(Press-News.org) Researchers from Memorial Sloan Kettering Cancer Center (MSK) have announced results from the first published basket study, a new form of clinical trial design that explores responses to drugs based on the specific mutations in patients' tumors rather than where their cancer originated.
Published in the New England Journal of Medicine, the early phase II study, led by MSK Physician-in-Chief and Chief Medical Officer José Baselga, MD, PhD, looked at the effect of vemurafenib (Zelboraf®) in multiple nonmelanoma BRAFV600-mutated cancers in 122 patients from 23 centers around the world. Vemurafenib previously has been proven to treat BRAFV600-mutated melanoma. People with lung, colorectal, and ovarian cancers were among those included in the study as well as people with rare diseases, such as Erdheim-Chester disease. Until this point, the efficacy of vemurafenib in nonmelanoma cancers has remained unexplored despite significant therapeutic potential.
"This study is the first deliverable of precision medicine. We have proven that histology-independent, biomarker-selected basket studies are feasible and can serve as a tool for developing molecularly targeted cancer therapy," said Dr. Baselga, the study's senior author. "While we can -- and should -- be cautiously optimistic, this is what the future of precision medicine looks like."
Basket studies permit the detection of early signals of activity across multiple tumor types simultaneously, while allowing for the possibility that tumor lineage might influence drug sensitivity. The first to follow this model, this new study aims to explore treatment responses among tumors based on their mutation types and to identify promising signals of activity in individual tumor types that could be pursued in subsequent studies. The results will ultimately guide researchers in looking for different drug targets or developing therapies that combine vemurafenib with complementary treatments.
Basket studies also have the ability to greatly increase the number of patients eligible to receive certain drugs. The mixed efficacy seen in this study proves that drugs can reach patients beyond the current approved use but, expectedly, do not work for everyone. As a pioneering trial, this data demonstrates the promising benefits of basket studies and the need for more work to be done with these types of trials.
The findings illustrate the preliminary clinical efficacy of vemurafenib in multiple nonmelanoma BRAFV600-mutated cancers. Of the 122 trial participants, clinical activity was observed in various tumor types. Preliminary vemurafenib activity was observed in non-small cell lung cancer as well as Erdheim-Chester disease and Langherhans cell histiocytosis. Response rate and median progression-free survival in non-small cell lung cancer was 42 percent and 7.3 months, respectively. In Erdheim-Chester disease and Langherhans cell histiocytosis, response rate was 43 percent; despite median treatment duration of 5.9 months, no patients progressed during therapy. Anecdotal responses were seen in anaplastic pleomorphic xanthoastrocytoma, anaplastic thyroid cancer, cholangiocarcinoma, salivary duct cancer, ovarian cancer, clear cell sarcoma, and colorectal cancer (cetuximab combination only).
"This kind of study is a beneficial way to do rare tumor research because it allows us to open the study to patients with diseases that are completely underrepresented in clinical trials in general, such as anaplastic thyroid cancer and glioblastoma," said David Hyman, MD, the study's first author and Acting Director of Developmental Therapeutics at MSK. "By broadening eligibility, we are translating potential benefits to as large a patient population as possible."
This clinical trial is the first in an impending wave of such studies focused on cancer-related mutations identified through the huge amounts of genomic data generated in recent years. It highlights the importance of further investigation into precision medicine, a promising area that has recently received attention from President Obama and the National Cancer Institute, among others. Last May, MSK launched an initiative in this space -- the Marie-Josée and Henry R. Kravis Center for Molecular Oncology -- that works to transform cancer care through genomic analysis of patient-derived tumors. Currently the center is analyzing 410 of the most important cancer genes in thousands of patients.
"We now have the landscape of all the most frequent cancer-causing mutations in the majority of tumor types, and we know there are a number of genes that are frequently mutated in some tumors and less frequently in others," explained Dr. Baselga. "The next step is exploring appropriate drug combinations, knowing that these cells have a finite number of pathways."
Full findings from the study can be found in the August 20 issue of the New England Journal of Medicine.
INFORMATION:
About Memorial Sloan Kettering Cancer Center
We are the world's oldest and largest private cancer center, home to more than 14,000 physicians, scientists, nurses, and staff united by a relentless dedication to conquering cancer. As an independent institution, we combine 130 years of research and clinical leadership with the freedom to provide highly individualized, exceptional care to each patient. And our always-evolving educational programs continue to train new leaders in the field, here and around the world. For more information, go to http://www.mskcc.org.
HOUSTON - (Aug. 20, 2015) - Texans without health insurance are twice as likely to skip seeking primary and mental health care because of cost. That's one of the findings of a new survey released today by Rice University's Baker Institute for Public Policy and the Episcopal Health Foundation.
The report found that in the past year 32 percent of uninsured adult Texans said they had skipped primary care due to costs, compared with 16 percent of adults who have health insurance. When it comes to mental health care or counseling, 12 percent of uninsured Texans said they had ...
NASA's CloudSat Satellite passed over Super Typhoon Atsani as it moved through the western North Pacific Ocean. CloudSat looked at the super typhoon from the side, revealing heavy rainfall in a sloping eyewall.
Typhoon Atsani strengthened into a super typhoon on August 19, 2015 at 0000 UTC. CloudSat flew over Atsani at 03:27 UTC, shortly after it became a super typhoon when maximum sustained winds were near 130 knots (150 mph). Atsani was equivalent to a category 4 strength hurricane.
CloudSat's cloud profiling radar (CPR), passed just to the west of Super Typhoon Atsani's ...
In the 1930s, Irving Langmuir and his colleague Katharine Blodgett were working long days in the General Electric Company's research laboratory. Together, they discovered that by spreading molecules with volatile organic solvents on the surface of water, they could create a one-molecule-thick film and use it as an anti-reflective coating for glass. Later named Langmuir-Blodgett assembly, this thin-film fabrication technique became popular for creating molecule or nanoparticle monolayers and is commonly used until this day.
Since Langmuir-Blodgett assembly was first reported ...
Reston, Va. (August 20, 2015) - A molecular imaging biomarker is able to detect fast-growing primary prostate cancer and distinguish it from benign prostate lesions, addressing an unmet clinical need. The new research, published in the July 2015 issue of The Journal of Nuclear Medicine, is significant for patients with suspected prostate cancer that has not been confirmed by standard biopsy.
"We were able to demonstrate in our research that PSMA PET imaging was more specific than MR imaging for detection of clinically significant high-grade prostate cancer lesions, and ...
WASHINGTON, D.C., August 20 -- A first-of-its-kind, nationally representative study of siblings supports previously published research on unrelated individuals that links specific genotypes to educational attainment among adults in their mid-20s to early 30s. The research, published today in AERA Open, a peer-reviewed journal of the American Educational Research Association, found that, within families, an adolescent with a higher "polygenic score"--which summarizes previously identified genome-wide associations for educational attainment--than her or his sibling tended ...
Summary: The ideal management strategy for primary cardiogenic shock is a matter of debate. After some early discouraging experiences, the use of extracorporeal life support for patients with cardiogenic shock is having a resurgence. A report from researchers in Padua, Italy finds that patients who have an acute onset of cardiogenic shock, for example following a heart attack, and are placed on extracorporeal life support, fare better than those who have a chronic cardiac pathology. In an accompanying editorial, Dr. Vivek Rao of the University of Toronto puts the findings ...
Virginia Tech researchers have identified a biomarker in pre-diabetic individuals that could help prevent them from developing Type II diabetes.
Publishing in Clinical Epigenetics, the researchers discovered that pre-diabetic people who were considered to be insulin resistant -- unable to respond to the hormone insulin effectively -- also had altered mitochondrial DNA.
Researchers made the connection by analyzing blood samples taken from 40 participants enrolled in the diaBEAT-it program, a long-term study run by multiple researchers in the Fralin Translational Obesity ...
(BOSTON) - Last year, a Wyss Institute team of scientists described the development of a new device to treat sepsis that works by mimicking our spleen. It cleanses pathogens and toxins from blood circulating through a dialysis-like circuit. Now, the Wyss Institute team has developed an improved device that synergizes with conventional antibiotic therapies and that has been streamlined to better position it for near-term translation to the clinic. The improved design is described in the October volume 67 of Biomaterials.
Sepsis is a common and frequently fatal medical ...
Having a baby at home can save thousands of dollars over a hospital birth and is just as safe for low-risk births, according to a new UBC study.
Researchers with UBC's School of Population and Public Health and the Child and Family Research Institute looked at all planned home births attended by registered midwives in B.C. between 2001 and 2004. They compared them to planned hospital births attended by registered midwives or physicians in which the mothers met the criteria for home birth.
For the first 28 days postpartum, they found planned home births saved an average ...
Amsterdam, August 20, 2015 - A new study published in Journal of Transport & Health reveals that middle-aged drivers are at higher risk of crashes because they use their cellphone regularly while driving. The research reveals that most drivers admit to using their cellphones regularly while driving, even with children in the car; drivers also feel pressured to answer work calls while driving.
The authors of the study, from the University of California San Diego, are now working with companies to teach employees about the risks associated with distracted driving, and show ...
