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The First Human Trial Of A New Approach To Cancer Treatment Has Scientists Very Excited

cancer therapy

Two of the most promising recent approaches to cancer treatment are immunotherapy, which harnesses the body’s own immune system to fight cancer, and personalized medicine, which involves therapeutics that are targeted to the genome of a particular patient and that patient’s cancer.

Now, scientists have combined those two strategies to create a novel treatment: a vaccine developed for a single patient that triggers an immune system attack that is laser-focused on that patient’s tumors.

In the very first human trial testing this approach, the personalized vaccines successfully activated an immune response in three patients with melanoma.

The research is in its earliest stages — it’s too soon to say if the treatment actually improved survival in the patients or whether it will work in others at all. Still, scientists say they are very excited about the idea and the proof-of-concept results, which were published in the journal Science on April 2.

In cancer, each tumor is unique, which is one reason that they can be so hard to treat. In this new approach, scientists have used that to their advantage. The broken genes that make a tumor grow out of control “can also be targeted by the immune system to control malignancies,” researchers from the Netherlands Cancer Institute and Washington University School of Medicine explained in a research review published alongside the study.

To create the personalized treatments, scientists determined the unique genetic make-up of each patient’s melanoma tumors, which had been surgically removed. They then identified special targets, called neoantigens, on the surface of each patient’s cancer cells. Using those targets, they developed a personalized vaccine for each patient that would hopefully encourage their immune system to attack these specific neoantigens on that patient’s cancer cells.

Selecting those unique-to-the-tumor targets helps to minimize adverse events or side effects, explained Elaine Mardis, a study coauthor and researcher at the Genome Institute at the Washington University School of Medicine. The immune response triggered by the personalized vaccines is designed to behave more like a sniper than a bomb — using the neoantigens as “flags” so it can specifically take out the cancerous cells.

Still, the process is complicated: developing each vaccine took the research team about three months — too long to wait for many cancer patients. They’re hoping a timeline of four to six weeks will become possible as they refine the process.

Even at this early and uncertain stage, scientists are encouraged by this approach because there’s a chance it could prove effective in patients who don’t respond to existing treatments. And while the trial was in melanoma, the researchers are eager to try it out on other cancers that are associated with carcinogens, like bladder, lung, and colorectal cancers.

“Many researchers have hypothesized that it would be possible to use neoantigens to broadly activate the human immune system, but we didn’t know that for sure until now,” said Dr. Gerald Linette, an oncologist at the Washington University School of Medicine and a coauthor on the paper. “We still have much more work to do, but this is an important first step and opens the door to personalized immune-based cancer treatments.”

His colleague, Dr. Beatriz Carreno, added: “These findings represent a significant step toward more personalized immunotherapies.”

It’s important to remember, though, that the Phase I trial was designed to evaluate safety, not long-term effectiveness, which will have to be assessed in future studies.

“At the moment it’s not clear how effective this immunotherapy would be at killing cancer cells in the body and improving survival, but this promising study sets the stage for creating vaccines that are designed to target each patient’s individual tumor in the future,” said Dr. Alan Worsley, of Cancer Research UK.

More testing will determine whether that immune response is tied to survival and whether it can be replicated in a wider variety of patients. The researchers are planning to conduct a larger trial within the year.



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"Science is a way of thinking much more than it is a body of knowledge." -- Carl Sagan


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