Sitoiganap is a therapeutic vaccine which used to be called Gliovac or ERC1671. This paper presents the results of the expanded access program for the vaccine on patients with recurrent glioblastoma. Expanded access programs are not as controlled as a traditional randomized controlled trial, in that they usually allow any patient to enter - whereas the traditional trials cherry pick those patients who would be expected to do well on the treatment. So these patients would be expected to do worse than the historical clinical trial statistics. Although this is a small study, the results are very exciting. The vaccine more than doubled the median survival to 19.6 months. Other trials are reporting a median survival of about 8 months for recurrent glioblastoma. The 2 year survival rate was 45% which is excellent.
With this data, they asked the European Medicines Agency (EMA) to approve the vaccine for the treatment of Glioblastoma. Strangely the EMA refused and wants more data. Seeing how there were minimal side effects and had very good results in a small group, I feel they should have received approval. Probably the same thing would happen here with our FDA. This is outrageous and we should fight to get the regulations changed. Our Promising Pathway Act is designed just for these types of treatments - it really is too early to tell how well it works, but with no side effects and early indication of success, how could a regulatory agency tell us that they will not allow us access to treatments like this. As with all previously approved brain tumor treatments, we never really find out how they work until after approval and they are used in combinations and on average patients.