Chapter Twenty-Eight: Gene Therapy and CRISPR
The first gene is found Woody Guthrie and Huntington’s Chorea
https://www.britannica.com/biography/Woody-Guthrie
http://news.mit.edu/1993/huntington-0331
https://www.genome.gov/25520322/online-education-kit-1983-first-disease-gene-mapped
Orphan Drug Report 2019
http://www.paradigmglobalevents.com/events/orphan-drugs-rare-diseases-2017-americas/
Gene therapy Aug 2019
https://www.nejm.org/doi/full/10.1056/NEJMra1706910
Genetic blindness: Leber’s Congenital Amaurosis
https://www.nejm.org/doi/full/10.1056/NEJMoa1414221
http://www.cnn.com/2017/10/12/health/fda-gene-therapy-blindness-vote/index.html
Cystic FibrosisThree drug treatment for cystic fibrosis nejm https://www.nejm.org/doi/full/10.1056/NEJMoa1908639
The Antidote by Barry Werth, Simon and Schuster, 2014 (see page 69–Victoria Sato, president of Vertex},
http://www.nejm.org/doi/full/10.1056/NEJMe1712335
https://www.nejm.org/doi/full/10.1056/NEJMoa1908639https://cysticfibrosisnewstoday.com/2020/08/24/vertex-triple-combo-kaftrio-approved-for-cf-patients-in-eu-with-f508del-mutation/
Europe approves triple therap. August 2020 According to Vertex, as many as 10,000 people in Europe ages 12 years and older have CF characterized by one F508del mutation and one minimal function mutation, or a mutation in which the resulting CFTR protein works minimally. The newly approved triple combination is the first therapy that works on the underlying cause of CF to be approved for such individuals.ttps://cysticfibrosisnewstoday.com/2020/08/24/vertex-triple-combo-kaftrio-approved-for-cf-patients-in-eu-with-f508del-mutation/
Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
SMA spinal muscular atrophy and POMPE’S
https://smanewstoday.com/2015/02/23/exclusive-new-insight-into-sma-from-dr-adrian-krainer/
https://www.nejm.org/doi/full/10.1056/NEJMoa1706198
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2692727/
https://www.jci.org/articles/view/1722
https://doi.org/10.1002/14651858.CD011539.pub2
Hemophilia
Nicholas and Alexandra by Raymond Massie. Random House; 1967
hemophila B N Engl J Med 2015
https://www.nejm.org/doi/full/10.1056/NEJMoa1708538
Gene transfer in severe hemophilia A https://www.nejm.org/doi/full/10.1056/NEJMoa1708483
As of 2016, BioMarin has six products on the market, each of which is an orphan drug. https://en.wikipedia.org/wiki.BioMarin_Pharmaceutical
Buy a gene cheap: Addgene
Amyloidosis
https://www.sfgate.com/health/article/The-Domino-Effect-Woman-gets-new-liver-gives-3238583.php
CRISPR
A Crack in Creation by Jennifer Doudna
Emmanuelle Charpentier co-discoverer of CRISPR
Charpentier rna https://embryo.asu.edu/pages/jennifer-doudna-and-emmanuelle-charpentiers-experiment-about-crisprcas-9-systems-role-adaptive
https://www.nytimes.com/2016/05/31/health/emmanuelle-charpentiers-crispr-dna-gene-editing.html https://www.nature.com/news/the-quiet-revolutionary-how-the-co-discovery-of-crispr-explosively-changed-emmanuelle-charpentier-s-life-1.19814
Sometimes, I then go to sleep again for an hour. I don’t have time to have a social life or even a cultural life.e.
In the early 1980s, Mario Capecchi discovered that sections of DNA that had been inserted into the cell nuclei of mammals could be incorporated into the cell’s genome.
https://www.nobelprize.org/prizes/medicine/2007/capecchi/facts/
Chapter Twenty-Nine: Gaming the System
Martin Shkreli and Daraprim
Gertrude Elion
daraprim https://theoncologist.onlinelibrary.wiley.com/doi/full/10.1634/theoncologist.11-9-961
Acthar ny times https://www.nytimes.com/2012/12/30/business/questcor-finds-profit-for-acthar-drug-at-28000-a-vial.html
https://www.britannica.com/biography/Gertrude-B-Elion https://www.nap.edu/read/9977/chapter/3#18
The Curious Case of Colchicine NEJM June 2010 https://www.nejm.org/doi/full/10.1056/NEJMp1003126
Acthar
60 minutes May 12, 2019
Chapter Thirty: Are Generic Drugs Safe and Effective?
Katherine Eban: NY Times May 12, 2019.
Bottle of Lies by Katherine Eban: Harper Collins 2019
https://www.fda.gov/news-events/congressional-testimony/securing-us-drug-supply-chain-
International regulations
https://www.panmobil.com/single-post/In-2019-pharmaceuticals-are-only-available-with-2D-Barcodes
Oversight of the FDA foreign inspection program Dec 10, 2019
Chapter Thirty-One: The FDA—The Fox That’s Guarding the Henhouse
Fastrack approval
Forbidden Cures. By Steven Fredman MD; Robert E Burger Stein and Day, 1976
The Rosiglitazone Story — Lessons from an FDA Advisory Committee Meeting. https://www.nejm.org/doi/full/10.1056/NEJMp078167
The Kefauver–Harris Amendments at 50
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4101807/
A Political History of Medicare and Prescription Drug Coverage
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2690175/
This article examines the history of efforts to add prescription drug coverage to the Medicare program.
https://www.patentdocs.org/followon_biologics/\
Overview of FDA’s Expanded Access Program for Investigational Drugs
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443564/
https://www.ncbi.nlm.nih.gov/pubmed/27917324
Indian Patent act of 1970
https://www.lexology.com/library/detail.aspx?g=3f92413f-107c-4886-aca7-24633a341e22
The Fate of FDA Postapproval Studies, by Steven Woloshin M.D. et al N Engl J Med 2017; 377:1114-1117
FRANCES KELSEY AND THALIDOMIDE
https://www.fda.gov/media/89162/download
Kefauver
Crime in America, by Estes Kefauver; paper back 1951
Sulfa in ethylene glycol
https://www.the-scientist.com/foundations/the-elixir-tragedy-1937-3
https://www.smithsonianmag.com/history/the-senator-and-the-gangsters-69770823/
Remdesivir