FDA to Fast-Track Possible Cure for Cancer

The new treatment has been granted "breakthrough" status by the FDA. This site criticizes CBS News for using that terminology so prominently, but not explaining what it means.

Though, to my ear, it still sounds pretty good:

For 40 seconds – an eternity by network TV standards – the words "CANCER BREAKTHROUGH" at the top of the newscast jumped out at the lay public. But what does it mean – and what might it NOT mean – when the FDA designates a procedure with "breakthrough status"?

An FDA Fact Sheet states:

A breakthrough therapy is a drug:

intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and

preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request.

Simply speaking, the FDA uses that designation to note that something has shown promise -- "it may demonstrate substantial improvement." It is not an announcement to the public that a breakthrough of substantial improvement has been declared.

Okay, fair enough -- it's just a bureaucratic designation for a treatment that seems promising, may treat a deadly disease better than the current ones, and which is given fast-track/expedited review to get it to market quickly.

But that does still sound very promising.

The treatment is like a black-humor joke -- they're going to treat your cancer by giving it polio.

But that is actually what they're doing, sort of. The treatment consists of injecting the patient with a modified form of the polio virus.

The virus is the creation of -- or, the obsession of -- molecular biologist Matthias Gromeier.

Gromeier re-engineered the virus, removing a key genetic sequence. The virus can't survive this way, so he repaired the damage with a harmless bit of cold virus. This new modified polio virus can't cause paralysis or death because it can't reproduce in normal cells.

But it can reproduce in cancer cells, and in the process of replicating, it releases toxins that poison the cell. This process also awakens the immune system to the cancer it never noticed before.

"Why didn't the immune system react to the cancer to begin with," CBS News' Scott Pelley asked Gromeier.

"All human cancers, they develop a shield or shroud of protective measures that make them invisible to the immune system," Gromeier explained. "This is precisely what we try to reverse with our virus. So by infecting the tumor, we are actually removing this protective shield. And enabling the immune system to come in and attack."

It appears the polio starts the killing, but the immune system does most of the damage. [Brain cancer patient] Stephanie Lipscomb's tumor shrank for 21 months until it was gone. Three years after the infusion, something unimaginable had happened.

An MRI in August of 2014 showed no active cancer cells at all.

There'll be a segment about this on 60 Minutes on Sunday.

posted by Ace at 01:12 PM

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