Patients battling incurable leukemia blood cancer have been given a lifeline, thanks to an experimental pill that has been described as “like something out of a science fiction movie”.
In one of the first tests of the drug, known as NX-5948 and so new it has no name, a 59-year-old patient who had run out of treatment options and had only a few months to live, has seen his cancer almost It disappeared after he started taking the pills.
Joe Murphy, from Hulme, Greater Manchester, is only the second patient in the world to be offered the treatment at The Christie NHS Foundation Trust, and says it has brought him “back from the brink”.
The former bar manager was diagnosed with an aggressive form of chronic lymphocytic leukemia (CLL) in 2015, which had recently spread to the cerebrospinal fluid and brain.
The disease, which causes abnormal buildup of white blood cells in the blood and bone marrow, can be controlled initially with tablets, allowing patients to live almost a decade longer after diagnosis.
Joe Murphy, from Hulme, Greater Manchester, is the second patient in the world to receive the revolutionary new pill and says it has brought him “back from the brink”.
Mr. Murphy was diagnosed with a very aggressive form of leukemia, which allows abnormal white blood cells to build up in the blood and bone marrow (artist’s impression above).
However, over time, cancer cells become resistant to treatments. This means that, for patients diagnosed at a younger age, there is a desperate need for more medications. CLL kills more than 1,000 people in the UK each year.
Last year, Joe stopped responding to the third medication he was taking to control his cancer. He lost more than a quarter of his body weight and, as his immune system weakened, he was hospitalized with sepsis and meningitis.
But today, thanks to being offered the pioneering drug, tests show almost no evidence of disease. “I wouldn’t be here today if it weren’t for the trial,” Joe said. ‘After the failure of the third treatment, I was preparing to die. I wasn’t in a good place.
‘But this drug has kept me alive and healthy since June last year. It’s incredible, like something out of a science fiction movie.
“I’m gaining weight back, my blood count is fine, my lymph nodes have shrunk to almost normal and it has worked successfully to get rid of the cancer in my brain, which is a huge relief.”
While doctors don’t know how long he could keep the disease at bay, Joe says he is now “hopeful” and can look forward to celebrating his 60th birthday in December.
CLL, which affects more than 4,500 Britons each year, is the most common blood cancer in adults, accounting for 38 per cent of all leukemia cases.
About 40 percent are 75 years old or older. People with a family history of CLL are more likely to get the disease.
It is more common among men, for reasons scientists do not yet understand.
Standard treatment for CLL includes inhibitors, which block the signals that make cancer cells grow, immunotherapy, which works by binding to cancer cells and killing them, and chemotherapy.
Patients may also be offered combinations of these treatments, which studies show give better results. NX-5948, developed by San Francisco-based pharmaceutical firm Nurix Therapeutics, belongs to a new class of treatments for blood cancers known as degraders.
They work by taking advantage of the natural elimination system within cells; In this case, they target a specific protein that CLL cancers need to grow and “tag” it to destroy it.
Dr Emma Searle, consultant hematologist at The Christie and principal investigator of the trial, said: “This could be the breakthrough we have been looking for in the treatment of CLL.
“The drug targets a pathway that CLL cells are particularly dependent on and basically destroys it.
‘As doctors, we are excited because we are seeing a response, even at low doses, in patients who have exhausted all standard care options and are very difficult to treat.
‘Seeing that some of our patients like Joe are already responding so well to treatment, with minimal side effects, is very promising.
“Only time will tell if this makes it to market, but it’s about as promising as an early-stage study can be.”
The US regulator, the Food and Drug Administration (FDA), has already given the treatment fast-track designation – meaning patients can access it more quickly – because early results are so promising.
Other phases of the trial are underway.
