UW Program for Advanced Cell Therapy

MSC v2.0 are a solution looking for a problem to fix. Randall Kimple is a UW School of Medicine and Public Health radiation oncology physician-scholar who provides the clinical context and patient perspective on matching MSC pharmacological attributes to the clinical investigational use case for treating severe dry mouth in throat cancer survivors that received radiation therapy to their necks. For academic and industry lab scientists, technologists, executives and regulators, the podcast provides the end user patient viewpoint and clinical care provider motivations and aspirations in developing impactful cell therapies that improve quality of life. This is not abstraction and is the thoughtful and on the ground informed podcast provides wonderful context for the development of MSC 2.0 for this and other clinical use cases for which they are well suited. Jacques Galipeau Associate Dean for Therapeutics Development UW School of Medicine and Public Health

UW Program for Advanced Cell Therapy Successfully Completes Phase I Dose Escalation Study of personalized cell therapy for dry mouth in throat cancer survivors and secures DSMB Approval to Proceed to Dose Expansion Cohort of 12 additional subjects. This is a single center academic clinical trial conducted at the UW Health and partnered with UW Carbone Cancer Center is open to recruitment of subjects far and wide. Let by Randall Kimple and Grace Blitzer, the study seeks to restore salivary gland function in individuals that have survived a diagnosis of throat cancer more than 2 years suffering from radiation-induced severe xerostomia (dry mouth).

Melissa Rohrer joins UW Program for Advanced Cell Therapy as incoming Director of Regulatory Affairs. Dr Rohrer brings a depth of expertise in industrial development of biologics and regulatory management to our program and will further advance the regulatory component of our mission to develop and de-risk academic cell therapy technologies through early phase clinical trials and future partnered commercial development. She joins a crack team of productive professionals engaged in the Wisconsin Idea as it applies to impactful cell pharmaceutical innovations spun from the UW School of Medicine and Public Health research enterprise assigned to Wisconsin Alumni Research Foundation (WARF).

Accompanied by UW School of Medicine and Public Health physician scholars Sara McCoy and Randall Kimple, Jacques Galipeau presented on October 7th at Alliance for Regenerative Medicine on the MESA meeting in Phoenix the results of of a Phase 0 study NCT04489732 of 6 subjects MSCG-001 (autologous MSC(M) activated with INFγ) to treat xerostomia. A call for partnership with CGT partners and funders was made to launch a new clinical stage biopharma spinoff arising from this academic IND sponsored trial. UW Program for Advanced Cell Therapy is an academic CGT incubator that vertically integrates clinical trial expertise, in-house GMP cell therapy manufacturing and FDA regulatory competencies bundled to advance IIT INDs through Phase I clinical trials. MSCG-001 provides an excellent safety profile and displays an anecdotal signal of efficacy as measured by QOL and saliva production outcomes and serves as the inaugural test case for our de-risking of academic CGTs on a trajectory for commercial development. A pipeline of academic INDs and IDEs enabled by a crack multidisciplinary PACT team are forthcoming.

Kimberly McDowell PhD is the inaugural Honorary Fellow of Regulatory Affairs as of October 1 2024, having retired as Director following five years of glorious service with UW Program for Advanced Cell Therapy. Dr. McDowell had previously overseen all things regulatory to ensure rigorous administrative FDA compliance for the PACT program, including crafting and managing Food and Drug Administration Center for Biologics Evaluation and Research investigational new drug and investigational device exemption applications for PACT-sponsored clinical trials and regulatory compliance. She will continue to contribute to PACT as an Emerita.

UW Program for Advanced Cell Therapy (PACT) secures new FDA IDE to restore innate immunity post allogeneic BMT. On July 2 2024 FDA CBER approved the PACT-sponsored Investigational Device Exemption (IDE) application # 30707 named “Allogeneic, TCR αβ and CD19+ cell-depleted peripheral blood mononuclear cells”. Allogeneic BMT patients receiving post-transplant cyclophosphamide to reduce risk of GVHD display impaired innate immunity manifest by an increased risk of viral reactivation. This single center phase I Investigator-Initiated clinical trial led by UW School of Medicine and Public Health BMT section chief Hongtao Liu will examine the safety and efficacy of related-donor day 30 TCR αβ and CD19+ cell-depleted DLI to accelerate innate immune recovery. This milestone was made possible by PACT team effort involving regulatory (Kimberly McDowell), cell manufacturing (Ross Meyers, Olga Ganz, Jessica Murphy) and by support from Hanssens Linda and Michaela Malchow from Miltenyi Biotec. Study activation and patient enrollment is expected in Q1/2025. UW Carbone Cancer Center partnered with UW Health lead the way in driving clinical care innovations and improve outcomes for curative allogeneic bone marrow transplant for the benefit of our patients and the healthcare community at large. #FDA #celltherapy #IDE #BMT #UWHealth #UWCCC #UWSMPH

UW Program for Advanced Cell Therapy joins Alliance for Regenerative Medicine as a Research Institution member. UW Program for Advanced Cell Therapy (PACT) is a partnership between UW School of Medicine and Public Health and UW Health. Our vision is to become the national leader in personalized cell therapies. In partnership with Wisconsin Alumni Research Foundation (WARF), our mission is to accelerate the commercial development of UW cell therapy innovations by engaging in first-in-human clinical trials at UW Health supported by PACT GMP facility. We are now expanding our commercial outreach through a new Research Institution membership with Alliance for Regenerative Medicine where the full commercial potential of our validated cell therapy platforms and clinical use cases can be brought to light. https://lnkd.in/etEb8nni

UW Program for Advanced Cell Therapy milestone met of sixth and last pilot study (NCT04489732) subject receiving a second administration of autologous MSC in salivary gland to treat radiation-induced dry mouth. In this pilot study, 6 subjects were to receive a single unilateral submandibular salivary gland injection to assess for safety in anticipation of a larger phase I trial to examine bilateral gland injection. All 6 subjects were offered post hoc to have their contralateral gland injected as part of of an amended protocol and 100% wished to do so and this was completed last week. Their outcomes as well as that of on-going larger phase I trial (NCT05820711) will be presented by study clinical PI Randall Kimple at the ISCT, International Society for Cell & Gene Therapy global meeting in Vancouver June 1 2024.

UW Program for Advanced Cell Therapy has an open position for a Regulatory Scientist. Join a dynamic team led by Jacques Galipeau pushing forward innovative cell therapies for cancer survivors and other disorders. Interested candidates should apply to the link posted below. https://lnkd.in/eqsstZV6

UW Program for Advanced Cell Therapy sponsored pilot clinical trial update gets oral presentation at ISCT, International Society for Cell & Gene Therapy global meeting in Vancouver on May 30, 2024. The abstract “A pilot, first in human study of autologous IFN-gamma stimulated, refreshed bone marrow mesenchymal stromal cells for treatment of radiation-induced xerostomia – 1 year results.” First authored by Grace Blitzer, the abstract will be presented by study PI Randall Kimple. In addition, Andrea Pennati PACT Director of R&D will present two posters at the same meeting: “Enhanced Transfection of Bone-Marrow Derived Mesenchymal Stromal Cells With Messenger RNA” co-authored with Adam Pagenkopf and Leisha Kopp as well as “Deciphering the secretome of mouse regulatory B cells on the polarization of lymphomyeloid cells in a mouse model of Multiple Sclerosis” co-authored with Xingyi Tang and Catigan Hedican. We are looking forward to a terrific meeting! Jacques Galipeau, Director, PACT