{"id":1333,"date":"2017-03-01T21:33:15","date_gmt":"2017-03-01T21:33:15","guid":{"rendered":"https:\/\/www.mc.vanderbilt.edu\/vanderbiltmedicine\/?p=1333"},"modified":"2021-08-11T19:40:23","modified_gmt":"2021-08-11T19:40:23","slug":"investigational-new-drug-for-alzheimers-scheduled-for-first-study-in-humans","status":"publish","type":"post","link":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/investigational-new-drug-for-alzheimers-scheduled-for-first-study-in-humans\/","title":{"rendered":"Investigational new drug for Alzheimer\u2019s scheduled for first study in humans"},"content":{"rendered":"
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Illustration by Dan Page<\/figcaption><\/figure>\n

Vanderbilt University scientists have received notification from the U.S. Food and Drug Administration (FDA) that testing in humans may proceed for an investigational new drug for Alzheimer\u2019s disease after more than 10 years of research by scientists at Vanderbilt University and Vanderbilt University Medical Center.<\/p>\n

It is relatively uncharted territory for an academic drug discovery group to take a molecule from the laboratory setting to the clinical trials stage.<\/p>\n

\u201cThis work exactly illustrates the critical role that basic science conducted in partnership with a world-class medical center can play in advancing knowledge in an attempt to fight a devastating disease,\u201d said Provost and Vice Chancellor for Academic Affairs Susan R. Wente, Ph.D.<\/p>\n

The aim is for the investigational drug to target major pathologies of Alzheimer\u2019s disease and selectively activate a key receptor in the brain. The Vanderbilt researchers believe that the current standard of care for the disease, cholinesterase inhibitors, has a different mechanism of action. They are hoping to establish through future clinical testing that the molecule is broadly effective across a number of cognitive and neuropsychiatric disorders, including schizophrenia.<\/p>\n

\u201cThis is the first instance I am aware of where an academic drug discovery group moved a molecule designed to hopefully treat a chronic brain disorder all the way from early discovery to human trials without there being, at some point along the way, a pharmaceutical partner,\u201d said P. Jeffrey Conn, Ph.D., Lee E. Limbird Professor of Pharmacology in the Vanderbilt University School of Medicine and director of the Vanderbilt Center for Neuroscience Drug Discovery (VCNDD).<\/p>\n

\u201cAnd that really is crossing what people refer to all of the time as the \u2018Valley of Death,\u2019 where good research discoveries have a hard time moving into the clinical testing phase due to lack of funding,\u201d he said. \u201cImportantly, at this early stage, the FDA has only granted permission to assess potential safety of this investigational new drug in healthy volunteers,\u201d said Conn. \u201cWe cannot predict the outcome, but if these studies are successful in demonstrating that the investigational drug can be safely administered to humans, this would pave the way to allow filing of additional applications with the FDA to seek permission to advance to testing for efficacy in improving cognitive function in patients suffering from Alzheimer\u2019s disease, and possibly schizophrenia or other brain disorders.<\/p>\n

VCNDD Co-Director Craig W. Lindsley, Ph.D., director of Medicinal Chemistry and William K. Warren Jr., Professor of Medicine, said Phase I testing will assess drug safety and tolerability in healthy volunteer participants, a process that could take a year. If successful, the Phase II and III studies would include efficacy assessments in patients with Alzheimer\u2019s disease and could take three to five years to complete.<\/p>\n

\u201cWe are hoping to address what we see as an unmet medical need,\u201d Lindsley said. \u201cFor Alzheimer\u2019s patients, the standard of care for symptomatic treatment remains cholinesterase inhibitors, which are 25 years old at this point. There hasn\u2019t been any real scientific advancement in this field in a long time.\u201d<\/p>\n

Lindsley and Conn credit The William K. Warren Foundation for its investments along the way to make clinical trials for this investigational drug a reality.<\/p>\n

\u201cOne of the most challenging things about doing this in an academic environment is funding,\u201d Lindsley said. \u201cEvery step requires funding and if there is a delay or break in funding, then everything sits idle and potentially innovative approaches for patient care do not advance.\u201d<\/p>\n

\u201cBeing matched with the Warrens happened serendipitously. They have invested so much in our programs, and it is wonderful to show them progress on their investments,\u201d he said. \u201cWithout the financial support from the Warrens, this investigational drug would not be poised to enter human clinical trials.\u201d<\/p>\n

The William K. Warren Foundation Chief Executive Officer John-Kelly Warren said he is gratified that FDA has allowed for the investigational drug to proceed to testing in human beings.<\/p>\n

\u201cAlthough this is an important sequential milestone, the only milestone that matters to us is the hope that one day we will learn that this investigational new drug has positively and safely changed the life of a patient suffering from a brain disorder such as schizophrenia or Alzheimer\u2019s disease,\u201d Warren said.<\/p>\n

\u201cThat day will warrant a celebration felt in the heavens. Until then, we are prepared to support the VCNDD research team until they can deliver the necessary results,\u201d he said.<\/p>\n

A NIH National Cooperative Drug Discovery\/Development grant funded the early basic science and discovery of this investigational drug and the Alzheimer\u2019s Drug Discovery Foundation and Harrington Discovery Institute helped support some of the key toxicity studies that FDA required, Conn said.<\/p>\n

\u201cThe investigational new drug has the potential to improve cognitive functions with fewer unwanted side effects. This could someday be an important advance for the treatment of cognitive deficits in psychiatric disorders and Alzheimer\u2019s disease,\u201d said Joshua Gordon, M.D., Ph.D., director of the National Institute of Mental Health, which co-funded the research.<\/p>\n

 <\/p>\n

 <\/p>\n","protected":false},"excerpt":{"rendered":"

Vanderbilt University scientists have received notification from the U.S. Food and Drug Administration (FDA) that testing in humans may proceed for an investigational new drug for Alzheimer\u2019s disease after more than 10 years of research by scientists at Vanderbilt University and Vanderbilt University Medical Center. It is relatively uncharted territory for an academic drug discovery…<\/p>\n","protected":false},"author":211,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"jetpack_post_was_ever_published":false,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_publicize_message":"","jetpack_publicize_feature_enabled":true,"jetpack_social_post_already_shared":false,"jetpack_social_options":{"image_generator_settings":{"template":"highway","default_image_id":0,"font":"","enabled":false},"version":2},"_links_to":"","_links_to_target":""},"categories":[11,31],"tags":[],"class_list":["post-1333","post","type-post","status-publish","format-standard","hentry","category-vm-around-the-medical-center","category-winter-2017"],"acf":[],"jetpack_publicize_connections":[],"jetpack_featured_media_url":"","jetpack_sharing_enabled":true,"jetpack_shortlink":"https:\/\/wp.me\/pcDnub-lv","_links":{"self":[{"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/posts\/1333","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/users\/211"}],"replies":[{"embeddable":true,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/comments?post=1333"}],"version-history":[{"count":1,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/posts\/1333\/revisions"}],"predecessor-version":[{"id":3085,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/posts\/1333\/revisions\/3085"}],"wp:attachment":[{"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/media?parent=1333"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/categories?post=1333"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medschool.vanderbilt.edu\/vanderbilt-medicine\/wp-json\/wp\/v2\/tags?post=1333"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}