Progressive Pediatric Cancer Consortium: Accelerating Leading Edge Pediatric Cancer Care to Those Who Need It Most

by Dorit Baxter and Marilyn Luce Robertson

As parents, we want to provide our children with the best of everything within our power. We sacrifice our own desires so they can have more than we had and become more than we’ve become. We move mountains with our love for them. After all, they are our future, and they deserve every opportunity to live their lives to the fullest in his or her own beautiful, one-of-a-kind way.

Now, imagine a visit to the pediatrician for what was thought to be shin splints or a tummy ache turning into the living nightmare of a pediatric cancer diagnosis. Suddenly, wanting the best for your child takes on a radically different meaning. You will do everything possible to make sure your child has access to the best doctors, leading facilities, and the most advanced treatments. You will likely spend hours researching every aspect of the cancer, deciphering medical abstracts and journals, and conferring in chat rooms with other parents across the globe who braved this journey before you. You amass an arsenal of well-researched medical opinions and possible treatment options, along with every ounce of hope you can muster, and present it to your child’s doctors.

What if they gently explain that the novel treatment showing promising results in decimating the same cancer your child has is only available to adults? Or, it’s available to children, but the only participating facility is on the opposite side of the country? Or, it isn’t covered by insurance, and even if you traded in your retirement and sold your house, you wouldn’t be able to afford it? Your child’s best option – best hope – is just beyond your grasp.

“Why not my child?” you ask. “Doesn’t my child deserve the best treatment possible?”


What is the Pediatric Cancer Consortium and why is it needed?

The Pediatric Cancer Consortium, a brave and forward-thinking collection of world-class pediatric oncologists, thinks every child deserves the best and most advanced treatments available. Formed by the Cancer Breakthroughs 2020 program led by Dr. Patrick Soon-Shiong, and joined by leading pediatric oncologists and medical centers across the country, the Consortium aims to speed delivery of the most innovative diagnostic science and therapies to target the most difficult cancers in children – all with the ultimate goal of revolutionizing the standard of cancer care for kids, offering more hopeful prognoses for difficult-to-treat cancers and dramatically improving the quality of life during treatment.

Step One: A Test that Tailors Treatment

Children are unique. So is their cancer. Over 15,000 children, ages 0-19, are diagnosed with cancer annually. With more than 100 types of pediatric cancer, not including sub-types, every case – and every patient – is different, and as such, no one cancer center has across the board expertise. Yet, today’s standard of care is largely a one-size-fits-all approach where children are treated with toxic chemotherapy and radiation (the same drugs administered to adults, only in smaller amounts) and sometimes invasive surgeries. As a first step toward a more precise, tailored treatment, children who are eligible will be offered a new test called GPS Cancer that combines the power of DNA and RNA sequencing with quantitative proteomics (vs. only genomic sequencing), thereby providing a more accurate picture of the cancer patient’s profile at the protein level. It is an invaluable tool in beginning to deliver more targeted therapies.

“It is simply the best diagnostic science available today,” says Leonard Sender, MD, a founding member of the Consortium and the medical director of the Hyundai Cancer Institute at Children’s Hospital Orange County (CHOC Children’s). Dr. Sender continues, “This is the first comprehensive analysis of tumors in conjunction with the evaluation of the normal genome of the patient offered in a CAP- and CLIA-approved Illumina X based lab facility. In addition, we will be evaluating the parents’ genomes as well to determine the basis of possible cancer predisposition genes.”

The utter comprehensiveness of the test coupled with the fact that it can be completed in one lab, with results returned in days instead of weeks, provides an invaluable jumpstart to developing a customized treatment as fast as possible, wasting no precious time in the fight to save children’s lives.

Dr. Leonard Sender, CHOC Children's 

Step 2: A Treatment That Promises Best Lifetime Outcomes

A major tenet of the Cancer Breakthroughs 2020 program is accelerating combination targeted therapy and immunotherapies, not just single agents, but “cocktails,” if you will, of approved and novel agents to “harness the immune system,” as Dr. Soon-Shiong says, not kill it.

The Pediatric Cancer Consortium believes the promise of immunotherapy is especially crucial to producing more positive outcomes for children. Not only does it offer hope for keeping more children alive and living with cancer by keeping it in check much as we would HIV or another chronic illness, but it also preserves the childhood of the patient during treatment by reducing negative side effects caused by more toxic therapies.

It’s tragic enough when full grown adults get cancer, but their bodies and brains are fully developed. Many of these adults have already started families and, once they have recovered from chemo or radiation, they may be able to return to active roles at home and work – returning to life they had before. But, for children whose bodies and brains have not yet fully developed, the effects are radically different. Traditional maximum chemotherapy and radiation doses kill the immune system as fast, if not faster, than they can kill the cancer. For growingchildren, the difference between these harsh therapies and immunotherapy is enormous. “When a child is treated for cancer with toxic therapies, the ramifications are tremendous,” explains Giannoula Laka Klement, MD, Pediatric Hematologist/Oncologist and Assistant Professor, Tufts University School of Medicine. “Sometimes cancer treatment leaves them sterile. They won’t be able to have children. They have to be out of school – out of their social circle for a long time. They may see a doctor monthly for the rest of their lives based on something caused by the drugs. They might be learning disabled. Their young bodies and minds are still developing. So, if we have the option to treat them with something that may not leave them sterile, may not leave them with learning disabilities, and may keep them from staying in a hospital for long periods of time, we should do it.”

Step 3: Accelerating Advances to Kids in Desperate Need

The formation of a separate Pediatric Consortium has been of the utmost importance as, unfortunately, children are often the last to see advancements in cancer treatments. Advancements collected via clinical trials often must be vetted with adults first – and the process is long. Then, even if the results show promise, and that promise becomes publicized, the new drugs must then go through a lengthy approval process to be used with children. Many new medications take 8-12 years to bring to the market, with some taking up to 19 years. Unless the trial agent being tested is specifically designed for pediatric use, given the way clinical trials are currently run, approval and access to new children’s medicines will almost always lag behind those for adults.

For many reasons, this tedious process is the right approach. As Dr. Sender points out, “Ethically, when we know a treatment already works most of the time, even if it is toxic and uses chemotherapy or radiation, we have to administer it. It wouldn’t be right to try a novel or unproven approach when we know the standard treatment will likely work.” But for the difficult cases, the clinical trials of Cancer Breakthroughs 2020 can help address the advancement gap that still exists with certain pediatric cancers, eventually reaching a point where targeted therapy can be used to treat disease initially.

As Dr. Sender further shares, “There are some cancers for which we have made no progress in 20 years – maybe longer. In situations where we know the standard therapy doesn’t always work, or there has been no improvement, or there is late stage or even current or refractory cancer, there is now some hope that we will find informative treatments. This (combination immunotherapy) can change that and bring advances to children faster.”

Supplementing an Already Dedicated System

It is important to note that, in spite of the current limitations of Pediatric Cancer Care, the global pediatric oncology community does make new breakthroughs. World class organizations such as the Children’s Oncology Group, or COG, have led the way in bringing Phase I and novel trials to children with cancer. In fact, children with cancer participate in clinical trials at a significantly higher rate than adults (about 80% children versus 3-5%, respectively). Therefore, the Pediatric Cancer Consortium is not seeking to replace the current approach – this is far from the case. Combination Immunotherapy is additive – meant to complement current treatments that are proving effective by providing hope for children with grim prognoses – who suffer with cancers that have no current effective treatment.

As Dr. Sender attests, “It is important to note that this is not in competition with the COG or trying to take anything away from that. There are many Phase I clinical trials, and we will continue to leverage that community. Again, this therapeutic approach is additive -- a supplemental approach to applying immunotherapy in the most difficult of cases where it was not available before, and then taking those results in real time and using that information to impact other children.” Additionally, the trials will be more accessible to kids – they won’t necessarily have to travel, and many hospitals are tackling the funding issue – ensuring kids who need the genomic testing will be able to get it.

Bringing New Hope to Patients and Families

When treating cancer, time is of the essence. Our children shouldn’t be the last to benefit from innovative, leading edge therapies. Through the combined efforts of the Pediatric Cancer Consortium, the treatment of childhood cancer is facing a new dawn filled with hope. As Dr. Giannoula Laka Klement has said, “This is not about just testing anybody’s drugs, but individualizing treatment so we are at a point where children will be living with cancer – with the emphasis on living. We will keep them alive in school. We will keep them alive with their friends, their family. This therapy potentially will not affect reproductive potential and will reduce long stints in a hospital. The hope is that our patients will live with cancer without the disease.”

This is our hope – for healing, for life, and for a future filled with opportunities to give the gift of childhood back to children suffering from cancer – a hope that one day soon, life-saving immunotherapies will be available to every boy and girl, and that no parent ever again has to ask, “why not my child?”