We recently received an update on a phase 2 FDA Trial for the treatment of glioblastoma multiforme (GBM), a highly aggressive brain tumor with a limited life expectancy of approx 12 months on current therapy available. This trial was a very successful trial in light of the small sample size of 21 patients, although that is in line with international standards for this type of trial for GBM.
In a randomized double blind placebo trial, 83% of the patients treated with the THC:CBD (cannabis compounds) had a one year survival rate vs 53% of the placebo population. Median survival for the THC:CBD group was greater than 550 days compared to the placebo group at 369 days. Adverse events were limited to common problems associated with the disease itself.
From the Press Release: Professor Susan Short, PhD, Professor of Clinical Oncology and Neuro-Oncology at Leeds Institute of Cancer and Pathology at St James’s University Hospital and principal investigator of the study. “Moreover, the cannabinoid medicine was generally well tolerated. These promising results are of particular interest as the pharmacology of the THC:CBD product appears to be distinct from existing oncology medications and may offer a unique and possibly synergistic option for future glioma treatment.”
What makes the study so interesting is the lack of secondary endpoints that many look for as additional signs of efficacy. When the company was asked about lack of data, the response was "It's not yet possible to work out statistics because there are still such a substantial number of them still alive." So, they can not give you all of the data yet in a deadly disease as the people expected to die have not died yet.
This type of drug is eligible for something called Advanced Approval from the FDA. The current top-line treatment TMZ has only extended life by 2.5 months on average and had received AA. AA is predominantly reserved for disease such as AIDS and certain forms of cancer. You can learn more about GBM trials here: http://www.intechopen.com/books/molecular-considerations-and-evolving-surgical-management-issues-in-the-treatment-of-patients-with-a-brain-tumor/clinical-trials-in-glioblastoma-designs-and-challenges