{"id":13021,"date":"2025-11-14T09:04:38","date_gmt":"2025-11-14T15:04:38","guid":{"rendered":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin-new\/?post_type=campus_story&p=13021"},"modified":"2025-11-14T09:05:10","modified_gmt":"2025-11-14T15:05:10","slug":"parkinsons-treatment-tested-at-uw-showing-promise-in-first-clinical-trial","status":"publish","type":"campus_story","link":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/story\/parkinsons-treatment-tested-at-uw-showing-promise-in-first-clinical-trial\/","title":{"rendered":"Parkinson\u2019s treatment tested at UW-Madison showing promise in first clinical trial"},"content":{"rendered":"
\"Image:<\/a>
Parkinson\u2019s Disease as a progressive neurological disorder affecting body movement and coordination as neuron cells losing function in the substantia nigra section of the human brain as a degenerative illness. ILLUSTRATION: iSTOCK\/ WILDPIXEL<\/figcaption><\/figure>\n

Parkinson\u2019s patients are receiving a new investigational treatment after a successful study at the University of Wisconsin\u2013Madison helped demonstrate the cell therapy\u2019s safety in non-human primates and refine the method for its delivery. Now, the biotechnology company running the trial is reporting encouraging results.<\/p>\n

Aspen Neuroscience<\/a>\u00a0recently announced they would enroll a third group of patients in their trial, called ASPIRO. The ASPIRO trial is referred to as Phase 1\/2a, a stage designed to evaluate an investigational treatment\u2019s safety and most effective dose in humans.<\/p>\n

The patients already enrolled in the trial are safely tolerating the treatment, in which new brain cells, called neurons, are grown from the patients\u2019 own cells and grafted into key parts of the brain. Their doctors have noted improvement in patients\u2019 Parkinson\u2019s symptoms, potentially providing promise for people living with this debilitating neurological disease.<\/p>\n

\u201cEarly data from the first four patients show that precision intracranial delivery of [the neurons] is safe and well tolerated,\u201d says Andr\u00e9s\u00a0Bratt-Leal, Aspen co-founder and senior vice president of research and development. \u201cBoth patient-reported and clinician-reported outcomes show early signs of changes from baseline in multiple clinically meaningful outcome measures. Autologous iPSC-derived therapy as a regenerative medicine approach has the unique advantage of not requiring immunosuppressive medications.\u201d<\/p>\n

In 2024, researchers at the\u00a0Wisconsin National Primate Research Center<\/a>\u00a0successfully grafted progenitor cells \u2014 which develop into the types of neurons that produce a brain chemical called dopamine \u2014 into the brains of macaque monkeys. California-based Aspen provided the cells, grown from multiple lines of human-induced pluripotent stem cells, along with the equipment for delivering them to specific parts of the brain. Key to Aspen\u2019s cell-therapy approach in people is deriving the new brain cells from cells donated by the patient who will receive the graft. Scientists call that an \u201cautologous\u201d transplant.<\/p>\n

\u201cUsing a patient\u2019s own cells avoids the need to use immunosuppression to keep the patient\u2019s body from rejecting or attacking the graft,\u201d says\u00a0Marina Emborg, a UW\u2013Madison professor of medical physics<\/a>. \u201cAspen has developed the technological methods for manufacturing, for quality control, that makes it feasible at scale to make autologous cells and get them to the patients.\u201d<\/p>\n

Parkinson\u2019s disease damages neurons that produce dopamine, a hormone that transmits signals between nerve cells. The disrupted signals make it progressively harder to coordinate even simple movements and cause rigidity, slowness and tremors that are the disease\u2019s hallmark symptoms. Patients are typically treated with drugs like L-DOPA to increase dopamine production. Although the drugs help many patients, they present complications and lose their effectiveness over time.<\/p>\n

\u201cBy the time of diagnosis, it is common for people with Parkinson\u2019s to have lost the majority of dopaminergic neurons, leading to progressive loss of motor and neurological function,\u201d explains Edward Wirth III, an expert in cell therapies, Aspen\u2019s chief medical officer and a collaborator on the 2024 study. \u201cTo replace these lost cells, we must target a very specific area of the brain with a high degree of surgical precision.\u201d<\/p>\n

Using the latest advances in MRI-guided surgical techniques, the patient\u2019s new cells are transplanted, a few microliters at a time, to the exact area where they are most needed.<\/p>\n

Working with potential cell therapies in pursuing treatments for Parkinson\u2019s disease is a particular specialty of the Emborg lab and other primate center colleagues. The researchers\u2019 results in non-human primates, which supported Aspen\u2019s successful application to begin human trials, were published\u00a0in the Journal of Neurosurgery<\/a>. The study followed Emborg\u2019s success, alongside\u00a0Su-Chun Zhang<\/a>\u00a0of the Wisconsin School of Medicine and Public Health,\u00a0reversing Parkinson\u2019s symptoms in monkeys<\/a>\u00a0with their own autologous cell graft approach.<\/p>\n

The 2024 study was designed to test the safety and efficacy of Aspen\u2019s human cells and a delivery method that allowed surgeons to reach their target in the brain with fewer insertions of the apparatus that delivers the new cells.<\/p>\n

\u201cThis study was an important step in our work to bring the promise of a cell-replacement therapy to people with Parkinson\u2019s disease,\u201d says Bratt-Leal, who co-authored the 2024 study. \u201cThe results were instrumental in opening our first-in-human trial and informing how we deliver patients\u2019 own cells to them in the study.\u201d<\/p>\n

It was a true collaboration according to Emborg \u2014 between the Aspen scientists, her lab and the Wisconsin National Primate Research Center \u2014 to validate the company\u2019s procedures and equipment. The work done to refine the logistics, surgical equipment and techniques in the animal procedures informed the way patients in the human trial have received and recovered from the new therapy, providing hope for those struggling with this debilitating disease.<\/p>\n

\u201cOur results were all so exciting,\u201d Emborg says. \u201cAnd then, when I saw they had been able to begin with a human patient \u2026 I just had tears in my eyes.\u201d<\/p>\n

\n

Written by Chris Barncard<\/p>\n

Link to original story: https:\/\/news.wisc.edu\/parkinsons-treatment-tested-at-uw-showing-promise-in-first-clinical-trial\/<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

Parkinson\u2019s patients are receiving a new investigational treatment after a successful study at the University of Wisconsin\u2013Madison helped demonstrate the cell therapy\u2019s safety in non-human primates and refine the method for its delivery. Now, the biotechnology company running the trial is reporting encouraging results. Aspen Neuroscience\u00a0recently announced they would enroll a third group of patients […]<\/p>\n","protected":false},"author":15,"featured_media":13024,"comment_status":"closed","ping_status":"closed","template":"","institution":[103],"story_category":[],"class_list":["post-13021","campus_story","type-campus_story","status-publish","has-post-thumbnail","hentry","institution-uw-madison"],"_links":{"self":[{"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/campus_story\/13021","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/campus_story"}],"about":[{"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/types\/campus_story"}],"author":[{"embeddable":true,"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/users\/15"}],"replies":[{"embeddable":true,"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/comments?post=13021"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/media\/13024"}],"wp:attachment":[{"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/media?parent=13021"}],"wp:term":[{"taxonomy":"institution","embeddable":true,"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/institution?post=13021"},{"taxonomy":"story_category","embeddable":true,"href":"https:\/\/wwwtest.wisconsin.edu\/all-in-wisconsin\/wp-json\/wp\/v2\/story_category?post=13021"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}