“I’m working to develop better treatments for children with devastating diseases”

Working with adult patients suffering from a debilitating disease is difficult. Working with children suffering from a debilitating disease?

“It’s devastating,” says Kerri Nottage, M.D.

During her days as a pediatric hematologist and oncologist at St. Jude Children’s Research Hospital, Dr. Nottage—now Global Medical Head, Oncology, Janssen Pharmaceutical Companies of Johnson & Johnson—cared for kids who’d undergone stem cell transplants. While the transplants often worked, anywhere from 52% to 65% of those patients would go on to develop chronic graft versus host disease (cGVHD), a condition that occurs if the stem cells that cured the cancer start attacking the body’s healthy tissue.

“You just want to do better for those patients,” Dr. Nottage says.

Kathleen Lomax, M.D., a pediatric gastroenterologist and Medical Director, Immunology, Janssen, relates to this sentiment, though her focus area within the company is slightly different: pediatric psoriatic arthritis (PsA), an autoimmune disease that causes chronic joint inflammation and swelling and can be difficult to treat.

“When you look at diseases in the pediatric population, they’re often more severe at presentation than what you find in adults, and it really pulls at your heartstrings,” says Dr. Lomax. “The need to develop medications for kids—ones that work and that they’re able to tolerate—is crucial.”

For Childhood Cancer Awareness Month, read on to find out more about the work of these physician-scientists and what motivates them to keep fighting for children’s futures, in their own words.

Innovating to Treat Children With Chronic Graft Versus Host Disease

Kerri Nottage, M.D., Global Medical Head, Oncology, Janssen

“During my time as a pediatric hematologist and oncologist at St. Jude Children’s Research Hospital, I treated a lot of young patients who had allogeneic stem cell transplants to treat their cancer. This procedure involves transferring stem cells from a healthy person—either a related or unrelated donor—to a patient after chemotherapy or radiation.

In the best-case scenario, those donated cells will move through the patient’s bloodstream to the bone marrow, where they’ll begin to grow and produce healthy new blood cells—a process called engraftment. This essentially creates a new immune system, which is then able to attack any remaining cancer cells in the patient’s body.

It’s an incredibly effective treatment for certain types of cancer, including leukemias and lymphomas. Unfortunately, the journey for these patients isn’t always over after a transplant. The sad truth is that the treatment that successfully cured their cancer can sometimes lead to another life-threatening disease.

An unfortunate complication that can happen is a condition called chronic graft versus host disease (cGVHD). The condition occurs when stem cells from the donor (a.k.a. the graft) start to attack healthy cells in the patient’s body (a.k.a. the host). While almost any organ can be affected, the most common areas that come under attack are the skin, mouth, liver and lungs. If the cGVHD is really bad, some kids can’t straighten their joints or breathe easily. I’ve had patients who needed a lung transplant after their bone marrow transplant because their cGVHD was so severe.

It’s heartbreaking for parents to watch their child struggle with the debilitating effects of cGVHD, especially since there are so few treatment options. Steroids are currently the first line of treatment for these children, but this can be challenging. cGVHD symptoms tend to flare and subside, meaning that patients are usually on and off steroids for years at a time, potentially making this an unsustainable treatment.

Another treatment option for children with cGVHD may include medicines called kinase inhibitors, which can be used when systemic therapy, like steroids, doesn’t work well enough. Until very recently, if a child was between ages 1 and 12 and the steroids didn’t work, we had no other U.S. Food and Drug Administration (FDA)-approved options for them.

You never forget the patients you treat. It’s important for us to continue our commitment to these children and their families to help ensure there are treatment options available that can make a difference. I’ll keep pushing until there’s something out there that can both prevent deaths and give those patients who do survive a better quality of life.”

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A New Way to Help Ease the Pain of Active Pediatric Psoriatic Arthritis

Kathleen Lomax, M.D., Medical Director, Immunology, Janssen

“Active psoriatic arthritis (PsA) shows up in so many different ways in young patients. Sometimes kids have psoriasis—a skin disease that causes itchy, scaly patches on the skin—and later they develop an arthritic component, where one or more of their joints start to ache. Sometimes symptoms start in the opposite order, with arthritic findings initially; later, the patient develops psoriasis.

That’s partially why active pediatric PsA can be a difficult disease to diagnose and to treat. Because it’s often an invisible disease, it can be especially tough for kids to deal with. So many young patients with active PsA talk about how nobody understands that they’re sick. They may be in a lot of pain, but because their joints aren’t necessarily visibly red or even obviously swollen, people may not believe them. I’ve heard families tell heartbreaking stories about these kids not getting the accommodations they need at school, for example. The concept that kids can have an inflammatory arthritis doesn’t make sense to people who think arthritis is something only the elderly experience.

This disease is quite rare: It affects just 5% to 8% of children and adolescents with chronic inflammatory arthritis. And while we don’t understand exactly why it happens, we do know it’s an autoimmune disease, which means for some reason the body starts attacking itself. In the case of active PsA, symptoms can show up in many areas of the body, though the most common areas are the joints, skin and eyes.

The main goals when treating psoriatic arthritis in pediatric patients are to reduce joint inflammation, maintain mobility and prevent deformity. For many years, we’ve had several approved prescription medications to treat psoriatic arthritis in adults, but we haven’t had the same number of options approved to treat kids. That’s because pediatric drug development is difficult. It often only happens after a medication for adults has been approved. Understandably, the FDA wants to ensure that medications for pediatric patients and their proper dosages are tested thoroughly so that they’re as safe and effective as they can be.

Given these (and other!) hurdles that come along with doing pediatric research, scientists often try to extrapolate information using data from previous research—something that helps us get drugs to the pediatric populations who need them quicker than we’d otherwise be able to.

That’s exactly what happened with this recent FDA approval of the pediatric indication of an existing Janssen therapy. The drug works as an anti-inflammatory in the body, blocking specific cytokines thought to play an important role in tempering the overactive inflammatory response in several autoimmune diseases, including active PsA. It was first approved to treat adults living with moderate to severe plaque psoriasis; then this indication was expanded to treat a pediatric population. Using the existing data we had from those psoriasis studies, as well as existing data in active PsA among adults, we were able to prove the drug’s effectiveness in treating active PsA in a certain pediatric population.

And that’s a beautiful thing! Of course, it’d be really exciting to have the answer to why kids develop PsA in the first place. If we knew exactly why some get it—and why it manifests in such different ways—it would help facilitate faster diagnoses and more innovative treatments. In the meantime, it feels good to work for a company committed to pediatrics and to getting life-changing medications to our youngest patients as fast as possible.”