FDA Grants Breakthrough Therapy Designation to Burtomab for Metastatic Neuroblastoma

By Jim Stallard,

Monday, June 12, 2017

Head shot of smiling female doctor in civilian clothes with arms folded.
Summary

Aggressive forms of neuroblastoma will spread to the brain even if initial treatment is effective. A new drug called burtomab offers real hope and even a cure for children with this disease. The drug delivers precision liquid radiation to strike the cancer cells dead. It was created at MSK and has received Breakthrough Therapy Designation from the FDA.

Highlights
  • Neuroblastoma is a rare cancer of nerve tissue.
  • The disease sometimes spreads to the brain.
  • A drug called burtomab, created and tested at MSK, is an effective treatment.
  • The drug received Breakthrough Therapy Designation from the FDA.

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, burtomab, Breakthrough Therapy Designation for the treatment of neuroblastoma that has spread to the brain.

Burtomab 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.

Burtomab 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.”

This treatment is evidence-based and offers real hope and even cure.
Nai-Kong V. Cheung
Nai-Kong V. Cheung Head, Neuroblastoma Program

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 burtomab 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 burtomab 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.”

Comments

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.

I hope you will find a diagnostic application Burtomab to detect spread or recurrence throughout the body.

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!!

Dear Noreen, we are very sorry for your loss. Thank you for your kind words, and best wishes to you.

So grateful for the research and treatment at MSKCC. You have given HOPE as well as given back a normal life to a very special 5 year old with Neuroblastoma.

Dear Barbara, thank you for your comment!

Is this treatment useful for adults?

Dear Albert, this treatment is only for neuroblastoma, which occurs almost exclusively in pediatric patients. Thank you for your comment.

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!

Dear Stacy, we're so glad to hear your daughter is a survivor! Thank you for your kind words.

Is Burtomab available for Neuroblastoma patients that has no cancer sites in their brain? My daughter has stage 4 Neuroblastoma but no cancer activity in her brain. Thank you MSK for your research.

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.

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.

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.

Dear Brittany, we are so happy to hear that Wyatt is doing well. We will forward your comment to Dr. Heaton. Thank you for your message, and best wishes to you and your family.

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