{"id":19172,"date":"2024-11-13T16:00:06","date_gmt":"2024-11-13T16:00:06","guid":{"rendered":"https:\/\/lpfch.org\/?p=19172"},"modified":"2025-05-30T20:44:10","modified_gmt":"2025-05-30T20:44:10","slug":"tumor-research-breakthrough","status":"publish","type":"post","link":"https:\/\/lpfch.org\/tl\/impact-stories\/tumor-research-breakthrough\/","title":{"rendered":"Ang Malawak na Suporta sa Donor ay Nagtutulak ng Mahalagang Pambihirang Pambihirang Pagsaliksik sa Pediatric Brain Tumor Research"},"content":{"rendered":"

This week, an extraordinary breakthrough at the Stanford School of Medicine brings hope to children and families facing devastating brain or spinal cord cancers typically considered incurable. The <\/span>new study, published in <\/span>Natur<\/span><\/i>e<\/span><\/a>, demonstrates exciting promise for an immune-cell therapy in treating childhood tumors for which no effective therapy currently exists.\u00a0<\/span>\u00a0<\/span><\/p>\n

An astounding level of supp<\/span>ort from a passionate donor base fueled this game-changing discovery. Donations of all sizes, alongside tissue donations from grieving families, propelled the research. Michelle Monje, MD, PhD, the <\/span>Milan Gambhir Professor of Pediatric Neuro-Oncology <\/span><\/i>and lead author of the study, says, \u201cThese results\u2014which make me more hopeful about a cure than I have ever been\u2014owe so much to philanthropy and the families and foundations that have put their faith in our research.\u201d <\/span>\u00a0<\/span><\/p>\n

The study explores the use of <\/span>CAR T-cell therapy<\/span><\/a> to treat deadly cancers of the brain or spinal cord occurring most commonly in children\u2014including diffuse intrinsic pontine glioma\u00a0(DIPG), which impacts the brainstem. Not only is the clinical trial one of the first to demonstrate CAR T-cell efficacy against any solid tumor, the results are more encouraging than even the researchers had dared imagine. \u201cOne of the biggest surprises was how much clinical benefit we saw,\u201d said the study\u2019s senior author, Crystal Mackall, MD, the <\/span>Ernest and Amelia Gallo Family Professor<\/span><\/i> and professor of pediatrics and of medicine. <\/span>\u00a0<\/span><\/p>\n

The therapy used in the clinical trial also recently received an FDA designation of regenerative medicine advanced therapy, or RMAT\u2014available only to a small group of therapies with immense potential to treat life-threatening conditions. This designation is a major milestone, signaling that the agency will work closely with researchers to fast-track the path to FDA approval. <\/span>\u00a0<\/span><\/p>\n

A Bleak Disease\u2014and a Ray of Hope\u00a0<\/strong><\/p>\n

A diagnosis of DIPG is exceptionally grim: The median survival time following a diagnosis is less than one year, and the five-year survival rate is below 1%. Existing chemotherapy drugs are useless against the tumors, radiation offers only temporary relief, and the tumors are inoperable because malignant cells are enmeshed with healthy cells in the brain and spinal cord. <\/span>\u00a0<\/span><\/p>\n

The disease can result in profound disability as the fast-growing tumors progress. Among other effects, patients may lose their ability to walk, smile, swallow, hear, and talk. They might also experience neuropathic pain resulting from spinal cord damage, as well as paralysis, loss of sensation, and incontinence.<\/span>\u00a0<\/span><\/p>\n

It\u2019s against this dire backdrop that the results of the new Stanford study arrive. In a clinical trial with 11 participants receiving the therapy, nine patients showed clinical and\/or imaging benefits, as well as improvement in disabilities. Additionally, in four participants, the volume of tumors decreased by <\/span>more than half<\/span><\/i>.<\/span> And one trial participant, <\/span><\/b>Drew<\/span><\/b><\/a>, had a complete response: His tumor disappeared entirely from brain scans.<\/span><\/b> He\u2019s healthy now, four years after his diagnosis. The magnitude of this result\u2014for a cancer that has until now been universally fatal\u2014is hard to overstate.<\/span><\/b>\u00a0<\/span><\/p>\n

An Outpouring of Donor Support\u00a0\u00a0<\/strong><\/p>\n

Over the last decade, donor support has catalyzed Monje\u2019s research. New research ideas typically do not qualify for federal funding, and in the early stages of her investigations, philanthropy was essential to getting the research off the ground. Now, the rapid pace of Monje\u2019s continuing discoveries\u2014urgently awaited by desperate families\u2014continues to owe much to passionate donor support.<\/span>\u00a0<\/span><\/p>\n

In fact, few areas of pediatric research have ever received the type of overwhelming support that has fueled the progress in Monje\u2019s lab over the last 14 years. The cumulative dollar amount of philanthropic support for her research\u2014more than $25 million\u2014is tremendous. <\/span>Equally staggering is the fact that Monje has received gifts at all levels, ranging from $5 to $5 million, from a vast donor base comprising <\/span><\/b>more than 1,000 individuals and foundations. <\/span><\/i><\/b>\u00a0<\/span><\/p>\n

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\n
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\n Bring Hope to Kids with Brain Tumors\n <\/h2>\n\n

\n Join us on this extraordinary journey. Make a contribution to drive life-saving research in the Monje lab.\n <\/p>\n \n

\n \n Give now\n \n <\/a>\n \n <\/div>\n <\/div>\n\n <\/div>\n <\/div>\n<\/section>\n\n

The story of one early donor, Maiy\u2019s Miracle, is especially poignant. In 2014, after 4-year-old <\/span>Maiyanna<\/span><\/a> passed away from DIPG, her mother, Mycah, donated her tumor tissue to Monje\u2019s lab. Mycah hoped the tissue donation would drive research and someday prevent other families from experiencing unbearable loss. <\/span>\u00a0<\/span><\/p>\n

Mycah also organized fundraisers in her Pittsburgh community, including a fashion show and a dance\u2014raising more than $6,000 toward Monje\u2019s research. This funding turned out to be pivotal. The money was used to award a summer research scholarship in 2016 to a Stanford undergraduate who screened DIPG tumor cultures in Monje\u2019s lab. His work that summer helped the team understand that a sugar molecule, GD2, was abundantly found on the surface of most DIPG tumors. Today, this sugar molecule is being targeted by the CAR T-cell therapy used in Monje and Mackall\u2019s clinical trial.<\/span>\u00a0<\/span><\/p>\n

\u201c<\/span>Even small gifts applied at important leverage points can make a big difference,\u201d says Monje.<\/span>\u00a0<\/span><\/p>\n

In several other instances, the timing of the gifts has been especially striking. For example, Unravel Pediatric Cancer\u2014a foundation started by another family who lost their child to DIPG\u2014approached Monje with a significant donation just as she was exploring the next big step: testing a GD2-targeting CAR T-cell therapy in mice.<\/span>\u00a0<\/span><\/p>\n

The incredible strides made by Monje\u2019s lab would not have been possible without support that ran deep. <\/span>Tissue d<\/span><\/b>onations from families suffering immense loss, as well as financial contributions from a wide base of donors\u2014many of whom made gifts in memory of a child or young adult\u2014helped launch and sustain the research at every stage.<\/span><\/b>\u00a0<\/span><\/p>\n

\u00a0\u201cSo many donors, foundations, and families have supported DIPG research,\u201d says Monje. \u201c[M]any families have even made the unthinkable gift of donating their child\u2019s brain tumor. They have all helped to expedite our work. Every single contribution, large or small, enables us to move forward without delay and do all we can to help families fighting this terrible disease.\u201d<\/span>\u00a0<\/span><\/p>\n

An Only-at-Stanford Collaboration\u00a0<\/strong><\/p>\n

The extensive show of donor support over the last decade is a testament not just to the desperate need for an effective treatment for DIPG and related cancers, but also to the trailblazing science at the heart of Monje\u2019s research. And Stanford, it turned out, was the perfect setting for this boundary-pushing science.<\/span>\u00a0<\/span><\/p>\n

Once her lab had identified a potential target in the sugar molecule GD2, Monje fostered a new relationship that would later seem fated. Mackall, a prominent investigator known for her work pioneering immune therapies for pediatric cancers, had recently come to Stanford\u2014and she just happened to work in the same building, one hall over. In fact, Mackall had already reached out to explore the potential of using immunotherapy on\u00a0childhood brain tumors. As Monje tells it, once her\u00a0student\u00a0discovered\u00a0GD2 on the surface of tumor cultures, the path was clear. Monje simply walked over to Mackall\u2019s office. Monje knew that Mackall\u2019s team had already engineered a CAR T-cell therapy targeting\u00a0GD2\u2014so she asked whether they could work together to test it on DIPG and related tumors. This casual overture ended up launching what has proven to be an incredibly rich and fruitful collaboration.<\/span>\u00a0<\/span><\/p>\n

In 2018, Monje and Mackall celebrated a significant milestone when they published a study in <\/span>Nature Medicine<\/span><\/i> showing that CAR T-cell therapy targeting GD2 could make<\/span> DIPG tumors disappear in mice<\/span><\/a>. The next stage of their collaboration\u2014a human trial\u2014was risky, due to the potential for swelling in areas like the brainstem. Monje and Mackall built in as many safeguards as possible and began the clinical trial in 2020. In 2022, they published <\/span>the first promising results of this trial in <\/span>Nature<\/span><\/i><\/a>. \u00a0<\/span>\u00a0<\/span><\/p>\n

Now, in the latest findings of the ongoing clinical trial, published this week in <\/span>Nature<\/span><\/i>, the research shows huge potential to change the course for children and families facing DIPG and similar cancers.\u00a0<\/span>\u00a0<\/span><\/p>\n

A Brighter Future Ahead\u00a0<\/strong><\/p>\n

Drew, the clinical trial participant who had a complete response, was diagnosed with DIPG in 2020, during his junior year of high school. As his tumor grew, he found he needed a wheelchair to get around. In June 2021, Drew received his first infusion of CAR T cells at Stanford. Though it was difficult, especially early on in the trial, he continued receiving infusions in his senior year, participating in classwork from home. <\/span>\u00a0<\/span><\/p>\n

By spring 2022, Drew had improved to the point of returning to school and was able to navigate the hallways with a rolling walker. That May, he walked across the graduation stage unassisted, to a standing ovation. Now, four years after his diagnosis, Drew\u2019s brain scans are tumor-free, and he is healthy, walking easily, and attending college. He dreams of making a career in restoring natural areas, a conservation strategy known as rewilding.<\/span>\u00a0<\/span><\/p>\n

\u201cI\u2019m hoping they\u2019ll learn from all my success to help other kids,\u201d says Drew. His family is also constantly thinking of others facing the same devastating diagnosis. \u201cThere\u2019s a lot of loss that led to this research,\u201d says Drew\u2019s dad. \u201cI hope those families know that this success is because of them.\u201d\u00a0<\/span>\u00a0<\/span><\/p>\n

For Monje, who first cared for a young child with DIPG 20 years ago as a medical student, and who has since devoted herself to finding effective treatments, Drew\u2019s result only adds to her determination. The team\u2019s analysis suggests the participants\u2019 responses fall on a normal bell curve\u2014meaning Drew\u2019s complete response is not a fluke. The researchers are working hard to investigate how they can improve on the CAR T-cell therapy\u2014as well as its application to cancers beyond DIPG\u2014with the goal of seeing more results like Drew\u2019s and answering the hopes of more families. <\/span>\u00a0<\/span><\/p>\n

Join us on this extraordinary journey and m<\/span>ake a contribution<\/span> to<\/span> <\/span>support <\/span>the<\/span> vital research<\/span> of the Monje lab<\/span><\/span><\/a>.<\/span><\/span>\u00a0<\/span><\/em><\/p>\n","protected":false},"excerpt":{"rendered":"

Sa linggong ito, ang isang pambihirang tagumpay sa Stanford School of Medicine ay nagdudulot ng pag-asa sa mga bata at pamilyang nahaharap sa mapangwasak na mga kanser sa utak o spinal cord na karaniwang...<\/p>","protected":false},"author":7,"featured_media":19173,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","footnotes":""},"categories":[164],"class_list":["post-19172","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-research","health_topic-pediatric-cancer"],"acf":[],"yoast_head":"\nExpansive Donor Support Drives Pivotal Breakthrough in Pediatric Brain Tumor Research - 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