The patient whose chronic granulomatous disease (CGD) was treated in a world first last year at CHU Sainte-Justine can essentially be considered \u201ccured,\u201d says the doctor who supervised his treatment in Montreal.<\/p>\n
Last spring, Dr. \u00c9lie Haddad and his colleagues used a gene therapy called \u201cprime editing\u201d to correct, directly in the patient\u2019s hematopoietic stem cells, the genetic mutation responsible for this serious immune system disease.<\/p>\n
And even though great caution must always be exercised before declaring a cure in connection with such a sophisticated therapy, for which there is little historical data, it is difficult, one year later, not to tell the patient that he is \u201ccured,\u201d admitted Dr. Haddad.<\/p>\n
\u201cIt\u2019s as if, even though we believe deep down that there is a strong chance he is cured, we refuse to give him the good news and instead leave him with a sword of Damocles hanging over his head,\u201d said the researcher, whose enthusiasm is palpable even over the phone.<\/p>\n
That being said, at this time, the 18-year-old patient shows \u201cno symptoms whatsoever,\u201d about three-quarters of the diseased white blood cells have been corrected (\u201cit\u2019s stable, it\u2019s exactly what we had at the beginning\u201d) and \u201cthis stability strongly suggests that it will last forever, so it\u2019s perfect, we\u2019re very, very happy and he has a normal life,\u201d applauded Dr. Haddad.<\/p>\n
In a recent update published by the prestigious New England Journal of Medicine, Dr. Haddad\u2019s team also reported extremely encouraging data for a second patient who benefited from the same therapy.<\/p>\n
\u201cThese results demonstrate that prime editing can achieve high levels of precise correction and combined with clinical data, support the development of this approach as a therapeutic platform for CGD and other genetic diseases,\u201d they write.<\/p>\n
Even though he lives in British Columbia, and even though everything that can be delegated to Vancouver is, the young patient continues to visit CHU Sainte-Justine regularly so that researchers can keep an eye on how things are progressing.<\/p>\n
\u201cWe monitor it like milk on the stove to make sure we don\u2019t miss anything,\u201d said Dr. Haddad, who is the principal investigator for the clinical trial at Sainte-Justine University Hospital Center and a professor at the Universit\u00e9 de Montreal\u2019s Faculty of Medicine. The best sign is when the patient gets tired of coming in. If they feel like they\u2019re coming in for nothing, that means they\u2019re on the road to recovery.\u201d<\/p>\n
From dream to reality<\/p>\n
The usual treatment for CGD is similar to that used for certain types of leukemia: the patient\u2019s bone marrow\u2014where the immune system\u2019s white blood cells are formed\u2014is destroyed with very aggressive chemotherapy, and then healthy bone marrow from a donor is transplanted.<\/p>\n
However, this is a very painful treatment for the patient and carries several risks, such as the body rejecting the transplant.<\/p>\n
The \u201cprime editing\u201d technique avoids some of these problems. First, the preparatory chemotherapy, while still unpleasant, is a little less \u201cbrutal\u201d for the patient. Subsequently, since it is the patient\u2019s own hematopoietic cells that are returned to them after gene editing in the laboratory, rejection is not possible. The survival rate should therefore, in theory, increase from 80 per cent or 90 per cent to 100 per cent.<\/p>\n
Gene therapy as we knew it until now, Dr. Haddad explained last year, consisted of replacing a defective gene with a new healthy gene.<\/p>\n
Prime editing takes this to the next level and makes it possible to correct virtually any mutation, said Dr. Haddad, who uses expressions such as \u201crevolution,\u201d \u201cmagic wand,\u201d \u201cabsolutely brilliant,\u201d and \u201ccompletely crazy\u201d when discussing the technique.<\/p>\n
And this is probably just the beginning: from genetic diseases present at birth to genetic mutations acquired along the way, such as cancer, we have yet to see the full potential of prime editing, he predicted.<\/p>\n
\u201cA success like this shows that we are capable of correcting a genetic abnormality,\u201d concluded Dr. Haddad. \u201cAnd given the number of absolutely incredible diseases that depend on a genetic mutation, it opens the door to correcting many other diseases, at least conceptually. (\u2026) We\u2019re no longer selling a dream, we\u2019re selling reality.\u201d<\/p>\n
\u2013This report by La Presse Canadienne was translated by CityNews<\/p>\n","protected":false},"excerpt":{"rendered":"The patient whose chronic granulomatous disease (CGD) was treated in a world first last year at CHU Sainte-Justine…\n","protected":false},"author":2,"featured_media":521490,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[194292],"tags":[49,48,2338],"class_list":["post-521489","post","type-post","status-publish","format-standard","has-post-thumbnail","category-montreal","tag-ca","tag-canada","tag-montreal"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts\/521489","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/comments?post=521489"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts\/521489\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/media\/521490"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/media?parent=521489"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/categories?post=521489"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/tags?post=521489"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}