Contributed by Eugenie Hsu, Ph.D.
In 2016, my son was 13 years old and a 5-year-survivor of a brain tumor called craniopharyngioma. While we were grateful that he had survived the tumor and surgery, his quality of life was poor due largely to hypothalamic obesity with hyperphagia, a cruel condition characterized by symptoms including uncontrollable hunger and rapid weight gain. To prevent him from eating as much as his ravenous appetite dictated, we resorted to locking up all food and keeping a constant watch of him lest he steal food. It was an exhausting, depressing, and unsustainable lifestyle.
In my desperation to find help for my son’s unrelenting hunger, I networked with other parents in Facebook support groups and read everything I could in the medical literature on the topic of hypothalamic obesity, hyperphagia, and panhypopituitarism. Despite my son’s complete loss of hormone production, I learned that not all hormones are replaced. To my surprise, I then learned about oxytocin, a neurohormone whose functions closely matched the untreated symptoms of my son’s hypothalamic obesity. Because Prader Willi syndrome (PWS), a rare congenital condition with many similar traits to craniopharyngioma (including hyperphagia), I also delved into reading research on PWS. Through my research, I came across Dr. Jennifer Miller, the beloved pediatric endocrinologist and the champion for kids with PWS. Dr. Miller had conducted research on the use of oxytocin for kids with PWS and after I contacted Dr. Miller and corresponded with her about her research with oxytocin, I became even more determined to try it. Being from California, I found a progressive endocrinologist in Los Angeles who was willing to prescribe off-label oxytocin to my son but he did not have experience using it for the treatment of hypothalamic obesity; thankfully, Dr. Miller was willing to be a medical advisor to me while I applied intranasal oxytocin as an experimental treatment for my son’s hypothalamic obesity and hyperphagia.
In the late spring of 2016, we began our journey with oxytocin (and later added naltrexone, an opiate antagonist, used to deter the cravings in alcohol and opiate addiction). During the experimental treatment, I also documented our experiences in real time in a blog, Hope for HO. Although I did not know the outcome of our experiment, I thought that it had enough promise when we experienced the HEFY or Half Eaten Frozen Yogurt (see this post for info) and I was excited to share our experiences with the world. In spite of the many ups and downs we experienced, the “oxytocin experiment” was a success; his relationship to food normalized which no longer required locking up all food and his weight was reduced to a normal range. With the support of co-authors, Drs. Christian Roth, Francisco Perez, and Jennifer Miller, I wrote and published our findings into a case report in the Journal of Clinical Endocrinology and Metabolism. To date, five years later, my son still takes intranasal oxytocin and naltrexone and has continued to have an improved relationship to food and to keep his weight within a normal range.
In spite of our successful experimental use of oxytocin, hypothalamic obesity continues to be an intractable medical condition, and oxytocin, although promising, is still very much needing more study. Fortunately, there are scientists and practitioners devoted to the research of oxytocin and metabolism and the Raymond A. Wood Foundation is pleased to present an online panel discussion by three such top researchers: Dr. James E. Blevins (University of Washington); Dr. Elizabeth Lawson (Harvard University); and Dr. Jennifer Miller (University of Florida). Please join us on March 25, 2021 at 7:30 ET for a stimulating Q and A with these experts. Register here and come prepared with your questions!