“This may be the cure for some types of cancer already,” said Dr. Joshua Brody.

Dr. Brody is director of the Lymphoma Immunotherapy Program at the Icahn School of Medicine at Mount Sinai in New York City. He talked with LifeZette in the wake of new findings presented this week on a promising immunotherapy treatment at the annual meeting for the American Association for the Advancement for Science (AAAS).

Immunotherapy, which uses the body’s own immune cells to treat diseases, is an exciting pathway in cancer research. And here’s what has Dr. Brody and many others excited: Dr. Stanley Riddell may just have hit the jackpot in regard to a cure.

Riddell has seen “extraordinary” results using T-cells to target blood cancer. Patients under his care have seen symptoms disappear or go into remission, even in cases in which they were told they would not survive much longer.

The therapy, which engineers T-cells with chimeric antigen receptors (CARs), involves removing a patient’s own immune cells, tagging them with receptor molecules to target a specific cancer, and injecting them back into the body. The immune system does the rest to attack blood based cancers.

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Dr. Riddell, a researcher at the Fred Hutchinson Cancer Research Center in Washington State, has been treating 35 patients with acute lymphoblastic leukemia.

Upon receiving CARs, 94 percent of these patients stopped experiencing symptoms. Many with lymphoma experienced remission. More than 80 percent of people in Dr. Riddell’s trials who had non-Hodgkin’s lymphoma saw symptoms diminish.

“These (results) are in patients that have failed everything. Most of the patients in our trial would be projected to have two to five months to live,” Riddell told The Guardian. “This is unprecedented in medicine, to be honest, to get response rates in this range in these very advanced patients,” he added.

The therapy isn’t for everyone, Riddell also said. In fact, it is only suitable for a small number of patients that have B-cell malignancies.

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Looking Ahead
Riddell’s CARs treatment isn’t as easy as injecting fighting cells back into the body. Reprogramming a patient’s immune system can cause serious side effects. One of them, cytokine release syndrome (sCRS), can be fatal.

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During his research, 20 patients experienced fever, hypotension and neurotoxicity from CARs; two of them actually died. It’s important to note, however, that the two patients did not see improvements when they underwent chemotherapy, and had no other treatment options.

Still, there’s more work to do to perfect CARs. Researchers say they want to address solid tumors and better monitor patients in remission after the therapy. Riddell said his team is conducting clinical trials in acute lymphoblastic leukemia, non-Hodgkin’s lymphoma and chronic lymphocytic leukemia.

“The next steps are to perform additional trials to optimize the therapy and eventually to get approval for widespread use,” he said.

While the results so far are incredibly positive, Riddell is hoping everyone keeps this in perspective. “I don’t want people with cancer to think this approach will apply to everyone,” he said. “The results are very encouraging, but a lot of research needs to be done to extend the work to other cancers.”

Cure for Cancer?
Dr. Brody said that overall, immunotherapy as a class of treatments is showing “drastic and miraculous results,” and that Riddell’s research is “a big deal.”

Another immunotherapy similar to CARs is known as checkpoint blockade, which involves using antibodies to drive T-cells to attack tumors. Brody said it has been helpful to fight conditions such as melanoma, bladder cancer, lung cancer, some lymphomas and other types of cancer.

While the research into immunotherapy is exciting and labs throughout the country like Riddell’s are plowing ahead to make advancements as quickly as possible in the field, others are tapping the brakes a bit.

Cai Xuan, Ph.D., an analyst for GlobalData covering oncology and hematology, said the potential cost of T-cell therapy, the scalability of this type of treatment, and the safety profile all remain major concerns according to pharmpro.com.

“The cost of T-cell therapy is currently estimated at a staggering $300,000 per patient, with some estimates pegging the cost at over $500,000. Given this, T-cell therapies must show curative ability in order to justify such a high price tag in comparison to existing treatment options such as stem cell transplantation, which can cost from around $100,000 to $200,000,” Xuan said.

“In order to do this, more trials must be conducted. To date, only a handful have gone ahead, in a limited number of patients, due to the high cost as well as the long and difficult manufacturing processes involved in administering T-cell therapy. This presents a huge scalability problem which cannot easily be solved.”

Dr. Brody and many others, including the patients who have recovered after being told there was nothing more that could be done for them, believe the developments are significant and have the potential to make real breakthroughs.

Earlier this week, another form of T-cell therapy received an FDA breakthrough therapy designation. This is for the treatment of patients with inoperable or metastatic pretreated synovial sarcoma, a soft tissue cancer.

“There’s a little bit of a hype, but there’s a lot of reality,” he said.