Cells and pieces of the tumors were detached from the tumor, and analysis of urine samples showed large amounts of cells that had taken up the medicine. The tumor also showed evidence of uptake of Alpha1H and apoptosis, which is a beneficial form of cell death. Advanced molecular analyses also showed interesting and specific responses in tumors treated with Alpha1H, for example that Alpha1H switching off the expression of a large number of different cancer genes, which proved to be an important milestone.

The figure gives an overview of the placebo-controlled study and treatment of bladder cancer with Alpha1H. The images show how Alpha1H is administered and how different effects can be evaluated using clinical techniques and in different patient samples. Patients secrete large amounts of cells, often containing the drug, the tumor shrinks in size and analysis of immune responses provides important information on the effect on tumor tissue.

Successful placebo-controlled study published in Nature Communications >>

1. Reduction in tumor size

• • Treatment resulted in a complete or partial response in 88% of tumors treated with 8.5 mM and in 47% treated with 1.7 mM Alpha1H.

2. Changes in the tumor

• • The treatment resulted in the dissolution of the tumor and the release of fragments and cells into the urine. This effect increased significantly with higher doses of Alpha1H.

• • The tumor cells died by a mechanism called apoptosis after taking up Alpha1H.

• • Pieces of tissue from the tumor that remained in the patient after the treatment had lost some of their tumor characteristics and become more like healthy tissue, as shown by gene expression analysis.

The manuscript describing this study has been published in Cancer Medicine >>

Researchers at Lund University, in collaboration with Hamlet BioPharma, have long experience in research on infections and immunity and have identified key control mechanisms for the balance between defense and disease. These molecular insights make it possible to select compounds that are suitable for controlling the immune response and reducing disease caused by infections. One example is the study of the immune molecule Interleukin-1 (IL-1) and its receptor IL-1RA. An existing drug, Kineret, contains the substance anakinra and inhibits the effects of IL-1.

The company has announced positive results from the controlled Phase II clinical trial in patients with recurrent acute cystitis. The study compared antibiotic treatment with immunotherapy (anakinra) in patients with recurrent urinary tract infections, a study conducted in collaboration with specialists in Giessen, Germany. Both treatments showed significant reduction of symptoms and improved quality of life, with no difference in efficacy between the two patient groups.

Immunotherapy with anakinra can thus reduce the need for antibiotics, which is an important aspect in the fight against resistant bacteria. In addition, the normal bacterial flora can be preserved, which in turn helps to counteract antimicrobial resistance. The next step is a more comprehensive analysis of the study results, which will be submitted for scientific publication.

This approach opens up new possibilities to treat antibiotic-resistant bacteria and shifts the focus from direct elimination of bacteria to strengthening the host’s antibacterial defences, offering an alternative solution to the growing global health threat of antibiotic resistance identified by the WHO.

Bladder Pain Syndrome (BPS), also known as interstitial cystitis, is a chronic disease involving bladder pain and discomfort estimated to affect between 4 to 12 million people in the United States, with the majority of cases being women (approximately 80-90%) (ICHelp)(BladderSmart). BPS causes chronic pain and discomfort in the bladder, often without an infection or other identifiable cause. The disease significantly affects quality of life and requires long-term management and treatment.

Patients with BPS have severe pain and other symptoms, which are socially disabling. As conventional painkillers are not effective, some patients are treated with morphine or surgery, often without lasting effects. The compound anakinra (IL-1RA), patented by the company for the treatment of bladder pain conditions, has shown promising effects in patients receiving off-label treatment. Pain scores decreased after treatment and quality of life increased in this severely disabled patient group. In addition, the laboratory tests showed a convincing reduction in pain molecules after treatment, suggesting a direct effect of the treatment at the molecular level.

Hamlet BioPharma is therefore continuing the clinical programme with a controlled phase II study in this patient group. The study was initiated with a screening phase, to identify patients who responded to treatment.  A significant proportion of patients treated with anakinra responded favorably to treatment.