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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: 0, Disagree) by Anonymous Coward on Thursday August 31 2017, @04:20PM (2 children)
We really don't know what the long term effects of these treatments can be. I'm not sure what the best way forward is, but as a species we should be very careful about changing our genetic code. Do these therapies mean that some non-human DNA gets introduced to the population? Or are the gene sequences reliably rewritten to match a healthy human's sequence?
The following is for discussion, I'm not necessarily advocating it: if gene therapy mixes in foreign DNA we may want to sterilize those individuals. They get to live, but after that point cannot possibly contaminate the human gene pool. What if some sequence gets turned on by accident? What if meiosis reorganizes the genes and the addition introduces something harmful? Are we able to reverse changes reliably?
(Score: 4, Informative) by Joe on Thursday August 31 2017, @05:18PM (1 child)
T cells do not undergo meiosis and modifications to them have no plausible mechanisms of spreading into the germ-line. T cells, as well as B cells, are already genetically distinct from the normal genome due to genetic rearrangements and mutations brought about through the actions of RAG proteins.
The modified DNA consists of human-derived sequences that code for a chimeric antigen receptor (CAR) - which is basically an antibody (in this case specific for CD19) fused to part of the cytoplasmic domain of the T cell receptor and other co-stimulatory proteins.
Hypothetically, if such sequences were to be directly introduced into the germ-line, the phenotype of the offspring would likely be similar to Omenn syndrome or other immunodeficiencies.
- Joe
https://en.wikipedia.org/wiki/Meiosis [wikipedia.org]
https://en.wikipedia.org/wiki/Recombination-activating_gene [wikipedia.org]
https://en.wikipedia.org/wiki/Chimeric_antigen_receptor [wikipedia.org]
https://en.wikipedia.org/wiki/Omenn_syndrome [wikipedia.org]
(Score: 0) by Anonymous Coward on Thursday August 31 2017, @07:04PM
Thanks for the details, my comment was more general and I didn't do much checking about this particular therapy. I guess my concerns could be covered by genetic testing, if people are worried about it they can request genetic tests to find out what they'd be exposing their offspring to.