Gorham-Stout Remedies
GSD (Gorham-Stout Disease) is a vascular anomaly thought to be an error in the development of the lymphatic system. As bones are routinely broken down, they are replaced with lymphatic vessels and a fibrous band of connective tissue instead of more bone. Also known as Vanishing Bone Disease, it is listed in the international directory of rare diseases (https://rarediseases.org/rare-diseases/gorham-stout-disease/ and https://www.childrenshospital.org/conditions/gorham-stout-disease#:~:text=How we care for Gorham-Stout disease).
Up until recently, it was thought to be a disease found exclusively in children, but now that more adults are reporting having symptoms, there is thought that it might even be a form of osteoporosis. Signs of the disease can vary, but for me, it first manifested as small divets or depressions in the skull I could feel with my fingers that grew larger with time. I wore hair extensions for years and thought it might have been caused by that.
My current understanding is that the diagnosis cannot be fully confirmed without doing a skull biopsy, and this is a high-risk procedure that often results in ongoing cerebral-spinal fluid leakage. It is discouraged by the doctors I have seen. Right now there is no known cure for Gorham-Stout. Current therapy is experimental and consists of a combination of bisphosphonates, interferon and an immunosuppressant drug called sirolimus. All have disturbing side-effects that seem - at least at this point in my situation - potentially worse than the disease itself. I personally have a poor tolerance of bisphosphonates, and my mother, in fact, died as a direct result of her taking Fosamax.
If anyone has knowledge of this disease and any recommendations or referrals for me to contact I would be extremely grateful. Any information at all would be helpful. Thank you.
Shana,
Sirolimus/Rapamycin does have significant side effect potential, however it has demonstrated effectiveness in GSD as discussed here :
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7446191/
Here is a relevant quote from the case report :
' We prescribed sirolimus, which is an oral mTOR inhibitor, for two consecutive years. The boy recovered well without other progressive bone lesions and participates in normal daily activities. His growth and development are the same as that of his peers.'
Interestingly, melatonin has shown the ability to reduce the side effects of Rapamycin while being additive to its positive health effects as discussed here :
https://scholars.uthscsa.edu/en/publications/combination-of-melatonin-and-rapamycin-for-head-and-neck-cancer-t
Moreover, melatonin has shown to exert protective effects against bone degeneration as discussed here :
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7979260/#:~:text=Melatonin also attenuates the autophagy, calcium metabolism and prevents osteoporosis.
Here is a relevant quote :
' Melatonin exerts numerous physiological effects, including inducing anti-inflammatory and antioxidative functions, resetting circadian rhythms and promoting wound healing and tissue regeneration. Melatonin also participates in the health management of bone and cartilage. In the present review, the potential roles of melatonin in the pathogenesis and progression of bone injury, osteoporosis, OA and periodontitis are summarized. Furthermore, the high efficiency and diversity of the physiological regulatory effects of melatonin are highlighted and the potential benefits of the use of melatonin for the clinical prevention and treatment of bone-related diseases are discussed. '
Since melatonin has synergy with Rapamycin, it is possible that that effect may allow you to use a lower dose of sirolimus, potentially further reducing the potential of any side effects. Melatonin has many know health benefits and works to maintain homeostasis in the body, which may mean that melatonin can work in trying to prevent the cause of GSD.
Art
(North Carolina)
04/24/2023
Thank you so much for your reply, Art. I am familiar with this research and am already on melatonin and a commercially available calcium w/strontium supplement program called AlgaeCal. The research studies look very good on paper, but my endocrinologist thinks the results are too good to be true. Still, she is supporting me in taking it because I cannot take the bisphosphonates. Current GSD doctors won't administer Sirolimus without the bisphosphonates, so this is something of a sticking point right now. Also, the docs I am seeing have not been able to show me "numbers", so that I can see what changes are occurring between dexa scans. Is there any other way to detect improvement or remission? How else do they know what is or isn't working?
(Kitchener On)
04/22/2023
(North Carolina)
04/24/2023
Yomama,
The bisphosphonates are used for osteopenia and osteoporosis, so this sounds like one part of your treatment is to improve the bone material itself. I believe that melatonin is better for this purpose as well as the common supplements used for osteoporosis such as boron, vitamin D, magnesium glycinate, vitamin K2 menaquinone 7, strontium, berberine, calcium, etc.
The bisphosphonates seem to have serious side effects and in some cases actually increase risk of fracture as outlined here :
Here is a relevant quote :
' In this subgroup of bisphosphonate-treated patients that suffered a fracture, the accumulation of microcracks following treatment with bisphosphonates may have compromised the trabecular microstructure. As a result, there may have been weakening of the bone and consequently, an increased risk of fracture. '
This is the opposite of what an osteoporosis treatment should do! I think the natural methods such as melatonin and ones listed above would be much better for the purpose of improving bone integrity and when you look at the listed side effects of bisphosphonates, it really makes you wonder. Here are side effects related to the bisphosphonate, Fosamax :
https://www.drugs.com/fosamax.html#side-effects
Fosamax side effects
Get emergency medical help if you have signs of an allergic reaction to Fosamax: hives; wheezing, difficulty breathing; swelling of your face, lips, tongue, or throat.
Stop using Fosamax and call your doctor at once if you have:
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chest pain, new or worsening heartburn;
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difficulty or pain when swallowing;
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pain or burning under the ribs or in the back;
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severe heartburn, burning pain in your upper stomach, or coughing up blood;
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new or unusual pain in your thigh, hip or groin;
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jaw pain, numbness, or swelling;
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severe joint, bone, or muscle pain; or
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signs of low calcium levels - muscle spasms or contractions, numbness or tingly feeling (around your mouth, or in your fingers and toes).
Common Fosamax side effects may include:
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heartburn, upset stomach;
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stomach pain, nausea;
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diarrhea, constipation; or
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headaches; or
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bone pain, muscle or joint pain.
This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
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Art
