Ok - I'm not a Dr.. but it is my understanding that Rett Syndrome is epigenetic, which would conform with it not being heritable and occurring after birth.
Now if it is epigenetic is there a possibility that that the following could occur?
1) early administration of 2-73 could limit damage done by the disease 2) reversal of some aspects of the disease is possible
I agree that the initial trial is probably partly because of safety, but information gathering will also be an important goal, consistent with Missling's precision medicine approach.
There is a reason why Kaufmann is CMO, and I don't think it is because he expects failure in Rett Syndrome:
Anavex Life Sciences Announces the Appointment of Walter E Kaufmann, MD as Chief Medical Officer
NEW YORK – January 2, 2019 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced the appointment of Walter E Kaufmann, MD to Chief Medical Officer (CMO).
Dr. Walter Kaufmann’s clinical focus is on developing novel therapies for genetic disorders associated with intellectual disability. Until recently, he was the Director of the Center for Translational Research at the Greenwood Genetic Center, where he also held the Ravenel Boykin Curry Chair in Genetic Therapeutics. He holds adjunct appointments at Emory University School of Medicine, where he is an Adjunct Professor of Human Genetics, and at the MIND Institute/University of California Davis School of Medicine, where he is a Visiting Scholar in the Department of Neurology. Dr. Kaufmann is also a Simons Investigator at the Massachusetts Institute of Technology’s Simons Center for the Social Brain. Before these academic appointments, Dr. Kaufmann was a Professor of Neurology at Harvard Medical School and a Professor of Pathology, Neurology, Pediatrics, Psychiatry, and Radiology at the Johns Hopkins University School of Medicine.
A major focus of Dr. Kaufmann’s research has been Rett syndrome, a field where he has published extensively on neurobiology and clinical aspects. He served as founder and leader of RettSearch, the international consortium of Rett syndrome clinical researchers. As such, he co-authored the current diagnostic guidelines for the disorder. He also edited the first clinical textbook on Rett syndrome, published in late 2017. Dr. Kaufmann has also served as Co-Principal Investigator of the NIH-funded Natural History Study of Rett syndrome (RDCRN program). Currently, he leads the Rett Syndrome Molecular Biomarkers Working Group for the Rettsyndrome.org foundation and the Rett Syndrome Behaviour Questionnaire Working Group. Dr. Kaufmann has played different roles, including site investigator, Principal Investigator, and DSMB member/chair, in almost 20 drug trials for neurodevelopmental disorders. In this context, he has been involved in virtually all neurobiologically-based drug trials for Rett syndrome.
Dr. Kaufmann has published more than 220 journal articles, most of them original publications. He has also served on several editorial boards and as reviewer for over 100 scientific journals.
“I am looking forward to working together with the Anavex team. I hope to bring expertise and experience to Anavex in order to succeed in the development of ANAVEX®2-73 for the severely devastating indication Rett syndrome but also Parkinson’s disease dementia and Alzheimer’s disease, since there is such a significant unmet medical need,” commented Walter Kaufmann, MD.
“We are pleased to welcome Dr Kaufmann to join Anavex’s team,” stated Christopher U Missling, PhD, President and CEO of Anavex. “Dr Kaufmann brings extensive experience in Rett syndrome and other related disorders to Anavex. I am looking forward to his involvement in execution of the ongoing clinical studies to the continued development of ANAVEX®2-73 for both neurodevelopmental and neurodegenerative diseases.”
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