https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm
The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," said FDA Commissioner Scott Gottlieb, M.D. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."
Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte. The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Also at NPR, CNN, BBC, and FierceBiotech.
Novartis press release.
(Score: 2) by Joe on Thursday August 31 2017, @01:20PM (2 children)
Stem cells typically dampen-down the immune response, but there have been efforts to use implanted scaffolds to improve CAR T cell therapies for solid tumors (link below).
There are lots of research groups that are trying to generate a one-size-fits-all version that uses donor-derived cells instead of patient-derived cells. Donor-derived would greatly lower costs and improve consistency, but they would also come with a much greater risk of off-target effects (such as a rapid and unstoppable graft-versus-host disease). Kill switches and other genetic modifications will mitigate the risks and improve the efficacy of CAR T cell therapies.
We are in the early days of this type of research and, despite its current success, there is even greater potential for it to be extended to other forms of cancer.
Scaffold: https://soylentnews.org/comments.pl?noupdate=1&sid=19218&page=1&cid=500984#commentwrap [soylentnews.org]
GVHD: https://en.wikipedia.org/wiki/Graft-versus-host_disease [wikipedia.org]
(Score: 2) by Geezer on Thursday August 31 2017, @01:27PM (1 child)
I was just thinking that perhaps CAR T cells are not so different structurally at the cellular from stem cells that the same 3d printing mechanics could be applied, with the resulting reduction in time and cost.
Again, I'm not a bio guy, just a lowly EE, and appreciate better-informed opinion.
(Score: 3, Informative) by Joe on Thursday August 31 2017, @02:02PM
These kind of T cells do not have and would not likely benefit from structural interactions with other cells. One of the great advantages of T cells is that they can "search and destroy" by homing-in on sites of cellular damage and infiltrate densely packed areas to "find" what is responsible and, directly or indirectly, kill it.
T cells already infiltrate tumors and kill cancer cells, but the tumor microenvironment as well as immunoediting can prevent their activity or convert them into a cancer-protecting state (Tregs). The, frankly, amazing success of cancer immunotherapy by blocking immune checkpoints relies on reversing the negative effects of the tumor microenvironment.
https://en.wikipedia.org/wiki/Tumor_microenvironment [wikipedia.org]
https://en.wikipedia.org/wiki/Immunoediting [wikipedia.org]
https://en.wikipedia.org/wiki/Regulatory_T_cell [wikipedia.org]
https://en.wikipedia.org/wiki/Cancer_immunotherapy#Immune_checkpoint_blockade [wikipedia.org]