[Apr.10, 2023: Staff Writer, The Brighter Side of News]
Four people who were successfully treated for rectal cancer in a clinical trial at Memorial Sloan Kettering. (L to R) Sascha Roth, Dr Luis Diaz, Imtiaz Hussain, Dr Andrea Cercek, Avery Holmes and Nisha Varughese. (CREDIT: Memorial Sloan Kettering Cancer Center)
Immunotherapy alone was successful in defeating rectal cancer in a clinical trial involving 14 patients. This groundbreaking research is the first to determine whether immunotherapy can defeat rectal cancer that has not spread to other tissues in a subset of patients whose tumor contains a specific genetic mutation.
The clinical trial, conducted by Memorial Sloan Kettering Cancer Center (MSK) in New York, achieved a 100% remission rate, with all patients experiencing complete cancer clearance following immunotherapy. The cancer did not come back in any of the patients who had been cancer-free for at least two years.
This trial is important because it paves the way for the use of immunotherapy as first-line treatment for certain types of rectal cancer, potentially sparing patients the debilitating side effects of standard radiation, surgery or chemotherapy treatments. The findings were published in The New England Journal of Medicine.
Patients in the trial had tumors with a specific genetic makeup known as mismatch repair defect (MMRd) or microsatellite instability (MSI). MMRd tumors develop a defect in their ability to repair certain types of mutations that occur in cells. When these mutations accumulate in the tumor, they stimulate the immune system, which attacks the cancer cells carrying the mutations.
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 patient received other standard treatments for rectal cancer, such as radiation therapy or surgery.
The results were significant, with all 14 patients in the trial achieving complete remission. The study’s principal investigator, MSK medical oncologist Andrea Cercek, said, “It’s incredibly gratifying to receive these tears of joy and emails of joy from the patients in this study who are completing treatment and realizing “Oh my God, I can keep all of my normal body functions that I was worried about losing due to radiation or surgery.
MSK medical oncologist Luis Diaz, Jr., the co-investigator, explained that the research was triggered by two key ideas. The first was to identify precisely which patients would benefit the most from immunotherapy to receive it right away, rather than as a second or third line treatment.
Sascha Roth, the trial’s first patient, unexpectedly learned she had rectal cancer in 2019. (CREDIT: Shuran Huang)
Diaz’s previous research has already shown that checkpoint inhibitors can help people with MMRd colorectal tumors that have spread. The second idea was to avoid the toxicity often associated with rectal cancer treatment, such as bowel and bladder dysfunction, incontinence, infertility, sexual dysfunction, etc.
According to Dr. Cercek, the standard treatment for rectal cancer with surgery, radiation therapy and chemotherapy can be particularly challenging for patients due to the location of the tumor. The goal of the clinical trial was to avoid these toxicities, and Dr. Cercek proposed using immunotherapy as a first-line treatment to shrink the tumor, allowing for more efficient surgery. Dr Diaz said: “I think this is a big step forward for patients.”
Rectal cancer is one of the most common cancers in the United States, with approximately 45,000 Americans diagnosed each year. The current standard of care is surgery, radiation therapy and chemotherapy. However, these treatments can cause significant side effects and may be ineffective in some patients.
The results of this clinical trial represent a significant breakthrough in the treatment of rectal cancer and pave the way for future research into the use of immunotherapy as a first-line treatment.
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.
Imtiaz Hussain takes a selfie with his MSK medical oncologist, Andrea Cercek. He says that when he was told the immunotherapy had worked, “the first thing I did was call my mom. We both cried. (CREDIT: Imtiaz Hussain)
The trial has already generated considerable interest in the medical community, with experts hailing it as a breakthrough development in the fight against cancer.
Dr. Scott Kopetz, a gastrointestinal medical oncologist at the University of Texas MD Anderson Cancer Center, said the results from the MSK clinical trial were “exciting and game-changing”.
“This is a potentially transformative approach to rectal cancer that could offer patients better outcomes and avoid the side effects of traditional treatments,” said Dr. Kopetz.
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 treatment and can have a significant impact on disease,” said Dr. Kopetz. “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 initial results of the clinical trial, but warned that further research was needed to confirm the effectiveness of immunotherapy as a first-line treatment for rectal cancer .
They said they continue to monitor study patients to make sure the cancer does not come back and to assess the long-term effects of the treatment.
“We are still in the early stages of this research, and we need to be careful about jumping to conclusions,” Dr. Cercek said. “But we hope 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 were life changing. Sascha said she was delighted to have participated in the study and to have had access to cutting-edge treatments.
“I am very grateful to the physicians and researchers at MSK who have worked tirelessly to develop new treatments and improve outcomes for cancer patients,” said Sascha. “This clinical trial has given me hope and a new breath of life.”
Sascha said she is now cancer free and looking forward to getting back to her normal routine, including her work 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 to live life to the fullest.”
The MSK clinical trial is just one of many ongoing studies exploring the potential of immunotherapy as a cancer treatment. 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 scientific news, see our New Innovations section on The bright side of the news.
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