• IU School of Medicine names first Walther Scholar in Psycho-Oncology

    IU School of Medicine names first Walther Scholar in Psycho-Oncology

    INDIANAPOLIS — Indiana University School of Medicine has named Shelley Johns, PsyD, ABPP, the Walther Scholar in Psycho-Oncology.

    Johns is the first person to hold this position, which was established by the transformative $14 million gift to IU School of Medicine from the Walther Cancer Foundation to create five endowed positions to develop a supportive oncology program that encompasses research and patient care. Supportive oncology goes beyond standard therapies such as surgery, chemotherapy and radiation and seeks to care for a patient’s overall physical, mental and spiritual well-being.

    The program intends to influence care for cancer patients and their families throughout Indiana and the country by providing expertise and best practices for other health systems to model, with particular attention to the underserved.

    “Thanks to the generous support of the Walther Cancer Foundation, I have the opportunity to explore new ideas to address problems that are most important to people whose lives are disrupted by cancer,” Johns said. “I want to capitalize on the synergy that I already see and feel between clinical practice and research so we can develop studies that are informed by people with cancer and then implement our research findings in clinical practice.”

    Johns is a nationally-recognized, board-certified, clinical health psychologist, an associate professor at IU School of Medicine in the Division of General Internal Medicine and Geriatrics, an IU Simon Comprehensive Cancer Center researcher and a research scientist at the William M. Tierney Center for Health Services Research at the Regenstrief Institute. Her research focuses on developing and testing mind-body interventions to improve the physical health and psychological well-being of adults with cancer. She currently holds a $2.6 million grant from the National Cancer Institute to lead a clinical trial to support breast cancer survivors struggling with fear of cancer recurrence.

    As the Walther Scholar in Psycho-Oncology, Johns will develop programs in psycho-oncology within the newly established Supportive Oncology Center of Excellence at the IU Simon Comprehensive Cancer Center.

    “We are very fortunate to recruit Dr. Johns to the newly established Supportive Oncology Center of Excellence in the cancer center, which will be the hub for our cutting-edge efforts in research, education and clinical care in supporting the important needs of our cancer patients and their families,” Kelvin Lee, MD, cancer center director, said. “Dr. Johns’ exceptional work in psycho-oncology will be a major pillar of the center.”

    “Dr. Johns is a great addition to improving the total care of patients living with cancer who we see at the cancer center. Shelley’s work will be critical to the Supportive Oncology Center of Excellence in improving clinical care, research and education,” James Cleary, MD, professor of medicine and Walther Senior Chair in Supportive Oncology at IU School of Medicine, said. Cleary was recruited in 2018 to IU as part of the Walther gift, which was believed to be the largest gift in the country to support a program of this kind.

    “Dr. Johns does very rigorous and sophisticated research aimed at providing the evidence base for treating highly prevalent and challenging symptoms in patients with cancer,” said Greg Sachs, MD, director of the Division of General Internal Medicine and Geriatrics. “She also represents another critical connection between our palliative care program and the cancer center.”

    “Shelley Johns is a highly talented and insightful researcher. The Walther Cancer Foundation has provided support for certain aspects of Dr. John’s work for more than ten years. We are truly pleased that Shelley has been selected as the first Walther Scholar in Psycho-Oncology,” said Tom Grein, president and CEO of the Walther Cancer Foundation.

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    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

  • Vera Bradley Foundation for Breast Cancer makes $12.5 million commitment to Indiana University School of Medicine

    Vera Bradley Foundation for Breast Cancer makes $12.5 million commitment to Indiana University School of Medicine

    INDIANAPOLIS — A gift commitment of $12.5 million to Indiana University School of Medicine from the Vera Bradley Foundation for Breast Cancer will support new research to harness immunotherapy for breast cancer treatment.

    This gift from the Vera Bradley Foundation brings the non-profit’s total commitments in breast cancer research funding since 2000 to $50 million. The Foundation’s ongoing support led to the 2018 creation of the Vera Bradley Foundation Center for Breast Cancer Research at the Indiana University Melvin and Bren Simon Comprehensive Cancer Center where more than 30 investigators collaborate on breast cancer discoveries.

    Support from the Vera Bradley Foundation has aided the IU School of Medicine in amassing talent, technology, and resources to become a national leader in targeted breast cancer therapies, specifically for triple negative breast cancer. For example, Bryan P. Schneider, MD, Vera Bradley Professor of Oncology at IU School of Medicine, is leading a nationwide study to develop personalized therapies for triple negative breast cancer patients with funding from the Vera Bradley Foundation. The study builds on groundbreaking findings by Schneider and other IU researchers.

    Since 2004, more than 1,300 discoveries have been published in peer-reviewed journals by IU breast cancer researchers.

    The Vera Bradley Foundation’s latest funding commitment will help launch immunotherapy research efforts to develop better therapies for triple negative breast cancer, an aggressive form of breast cancer that is often not responsive to hormone therapies and is resistant to chemotherapy. Immunotherapy uses the body’s immune system to target and destroy cancer cells.

    “To say we’ve ‘donated’ $37.5 million for breast cancer research doesn’t tell our story. To date, the foundation has provided $37.5 million of life changing research that has and will continue to impact women and their loved ones all over the globe,” Ruth Cook, chair of the Vera Bradley Foundation board, said. “We’ve invested in a team of the best, the brightest, and the most passionate researchers in the world. Our research team is relentlessly pursuing critical advancements that have and will continue to change the face of breast cancer forever.

    “Our goal is to have a breast cancer diagnosis become an inconvenience rather than an overwhelming presence,” she added. “We are confident our new pledge of $12.5 million will mean more women can enjoy long and thriving lives well beyond their breast cancer diagnosis.”

    “The Vera Bradley Foundation has been an unwavering source of support and inspiration at IU School of Medicine for the last 23 years. This new gift will help us take the next step in conquering breast cancer—one we’ve had in our sights for years,” said Jay L. Hess, MD, PhD, MHSA, dean of IU School of Medicine and IU’s executive vice president for university clinical affairs. “The foundation has benefitted and provided hope for countless women with the most difficult-to-treat breast cancers. We are tremendously grateful for their partnership and generosity.”

    Earlier this year, Mateusz Opyrchal, MD, PhD, joined IU as the inaugural Vera Bradley Foundation Scholar in Breast Cancer Discovery at IU School of Medicine and as co-leader of the cancer center’s experimental and development therapeutics research program. He most recently was at Washington University School of Medicine in St. Louis, where he was an associate professor of medicine and an associate director of cellular and gene therapeutics program in solid tumors.

    Opyrchal’s research focuses on novel therapeutic approaches and enhancing immune responses in triple negative breast cancer. His recruitment, made possible by Vera Bradley Foundation funding, expands IU’s leadership and knowledge base for the immunotherapy initiative with the goal of developing innovative approaches to help patients with this disease.

    In addition to precision medicine and immunotherapy, research at IU’s Vera Bradley Foundation Center for Breast Cancer Research includes efforts in prevention, early detection, improved treatments for curable disease, and survivorship.

    “I and the entire cancer research and care team here at the IU Simon Comprehensive Cancer Center are enormously grateful for the significant and sustained support we have received from the Vera Bradley Foundation,” said Kelvin Lee, MD, director of both the IU Simon Comprehensive Cancer Center and IU’s Vera Bradley Foundation Center for Breast Cancer Research. “This remarkable gift will enable our researchers in the Vera Bradley Foundation Center for Breast Cancer Research to continue their pioneering work in lifting the burden of breast cancer. Research cures cancer and such generous philanthropy fuels that research.”

    View and download photos, videos and more information about the Vera Bradley Foundation and breast cancer research at IU School of Medicine and IU Simon Comprehensive Cancer Center.

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    ABOUT VERA BRADLEY FOUNDATION FOR BREAST CANCER

    The Vera Bradley Foundation for Breast Cancer raises funds for breast cancer research to find a cure and to improve the lives of the many affected by this disease. The Foundation has contributed $37.5 million to the Vera Bradley Foundation Center for Breast Cancer Research at the Indiana University School of Medicine. The Center is focused on developing and dramatically improving therapies for some of the most difficult-to-treat types of breast cancer. Funds are raised through special events, partner events, and individual donations. Learn more about the Vera Bradley Foundation for Breast Cancer at www.verabradley.org.

    ABOUT INDIANA UNIVERSITY SCHOOL OF MEDICINE

    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

    Press Contacts:

    Stephanie Scheele, Vera Bradley Chief Purpose and Communication Officer
    sscheele@verabradley.com
    260.207.5185

    Anna Shelton, Vera Bradley Foundation Communication Specialist
    ashelton@verabradley.com
    260.207.5252

    Candace Gwaltney, IU Simon Comprehensive Cancer Center science writer
    cmgwaltn@iu.edu
    317.278.4322

  • Study: Vaccine protects most cancer patients from COVID, but risk remains higher for patients with blood cancers

    Study: Vaccine protects most cancer patients from COVID, but risk remains higher for patients with blood cancers

    INDIANAPOLIS—Using the nation’s largest COVID-19 data resource, a researcher at the Indiana University Melvin and Bren Simon Comprehensive Cancer Center found the COVID-19 vaccine protected most cancer patients from getting COVID. However, patients with certain types of cancer have a higher and widely varied risk of breakthrough COVID infections after receiving the COVID vaccine.

    Jing Su, PhD, assistant professor in the Indiana University School of Medicine Department of Biostatistics was the lead investigator for the study published in the Journal of Clinical Oncology. He is also the core associate director of real-world data for the cancer center’s Biostatistics and Data Management Core.

    Su led a team of 13 investigators from 10 research institutes across the country to analyze data from the National COVID Cohort Collaborative (N3C) at the National Institutes of Health, including another researcher at IU School of Medicine, Xiaochun Li, PhD, a professor of biostatistics and health data sciences.

    “This is one of the largest COVID real-world data resources in the world and the largest in the United States,” Su said. It includes more than 12.5 million patients and 4.5 million COVID patients. Researchers examined more than 64,000 cancer patients who were vaccinated against COVID-19.

    “We systematically screened major cancer types and major treatment types, as well as other risk factors such as age, comorbidities, sex, race, geographic locations and others to qualitatively know the contribution of each risk factor and the specific rates of each cancer subgroup as well as the contribution of treatment categories for cancer patients,” Su said. “This type of analysis is only possible because we have a huge COVID cohort and control cohort.”

    Among key findings were:

    • The risk of breakthrough infection was reduced after the second vaccine dose for all cancers.
    • Patients with hematologic cancers, or blood cancers, including leukemia, multiple myeloma and lymphoma, were at a higher risk of breakthrough COVID; those with blood cancers had a greater risk than solid cancers.
    • The Moderna vaccine was more effective than the Pfizer vaccine for protecting patients with hematologic cancers, especially patients with multiple myeloma.

    These findings could help guide clinical care and treatment for cancer patients with COVID, Su said. Beyond the pandemic, this research could also help when developing immune-based cancer treatments. Some immunotherapies rely on a patient’s immune capacities, and these findings could help researchers predict which patient populations may respond best to specific treatments.

    “In fact, the COVID pandemic provides a unique opportunity for us to screen the immune competence among all cancer patients at a national level,” Su said. “We could use this to imitate the differential immune capacities among cancer patients. This could guide us to better understand whether cancer patients will have good responses to cancer vaccines and if they are at a higher risk of infection of other viruses, such as the flu.”

    “Risk and Outcome of Breakthrough COVID-19 Infections in Vaccinated Patients WithCancer: Real-World Evidence From the National COVID Cohort Collaborative” is the second Journal of Clinical Oncology paper published by Su and colleagues using the N3C data. The group is now working to answer additional questions about waning immunity and the effectiveness of booster shots.

    “With the surging of new variants, especially the BA.2, we don't know whether there will be another wave down the road,” Su said. “We are monitoring the situation to see what new variants will mean for cancer patients and how to best protect them through vaccination.”

    This research was supported by the Indiana University Melvin and Bren Simon Comprehensive Cancer Center Support Grant from the National Cancer Institute (P30 CA 082709) and the Indiana University Precision Health Initiative.

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    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

  • Researchers make important new findings about how to test cancer-fighting drugs

    Researchers make important new findings about how to test cancer-fighting drugs

    INDIANAPOLIS—Researchers from Indiana University School of Medicine are discovering new ways to find out how effective a drug might be against cancer. Their findings are detailed in a new paper published by Science Advances.

    “This paper completely changes the way we need to collect tumor tissues and test for drug sensitivity,” said Harikrishna Nakshatri, PhD, a senior author of the paper. Nakshatri is the Marian J. Morrison professor of breast cancer research at IU School of Medicine and a researcher with the Vera Bradley Foundation Center for Breast Cancer Research at the Indiana University Melvin and Bren Simon Comprehensive Cancer Center. Hal Broxmeyer, PhD, a distinguished professor at IU School of Medicine who passed away in December 2021, also contributed to this study.

    Typically, tumors are collected and exposed to room oxygen, which is about 21 percent. However, different organs in the body have different oxygen levels. For example, the brain has 4.4 percent oxygen, blood 5.3 percent, and liver 5.4 percent. When cancer drugs are used on tumors in the clinical setting, they’re still in a patient’s body and are not exposed to ambient air.

    “The oxygen level in our different parts of the body is almost half of what we find in ambient air,” Nakshatri said. “Oxygen can have a different effect on the function of different proteins in the tumors. They may get activated, lose their activity level, get degraded or get stabilized. We wanted to test the tumors in a way that more closely resembles how they are in the body, so we know more about what drugs to use.”

    Researchers tested three different drugs on two different types of tumors. They split the tumors in half and tested one part in 5 percent oxygen, since that is an average oxygen level in the body, and exposed the other part to room oxygen before testing. They looked at the difference in the cancer stem cells, signaling pathways and how drugs behaved in the different oxygen levels. They found the sensitivy level of the tumor cells was different in 5 percent oxygen versus room oxygen.

    “This is a study that is now raising more questions we need to answer,” Nakshatri said. “Why do the cells react differently? Are we screening the drugs against cancer cells the right way? If we screen for drugs at the physiologic oxygen level, are we going to find different drugs that we may have missed all these years by doing the experiments at 21 percent oxygen?”

    In the future, researchers hope to study the different reactions tumors have to other various oxygen levels, like 1 percent or 20 percent. Nakshatri explained this kind of testing could act as another method of screening to determine a drug’s efficacy.

    “Suppose we identify a drug with the way we are doing right now in room oxygen, then add another layer of testing in the lab where we keep the cells at the physiologic oxygen level and compare whether the drug is working or not,” Nakshatri said. “If it works, then we can move forward to the clinical setting and it increases the chances of the drug being successful.”

    Other study authors from IU School of Medicine include Brijesh Kumar, PhD, Maegan Capitano, PhD, Yunlong Liu, PhD, Constance Temm, PhD, George Sandusky, PhD, and Amber Mosley, PhD. Read the full publication in Science Advances.

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    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

  • IU School of Medicine research provides advances in treatment of triple negative breast cancer

    IU School of Medicine research provides advances in treatment of triple negative breast cancer

    Findings published in influential Journal of Clinical Oncology

    INDIANAPOLIS – Indiana University School of Medicine physician scientist Bryan Schneider, MD is the principal investigator of clinical trial BRE12-158, a randomized clinical study published in the prominent Journal of Clinical Oncology the primary goal of which was to compare survival in women with high-risk (those who did not fully respond to chemotherapy prior to surgery) triple negative breast cancer (TNBC) with a genomically directed therapy versus standard of care.

    The study found that in the adjuvant (following chemotherapy and surgery) setting, a single genomically directed therapy was not better than a current standard of care option, which was capecitabine. 

    “Although this study did not prove genomically directed therapy was significantly superior alone, it did provide many advances for the treatment of high-risk triple negative breast cancer. We are thankful for the 200 patients and study teams across the United States who participated in this trial,” said Schneider, a researcher at the Vera Bradley Foundation Center for Breast Cancer Research at the IU Melvin and Bren Simon Comprehensive Cancer Center; also the location where the lab research portion of this study was conducted.

    Until 2015, no therapy, in the adjuvant setting had been shown to improve outcomes for patients with TNBC. This study showed that therapy (both the targeted therapy and the standard therapy option capecitabine) was markedly better than no therapy at all.  

    “Patients with high risk TNBC who received genomically targeted therapies had superior outcomes when compared with those who received no therapy. Additionally, patients who received capecitabine – a current standard of care option for TNBC – also had superior results when compared to those who received no therapy at all,” said Schneider, who is also the Vera Bradley Chair of Oncology at IU School of Medicine.

    Twenty percent of patients who were in the study did not receive therapy of any kind, in the adjuvant setting, for the treatment of their high risk TNBC. “We found that despite a study from 2015 which showed capecitabine was superior to receiving no therapy in patients with high risk TNBC, some were still receiving no therapy,” said Schneider. “When we observe a finding like this in a clinical study, experience shows us that in the real world, the numbers are actually much higher. Hopefully, these findings will further support that all patients with high risk TNBC in this setting will be considered for additional therapy as deemed appropriate by the treating oncologist. 

    This study also found that race did not appear to impact outcomes in this high-risk population. “This finding shows that the biology of TNBC is incredibly important and clinical trials should be focused in settings where disparate outcomes are most prevalent,” said Schneider. 

    Schneider also presented these findings as part of a spotlight session at the December 7-10, 2021 San Antonio Breast Cancer Symposium, an international scientific exchange among basic scientists and clinicians, working in breast cancer. 

    Clinical study BRE-158 has yielded an important discovery and provided valuable findings for oncologists and patients with triple negative breast cancer, said Tatiana Foroud, PhD, executive associate dean for research affairs at IU School of Medicine and leader of the IU Grand Challenge Precision Health Initiative. Results from this study, which are being published in prestigious journals read around the world, are doing what we promised to do, advance treatment options for patients with this terrible disease.”
     
    An initial discovery from clinical study BRE12-158  was published in JAMA Oncology. Schneider and his colleagues found that circulating tumor DNA (ctDNA) found in a patient’s blood, in the adjuvant setting, is a marker for residual disease; meaning patients who test positive for ctDNA are much more likely to have disease recurrence than those who test negative for ctDNA.

    “Circulating tumor DNA status remains a significant predictor of long-term outcomes in these patients,” said Schneider. It should be used to stratify patients in cancer clinical trials moving forward.”   

    The findings from BRE12-158 provided the needed rationale to launch the PERSEVERE  (BRE18-334) trial, which tests genomically targeted therapies in combination with capecitabine. PERSEVERE has now opened with four patients in the study. Using ctDNA technology, PERSEVERE is enrolling and following ctDNA-positive and negative patients. “Our hope is that these patients will continue to do well, enabling us to design future clinical trials of personalized therapy or no therapy that will further improve quality of life,” added Schneider.

    Study BRE12-158 was funded by the Vera Bradley Foundation for Breast Cancer in Roanoke and the Walther Cancer Center. BRE-158 is part of the Indiana University Precision Health Initiative Grand Challenge. The study was managed by the Hoosier Cancer Research Network and enrolled at 22 clinical sites across the United States.

    Media contact: Christine Drury; Email: cldrury@iu.edu; Cellphone: 317-385-9227

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

  • IU Simon Comprehensive Cancer Center enters into sponsorship with Indiana racing driver Jackson Lee

    IU Simon Comprehensive Cancer Center enters into sponsorship with Indiana racing driver Jackson Lee

    INDIANAPOLIS -- Marking 30 years of accelerating lifesaving research, the Indiana University Melvin and Bren Simon Comprehensive Cancer Center is putting the pedal to the metal through a new auto racing sponsorship. 

    The cancer center will join formula car driver Jackson Lee as the primary sponsor on his No. 2 Cape Motorsports entry in the 2022 Cooper Tires USF2000 championship. Jackson, a 19-year-old from Avon, Indiana, has been racing formula cars for four years, and will be starting as a freshman at IUPUI in 2022. He is entering his second season in USF2000, which is part of the Road to Indy, and races during INDYCAR weekend events at tracks around the country, including the Indianapolis Motor Speedway road course event in mid-May. 

    “We are excited to support Jackson and his team as there are many parallels between racing a car and racing to save lives,” Kelvin Lee, MD, cancer center director, explained. “When you watch a race team compete, you see the precision, determination and the science behind the sport. That’s a lot like cancer research and care.” (Note: Jackson Lee and Kelvin Lee have no familial relationship.)

    Through the sponsorship, Dr. Lee, who became cancer center director on Feb. 1, envisions connecting with the community to tout the cancer center’s research accomplishments that have benefited countless people over the last three decades.

    “I have quickly learned that Hoosiers are far too humble,” Dr. Lee said. “I am looking forward to this sponsorship allowing us to share just how much cancer expertise exists right here in Indiana.”

    IU’s physician-scientists have altered or defined treatment standards for such cancers as breast, pancreatic, thoracic, testis and colon as well as thymoma and thymic carcinoma and tumors associated with neurofibromatosis type 1, primarily in children, among others.   

    The IU Simon Comprehensive Cancer Center, which was established in 1992, serves as a central hub of cancer research across Indiana University. The center has more than 260 researchers who conduct all phases of cancer research, from laboratory studies to clinical trials to population-based studies that address environmental and behavioral factors that contribute to cancer. It is Indiana’s only Comprehensive Cancer Center designated by the National Cancer Institute, the nation’s top cancer agency, and one of only 51 in the nation to hold that status. 

    The prestigious designation recognizes its excellence in basic, clinical, and population research, its outstanding educational activities and effective community outreach program across the state.

    Dr. Lee also hopes that the year-long sponsorship will create opportunities for teambuilding within the cancer center. 

    “The cancer center is a large organization composed of laboratory scientists, physicians, nurses and support staff, among others,” Dr. Lee explained. “The pandemic has made it challenging to carry on our sense of community and camaraderie, so it’s my hope that we can all rally around Jackson’s team as we celebrate 30 years of improving the lives of cancer patients.”

    Like too many others, Jackson’s family has been impacted by cancer, as he lost his grandfather to lung cancer nearly two decades ago. 

    “Cancer has unfortunately impacted almost everyone’s family, mine included,” Jackson said. “I’ve enjoyed learning about the progress being made in cancer research and treatment, but there is much more that can be done. I’m excited to help in any way I can to encourage donations for research, raise awareness about the availability of the best treatment here in Central Indiana and to help celebrate the 30th anniversary of the cancer center at racetracks and other events throughout the year. I’m extremely grateful to the IU Simon Comprehensive Cancer Center for its support.”

    Jackson’s goal is to reach INDYCAR and the Indy 500 by at least 2026. Jackson won a karts to cars scholarship, has won four formula car races with 16 podiums (top-three finishes) and won a Team USA Scholarship in 2020 to race in several prestigious events in England.

    The 18-race 2022 USF2000 season begins Feb. 26 and 27 during the INDYCAR season opening weekend in St. Petersburg, Florida. 

    Media contact: Michael Schug; Email: maschug@iu.edu

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability

  • IU cancer researcher leads national work focused on developing treatments for inherited childhood cancers

    IU cancer researcher leads national work focused on developing treatments for inherited childhood cancers

    INDIANAPOLIS – An Indiana University cancer researcher is leading an $11.4 million grant from the National Cancer Institute in which he and others across the country will work on identifying new treatments for tumors that develop in children, adolescents and adults with a common genetic condition.

    First awarded in 2015 by the National Cancer Institute, the prestigious Specialized Programs of Research Excellence (SPORE) is led by D. Wade Clapp, MD. Clapp is chairman of the Department of Pediatrics at IU School of Medicine, the physician-in-chief at Riley Hospital for Children at Indiana University Health and a member of the IU Melvin and Bren Simon Comprehensive Cancer Center and the Herman B Wells Center for Pediatric Research. The project co-principal investigator is Kevin Shannon, MD, professor of pediatrics at the University of California, San Francisco (UCSF) and a member of the Helen Diller Family Comprehensive Cancer Center. 

    Clapp will lead the collaborative research effort to develop new treatments for tumors that develop in neurofibromatosis type 1 (NF1), the most common inherited syndrome causing a predisposition to cancer. It affects one in every 3,000 people worldwide, with about 100,000 individuals living with the syndrome in the United States. Individuals with NF1 have an increased risk of developing specific types of benign tumors and cancers including some brain tumors, a rare childhood leukemia and tumors that grow along the nerves and can progress to become sarcomas. 

    “Our SPORE grant is distinguished by its strong childhood cancer focus and multi-institutional effort, which brings together a coalition of the best NF1 investigators in the United States and likely the world,” Clapp said. “The grant involves six major institutions and the Pediatric Oncology Branch of the National Cancer Institute. The number of children with these genetic diseases is relatively small, so this sort of structure allows us to get the trials done efficiently and expediently.”

    A common underlying feature of these cancers is abnormal activity of a key signaling protein called Ras. The protein made by the NF1 gene normally restrains Ras signaling and this key “brake” is lost in tumors with NF1 mutations. In addition to playing a central role in tumors that develop in people with NF1, the NF1 gene is frequently mutated in cancers that develop in the general population such as breast and lung cancers, pediatric and adult brain tumors, melanoma and acute myeloid leukemia.

    The Ras protein is involved with more than a third of all cancers, and this research could lead to new therapies for many other cancers, Clapp said.

    “Our SPORE team applies data generated in the laboratory to guide the development of innovative clinical trials,” Shannon said. “This new NCI grant will allow us to extend this ground-breaking work that is evaluating a number of promising treatments for decreasing abnormal Ras signaling.”

    In addition to Indiana University and UCSF, collaborating institutions include the Pediatric Oncology Branch of the National Cancer Institute (NCI), Johns Hopkins University (JHU), Memorial Sloan Kettering Cancer Center (MSK), UT Southwestern Harold C. Simmons Comprehensive Cancer Center (UTSW), and Children’s Hospital of Philadelphia (CHOP) aligned in collaboration with the University of Pennsylvania Abramson Cancer Center. 

    One of 63 SPORE grants throughout the country, the IU grant remains the only one focused on childhood cancers. Additionally, while most SPORE efforts focus on a single cancer, the IU-led program—called the Developmental and Hyperactive Ras Tumor (DHART) SPORE—is working to develop new treatments for different tumors and cancers that develop due to NF1 mutations.

    “In the past five years, the group has completed a series of basic studies and clinical trials that have led to the first-ever FDA drug approval for NF1 and successful trials that are encouraging for new treatments in peripheral nerve tumors and myeloid leukemia,” Clapp said. “In this renewal, we are building on that work as well as initiating a new project focused on brain tumors that occur at higher rates in adolescents and young adults with NF1.”

    The grant includes three research projects: 

    • Project 1: Up to 50 percent of NF1 patients develop nerve tumors called plexiform neurofibromas (PNF). Researchers on this project have identified two effective drugs for treating PNF, including efforts that led to the FDA approval of selumetinib, the first drug specifically for NF1. They will build on that work to explore combination therapies and other new single drug therapies. Lead investigators on this project include Clapp; Lu Le, MD, PhD, (UTSW); Brigitte Widemann, MD, (NCI); Jaishri Blakeley, MD, (JHU); and Michael Fisher, MD, (CHOP). Luis F. Parada, PhD, (MSK) is also an investigator on this project.
    • Project 2: Young adults with NF1 are five to 10 times more likely to develop brain tumors called gliomas and glioblastoma. Additionally, the NF1 mutation can appear in gliomas even when the patient does not have the genetic syndrome. Project co-leaders Luis F. Parada, PhD, and Ingo K. Mellinghoff, MD, (both with MSK) will study the biology and possible therapies for these NF1-associated brain tumors. Jaishri Blakeley, MD, (JHU) is also an investigator on this project.
    • Project 3: Children with NF1 are also at high risk of developing juvenile myelomonocytic leukemia (JMML). Shannon and Mignon Loh, MD, (both of UCSF) are working to develop more effective and less toxic therapy for infants and children with JMML. Their findings have already led to an ongoing Phase 2 clinical trial.

    In addition, the SPORE grant includes a Development Research Program and a Career Enhancement Program, which provide funding to early-stage and established investigators to conduct research on NF1 and contribute to the overall goals of this work. 

    “The groundbreaking work done by this group in this SPORE has already been paradigm- and practice-changing and has fundamentally impacted patients in the most positive of ways,” said Kelvin Lee, MD, director of the IU Simon Comprehensive Cancer Center. “I believe the advances by Dr. Clapp and his team are only going to accelerate with this renewal.”

    Available for interview: 

    • D. Wade Clapp, MD, leader of the Specialized Programs of Research Excellence (SPORE). Clapp is chairman of the Department of Pediatrics at IU School of Medicine, the physician-in-chief at Riley Hospital for Children at Indiana University Health and a member of the IU Melvin and Bren Simon Comprehensive Cancer Center and the Herman B Wells Cancer for Pediatric Research. 
    • Mindi Hampton, a resident of southern California who brought her daughter, at the time a preschooler, to Indianapolis to take part in Clapp’s clinical trial. Hampton’s daughter Emily, now 17 and a high school senior, is alive today because of research conducted at IU. 

    Media contact: 

    Candace Gwaltney; Email: cmgwaltn@iu.edu

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.

  • $2 million gift supports lung, pancreatic cancer research at IU

    $2 million gift supports lung, pancreatic cancer research at IU

    INDIANAPOLIS—In a gesture that defines gratitude and respect spanning more than 23 years, Carmel resident Ruth “Rikki” Kutcher Goldstein has made a $2 million gift to support cancer research at Indiana University School of Medicine.

    The Rikki and Leonard Goldstein Chair in Cancer Research will be held by a lung or pancreatic cancer researcher at the Indiana University Melvin and Bren Simon Comprehensive Cancer Center. The gift honors Lawrence H. Einhorn, MD, for his care of her son-in-law, S. Wyle Weiman, more than 20 years ago. Though Wyle unfortunately died of lung cancer, the family remains grateful for the expertise and compassion that Einhorn provided. Since 1998, the Goldsteins have provided annual support to Einhorn’s research.

    “All Hoosiers should be grateful for the impact Larry has had on cancer research during his career at IU,” Rikki Goldstein said. “Our family is thrilled to honor him by ensuring that IU can continue to recruit and retain top-notch researchers like him.” 

    Einhorn, who is internationally known for his role in developing the chemotherapeutic cure for testis cancer in the 1970s, has been a faculty member at IU School of Medicine since 1973. Over his career, he has mentored and trained more than 100 oncologists and provided care to countless patients. His work continues to focus on testis and lung cancers. 

    The Goldsteins have a long history of generous support of Indiana University. Rikki Goldstein and her late husband Leonard (Len) generously supported the IU Borns Jewish Studies Program and established the Leonard M. and Ruth K. Goldstein Matching the Promise Scholarship in Jewish Studies.

    “In our family, we believe that giving and volunteering are important traditions," she said. “My parents taught me to support those in need. As a matter of fact, it's almost a requirement in Judaism. The word is ‘tzedakah,’ and it means responsibility. Being able to do this is really exciting for me and for all our kids, too.” 

    The Goldsteins, who were married for 71 years before Len’s death at the age of 97 in 2018, are the parents of four adult children: Michael, Steven and Jan Goldstein and Lisa Post. They also have eight grandchildren and four great-grandchildren. 

    After being an active volunteer at her children’s schools, Rikki Goldstein re-entered the workforce and, while working, earned her bachelor’s degree at age 50 from Indiana University Purdue University Fort Wayne (now Purdue University Fort Wayne). In 1976, she was a founding mother of the Fort Wayne Women’s Bureau, where she worked as director of peer counseling supervising programs that helped women re-enter the workforce after a divorce or death of a spouse.

    In 1996, at age 70, Rikki Goldstein began working for the Neighborhood Health Clinics in Fort Wayne. For 20 years, she served as a medical social worker and directed outreach programs before retiring in August 2016.

    “Medical research is very meaningful to me professionally as well as personally,” she explained. “That’s one of the reasons I am particularly delighted to make this gift.”

    Rikki Goldstein helped found the Fort Wayne Ballet and served on its board of directors. The Goldsteins both served on the board of directors of the Planned Parenthood organization during its early years in Fort Wayne.

    In 1994, Rikki and Len Goldstein were each appointed Sagamores of the Wabash by then-Governor Evan Bayh. One of the highest honors an Indiana governor can give, the award is presented for distinguished service to the state or governor.

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.
  • IU School of Medicine launches new study to develop personalized therapies for triple-negative breast cancer patients

    IU School of Medicine launches new study to develop personalized therapies for triple-negative breast cancer patients

    Stratification of at-risk patients made possible by building on foundation of previous discovery made by IU research team 

    INDIANAPOLIS – Indiana University School of Medicine researcher Bryan P. Schneider, MD, is leading a novel nationwide study to better understand how to treat patients with triple-negative breast cancer based on their own unique genetic data. 

    PERSEVERE is a phase 2 clinical trial with the goal of studying personalized cancer treatment combinations when compared to standard cancer treatment. 

    “There is a tremendous need for successful triple-negative breast cancer treatments,” said Schneider, who is the Vera Bradley Professor of Oncology at IU School of Medicine and a physician-scientist at the IU Melvin and Bren Simon Comprehensive Cancer Center and the Vera Bradley Foundation Center for Breast Cancer Research. “Recurrence and death rates are still too high, and novel strategies to improve that are markedly needed. We feel PERSEVERE is an innovative trial to try to help meet those needs.”

    While approximately one-third of patients with triple-negative breast cancer will achieve remission after surgery and chemotherapy, two-thirds will have some cancerous tissue in their body. Recent research shows that patients who test positive for circulating tumor DNA (ctDNA) – tumor cell pieces found in the blood stream – after surgery are at higher risk of their cancer returning. PERSEVERE will enroll 200 participants and test whether there is ctDNA in their blood. 

    If a participant tests positive for ctDNA, the researchers will look at the DNA of their blood and tumor to see if there are any genetic differences that can be treated with a genomically targeted therapy or a standard treatment. There are several possible combination therapies that these participants may receive based on their genetic differences. 

    If a participant tests positive for ctDNA but researchers determine they do not have a specific genomic target in their blood or cancer tissue, they will receive a standard treatment recommended by their doctor. Participants who test negative for ctDNA may receive a standard treatment, but they will also have the option to receive no treatment and remain in the study for observation. 

    “Based on the data that came out of our group, we have shown that circulating tumor DNA is also a very powerful risk stratifier,” said Schneider. “In this trial, we’ll capitalize on that powerful ability to better focus our attention on novel therapies for those patients at extraordinarily high risk who have ctDNA positivity. Equally importantly, it will provide the opportunity for us to focus on those who do not test positive for ctDNA, a group that may be expected to do quite well in terms of deescalating therapy and improving quality of life.” 

    PERSEVERE builds on a previous discovery by Schneider and colleagues in which they found that the presence of ctDNA and circulating tumor cells (CTCs) in the plasma of women’s blood who have undergone chemotherapy before surgery for triple-negative breast cancer treatment are critical indicators for the prediction of disease recurrence and disease-free survival. 

    Their findings from the BRE12-158 study were first presented at the 2019 San Antonio Breast Cancer Symposium, the most influential gathering of breast cancer researchers and physicians in the world, and then published in the prestigious medical journal JAMA Oncology

    Researchers will follow up with all participants for up to five years to check for side effects, as well as signs of cancer recurrence. This study hopes to determine better treatments for those fighting triple-negative breast cancer by looking at characteristics that are unique to each participant’s cancer. 

    The PERSEVERE study is funded by the Indiana University Precision Health Initiative Grand Challenge, the Vera Bradley Foundation for Breast Cancer, Genentech Roche, Pfizer, Foundation Medicine and Epic Sciences. The study is being managed by the Hoosier Cancer Research Network and will enroll at 20 clinical sites across the United States. 

    Media Contacts: 

    Anna Carrera | acarrer@iu.edu, 614-570-6503 (cell)
    Christine Drury | cldrury@iu.edu, 317-385-9227 (cell)

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News and World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability. 

  • IU School of Medicine researchers discover small molecule degrader as potential anticancer drug

    Unique approach unlocks opportunity for previously undruggable cancerous targets to be discovered

    INDIANAPOLIS—Researchers in the Department of Pathology and Laboratory Medicine at Indiana University School of Medicine have discovered a novel anti-cancer drug, using a unique approach that has allowed them to target previously undruggable cancerous proteins. When traditional drug discovery approaches failed, Anita Bellail, PhD, Chunhai Charlie Hao, MD, PhD, and their team developed a cancer cell-based screening to identify a lead compound known as HB007, which slowed cancer growth in models. Their findings were published this month in Science Translational Medicine. 

    “All proteins go through a life cycle from gene transcription to protein degradation,” said Bellail, the lead author who is an assistant professor of pathology and laboratory medicine at IU School of Medicine. “The cancerous proteins are highly expressed in part due to the blockage of degradation. Targeted protein degraders such as HB007 can release the blockage and send the proteins to the trash can.”

    In the past, researchers have searched for compounds known as inhibitors to directly bind to and inhibit cancerous proteins, but the vast majority of proteins lack binding pockets. The next generation of drugs that Bellail, Hao and team are working to identify are small molecule degraders that target the protein degradation pathway for the destruction of cancerous proteins and shutdown of cancer growth. 

    Eighty percent of proteins in the human body are currently undruggable. The team led by Bellail and Hao, who are members of the IU Melvin and Bren Simon Comprehensive Cancer Center,  designed and utilized a cancer cell-based drug screening process to identify the first small-molecule degraders of a cancerous protein called SUMO1, which was previously considered undruggable. The team has also discovered the novel CAPRIN1-CUL1 ubiquitin ligase that compound binds to and leads to SUMO1 recruitment to the ligase for destruction.  

    “We believe we are the first ones to report the step-by-step process of how to find the small molecule degraders of undruggable proteins,” said Hao, a senior author who is the Bicentennial Professor of Pathology and Laboratory Medicine and Neurological Surgery at IU School of Medicine and a member of the Vera Bradley Foundation Center for Breast Cancer Research at the cancer center. “This will generate a strategy that can help researchers find small molecule degraders as novel drugs. Since the degraders can destroy the proteins, the dose can be much lower, so efficacy is also much better.”

    Bellail, Hao and colleagues tested their theory by observing patient-derived brain, breast, colon and lung cancers in animal models. Their approach suppressed the cancers and increased the survival of the animals. This approach may be useful for identifying other small-molecule degraders of cancerous proteins as the next generation of anticancer drugs. 

    Bellail and Hao came to IU School of Medicine in 2018 with the goal of building up novel drug discovery programs. 

    This work was supported in part by the Pathology Bicentennial Chair endowment and start-up fund, the Indiana University School of Medicine Physician Scientist Initiative funded by Lilly Endowment Inc., IU Simon Comprehensive Cancer Center’s 100 Voices of Hope program, the Vera Bradley Foundation for Breast Cancer Scholar fund, and National Institutes of Health grants R01CA203893 and R43CA224461.   

    Media contact
    Anna Carrera | Email: acarrer@iu.edu | Cellphone: 614-570-6503

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    About IU School of Medicine
    IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.