Lurie Comprehensive Cancer Center

Chicago, IL, United States

Lurie Comprehensive Cancer Center

Chicago, IL, United States
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News Article | May 11, 2017
Site: www.eurekalert.org

Northwestern took drug from discovery to FDA approval of investigational drug for use in a clinical trial for fatal disease CHICAGO --- The first drug using spherical nucleic acids to be systemically given to humans has been developed by Northwestern University scientists and approved by the Food and Drug Administration as an investigational new drug for an early-stage clinical trial in the deadly brain cancer glioblastoma multiforme. A clinical trial has just been launched at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and Northwestern Medicine. The new drug is able to cross the challenging blood-brain barrier to reach tumors in animals, where it turns down a critical cancer-causing gene. Now, the Phase 0 clinical trial will investigate the drug's ability to reach tumors in humans. The glioblastoma drug represents a revolutionary new class of drugs. The novel spherical nucleic acid platform it is based on can be applied to other types of neurological diseases, such as Alzheimer's and Parkinson's, by similarly turning down the genes that lead to those diseases. It's highly unusual for a drug to be developed in preclinical research at a university, shepherded through FDA approval as an investigational new drug and studied in a clinical trial -- all within the same university and without funding from a pharmaceutical company. In most cases, a drug is developed and licensed to a pharmaceutical company. "We want to get the drug to patients as quickly as possible," said Jay Walsh, vice president for research at Northwestern. "We want to move the drug forward because there are patients with a disease with no current cure." The drug, called NU-0129, will have to go through many more phases before it is approved for general use in a clinical environment. "We know this drug works in mice. Now we need to know if it can cross the human blood-brain barrier and accumulate in the tumor of a human being," said Alexander Stegh, an assistant professor of neurology at Northwestern University Feinberg School of Medicine. Stegh developed the drug with Chad A. Mirkin, the George B. Rathmann Professor of Chemistry at the Weinberg College of Arts and Sciences and director of Northwestern's International Institute for Nanotechnology (IIN). "If the spherical nucleic acids cross the barrier and localize in the brain, the implications go beyond glioblastoma," Mirkin said. "This would give us the ability to target diseases of the brain by targeting pathways that we know are associated with different diseases, including Huntington's, Parkinson's and Alzheimer's diseases." The drug, which consists of short snippets of RNA densely arranged on the surface of spherical gold nanoparticles, changes the genetic makeup of the tumor cells and dampens their ability to divide. The drug targets the gene BCL2L12, which is involved in apoptosis, or programmed cell death. Mirkin and his group invented spherical nucleic acids, and Stegh identified the gene to target. Glioblastoma patients are treated with chemotherapy, radiation and surgery, but there is no cure. The life expectancy for the estimated 30,000 people in the U.S. who are diagnosed with the aggressive brain cancer each year is 14 to 16 months. "We desperately need an effective treatment for this deadly disease," said Dr. Priya Kumthekar, an assistant professor of neurology and hematology-oncology at Feinberg and a Northwestern Medicine neuro-oncologist. Kumthekar is the lead investigator of the Northwestern trial, which will recruit six to eight glioblastoma patients. The study will recruit individuals who have had regrowth of their tumors and are candidates for tumor removal. They will receive the drug intravenously prior to surgery. Following tumor removal, Kumthekar and her team will study the tumor's drug content to determine how well the drug crossed the blood-brain barrier in patients. "Based on animal studies, there is good penetration into the tumor," Kumthekar said. "We predict that will be the case here, too." "Northwestern is very focused on delivering better therapy to patients," said Dr. Frank Giles, who serves as Northwestern University's sponsor representative on the investigational new drug application and clinical study. "Applying new scientific knowledge is the best way to develop truly paradigm-changing novel therapies." Kumthekar, Stegh, Mirkin and Giles are members of the Lurie Cancer Center. "This was a complex, meticulous process requiring intense collaboration across many facets and groups within the University and Northwestern Medicine," noted Ann Adams, associate vice president for research at Northwestern and one of the quarterbacks who led the FDA application process with Giles and Caroline Ko, a research associate in the department of chemistry and chief scientific officer of the IIN. Dr. Leon Platanias, director of the Lurie Cancer Center, said, "The technology of spherical nucleic acids used in this first-in-human study is very powerful and may result in new, more effective approaches for the treatment of brain tumors and possibly other tumors in the future." The birth of the drug: spherical nanoparticles meet genetics Stegh and Mirkin have been collaborating since 2009 to tackle the difficult problem of developing better treatments for glioblastoma. They first came together through Northwestern's Center of Cancer Nanotechnology Excellence, funded by the National Cancer Institute. Mirkin had the perfect tool: spherical nucleic acids, new globular forms of DNA and RNA -- rather than linear -- which are nontoxic to humans. The nucleic acid sequence is designed to match the target gene. Stegh had the gene: in 2007, he and colleagues identified the gene BCL2L12 as one that is overexpressed in glioblastoma tumors and related to glioblastoma's resistance to conventional therapies. The therapeutic, because of its novel structure, is actively taken across the blood-brain barrier, allowing it to reach the brain tumor. Once inside cancer cells, the drug silences the disease-causing gene. The RNA's programmed sequence acts as a dimmer switch, selectively impeding the cells from producing specific proteins related to the disease. The result is selective cancer cell death and reduced cell growth. Key to the drug's success is the nanostructure's 3-D spherical shape and nucleic acid density. Normal (linear) nucleic acids cannot get into cells or cross the blood-brain barrier, but these spherical nucleic acids can. Strands of RNA attach to and surround a gold nanoparticle like a shell; the nucleic acids are densely packed and form a tiny sphere. The gold nanoparticle core is only 13 nanometers in diameter. In 2013, Mirkin and Stegh were the senior co-authors of an animal study published in Science Translational Medicine. Their research team was the first to demonstrate delivery of a drug by intravenous injection that turns off a critical cancer-causing gene in glioblastoma, reducing tumor progression and increasing survival rates significantly in mice with the deadly disease. In mice with glioblastoma, approximately 1 percent of the injected dose accumulated in the intracranial brain tumor tissue. Interestingly, once in the brain, the spherical nucleic acids selectively accumulated in tumor tissue over normal brain tissue. This is likely due to the ability of nanoparticles to accumulate in tumor tissue because of leaky blood vessels created by the tumor. The survival rate of the animals increased nearly 20 percent, and tumor size was reduced three to four fold, as compared to the control group. Mirkin first developed the nanostructure platform used in this study at Northwestern in 1996. This new development targeting glioblastoma was the first realization that the nanostructures injected into an animal naturally find their target in the brain and can deliver an effective payload of therapeutics. Mirkin also is a professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering. Editor's note: Northwestern University and some of the researchers involved in the development of the drug have financial interests relative to the drug used in this research study. As a result of these interests, Northwestern University and/or some researchers could ultimately benefit financially from the outcomes of this research.


"NCCN ORP congratulates Dr. Gomez and Dr. Jänne on their awards and looks forward to the commencement of their studies," said Susan Most, RN, MBA, Director, Clinical Operations, NCCN ORP. "We are pleased to be working with AstraZeneca on further evaluation of osimertinib, an important targeted agent for the treatment of EGFRm+ non-small cell lung cancer. This project furthers NCCN's commitment to improving the lives of patients with cancer by providing opportunities to identify additional treatment options in lung cancer." Submissions were peer reviewed by the NCCN Osimertinib Scientific Review Committee. The funded concepts were selected based on several criteria, including scientific merit, existing data, and the types of studies necessary to further evaluate the efficacy of osimertinib. NCCN ORP draws upon the expertise of investigators at the NCCN Member Institutions and their affiliates to facilitate all phases of clinical research. This research is made possible by collaborations with pharmaceutical and biotechnology companies in order to advance therapeutic options for patients with cancer. To date, this successful research model has received more than $60 million in research grants and supported more than 140 studies, producing a number of publications in peer-reviewed journals. To learn more about the NCCN ORP and ongoing clinical trials, visit NCCN.org/ORP. The National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 27 leading cancer centers devoted to patient care, research, and education, is dedicated to improving the quality, effectiveness, and efficiency of cancer care so that patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. As the arbiter of high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers. The NCCN Member Institutions are: Fred & Pamela Buffett Cancer Center, Omaha, NE; Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; City of Hope Comprehensive Cancer Center, Los Angeles, CA; Dana-Farber/Brigham and Women's Cancer Center | Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA; Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT; Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Mayo Clinic Cancer Center, Phoenix/Scottsdale, AZ, Jacksonville, FL, and Rochester, MN; Memorial Sloan Kettering Cancer Center, New York, NY; Moffitt Cancer Center, Tampa, FL; The Ohio State University Comprehensive Cancer Center - James Cancer Hospital and Solove Research Institute, Columbus, OH; Roswell Park Cancer Institute, Buffalo, NY; Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO; St. Jude Children's Research Hospital/The University of Tennessee Health Science Center, Memphis, TN; Stanford Cancer Institute, Stanford, CA; University of Alabama at Birmingham Comprehensive Cancer Center, Birmingham, AL; UC San Diego Moores Cancer Center, La Jolla, CA; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA; University of Colorado Cancer Center, Aurora, CO; University of Michigan Comprehensive Cancer Center, Ann Arbor, MI; The University of Texas MD Anderson Cancer Center, Houston, TX; University of Wisconsin Carbone Cancer Center, Madison, WI; Vanderbilt-Ingram Cancer Center, Nashville, TN; and Yale Cancer Center/Smilow Cancer Hospital, New Haven, CT. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/nccn-awards-grants-to-investigators-at-member-institutions-to-study-osimertinib-in-lung-cancer-300463998.html


CHICAGO, May 11, 2017 (GLOBE NEWSWIRE) -- Tempus, a technology company focused on helping doctors personalize cancer care by collecting, sorting and analyzing clinical and molecular data and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University announce a second collaboration aimed at personalizing care for cancer patients. As part of the newest collaboration, Tempus will provide molecular sequencing and analysis for patient derived xenograft (PDX) models and patient derived organoid models, which will allow researchers to better understand how representative in-vivo modeling systems are of a patient’s actual biology.  By sequencing and analyzing large cohorts of these patient derived biological models, researchers and physicians should be able to better predict how a patient’s cancer will progress and which specific clinical treatments may be more likely to lead to positive responses. “Oftentimes, researchers and physicians working with PDX and organoid models are starting from an imperfect place,” said Leonidas Platanias, MD, PhD, Director of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. “Obtaining molecular information on the models increases the utility to researchers and physicians who will be able to better understand how representative they are of individual patients.” “Northwestern’s Lurie Cancer Center is one of the country’s leading academic medical centers and we are thrilled to have been chosen to help them bring cutting edge technology to advance both their clinical and research efforts,” said Eric Lefkofsky, co-founder and CEO of Tempus. “We are eager to strengthen our partnership by combining our advanced modeling infrastructure and the hospital’s deep expertise to advance treatment for patients battling this disease.” Tempus currently serves as a preferred partner to handle genomic sequencing and analysis as part of Lurie Cancer Center’s OncoSET initiative. Lurie Cancer Center launched the OncoSET (Sequence, Evaluate, Treat) Program to provide personalized care and customized treatments for patients with cancer.   Lurie Cancer Center is one of only 47 Comprehensive Cancer Centers in the U.S. designated by the National Cancer Institute. Tempus provides molecular sequencing and clinical analytic solutions for top academic centers, hospital systems, associations, and healthcare providers. About Tempus: Tempus is a technology company that is building the world’s largest library of molecular and clinical data and an operating system to make that data accessible and useful. We enable physicians to deliver personalized cancer care for patients through our interactive analytical and machine learning platform. We provide genomic sequencing services and analyze molecular and therapeutic data to empower physicians to make real-time, data-driven decisions. Our goal is for each patient to benefit from the treatment of others who came before by providing physicians with tools that learn as we gather more data. For more information, visit tempus.com and follow us on Facebook (Tempus Labs) and Twitter (@TempusLabs). For more information on Eric Lefkofsky, visit lefkofsky.com. About Lurie Cancer Center: The Robert H. Lurie Comprehensive Cancer Center of Northwestern University, one of only 47 National Cancer Institute-designated Comprehensive Cancer Centers in the country, is committed to being a national leader in the battle to overcome cancer. To this end, the Lurie Cancer Center is dedicated to scientific discovery, advancing medical knowledge, providing compassionate, state-of-the-art cancer care, and training the next generation of clinicians and scientists.  For more information, visit cancer.northwestern.edu.


"NCCN Foundation is proud to support this important resource which indeed will empower patients with thyroid cancer and their caregivers to make informed choices about their care," said Marcie R. Reeder, MPH, Executive Director, NCCN Foundation. "We are incredibly grateful for the generous support from our sponsors, ThyCa and Rockin' for the Cure, as their sponsorship for these resources gives patients access to the same treatment information that their doctors use." "Thyroid Cancer is challenging because it affects people of all ages from young children through seniors, is especially life-disrupting and stressful when diagnosed in younger patients, and needs lifelong monitoring and management even in low-risk patients," said Gary Bloom, ThyCa Executive Director, and Thyroid Cancer survivor of 21 years. "ThyCa is very pleased to support these important guidelines, which will be helpful resources for both patients and their caregivers." NCCN Guidelines for Patients are easy-to-understand adaptations based on the same clinical practice guidelines used by health care professionals around the world to determine the best way to treat a person with cancer. Each resource features unbiased expert guidance from the nation's leading cancer centers designed to help people living with cancer understand and discuss their treatment options with their providers. NCCN Guidelines for Patients and NCCN Quick Guide™ sheets—one-page summaries of key points in the patient guidelines—are written in plain language and include patient-friendly tools, such as questions to ask your doctor, a glossary of terms, and medical illustrations of anatomy, tests, and treatment.  NCCN Guidelines for Patients and NCCN Quick Guide™ sheets DO NOT replace the expertise and clinical judgment of the clinician. NCCN currently offers NCCN Guidelines for Patients for the following: Brain, Breast, Colon Distress, Esophageal, Kidney, Non-Small Cell Lung, Ovarian, Pancreatic, Prostate, Stomach, and Thyroid Cancers; Acute Lymphoblastic Leukemia; Adolescents and Young Adults with Cancer; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Hodgkin Lymphoma; Lung Cancer Screening; Malignant Pleural Mesothelioma; Melanoma; Multiple Myeloma; Myelodysplastic Syndromes; Nausea and Vomiting; Non-Hodgkin's Lymphomas; Soft Tissue Sarcoma; and Waldenström's Macroglobulinemia. The NCCN Guidelines for Patients and NCCN Quick Guide™ sheet for Thyroid Cancer are available to download for free at NCCN.org/patients and on the NCCN Patient Guides for Cancer mobile app. NCCN Foundation® was founded by the National Comprehensive Cancer Network® (NCCN®) to empower people with cancer and advance oncology innovation. NCCN Foundation supports people with cancer and their caregivers at every step of their treatment journey by delivering unbiased expert guidance from the world's leading cancer experts through the library of NCCN Guidelines for Patients® and other patient education resources. NCCN Foundation is also committed to advancing cancer treatment by funding the nation's promising young investigators at the forefront of cancer research, initiating momentum in their careers and furthering the betterment of patients through their groundbreaking innovations. For more information about NCCN Foundation, visit NCCNFoundation.org. ThyCa: Thyroid Cancer Survivors' Association, Inc., an international nonprofit organization founded in 1995 and advised by thyroid cancer specialists, educates and supports patients and families through its website, online and face-to-face support groups, one-to-one support, over 50 videos with experts on its YouTube Channel, handbooks on all thyroid cancer types, downloadable low-iodine cookbook, online newsletter, and materials in 10 languages. ThyCa sponsors seminars, workshops, and the annual International Thyroid Cancer Survivors' Conference, as well as Thyroid Cancer Awareness Month, year-round awareness programs for early detection, and thyroid cancer research funds and research grants. For more information visit http://www.thyca.org. About the National Comprehensive Cancer Network The National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 27 leading cancer centers devoted to patient care, research, and education, is dedicated to improving the quality, effectiveness, and efficiency of cancer care so that patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. As the arbiter of high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers. The NCCN Member Institutions are: Fred & Pamela Buffett Cancer Center, Omaha, NE; Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; City of Hope Comprehensive Cancer Center, Los Angeles, CA; Dana-Farber/Brigham and Women's Cancer Center | Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA; Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT; Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Mayo Clinic Cancer Center, Phoenix/Scottsdale, AZ, Jacksonville, FL, and Rochester, MN; Memorial Sloan Kettering Cancer Center, New York, NY; Moffitt Cancer Center, Tampa, FL; The Ohio State University Comprehensive Cancer Center - James Cancer Hospital and Solove Research Institute, Columbus, OH; Roswell Park Cancer Institute, Buffalo, NY; Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO; St. Jude Children's Research Hospital/The University of Tennessee Health Science Center, Memphis, TN; Stanford Cancer Institute, Stanford, CA; University of Alabama at Birmingham Comprehensive Cancer Center, Birmingham, AL; UC San Diego Moores Cancer Center, La Jolla, CA; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA; University of Colorado Cancer Center, Aurora, CO; University of Michigan Comprehensive Cancer Center, Ann Arbor, MI; The University of Texas MD Anderson Cancer Center, Houston, TX; University of Wisconsin Carbone Cancer Center, Madison, WI; Vanderbilt-Ingram Cancer Center, Nashville, TN; and Yale Cancer Center/Smilow Cancer Hospital, New Haven, CT. [1] Robert I. Haddad, MD, et. al., NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Thyroid Carcinoma, Version 1.2017. © 2017 National Comprehensive Cancer Network, Inc.  Available at NCCN.org. Accessed: May 8, 2017 [2] "Cancer Stat Facts: Thyroid Cancer." Surveillance, Epidemiology, and End Results Program. National Cancer Institute, n.d. Web. 08 May 2017. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/nccn-publishes-new-patient-education-resources-for-thyroid-cancer----one-of-the-most-commonly-diagnosed-cancers-in-women-in-the-united-states-300456776.html

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