The Advanced Therapy Production Unit (UPTA) is a pharmaceutical facility certified under Legislative Decree 219/2006 (Directive 2001/83/EC). The Italian Medicines Agency (AIFA) have authorized the Unit to manufacture neural stem cell-based medicinal products for clinical trials, with initial authorization granted in 2019 (aM 118/2019) and renewed in 2023 (aM 153/2023).
Currently, UPTA is focused on producing neural stem cells for the Phase II clinical trial EudraCT 2018-002373-22, titled "Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A multicenter, randomized, placebo-controlled, and biological endpoints clinical trial." This multicenter, randomized, placebo-controlled study investigates the use of neural stem cells as a treatment for amyotrophic lateral sclerosis (ALS).
In addition to its role in producing cellular medicines for clinical trials, the Unit conducts foundational research on neural stem cells, with a particular emphasis on exploring the neuroinflammatory mechanisms associated with these cells. In 2023, UPTA launched the development of a production process for induced pluripotent stem cells (iPSCs), intended to serve as starting material for advanced therapy medicinal products. This initiative marks a significant expansion of the Unit’s capabilities, creating new opportunities for the development of innovative stem cell-based therapies.
Name: Daniela Celeste Profico
University and Training: Degree in Medical Biotechnology obtained at the University of Teramo in 2008. Authorization as Qualified Person granted from the Italian Drug Agency in 2013. Master’s degree in Preclinical and Clinical Research obtained at University of Parma in 2021.
Work Experience: From 2008 to 2013 Head of Production at “Laboratorio Cellule Staminali, Cell Factory e Biobanca” of S. Maria Hospital in Terni.
Scientific Area of Interest: Production and characterization of advanced therapies medicinal products, development of new drugs for neurodegenerative diseases.
Collaborations: University Milano Bicocca, Laboratorio Cellule Staminali, Cell Factory e Biobanca (AOSP S. Maria, Terni), Università degli studi di Bari.
otal list of Publications from 2011: n.8
- Culturing and Expansion of "Clinical Grade" Neural Stem Cells from the Fetal Human Central Nervous System https://pubmed.ncbi.nlm.nih.gov/34558001/
- Retrieval of germinal zone neural stem cells from the cerebrospinal fluid of premature infants with intraventricular hemorrhage https://pubmed.ncbi.nlm.nih.gov/32475061/
- Transplantation of clinical-grade human neural stem cells reduces neuroinflammation, prolongs survival and delays disease progression in the SOD1 rats https://pubmed.ncbi.nlm.nih.gov/31024007/
- Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long-Term Outcome https://pubmed.ncbi.nlm.nih.gov/31104357/
- Establishment of stable iPS-derived human neural stem cell lines suitable for cell therapies https://pubmed.ncbi.nlm.nih.gov/30224709/
- Human Fetal Neural Stem Cells for Neurodegenerative Disease Treatment https://pubmed.ncbi.nlm.nih.gov/30209666/
- Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials? https://pubmed.ncbi.nlm.nih.gov/27720637/
- Human neural stem cell transplantation in ALS: Initial results from a phase I trial https://pubmed.ncbi.nlm.nih.gov/25889343/
- Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis https://pubmed.ncbi.nlm.nih.gov/38016468/
- Human Neural Stem Cell-Based Drug Product: Clinical and Nonclinical Characterization https://pubmed.ncbi.nlm.nih.gov/25889343/
- PNRR-MR1-2022-12375938 dal titolo Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis: A multicenter, randomized placebo controlled and biological endpoints clinical trial
- PNC PROGETTO HUB LIFE SCIENCE – TERAPIA AVANZATA (LSH-TA) PNC-E3-2022-23683269 finanziato dal Ministero della Salute nell’ambito del Piano Nazionale Complementare Ecosistema Innovativo della Salute” - Codice univoco investimento: PNC-E.3
Equipe
Gelati Maurizio
Ripoli Maria
- Biostatistic unit
- Bioinformatics Research Unit
- Cell reprogramming unit
- Medical genetics laboratory
- Functional genomics laboratory
- Huntington research and care unit
- Hemostasis and thrombosis laboratory
- Diabetes and endocrinology research laboratory
- Gastroenterology laboratory
- Hepatology laboratory
- Geriatrics laboratory
- Oncology laboratory
- Cancer Stem Cells unit
- Cancer Biomarkers unit
- Hematopathology unit
- Tissue engineering unit
- Advanced Therapies Production Unit