Cancer Cell Therapy Trials Spike in Last Six Months, New Report Finds

In just the six months from last September to March, the number of cancer cell therapies in development has increased by 87%, though about half of the total tallied is in the preclinical phase, according to a new report from the non-profit Cancer Research Institute in Nature Reviews Drug Discovery.

The Cancer Research Institute’s database is now tracking 753 cell therapies, pursuing 113 different targets, and of which 375 are in clinical trials and 378 are in the preclinical phase.

The US and China are developing more than two-thirds of the cancer cell therapies, and one third in the database are being developed by academic centers in both countries.

The cell therapies were classified into seven categories based on the mechanism of action:

• chimeric antigen receptor T-cell (CAR-T)
• T cell receptor (TCR) T
• autologous circulating T cells targeting an unspecified tumor-associated antigen (TAA) or a tumor-specific antigen (TSA)
• tumor-infiltrating T cells (TIL)
• T cell therapies based on new technologies (ie. Induced pluripotent stem cells, CRISPR)
• Cell therapies derived from natural killer cells (NKT cells)
• And other cell types (macrophages or stem cells)

“Most of the CAR T [chimeric antigen receptor T-cell] agents developed in China are not innovative therapies, but locally produced agents with properties similar to those already approved or in late stage clinical development in the US,” the article says.

The US Food and Drug Administration (FDA) also has said it has its own database of CAR-T therapies – previously noting that it contained more than 100 investigational new drug (IND) applications.

And three cell and gene therapies have won FDA approval: two Car-T cell therapies, including Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL) and Gilead’s Yescarta (axicabtagene ciloleucel) for adults with certain types of large B-cell lymphoma; and a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness.

Earlier this week, FDA Commissioner Scott Gottlieb discussed several new cell and gene therapy guidance documents that are expected soon.

Gottlieb also said the agency may be willing to accept more uncertainty for certain gene and cell therapies, particularly with those earning the new Regenerative Medicine Advanced Therapy (RMAT) designation. He noted that as of the end of April, there had been 62 RMAT designation submissions since the inception of the program in December 2016, and 19 designations have been granted (see 16 of them here).

FDA Cellular & Gene Therapy Guidances

Cancer Research Institute