{"id":1329,"date":"2023-08-10T15:23:56","date_gmt":"2023-08-10T15:23:56","guid":{"rendered":"https:\/\/pediatricbrainfoundation.org\/archive\/stem-cells-therapeutic-treatment-pediatric-neurological-disorders-planning-phase-i\/"},"modified":"2023-08-10T15:23:56","modified_gmt":"2023-08-10T15:23:56","slug":"stem-cells-therapeutic-treatment-pediatric-neurological-disorders-planning-phase-i","status":"publish","type":"page","link":"https:\/\/pediatricbrainfoundation.org\/archive\/stem-cells-therapeutic-treatment-pediatric-neurological-disorders-planning-phase-i\/","title":{"rendered":"Stem Cells as a Therapeutic Treatment for Pediatric Neurological Disorders: Planning a Phase I Clinical Trial"},"content":{"rendered":"
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\u00ab Back to Workshops<\/a><\/div>\n<\/section><\/div>\n
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Stem Cells as a Therapeutic Treatment for Pediatric Neurological Disorders: Planning a Phase I Clinical Trial<\/h1>\n<\/section><\/div>\n
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Subject:<\/b> \u00a0Stem Cells as a Therapeutic Treatment for Pediatric Neurological Disorders: Planning a Phase I Clinical Trial<\/i><\/p>\n

Location: \u00a0<\/b>Tarrytown, New York<\/p>\n

Attendees:<\/b> \u00a0A-T Children\u2019s Project representatives, Pediatric Brain Foundation representatives,\u00a0scientists, clinicians, representatives from the Food and Drug Administration\u00a0<\/span>(FDA), National Institutes of Health (NIH), biotechnology companies, and pediatric organizations.\u00a0<\/span><\/p>\n

Recap: \u00a0<\/b>In collaboration with the A-T Children\u2019s Project, Pediatric Brain Foundation sponsored a three-day conference from June 15-18, 2004 in Tarrytown, New York. The objective of this meeting was to plan research projects needed to move neural stem cell therapy from laboratory animals to Phase I clinical trials in children within 2-3 years.<\/p>\n

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This workshop, Stem Cells as a Therapeutic Treatment for Pediatric Neurological Disorders: Planning a Phase I Clinical Trial<\/i>\u00a0was attended by 36 of the most distinguished scientists and clinicians in the nation that included representatives from the Food and Drug Administration (FDA), National Institutes of Health (NIH), biotechnology companies, and a number of pediatric organizations. The participants, all of whom were engaged in research involving stem cells and\/or pediatric neurological disorders, spent the first two days eagerly exchanging presentations regarding their current work. The synergy created by bringing together these leading scientists led to discussions that were both exciting and productive.\u00a0<\/p>\n

After reviewing the current state of neural stem cell research, the participants began an important discussion of how to bring stem cell therapies to neurologically impaired children as quickly and effectively as possible. The immediate consensus for a first step was to identify a particular model of pediatric neurological disease that could be treated by stem cell therapy within the next two years. Participants methodically discussed the medical, scientific, regulatory and ethical issues that must be considered for such an ambitious undertaking.\u00a0<\/p>\n

By the end of the workshop, participants made significant strides in:<\/b>\u00a0<\/p>\n

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  1. Defining the key aspects of a workable disease model\u00a0<\/span>
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  2. Identifying the types of stem cells available for therapies<\/span>
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  3. Discussing strategies for ensuring that transplanted cells are not rejected by their recipient\u2019s immune system\u00a0<\/span>
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  4. Identifying the specific experiments that must be performed prior to approaching the FDA for approval of a clinical trial<\/span>
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  5. Outlining various other requirements for conducting clinical trials in children<\/span>
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    The workshop was universally hailed as a success. While there was incredible optimism that scientists are on the cusp of an extraordinary breakthrough in their ability to treat childhood neurological disorders with stem cells, there was also a sense that much work must be done before victory can be declared in earnest. Participants left with the resolve to do whatever is necessary to bring these therapies to children as quickly as possible.\u00a0<\/p>\n

    By bringing leading scientists together to chart this revolutionary course, Pediatric Brain Foundation is turning the hope of science into reality for children living with devastating neurological conditions. Unfortunately, despite the progress and dedication of these scientists, their research is severely under-funded. By supporting their work, Pediatric Brain Foundation is an advocate for the millions of children who anxiously await the fruits of their labor. These scientific breakthroughs will bring children living with neurological diseases ever closer to therapies and cures addressing the underlying causes of their disorders, not just the symptoms.<\/p>\n


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