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Partners discover new drug to treat leukemia

Thanks to the work of British and Spanish partners, a new drug to treat cancer patients will soon be on the market.

Epileth, which was funded through EUREKA’s Eurostars programme, featured the combined work of Spanish biopharmaceutical company ORYZON Genomics and The University of Manchester in the UK.

As a result of the project, it was discovered that Leukemia could be treated by a drug which until now, had been used for other purposes.

A Dream Team

The hugely successful partnership was made possible through the work that both partners had been doing prior to the collaboration. 

In 2012, The University of Manchester proved that an enzyme called ‘LSD1’ was contributing to the onset and progression of a blood cancer called acute myeloid leukemia (AML).

Separately, ORYZON had been developing a drug that inhibited the impact of that very enzyme.

Given this blatant crossover in their areas of interest, it was only a matter of time before their individual work would bring them together.

“As humans, we rely on the ability of our genes to produce proteins, which we need to build and repair our muscles, skin and blood”, says Tim Somervaille, Senior Group Leader at the Cancer Research UK Manchester Institute. “However”, he continued, “sometimes a gene can be turned on too much, or turned on in the wrong place, a phenomenon which can sometimes contribute to the onset of cancer.”

So logically, when researchers are looking into the causes of any cancer, they need to uncover what exactly is keeping a gene ‘turned on’ in this way. In a report published by Somervaille’s laboratory in 2012, it was revealed that in the case of AML, the enzyme LSD1 was turned on too much in blood cells, contributing to the cancer by causing ‘poor differentiation of cells’.

“In AML the problem is that there are too many poorly differentiated, immature blood cells and not enough well differentiated, mature blood cells,” Somervaille explains. “By blocking the activity of the enzyme LSD1, by turning it off, we can convert the immature cells in to well differentiated mature cells. This is essentially a therapeutic benefit to the patient, so inhibitors of LSD1 help leukemia cells turn into something less dangerous.”

Major opportunities

Meanwhile, in Barcelona, ORYZON had been developing what is now known as ORY-1001, a drug designed specifically to inhibit the offending LSD1 enzyme. This LSD1 inhibitor, as the name suggests, is a drug designed to negate its negative effect.

“Before Epileth, we had been developing LSD1 inhibitors insofar as they related to genetic disorders like Parkinson's and Dementia,” says Carlos Buesa, CEO of ORYZON. “And although we had suspected that LSD1 might have been contributing to certain types of cancer, the conclusive evidence uncovered at The University of Manchester gave us an excellent reason to start testing the impact of our inhibitor on leukemia specifically.”

A major opportunity beckoned for both partners and following a meeting in 2012, it was quickly agreed that their shared areas of interest could lead to hugely important and pioneering work. A subsequent application to the Eurostars programme resulted in a project worth €1.5 million to begin performing clinical trials.

These trials would set out to answer a range of important questions; How safe would it be to administer this drug to AML patients? Would it be effective in the treatment of leukemia? How long would its effect last in the body? What would be the most appropriate dosage?

“The project results are very exciting,” Buesa says. “After having treated 41 patients with different sets of leukemia during the first round of clinical trials, we can now confirm, most importantly, that the drug is safe. We can also confirm that, as predicted by the earlier laboratory-based studies, the drug is effective, producing differentiation of AML in patients.”

“This has only been the first step of many but Epileth has given us a very realistic hope that this drug will be available as a reliable and effective treatment to cancer patients in the near future.”