CARMIT (Children’s Access to Regenerative Medicine in Texas) is a core service of the Cancer Prevention and Research Institute of Texas (CPRIT). Its aim is to provide cellular therapy products and viral vectors (retrovirus, adenovirus and plasmids) to treat pediatric cancer patients at Texas Academic Institutions. Manufacturing of these products must be performed under FDA regulations and Good Manufacturing Practices (GMP). The GMP facility at Baylor College of Medicine Center for Cell and Gene Therapy (CAGT) is one of the largest and most modern academic centers in the country. The staff is highly experienced in manufacturing a very wide variety of cellular therapy products and viral vectors used for genetic modification of cells. CARMIT will cover consultations regarding Chemistry, Manufacturing and Controls section of the Investigational New Drug applications with the FDA and some of the cost of making (mainly labor) of your product for your clinical trial. You will have to obtain funding for supplies and testing from other resources.
To be eligible to apply for CARMIT support you must be a physician scientist at a Texas Academic Center. Initial applications are short and are reviewed by a CAGT Internal Review Panel for scientific content and manufacturing feasibility. Successful applicants are then requested to submit a slightly longer application form describing the product to be made and the pediatric clinical protocol in which it is to be used, IND status etc. This is reviewed by an External Advisory Board composed of cellular therapy experts at renowned institutions. To apply, please complete the initial application located below, and submit it to Dr. Natasha Lapteva at nxlaptev@txch.org.
Successful applicants will work with a CARMIT representative to develop a manufacturing timeline and budget.
This work is funded by CPRIT Core Facilities Support Award RP240545 (renewal RP180785).
Applications
Primary Location
- Feigin Center at Texas Children's Hospital
- 16th Floor Offices and GMP facilities
- 17th Floor Translational Research
- 17th Floor Protocol & Regulatory Affairs
Services
Cellular therapy products manufactured include hematopoietic progenitor cells, donor leukocyte infusions, chimeric antigen receptor (CAR) T cells, virus and tumor specific T cells, NK and NKT cells, dendritic cells, and mesenchymal stromal cells. The staff will work with you to prepare the cell type used in your protocol.
Analytical services include all of the testing required to release the cell product or vector for clinical use. Testing is performed internally or contracted out to commercial testing companies. Multicolor and multiparameter flow cytometric analysis is performed on site. Manufacturing records are reviewed by our independent Quality Assurance group, who will generate Certificate of Analysis to facilitate clinical use of the products. All services are in compliance with GMP regulations as they apply to products used in Phase I/early Phase II clinical trials.
Products Manufactured
- Stem Cell Products for Transplant
- Virus-directed T cells
- Virus/Tumor-directed T cells
- NK cells (auto/allo/cell lines)
- NKT Cells
- Regenerative Medicine products (BM MNC & MSC )
- Viral vectors (Adenoviral and Retroviral), Master & Working Cell Banks
- Dendritic Cells
Frequently Asked Questions
Applicants should be physician scientists at Texas Academic Institutions who require cell therapy products or viral vectors to initiate an IND (Investigational New Drug) application to the FDA to perform a Phase I / early Phase II clinical trial on pediatric patients.
The level of support from CARMIT will be determined on an individual application basis and will depend on the number of applications received. The anticipated funding level will be discussed with successful applicants.
This will depend on the type of product requested. Once an application is approved a CARMIT representative will work with the applicant to develop a timeline.
CARMIT staff will assist you with development of the Chemistry Manufacturing and Control section of your IND application.
Core Staff
Publications
Zhou X, Dotti G, Krance RA, Martinez CA, Naik S, Kamble RT, Durett AG, Dakhova O, Savoldo B, Di Stasi A, Spencer DM, Lin YF, Liu H, Grilley BJ, Gee AP, Rooney CM, Heslop HE, Brenner MK. Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation. Blood. 2015;125(26):4103-13. Epub 2015/05/14; PMCID: PMC4481597.
Naik S, Martinez C, Leung K, Sasa G, Nguyen NY, Wu MF, Gottschalk S, Brenner M, Heslop H, Krance R. Outcomes after Second Hematopoietic Stem Cell Transplantations in Pediatric Patients with Relapsed Hematological Malignancies. Biol Blood Marrow Transplant. 2015;21(7):1266-72. Epub 2015/03/10; PMID: 25765555.
Lindblad RW, Ibenana L, Wagner JE, McKenna DH Jr, Hei DJ, Hematti P, Couture LA, Silberstein LE, Armant M, Rooney CM, Gee AP, Welniak LA, Heath Mondoro T, Wood DA, Styers D. Cell therapy product administration and safety: data capture and analysis from the Production Assistance for Cellular Therapies (PACT) program. Transfusion. 2015;55(3):674-9. Epub 2014/10/14; PMCID:PMC4357544.
Sun J, Huye LE, Lapteva N, Mamonkin M, Hiregange M, Ballard B, Dakhova O, Raghavan D, Durett AG, Perna SK, Omer B, Rollins LA, Leen AM, Vera JF, Dotti G, Gee AP, Brenner MK, Myers DG, Rooney CM. Early transduction produces highly functional chimeric antigen receptor-modified virus specific T-cells with central memory markers: a Production Assistant for Cell Therapy (PACT) translational application. J Immunotherapy Canc. 2015;3:5. Epub 2015/02/18; PMCID: 4346112.
Ahmed N, Brawley VS, Hegde M, Robertson C, Ghazi A, Gerken C, Liu E, Dakhova O, Ashoori A, Corder A, Gray T, Wu MF, Liu H, Hicks J, Rainusso N, Dotti G, Mei Z, Grilley B, Gee A, Rooney CM, Brenner MK, Heslop HE, Wels WS, Wang LL, Anderson P, Gottschalk S. Human Epidermal Growth Factor Receptor 2 (HER2) -Specific Chimeric Antigen Receptor-Modified T Cells for the Immunotherapy of HER2-Positive Sarcoma. J Clin Oncol. 2015;33(15):1688-96. Epub 2015/03/23; PMCID: PMC4429176.
Hanley PJ, Melenhorst JJ, Nikiforow S, Scheinberg P, Blaney JW, Demmler-Harrison G, Cruz CR, Lam S, Krance RA, Leung KS, Martinez CA, Liu H, Douek DC, Heslop HE, Rooney CM, Shpall EJ, Barrett AJ, Rodgers JR, Bollard CM. CMV-specific T cells generated from naive T cells recognize atypical epitopes and may be protective in vivo. Sci Transl Med. 2015;7(285):285ra63; PMCID: PMC4479400.
Szmania S, Lapteva N, Garg T, Greenway A, Lingo J, Nair B, Stone K, Woods E, Khan J, Stivers J, Panozzo S, Campana D, Bellamy WT, Robbins M, Epstein J, Yaccoby S, Waheed S, Gee A, Cottler-Fox M, Rooney C, Barlogie B, van Rhee F. Ex vivo-expanded natural killer cells demonstrate robust proliferation in vivo in high-risk relapsed multiple myeloma patients. J Immunother. 2015;38(1):24-36; PMCID: PMC4352951.
Ramos CA, Savoldo B, Torrano V, Ballard B, Zhang H, Dakhova O, Liu E, Carrum G, Kamble RT, Gee AP, Mei Z, Wu MF, Liu H, Grilley B, Rooney CM, Brenner MK, Heslop HE, Dotti G. Clinical responses with T lymphocytes targeting malignancy-associated kappa light chains. J Clin Invest. 2016;126(7):2588-96. Epub 2016/06/06; PMCID: PMC4922690.
Ahmed N, Brawley V, Hegde M, Bielamowicz K, Kalra M, Landi D, Robertson C, Gray TL, Diouf O, Wakefield A, Ghazi A, Gerken C, Yi Z, Ashoori A, Wu MF, Liu H, Rooney C, Dotti G, Gee A, Su J, Kew Y, Baskin D, Zhang YJ, New P, Grilley B, Stojakovic M, Hicks J, Powell SZ, Brenner MK, Heslop HE, Grossman R, Wels WS, Gottschalk S. HER2-Specific Chimeric Antigen Receptor-Modified Virus-Specific T Cells for Progressive Glioblastoma: A Phase 1 Dose-Escalation Trial. JAMA Oncol. 2017;3(8):1094-101; PMCID: PMC5747970.
Tanaka M, Tashiro H, Omer B, Lapteva N, Ando J, Ngo M, Mehta B, Dotti G, Kinchington PR, Leen AM, Rossig C, Rooney CM. Vaccination Targeting Native Receptors to Enhance the Function and Proliferation of Chimeric Antigen Receptor (CAR)-Modified T Cells. Clin Cancer Res.2017;23(14):3499-509; PMCID: PMC5511585. Szoor A, Vaidya A, Velasquez MP, Mei Z, Galvan DL, Torres D, Gee A, Heczey A, Gottschalk S. T Cell-Activating Mesenchymal Stem Cells as a Biotherapeutic for HCC. Mol Ther Oncolytics. 2017;6:69-79. Epub 2017/07/28; PMCID: PMC5562179.
Ramos CA, Ballard B, Zhang H, Dakhova O, Gee AP, Mei Z, Bilgi M, Wu MF, Liu H, Grilley B, Bollard CM, Chang BH, Rooney CM, Brenner MK, Heslop HE, Dotti G, Savoldo B. Clinical and immunological responses after CD30-specific chimeric antigen receptor-redirected lymphocytes. J Clin Invest. 2017;127(9):3462-71. Epub 2017/08/14; PMCID: PMC5669573.