Wilms Tumor 1 (WT1) Peptide Vaccine

Property	Information
Drug Name	Wilms Tumor 1 (WT1) Peptide Vaccine
FDA Approval	In clinical trials; not yet FDA-approved
Used for	Newly diagnosed and recurrent Glioblastoma Multiforme (GBM)
Clinical Trial Phase	Phase II (Based on the latest clinical trial data available)
Clinical Trial Explanation	Not specified
Common Side Effects	Local erythema at the injection site; overall, the treatment is well-tolerated
OS without	Typical median overall survival for GBM with standard treatment is about 15-17 months
OS with	Early trials reported a median progression-free and overall survival of at least 43.5 months, with a later update indicating median PFS over 48 months
PFS without	Standard treatments usually achieve a median progression-free survival of around 6.9 months
PFS with	Reported as over 48 months in a Phase II trial, indicating a significant improvement over standard treatments
Usefulness Rating	4
Usefulness Explanation	The WT1 peptide vaccine shows considerable promise in extending progression-free and overall survival in GBM patients. These results suggest a major breakthrough, especially considering the extended PFS and OS compared to standard therapies.
Toxicity Level	1
Toxicity Explanation	The vaccine is generally well-tolerated, with the most common side effect being local erythema at the vaccine injection site, indicating a low toxicity profile.

Links: https://thejns.org/abstract/journals/j-neurosurg/108/5/article-p963.xml

From Ben Williams Book: In March of 2015, a Japanese group published results of a trial that tested a Wilms tumor 1(WT1) peptide vaccine in addition to radiation and chemotherapy for newly diagnosed glioblastoma (341). Wilms Tumor 1 (WT1) is a protein overexpressed in various types of solid and liquid cancers, not to be confused with the pediatric cancer it is named after (Wilms’ Tumor). Seven patients were included in the analysis, with four having undergone total tumor resection, two having partial resection, and one having biopsy only. None of the tumors were positive for IDH1 mutation. Patients received up to 24 monthly temozolomide cycles, the standard at this institution. Remarkably, five of these seven patients (71%) were still disease-free at three years or more. Only one patient had experienced disease progression at the time of analysis and all were still alive. Median progression-free and overall survival were at least 43.5 months (about 3 and a half years), which was the median follow-up time at the time of the analysis. These very impressive results were not likely simply due to the prolonged cycles of temozolomide: at the same institution, median PFS and OS with up to 24 cycles of TMZ (but no vaccine) is 10.7 and 21 months.

An abstract (reference 342, abstract IMCT-09) for the SNO 2015 meeting tells us that median progression-free survival for the seven patients is now over 48 months (4 years) as five of seven patients were still without progression at that time point.Property "Has original text" (as page type) with input value "In March of 2015, a Japanese group published results of a trial that tested a Wilms tumor</br>1(WT1) peptide vaccine in addition to radiation and chemotherapy for newly diagnosed</br>glioblastoma (341). Wilms Tumor 1 (WT1) is a protein overexpressed in various types of</br>solid and liquid cancers, not to be confused with the pediatric cancer it is named after</br>(Wilms’ Tumor). Seven patients were included in the analysis, with four having</br>undergone total tumor resection, two having partial resection, and one having biopsy</br>only. None of the tumors were positive for IDH1 mutation. Patients received up to 24</br>monthly temozolomide cycles, the standard at this institution. Remarkably, five of these</br>seven patients (71%) were still disease-free at three years or more. Only one patient had</br>experienced disease progression at the time of analysis and all were still alive. Median</br>progression-free and overall survival were at least 43.5 months (about 3 and a half years),</br>which was the median follow-up time at the time of the analysis. These very impressive</br>results were not likely simply due to the prolonged cycles of temozolomide: at the same</br>institution, median PFS and OS with up to 24 cycles of TMZ (but no vaccine) is 10.7 and</br>21 months.</br></br>An abstract (reference 342, abstract IMCT-09) for the SNO 2015 meeting tells us that</br>median progression-free survival for the seven patients is now over 48 months (4 years)</br>as five of seven patients were still without progression at that time point." contains invalid characters or is incomplete and therefore can cause unexpected results during a query or annotation process.

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