In 2017, a treatment developed at MSK for children with a rare nerve tissue cancer that spreads to the brain received Breakthrough Therapy Designation from the FDA. The hope is that the drug will soon be available for patients everywhere.
Researchers at MSK have made huge strides in treating neuroblastoma, a rare nerve-tissue cancer that often spreads to the brain and is most common in young children. The disease was once uniformly fatal, but now, many children can survive it and go on to lead full, happy lives. MSK treats more people with neuroblastoma than any other institution in the world.
Medical oncologist Nai-Kong Cheung, head of MSK’s neuroblastoma program, created and tested a drug to treat neuroblastoma called omburtamab in clinical trials. When linked to a radioactive element and injected into the spinal fluid, the drug delivers precision liquid radiation to strike cancer cells dead. In 2017, the US Food and Drug Administration granted the drug a Breakthrough Therapy Designation for neuroblastoma that has spread to the brain. In 2018, the same designation was given to naxitamab, another drug born out of MSK research. The designation is given when early clinical data indicate a drug demonstrates substantial improvement over existing therapies. Both of these drugs have been licensed to a biopharmaceutical company called Y-mAbs, which is focused on further developing these treatments and bringing them to patients outside of MSK. Y-mAbs was founded by the father of an MSK neuroblastoma survivor.
Pediatric oncologist Kim Kramer headed the clinical trial involving 105 children that led the FDA to give omburtamab Breakthrough Therapy Designation. Dr. Kramer is also researching new treatments to eliminate microscopic tumor cells in the central nervous system.
The researchers sat down to talk about their efforts, what keeps them going when treatments don’t go as planned, and what they appreciate about doing this work at MSK.
Nai-Kong Cheung: I bet you often get asked, “Why did you go into oncology?” But I have a different question: What keeps you in oncology? How do you sustain yourself?
Kim Kramer: I’m really moved by the strides we’ve made in treating neuroblastoma. It used to be a death sentence. But now we have kids who have survived for decades, grown into adults, and gone on to have their own kids. And the FDA giving a Breakthrough Therapy Designation to omburtamab gives us even more cause for hope.
This fall will be the 20th anniversary of our first celebration for survivors of neuroblastoma. It’s been a real privilege to see these kids who had previously been given fatal diagnoses grow up and visit us to celebrate.
Dr. Cheung: I think pediatric oncology faces a lot of challenges. First, in young patients it’s often difficult to give anything that is really toxic. They may have to live with the consequences for a long time.
The second thing is that there are very few pediatric patients. So that means the pharmaceutical industry doesn’t pay as much attention. They’re not going to make as much money developing drugs for pediatric patients. That’s the way it is. Numbers count. This is the problem those of us in pediatric oncology have faced for decades. How do you get anything done, make a drug, prove that it works, and after all that have someone make it? Because a therapy can be great and help children on clinical trials at MSK, but it cannot be used anywhere else.
That’s why it’s so important when treatments like omburtamab reach important milestones in approval from the Food and Drug Administration, so they can hopefully help more kids.
Dr. Kramer: There are some organizations that are trying to make drug accessibility equal for adults, young adults, and children. There’s the RACE for Children Act [Research to Accelerate Cures and Equity] that was just passed earlier in 2018, which will mandate that drugs in development are accessible for pediatric patients as well. So hopefully the increased awareness at that level will improve things.
When it comes to developing great new therapies, there really is a multidisciplinary team effort. My department, pediatric oncology, is just one part of this. It’s the nuclear medicine team, the medical physics team, the pharmacology team, the radiation safety officers, and all the other departments that buy into putting forth the time and effort to make something succeed. The dedication and loyalty and effort and diligence and experience are phenomenal here.
Dr. Cheung: This brings us full circle to the question we began with, about why we stay in oncology. For me, I know that I need to meet a standard. It’s almost as if you’re trying to improve on your own self. In fact, you try to surpass yourself. You want to be a better doctor. And as you get better, the institution gets better. And that is the goal.
Nai-Kong Cheung has received commercial research grants from Y-mAbs Therapeutics and is a part owner of the company. Dr. Cheung is the inventor and owner of issued patents licensed by MSK to Y-mAbs.