Advanced therapy medicinal products and exemptions to the Regulation 1394/2007: How confident can we be? An exploratory analysis

Frontiers in Pharmacology

Volumn 3 FEB, Issue , 2012, Pages

  Van Wilder, Philippe ^a  

^a TiGenix   (Belgium)

Author keywords

Advanced therapy; Exemption; Patient outcome; Regulation 1394 2007

Indexed keywords

ALIPOGENE TIPARVOVEC; CHONDROCYTE IMPLANT; CONTUSUGENE LADENOVEC; SITIMAGENE CERADENOVEC;

ADVANCED THERAPY MEDICINAL PRODUCT; ARTICLE; AUTOLOGOUS CHONDROCYTES IMPLANTATION; CHONDROCYTE IMPLANTATION; CHONDROPATHY; DISORDERS OF LIPID METABOLISM; DRUG EFFICACY; DRUG LEGISLATION; DRUG MARKETING; DRUG QUALITY; DRUG SAFETY; GENE PRODUCT; GENE THERAPY; GLIOMA; HEAD AND NECK SQUAMOUS CELL CARCINOMA; HUMAN; MICROFRACTURE; OSTEOARTHRITIS; PATIENT CARE; SOMATIC CELL; SOMATIC CELL THERAPY; SYSTEMATIC REVIEW; TISSUE ENGINEERING;

EID: 84865971643     PISSN: None     EISSN: 16639812     Source Type: Journal    
DOI: 10.3389/fphar.2012.00012     Document Type: Article

References (50)

1. Alberti, C. (2009). Tissue engineering technologies: just a quick note about transplantation of bioengineered donor trachea and augmentation cystoplasty by de novo engineered bladder tissue. G. Chir. 30, 514-519.
2. Assche, D. V., Caspel, D. V., Staes, F., Saris, D. B., Bellemans, J., Vanlauwe, J., and Luyten, F. P. (2011). Implementing one standardized rehabilitation protocol following autologous chondrocyte implantation or microfracture in the knee results in comparable physical therapy management. Physiother. Theory Pract. 27, 125-136.
3. Basad, E., Ishaque, B., Bachmann, G., Stürz, H., and Steinmeyer, J. (2010). Matrix-induced autologous chondrocyte implantation versus microfracture in the treatment of cartilage defects of the knee: a 2-year randomised study. Knee Surg. Sports Traumatol. Arthrosc. 18, 519-527.
4. Bekkers, J. E., Inklaar, M.,and Saris, D. B. (2009). Treatment selection in articular cartilage lesions of the knee: a systematic review. Am.J.SportsMed. 37(Suppl. 1), 148S-55S.
5. Brun, P., Dickinson, S. C., Zavan, B., Cortivo, R., Hollander, A. P., and Abatangelo, G. (2008). Characteristics of repair tissue in secondlook and third-look biopsies from patients treated with engineered cartilage: relationship to symptomatology and time after implantation. Arthritis Res. Ther. 10, R132.
6. Candrian, C., Bonacina, E., Frueh, J. A., Vonwil, D., Dickinson, S., Wirz, D., Heberer, M., Jakob, M., Martin, I., and Barbero, A. (2009). Intra-individualcomparison of human ankle and knee chondrocytes in vitro: relevance for talar cartilage repair. Osteoarthr. Cartil. 17, 489-496.
7. Choi, Y. S., Potter, H. G., and Chun, T. J. (2008). MR imaging of cartilage repair in the knee and ankle. Radiographics 28, 1043-1059.
8. Chubinskaya, S., Hakimiyan, A. A., Rappoport, L., Yanke, A., Rueger, D. C., and Cole, B. J. (2008). Response of human chondrocytes prepared for autologous implantation to growth factors. J. Knee. Surg. 21, 192-199.
9. Domayer, S. E., Trattnig, S., Stelzeneder, D., Hirschfeld, C., Quirbach, S., Dorotka, R., Nehrer, S., Pinker, K., Chan, J., Mamisch, T. C., Dominkus, M., and Welsch, G. H. (2010). Delayed gadolinium-enhanced MRI of cartilage in the ankle at 3 T: feasibility and preliminary results after matrix-associated autologous chondrocyte implantation. J. Magn. Reson. Imaging 31, 732-739.
10. Drobnic, M., Radosavljevic, D., Cör, A., Brittberg, M., and Strazar, K. (2010). Debridement of cartilage lesions before autologous chondrocyte implantation by open or transarthroscopic techniques: a comparative study using postmortem materials. J. Bone Joint Surg. Br. 92, 602-608.
11. Ebert, J. R., Robertson, W. B., Lloyd, D. G., Zheng, M. H., Wood, D. J., and Ackland, T. (2008). Traditional vs accelerated approaches to post-operative rehabilitation following matrix-induced autologous chondrocyte implantation (MACI): comparison of clinical, biomechanical and radiographic outcomes. Osteoarthr. Cartil. 16, 1131-1140.
12. Erggelet, C., Kreuz, P. C., Mrosek, E. H., Schagemann, J. C., Lahm, A., Ducommun, P. P., and Ossendorf, C. (2010). Autologous chondrocyte implantation versus ACI using 3D-bioresorbable graft for the treatment of large full-thickness cartilage lesions of the knee. Arch. Orthop. Trauma Surg. 130, 957-964.
13. Ferruzzi, A., Buda, R., Faldini, C., Vannini, F., Di Caprio, F., Luciani, D., and Giannini, S. (2008). Autologous chondrocyte implantation in the knee joint: open compared with arthroscopic technique. Comparison at a minimum follow-up of five years. J. Bone Joint Surg. Am. 90(Suppl. 4), 90-101.
14. Genovese, E., Ronga, M., Angeretti, M. G., Novario, R., Leonardi, A., Albrizio, M., Callegari, L., and Fugazzola, C. (2011). Matrixinduced autologous chondrocyte implantation of the knee: mid-term and long-term follow-up by MR arthrography. Skeletal Radiol. 40, 47-56.
15. Giannini, S., Buda, R., Vannini, F., Cavallo, M., and Grigolo, B. (2009). One-step bone marrow-derived cell transplantation in talar osteochondral lesions. Clin. Orthop. Relat. Res. 467, 3307-3320.
16. Gomoll, A. H., Probst, C., Farr, J., Cole, B. J., and Minas, T. (2009). Use of a type I/III bilayer collagen membrane decreases reoperation rates for symptomatic hypertrophy after autologous chondrocyte implantation. Am.J.SportsMed.37(Suppl. 1), 20S-23S.
17. Gonda, K., Shigeura, T., Sato, T., Matsumoto, D., Suga, H., Inoue, K., Aoi, N., Kato, H., Sato, K., Murase, S., Koshima, I., and Yoshimura, K. (2008). Preserved proliferative capacity and multipotency of human adipose-derived stem cells after long-term cryopreservation. Plast. Reconstr. Surg. 121, 401-410.
18. Harris, J. D., Siston, R. A., Pan, X., and Flanigan, D. C. (2010). Autologous chondrocyte implantation:a systematic review. J. Bone Joint Surg.Am. 92, 2220-2233.
19. Hwang, N. S., and Elisseeff, J. (2009). Application of stem cells for articular cartilage regeneration. J. Knee Surg. 22, 60-71.
20. Kon, E., Delcogliano, M., Filardo, G., Montaperto, C., and Marcacci, M. (2008). Second generation issues in cartilage repair. Sports Med. Arthrosc. 16, 221-229.
21. Kon, E., Gobbi, A., Filardo, G., Delcogliano, A., Zaffagnini, S., and Marcacci, M. (2009). Arthroscopic second-generation autologous chondrocyte implantation compared with microfracture for chondral lesions of the knee: prospective nonrandomized study at 5 years. Am.J.SportsMed.37, 33-41.
22. Kreuz, P. C., Müller, S., Ossendorf, C., Kaps, C., and Erggelet, C. (2009). Treatment of focal degenerative cartilage defects with polymer-based autologous chondrocyte grafts:fouryear clinical results. Arthritis Res. Ther. 11, R33.
23. Lee, K. T., Choi, Y. S., Lee, Y. K., Kim, J. S., Young, K. W., and Kim, J. H. (2010). Comparison of MRI and arthroscopy after autologous chondrocyte implantation in patients with osteochondral lesion of the talus. Orthopedics 33. doi: 10.3928/01477447-20100625-12
24. Malicev, E., Kregar-Velikonja, N., Barlic, A., Alibegovic, A., and Drobnic, M. (2009). Comparison of articular and auricular cartilage as a cell source for the autologous chondrocyte implantation. J. Orthop. Res. 27, 943-948.
25. Mithoefer, K., Hambly, K., Della Villa, S., Silvers, H., and Mandelbaum, B. R. (2009). Return to sports participation after articular cartilage repair in the knee: scientific evidence. Am. J. Sports Med. 37(Suppl. 1), 167S- 176S.
26. Mobasheri, A., Csaki, C., Clutterbuck, A. L., Rahmanzadeh, M.,and Shakibaei, M. (2009). Mesenchymal stem cells in connective tissue engineering and regenerative medicine: applications in cartilage repair and osteoarthritis therapy. Histol. Histopathol. 24, 347-366.
27. Moseley, J. B. Jr., Anderson, A. F., Browne, J. E., Mandelbaum, B. R., Micheli, L. J., Fu, F., and Erggelet, C. (2010). Long-term durability of autologous chondrocyte implantation: a multicenter, observational study in US patients. Am. J. Sports Med. 38, 238-246.
28. Munirah, S., Ruszymah, B. H., Samsudin, O. C., Badrul, A. H., Azmi, B., and Aminuddin, B. S. (2008). Autologous versus pooled human serum for articular chondrocyte growth. J. Orthop. Surg. (Hong Kong) 16, 220-229.
29. Niemeyer, P., Pestka, J. M., Erggelet, C., Steinwachs, M., Salzmann, G. M., and Südkamp, N. P. (2011). Comparison of arthroscopic and open assessment of size and grade of cartilage defects of the knee. Arthroscopy 27, 46-51.
30. Pinker, K., Szomolanyi, P., Welsch, G. C., Mamisch, T. C., Marlovits, S., Stadlbauer, A., and Trattnig, S. (2008). Longitudinal evaluation of cartilage composition of matrix-associated autologous chondrocyte transplants with 3-T delayed gadolinium-enhanced MRI of cartilage. AJR Am. J. Roentgenol. 191, 1391-1396.
31. Potter, H. G., Chong le, R.,and Sneag, D. B. (2008). Magnetic resonance imaging of cartilage repair. Sports Med. Arthrosc. 16, 236-45.
32. Quirbach, S., Trattnig, S., Marlovits, S., Zimmermann, V., Domayer, S., Dorotka, R., Mamisch, T. C., Bohndorf, K., and Welsch, G. H. (2009). Initial results of in vivo high-resolution morphological and biochemical cartilage imaging of patients after matrix-associated autologous chondrocyte transplantation (MACT) of the ankle. Skeletal Radiol. 38, 751-760.
33. Rosenberger, R. E., Gomoll, A. H., Bryant, T., and Minas, T. (2008). Repair of large chondral defects of the knee with autologous chondrocyte implantation in patients 45 years or older. Am.J.SportsMed.36, 2336-2344.
34. Rue, J. P., Yanke, A. B., Busam, M. L., McNickle, A. G., and Cole, B. J. (2008). Prospective evaluation of concurrent meniscus transplantation and articular cartilage repair: minimum 2-year follow-up. Am. J. Sports Med. 36, 1770-1778.
35. Saris, D. B., Vanlauwe, J., Victor, J., Almqvist, K. F., Verdonk, R., Bellemans, J., Luyten, F. P., and TIG/ACT/01/2000&EXT Study Group. (2009). Treatment of symptomatic cartilage defects of the knee: characterized chondrocyteimplantation results in better clinical outcome at 36 months in a randomized trial compared to microfracture. Am.J.SportsMed. 37(Suppl. 1), 10S-19S.
36. Saris, D. B., Vanlauwe, J., Victor, J., Haspl, M., Bohnsack, M., Fortems, Y., Vandekerckhove, B., Almqvist, K. F., Claes, T., Handelberg, F., Lagae, K., van der Bauwhede, J., Vandenneucker, H., Yang, K. G., Jelic, M., Verdonk, R., Veulemans, N., Bellemans, J., and Luyten, F. P. (2008). Characterized chondrocyte implantation results in better structural repair when treating symptomatic cartilage defects of the knee in a randomized controlled trial versus microfracture. Am.J.SportsMed.36, 235-246.
37. Schmal, H., Mehlhorn, A., Stoffel, F., Köstler, W., Südkamp, N. P., and Niemeyer, P. (2009). In vivo quantification of intraarticular cytokines in knees during natural and surgically induced cartilage repair. Cytotherapy 11, 1065-1075.
38. Schmal, H., Niemeyer, P., Zwingmann, J., Stoffel, F., Südkamp, N. P., and Mehlhorn, A. T. (2010). Association between expression of the bone morphogenetic proteins 2 and 7 in the repair of circumscribed cartilage lesions with clinical outcome. BMC Musculoskelet. Disord. 11, 170. doi:10.1186/1471-2474-11170
39. Selmi, T. A., Verdonk, P., Chambat, P., Dubrana, F., Potel, J. F., Barnouin, L., and Neyret, P. (2008). Autologous chondrocyte implantation in a novel alginate-agarose hydrogel: outcome at two years. J. Bone. Joint. Surg. Br. 90, 597-604.
40. Steinhagen, J., Bruns, J., Deuretzbacher, G., Ruether, W., Fuerst, M., and Niggemeyer, O. (2010). Treatment of osteochondritis dissecans of the femoral condyle with autologous bone grafts and matrix-supported autologous chondrocytes. Int. Orthop. 34, 819-825.
41. Tohyama, H., Yasuda, K., Minami, A., Majima, T., Iwasaki, N., Muneta, T., Sekiya, I., Yagishita, K., Takahashi, S., Kurokouchi, K., Uchio, Y., Iwasa, J., Deie, M., Adachi, N., Sugawara, K., and Ochi, M. (2009). Atelocollagen-associated autologous chondrocyte implantation for the repair of chondral defects of the knee: a prospective multicenter clinical trial in Japan. J. Orthop. Sci. 14, 579-588.
42. Trattnig, S., Burstein, D., Szomolanyi, P., Pinker, K., Welsch, G. H., and Mamisch, T. C. (2009). T1(Gd) gives comparable information as Delta T1 relaxation rate in dGEMRIC evaluation of cartilage repair tissue. Invest Radiol. 44, 598-602.
43. Van Assche, D., Staes, F., Van Caspel, D., Vanlauwe, J., Bellemans, J., Saris, D.B., and Luyten, F. P. (2010). Autologous chondrocyte implantation versus microfracture for knee cartilage injury: a prospective randomized trial, with 2-year follow-up. Knee Surg. Sports Traumatol. Arthrosc. 18, 486-495.
44. van Bergen, C. J., de Leeuw, P. A., and van Dijk, C. N. (2008). Treatment of osteochondral defects, of the talus. Rev. Chir. Orthop. Reparatrice Appar. Mot. 94(Suppl. 8), 398-408.
45. Welsch, G. H., Mamisch, T. C., Domayer, S. E., Dorotka, R., Kutscha-Lissberg, F., Marlovits, S., White, L. M., and Trattnig, S. (2008). Cartilage T2 assessment at 3-T MR imaging: in vivo differentiation of normal hyaline cartilage from reparative tissue after two cartilage repair procedures - initial experience. Radiology 247, 154-161.
46. Welsch, G. H., Zak, L., Mamisch, T. C., Resinger, C., Marlovits, S., and Trattnig, S. (2009a). Three-dimensional magnetic resonance observation of cartilage repair tissue (MOCART) score assessed with an isotropic three-dimensional true fast imaging with steady-state precession sequence at 3.0 Tesla. Invest Radiol. 44, 603-612.
47. Welsch, G. H., Mamisch, T. C., Marlovits, S., Glaser, C., Friedrich, K., Hennig, F. F., Salomonowitz, E., and Trattnig, S. (2009b). Quantitative T2 mapping during follow-up after matrixassociated autologous chondrocyte transplantation (MACT): fullthickness and zonal evaluation to visualize the maturation of cartilage repair tissue. J. Orthop. Res. 27, 957-963.
48. Wondrasch, B., Zak, L., Welsch, G. H., and Marlovits, S. (2009). Effect of accelerated weightbearing after matrix-associated autologous chondrocyte implantation on the femoral condyle on radiographic and clinical outcome after 2 years: a prospective, randomized controlled pilot study. Am.J.SportsMed. 37(Suppl. 1), 88S-96S.
49. Zaslav, K., Cole, B., Brewster, R., DeBerardino, T., Farr, J., Fowler, P., Nissen, C., and STAR Study Principal Investigators. (2009). A prospective study of autologous chondrocyte implantation in patients with failed prior treatment for articular cartilage defect of the knee: results of the Study of the Treatment of Articular Repair (STAR) clinical trial. Am. J. Sports Med. 37, 42-55.
50. Zeifang, F., Oberle, D., Nierhoff, C., Richter, W., Moradi, B., and Schmitt, H. (2010). Autologous chondrocyte implantation using the original periosteum-cover technique versus matrix-associated autologous chondrocyte implantation: a randomized clinical trial. Am.J.SportsMed.38, 924-933.