
Pediatric oncologist Kim Kramer led the clinical study demonstrating the effectiveness of a new drug for neuroblastoma that has spread to the brain.
Editor’s note: When the original version of this story was published in June 2017, the drug was named burtomab. The name was changed to omburtamab in December 2017.
Great strides have been made in the treatment of neuroblastoma, a rare cancer of nerve tissue that usually occurs in children and typically arises in the abdomen. However, with current therapies, the aggressive form of this cancer will spread, or metastasize, to the brain. At that point, it becomes nearly impossible to control.
Now, there is hope for people facing this devastating situation. A drug created at Memorial Sloan Kettering has reached an important milestone toward becoming a treatment option for patients. The FDA has granted the drug, omburtamab, Breakthrough Therapy Designation for the treatment of neuroblastoma that has spread to the brain.
Omburtamab is a monoclonal antibody that binds to the surface of neuroblastoma cells. When it is linked to a radioisotope — a version of a chemical element that emits radiation — it becomes a drug suitable for a treatment known as radioimmunotherapy. It is injected into the spinal fluid and delivers precision liquid radiation to strike the cancer cells dead.
Omburtamab was created and tested under the name 8H9 by MSK physician-scientist Nai-Kong Cheung, who heads MSK’s Neuroblastoma Program. The drug has been licensed by MSK to Y-mAbs Therapeutics.
“Now, we have children living for years after receiving 8H9,” Dr. Cheung says. “This treatment is evidence-based and offers real hope and even cure that was previously unthinkable. I’m so glad the FDA has granted the Breakthrough Therapy Designation, since there is no approved drug to treat neuroblastoma that has spread to the brain.”
The designation was given to the drug on the basis of a clinical study of 105 children with metastatic neuroblastoma who had all been treated at MSK since 2003. Those who received it in addition to conventional treatment with chemotherapy and radiation survived significantly longer than children who never received the drug. Celebrating the ten-year cancer-free milestone is now a reality for those first recruits.
The FDA grants Breakthrough Therapy Designation when early clinical data indicate that a drug may demonstrate substantial improvement over existing therapies. The designation is intended to speed up the review process of these drugs.
In addition to demonstrating the drug’s effectiveness, the trial also showed it to be safe. The approach targets the cancer cells with precision, while sparing the surrounding brain tissues from damage. This is especially important for pediatric patients because more conventional treatments, especially external beam radiation therapy, can cause learning deficits and developmental delays in young children.
Pediatric oncologist Kim Kramer, who led the clinical study, says the results of the trial — and possible approval of omburtamab by the FDA — could change the course of the disease at this late stage. Doctors at other institutions are beginning to take notice and express interest in getting this therapy to their patients — which is now routinely given in the outpatient Pediatric Day Hospital at MSK — to maximize their quality of life.
“We have met with several colleagues in the United States and Europe to help facilitate a formal collaboration that will permit omburtamab to be administered at their centers,” Dr. Kramer says. “It’s super exciting to move this forward beyond the success that we’ve had with the drug at MSK, and to enable children throughout the world the chance to receive this lifesaving treatment.”
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.
Comments
Cathy Rezmer
Jun 14, 2017 • 2:11 PM
James Cucco
Jun 23, 2017 • 9:33 AM
Noreen Slager
Jun 23, 2017 • 10:08 AM
I too lost a child to brain cancer. Dawn passed away in 1987, at the age of 13. Never a day goes by that I don't think of her, and think of how different things could have been with some effective treatment! There was no treatment for her at all, back in 1987. This article made me feel very hopeful. Not for my Dawn, of course, but for the so many other children suffering from brain cancer. Life extended 10 years- WOW. Dawn lived every minute of the one year she fought so hard for. Imagine the quality of life for these kids and their families! Imagine the joy and the gratitude all around. Thank you, MSK!!
Memorial Sloan Kettering
Jun 23, 2017 • 1:24 PM
In reply to I too lost a child to brain… by Noreen Slager
Barbara Spieth
Jun 23, 2017 • 11:23 AM
Memorial Sloan Kettering
Jun 23, 2017 • 1:21 PM
In reply to So grateful for the research… by Barbara Spieth
Albert Promislow
Jun 27, 2017 • 2:51 PM
Memorial Sloan Kettering
Jun 27, 2017 • 3:23 PM
In reply to Is this treatment useful for… by Albert Promislow
Stacy Rocco
Jul 8, 2017 • 10:37 AM
My daughter is a survivor of NB...you all are the true heroes in our society! You sacrifice everyday to try and find a cure to help these little warriors! You all should be the ones that we tell our children to look up to and to celebrate! Thank you for all you do every day unnoticed and without wanting anything in return...except a cure!
Memorial Sloan Kettering
Jul 10, 2017 • 11:02 AM
In reply to My daughter is a survivor of… by Stacy Rocco
Sarita Sasa
Aug 5, 2017 • 7:42 PM
Memorial Sloan Kettering
Aug 7, 2017 • 3:45 PM
In reply to Is Burtomab available for… by Sarita Sasa
Sarita, thank you for reaching out. We are sorry to hear about your daughter’s condition. Currently burtomab is being used only for neuroblastoma that has spread to the brain.
If you are interested, you can learn about neuroblastoma treatment at MSK here:
https://www.mskcc.org/pediatrics/cancer-care/types/neuroblastoma
Michiel Coene
Aug 10, 2017 • 5:46 AM
My son (2 years and four months old ) was diagnosed with stage IV Neuroblastoma at the beginning of 2017. In May we've learned that, in spite of a very good response during induction therapy, the cancer had become refractory and metastasised in the brain.
He is currently undergoing additional TVD treatment under SIOPEN 1.7, but the cancer has failed to respond in a satisfactory manner. Reading this has me wondering if he is eligible for treatment with Burtomab.
Memorial Sloan Kettering
Aug 10, 2017 • 12:05 PM
In reply to My son (2 years and four… by Michiel Coene
Dear Michiel, we’re very sorry to hear about your son’s diagnosis. If you would like to have a consultation with someone at MSK about the possibility of him being treated here, you can call 800-525-2225 or go to https://www.mskcc.org/experience/become-patient/appointment for more information on making an appointment. Thank you for your comment, and best wishes to you and your family.
Brittany Brown
Dec 20, 2017 • 3:32 PM
My son Wyatt was diagnosed with stage 4 neuroblastoma at 9 months. MSKCC you are all amazing and because of you and our local children's hospital Wyatt is now playing with his brother and sister like a regular kid. Thank you Dr. Heaton for getting the last bit of cancer out of my baby! I am forever grateful for each of you. I pray this new treatment gives lots of years, laughs and hope back to families fighting this horrible cancer.
Memorial Sloan Kettering
Dec 21, 2017 • 10:54 AM
In reply to My son Wyatt was diagnosed… by Brittany Brown
Michael N
Oct 12, 2018 • 4:13 AM
I am reading some of the replies above from MSK saying that omburtamab or the 8H9 is currently only offered for neuroblastoma patients. Dear MSK, can you confirm if that really is the case? As far as I know many kids with all kinds of brain malignancies expressing B7-H3 have been treated with the 8H9. It would also appear that B7-H3 is expressed in most adult brain malignancies. Does MSK have any plans to expand the 8H9 studies to adults as a result? Thank you
Beethoven Coronel
May 14, 2019 • 3:01 PM
Memorial Sloan Kettering
May 15, 2019 • 9:25 AM
In reply to There is a metastatic… by beethoven coronel
Dear Beethoven, we’re very sorry to hear about this. If this patient or his or her parents are interested in learning more about treatment at MSK, they can contact our International Center at international@mskcc.org. Thank you for your comment.
My son, Shane, was treated at MSKCC for Neuroblastoma back in 2003. Shane's cancer was too advanced back then for any of the options that were available. I am SO happy that new treatments are still being discovered to give the kids a chance to live longer lives.