“The damage caused by steroid exposure is not surprising - it is expected.”
Deborah Gelinas, MD is a Neuromuscular Expert Medical Affairs for Argenx. She also serves as an Associate Professor in the Department of Neurology at the University of North Carolina, Chapel Hill, where she actively sees patients in the Amyotrophic Lateral Sclerosis (ALS) multidisciplinary clinic.
After earning her medical degree from New York Medical College in Valhalla, NY, she completed her residency in neurology at the Letterman Army Medical Center in affiliation with the University of California, San Francisco. She is a veteran of the Army Medical Corps and has received the Army Achievement Medal, the Army Commendation Medal, and the Meritorious Service Medal.
During Dr Gelinas’ time as an army physician, she took care of patients who were both active duty and retired service personnel. While training, she had completed a rotation with Robert Miller MD, at the California Pacific Medical Center, San Francisco, CA and whenever she was faced with a challenging neuromuscular disease that was beyond her brief as a general neurologist she would refer patients to him. It was a natural progression to go work with Dr Miller when it became time for her to move on from being an army physician.
“I took care of many patients with myasthenia gravis, and there are certain things Dr Miller told me to always keep in mind - most importantly that whenever you start a patient on steroids, you should be already thinking about the non-steroidal, immunosuppressive agent that you're going to migrate to so that you can get them off steroids.”
“Our protocol would be to add a non-steroidal, immunosuppressant once it was demonstrated that steroids were needed to achieve disease control, and then, as soon as possible, start the steroid taper. We had a very careful regimen for steroid taper - you would have to taper more slowly as you got lower and lower on dose for fear of a flare-up.”
During her time in academic research, she co-authored multiple publications involving amyotrophic lateral sclerosis (ALS), myasthenia gravis (MG), and chronic inflammatory demyelinating polyneuropathy (CIDP) in peer-reviewed journals including Neurology, the Journal of Neuroimmunology, and the Journal of Palliative Medicine. Dr Gelinas held a faculty position at Michigan State University College of Human Medicine, and was the Clinical Neuroscience Research Director at Mercy Health Saint Mary’s in Grand Rapids, MI.
“Over time I realized how infrequently you could ever get a patient completely off steroids. Once a patient was started on steroids, steroid dependence was the rule rather than the exception.”
“As I saw more and more patients, I saw more and more patients develop steroid toxicities, almost universally. It was common for people to develop a moon face, gain weight, encounter hair distribution changes, and complain about their skin. I felt these “minor side effects” were a small price to get control over a very, very serious disease.”
“But as I started to follow patients who had been on steroids longer, I started to see that there were fractures; hip fractures, vertebral fractures, and I started to really, really hate having to use steroids.”
Dr Gelinas continues by explaining that toxicity is related to both dose and duration, and unless you can get the dose to approximately 10 milligrams every two days, the steroids are going to eventually cause problems. Sadly, that goal is not often accomplished in myasthenia gravis patients.
“The damage caused by steroid exposure is not surprising - it is expected,” she says.
Following Dr Gelinas’ move to biopharma she continued to work with neuromuscular disease patients and her work with Argenx has enabled her to continue her important work in this area.
“When I looked at the Argenx ADAPT trial, which investigated the safety and efficacy of a new treatment for patients with myasthenia gravis, the steroids patients were taking at the start of the trial was shocking - 70% of them were still on steroids despite the average mean duration since diagnosis being nine and a half years!”
Dr Gelinas sees the development of the Steritas Glucocorticoid Toxicity Index (GTI) as incredibly beneficial to helping study the benefits of newer therapeutics as well as helping encourage and educate physicians about the need to measure and monitor steroid-toxicity with the aim of limiting the damage these powerful therapeutics can do. She believes it would also guide the taper process and help patients see that a potentially slow taper was yielding benefits.
Dr Gelinas sees the problem of steroid-toxicity as a global steroid pandemic - especially for myasthenia gravis patients, where often the first immunosuppressant a patient will be given is a steroid and there is often no off-ramp.
“There's really only one country that impresses me as making a careful attempt to avoid steroids entirely, and not rely upon steroids for disease control, and that's Japan. The Japanese guidelines are different from the international guidelines, in that they truly are very cognizant of the fact that the burden on the patient will be greater with steroids, and so they go out of their way to avoid them.”