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Crime against Humanity
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Abstract:
The world needs to know the facts as to how the FDA “protects” the public health by assuring the safety, effectiveness, and security of human drugs and biological products for human use. Recent uproar has surfaced over the FDA’s approval process on the unproven Duchenne Muscular Dystrophy (DMD) drug, Exondys 51 (Sarepta Therapeutics, Cambridge, MA, USA), but what many people do not know are the unlawful events that occurred nearly two decades ago on a proven and promising treatment for DMD.
| [1] | Law, P.K., Bertorini, T., Goodwin, T.G., et al. (1990) Dystrophin Production Induced by Myoblast Transfer Therapy in Duchenne Muscular Dystrophy. The Lancet, 336, 114-115. https://doi.org/10.1016/0140-6736(90)91628-N |
| [2] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1997) First Human Myoblast Transfer Therapy Continues to Show Dystrophin after 6 Years. Cell Transplant, 6, 95-100. |
| [3] | Law, P.K. (1995) Methods for Human Myoblast Culture and Transplantation. In: Ricordi, C., Ed., Methods in Cell Transplantation, R.G. Landes, Austin, TX, 707-735. |
| [4] | Law, P.K., Li, H., Chen, M., Fang, Q. and Goodwin, T. (1994) Myoblast Injection Method Regulates Cell Distribution and Fusion. Transplantation Proceedings, 26, 3417-3418. |
| [5] | Law, P.K. (1992) Myoblast Transplantation. Science, 257, 1329. https://doi.org/10.1126/science.1529326 |
| [6] | Law, P.K. (1993) Myoblast Transfer Therapy. Lancet, 341, 247. https://doi.org/10.1016/0140-6736(93)90115-W |
| [7] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1992) Feasibility, Safety, and Efficacy of Myoblast Transfer Therapy on Duchenne Muscular Dystrophy Boys. Cell Transplant, 1, 235-244. https://doi.org/10.1177/0963689792001002-305 |
| [8] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1991) Pioneering Development of Myoblast Transfer Therapy. In: Angelini, C., Darrieli, G.A. and Fontanan, D., Eds., Muscular Dystrophy Research, Elsevier Science Inc., New York, 109-116. |
| [9] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1991) Long-Term Improvement in Muscle Function, Structure and Biochemistry Following Myoblast Transfer in DMD. Acta Cardiomiologica, 1, 281-301. |
| [10] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1993) Cell Transplantation as an Experimental Treatment for Duchenne Muscular Dystrophy. Cell Transplant, 2, 485-505. https://doi.org/10.1177/096368979300200607 |
| [11] | Law, P.K., Goodwin, T.G., Fang, Q., et al. (1998) Myoblast Transfer as a Platform Technology of Gene Therapy. Gene Therapy and Molecular Biology, 1, 345-363. |
| [12] | US Food & Drug Association (2016) FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm521263.htm |
| [13] | Perrone, M. (2016) FDA Oks 1st Muscular Dystrophy Drug; Awaits Proof It Works. Associated Press. https://apnews.com/9c4e469479ea438eb42957ba6ecd4710/fda-tentatively-approves-first-drug-muscular-dystrophy |
| [14] | Thomas, K. (2017) Insurers Battle Families over Costly Drug for Fatal Disease. The New York Times. https://www.nytimes.com/2017/06/22/health/duchenne-muscular-dystrophy-drug-exondys-51.html |
| [15] | Edlin, M. (2017) Controversy Surrounds Exondys 51 Approval: What to Know. Managed Healthcare Executive. http://managedhealthcareexecutive.modernmedicine.com/managed-healthcare-executive/news/controversy-surrounds-exondys-51-approval-what-know?page=0,1 |
| [16] | Swaminathan, N. (2016) Precision Medicine May Be Health Care’s Next Big Thing, But We Don’t Yet Know How to Evaluate Precision Drugs. Quartz. https://qz.com/824150/precision-medicine-may-be-health-cares-next-big-thing-but-we-dont-yet-know-how-to-evaluate-precision-drugs/ |
| [17] | Silverman, E. (2016) Behind the Sarepta Drug Approval Was Intense FDA Bickering. STAT. https://www.statnews.com/pharmalot/2016/09/19/sarepta-fda-duchenne-behind-the-decision/ |
| [18] | Seife, C. (2016) FDA Documents Reveal Depths of Internal Rancor over Drug’s Approval Process. Undark. https://undark.org/article/fda-eteplirsen-janet-woodcock/ |
| [19] | Adams, B. (2016) FDA New Drugs Director Slams Sarepta Approval as “NOT a Good Model”. FierceBi-otech. http://www.fiercebiotech.com/biotech/fda-news-drugs-director-slams-sarepta-says-biotech-s-approval-not-a-good-model |
| [20] | Silverman, E. (2016) FDA Official Warns Other Drug Makers Not to Copy Sarepta. STAT. https://www.statnews.com/pharmalot/2016/10/20/fda-warns-drug-companies-about-sarepta/ |
| [21] | Carroll, J. (2017) Top FDA Official Accused CDER Chief Woodcock of Appearing Biased, Browbeating Reviewers in Demanding Eteplirsen OK. Endpoint News. https://endpts.com/top-fda-official-accused-cder-chief-woodcock-of-appearing-biased-alienating-reviewers-in-demanding-eteplirsen-ok/ |
| [22] | Henriques, C. (2017) FDA’s Approval of Duchenne Therapy Exondys 51 Sparks Lawsuit by NYU Professor. Muscular Dystrophy News Today. https://musculardystrophynews.com/2017/05/30/nyu-professor-files-lawsuit-against-fda-for-records-on-duchenne-md-exondys-51-approval/ |
| [23] | Mosher, D. (2016) A Top Journalist Is Suing the FDA over Its Alleged Use of a Banned and Secretive Practice to Manipulate the News. Business Insider. http://www.businessinsider.com/fda-journalist-manipulation-embargoes-2016-9 |
| [24] | Stennes, M.L. (1997) The Criminalization of Innovation: FDA Misdirection in the Najarian and Burzynski Cases (1997 Third Year Paper) Harvard Law School. http://nrs.harvard.edu/urn-3:HUL.InstRepos:9453691 |
| [25] | Law, P., Weinstein, J., Ben Hain, S., et al. (2000) World’s First Human Myoblast Transfer into the Heart. Acta Physiologica Scandinavica, A1-A114. |
| [26] | Law, P.K., Haider, K., Fang, G., et al. (2002) Mechanisms of Myoblast Transfer in Treating Heart Failure. In: Kimchi, A., Ed., Advances in Heart Failure, Medimont, New York, 43-48. |
| [27] | Law, P.K., et al. (2003) Myoblast Genome Therapy and the Regenerative Heart. In: Kipshidze, N. and Serruys, P., Eds., Handbook of Cardiovascular Cell Transplantation, Martin Dunitz, Ltd., London, 241-257. |
| [28] | Haider, K.H., Ye, L., Jiang, S.J., Law, P.K. and Sim, E.K.W. (2003) My-oblast Transplantation for Cardiac Repair using Transient Immunosuppression. Basic and Applied Myology, 13, 45-52. |
| [29] | Law, P.K., Fang, G., Chua, F., Kakuchaya, T. and Bockeria, L.A. (2003) First-in-Man Myoblast Allografts for Heart Degeneration. The International Journal of Medical Implants Devices, 1, 100-155. |
| [30] | Law, P.K., et al. (2008) Delivery of Biologics for Angiogenesis and Myogenesis. In: Nguyen, T., Colombo, A., Hu, D., et al., Eds., Practical Handbook of Advanced Interventional Cardiology, 3rd Edition, Blackwell Futura, Malden, 584-596. |
| [31] | Ma, J.H., Su, L.P., Zhu, J., et al. (2013) Skeletal Myoblast Transplantation on Gene Expression Profiles of Insulin Signaling Pathway and Mitochondrial Biogenesis and Function in Skeletal Muscle. Diabetes Research and Clinical Practice, 102, 43-52. |
| [32] | Law, P.K. (2016) Disease Prevention and Alleviation by Human Myoblast Transplantation. Open Journal of Regenerative Medicine, 5, 25-43. https://doi.org/10.4236/ojrm.2016.52003 |
| [33] | Law, P.K., Song, S.J., Lu, P., et al. (2017) World’s First Myoblast Treatment of Human Cancer Found Safe and Efficacious. Open Journal of Regenerative Medicine, 6, 1-16. https://doi.org/10.4236/ojrm.2017.61001 |
