Materiały źródłowe

• #1 Medulloblastoma Treatment | St. Jude Care & Treatment

  https://www.stjude.org/care-treatment/treatment/childhood-cancer/brain-tumors/medulloblastoma.html

  Treatment of medulloblastoma includes surgery, chemotherapy, and in most cases radiation therapy. […] Surgery removes part or all of the tumor tissue. This takes out cancerous tissue and helps determine the type of tumor. […] Chemotherapy (chemo) is important in the treatment of these tumors. The exact type of chemotherapy and the length of treatment will depend on your child’s age and type of tumor. […] Radiation: The dose, location, and timing of radiation will depend on many factors. These include your child’s age, type of tumor, and if it has spread to other parts of the brain or spine. […] At St. Jude, the treatment of medulloblastoma differs by the molecular group and subgroup, extent of its spread at the time of diagnosis (metastatic state), and age at diagnosis. […] Our goal is to give each child the most effective therapy to cure their tumor while sparing them unnecessary treatment to improve their long-term quality of life.

• #1 Medulloblastoma: Diagnosis and Treatment – NCI

  https://www.cancer.gov/rare-brain-spine-tumor/tumors/medulloblastoma

  The first treatment for medulloblastoma is surgery, if possible. The goal of surgery is to obtain tissue to determine the tumor type and remove as much tumor as possible without causing more symptoms for the person. […] Most people with medulloblastomas also receive additional treatments. These may include radiation, chemotherapy, or clinical trials. Because this disease tends to spread in the brain and spine, surgery is usually followed by radiation to the entire brain and spine (called craniospinal radiation). Proton beam radiation therapy is often recommended, because of problems with bone marrow and surrounding organs that occur due to brain and spine radiation. […] Chemotherapy is often part of the treatment plan, depending on the tumor subtype, how much the tumor has spread, how the person responds to treatment, the side effects, and the impact on their quality of life.

• #1 Medulloblastoma – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/medulloblastoma/diagnosis-treatment/drc-20579280

  Treatment for medulloblastoma usually includes surgery followed by radiation or chemotherapy, or both. Your healthcare team considers many factors when creating a treatment plan. These factors might include the tumor’s location, how fast it’s growing, whether it has spread to other parts of the brain and the results of tests on the tumor cells. Your care team also considers your age and your overall health. […] Treatment options include: […] Surgery to relieve fluid buildup in the brain. A medulloblastoma may grow to block the flow of cerebrospinal fluid. This can cause a buildup of fluid that puts pressure on the brain. To reduce the pressure, a surgeon can create a pathway for the fluid to flow out of the brain. Sometimes this procedure can be combined with surgery to remove the tumor.

• #1 Treatment Options for Medulloblastoma and CNS Primitive Neuroectodermal Tumor (PNET)

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5026188/

  The amount of residual disease after surgery better correlates with survival for medulloblastomas than for CNS PNETs. Maximal surgical resection of tumor should be done, only if additional permanent, neurologic deficits can be spared. Patients should have a staging work-up to assess the extent of disease. This includes postoperative magnetic resonance imaging (MRI) of the brain, MRI of the entire spine and lumbar cerebrospinal fluid (CSF) sampling for cytological examination, if deemed safe. […] Radiation therapy to the entire CNS axis is required, with a greater dose (boost) given to the region of the primary site or any bulky residual disease for older children. Adjuvant chemotherapy must be given even if no evidence of disease after radiation therapy exists, as the risk of relapse is substantial after radiation alone.

• #1 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  Studies that incorporate molecular profiling to guide adjuvant therapy are underway. […] The current recommendation is 23.4 Gy to the craniospinal axis, followed by a boost of 30.6 Gy to the primary tumor site. […] In general, the start of radiation should be timed for no later than 4 weeks from surgery to maximize clinical outcomes. […] Reducing the amount of craniospinal radiation in an attempt to decrease morbidity without jeopardizing survival appears to be successful in this group. […] In a report by the International Society of Pediatric Oncology, children with average-risk medulloblastoma randomly received either the standard 36 Gy or a reduced dose of 24 Gy to the neuraxis. […] It was found that no statistical difference in progression-free survival rates was demonstrated between the groups as long as the initiation of radiotherapy was not delayed by the administration of chemotherapy before radiation.

• #1 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  Following upfront maximal safe surgical resection, the standard of care approach for medulloblastoma is dependent on risk stratification and generally consists of chemotherapy and radiotherapy. […] New treatment approaches are being developed in an attempt to reduce long-term toxicity of therapy and include reduction of the total dose or volume of radiotherapy, use of less neurotoxic chemotherapeutic agents and newer targeted therapeutic agents, and use of high-dose chemotherapy with autologous stem cell rescue in lieu of craniospinal irradiation (CSI). […] Although there is insufficient evidence to routinely recommend proton therapy, for younger children with medulloblastoma, strong consideration should be given to proton-beam radiotherapy when available. Compared with conventional radiotherapy, proton-beam therapy provides similar efficacy but offers the advantage of minimizing the risk of long-term adverse effects including but not limited to ototoxicity, cardiopulmonary toxicity, and secondary malignancies.

• #1 Childhood Medulloblastoma and Other CNS Embryonal Tumors (PDQ®) – NCI

  https://www.cancer.gov/types/brain/hp/child-cns-embryonal-treatment-pdq

  The treatment of younger children with newly diagnosed medulloblastoma continues to evolve. Results have been variable, and comparison across studies has been difficult because of differences in the drug regimens used and the utilization of craniospinal and local boost radiation therapy at the end of chemotherapy or when children reached age 3 years in some studies. […] Standard treatment options for younger children with newly diagnosed medulloblastoma include the following: Surgery. Adjuvant chemotherapy. […] In patients between the ages of 3 and 4 years, and possibly as old as age 5 years, some investigators have recommended that radiation therapy be delayed or omitted entirely. […] For children older than 3 years with average-risk medulloblastoma, standard treatment options include Surgery, Adjuvant radiation therapy, and Adjuvant chemotherapy.

• #1 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  Radiation therapy for patients younger than 3 years, the poorest risk group, is generally avoided because of its deleterious effects on intellectual development, especially in this age group. […] In lieu of this, in an effort to delay or omit radiation, current protocols utilize high-dose chemotherapy approaches. […] Cytotoxic chemotherapy may be used in the initial treatment (in infants), as maintenance therapy (for average and high-risk disease) or for disease recurrence. […] In patients less than 3 years of age, incorporation of high dose chemotherapy strategies may delay or obviate the need for radiation therapy. […] Standard therapy post-radiation is with the use of cisplatin-based regimens. […] Many contemporary chemotherapy protocols use a combination of cisplatin, vincristine, lomustine (CCNU) and cyclophosphamide as maintenance therapy for approximately one year following radiation therapy.

• #1 Advances in medulloblastoma treatment for children – Mayo Clinic Comprehensive Cancer Center Blog

  https://cancerblog.mayoclinic.org/2024/05/29/advances-in-medulloblastoma-treatment-for-children/

  Though rare, medulloblastoma is the most common cancerous brain tumor in children. Medulloblastoma treatment depends on tumor type and your child’s age. „Treatment usually involves safely removing as much of the tumor as possible, followed by radiation therapy and chemotherapy,” says Dr. Schwartz. […] In children younger than 4, Dr. Schwartz says care teams use radiation-sparing techniques, which reduce damage to healthy tissue, and high doses of chemotherapy followed by stem cell transplant (bone marrow transplant). […] If medulloblastoma recurs, Dr. Schwartz says care teams turn to other aggressive treatments, including chemotherapy and clinical trials for vaccines, chimeric antigen receptor (CAR)-T cell therapy, and other viral therapies. „It’s an aggressive disease. That’s why we treat it aggressively. If medulloblastoma recurs, we know treatment is generally not curative,” he says.

• #1 Treating Specific Types of Childhood Brain and Spinal Cord Tumors | American Cancer Society

  https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-children/treating/specific-types-of-tumors.html

  Embryonal tumors (including medulloblastomas) tend to grow quickly and spread through the spread to cerebrospinal fluid (CSF). […] Medulloblastomas: These tumors start in the cerebellum. They tend to grow quickly and are among those most likely to spread outside the brain (usually to the bones or the bone marrow). But they also tend to respond well to treatment. […] Children with medulloblastoma are often divided into standard-risk and high-risk groups, depending on certain factors. Those in the high-risk group usually get more intensive treatment than children in the standard-risk group. […] Medulloblastomas are removed with surgery when possible, followed by radiation therapy to the area where they started. High doses of radiation are aimed at the area of the tumor. […] For children younger than 3, doctors try to use as little radiation as possible. Chemotherapy is typically the first treatment given after surgery. Depending on how the tumor responds, chemotherapy might be followed by radiation therapy. […] There are some reports that giving high-dose chemotherapy followed by an autologous stem cell transplant may be helpful for some children with medulloblastomas. Several clinical trials are now studying this.

• #1 Advances in medulloblastoma treatment for children – Mayo Clinic Comprehensive Cancer Center Blog

  https://cancerblog.mayoclinic.org/2024/05/29/advances-in-medulloblastoma-treatment-for-children/

  Based on molecular diagnostic tests, medulloblastoma tumors are now categorized into four subgroups, and healthcare professionals understand that each requires a different treatment approach. „We now know that you shouldn’t treat them all the same. We know that the five-year overall survival for WNT-activated medulloblastoma is 100%. Other subgroups have lower survival or are likely to recur,” says Dr. Schwartz. […] Dr. Schwartz is conducting a clinical trial to study whether reduced doses of radiation therapy and chemotherapy can effectively treat patients with newly diagnosed medulloblastoma tumors in WNT-driven medulloblastoma, the least aggressive tumor subgroup. […] Care teams now have a tool to protect children with medulloblastoma from chemotherapy-related hearing loss. In 2022, the FDA approved the use of sodium thiosulfate to reduce the risk of cisplatin-associated hearing loss in children one month and older who have solid tumors that have not spread.

• #1 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  In the largest trial conducted for average-risk medulloblastoma by the Children’s Oncology Group (COG), survival rates following reduced radiation therapy boost volumes were comparable to standard treatment volumes for the primary tumor site, but lower CSI doses were associated with higher relapse rates and worse survival outcomes. […] Studies to determine the feasibility of avoiding radiation therapy altogether in WNT-activated medulloblastoma, a subgroup with excellent prognosis, are ongoing. […] The current recommendation is 36 Gy to the craniospinal axis, followed by a boost of 19.8 Gy to the primary tumor site and an additional 19.8 Gy to focal metastatic sites. […] In general, the start of radiation should be timed for no later than 4 weeks from surgery to maximize clinical outcomes.

• #1 Childhood Medulloblastoma and Other CNS Embryonal Tumors (PDQ®) – NCI

  https://www.cancer.gov/types/brain/hp/child-cns-embryonal-treatment-pdq

  In high-risk patients, numerous studies have demonstrated that multimodality therapy improves the duration of disease control and overall disease-free survival. […] The optimal treatment for children with SHH-activated, TP53-altered medulloblastoma has not been determined, as less than 30% of patients are expected to survive 5 years from diagnosis after treatment with current therapy. […] There are no standard treatment options for recurrent childhood CNS embryonal tumors. […] Treatment approaches may include Surgery, Radiation therapy, Chemotherapy, High-dose chemotherapy with stem cell rescue, and Molecularly targeted therapy.

• #1 The Cure for Childhood Cancer | The Medulloblastoma Initiative

  https://mbinitiative.org/what-is-medulloblastoma/

  Clinical trials with new medications may also be an option for some patients. Clinical trials enroll eligible participants to study new treatments, such as immunotherapy, or new ways of using existing treatments, such as different combinations or durations of radiotherapy and chemotherapy. These studies offer patients the opportunity to try the most recent treatment options, although the risks of side effects may not be fully known. […] Since its creation in 2021, the MBI has been raising resources to find a cure for Group 4 medulloblastomas. MBI has successfully secured financial support for a consortium of 14 laboratories in the United States, Germany, and Canada (the Cure Group 4 Consortium), led by Dr. Roger Packer from Childrens National Hospital. […] In addition, in under 3 years, two clinical trials have also already been approved by the U.S. Food and Drug Administration (FDA) to test two new treatments:

• #1 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  Medulloblastoma is the commonest malignant brain tumour of childhood. Disease relapse following maximal multi-modal therapy including upfront craniospinal irradiation (CSI) is almost always fatal and occurs in approximately 30% of patients. Importantly, patients rarely die of other causes, and consequently relapsed medulloblastoma (rMB) accounts for 10% of all childhood cancer deaths. There is a variety of life-prolonging treatment options at relapse including neurosurgery, re-irradiation, early-phase trials, and chemotherapy. Here, we summarise best practice for investigations including re-biopsy and molecular characterisation of rMB, treatment decision making, and available treatment options. We also highlight advances in the understanding of rMB disease biology and prognostic factors, and look towards future developments such as targeted therapies, liquid biopsies and disease modelling strategies to improve outcomes in this area of unmet clinical need.

• #1 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  The choice to either not commence or discontinue ‘active’ treatment and provide symptom-modifying treatment as best supportive care only should always be considered and openly discussed. […] Neurosurgery should be considered to resect an isolated nodular lesion as this treatment may prolong survival, re-biopsy for the purpose of detailed pathological and molecular characterisation including exclusion of second malignancy, and palliative diversion of CSF to enhance quality of life. […] Re-irradiation offers the potential to decelerate tumour progression, reduce symptoms, and in some cases, provide long-term disease control. […] Given the dire prognosis of rMB following upfront multi-modal standard therapy, the aim of chemotherapy either as the sole treatment at relapse or combined with local therapy (neurosurgery and/or re-irradiation) is typically to prolong life with minimal side-effects/hospital admissions.

• #1 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  High-dose chemotherapy, defined as administration of intensive chemotherapy with autologous stem-cell support has been utilised in rMB for over two decades. […] Several case series have reported the use of temozolomide, irinotecan and bevacizumab for rMB. […] Immunotherapy is under development in many different forms, and has shown great potential in pre-clinical and early-phase clinical trials. However, despite the remarkable clinical efficacies observed in a number of malignancies, the use of immunotherapy alone may not be sufficiently active for many patients. Therefore, it is likely that the real potential for most patients, including those with r/rMB, lies in combining different immunotherapies with other treatments such as radiotherapy and chemotherapy to target tumour heterogeneity, overcome immunosuppression, and improve immune infiltration.

• #1 New Approaches in Targeted Therapy for Medulloblastoma in Children | Anticancer Research

  https://ar.iiarjournals.org/content/41/4/1715

  Medulloblastoma (MB) is the most frequent malignant brain tumor in children. Treatment of MB is based on histopathological and molecular stratification, and includes surgical intervention, often with craniospinal irradiation and adjuvant chemotherapy. Unfortunately, however, this treatment leads to a high morbidity rate, and it does not cure all patients either, with around 30% succumbing to their disease. […] With improved cancer genomics and better molecular characterization, MB has been classified into four major subgroups, wingless-activated, sonic hedgehog-activated, Group 3, and Group 4, with each group consisting of additional subtypes. Recently disclosed genetic drivers of MB may in the future help improve treatment, and in this way reduce therapy-related toxicity. In this review, we describe the heterogeneity of the MB subgroups, and potential new options for targeted therapy.

• #1 New Approaches in Targeted Therapy for Medulloblastoma in Children | Anticancer Research

  https://ar.iiarjournals.org/content/41/4/1715

  Current standard therapy, mainly composed of maximal surgical resection followed by CSI and chemotherapy, presents severe treatment-related toxicity, and secondary tumors may also occur. Reducing these consequences without affecting treatment efficacy is the focus of targeted therapy, aiming to reduce the doses of cytotoxic agents and radiation, as well as optimizing overall survival. […] In WNT and SHH MB, some driver mutations and inhibitors interfering in aberrant signaling pathways or targeting somatic mutations have been identified, and are intensively studied in vitro and in vivo. […] One approach is to epigenetically or directly inhibit the transcription factor GLI, which is considered to be much more efficient than inhibition of SMO, since GLI is a terminal effector of HH signaling and initiates the transcription of HH target genes.

• #1 5 Innovative Medulloblastoma Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/medulloblastoma/medulloblastoma-treatment.html

  At MD Anderson, medulloblastoma treatment is carefully designed by a team of highly specialized experts. […] Advanced technologies and less-invasive treatment options, such as proton therapy, BrainSuite (intraoperative MRI) and new targeted therapies provide the best hope for successful treatment with the least impact on physical and mental function. […] We are also pioneering the use of immunotherapy, which unleashes the body’s own immune system against disease, as a treatment for recurrent medulloblastoma. […] Thanks to advanced treatments, more than two-thirds of children with medulloblastoma can be treated successfully. […] Medulloblastoma treatment is customized based on several factors, including, the patients age and health, the size and location of the tumor, and whether the cancer has spread to other parts of the nervous system or the body.

• #1 Overcoming Treatment Resistance in Medulloblastoma: Underlying Mechanisms and Potential Strategies

  https://www.mdpi.com/2072-6694/16/12/2249

  The blood-brain barrier (BBB) is a layer of specialized cells that surround the brain and protects it from pathogens and toxic metabolites. […] The blood-brain barrier becomes problematic during the management of different brain cancers—including medulloblastoma—because of how it can inhibit chemotherapeutic drug delivery. […] One prominent driver of treatment resistance in cancers is the ATP-binding cassette transporter superfamily. […] Several efforts have been channeled towards targeting the different drivers that promote the development and maintenance of treatment resistance in medulloblastoma. […] The mTOR pathway is also frequently associated with all subgroups of medulloblastoma. […] The ABCB receptor family, which act as ATP-dependent multidrug pumps, are also key regulators of chemoresistance in SHH medulloblastoma.

• #1 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  Medulloblastoma is a type of brain cancer found mostly in children. UCSF is studying a drug called DFMO to see if it can help patients with high-risk or returning medulloblastoma. Another study is testing a flavored oral medicine, Irinotecan, with Temozolomide for treating recurring pediatric tumors. UCSF is also creating personalized treatment plans based on each patient’s tumor. […] Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma. […] A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally administered irinotecan VAL-413 (Orotecan) given with temozolomide for treatment of recurrent pediatric solid tumors including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma and medulloblastoma.

• #1 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  Standard therapy for newly diagnosed average-risk or low-risk medulloblastoma includes surgery, radiation therapy, and chemotherapy (including cisplatin). […] The overall goals of this study are to see if giving STS along with standard treatment (radiation therapy and chemotherapy) will reduce hearing loss in medulloblastoma patients and to compare the overall outcome of patients with medulloblastoma treated with STS to patients treated without STS on a previous study in order to make sure that survival and recurrence of tumor is not worsened. […] This phase III trial studies different chemotherapy and radiation therapy regimens to compare how well they work in treating young patients with newly diagnosed, previously untreated, high-risk medulloblastoma. […] This randomized phase III trial is studying two different combination chemotherapy regimens to compare how well they work in treating young patients with newly diagnosed supratentorial primitive neuroectodermal tumors or high-risk medulloblastoma when given before additional intense chemotherapy followed by peripheral blood stem cell rescue.

• #1 Medulloblastoma Treatment | St. Jude Care & Treatment

  https://www.stjude.org/care-treatment/treatment/childhood-cancer/brain-tumors/medulloblastoma.html

  All patients receive surgery, radiation to the brain and spine, and chemotherapy. But the doses of radiation and the number of chemotherapy courses depend on the molecular group of medulloblastoma. […] St. Jude is a leader in the treatment of certain molecular groups, such as WNT-activated medulloblastoma. […] For other more aggressive molecular groups, such as group 3, we also lead the way in adding new therapies to help improve prognosis. […] All medulloblastoma patients get supportive care that includes physical therapy, speech therapy, occupational therapy, and neurocognitive therapy. […] St. Jude clinical trials were the first to introduce treatment of medulloblastoma by the tumors molecular type. This continues today. […] We offer clinical trials and research studies for children, teens, and young adults with medulloblastoma. […] Our brain tumor clinical trials in medulloblastoma have led to lower-intensity treatments based on molecular class of medulloblastoma.

• #1 Treatment Options for Medulloblastoma and CNS Primitive Neuroectodermal Tumor (PNET)

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5026188/

  The treatment of medulloblastoma and CNS PNET can cause significant impairment in neurologic function. Evaluations by physical therapy, occupational therapy, speech therapy and neurocognitive assessments should be obtained, as needed. After therapy is completed, survivors need follow-up of endocrine function, surveillance scans and psychosocial support. […] Tissue must be obtained via neurosurgery to render a definitive diagnosis. Most medulloblastoma tumors are amenable to total resection if they do not extensively infiltrate the cerebellum and brainstem. […] Medulloblastoma and CNS PNET are radiosensitive tumors and RT remains the single most effective postoperative treatment. It is conventional practice to irradiate the entire craniospinal axis. […] Chemotherapy given during and after RT for patients with standard risk medulloblastoma has resulted in 5-year event free survival and overall survival (OS) of 81 % and 86 % in a cohort of 379 patients treated in prospective, phase III trial.

• #1 Medulloblastoma | Radiology Reference Article | Radiopaedia.org

  https://radiopaedia.org/articles/medulloblastoma?lang=us

  Medulloblastomas are the most common malignant brain tumors of childhood, most often presenting as midline masses in the roof of the 4th ventricle (at the superior medullary velum) with associated mass-effect and hydrocephalus. Treatment typically consists of surgical resection, radiation therapy, and chemotherapy, with the prognosis strongly influenced by surgical resection, the presence of CSF metastases at the time of diagnosis, molecular and histological features and expression of the c-erbB-2 (HER2/neu) oncogene. […] Treatment typically consists of surgical resection, radiation therapy, and chemotherapy. In general, the tumors are quite radiosensitive. A VP shunt is required in approximately 35% of children who undergo resection. […] Prognosis is most strongly influenced by molecular subtype: WNT: very good; SHH: infants good, others intermediate; group 3: poor; group 4: intermediate.

• #1 Medulloblastoma: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/22591-medulloblastoma

  Length of treatment can vary from person to person. Those who undergo any type of brain surgery usually need at least four to eight weeks to recover. For chemotherapy or radiation therapy, treatment could last several weeks or several months, depending on the case. […] In many cases, yes. While medulloblastoma has the potential to spread throughout your entire nervous system, many people can be cured. There’s a higher chance of survival if the medulloblastoma hasn’t spread to other parts of your brain and spinal cord. […] The five-year medulloblastoma survival rate is over 80%. This means that over 80% of all people diagnosed with medulloblastoma are still alive five years later.

• #1 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  Prognosis following relapse is dismal, with less than 5% of patients surviving; disease relapse is therefore the most adverse prognostic factor in MB. Furthermore, patients diagnosed with MB rarely die of other causes; less than 2% of patients who relapse die of treatment complications. Consequently, rMB accounts for a considerable, and disproportionate amount of childhood cancer deaths (~10%) and remains an urgent area of clinical unmet need. […] Here, we describe the current understanding of prognostic factors at disease relapse such as principal molecular group, adverse molecular biology, and timing of relapse. We provide an overview of the clinical diagnostic process including signs and symptoms, staging investigations, and molecular pathology, followed by a summary of treatment modalities and considerations. Finally, we summarise future directions to progress understanding of treatment resistance and the biological mechanisms underpinning early therapy-refractory and relapsed disease.

• #1 Treatment and prognosis of medulloblastoma – UpToDate

  https://www.uptodate.com/contents/treatment-and-prognosis-of-medulloblastoma

  Treatment consists of a combined-modality approach that includes surgery, radiation therapy (RT), and chemotherapy in most patients. […] Long-term survival is now achieved in approximately three-quarters of all patients with medulloblastoma, though specific risk groups have varied survival outcomes. […] The optimal initial treatment of patients with medulloblastoma includes both general measures to alleviate increased intracranial pressure and specific therapy directed against the tumor. […] Based upon results from multiple cooperative group trials, the preferred approach in most patients includes a combination of maximal safe surgical resection, radiation therapy (RT) to both the tumor site and the craniospinal axis, and systemic chemotherapy. […] The application of this combined-modality approach to different risk groups is discussed below.

• #1 How we treat medulloblastoma in adults

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8207184/

  Due to the tumour’s typical location in the posterior fossa, patients with medulloblastoma often present with symptoms of increased intracranial pressure, hydrocephalus, cerebellar signs with gait disturbances or ataxia of the extremities and signs of leptomeningeal disease. […] Magnetic resonance imaging (MRI) is the modality of choice to assess and follow up medulloblastoma. […] Gross total resection should be carried out, if possible, without harming the patient. In cases where a gross total resection is not possible, maximum safe resection should be carried out. […] Adults with medulloblastoma should be treated with radiotherapy of the craniospinal axis, with a boost to the tumour bed. […] Adults with medulloblastoma should be treated with systemic therapy, regardless of their risk category.

• #2 Treatment and prognosis of medulloblastoma – UpToDate

  https://www.uptodate.com/contents/treatment-and-prognosis-of-medulloblastoma

  Treatment consists of a combined-modality approach that includes surgery, radiation therapy (RT), and chemotherapy in most patients. […] Long-term survival is now achieved in approximately three-quarters of all patients with medulloblastoma, though specific risk groups have varied survival outcomes. […] The optimal initial treatment of patients with medulloblastoma includes both general measures to alleviate increased intracranial pressure and specific therapy directed against the tumor. […] Based upon results from multiple cooperative group trials, the preferred approach in most patients includes a combination of maximal safe surgical resection, radiation therapy (RT) to both the tumor site and the craniospinal axis, and systemic chemotherapy. […] The application of this combined-modality approach to different risk groups is discussed below.

• #2 5 Innovative Medulloblastoma Treatment Options | MD Anderson Cancer Center

  https://www.mdanderson.org/cancer-types/medulloblastoma/medulloblastoma-treatment.html

  Medulloblastoma treatment almost always includes surgery to remove as much of the tumor as possible as safely as possible to minimize damaging the surrounding brain tissue. […] After surgery, most children receive chemotherapy to destroy any remaining cancer cells. […] All adults with medulloblastoma receive radiation therapy to the brain and the spine (craniospinal radiation) after surgery to stop or slow the growth of any residual tumor that cannot be removed surgically and any tumor cells that may have spread into the cerebrospinal fluid. […] MD Anderson uses the most advanced radiation treatment methods, including proton therapy, which delivers high radiation doses directly to the brain tumor site, minimizing damage to nearby healthy tissue. […] Targeted therapies are specially designed to treat each cancers specific genetic/molecular profile to help the body fight the disease. […] Medulloblastoma is treated at our Children’s Cancer Hospital and our Proton Therapy Center. […] MD Anderson patients have access to clinical trials offering promising new treatments that cannot be found anywhere else.

• #2 Medulloblastoma: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/22591-medulloblastoma

  Medulloblastoma is a cancerous brain tumor that starts near your brainstem in your cerebellum. The tumor is fast-growing and can spread to other areas of your brain and spinal cord. Treatments include surgery, radiation therapy and chemotherapy. […] The most common medulloblastoma treatment involves surgery followed by chemotherapy, radiation therapy or both. […] Your oncologist may give you chemotherapy drugs after surgery, after radiation therapy or in combination with radiation therapy. In some instances, a stem cell transplant is done after chemotherapy. […] This treatment can get rid of cancer cells that remain after surgery. Your healthcare provider may use it in combination with chemotherapy. […] A neurosurgeon can do surgery to relieve the pressure buildup on your brain or to remove the tumor. Sometimes, complete removal of the tumor isn’t possible because it forms near structures deep within your brain.

• #2 Childhood Medulloblastoma and Other CNS Embryonal Tumors (PDQ®) – NCI

  https://www.cancer.gov/types/brain/hp/child-cns-embryonal-treatment-pdq

  Surgery is considered a standard part of treatment for histological confirmation of tumor type and as a means to improve outcome. Total or near-total resections are considered optimal if they can be performed safely. […] Radiation therapy to the primary tumor site is usually in the range of 54 Gy to 55.8 Gy. In most instances, this therapy is given with a margin of 1 cm to 2 cm around the primary tumor site, preferably by conformal techniques. […] Chemotherapy, usually given during and after radiation therapy, is a standard component of treatment for older children with medulloblastoma and other embryonal tumors. Chemotherapy can be used to delay and sometimes obviate the need for radiation therapy in 20% to 40% of children younger than 3 to 4 years with nondisseminated medulloblastoma.

• #2 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  In the largest trial conducted for average-risk medulloblastoma by the Children’s Oncology Group (COG), survival rates following reduced radiation therapy boost volumes were comparable to standard treatment volumes for the primary tumor site, but lower CSI doses were associated with higher relapse rates and worse survival outcomes. […] Studies to determine the feasibility of avoiding radiation therapy altogether in WNT-activated medulloblastoma, a subgroup with excellent prognosis, are ongoing. […] The current recommendation is 36 Gy to the craniospinal axis, followed by a boost of 19.8 Gy to the primary tumor site and an additional 19.8 Gy to focal metastatic sites. […] In general, the start of radiation should be timed for no later than 4 weeks from surgery to maximize clinical outcomes.

• #2 Advances in medulloblastoma treatment for children – Mayo Clinic Comprehensive Cancer Center Blog

  https://cancerblog.mayoclinic.org/2024/05/29/advances-in-medulloblastoma-treatment-for-children/

  Dr. Schwartz says families should seek care from healthcare professionals who can offer a comprehensive team approach and the latest technology. […] Proton beam therapy, which delivers precise radiotherapy with lower doses of radiation to healthy tissue, is also important for children whose organs are still developing, says Dr. Schwartz.

• #2 Medulloblastoma | Children’s brain tumours

  https://www.cancerresearchuk.org/about-cancer/childrens-cancer/brain-tumours/types/medulloblastoma

  The doctor decides on further treatment based on the results of the tests and other factors. […] Your child’s specialist usually avoids using radiotherapy to the whole brain and spine if your child is younger than 3. This is to reduce the risk of developing long term side effects, which is higher in children younger than 3. […] Chemotherapy is the use of anti cancer drugs to destroy cancer cells. […] Your child’s specialist might recommend chemotherapy instead of whole brain and spine radiotherapy. This aims to keep your child’s tumour under control until radiotherapy is likely to cause less damage. […] Generally, children over 3 years old have: […] Some children aged between 3 and 6 years have chemotherapy only without radiotherapy. The doctor will go over the risks and benefits of this treatment with you, if this is an option for your child.

• #2 Medulloblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/brain-and-spinal-cord-childhood/treatment/medulloblastoma

  Surgery is usually the first treatment used. The goal of surgery is to remove as much of the tumour as possible. Surgery is followed with radiation therapy. […] Radiation therapy uses high-energy rays or particles to destroy cancer cells. Radiation therapy is usually given after surgery for children older than 3 years. Even if the tumour was completely removed, the entire brain and spinal cord are usually treated with low-dose radiation (called craniospinal radiation) because medulloblastomas often spread to these areas. A higher-dose boost of radiation is given to the area from where the tumour was removed. […] Chemotherapy uses drugs to destroy cancer cells. It is usually given during and after radiation therapy to treat medulloblastomas. In children younger than 3 years old, chemotherapy is given to try to delay or avoid radiation therapy. Chemotherapy may also be given directly into the CSF (called intrathecal or intraventricular chemotherapy).

• #2 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  As in average risk medulloblastoma, following induction therapy with radiation therapy, the most effective chemotherapy agents utilized are cisplatin, vincristine, lomustine (CCNU) and cyclophosphamide. […] Risk adapted radiotherapy followed by a shortened schedule of four consecutive high dose cyclophosphamide-based chemotherapy with autologous stem cell rescue has been shown to improve outcome of patients with high-risk medulloblastoma. […] Intensive chemotherapy with stem cell support has shown promise in recurrent and infant medulloblastoma cases. […] High-dose chemotherapy with autologous stem cell rescue has shown potential benefit in patients who are able to achieve minimal residual disease following salvage chemotherapy but carry significant morbidity. […] Alternatively, metronomic chemotherapy, an approach where low daily doses of drug are used to reduce tumor angiogenesis and promote cancer apoptosis, with or without the addition of intrathecal chemotherapy, has also shown promise with few reports of long-term survivors.

• #2 Medulloblastoma | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/brain-and-spinal-cord-childhood/treatment/medulloblastoma

  High-dose chemotherapy followed by stem cell transplant may also be used to treat medulloblastoma, especially in children younger than 3 years old. […] A stem cell transplant replaces stem cells. High-dose chemotherapy followed by stem cell transplant, or rescue, may be used to treat high-risk medulloblastoma, especially in children younger than 3 years of age.

• #2 Advances in medulloblastoma treatment for children

  https://medicalxpress.com/news/2024-06-advances-medulloblastoma-treatment-children.html

  Advances in medulloblastoma treatment for children […] Medulloblastoma treatment depends on tumor type and your child’s age. „Treatment usually involves safely removing as much of the tumor as possible, followed by radiation therapy and chemotherapy,” says Dr. Schwartz. […] In children younger than 4, Dr. Schwartz says care teams use radiation-sparing techniques, which reduce damage to healthy tissue, and high doses of chemotherapy followed by stem cell transplant (bone marrow transplant). In children older than 4, treatment also depends on whether the tumor is average or high risk. […] „If you leave more than 1.5 cubic centimeters of residual disease, we call that high risk. If it’s metastatic and has spread anywhere else, it’s high risk,” says Dr. Schwartz. […] If medulloblastoma recurs, Dr. Schwartz says care teams turn to other aggressive treatments, including chemotherapy and clinical trials for vaccines, chimeric antigen receptor (CAR)-T cell therapy, and other viral therapies. „It’s an aggressive disease. That’s why we treat it aggressively. If medulloblastoma recurs, we know treatment is generally not curative,” he says.

• #2 Advances in medulloblastoma treatment for children – Mayo Clinic Comprehensive Cancer Center Blog

  https://cancerblog.mayoclinic.org/2024/05/29/advances-in-medulloblastoma-treatment-for-children/

  Though rare, medulloblastoma is the most common cancerous brain tumor in children. Medulloblastoma treatment depends on tumor type and your child’s age. „Treatment usually involves safely removing as much of the tumor as possible, followed by radiation therapy and chemotherapy,” says Dr. Schwartz. […] In children younger than 4, Dr. Schwartz says care teams use radiation-sparing techniques, which reduce damage to healthy tissue, and high doses of chemotherapy followed by stem cell transplant (bone marrow transplant). […] If medulloblastoma recurs, Dr. Schwartz says care teams turn to other aggressive treatments, including chemotherapy and clinical trials for vaccines, chimeric antigen receptor (CAR)-T cell therapy, and other viral therapies. „It’s an aggressive disease. That’s why we treat it aggressively. If medulloblastoma recurs, we know treatment is generally not curative,” he says.

• #2 New Approaches in Targeted Therapy for Medulloblastoma in Children | Anticancer Research

  https://ar.iiarjournals.org/content/41/4/1715

  Current standard therapy, mainly composed of maximal surgical resection followed by CSI and chemotherapy, presents severe treatment-related toxicity, and secondary tumors may also occur. Reducing these consequences without affecting treatment efficacy is the focus of targeted therapy, aiming to reduce the doses of cytotoxic agents and radiation, as well as optimizing overall survival. […] In WNT and SHH MB, some driver mutations and inhibitors interfering in aberrant signaling pathways or targeting somatic mutations have been identified, and are intensively studied in vitro and in vivo. […] One approach is to epigenetically or directly inhibit the transcription factor GLI, which is considered to be much more efficient than inhibition of SMO, since GLI is a terminal effector of HH signaling and initiates the transcription of HH target genes.

• #2 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma. […] This phase II pediatric MATCH treatment trial studies how well selpercatinib works in treating patients with solid tumors that may have spread from where they first started to nearby tissue, lymph nodes, or distant parts of the body (advanced), lymphomas, or histiocytic disorders that have activating RET gene alterations. […] A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors (phase 1).

• #2 Pediatric Medulloblastoma Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/987886-treatment

  Radiation therapy for patients younger than 3 years, the poorest risk group, is generally avoided because of its deleterious effects on intellectual development, especially in this age group. […] In lieu of this, in an effort to delay or omit radiation, current protocols utilize high-dose chemotherapy approaches. […] Cytotoxic chemotherapy may be used in the initial treatment (in infants), as maintenance therapy (for average and high-risk disease) or for disease recurrence. […] In patients less than 3 years of age, incorporation of high dose chemotherapy strategies may delay or obviate the need for radiation therapy. […] Standard therapy post-radiation is with the use of cisplatin-based regimens. […] Many contemporary chemotherapy protocols use a combination of cisplatin, vincristine, lomustine (CCNU) and cyclophosphamide as maintenance therapy for approximately one year following radiation therapy.

• #2 Azthena logo with the word Azthena

  https://www.news-medical.net/news/20241009/Major-studies-reveal-improved-treatment-for-children-with-Group-3-medulloblastoma.aspx

  Vital research findings pointing to improved treatment for children with Group 3 medulloblastoma brain tumors have been revealed in two major studies published in the journal Neuro-Oncology. […] Now, findings from the two-phase 5 million INSTINCT studies could form the foundation of more targeted treatments for many children, leading to improved survival, less severe long-term side effects and improved quality of life. […] The two studies, which began in 2015, have provided critical evidence to help guide diagnosis and consider appropriate treatment dependent on the genetic make-up of the tumor. […] New therapies are urgently required to treat these tumors, but there has been a lag in their development. […] Together, these studies provide the essential diagnostic characteristics that can immediately be used to identify this critical tumor group in the clinic, as well as an important targetable mechanism for the development of new therapies aimed at improving their outcomes.

• #2 The Cure for Childhood Cancer | The Medulloblastoma Initiative

  https://mbinitiative.org/what-is-medulloblastoma/

  Study 1: Also called MATCHPOINT it will attack the tumor in two ways: 1) by training the patients own cells to identify and destroy cancer cells, and 2) by using an additional drug to disrupt the tumors defense system. […] Study 2: It will test an mRNA vaccine, similar to the COVID-19 vaccine, but more personalized, as it uses the patients own cells. Nanotechnology is used to create the vaccine. Once injected, the vaccine generates a targeted immune response to destroy the tumors. […] Three more clinical trials will be submitted between 2024 and 2025. This effort supported by philanthropy is expected to find a cure for Group 4 medulloblastomas.

• #2 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  Prognosis following relapse is dismal, with less than 5% of patients surviving; disease relapse is therefore the most adverse prognostic factor in MB. Furthermore, patients diagnosed with MB rarely die of other causes; less than 2% of patients who relapse die of treatment complications. Consequently, rMB accounts for a considerable, and disproportionate amount of childhood cancer deaths (~10%) and remains an urgent area of clinical unmet need. […] Here, we describe the current understanding of prognostic factors at disease relapse such as principal molecular group, adverse molecular biology, and timing of relapse. We provide an overview of the clinical diagnostic process including signs and symptoms, staging investigations, and molecular pathology, followed by a summary of treatment modalities and considerations. Finally, we summarise future directions to progress understanding of treatment resistance and the biological mechanisms underpinning early therapy-refractory and relapsed disease.

• #2 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  High-dose chemotherapy, defined as administration of intensive chemotherapy with autologous stem-cell support has been utilised in rMB for over two decades. […] Several case series have reported the use of temozolomide, irinotecan and bevacizumab for rMB. […] Immunotherapy is under development in many different forms, and has shown great potential in pre-clinical and early-phase clinical trials. However, despite the remarkable clinical efficacies observed in a number of malignancies, the use of immunotherapy alone may not be sufficiently active for many patients. Therefore, it is likely that the real potential for most patients, including those with r/rMB, lies in combining different immunotherapies with other treatments such as radiotherapy and chemotherapy to target tumour heterogeneity, overcome immunosuppression, and improve immune infiltration.

• #2 New Approaches in Targeted Therapy for Medulloblastoma in Children | Anticancer Research

  https://ar.iiarjournals.org/content/41/4/1715

  This leads to considerable morbidity among the 70% of patients that are cured, and leaves the remaining 30% in need of better therapeutic options since they are not cured. […] Genomic data have also identified genetic drivers for MB, and, as specific targets, these could potentially help improve present treatment options considerably and thereby reduce therapy-related toxicity. […] We also focus on pathways that drive MB tumorigenesis, and present an overview of some current preclinical and clinical studies targeting the hedgehog (HH), phosphoinositide 3-kinase (PI3K), cyclin-dependent kinase 4 and 6 (CDK4/6) pathways, as well as approaches in immunotherapy. […] There is therefore a strong motivation to identify targeted therapies that are less toxic and more effective, in order to reduce the resulting long-term side-effects.

• #2 Overcoming Treatment Resistance in Medulloblastoma: Underlying Mechanisms and Potential Strategies

  https://www.mdpi.com/2072-6694/16/12/2249

  The blood-brain barrier (BBB) is a layer of specialized cells that surround the brain and protects it from pathogens and toxic metabolites. […] The blood-brain barrier becomes problematic during the management of different brain cancers—including medulloblastoma—because of how it can inhibit chemotherapeutic drug delivery. […] One prominent driver of treatment resistance in cancers is the ATP-binding cassette transporter superfamily. […] Several efforts have been channeled towards targeting the different drivers that promote the development and maintenance of treatment resistance in medulloblastoma. […] The mTOR pathway is also frequently associated with all subgroups of medulloblastoma. […] The ABCB receptor family, which act as ATP-dependent multidrug pumps, are also key regulators of chemoresistance in SHH medulloblastoma.

• #2 Medulloblastoma: Brain Tumours | Children with Cancer UK

  http://www.childrenwithcancer.org.uk/childhood-cancer-info/cancer-types/brain-spinal-tumours/medulloblastoma/

  Using cutting edge molecular genetic techniques, Professor Pilkingtons team is unravelling the biology of medulloblastoma. […] Dr Amin is working on the development of a new way to deliver drugs to the brain bypassing the blood-brain barrier, carried by a special virus. […] Dr Maria is studying how p73 is involved in MB and in particular its role in metabolic adaptation of these tumours. Her final goal is to understand whether targeting p73 could help treat them.

• #2 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  The current study will use a new treatment approach based on the molecular characteristics of each participant’s tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. […] This phase III trial tests two hypotheses in patients with low-risk and average-risk medulloblastoma.

• #2 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  This phase II Pediatric MATCH treatment trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back (recurrent) or does not respond to treatment (refractory) and may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). […] This phase II Pediatric MATCH trial studies how well erdafitinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with FGFR mutations that have spread to other places in the body and have come back or do not respond to treatment. […] This trial studies how well fimepinostat works in treating patients with newly diagnosed diffuse intrinsic pontine glioma, or medulloblastoma, or high-grade glioma that have come back.

• #2 How we treat medulloblastoma in adults

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8207184/

  Early and late side-effects are important issues in adults with medulloblastoma. These include neurotoxicity and systemic toxicity as well as psychosocial and fertility issues. Psychological and social support should be offered to all patients. […] Post-operative craniospinal irradiation (CSI) with adequate target volume coverage and sparing of organs at risk is mandatory. […] Medulloblastomas are chemosensitive tumours. Adult patients with medulloblastoma should be treated with systemic therapy in addition to resection and radiotherapy, irrespective of their risk category. […] The Packer chemotherapy regimen, which is based on lomustine, vincristine and cisplatin, is commonly used in adults, at least in Europe. […] With the recognition of medulloblastoma subtyping, targeted therapies can be considered in individual cases and are part of recent prospective clinical trials.

• #2 Medulloblastoma/PNET | The Royal Marsden

  https://www.royalmarsden.nhs.uk/your-care/cancer-types/paediatric-cancers/medulloblastomapnet

  The majority of children with a localised tumour removed by the surgeon and who also have intensive chemotherapy and radiotherapy in a reduced dose to the brain and spine are cured. Children who have PNET, an incomplete removal of tumour at initial surgery, or evidence of the tumour having spread, have a poorer outlook. […] The treatment of infants is difficult. This is because of the need to delay, avoid or reduce radiotherapy to the brain and spine, because of the damaging effects of radiation on the young developing brain. […] As medulloblastomas are more common in young children and the treatment is given at an important time of the child’s development there may be some long-term effects of treatment. These could include growth and hormonal changes, reduced fertility, behavioural changes, possible learning problems and difficulties with coordination. Hearing and visual disturbances can result from both tumour and treatments. There is a small risk of developing a second cancer later in life.

• #2 Medulloblastoma: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/22591-medulloblastoma

  Length of treatment can vary from person to person. Those who undergo any type of brain surgery usually need at least four to eight weeks to recover. For chemotherapy or radiation therapy, treatment could last several weeks or several months, depending on the case. […] In many cases, yes. While medulloblastoma has the potential to spread throughout your entire nervous system, many people can be cured. There’s a higher chance of survival if the medulloblastoma hasn’t spread to other parts of your brain and spinal cord. […] The five-year medulloblastoma survival rate is over 80%. This means that over 80% of all people diagnosed with medulloblastoma are still alive five years later.

• #2 Treatment Options for Medulloblastoma and CNS Primitive Neuroectodermal Tumor (PNET)

  https://pmc.ncbi.nlm.nih.gov/articles/PMC5026188/

  For patients with medulloblastoma less than 3 years of age, PFS is 30-40 % at 5 years. […] Patients with high-risk medulloblastoma are treated with 36 Gy CSI, although data, in nondisseminated patients, to confirm the efficacy of higher-doses of radiotherapy, are lacking. […] The classification and treatment of medulloblastomas is likely to change in the coming years, due to new insights into the molecular biology of medulloblastoma.

• #2 Relapsed Medulloblastoma in Pre-Irradiated Patients: Current Practice for Diagnostics and Treatment

  https://www.mdpi.com/2072-6694/14/1/126

  In summary, while rMB remains a significant area of unmet clinical need in paediatric oncology, current and future initiatives provide hope for new approaches to modify outcomes. Current state-of-the-art approaches to the diagnosis and management of rMB comprise comprehensive radiological, histological and molecular assessments, consideration of local therapy options (surgery and/or re-irradiation) and a selection of chemotherapy regimens which can be utilised on an individual patient basis.

• #2 Medulloblastoma – Overview of Information and Clinical Research

  https://clinicaltrials.eu/disease/medulloblastoma/

  Innovative treatments, such as targeted therapies and immunotherapy, are being explored to improve outcomes for medulloblastoma patients. […] Supportive care is an integral part of the treatment plan, aimed at enhancing the patient’s quality of life and managing side effects. […] The treatment of medulloblastoma is a complex process that requires a comprehensive approach to improve patient outcomes and quality of life. Surgical intervention is often the first step, aiming to remove as much of the tumor as possible while preserving brain function. Radiation therapy follows, targeting any remaining cancer cells with precision techniques like proton beam therapy, especially beneficial for children. Chemotherapy is a cornerstone of treatment, with regimens tailored to individual patient needs and sometimes combined with stem cell transplants to enhance effectiveness. Advanced therapies, such as targeted therapies and immunotherapy, offer hope for more personalized and effective treatment options. […] Clinical trials are crucial for testing new therapies and improving survival rates, with various phases focusing on safety, efficacy, and long-term outcomes.

• #3 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  This is a prospective randomized clinical trial, to determine whether dose-intensive tandem Consolidation, in a randomized comparison with single cycle Consolidation, provides an event-free survival (EFS) and overall survival (OS). […] This phase II Pediatric MATCH trial studies how well ivosidenib works in treating patients with solid tumors that have spread to other places in the body (advanced), lymphoma, or histiocytic disorders that have IDH1 genetic alterations (mutations). […] This phase II Pediatric MATCH trial studies how well larotrectinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with NTRK fusions that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) and have come back (relapsed) or does not respond to treatment (refractory).

• #4 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma. […] This phase II pediatric MATCH treatment trial studies how well selpercatinib works in treating patients with solid tumors that may have spread from where they first started to nearby tissue, lymph nodes, or distant parts of the body (advanced), lymphomas, or histiocytic disorders that have activating RET gene alterations. […] A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors (phase 1).

• #5 UCSF Medulloblastoma Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/medulloblastoma

  This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. […] This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a genetic alteration in the gene HRAS. […] This phase II Pediatric MATCH trial studies how well ulixertinib works in treating patients with solid tumors that have spread to other places in the body (advanced), non-Hodgkin lymphoma, or histiocytic disorders that have a genetic alteration (mutation) in a signaling pathway called MAPK.

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