Healing kids. Precisely.
What if we had the diagnostic capabilities to understand the differences in seemingly similar diseases? What if we knew which children with similar diseases would respond better to which treatments? What if by understanding these variations we were able to design more precise treatments for a child that gave them a better chance of survival, reduced the toxicity of their treatments, allowed them to recover more quickly, and gave them a better childhood?
The answers to each of these questions can be found in precision medicine.
Tailoring treatments for every cancer
Hundreds of children are treated for cancer at Children’s Hospital Colorado every year, and their likelihood of beating this illness has never been better. Four decades ago, a cancer diagnosis was almost certainly terminal. Today, more than 85% of pediatric cancer patients survive.
This pace of progress is a testament to the unselfish collaboration of the pediatric oncology community. Hundreds of hospitals, dozens of industry partners, and thousands of experts have joined forces to share data, standardize treatments, expand clinical trials, and develop life-saving drugs. With every advance, survival rates rise; families find new hope.
But still, 15% of children do not survive a cancer diagnosis. Three difficult questions have stalled progress toward a world in which every child experiences a healthy, hopeful future.
How can we reduce the unintended burden of treatment?
95% of childhood cancer survivors develop significant health-related issues resulting from treatment. These challenges — hearing, vision, and dental problems, infertility, new cancers, stunted bone growth — can impact children for the rest of their lives. They can put kids at risk for other complications. At their most devastating, they are life-threatening.
How can we improve survival rates for the most resistant cancers?
For the 10 to 20% of children whose cancer is resistant to traditional treatment, repeatedly returning, or high risk, outcomes are far less promising. Of treatments that do exist, many are more experimental and less effective. And diagnoses of some cancers still offer no chance of survival.
How can we pinpoint which treatment will be most effective?
While physician-scientists diagnose and treat pediatric cancers using all the information at hand, they cannot guarantee how a child will respond to a drug, or pinpoint with certainty the therapy that will prevent a future relapse. Treating pediatric cancer still involves extensive research and experimentation.
An investment that will change individual lives and advance pediatric health
The more we know about a patient’s genomic sequencing alongside their cancer diagnosis, the better we can target and treat the disease they are facing. The ability to conduct this sequencing would enable us to accomplish the following:
- Ensure 100% of cancer patients receive genomic testing. Only 50% of patients receive testing today.
- Enable significantly more sophisticated testing. Next-generation sequencing provides faster, more comprehensive information, both about the patient and the cancer. The resulting data will be analyzed throughout the patient’s journey: as they progress, or if they relapse. Like a series of school photos documenting a student’s physical growth, these genetic snapshots will create a valuable pool of information.
- Reduce reporting time from 30 days to 10 days. Quicker diagnoses expedite decision making.
- Reduce the cost of each test by 50%
Next-generation sequencing means the ability to administer more effective treatments, immediately.
To have a robust dataset is not enough. We must be able to use our sequencing data to improve care and advance treatments. Donors will create a state-of-the-art system for analyzing, understanding, and using sequencing results. Investments will:
- Organize and store clinical sequencing data in a secure platform accessible to researchers.
- Build and train a data model, in partnership with other leading pediatric cancer centers, that assesses the effectiveness of treatments based on patients’ genetic profiles, diagnoses, and tumors.
- Through modeling and analytics, uncover new genomic-disease associations — in other words, the specific genetic variations that are connected to particular diseases.
Data profiling means that physicians can view—in real time—how other patients with similar genetic profiles and tumors have responded to different treatments. It means we can analyze the factors that cause treatments to be toxic or effective. It means we can learn, adjust treatments, and proactively establish contingency plans based on a patient’s genetic profile.
Armed with the precious information provided by genetic testing and intelligent analysis, researchers can design better clinical trials. Physicians can direct the right patients to the right clinical trials. Investments in these smarter, more precise research projects and trials will accelerate the discovery, development, and deployment of novel therapies. Philanthropy will help to:
- Attract and retain the experts and key personnel necessary to scale and accelerate research.
- Provide pilot awards, which provide the early funding necessary to seed innovative research ideas, especially for early-career investigators. With pilot funds—awarded on a competitive basis to the most compelling, scientifically meritorious ideas—researchers can explore new hypotheses, grow as national thought leaders, and pursue even larger grants from extramural funders like the NIH. Pilot awards have helped launch ideas that spurred major breakthroughs.
- Ensure sufficient space, technology, and equipment to support groundbreaking research.
Accelerated therapy development means better treatments, higher survival rates, and greater hope for families. It means progress toward a world in which every child survives a cancer diagnosis.
Propelling the promise of precise solutions
Equipped with the information that genomic medicine reveals, physicians can administer targeted care, tailored to a child’s genetic fingerprint. They can pinpoint the treatments most likely to help or harm a patient or direct a child to the right clinical trial. They can more accurately predict the chance of relapse, then modify treatment accordingly.
Genomic medicine offers the promise of precise solutions. Its power to impact pediatric cancer is amplified by:
- The potential for learning. The genetic basis of even the most common pediatric cancers is not well understood. Every discovery in this field will represent a significant leap forward, unlocking a new world of information for researchers to build upon. Answers for one cancer will be parlayed into answers for other cancers.
- Fewer complicating mutations. Pediatric cancers have few genetic mutations compared to adult cancers. With fewer mutations to navigate, physician-scientists can more easily isolate the genetic changes responsible for tumor growth. They can draw cleaner, clearer conclusions.
While we begin this work in pediatric cancer, it will ultimately cross all our service lines and disease states so we can provide the right treatment to the right children using the best information and treatments available.
Beyond cancer and blood disorders…
A child enters Children’s Colorado with a rare neurodegenerative disease, cystic fibrosis, or a serious heart condition like cardiomyopathy. Their genome is immediately sequenced. A profile is built, then updated and improved throughout the child’s life. The information is added to the most sophisticated, secure database of pediatric genomic data in the country. Because of this, the child’s physician can offer better, more personalized treatments—the particular medicine, for example, that the child’s specific type of cystic fibrosis will respond best to. Or the protein-inhibiting drug that will target the genetic cause of the child’s spinal disorder. On a broader level, we can better understand the illness. We can improve upon existing treatments, while developing more precise, harm-free therapies. This is what significant investments in precision medicine will catalyze.
Building on a proven framework
Our bold vision—to create a new model for treating pediatric cancer, and eventually all childhood disease—demands a unique intersection of strengths. Its success will require an established cancer program that has earned the respect of partners and peers, deep analytical talent, and a powerful foundation for genomic medicine. Children’s Colorado has cultivated these strengths, and more.
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