Startup targets glioblastoma tumors with CAR-T therapy – FierceBiotech

One of the major breakthroughs in cancer treatment is CAR-T technology, which involves genetically modifying a patient’s own immune cells so they can recognize and attack cancer. But while the innovation has benefited patients with certain blood malignancies, progress in solid tumors remains limited.

Now, scientists at McMaster University and the University of Toronto have developed a CAR-T therapy for the aggressive brain cancer glioblastoma. It helped reduce tumor burden and improved survival in mouse models, according to a new study published in the journal Cell Stem Cell.

The researchers were so encouraged by the findings that they launched a startup called Empirica Therapeutics, which aims to bring the CAR-T drug into clinical trials in recurrent glioblastoma patients by 2022.

For each CAR-T construct, T cells are modified to produce a special structure called a chimeric antigen receptor (CAR) that gives the cells the ability to recognize a specific protein on cancer cells. The two FDA-approved CAR-Ts, Novartis’ Kymriah and Gilead Sciences’ Yescarta, are directed toward CD19. The CAR-T cell Empirica is developing targets CD133, also known as prominin-1.

In a 2003 Cancer Research study, a McMaster University team identified a group of neural stem cells from human brain tumors that bear CD133 on their surface. They found these CD133-expressing cells could differentiate into cells identical to the original tumor, suggesting these stem cells are necessary for glioblastoma tumor growth.

For the current study, the team tested three types of treatments in lab dishes and in mice. The first was a human IgG antibody
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