Saol Therapeutics News

Saol Therapeutics enhances its IP portfolio for Dichloroacetate (DCA; SL-1009) in Glioblastoma and Rare Pediatric Cancers

  • By signing an agreement with Medosome Biotec
  • By providing support for clinical trial treatment of GBM at the University of Florida
  • By starting a collaborative research project in rare pediatric cancers with a leading cancer center

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, March 15, 2023 / PRNewswire/ –

Saol Therapeutics (“Saol”), a privately held, clinical-stage pharmaceutical company, announced today the signing of an agreement with Medosome Biotec to provide Saol the rights to use their patented genotype test for all potential indications where SL-1009 might be used as a therapy.   The test is performed to identify potential mutations at GSTZ1 (glutathione S-transferase zeta 1 human enzyme) that categorize individuals as fast or slow metabolizers of DCA (SL-1009).   Utilization of the genotype testing enables individual dosing, potentially reducing the incidence of treatment related adverse events.  Prior to this agreement Saol only had rights to the patented genotype test for the indication of Pyruvate Dehydrogenase Complex Deficiency (PDCD).

As part of the agreement Saol will aid patient recruitment efforts for the Phase IIA Trial of DCA in Glioblastoma Multiforme (GBM) by providing additional funding and resource support.  Glioblastoma is aggressive and the most common type of primary brain cancer in adults.  GBM is difficult to treat and there is no known cure.  This study, sponsored by the University of Florida and the Food and Drug Administration (FDA) (NCT05120284), is further supported in partnership with Medosome Biotec and Saol.

Dr. Peter Stacpoole is Principal Investigator for the PDCD trial and the GBM trial and is Professor of Medicine at University of Florida. Dr. Stacpoole stated, “We are pleased to expand our partnership with Saol Therapeutics.  Our partnership has led to over-enrollment of our Phase III study in PDCD (NCT02616484) and we mutually see the broader potential application of DCA as the prototypic inhibitor of Pyruvate Dehydrogenase Kinases (PDKs) that, in turn, inhibit PDC enzymatic activity. We look forward to collaborating with Saol on potential additional indications for DCA.  Expansion of our collaboration on the GBM trial will result in the addition of more study sites, allowing for quicker recruitment of patients.  We are hopeful that DCA will be an important addition to the treatment of GBM.”

Saol also recently signed a sponsored research agreement with a leading cancer center to evaluate DCA in rare pediatric solid tumors.  The preclinical research will investigate DCA alone and in combination with chemotherapy in several different pediatric tumor models, such as Wilms, Neuroblastoma, Rhabdoid Tumor, Osteosarcoma, Ewing Sarcoma, and Rhabdomyosarcoma.

Literature suggests that DCA could be effective in several rare, pediatric, solid tumors.  The rationale for the pediatric tumor screening includes the fact that DCA has been widely studied in animals and humans and has a mechanism of action that might override the Warburg effect seen in cancer cells1,2,3,4.  Saol is currently studying this medicine in children using a proprietary formulation developed for pediatric use (NCT02616484).  “If the pre-clinical assessment provides encouraging data, the work already in pediatric patients may allow us to quickly transition toward clinical investigation”, commented Dr. Virinder Nohria, Chairman and Chief Medical Officer at Saol.

These agreements, and the additional investments behind them, represent an important next step in the evolution of Saol Therapeutics as an emerging pharmaceutical company.  “Now that we have completed enrollment in the PDCD trial we are rapidly transitioning to other important therapeutic areas, and are evaluating the utility of DCA (SL-1009) in adult and pediatric oncology indications with limited treatment options”, said Saol CEO David Penake. “We are pleased to have executed these agreements to support our pursuit of expanded opportunities beyond PDCD”.

Saol expects to have topline data in the PDCD trial in the 3rd quarter of 2023. Should the results of the trial support a subsequent FDA approval, it will be the first and only medicine approved for this rare, pediatric indication.


  1. 1. Aminzadeh S, Vidali S, Sperl W, Kofler B, Feichtinger RG. Energy metabolism in neuroblastoma and Wilms tumor. Transl Pediatr. 2015 Jan;4(1):20-32. doi: 10.3978/j.issn.2224-4336.2015.01.04.
  2. 2. Guo JQ, Tang HY, Wang CD, Sang BT, Liu X, Yi FP, Liu GL, Wu XM. Influence of Dichloroacetate on Wilms' Tumor in vitro. Ann Clin Lab Sci. 2022 Jan;52(1):101-108. PMID: 35181623.
  3. 3. Kankotia S, Stacpoole PW. Dichloroacetate and cancer: new home for an orphan drug? Biochim Biophys Acta. 2014 Dec;1846(2):617-29. doi: 10.1016/j.bbcan.2014.08.005.
  4. 4. Tataranni T, Piccoli C. Dichloroacetate (DCA) and Cancer: An Overview towards Clinical Applications. Oxid Med Cell Longev. 2019 Nov 14; 2019:8201079. doi: 10.1155/2019/8201079. PMID: 31827705; PMCID: PMC6885244.


About Dichloroacetate (DCA; SL-1009) 

DCA, a pan- PDK inhibitor, has the potential to be the 1st approved medication for the mitochondrial disease PDCD and will be available as an oral solution.  Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. However, PDC is also inhibited by PDKs, that may be over-expressed in PDCD. DCA inhibits PDKs to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. DCA dosing is based on a proprietary genetic test that dichotomizes subjects into “fast” and “slow” drug metabolizers, providing individualized dosing.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda.  Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases.  Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations.  For more information, visit

About Medosome Biotec

Medosome Biotec, LLC is a privately held, preclinical and early-stage clinical pharmaceutical company with operations in Alachua, FL and Bloomington, IN.  The Company focuses on pediatric diseases with an emphasis on developing and offering genetic tests for diagnosing rare diseases and providing personalized dosing of pharmaceutical drugs.  For more information, visit

Media contact:  Brian Nappi, Senior Vice President Strategy,