease and metabolic syndrome.
The terms of the agreement dictate that SteroTherapeutics will supply both the placebo and the medication, while the NICHD will actually run the study, which has been sanctioned under SteroTherapeutic’s Investigational New Drug authorization. The hope is that this trial is successful and allows for a Phase 3 trial, which will then lead to marketing authorization by 2023 or 2024.
Many Cushing syndrome patients are unable to utilize existing treatments, which makes the developments of this DHEA so important. Hopefully all further data regarding this therapy are positive.
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