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Interim Data Show Increased Median Overall Survival of Brain Cancer Patients Treated with Tocagen’s Toca 511 & Toca FC, Compared to Published Benchmarks

Al's Comment:

Impressive results.. Especially the 6 month survival rate (for recurrent glioblastoma)  of 93.8% compared to historical rate of 59%.  It also did well with toxicity.  This is a gene therapy. First the virus has to be injected - either intra-tumorally, at the time of surgery or IV (right now they are doing the IV study combined with intra-tumoral). Then they wait and give the virus time to infect the tumor cells where it inserts a gene that produces an enzyme that converts a safe, low toxicity antifungal drug into a toxic chemotherapy.  . It only infects tumor cells and not normal cells. Visit their website to see how they made that possible!   Then they give the oral anti-fungal drug which is relatively non-toxic. This drug crosses the blood brain barrier and is harmless to normal human cells, but when it gets to a cell that was infected and has the enzyme, this drug, 5fc gets converted to the chemotherapy drug 5fu, and it kills those cells.   In theory it is the perfect magic bullet -  killing all tumor cells with minimal side effects.  In practice it doesn't kill all of the tumor cells, but those cells that aren't killed act as a reservoir for the virus and they wait another month and then repeat the 5FU. After a few cycles, most or all of the tumor cells should be gone. It worked well in animal models.  Too early to tell how it will work in people but this interim data looks pretty good.

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