Cancer Treatment Revolution: Gene Therapy Works and is Already Changing The Future


Intravenous chemotherapy has for decades been a standard treatment for cancer. But it may soon be considered as archaic as the telegraph, says a top medical analyst.

Robert Goldberg of the Center for Medicine in the Public Interest says cancer treatment is on the cusp of huge advances. Targeting cancer-causing gene mutations – instead of a malignancy’s site of origin – is the next frontier, he says. Targeted therapy, which comes in pill form, has already proven to be a game-changer for some of the most virulent types of the disease, including pancreatic cancer.

“We’re entering an era that isn’t a matter of where the cancer is but the mutation that causes the cancer,” Goldberg says. “One genetic mutation can cause a lot of different cancers.”

Even the language of cancer will change, Goldberg says. Instead of talking about site of the cancer – breast cancer, lung cancer, prostate cancer, etc. – doctors will refer to the gene mutation that causes it.

For example, a mutation known as the “sonic hedgehog pathway” causes cancers of the skin, pancreas, stomach, lung, brain, and possibly prostate, according to Goldberg.

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Already, malignancies historically unresponsive to treatment, such as cancer of the pancreas and multiple myeloma, are not just being controlled but in some cases cured, he says. And targeted treatment – pills swallowed just like aspirin – is far less toxic than traditional IV chemotherapy, mitigating both side effects as well as damage to the body’s healthy cells. The net result? Extended quantity and quality of life.

Targeted treatment works by identifying genetic abnormalities with a simple blood or tissue sample, which then provides doctors with a patient’s genetic roadmap. Treatment is customized accordingly.

“It’s like going into a shoe store and instead of being handed a size six shoe because it may be the average size, you actually measure and get it fitted more exactly to your foot,” Goldberg says.

Targeted therapy pills are formulated to destroy the cancer cells and bypass the healthy ones. There are currently 150 known cancer-causing mutations with thousands of others playing a supporting role, according to Goldberg. Researchers are working to target the “driver mutations” that control the spread of tumors.

The success stories are abundant:

·         In 2012, the Food and Drug Administration approved the drug Erivedge to treat advanced basal cell skin cancer, one of the sonic hedgehog mutations. It was later found to help pancreatic cancer patients, a diagnosis known for its dismal prognosis. Erivedge is currently being studied for treating other sonic hedgehog pathway cancers.

·         The remarkably successful drug Gleevec, first approved in 2001 to treat chronic myeloid leukemia (CML), was later approved for some types of stomach cancer. Goldberg says the next generation of drug attacking this mutation could be “an effective cure.”

·         Tykerb, approved for breast cancer, has shown promise treating other solid tumors.

·         In February, the FDA approved Pomalyst (a more powerful form of the drug Revlimid) to treat multiple myeloma, cancer of the plasma cells.

“Multiple myeloma used to be incurable. Now, if you get it early enough you can expect to live a normal life span,” Goldberg says.

Though there has been marked progress, many mutations remain a mystery. Researchers not only need to identify the myriad mutations, they have to figure out what medicines will block them in the safest, fastest way, according to Goldberg.

Mutation testing is still not standard protocol for many doctors, Goldberg says, and even if it was, securing targeted treatment is cost prohibitive for many people. Insurance benefits lag behind treatment advances and the newer drugs come with a hefty price tag.

Many breakthrough oral treatments are covered at just 50 percent compared to more generous benefits for old-line IV chemotherapy.

Goldberg thinks market demand will ultimately ensure emerging testing and that treatments are within patients’ reach.

In the cancer clinic of the future, Goldberg predicts there will be few IV stations. Instead, there will be pills dispensed that cancer patients will take at home. And those pills will cure them.


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