2 ms·
And congratulations once again. This kind of therapy is a big deal because of just how different it is from standard 'drugs'. This is a precise and intentional
by jfarlow 9y ago
And congratulations once again. This kind of therapy is a big deal because of just how different it is from standard 'drugs'. This is a precise and intentional genetic change to a patient's own immune cells to provide it a new capability. This new capability permits one's own immune system to detect a cancer with precision, clearing it from the body without harming other non-cancerous cells. The same protein doing the work here can be easily tuned to become more and more precise, and the scaffold of the technique can be ported to other targets relatively easily - again very much unlike most small molecule drugs. In this way it is nothing like most any drug of the 20th century. And there are many more to come.
There are >100 in the FDA pipeline right now for various types of cancer [1]. And the concepts used here to introduce DNA encoding proteins that confer NEW functions can be used to introduce any number of new functions.
So in less than 2 months, the FDA has approved 3 gene therapies:
1) Cure for Leukemia (Novartis' CAR)
2) Cure for Blindness (Spark's hRpe65)
3) Cure for Lymphoma (Gilead's (nee Kite) CAR)
Here's our description of the designed protein in case you want to learn what it does at a molecular level, or want to design your own:
https://serotiny.bio/notes/proteins/car19/ https://serotiny.bio/notes/proteins/car19/
[1] (paywalled CAR trails list): https://www.ncbi.nlm.nih.gov/pubmed/27613725 https://www.ncbi.nlm.nih.gov/pubmed/27613725