Augusta University

NEVER SAY NO

Dr. Ted Johnson in lab

Pediatric Immunotherapy:
The Time for YES

One of the most heartbreaking words a parent of a child with cancer can hear is “No.” No more treatment options to cure your child’s cancer. No more clinical trials to slow down the relapsed cancer. No more hope.

Here at the Georgia Cancer Center, our heart’s desire is to Never Say No. We are striving to offer a complete array of clinical trials to families looking for immunotherapy options for their child with relapsed cancer. And, as some of the world leaders in helping the immune system control cancer in children, who better to lead the way?

Help us say Yes. Itʹs time.

Dr. Davin Munn in labClinical Trials for Children with Brain Tumors

Based on preclinical laboratory results, the clinical trials are designed to test the concept that this class of drugs (IDO-inhibitor drugs) creates a synergy with standard treatments that:

Makes the combined treatment work MORE POTENTLY against the brain tumor and requires lower doses of chemotherapy and radiation to achieve the more potent effect; therefore, the treatment may be LESS TOXIC and the children may have fewer side effects and BETTER QUALITY OF LIFE.

“We have been able to design regimens that produce an excellent quality of life, with minimal well-tolerated side effects, yet still access the tumor-killing power of chemotherapy and radiation,” says Dr. David Munn. “Our ultimate goal is to achieve ten times the potency at one-tenth of the side effects.”  Dr. David Munn

Dr. Ted Johnson and Coulter Hnatt

Dr. Johnson and Dr. Munn have the following goals for quality-of-lifefeatures in all of their immunotherapy trials:

  • Use oral medications whenever possible and focus on moderate doses of chemotherapy
  • Have as much of the treatment as possible taken at home (keeping hospital stays to a minimum) and work closely with the referring centers to decrease the amount of travel for long-distance patients
  • Use immunotherapy to help out the chemotherapy and radiation so that ultra-high doses can be avoided (important for minimizing side effects like nausea, vomiting, hair-loss, etc.)
  • Focus on maintaining a good quality of life — the goal is to have children returning to school, playful and happy, not wiped-out and miserable

Treating More Pediatric Cancers

The Georgia Cancer Center is the first – and currently only – program integrating chemotherapy, immunotherapy and radiation to treat children with relapsed brain tumors. We have plans for even more children to benefit from the promise of pediatric immunotherapy by:

  • Increasing the number of children in the existing Indoximod trial for brain tumors
  • Starting clinical trials using an infant-friendly (liquid) formulation of Indoximod
  • Expanding the existing Indoximod trial into Phase II and adding especially difficult-to-treat brain tumors such as pontine gliomas (DIPG)
  • Adding more participating Centers around the country
  • Beginning a first-in children trial of IDO-inhibitor in pediatric solid tumors (neuroblastoma, osteogenic and Ewing’s sarcomas, rhabdomyosarcoma, and others)
  • Beginning a first-in children trial of IDO-inhibitor in relapsed/refractory AML (acute myeloid leukemia)
  • Developing a new class of immunotherapy drug (PTEN-inhibitor agents) that will let us take chemotherapy to the next level of effectiveness
  • Developing a continuing pipeline of powerful new drugs through discovery research
  • Pioneering new ways of monitoring a child’s immunity against their cancer
  • Ultimate goal: offering a treatment option for every child with cancer that incorporates some component of immunotherapy to harness the power of the patients' own immune system.

Be Nimble, Be Quick

These additional clinical trials are ready and waiting to open. But we can’t enroll new patients without the resources we need for success. There is a pent-up demand for this exciting program – these discoveries and drugs are not available anywhere else in the world. We are receiving referrals from major cancer centers around the country and many families fly to Augusta for treatment.

Yet, we have had to turn children away because we do not have enough staff and other resources to handle the demand for even our existing brain-tumor trials. With more resources, we could significantly increase the number of children who could have access to these promising treatments.

We have the science ready and waiting to quickly offer additional clinical trials for other kinds of pediatric cancers; but, again, we need staff and other resources to implement them.

Pediatric "Piggyback" Model

Nationwide, children almost always lag many years behind adults in gaining access to new drugs and treatments. Through the Pediatric Immunotherapy Program at the Georgia Cancer Center, we have developed an innovative model to rapidly bring forward promising new immunotherapy drugs from the pre-clinical pipeline into pediatric clinical trials. We call this the “pediatric piggyback” approach. Our goal is to begin a “piggyback” pediatric trial of a given immunotherapy drug just as soon as suitable safety data are available from a corresponding adult trial in the analogous tumor – without having to wait for the drug to eventually reach the commercial market in adults.

This “pediatric piggyback” strategy works because of the excellence in preclinical research and drug development in the Pediatric Immunotherapy program. Discoveries at Augusta University are moving the field forward, and this is being translated into a place at the table for children in the newest clinical trials.

But the immune system is capable of doing much more than we are currently asking it to do. One drug – no matter how good – is not enough to tap the whole power of the immune system. We have several other potent drugs in the development pipeline, moving toward their first clinical trials. To ensure that children keep their place at the table with these drugs as well, our pediatric-focused preclinicaldevelopment laboratories are key drivers of the “pediatric piggyback” pipeline.

Our goal is to "Never Say No" to children with cancers that have defied standard treatments

We can do this with more staff support, funds and resources

Making an investment in the Pediatric Immunotherapy Program is making an investment in a child’s future.

How to make an Impact:

CONTRIBUTE - Seed funding for clinical trials to support launch of early-phase immunotherapy trials in children (data collection, sample collection, e-rays, etc.) $3 million over 5 years ($600,000/year)

GIVE - Operating funds to continue nimble development of state-ofthe-art new immunotherapy drugs for children, especially to quickly translate breakthroughs and opportunities into first-in-children trials in childhood cancer. $5 million endowment

PROVIDE - Infrastructure support to add full-time, pediatric-focused personnel, including a pediatric nurse practitioner; oncology clinical-trials nurse-coordinator; data manager; and regulatory/compliance staff for first-in-children trials. $2 million over 5 years ($400,000/year)

You can make a give using cash, securities, estate gifts, real estate or other assets, payable to Georgia Health Sciences Foundation. You may also pledge an amount to be given over time.

Naming Opportunities are available.

giving.augusta.edu/neversayno

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