Estriol (also oestriol or E3) is one of the three main estrogens produced by the human body. Estriol is only produced in significant amounts during pregnancy as it is made by the placenta.
A study published online in Lancet Neurology, a team of UCLA took observations from the bedside, tested them in the laboratory and took those findings back again to patients in clinical trials, said the study’s lead author Dr. Rhonda Voskuhl, professor in the UCLA Department of Neurology and director of UCLA’s Multiple Sclerosis Program.
Women who are pregnant with multiple sclerosis often see little to no disease activity during pregnancy.
In a Phase II randomized, placebo controlled study researchers at UCLA look to discover why women with relapsing-remitting MS have a reduction in relapses. Interestingly this protection also occurs in other autoimmune disorders scuh as rheumatoid arthritis and psoriasis.
The Phase II trial was begun back in 2007 at UCLA and 15 other sites across the United States.
“The beauty of estriol is that it is not a shot and can be taken in pill form, and also that it’s not a new drug. It has decades of safety behind it,” said Voskuhl, who holds the Jack H. Skirball Chair for Multiple Sclerosis in the UCLA Department of Neurology. “Also, current MS treatments are very complex to manufacture. These findings hopefully will pave the way for oral, safe treatments that are more widely accessible, since estriol is simple and naturally occurring.”
Voskuhl and her team discovered that estriol potentially both reduces the ability for immune cells to attack the brain but also makes brain cells resist damage. Estriol treatments improved cognition and reduced atrophy as well. It is suspected that estriol protects a fetus brain and prevents rejection of the fetus as a foreign item. It appears as though Estriol also protects the mother from immune attacks on the brain.
Back in 2002, Voskuhl performed a small pilot study with 10 women with MS who were given estriol, she witnessed a 70 percent drop in inflammatory lesions in the brain within only six months.
This Phase II study had 164 MS patients where 83 were given estriol and 81 given placebo. All of the participants continued their injections of glatiramer acetate (Copaxone). Th research found that the participants taking estriol had 1/3 to half as many relapses as those taking placebo. These results additive to the impact of the disease modifying therapy. When estriol levels were at their highest levels cognition was also improved and brain atrophy was shown to be reduced.
The study proceeded for two years and the only side effect witnessed was some irregular menstruation. Additionally no portions of the pilot trial or this phase II trial were in any way funded by any pharmaceutical companies but were funded by the National Institutes of Health and the Multiple Sclerosis Society.
Synthetic Biologics, Inc., provided estriol and placebo for the multicenter trial and has licensed certain rights from UCLA calling their medication “Trimestra”
ROCKVILLE, Md., Nov. 25, 2015 /PRNewswire/ — Synthetic Biologics, Inc. (NYSE MKT: SYN), a clinical stage company focused on developing therapeutics to protect the gut microbiome while targeting pathogen-specific diseases, announced today that Rhonda Voskuhl, M.D., Professor, Department of Neurology, Jack H. Skirball Chair in Multiple Sclerosis Research and Director, Multiple Sclerosis Program at the University of California, Los Angeles (UCLA) David Geffen School of Medicine, and the lead principal investigator of the Phase 2 clinical trial that evaluated Trimesta™ as a treatment for relapsing-remitting multiple sclerosis (RRMS) in women, published additional clinical trial data in the journal, Lancet Neurology.
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As previously disclosed in July 2015, Synthetic Biologics was informed by UCLA that magnetic resonance imaging (MRI) analyses were ongoing to evaluate changes in the brain that correlate with improvements seen in clinical outcomes. With the publication of the data by Dr. Voskuhl, the Company has received topline MRI data and UCLA’s related analysis. Upon receipt of the MRI data set, as is typical with academic collaborations, Synthetic Biologics commenced a thorough third party analysis in order to confirm UCLA’s results for potential business development discussions. The Company continues discussions with the neurology community and potential strategic partners, as it determines next steps for Trimesta.
Synthetic Biologics, Inc. is a clinical-stage company developing therapeutics to protect the gut microbiome while targeting pathogen-specific diseases. The Company’s lead candidates in Phase 2 development include SYN-004 which is designed to protect the gut microbiome from the effects of certain commonly used intravenous (IV) antibiotics for the prevention of C. difficile infection and antibiotic-associated diarrhea (AAD), and SYN-010 which is intended to reduce the impact of methane producing organisms in the gut microbiome to treat the underlying cause of irritable bowel syndrome with constipation (IBS-C).
In addition, the Company is developing a Phase 2 oral estriol drug for the treatment of relapsing-remitting multiple sclerosis (MS) and cognitive dysfunction in MS, and in collaboration with Intrexon Corporation (NYSE:XON), a preclinical stage monoclonal antibody for the prevention and treatment of Pertussis, and novel discovery stage biotherapeutics for the treatment of phenylketonuria (PKU).