Glioblastoma multiforme (GBM) is the most aggressive form of primary brain tumor and represents a critical area of unmet medical need. Despite advances, the prognosis for glioblastoma patients remains dismal, with a median survival rate of just 15-18 months post-diagnosis. This stark reality underscores the urgent need for innovative therapeutic approaches and a deeper understanding of the disease’s underlying mechanisms.

The greatest barrier to treating CNS disorders has been the inability of therapeutic molecules to bypass the blood brain barrier (BBB). That’s because the blood brain barrier actively works to prevent foreign substances from entering the central nervous system (CNS).

The standard treatment for GBM often involves a combination of surgery, radiation, and chemotherapy. However, these approaches have limitations:

• Surgery: Complete removal is rarely possible due to the tumor’s invasive nature.
• Radiation: Brain tissue is sensitive to radiation, leading to potential side effects.
• Chemotherapy: The blood-brain barrier (BBB) restricts drug delivery to the tumor site, hindering the effectiveness of many chemotherapeutic agents.
• Limited treatment options for recurrent GBM: GBM has a high rate of recurrence, and treatment options for recurrent tumors are even more limited.

The limitations of current treatments highlight the urgent need for novel approaches in GBM therapy. Great clinical (and commercial) opportunities exist in these two areas:

Improved Drug Delivery: Developing strategies to bypass the BBB or utilizing targeted drug delivery systems could significantly improve treatment efficacy.

Immunotherapy: Harnessing the immune system to fight GBM holds immense promise, with ongoing research exploring immune therapies.

Several therapeutics are currently approved to treat glioblastoma representing a total market size of nearly $2 billion