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Pharmaxis discoveries showing promise in bone marrow cancer, according to Boston University School of Medicine publication

Published: 13:21 24 Mar 2021 AEDT

Pharmaxis Ltd - Pharmaxis discoveries showing promise in bone marrow cancer, according to Boston University School of Medicine publication
Based on this study, Pharmaxis has initiated Phase 1-2 clinical trials in humans.

Drugs from Pharmaxis Ltd’s lysyl oxidase inhibitor drug discovery program, have been found to be capable of slowing the progression of primary myelofibrosis (PMF), a relatively rare but painful type of bone marrow cancer, according to a new study by the Boston University School of Medicine (BUSM).

The study, led by corresponding author Professor Katya Ravid, DSc, was funded by the National Heart, Lung and Blood Institute and the findings were published online in the journal Archives of Stem Cell and Therapy.

The researchers hope that the study will contribute to the development of novel treatment options for primary myelofibrosis.

Based on its own extensive pre-clinical program and the results from this study, Pharmaxis has initiated Phase 1c/2 clinical trials in humans with its lead drug from this program, PXS-5505.

Unique drugs

According to Katya Ravid, Professor of Medicine and Biochemistry at BUSM and Professor of Biology at Boston University, these drugs are unique because they are able to inhibit their target, a protein called lysyl oxidase (LOX), with a combination of specificity and potency not seen in previously tested drugs. 

These current findings build upon previous discoveries and publications by Ravid and her group, showing what specific proteins in the bone marrow cause the scaring effect observed in PMF.

The study

Three experimental models were used for this study: the first group was normal, while the other two had gene alterations that caused them to develop cancer in their bone marrow. 

One of Pharmaxis LOX inhibitors (PXS-LOX_1) was given to all three groups. 

While it caused no significant changes in the normal models, it reduced the severity of the bone marrow cancer symptoms in both altered groups. 

In a separate experiment, another Pharmaxis drug (PXS-LOX_2 also known as PXS-5505) was given to the normal set and one of the genetically altered groups. 

This test resulted in no change in the normal group, but a reduction in the severity of the cancer symptoms of the altered models.

Findings significant

Professor Ravid, believes these findings are significant because they represent a possible novel avenue of treatment for PMF, since there are currently no available drugs that fight the diseased bone marrow matrix proteins in the way that the Pharmaxis LOX inhibitors fight it.

"Should these drugs eventually be approved for human use, they could help to slow the progression of the cancer and lessen the painful symptoms of the disease."

PMF 

PMF disrupts the body's normal production of blood cells by causing extensive scarring in the bone marrow. 

At the moment, few treatment options are available, with most of them being palliative in nature. 

The only curative treatment is a stem cell transplant, for which few patients are eligible.

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on 14/2/24