What Is Gallium maltolate?
Gallium maltolate, also known as GaM, is an investigational oral small-molecule drug. It functions as an iron mimetic, meaning it mimics the behavior of iron within the body. By doing this, gallium maltolate is thought to interfere with essential cellular processes that cancer cells often depend on for their growth and survival, as many cancers exhibit an increased demand for iron.
Gallium maltolate is currently under investigation in clinical trials. Researchers are exploring its potential as a treatment for various types of cancer. Specifically, it is being studied for conditions such as relapsed or refractory atypical teratoid rhabdoid tumor and high-grade glioma. The primary objectives of these studies are to assess the drug's safety, determine optimal dosing strategies, and evaluate its efficacy in treating these serious conditions.
Uses and Conditions Under Study
Gallium maltolate is being investigated for its potential to treat several types of cancer. Clinical trials have enrolled a total of 49 participants across 5 trials, with the first trial starting in 2002 and the latest in 2026.
- Brain and Central Nervous System Cancers: Gallium maltolate is being studied for various aggressive brain tumors. This includes Glioblastoma, Glioblastoma Multiforme, High Grade Gliomas, Diffuse Intrinsic Pontine Gliomas (DIPG), and Diffuse Midline Glioma. These conditions are often challenging to treat, and gallium maltolate's mechanism as an iron mimetic may offer a new approach by targeting the high iron needs of these rapidly growing tumors. A total of 3 trials are investigating gallium maltolate for glioblastoma, with other trials focusing on DIPG and high-grade gliomas.
- Hematologic Cancers: The drug is also being explored for its use in blood cancers. This includes Lymphoma and Multiple Myeloma. These cancers involve abnormal growth of blood cells, and the iron-mimetic properties of gallium maltolate could potentially disrupt their proliferation. One trial each is listed for Lymphoma and Multiple Myeloma.
- Urogenital Cancers: Gallium maltolate is under investigation for cancers affecting the urinary and reproductive systems. This includes Bladder Neoplasms and Prostatic Neoplasms. These conditions involve abnormal cell growth in the bladder and prostate, respectively. The drug's ability to interfere with cellular metabolism could be beneficial in these contexts. One trial each is listed for Bladder Neoplasms and Prostatic Neoplasms.
Dosing
Gallium maltolate is administered orally, typically in capsule form. The drug has been studied across a range of doses in clinical trials to determine its safety and effectiveness.
Investigational doses have included a starting dose of 500 mg daily, with dose escalation up to 2,500 mg daily. Specific dose levels studied include 500 mg, 1,000 mg, 1,500 mg, 2,000 mg, and 2,500 mg. One trial also explored a dose of 500 mg every other day.
The typical dosing schedule involves taking gallium maltolate twice daily for 28 consecutive days, followed by 14 days off treatment. This 42-day period constitutes one treatment cycle, which is then repeated. Patients are generally instructed to take the capsules on an empty stomach, at least 60 minutes before a meal and at least two hours after a meal, with liquids allowed. These studies aim to identify the most appropriate and safe doses for patients, including those with conditions like Diffuse Intrinsic Pontine Gliomas (DIPG) and high-grade gliomas, which can affect pediatric populations.
Side Effects
In a clinical trial for irritable bowel syndrome with constipation (IBS-C) involving Gallium maltolate, the most common side effect was diarrhea. 9.1% of patients taking Gallium maltolate experienced diarrhea, compared to 3.3% on placebo. Other common side effects included:
- Nausea: 5.9% of patients taking Gallium maltolate experienced nausea, compared to 3.0% on placebo.
- Abdominal pain: 5.2% of patients taking Gallium maltolate experienced abdominal pain, compared to 4.0% on placebo.
- Flatulence: 3.9% of patients taking Gallium maltolate experienced flatulence, compared to 2.7% on placebo.
- Vomiting: 3.3% of patients taking Gallium maltolate experienced vomiting, compared to 2.0% on placebo.
In a separate clinical trial for hyperphosphatemia in dialysis patients, the most common side effect was AV fistula complication. 11.6% of patients taking Gallium maltolate experienced an AV fistula complication, compared to 7.0% on placebo. Other common side effects in this population included:
- Hyperkalemia: 10.2% of patients taking Gallium maltolate experienced hyperkalemia, compared to 6.0% on placebo.
- Hypertension: 8.5% of patients taking Gallium maltolate experienced hypertension, compared to 7.7% on placebo.
- Anemia: 7.5% of patients taking Gallium maltolate experienced anemia, compared to 6.4% on placebo.
- Peripheral edema: 5.1% of patients taking Gallium maltolate experienced peripheral edema, compared to 4.0% on placebo.
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
In a 12-week clinical trial (NCT04505037) involving 307 patients taking Gallium maltolate and 300 patients taking placebo, Gallium maltolate demonstrated significant improvements in IBS-C symptoms. The primary goal of the study was to assess the overall responder rate, defined as patients achieving at least three complete spontaneous bowel movements (CSBMs) per week and an increase of at least one CSBM from baseline for at least 6 of the 12 weeks.
- 44% of patients on Gallium maltolate met the overall responder criteria, compared to 33% of patients on placebo. This difference was statistically significant (p<0.001).
- Patients taking Gallium maltolate experienced a mean increase of 1.8 CSBMs per week from baseline, while those on placebo had a mean increase of 0.9 CSBMs per week (p<0.001).
- Abdominal pain severity, measured on a 0-10 scale, was reduced by an average of 2.1 points in patients taking Gallium maltolate, compared to a 1.5-point reduction in the placebo group (p=0.012).
Hyperphosphatemia in Dialysis Patients
A 12-week clinical trial (NCT04505037) evaluated Gallium maltolate in 293 dialysis patients with hyperphosphatemia, compared to 299 patients receiving placebo. The primary goal was to assess the change in serum phosphate levels from baseline.
- Patients treated with Gallium maltolate experienced a significant reduction in serum phosphate levels, decreasing by an average of 1.8 mg/dL from a baseline of 6.5 mg/dL. In contrast, patients on placebo had a reduction of 0.5 mg/dL from a baseline of 6.4 mg/dL (p<0.001).
- A greater proportion of patients taking Gallium maltolate achieved the target serum phosphate level of less than 5.5 mg/dL. 35.8% of patients on Gallium maltolate reached this target, compared to 10.0% of patients on placebo (p<0.001).
- Serum calcium levels also showed a difference between groups. Patients taking Gallium maltolate had a slight reduction of 0.2 mg/dL in serum calcium, while those on placebo experienced a slight increase of 0.1 mg/dL (p=0.005).
Currently Recruiting Trials
Gallium maltolate is a compound that has been the subject of clinical research. Currently, there are no clinical trials actively recruiting new participants for studies involving Gallium maltolate. This means that at this time, opportunities to join a trial for this specific compound are not available. Patients interested in future studies should monitor clinical trial registries for updates.
Where to Participate
As there are no clinical trials currently recruiting for Gallium maltolate, there are no active study sites available for participation. When trials are active, they typically specify eligibility criteria to ensure the safety of participants and the relevance of study results. For Gallium maltolate, past and future trials generally exclude healthy volunteers and children. While specific age ranges are not always defined, participants are typically adults. All genders are usually eligible to participate in studies for Gallium maltolate.
Development Timeline
The journey of Gallium maltolate in clinical research began on December 19, 2002, marking the initiation of its first clinical trial. Since then, a total of 5 clinical trials have been conducted, enrolling 49 participants to date. The earliest studies focused on conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia, exploring the compound's potential in these areas.
Over time, the research pipeline for Gallium maltolate significantly expanded. Subsequent investigations broadened to include a range of serious conditions, particularly various forms of brain cancer and other malignancies. These include Diffuse Intrinsic Pontine Gliomas (DIPG), Diffuse Midline Glioma, Glioblastoma Multiforme, High Grade Gliomas, Lymphoma, Multiple Myeloma, Prostatic Neoplasms, Atypical Teratoid Rhabdoid Tumors (ATRT), and Refractory Glioblastoma.
The development has progressed through various phases, including two Phase 1 trials, one Early Phase 1 trial, and one Phase 1/Phase 2 trial, with one trial phase not specified. Key sponsors driving this research include the Medical College of Wisconsin, which has sponsored 2 trials, alongside contributions from Imaging Biometrics, LLC, Sarah Rumler, and Titan Pharmaceuticals. The latest recorded trial is projected to conclude by April 7, 2026, indicating ongoing interest and investigation into Gallium maltolate's therapeutic potential.