While many drugs have helped improve the quality of life for patients, few of these could–or should–be claimed as “miracle drugs.”
The phrase “miracle drug” rightfully raises eyebrows in most medical contexts, although a new study involving an experimental new cancer treatment may actually qualify, as data shows that a new immunotherapy drug cured 100% of patients in its trial study.
Background: Fighting Rectal Cancer
The study, conducted by oncologists at Memorial Sloan Kettering Cancer Center (MSK) in New York, focused on patients battling rectal cancer. With around 45,000 Americans diagnosed with rectal cancer annually, this cancer is rather uncommon, but its symptoms can be severe.
According to one researcher in the study, Dr. Luiz Diaz Jr., “[Patients] can suffer life-altering bowel and bladder dysfunction, incontinence, infertility, sexual dysfunction, and more.” Patients with rectal cancer try to get radiation or chemotherapy treatment to help, but this can be difficult depending on the location of the tumor. The researchers at MSK hoped to try something new by using an innovative immunotherapy drug.
Analysis: Treating Tumors
Immunotherapy is a form of cancer treatment that aids the immune system in fighting the disease. To leverage this treatment, the researchers looked at 12 patients with tumors that had mismatched repair deficient (MMRd) genes. These genes helped make the tumors more sensitive to immunotherapy.
“Immunotherapy has proven successful in treating a subset of patients with colon and rectal cancer that has metastasized, meaning spread to other tissues,” explained Dr. Diaz. The 12 patients were required to have the MMRd tumors in either stage 2 or 3 cancer. These patients were then administered the immunotherapy drug every three weeks for a six-month period. The tumors were monitored using medical imaging throughout the whole period.
“We initiated a prospective phase 2 study in which single-agent dostarlimab, an anti–PD-1 monoclonal antibody, was administered every 3 weeks for 6 months in patients with mismatch repair–deficient stage II or III rectal adenocarcinoma,” the study’s authors wrote. Anti–PD-1 (programmed death receptor-1)–blocking monoclonal antibodies are a variety of what are known as immune checkpoints inhibitors (ICIs), which past studies have shown to have proven antitumor abilities.
Following the administration of the experimental drug, standard chemotherapy and surgery were to be administered, according to the study. However, at the end of the six-month trial period, the researchers found that none of the twelve patients had tumors; all had completely disappeared. As researcher Dr. Andrea Cercek stated, “One man and his family just sat in stunned silence when I told them his cancer had disappeared. Then they thanked us over and over.”
The immunotherapy drug seemed to allow the patients to avoid radiation or surgery, and restored normal body processes again. “They have preserved normal bowel function, bladder function, sexual function, fertility. Women have their uterus and ovaries,” Dr. Cercek added.
While the researchers published their results in The New England Journal of Medicine, it may be premature to label treatment a “miracle drug.” The small sample size of only 12 individuals suggests that more research is needed. However, the study does bear promise for a new type of treatment that could help a growing number of cancer patients.
“We are seeing more and more young people with rectal cancer, including people in their 20s in our trial,” said Dr. Cercek. “Immunotherapy might be an important new option for them.”
Kenna Hughes-Castleberry is a staff writer at the Debrief and the Science Communicator at JILA (a partnership between the University of Colorado Boulder and NIST). She focuses on deep tech, the metaverse, and quantum technology. You can find more of her work at her website: https://kennacastleberry.com/