A drug that was being developed in Scotland to treat a neglected disease could be repurposed to treat leukaemia after it was given fast track designation by US authorities.
Researchers at the University of Dundee said they are “delighted” after the drug was given the designation by the US Food and Drug Administration (FDA).
Originally known as DDD86481 and now named PCLX-001, it will be trialled in adult patients with relapsed or refractory acute myeloid leukaemia (AML).
PCLX-001 was originally developed by Dundee’s Drug Discovery Unit as part of a programme to treat African sleeping sickness.
This is another important step towards delivering real benefits to cancer patients— Professor David Gray, University of Dundee
The compound is an analogue of DDD85646, which inhibited an essential function in the parasite that causes sleeping sickness but was found not to be suitable to treat the latter stages of the disease that affect the brain.
During their research, the Dundee team discovered the compound could also kill some human cancer cells by the same process, but they could not identify which cancers were appropriate for clinical trials.
The entire family of related “NMT-inhibiting” compounds was then licensed by Canadian pharmaceutical company Pacylex Pharmaceuticals, which is developing PCLX-001 as a once-a-day oral pill initially to treat leukaemia and lymphoma.
Professor David Gray, head of biology at the Drug Discovery Unit (DDU) in Dundee, said: “This is an exciting development that demonstrates how complementary scientific research can have tremendous impact at the global level.
“We were frustrated in our efforts to develop DDD85646 as a drug to use against African sleeping sickness.
“At the same time, Dr Luc Berthiaume, from the University of Alberta, had a deep understanding of cancers he believed such compounds could work against, but did not have the compounds to prove his theories.
“It was therefore serendipitous that Luc was introduced to PCLX-001, then known as DDD86481, at a seminar he gave at Dundee and more fully at a subsequent conference he invited me to speak at.
“Everyone at the DDU is delighted to hear that PCLX-001 has received fast track designation. This is another important step towards delivering real benefits to cancer patients.”
Fast track designation facilitates drug development and speeds up the review of new therapies intended to treat or prevent serious conditions and address unmet medical needs.
In 2020, more than 474,500 new cases of leukaemia were reported globally with more than 311,500 deaths.
AML is one of the most common types of leukaemia in adults.
Pacylex, where Dr Berthiaume is chief scientific officer, is a clinical-stage company that develops therapies for leukaemia, lymphoma, and solid tumour cancers.
The FDA also recently approved PCLX-001 for phase one clinical study to start in AML patients in the coming months and granted it orphan drug designation.
Pacylex chief medical officer John Mackey said: “Acute myeloid leukaemia is a rapidly progressing, difficult-to-treat blood cancer with a poor prognosis.
“Because Pacylex’s nonclinical data with PCLX-001 suggest that AML may be the cancer type most sensitive to NMT inhibitors, we have been eager to advance PCLX-001 into clinical studies in AML patients in the near future.”
