Fighting brain cancer

Sience

An alternative treatment

Glioblastoma is one of the most aggressive brain cancers, growing and spreading quickly due to underlying genetic changes. This fast growth means tumor cells need large amounts of nutrients, especially amino acids, to survive. To meet this demand, tumor cells upregulate transport mechanisms, often bypassing normal regulatory controls. This vulnerability can be exploited through the use of an amino acid analogue that mimics natural substrates but cannot be metabolized. Once taken up, the analogue accumulates intracellularly, creating an osmotic imbalance that ultimately leads to tumor cell lysis.

This novel therapeutic modality offers an alternative to traditional chemotherapeutics and radiation therapy, which are often associated with significant side effects.

The drug candidate is based on diaminobutyric acid (DAB), an amino acid analogue that selectively targets the System A amino acid transport complex, which is upregulated in rapidly dividing glioblastoma cells. By exploiting this transport system, the compound is preferentially taken up by tumor cells, sparing healthy tissue.

Key competitive advantages of the Ambusol platform include:

  • A favorable safety profile, minimizing off-target toxicity
  • Scalable manufacturing suitable for large-volume production
  • Stable at ambient temperature, simplifying logistics and storage

Selected scientific publications to date:

  1. Alpha-aminoisobutyric acid transport into human glia and glioma cells in culture. Ronquist G, Agren G, Ponten J,  Westermark B. J Cell Physiol. 1976;89(3):433-9. doi: 10.1002/jcp.1040890309.
  2.  Induction of complete and irreversible damage to malignant glioma cells by L-2,4 diaminobutyric acid. Ronquist G, Westermark B, Hugosson R. Anticancer Res. 1984;4(4-5):225-8.
  3. Treatment of malignant glioma by a new therapeutic principle. Ronquist G, Hugosson R, Sjölander U, Ungerstedt U. Acta Neurochir (Wien). 1992;114(1-2):8-11. doi: 10.1007/BF01401106.
  4. Metabolic manipulation of glioblastoma in vivo by retrograde microdialysis of L-2, 4 diaminobutyric acid (DAB). Bergenheim AT, Roslin M, Ungerstedt U, Waldenström A, Henriksson R, Ronquist G. J Neurooncol. 2006 Dec;80(3):285-93. doi: 10.1007/s11060-006-9186-1.
  5. Membrane transport properties of L-2,4-diaminobutyrate revisited. Christensen HN, Ronquist G. J Membr Biol. 1992;127(1):1-7. doi: 10.1007/BF00232753.
  6. Post-resection Cavity Lavage of High Grade Glioma With a Novel Drug Combination: A Case Report. Ganapathy S, Venkataramana NK, Rao SA, Nagaraj A, Panayotou G, Panotopoulos C, Ronquist G. Anticancer Res. 2023;43(8):3583-3588. doi: 10.21873/anticanres.16537.
  7. Elimination of High-grade Gliomas Through Induced Cytolysis, Elucidated by Two Patient Cases. Panotopoulos C, Athanasiou G, Stavropoulos S, Boutos N, Rao S, Panayotou G, Venkataramana NK, Ronquist G. Anticancer Res. 2025 Jan;45(1):201-207. doi: 10.21873/anticanres.17405.