Personalized cancer vaccine shows potential in myeloma, solid tumors
Immune responses seen in all 13 at-risk patients given PGV001 in Phase 1 trial
A personalized cancer vaccine called PGV001 showed potential in combating several types of cancer, both solid tumors and blood malignancies like myeloma, in a small Phase 1 clinical trial.
The vaccine targets neoantigens, proteins able to trigger an immune response against a cancer, that were specific to each of the treated patients, all considered at high risk for disease recurrence.
Results were in the study “PGV001, a multi-peptide personalized neoantigen vaccine platform: Phase I study in patients with solid and hematological malignancies in the adjuvant setting,” published in Cancer Discovery.
Anticancer vaccines, like all vaccines, work to train person’s immune system
Vaccines are a type of treatment that works to train the immune system to attack a specific molecular target. They have revolutionized care for infectious diseases, and researchers have begun to explore whether they might also help in fighting cancers.
An anticancer vaccine would have the capacity to train the immune system to recognize and eliminate tumor cells. However, developing an effective anticancer vaccine is far more complex than creating one for a virus. This is because each person’s cancer presents a unique set of neoantigens, tiny molecular changes specific to these cancer cells, to be targeted.
PGV001 works by taking a sample of the patient’s cancer and performing in-depth molecular profiling to identify the exact neoantigens that could be attacked. Then, a personalized vaccine targeting up to 10 different neoantigens is synthesized to treat the individual.
In this Phase 1 trial (NCT02721043), led by scientists at the Icahn School of Medicine at Mount Sinai in New York, the PGV001 approach was used to treat people with different types of cancer, including myeloma, lung cancer, head and neck cancer, urothelial cancer, and breast cancer.
A total of 14 patients enrolled in the trial; 13 of them received the PGV001 vaccine, and 11 finished the full course of treatment. Data showed that the anticancer vaccine was generally tolerated well, with no serious side effects reported in the study.
“Our data indicates that PGV001 is feasible and safe,” the researchers wrote.
Responses support vaccine helping the immune system to target cancer
Preliminary findings also showed that, in all patients who received the vaccine, the immune system showed signs of a response against the neoantigens, implying that the vaccine was activating the immune system to go after the cancer as designed. These data highlight “the potential of PGV001 for safely inducing targeted immunity,” the researchers wrote.
At five years of follow-up, six of the trial’s participants are alive, and three remain free of cancer.
“This study shows that making personalized cancer vaccines is possible and safe,” Nina Bhardwaj, MD, PhD, the study’s lead author and a professor of hematology and medical oncology at Mount Sinai, said in a news release from the Icahn school. Bhardwaj stressed that this was an early study in a small number of patients, but she considered its findings “an exciting step toward using the immune system to help people live cancer-free, longer.”
The National Cancer Institute at the National Institutes of Health, the Cancer Research Institute, and the Parker Institute of Cancer Immunotherapy helped to support this work.
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