FDA Approves a Second CAR-T

choi

Denise Grady, New Therapy to Kill Cancer Is Approved. New York Times, Oct 19, 2017.
https://www.nytimes.com/2017/10/ ... py-cancer-kite.html

Quote:

(a) "The Food and Drug Administration on Wednesday [Oct 18] approved the second [CAR-T cell therapy] * * * The new therapy, Yescarta, made by Kite Pharma, was approved for adults with [DLBCL].  who have undergone two regimens of chemotherapy that failed. * * * The cost will be $373,000.

(b) "Tina Bureau, a fifth-grade teacher from Queensbury, NY, was one of the lymphoma patients in the study. Previously, she'd had several types of chemotherapy.

" 'The cancer would shrink but then it would come right back,' she said.

"Last spring [2017], she had the T-cell treatment at the Dana-Farber Cancer Institute and Brigham and Women's Hospital in Boston. The side effects were ferocious.

" 'You don’t even recognize your family members,' Ms Bureau said. 'I had some bleeding on my brain, and had to be put in intensive care. The week it was happening, I don't remember a lot. It was much more difficult for my family than me.'

"Within a month, she had a complete remission, which has continued. She is back at work, full time.

" 'Yes, it can pose life-threatening problems,' Ms Bureau said. “But when you're in a situation where your life's threatened anyway, I don't feel you have anything to lose.'

Note:
(a) There is no need to read the rest of the NYT report.
(b) News release: FDA Approves CAR-T Cell Therapy to Treat Adults with Certain Types of Large B-cell Lymphoma; Yescarta is the second gene therapy product approved in the US. fda, oCT 18, 2017
("Diffuse large B-cell lymphoma (DLBCL) is the most common type of NHL [non-Hodgkin lymphoma] in adults")
(c) CAR-T stands for chimeric antigen receptor (CAR) technology.

(d) from en.wikipedia.org:
(i) Kite Pharma (2009- ; based in Santa Monica, Calif; founded by Arie Belldegrun, MD, an Israeli-American oncologist)
(ii) "In August 2017, the company [Gilead Sciences, Inc]announced it would acquire Kite Pharma for $11.9 billion."
(iii) Gilead Sciences's website announced on Oct 3, 2017 that the acquisition was completed that day.
(iv) Gilead Sciences, Inc (1987- ; headquarters Foster City, Calif [on the western edge of Silicon Valley])
(v) I fail to find out why the company named itself Gilead. But see Gilead
https://en.wikipedia.org/wiki/Gilead

(e) What is diffuse large B-cell lymphoma (DLBCL)?
(i) A median age at the time of diagnosis is 64 or 70, depending on two different sources. So far (before introduction of CAR-T), the five-year survival rate with treatment is 58% overall.
(ii) "an aggressive (fast-growing) lymphoma. It can arise in the lymph nodes or outside of the lymphatic system in the gastrointestinal tract, testes, thyroid, skin, breast, bone, or brain. Often, the first sign of DLBCL is a painless rapid swelling in the neck, armpit, abdomen, or groin which is caused by enlarged lymph nodes."
National Center for Advancing Translational Sciences (NCATS), National Institute of Health (NIH), June 28, 2016
https://rarediseases.info.nih.go ... rge-b-cell-lymphoma
(iii) "Diagnostic Criteria: [A] Diffuse effacement of normal architecture [; B] Large non-cohesive cells make up over 50% of population [among other things]”
Surgical Pathology Criteria. Stanford University, undated
http://surgpathcriteria.stanford.edu/bcell/dlbcl/
(iv) What does it mean, by "diffuse effacement of normal architecture" in (d)(ii)?

Hunt KE and Reichard KK, Diffuse Large B-Cell Lymphoma. Arch Pathol Lab Med, 132: 118-24 (2008)
www.archivesofpathology.org/doi/pdf/10.1043/1543-2165(2008)132[118LBL]2.0.CO;2
("Diffuse large B-cell lymphoma most commonly completely effaces the normal lymph node architecture, although partial nodal involvement can be seen.")
(v) a pathological slide:

Diffuse Large B-cell Lymphoma, NOS
www.pathpedia.com/education/eatl ... l_lymphoma_nos.aspx
("large lymphocytes (defined as >2 times larger than a small mature lymphocyte)" )

Notable are those large lymphocytes with white halos (which are cytoplasm).