Gallium Maltolate: A New Hope in the Treatment of Glioblastoma?

In recent months I have received several requests for information on gallium maltolate in the treatment of glioblastoma and so with the help of a volunteer, Mario Iannaccone who took an interest and studied the topic in depth, I decided to write this article.
We all know that glioblastoma multiforme (GBM) is known for its poor prognosis and poor long-term survival. In recent decades, progress in treatment has been limited, with the Stupp protocol, which combines surgery, temozolomide (TMZ) and radiotherapy, remaining the standard of care. However, a new frontier in research may offer unexpected hope: gallium maltolate.

Gallium maltolate is a promising compound in the treatment of glioblastoma. Wisconsin College of Medicine professor Jennifer Connelly has led numerous studies, indicating the potential of this compound in the treatment of GBM. But why gallium? Gallium, similar to iron in the periodic table, can “trick” tumor cells that require iron to grow, entering the cells through transferrin receptors and disrupting vital tumor processes.

Preclinical studies have shown that gallium maltolate taken orally effectively inhibits the growth of glioblastoma by targeting iron metabolism in tumor cells. Experiments on tumor cells and animal models have shown that gallium maltolate can significantly reduce the growth of GBM. In preclinical studies, glioblastoma cells treated with gallium showed a reduction in tumor volume and growth rate.

In 2022, the first phase 1 clinical trial of gallium maltolate for recurrent glioblastoma was launched. Preliminary results have been encouraging, showing not only good tolerability of the drug but also potential efficacy in reducing tumor size. In addition, the Food and Drug Administration (FDA) recently granted orphan drug designation to gallium maltolate for the treatment of relapsed and refractory glioblastoma.

An “orphan drug” is a drug developed to treat a rare disease, often defined as one that affects a limited number of people. Orphan drug designation is granted by regulatory agencies such as the Food and Drug Administration (FDA) in the United States or the European Medicines Agency (EMA) in Europe. This designation comes with several benefits for pharmaceutical companies. Orphan drug designation is important because rare diseases often do not receive the same attention and investment in research and development as more common conditions. These incentives help promote the development of treatments for rare diseases, offering new hope to patients with these conditions.

Another gallium-based compound, called hrBMP4, has been shown to act on brain cancer stem cells, blocking their growth without causing toxicity in the body. A phase 1 clinical trial has reported very promising results, paving the way for a phase 2 trial to confirm the efficacy of this new therapeutic approach.

The next step is the ongoing phase 2 clinical trial, which aims to further evaluate the efficacy and safety of gallium maltolate in patients with newly diagnosed and relapsed glioblastoma. This study is crucial to determine the optimal dose and verify the therapeutic activity of the drug.

The Medical College of Wisconsin, together with various partners and funders, is working hard to advance this research. If the results continue to be positive, gallium maltolate could represent a breakthrough in the treatment of GBM, offering new hope to patients suffering from this devastating tumor. At the moment, unfortunately there are no known active clinical trials in Italy for gallium maltolate in the treatment of glioblastoma.

There is still a long way to go to find a cure for glioblastoma, but gallium maltolate could be a significant step in that direction. With further research and clinical trials, it could become an integral part of GBM treatment, improving the quality of life and survival of patients. The fight against glioblastoma continues, and each new development brings with it the hope of a better future. Below are some useful references to delve deeper into what we have discussed in this article.

[1] Malignant brain tumors: clinical trial identifies a promising new biologic drug against cancer stem cells in the treatment of glioblastoma
[2] Clinical Trials Registry – NCT04319276
[3] IQ-AI has Orphan Drug Designation Obtained for Gallium Maltolate
[4] Glioblastoma: New Therapy Heading to Clinical Trial