This leading study is the first-ever investigation into the potential of immunotherapy to combat non-metastatic rectal cancer in patients. (CREDIT: iStock Images)
In a clinical trial involving 14 participants, immunotherapy on its own effectively eradicated rectal cancer. This leading study is the first-ever investigation into the potential of immunotherapy to combat non-metastatic rectal cancer in patients with a particular genetic mutation in their tumors.
Conducted by New York's Memorial Sloan Kettering Cancer Center (MSK), the trial reported a complete remission in all participants. Following immunotherapy, the cancer entirely vanished and has remained absent in all patients, some of whom have been cancer-free for as long as two years.
This trial is significant because it paves the way for immunotherapy to be used as a first-line treatment for certain types of rectal cancer, potentially sparing patients from the debilitating side effects of standard treatments of radiation, surgery, or chemotherapy. The findings were published in The New England Journal of Medicine.
The patients in the trial had tumors with a specific genetic makeup known as mismatch repair-deficient (MMRd) or microsatellite instability (MSI). MMRd tumors develop a defect in their ability to repair certain types of mutations that occur in cells. When those mutations accumulate in the tumor, they stimulate the immune system, which attacks the mutation-ridden cancer cells.
The clinical trial, included patients between the ages of 18 and 65. All patients received treatment with a checkpoint inhibitor, a type of immunotherapy that blocks the PD-1 protein on T-cells, allowing the immune system to attack the cancer. No patients received any other standard treatments for rectal cancer, such as radiation or surgery.
The results were significant, with all 14 patients in the trial achieving complete remission. The study’s lead investigator, MSK medical oncologist Andrea Cercek, said, “It’s incredibly rewarding to get these happy tears and happy emails from the patients in this study who finish treatment and realize, ‘Oh my God, I get to keep all my normal body functions that I feared I might lose to radiation or surgery.’”
MSK medical oncologist Luis Diaz, Jr., the co-investigator, explained that the research was sparked by two key ideas. The first was to identify precisely which patients would benefit most from immunotherapy to receive it right away, rather than as a second-line or third-line therapy.
Imtiaz Hussain takes a selfie with his MSK medical oncologist, Andrea Cercek. He says that when he was told the immunotherapy was successful, “the first thing I did was call my mom. We both cried.”. (CREDIT: Imtiaz Hussain)
Diaz’s previous research had already shown that checkpoint inhibitors could help people with MMRd colorectal tumors that have spread. The second idea was to avoid the toxicity often associated with treatment for rectal cancer, such as life-altering bowel and bladder dysfunction, incontinence, infertility, sexual dysfunction, and more.
According to Dr. Cercek, the standard treatment for rectal cancer with surgery, radiation, and chemotherapy can be particularly hard on patients due to the location of the tumor. The clinical trial’s aim was to avoid these toxicities, and Dr. Cercek proposed using immunotherapy as a first-line treatment to shrink the tumor, enabling a more successful surgery. Dr. Diaz said, “I think this is a great step forward for patients.”
Rectal cancer is one of the most common cancers in the United States, with an estimated 45,000 Americans diagnosed each year. The current standard of care is surgery, radiation, and chemotherapy. However, these treatments can cause significant side effects and can be ineffective in some patients.
The findings of this clinical trial represent a significant breakthrough in the treatment of rectal cancer and pave the way for future research on the use of immunotherapy as a first-line treatment.
Four people who were successfully treated for rectal cancer in a clinical trial at Memorial Sloan Kettering. (Left to right) Sascha Roth, Dr. Luis Diaz, Imtiaz Hussain, Dr. Andrea Cercek, Avery Holmes and Nisha Varughese. (CREDIT: Memorial Sloan Kettering Cancer Center)
The clinical trial is ongoing, and MSK is currently recruiting new patients to join the study, with the aim of expanding their knowledge of the potential of immunotherapy as a first-line treatment for rectal cancer.
The trial has already generated significant interest in the medical community, with experts hailing it as a groundbreaking development in the fight against cancer.
Sascha Roth, the first patient in the trial, unexpectedly learned she had rectal cancer in 2019. (CREDIT: Shuran Huang)
Dr. Scott Kopetz, a gastrointestinal medical oncologist at The University of Texas MD Anderson Cancer Center, said the results of the MSK clinical trial were “exciting and game-changing.”
“This is a potentially transformative approach to rectal cancer that could provide patients with better outcomes and avoid the side effects of traditional treatments,” Dr. Kopetz said.
Dr. Kopetz noted that the MSK study was the first to demonstrate that immunotherapy alone could eradicate rectal cancer in patients with MMRd tumors. He said the findings could also have implications for the treatment of other types of cancer.
“This study shows that immunotherapy can be used earlier in the treatment course, and that it can have a significant impact on the disease,” Dr. Kopetz said. “It also highlights the importance of identifying specific genetic mutations in patients, which can help guide treatment decisions and improve outcomes.”
Dr. Cercek and Dr. Diaz said they were encouraged by the early results of the clinical trial, but cautioned that more research was needed to confirm the effectiveness of immunotherapy as a first-line treatment for rectal cancer.
They said they were continuing to monitor the patients in the study to ensure that the cancer did not return, and to assess the long-term effects of the treatment.
“We’re still in the early stages of this research, and we need to be cautious about drawing conclusions,” Dr. Cercek said. “But we’re hopeful that this approach could provide a new treatment option for patients with rectal cancer.”
For patients like Sascha Roth, the results of the MSK clinical trial have been life-changing. Sascha said she was thrilled to have been part of the study and to have had access to cutting-edge treatments.
“I’m so grateful to the doctors and researchers at MSK who have worked tirelessly to develop new treatments and improve outcomes for patients with cancer,” Sascha said. “This clinical trial has given me hope and a new lease on life.”
Sascha said she was now cancer-free and looking forward to returning to her normal routine, including her job as a freelance writer.
“I’m so happy to be healthy again and to be able to focus on the things that matter most to me,” Sascha said. “I’m excited to see what the future holds and to continue living life to the fullest.”
The MSK clinical trial is just one of many ongoing studies exploring the potential of immunotherapy as a treatment for cancer. As researchers continue to make progress in this area, there is growing optimism that this approach could revolutionize cancer treatment and offer hope to millions of patients around the world.
For more science news stories check out our New Innovations section at The Brighter Side of News.
Note: Materials provided above by The Brighter Side of News. Content may be edited for style and length.
Like these kind of feel good stories? Get the Brighter Side of News' newsletter.