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IU announces progress toward its Precision Health Grand Challenge: cancers it aims to cure, diseases it is working to prevent

IU School of Medicine • 8/6/18

Today Indiana University researchers and physicians announced progress toward their bold goals of developing cures and preventive treatments for devastating diseases prevalent among Indiana residents.

IU Precision Health leaders announced they have recruited 33 new research faculty from throughout the country and built some of the most advanced research and clinical capabilities in the Midwest with the goals of:

  • Developing new approaches for treating triple negative breast cancer and multiple myeloma that that will cure more patients with these cancers
  • Curing more children with pediatric sarcoma, a particularly deadly cancer found in tissues such as tendons, bones and muscle
  • Preventing the onset and progression of Type 2 diabetes by discovering what biological factors trigger the disease and tailoring treatments to individuals
  • Slowing the progression of Alzheimer’s disease by researching the role of the immune system and developing new immunotherapies

In addition, the university unveiled that a team led by IU Bloomington social scientists will soon begin canvassing rural Indiana to collect information and DNA samples from residents of all backgrounds. The data collected will help researchers understand how genetic, behavioral and environmental factors influence a person’s health.

Led by IU School of Medicine, the Grand Challenge Precision Health Initiative is IU’s big health care solution. It was established in 2016 with a $120 million investment––$80 million of which has already been committed.

“The progress made over the last two years is already impacting the care we provide patients,” said IU School of Medicine Dean Jay L. Hess, MD, PhD, MHSA. “The Precision Health Initiative ensures Hoosiers do not have to travel to receive leading-edge care and participate in clinical trials. In partnership with IU Health, we are now using genomics to personalize care for thousands of patients, and we are the only place in Indiana to offer cellular immunotherapy – the ability to reprogram a patient’s immune system to fight disease.” 

Statewide Person-to-Person Health Interview Study

Beginning in August, Precision Health Initiative social scientists led by Bernice Pescosolido, PhD, Distinguished Professor of Sociology at IU Bloomington, will begin an effort to reach a statistically representative sample of 2,000 Indiana residents that reflects the demographic characteristics of Hoosiers. Based on recent U.S. Census data, residents will be selected to participate in the initiative’s “Person to Person Health Interview Study.”

Households that are selected will be sent letters of invitation in the mail, letting them know that credentialed interviewers will soon be arriving at their door. Residents will then be asked to complete a questionnaire covering a variety of health topics such as their exposure to environmental contaminants and their attitudes toward mental health treatment. Participants will also be asked to discuss their social networks and provide a saliva sample for DNA collection.

IU Bloomington Provost and Executive Vice President Lauren Robel said input from residents across Indiana is critical to the long-term success of the IU Precision Health Initiative.

“People turn to their social networks to support their well-being, health and pathways to care, so it matters who is in your network,” Robel said. “This unique approach will allow IU researchers to bring the voices and experiences of Indiana’s people into the Precision Health Initiative’s efforts to improve health.”

The IU Precision Health approach

Anantha Shekhar, MD, PhD, principal investigator of the I Precision Health Initiative and executive associate dean for research affairs at IU School of Medicine said, “the IU Precision Health Initiative incorporates a multi-disciplinary view of health, illustrated by the initiative’s several scientific pillars of investigation: genomic medicine; cell, gene and immunotherapies; chemical biology and biotherapeutics; data and informatics; and psychosocial, behavioral and ethics.”

“Identified diseases are taken through each pillar using a team science approach as we work to treat, cure and prevent diseases,” Dr. Shekhar said.

He cited the university’s approach to Alzheimer’s disease as an example of how different pillars––or scientific areas of focus––of the IU Precision Health Initiative will work together.

Researchers are studying how the body’s own immune system may impact the underlying biology associated with cognitive decline, the primary symptom of Alzheimer’s disease. The team is planning to establish new immune biomarkers associated with Alzheimer’s disease that together, with existing Alzheimer’s biomarkers, will help better predict onset and progression of the disease.

Once those biomarkers are determined, researchers will investigate and develop immune-based therapies that not only target biomarkers of the disease, but are also tailored precisely to an individual’s own biology.

Bruce Lamb, PhD, co-leader of the Precision Health Alzheimer’s disease research team and executive director of IU School of Medicine’s Stark Neurosciences Research Institute, said, “Previous approaches to treatment or prevention of Alzheimer’s disease may have failed because they only focused on one indicator of disease, such as amyloid or tau.”

“Our approach focuses on altering the body’s own immune system to positively impact all three biomarkers of amyloid, tau and inflammation,” Lamb said. “If we can identify new methods to slow Alzheimer’s disease from progressing to a fully debilitating disease of the mind and body, we’ll have made a great step forward.”

Indiana resident Mary Estrada, 62, lost her mother to Alzheimer’s disease and was recently diagnosed with early onset Alzheimer’s disease. Estrada is currently participating in Alzheimer’s disease research as part of the Precision Health Initiative at IU School of Medicine.

“I’m concerned (about my diagnosis), but I also feel good that there is so much being done these days and so many people are working on it; that gives me reason for hope and reason to be proud that I get to be a part of it,” Estrada said.

Stay up-to-date with progress

Because the IU Precision Health Initiative is a long-term effort, IU School of Medicine is sponsoring monthly podcasts on the topics of diseases that IU researchers aim to cure, treat or prevent. The podcasts, produced by the award-winning show “Healthcare Triage,” are anchored by IU School of Medicine faculty and social media star Aaron E. Carroll, MD.

You can download the first podcast, focused on Alzheimer’s disease, on the IU School of Medicine website: medicine.iu.edu/expertise/precision-health-initiative.

Additionally, anyone interested in getting involved in improving health in Indiana is encouraged to visit www.allin4health.info and sign up to receive information about health resources and to participate in research happening across the state.

PROGRESS TO DATE:

Cancer

  • Today, IU Precision Health Initiative partner Indiana University Health plans to administer CAR T-cell therapy to its first relapsed diffuse large B cell lymphoma patient. As a result of the IU Precision Health Initiative, IU Health is now the only approved site in Indiana to administer FDA-approved CAR T-cell therapies, widely considered by some as a cure for certain types of leukemia and known for improved remission rates in certain lymphomas, as a result of the IU Precision Health Initiative.
  • Pediatric patients will be able to receive CAR T-cell therapy as early as this fall at IU Precision Health Initiative partner Riley Hospital for Children at IU Health.
  • The triple negative breast cancer research team is about to wrap up its first Phase II Precision Health Initiative clinical trial, which was funded by the Vera Bradley Foundation. The trial, called BRE 12-158, compares precision-guided therapy versus current standard of care. By raising awareness through social media, the team doubled its patient enrollment rate and shaved two years off the clinical trial timeline, potentially speeding the time for the treatments to reach more patients.
  • Institutional Review Board approval has now been secured for a clinical study aimed at screening patients awaiting bariatric surgery for the presence of monoclonal gammopathy of undetermined significance, or MGUS, a protein known to progress to multiple myeloma in certain cases. This study will investigate the incidence of MGUS in an obese population and see if weight loss following weight reduction surgery has an effect on MGUS. This study will contribute to knowledge about MGUS and the prevention of multiple myeloma.
  • The multiple myeloma disease research team has just opened the Indiana multiple myeloma biobank study, which is currently enrolling 1,000 multiple myeloma patients across Indiana who are willing to donate bio specimens, such as blood and saliva, and complete a questionnaire. The goal of the study is to evaluate patients’ clinical, genomic and environmental data to advance clinical trials aimed at developing what could eventually be considered a cure.

Immunotherapy

  • IU School of Medicine opened its first Good Manufacturing Practice Cell Therapy Lab, which allows IU scientists to research and develop new cell, gene and immunotherapy-based treatments on campus. The new lab will focus on diseases in scope for the IU Precision Health Initiative.
  • IU School of Medicine was recently selected as a site to participate in a Phase III clinical trial of CAR T-cell therapy versus a monoclonal antibody in late-stage relapsed or refractory multiple myeloma patients. Clinical trials such as this seek to identify the most effective therapies and potential cures available.

Genomic medicine

  • IU School of Medicine acquired technology that can perform a staggering 200 whole genome sequences per week, bringing the most powerful genome sequencing capabilities in the Midwest to the university.
  • Key Precision Health Initiative partner IU Health has expanded its precision genomics program beyond Indianapolis to Muncie, West Lafayette and Bloomington, bringing the total number of clinics to five statewide.
  • The Pediatric Precision Genomics Clinic, established as a result of IU’s Precision Health Initiative, allows IU researchers to test the DNA of every child at IU Health with aggressive or relapsed sarcomas. This enhanced capability, combined with the ability to assess a child’s RNA and proteomics, has led to the identification of a potentially useful FDA-approved treatment in greater than 90 percent of cases—up 20 percent since the launch of the Grand Challenge.
  • The pediatric sarcoma team is planning to launch a clinical trial testing molecularly targeted maintenance therapy for metastasized sarcoma at the time of diagnosis. Unlike adults, children do not receive maintenance therapy after chemotherapy and surgery. The goal of the study is developing curative therapies for rhabdomyosarcoma, osteosarcoma and Ewing’s sarcoma.

Diabetes

  • The Type 2 diabetes research team is focused on the prevention and progression of this chronic disease with a special focus on women with gestational diabetes, who are at very high risk of developing Type 2 diabetes later in life. The team is currently analyzing the genomics of 10,000 pregnant women through blood samples made available through the NIH’s nuMoM2b study to understand what biological factors lead some of them to develop Type 2 diabetes. Researchers are also investigating RNA to understand the environmental components of the disease. Prevention studies in people at higher risk of developing Type 2 diabetes will be focused on identifying and overcoming individual factors that interfere with treatment. 

Talent recruitment highlights

  • Kun Huang, PhD, was recruited as Data and Informatics pillar leader of the IU Precision Health Initiative.
  • Michael Weiss, MD, PhD, MBA, has been recruited as the leader of the Chemical Biology and Biotherapeutics pillar of the IU Precision Health Initiative.

What leaders are saying…

  • “Leveraging the deep research expertise in each of these diseases through IU School of Medicine, combined with the population health experience of IU’s Bloomington campus, is what led us to choose these as areas of focus for IU’s first Grand Challenge. It starts with the idea that we have the right expertise and some of the country’s top researchers in these diseases that we believe will help make these incredible goals a reality.” – IU President Michael A. McRobbie
  • “All knowledge from the initiative is being integrated into research training and clinical education to further propel IU’s reputation as one of the leading universities with one of the nation’s top medical schools.” – Fred Cate, IU vice president for research and leader of IU’s Grand Challenges program