Headaches. Blurred vision. Nausea. No or poor appetite. Behavioral changes. Trouble thinking. These are some common problems people experience in their daily lives; however, are easy to write off as momentary or sideline in the name of stress. Yet, these constitute some of the common early symptoms of Glioblastoma multiforme (GBM), a form of aggressive and lethal tumor of the brain and spinal cord. These are the most malignant form of human tumor delineated by rapid progression, invasion, and intense angiogenesis. The onset of vague symptoms as the early signs of Glioblastoma, and also as these are in the mind, literally and figuratively, any changes that take place go undetected, is what makes the tumor deadly. 

GBMs are rare tumors with a global incidence of less than 10 per 100,000 people.

The total Glioblastoma multiforme incident population in the 7MM (the US, EU5 (the UK, Germany, Italy, Spain, and France) and Japan) was estimated to be around 2900 in 2017. Further, in 2020, the GBM incident poulation in the 7MM is expected to be 29,440.


Furthermore, Glioblastoma multiforme appeared to have a higher male predisposition. In a symposium presented at the Society of NeuroOncology in 2018, gender discrepancy at all the levels – cellular, systemic, and genetic, in Glioblastoma multiforme was largely pressed, with over 60% of the diagnosed population constituting males. It could be either due to sex hormones or due to IDH-1 mutation, the frequency of which is quite higher in males.

Glioblastoma multiforme epidemiology analysis further revealed that the majority of the GBM cases, more than 90%, were reported to have  Primary glioblastoma. GBMs either develop de novo, known as or they may progress from low-grade or anaplastic astrocytomas, termed as Secondary glioblastoma. Although the tumors share similar histologic appearance, they differ largely in terms of precursor cells, thus, calls for different therapeutic approaches.  

Even though GBM tumors are rare, the median survival rate of Glioblastoma multiforme is 10-15 months after diagnosis – attributable to insufficient knowledge of the early stages of Glioblastomas. Delayed diagnosis is one of the significant reasons behind poor patient outcomes and a major unmet need in the market, besides, of course, treatment. To date, Glioblastomas remain incurable and available treatment is palliative in nature. The cardinal aim of the treatment of patients newly diagnosed with GBM is to achieve “maximum safe resection” by performing surgery. Although the metastatic property of Glioblastoma multiforme is a rare scenario, i.e., they never leave the nervous tissue, however, the tentacles and finger-like projections of the brain cells infiltrate the brain holistically, making it difficult to remove altogether. In the cases where surgery seems to be out of the question or fails in achieving optimal results, physicians turn to Radiotherapy and chemotherapy as concomitant or adjuvant therapy. At present, the Glioblastoma multiforme treatment market is inhabited with a few FDA approved chemotherapy drugs namely BiCNU (carmustine), Avastin (bevacizumab), and Temodar (temozolomide). Schering-Plough’s Temodar is the most frequently used chemo drug. Temodar is the most widely used chemotherapy and manages to increase the median survival rate by a few weeks, and so does the other two. 

Regardless of advanced technology, diagnostic modalities, R&D in brain neoplasms, carefully weighted multidisciplinary treatment approach, the overall five-year survival rate is still around 5% from the past decades, which is one of the huge unmet needs in the GBM market. Average survival does not traverse the one-year mark; and if the tumor recurs, this survival rate pales further, with almost all the GBM patients’ tumor progressing and ending with only the death of the patient. 

The treatment of brain tumors faces unique challenges; the presence of the blood-brain-barrier (BBB) is one of the most notable ones. Unfortunately, therapeutic options available in the current Glioblastoma multiforme find themselves incapable of crossing it. It is highly argued that the GBM is responsible for a leaky BBB; however, it does not help as the cancerous cell remains within the precincts of compact BBB. Another hurdle that perverses in the Glioblastoma multiforme treatment market is Intertumor heterogeneity, and this is why in the very first place, the tumor is termed as Glioblastoma ‘multiforme’. Intertumor heterogeneity is a root cause of treatment resistance and failure resulting in mean survival less than 14 months. Further, there is no therapeutic option or standard care available to approach recurring GBM cases. 

Unmet Needs in GBM Treatment Market

To address Unmet needs in the Glioblastoma multiforme treatment market, there is a dire need of funding. Being a rare disease, there is already a dearth of patient accrual, and understanding of tumor and the current scenario does not look promising even in terms of funds needed for the development of novel therapeutic targets. Innumerous clinical trials are underway that are majorly led by Industries, followed by academia with a focus on systemic therapies. There is no remarkable progress in patient outcome, rather the results appear to be quite disappointing. Not to forget, clinical trial enrollment for such a rare brain tumor is not less than an uphill struggle. The studies underway, and in the pipeline, always face the issues with selection bias, confounders, and inappropriate historic controls. 

Although several pharmaceutical companies are including, Bristol-Myers Squibb, DelMar Pharmaceuticals, AbbVie, Aivita Biomedical, Diffusion Pharmaceuticals, MedImmune, DNAtrix, Apogenix, Kazia Therapeutics, Medicenna Therapeutics, and many others are running clinical trials investigating their candidates in different stages of clinical development, but most of them are in Phase II stage of development. However, with a bleak rate of only a few reaching Phase III clinical development, it is yet to see which candidate stands the test of time.  

Advances have been made in neuroimaging, in the field of surgical techniques, however, none seems to have addressed the survival crisis. The grim prognosis, miserable patient outcomes, and temporizing treatment approaches scream for distinct options in the GBM treatment market landscape to budge the five-year survival rate. With only a handful of the approved therapies available in the present GBM treatment market, it is imperative to understand the challenges that plague the emergence of new therapeutic targets. Comprehensive knowledge of the molecular pathology or etiology of the disease further offers new handles in moving the needle in the Glioblastoma multiforme treatment market and meeting the unmet needs.