New Compound Could Become a Transformational Brain Cancer Pill
Researchers at the University of Chicago have identified a compound that has shown efficacy in shrinking tumors in the brains of mice. This has created hope that the compound could, in future, be made into a pill that can treat glioblastoma, one of the deadliest forms of brain cancer.
Hui Li, PhD, who led the study team, explains that compound A, as the substance is known, attached to a target on glioblastoma that hadn’t been known to serve as a therapeutic target. The researchers used mouse models in their study and they are now working to develop variants of compound A that are suitable for use in clinical trials. So far, 160 versions of this molecule have been created.
Back in 2020, Li’s team identified the AVIL (advillin) protein as being excessively abundant within glioblastoma tumors. Their work explaining the role of this protein in the progress of the cancer led many desperate patients and their family members to constantly call or email Li asking for a new treatment for this devastating brain cancer. These calls overwhelmed Li and drove him to look for ways in which the discovery of AVIL could form the basis of a new drug indicated for glioblastoma.
Brain cancers like GBM are particularly challenging to treat for several reasons. First, any drug must be able to go through the blood-brain barrier, a huge roadblock to overcome. Drugs shouldn’t also impair the functioning of normal brain tissues as they attack tumors. Additionally, the drug faces the challenge of the cancer becoming resistant to it, as often happens when patients are started on temozolomide, a chemotherapy drug currently available for brain cancer.
Li worked with Angela Koehler, PhD., at MIT. She operates a lab at MIT that focuses on discovering drugs for cancer. They screened over 50,000 compounds searching for any that could inhibit or bind to advillin. This was a desperate effort because that library was too small for the task and the molecule hasn’t been leveraged as a drug target. Additionally, the structure of this protein hasn’t been published, so the team was probing in the dark.
Fortunately, the team of researchers was lucky to land upon compound A during the screening exercise. They conducted lab experiments and assay tests on cancerous and noncancerous tissues to confirm that the compound could indeed target advillin. The tests showed that this substance targeted the protein.
The scientists then conducted a variety of tests on mice using compound A and found that it met the requirements of lowering the volume and size of GBM tumors. That happened even in tests in which the cancer had developed resistance to the drug temozolomide. Another exciting discovery was that the compound was even more effective at killing the stem cells of the cancer, which offered hope that the cancer could struggle to develop resistance to compound A.
This preclinical study is significant in many ways, including the compound having the capability of being administered orally, showing efficacy against many GBM cell lines while also being able to cross the BBB (blood-brain barrier).
The scientific community is eager to see how the drug will perform in the planned clinical trials once the researchers complete their experiments geared at having versions suitable for use by humans. Entities like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) that are also engaged in developing drugs for brain cancer are likely to be rooting for this team since they are all working to transform brain cancer care, in different ways.
NOTE TO INVESTORS: The latest news and updates relating to CNS Pharmaceuticals Inc. (NASDAQ: CNSP) are available in the company’s newsroom at https://ibn.fm/CNSP
Please see full terms of use and disclaimers on the BioMedWire website applicable to all content provided by BMW, wherever published or re-published: http://BMW.fm/Disclaimer
Researchers at the University of Chicago have identified a compound that has shown efficacy in shrinking tumors in the brains of mice. This has created hope that the compound could, in future, be made into a pill that can treat glioblastoma, one of the deadliest forms of brain cancer.
Hui Li, PhD, who led the study team, explains that compound A, as the substance is known, attached to a target on glioblastoma that hadn’t been known to serve as a therapeutic target. The researchers used mouse models in their study and they are now working to develop variants of compound A that are suitable for use in clinical trials. So far, 160 versions of this molecule have been created.
Back in 2020, Li’s team identified the AVIL (advillin) protein as being excessively abundant within glioblastoma tumors. Their work explaining the role of this protein in the progress of the cancer led many desperate patients and their family members to constantly call or email Li asking for a new treatment for this devastating brain cancer. These calls overwhelmed Li and drove him to look for ways in which the discovery of AVIL could form the basis of a new drug indicated for glioblastoma.
Brain cancers like GBM are particularly challenging to treat for several reasons. First, any drug must be able to go through the blood-brain barrier, a huge roadblock to overcome. Drugs shouldn’t also impair the functioning of normal brain tissues as they attack tumors. Additionally, the drug faces the challenge of the cancer becoming resistant to it, as often happens when patients are started on temozolomide, a chemotherapy drug currently available for brain cancer.
Li worked with Angela Koehler, PhD., at MIT. She operates a lab at MIT that focuses on discovering drugs for cancer. They screened over 50,000 compounds searching for any that could inhibit or bind to advillin. This was a desperate effort because that library was too small for the task and the molecule hasn’t been leveraged as a drug target. Additionally, the structure of this protein hasn’t been published, so the team was probing in the dark.
Fortunately, the team of researchers was lucky to land upon compound A during the screening exercise. They conducted lab experiments and assay tests on cancerous and noncancerous tissues to confirm that the compound could indeed target advillin. The tests showed that this substance targeted the protein.
The scientists then conducted a variety of tests on mice using compound A and found that it met the requirements of lowering the volume and size of GBM tumors. That happened even in tests in which the cancer had developed resistance to the drug temozolomide. Another exciting discovery was that the compound was even more effective at killing the stem cells of the cancer, which offered hope that the cancer could struggle to develop resistance to compound A.
This preclinical study is significant in many ways, including the compound having the capability of being administered orally, showing efficacy against many GBM cell lines while also being able to cross the BBB (blood-brain barrier).
The scientific community is eager to see how the drug will perform in the planned clinical trials once the researchers complete their experiments geared at having versions suitable for use by humans. Entities like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) that are also engaged in developing drugs for brain cancer are likely to be rooting for this team since they are all working to transform brain cancer care, in different ways.
NOTE TO INVESTORS: The latest news and updates relating to CNS Pharmaceuticals Inc. (NASDAQ: CNSP) are available in the company’s newsroom at https://ibn.fm/CNSP
Please see full terms of use and disclaimers on the BioMedWire website applicable to all content provided by BMW, wherever published or re-published: http://BMW.fm/Disclaimer
