A woman standing on top of a mountain holding the approved FDA therapy

The FDA Approves the First Targeted Therapy for Bladder Cancer

We hear often in recent years about “personalized medicine” and “targeted therapies” for cancer. These terms typically refer to treatments that target specific genetic mutations in a person’s cancer.

For some cancers, there are FDA-approved drugs to treat patients with a given and specific genetic mutation. That hasn’t historically been the case in bladder cancer, though.

An early version of targeted therapy

My first husband, Ahmad, received a very early version of targeted therapy in late 2013. At that time, there were no targeted therapies for bladder cancer. But a clinical trial at Memorial Sloan Kettering in New York had tested a drug—typically used to treat kidney and pancreatic cancers—in a clinical trial of bladder cancer patients with advanced disease.1

Why did the treatment work for one patient?

For most of the patients in that clinical trial, the drug did not work and they died from their disease. But one patient, a woman in her 70s, experienced a miraculous and, what oncologists call, durable remission.

Of course, the doctors wanted to know why this woman’s response was so remarkable when most of the patients did not respond at all. They sequenced her genome and found a mutation (in a gene called TSC1) that fewer than 10% of bladder cancer patients have.

My husband underwent genetic testing

It turned out that my husband had that same mutation. Genomic sequencing in 2013, cutting edge and not routinely performed at that time, revealed the mutation. We and our oncologist were hopeful that he would have a similar response.

Unfortunately, I think it was too late in the trajectory of his disease. This was the only drug that did actually shrink his tumors—the standard chemotherapy had done nothing. So that was hopeful but it wasn’t enough to save his life.

But the exciting and hopeful news for current advanced bladder cancer patients is that the FDA has just approved a different drug targeting a different mutation known as FGFR3 or FGFR2.2 It is estimated that about 20% of bladder cancer patients have this mutation. The overall response rate in these patients was about 32%. Most had only a partial response but a small percentage had a complete response.

Hopeful news for advanced bladder cancer

Some of these patients had been treated with immunotherapy drugs before treatment with Balversa, the new targeted therapy. Just a few short years ago—when Ahmad had advanced bladder cancer—the only tool that Western medicine could offer was platinum-based chemotherapy. It was cutting edge and considered “investigational” to get the targeted treatment that he got. “Investigational” meant that our insurance wouldn’t cover it until they were forced to by a government regulator.

Immunotherapies and this new targeted treatment are not cures. They aren’t suited for everyone and they aren’t working for everyone. And they do have side effects.

An exciting drug breakthrough

But it is tremendous progress that these new “tools” continue to emerge. Each is a step forward that will give doctors and patients more options. And more options eventually translate into more hope. And that will eventually translate into fewer deaths from bladder cancer.

Let’s hope that comes as soon as possible.

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