L.A. children’s hospital gets personal

February 17, 2015 in Medical Technology

Children’s Hospital Los Angeles announced it has committed $50 million to expand its Center for Personalized Medicine.

The investment in research and innovation will help unlock the human genome’s potential with the goal of making diagnoses more effective, therapies more targeted and healthcare more personalized for children, officials say. 

The money will be disbursed over five years. The hospital will seek an additional $50 million in philanthropic funding from the community to support the translation of research outcomes in the lab into bedside care for infants, children and adolescents.

Medicine is on the verge of a new era as game-changing as the discovery of antibiotics,” said Richard D. Cordova, president and CEO of the hospital, in a statement announcing the plans.

With President Obama’s recent announcement of support for the Precision Medicine Initiative, I am pleased to know that our institution has the capacity to lead the development of better treatments and cures for children,” he added. “We have ambitious goals and are uniquely poised to deliver on them in support of personalized medicine research and clinical care.”

[See also: Obama puts precision medicine on radar.]

Personalized, or precision, medicine is a revolutionary way of practicing medicine in which a patient’s biological profile, as determined by her or his genome, is used to develop individualized lifelong healthcare.

“In the near future, a newborn’s genome will be sequenced at birth – or even before – permitting clinicians to plan a lifetime of personalized, preventive healthcare that focuses on preventing, rather than reacting to, illness,” explained Alexander R. Judkins, MD, executive director of the Center for Personalized Medicine at CHLA and head of the hospital’s department of pathology and laboratory medicine. “Physicians and scientists at The Saban Research Institute at CHLA are in a position to research and develop treatments that are relevant for children here and across the globe. When we look at our peers using personalized medicine for children, the area where CHLA will be investing its efforts is in taking research outcomes and innovations and translating them into improvements in bedside care – an area where we already excel. This is where the real impact for children will be.”

The Center is led by Judkins and will be part of the hospital’s department of pathology and laboratory medicine. The Center’s team will include physicians, scientists, genetic counselors and staff who are internationally recognized for their expertise in genomics, clinical genetics, bioinformatics and molecular diagnostics. Children’s Hospital will also be exploring strategic collaborations with academic, government and healthcare organizations to share resources and expand the impact that the Center can have on the care of children.

[See also: Deloitte taps the Zen of data analytics.]

Children’s Hospital’s investment in the center will focus on three areas with the greatest potential to positively impact children’s health: cancer, inherited diseases and infectious diseases. CHLA has leading clinical and research programs in each of these areas, and the hospital will leverage its existing resources and expertise to use genetic testing to refine and make treatment and care more precise for each.

The Center will initially focus on pediatric cancer. The Vision Center and the Department of Pathology and Laboratory Medicine at CHLA have already developed a new gene sequencing test that will identify all changes related to the retinoblastoma gene – RB1 – in eye cancer patients, using leading-edge genomic sequencing technology and bioinformatics.

CHLA treats one-fifth of all the retinoblastoma patients in the U.S.

“This is just one example of bench-to-bedside translational research involving pediatric cancer genomics already under way at CHLA,” Judkins said. “Our expansion will provide us with the opportunity to study genomic features of all new and recurrent cancers treated at CHLA and support our Children’s Center for Cancer and Blood Diseases, led by Director Alan Wayne, MD, in discovering causes and novel therapies for pediatric cancer.”

As the program develops, CHLA plans to expand its efforts to include genetic conditions such as epilepsy, autism, neurocognitive disorders, congenital heart disease and cleft palate.

“Personalized medicine holds the potential to reduce the risk of developing some health disorders for which people are genetically predisposed, such as obesity, diabetes and hypertension,” Judkins said. “Yet, we have unlocked only a fraction of the information that our genes can reveal. At CHLA, we have unique expertise and are internationally recognized for treating many of these disorders in children.”

With the use of personalized medicine, the Center predicts that, in the future:

  • Diseases will be diagnosed earlier and more accurately.
  • Treatments will be safer and more effective.
  • Visits to the doctor will focus on prevention.
  • Some conditions will be treated before symptoms ever emerge.

Healthcare costs could decrease significantly due to early diagnoses, intervention and preventive medicine. To this end, the Center’s services and objectives include:

  • Using research to unravel the genetic basis of disease and create treatment options based on genetic profiles for subsets of patients and, ultimately, individuals
  • Leading genetics-based pediatric clinical trials
  • Setting new care standards for the use of personalized medicine in effective diagnosis, treatment and clinical care for children
  • Discovering therapies, and even cures, for diseases that currently have no effective treatment
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