SurVaxM Vaccine for Brain Cancer

Recruiting in Palo Alto (17 mi)

+12 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Pediatric Brain Tumor Consortium

Must not be taking: Cannabidiol, Medical marijuana, others

Disqualifiers: Active infection, Autoimmune disease, others

No Placebo Group

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This trial tests the SurVaxM vaccine, which helps the immune system target and destroy cancer cells, in children and young adults with certain difficult-to-treat brain cancers. The vaccine works by teaching the immune system to recognize a protein found in cancer cells. Additional substances are used to make the immune response stronger.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot be on any other anti-cancer or investigational drug therapy, and certain medications like cannabidiol (CBD) or medical marijuana are not allowed. It's best to discuss your current medications with the study team to ensure eligibility.

What data supports the effectiveness of the SurVaxM treatment for brain cancer?

Research shows that SurVaxM, a vaccine targeting the survivin protein in cancer cells, can activate the immune system in patients with glioblastoma, a type of brain cancer. In clinical studies, some patients experienced stable disease or partial response, with a median overall survival of 86.6 weeks, suggesting potential benefits of this treatment. ^{1 2 3 4 5}

Is the SurVaxM vaccine safe for humans?

SurVaxM has been tested in clinical studies for brain cancer and was generally well tolerated. Most side effects were mild, like injection site reactions and fatigue, and no serious side effects were linked to the vaccine. ^{1 2 5 6 7}

How is the SurVaxM treatment different from other treatments for brain cancer?

SurVaxM is unique because it is a peptide vaccine that activates the immune system to target survivin, a protein highly expressed in glioblastoma cells, unlike traditional treatments that focus on surgery, radiation, and chemotherapy. This approach aims to stimulate the body's own immune response to fight the cancer. ^{1 2 5 6 7}

Research Team

Clare Twist, MD

Principal Investigator

Roswell Park Comprehensive Cancer Center

Eligibility Criteria

This trial is for children and young adults aged 1 to 21 with specific brain tumors (like medulloblastoma, high-grade glioma, ependymoma) that are progressive or relapsed. They must have completed prior treatments without severe side effects, be HIV-negative or well-controlled on therapy, and have good organ function. Participants need to agree to use birth control and provide consent.

Inclusion Criteria

I have a high-grade brain tumor with clear signs on an MRI.

My hepatitis B virus load is undetectable with treatment.

I have a newly diagnosed brain tumor in the pons without needing a biopsy.

Exclusion Criteria

Patients living outside the US

I have had a condition where my lymphocytes grow abnormally.

I do not have an active infection needing treatment.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Priming

Patients receive one dose of SurVaxM combined with Montanide ISA 51 and sargramostim every 2 weeks for 6 weeks (total of 4 doses)

8 weeks

4 visits (in-person)

Maintenance

Patients receive a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years

Up to 2 years

Every 8 weeks (in-person)

Follow-up

Patients are monitored for safety and effectiveness after treatment, with clinic visits every 3 months

Up to 3 years

Every 3 months (in-person)

Treatment Details

Interventions

• SurVaxM (Cancer Vaccine)

Trial Overview The study tests SurVaxM vaccine combined with Montanide ISA 51 in two phases: Priming (4 doses every 2 weeks) and Maintenance (doses every 8 weeks up to two years). It aims to train the immune system to attack tumor cells expressing survivin protein. Sargramostim is also given to boost the immune response.

Participant Groups

Group I: SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥10 and ≤21 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

Group II: SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥1 and <10 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

Group III: SurVaxM for patients with non-relapsed DIPG post radiation-therapy ages ≥1 and ≤21 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

Who Is Running the Clinical Trial?

Pediatric Brain Tumor Consortium

Lead Sponsor

Trials

38

Recruited

1,600+

American Lebanese Syrian Associated Charities (ALSAC)

Collaborator

Trials

9

Recruited

670+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Dr. Douglas R. Lowy

National Cancer Institute (NCI)

Chief Executive Officer since 2023

MD from New York University School of Medicine

Dr. Monica Bertagnolli

National Cancer Institute (NCI)

Chief Medical Officer since 2022

MD from Harvard Medical School

American Lebanese Syrian Associated Charities

Collaborator

Trials

9

Recruited

670+

Roswell Park Cancer Institute

Collaborator

Trials

427

Recruited

40,500+

Dr. Julia Faller

Roswell Park Cancer Institute

Chief Medical Officer since 2024

DO from an unspecified institution

Dr. Candace S. Johnson

Roswell Park Cancer Institute

Chief Executive Officer since 2015

PhD in Immunology from The Ohio State University

Findings from Research

SurVaxM, a peptide vaccine targeting the survivin protein in glioblastoma cells, was found to be safe and well tolerated in a phase IIa trial with 64 patients, showing no serious adverse events related to the vaccine.

The combination of SurVaxM with adjuvant temozolomide (TMZ) resulted in a high progression-free survival rate of 95.2% at 6 months and a median overall survival of 25.9 months, indicating promising efficacy in treating newly diagnosed glioblastoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase IIa Study of SurVaxM Plus Adjuvant Temozolomide for Newly Diagnosed Glioblastoma. Ahluwalia, MS., Reardon, DA., Abad, AP., et al.[2023]

Immunotherapy, particularly using active specific vaccination like the survivin-targeted peptide vaccine (SurVaxM), shows promise as a potential adjunctive treatment for malignant gliomas, especially given the limitations of conventional therapies.

Despite the optimism surrounding immunotherapy for gliomas, significant challenges remain in translating laboratory findings into effective Phase I and II clinical trials, highlighting the complexity of developing these treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Challenges in the development of a survivin vaccine (SurVaxM) for malignant glioma. Fenstermaker, RA., Ciesielski, MJ.[2022]

The DCVax-L trial indicated a potential survival benefit for glioblastoma patients receiving dendritic cell vaccination alongside standard care, showing improved overall survival in both newly diagnosed (19.3 months vs. 16.5 months) and recurrent settings (13.2 months vs. 7.8 months).

However, the trial's design and methodology raised significant concerns, including changes to endpoints and the use of external controls, which may have biased the results and limited the ability to draw reliable conclusions about the efficacy of the treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous tumor lysate-loaded dendritic cell vaccination in glioblastoma: What happened to the evidence? Olivier, T., Migliorini, D.[2023]

Vaccination strategies targeting cancer-associated antigens have shown promise in preclinical brain tumor models, demonstrating the immune system's ability to safely eliminate established tumors in the central nervous system.

Early phase clinical trials, particularly in glioblastoma patients, have confirmed the safety and immunogenicity of these vaccines, with initial results suggesting potential clinical benefits, although further studies are needed to establish definitive outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vaccination strategies for neuro-oncology. Sampson, JH., Mitchell, DA.[2018]

The SurVaxM vaccine, targeting the survivin protein, was well tolerated in recurrent malignant glioma patients, with mostly mild side effects and no serious adverse events, indicating a favorable safety profile.

Immunologically, the vaccine successfully elicited immune responses in six out of eight evaluable patients, and showed promising clinical outcomes, with some patients maintaining stable disease for over 6 months and a median overall survival of 86.6 weeks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical study of a survivin long peptide vaccine (SurVaxM) in patients with recurrent malignant glioma. Fenstermaker, RA., Ciesielski, MJ., Qiu, J., et al.[2023]

DCVax-Brain, a personalized treatment using dendritic cells to target brain tumors, shows promise in inducing antitumor immunity with low toxicity in patients with glioblastoma multiforme (GBM).

Recent trials indicate that DC vaccine therapy correlates with clinical improvements in GBM patients, although some reports suggest possible selection bias in the findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

DCVax-Brain and DC vaccines in the treatment of GBM. Wheeler, CJ., Black, KL.[2019]

DCVax-L is a personalized cancer vaccine that uses a patient's own dendritic cells and tumor lysate to stimulate an immune response against glioblastoma multiforme (GBM).

Preliminary results from clinical trials, including ongoing Phase III studies, suggest that DCVax-L may offer a promising treatment option for patients with both newly diagnosed and recurrent GBM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dendritic cell immunotherapy for solid tumors: evaluation of the DCVax® platform in the treatment of glioblastoma multiforme. Hdeib, A., Sloan, AE.[2019]

References

1. United States pubmed.ncbi.nlm.nih.gov

Phase IIa Study of SurVaxM Plus Adjuvant Temozolomide for Newly Diagnosed Glioblastoma. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Challenges in the development of a survivin vaccine (SurVaxM) for malignant glioma. [2022]

3. France pubmed.ncbi.nlm.nih.gov

Autologous tumor lysate-loaded dendritic cell vaccination in glioblastoma: What happened to the evidence? [2023]

4. England pubmed.ncbi.nlm.nih.gov

Vaccination strategies for neuro-oncology. [2018]

5. Germany pubmed.ncbi.nlm.nih.gov

Clinical study of a survivin long peptide vaccine (SurVaxM) in patients with recurrent malignant glioma. [2023]

6. England pubmed.ncbi.nlm.nih.gov

DCVax-Brain and DC vaccines in the treatment of GBM. [2019]

7. England pubmed.ncbi.nlm.nih.gov

Dendritic cell immunotherapy for solid tumors: evaluation of the DCVax® platform in the treatment of glioblastoma multiforme. [2019]