Drug Delivery Approach Shows Promise for Treating a Deadly Brain Tumor in Children

Illustration of brain in green with pons area highlighted in orange.

DIPG tumors begin in the middle region (orange) of the brain stem, which regulates many critical body functions. A new study suggests that a method for delivering drugs to these pediatric tumors could be safe and effective.

Brain tumors are notoriously hard to treat with drugs. Treatments such as chemotherapy have trouble getting through the aptly named blood-brain barrier. This membrane is very selective about allowing substances to pass from the bloodstream into the brain. Most drugs given by IV never make it to tumors in high enough concentrations to be effective. As a result, progress in treating some tumors has been agonizingly slow or even nonexistent.

Results from a phase I clinical trial led by researchers at Memorial Sloan Kettering and Weill Cornell Medicine now suggest that treatment of some of the most difficult brain tumors may have taken a major step forward. The study tested a new drug delivery technique called convection enhanced delivery (CED). The findings indicate that CED appears safe and effective at distributing a drug throughout a fatal pediatric brain tumor called diffuse intrinsic pontine glioma (DIPG). The encouraging results are published in the journal The Lancet Oncology.

This is the most exciting thing I've done in my career by far.
Mark M. Souweidane pediatric neurosurgeon

DIPG tumors begin in the brain stem. This area at the base of the brain regulates many critical body functions, such as breathing, heart rate, and swallowing. DIPG is very difficult to treat because of its location and because the tumor cells can infiltrate normal brain tissue. Surgery is out of the question. The only traditional option has been radiation treatment, which has a minimal effect. Children with DIPG tend to live just a year or less.

The new study provides hope that drugs can be delivered more efficiently to DIPG tumors — and possibly other tumors located deep in the brain.

“This is somewhat groundbreaking because no one has taken CED into the brain stem with any type of systematic clinical trial,” says Mark Souweidane, a pediatric neurosurgeon at MSK and Weill Cornell who led the study. “All we had before were a few anecdotal cases. This trial shows we can use this very powerful drug-delivery platform repeatedly and safely.”

Slowly Pushing from Cell to Cell

The CED approach for DIPG involves slowly infusing the drug through tubes inserted deep into the brain stem. The delivery time lasts up to 12 hours. This extended flow allows the drug to gently push through the fluid compartment between cells in the tumor due to tiny differences in pressure. The drug saturates more of the tumor than has been possible through other delivery techniques.

Mark Souweidane, DIPG specialist and pediatric neurosurgeon at Memorial Sloan Kettering Cancer Center
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So far, CED has been tested more on adult brain tumors, such as glioblastoma, another aggressive cancer. Dr. Souweidane thinks the drug delivery technique is better suited for DIPG because these tumors are smaller and restricted to a tighter area.

The research underpinning the use of CED for DIPG was an exhaustive effort conducted at Weill Cornell, where Dr. Souweidane is Director of Pediatric Neurological Surgery and Co-Director of the Children’s Brain Tumor Project. The clinical trial, which began in May 2012, took place at MSK.

In the trial, 28 children with DIPG who had already received radiation therapy to the tumor were given a drug called 124I-8H9 using CED. This drug consists of an antibody linked to a radioactive substance. The antibody binds to a protein on the surface of brain tumor cells, and the radiation emitted kills the cancerous cells. MSK physician-scientist Nai-Kong Cheung created 124I-8H9. The drug has already proven effective in treating metastatic neuroblastoma to the brain.

At seven different dose levels, the delivery method appeared safe in children with DIPG. Researchers determined that 124I-8H9 was well distributed through the tumors by tracking the radioactive substance using PET/CT scans and MRI. Most impressively, the investigators were able to prove that drug concentrations in the tumor were more than a thousandfold higher than anywhere else in the body — a remarkable improvement over what is typical. These results validated using CED for children with DIPG.

The trial did not examine whether this drug delivery approach caused the children to survive longer. It did establish that CED merits further development as a treatment for children with DIPG.

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Remaining Challenges

Dr. Souweidane says that data gathered from this trial will guide the next steps toward refining the technique. How much of the drug made it into the tumor, how long it stayed there, the best ways to image and measure results, and other vital information will be assessed to come up with the best therapeutic strategy.

There are still many challenges to overcome. Researchers need to know how much of the tumor must be permeated for the drug to be effective. It also needs to be firmly established that children with DIPG truly benefit from this delivery approach. But proving the feasibility of the technique was a major hurdle.

“This has been revolutionary in my mind,” Dr. Souweidane says. “Our pharmacologists look at the results and say, ‘Where has this been for as long as we’ve been trying to treat these brain tumors?’”

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A Long Journey

The success of the trial represents a milestone in a long journey for Dr. Souweidane. He has been studying DIPG and CED’s therapeutic potential for more than two decades. Preclinical work at Weill Cornell enabled Dr. Souweidane to test CED’s safety, efficacy, and proper dosage in rodents, using the findings to refine the technique. The lack of progress in treating the disease, especially compared with other childhood cancers, has taken a heavy emotional toll.

In 2016, Dr. Souweidane’s mission to cure DIPG was highlighted by photographer Brandon Stanton for the popular photo blog Humans of New York as part of a pediatric cancer series Mr. Stanton was shooting at MSK. The stories inspired a huge number of people to take action: Overnight, Dr. Souweidane’s laboratory received $1.2 million in donations to help him find a cure for the devastating disease.

These funds were not used for the clinical trial, which was already well under way. But they are now being applied to accelerate the next steps of the process to make it better.

Dr. Souweidane says this will involve a soon-to-be-opened trial through the Pediatric Brain Tumor Consortium, a collaboration among 11 academic centers and children’s hospitals in the United States. The trial could begin as early as late 2018.

“This is the most exciting thing I’ve done in my career by far,” he says. “I’ve been in this for 30 years, and you just watch these kids die with no alternative. It’s constant, constant turmoil and tragedy. It’s amazing to think you’re on the verge of something big.”

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This research was supported by NIH grant P30CA008748, the Dana Foundation, The Cure Starts Now, Solving Kids’ Cancer, the Lyla Nsouli Foundation, Cookies for Kids’ Cancer, the Cristian Rivera Foundation, Battle for a Cure, Cole Foundation, Meryl & Charles Witmer Charitable Foundation, Tuesdays with Mitch Charitable Foundation, and Memorial Sloan Kettering.


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How could one enter the new clinical trial? 11 year old girl with DIPG

Thank you for this information; I am delighted that you are finding some promising results in your research! A dear friend's 26-year-old son passed away from DIPG just recently, after fighting it for 40 months post diagnosis. Your work is necessary to give families at least some hope. Too late for Ryan, sadly, but I have tears in reading this report, knowing that some day we may have an effective treatment. Thank you and God Bless each of you!

Dear Joyce, we are very sorry to hear about your friend’s son. Thank you for your comment.

Could this be possibly effective for a hemangiopericytoma? My brother has one diagnosed 1996, has had 3 resection attemps, 4 gamma knife treatments, and finally chemo most recently...all attempts have now ceased and the tumor has now doubled since 10/2107 and further infiltrated the brainstem.

Dear Susan, we’re sorry to hear your brother has been through all this. If he would like to come to MSK for a consultation and to find out about treatment options, including clinical trials, the number to call is 800-525-2225. You can go to https://www.mskcc.org/experience/become-patient/appointment for more information or to schedule online. Thank you for your comment, and best wishes to you and your brother.

Hello, I have called once before and was told that my daughter did not qualify for dipg trial not sure why, could we please try again we are in desperate need of help! We are willing to do anything at this point she is my only child and radiation/chemo not working. Thank you!

Dear Amber, please call our Department of Pediatrics at 833-MSK-KIDS. Thank you for your comment, and best wishes to you and your family.

Hi, is this clinical trial available for an adult with DIPG?

Angelica, thank you for reaching out. This particular trial is open to patients from 2 to 21 years old:


A similar trial has just opened for patients 3 to 21 years old:


You can learn about other DIPG trials at these links:



Best wishes to you.

My daughter 7 years old just diagnosed with DIPG , we are having a lot issues since we don’t have insurance, can we still participate in the study ???

Hello, I’m excited to read about this research. Can this trial be applied to an adult who is 30 years old? He just got diagonized 3 months ago. What else could be done? Appreciate your response. Looking for some hope.

Dear Urvish, our current protocol is only open for patients up to 2 years of age. We are excited about the results, however, and hope to extend the indications soon for other tumor types and ages. Thank you for your comment and best wishes to you.

My son has been diagnosed with dipg at the beginning of august,I live in south africa I want to if he is qualified to use the drug

Hello. My 5 year old son has just been diagnosed yesterday. We are starting radiotherapy on monday. We live in Switzerland. Do we qualify for the trial after the 6 weeks?

Hi my son Steven got diagnosed with DIPG on June 7 of this year, 5 days after his wedding. steve is 28 and lives in Buford Ontario
He has had 6 weeks of radiation and tumour is
still swollen from the radiation.
Would he qualify for CED or any other type of treatment

Dear Barb, we’re very sorry to hear about your son’s diagnosis. Unfortunately this trial would not be open to your son because of his age. If he would like to speak with someone at MSK about what other treatment options he may have, he can contact our International Center at [email protected] or go to https://www.mskcc.org/experience/become-patient/international-patients for more information. Thank you for your comment and best wishes to you and your family.

My 7 year old granddaughter has a friend, same age, who was diagnosed with a DIPG about two months ago. Will one of the 11 new trial sites to possibly begin late 2018 be at Children's Hospital of Pittsburgh?

Dear Sherry, we’re sorry to hear about your granddaughter’s friend. To learn about the locations for this upcoming trial, we recommend that you read out to the Pediatric Brain Tumor Consortium. You can find out how to reach them on their website, https://www.pbtc.org/.

Thank you for your comment.

My 51 year old brother has been diagnosed with a very rare case of medulloblastoma as an adult. Unfortunately, since he is an adult with a pediatric cancer, he is unable to be in exciting studies like yours. Maybe in the future, you may consider opening up your studies to advance the treatment of the disease in general, as I imagine in the future the victims will extend to a broader population.

Dear Emily, we’re very sorry to hear about your brother’s diagnosis. Although most clinical trials for pediatric cancers are limited by age, we may still have treatment options available for him. If he would like to schedule an appointment to learn more, he can call 800-525-2225 or go to https://www.mskcc.org/experience/become-patient/appointment for more information. Thank you for your comment, and best wishes to both of you.

Dear all,
My brother is 40 years old and has a DIPG. He is been treated with chemiotherapy and I wonder if there is more new about this news of last year.
Is it still in trials? There has been some developmemt in the research?
Thank you so mucho,
Kind regards,

Dear Alejandra, we’re very sorry to hear about your brother’s diagnosis. If he would like to arrange a consultation with an MSK expert to talk about treatment options, he can make an appointment online or call 800-525-2225. Thank you for your comment and best wishes to both of you.