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Experimental pill sends a THIRD of patients with leukemia into complete remission in early trial


An experimental leukemia pill has shown promise in early clinical trials, bringing a third of patients with aggressive disease into complete remission.

The drug, revumenib, was also credited with putting half of acute myeloid leukemia (AML) patients in partial remission during phase one clinical trials.

Dr. Scott Armstrong, a blood cancer expert at the Dana-Farber Cancer Institute in Boston, Massachusetts, who is leading the trial, called the results “very encouraging.” But he said more research was needed to show that the drug worked.

If more rigorous trials are successful, he said, his team could apply for Food and Drug Administration (FDA) approval by the end of this year. The initial trial did not include a control group, meaning the drug was not tested against current treatments.

Researchers at the Dana-Farber Cancer Institute in Boston, Massachusetts, said a third of patients treated with revumenib went into complete remission (file image)

Acute myeloid leukemia is a type of blood cancer that affects white blood cells, causing them to start dividing uncontrollably and become abnormal, leaving them unable to fight infection.

There are about 59,000 cases diagnosed each year in the United States and 23,700 people die from cancer.

It is notoriously difficult to treat because the cells involved are diverse and can mutate rapidly, making them hard to target with novel treatments like immunotherapy.

They also divide quickly and spread to different areas of the body, making doctors scramble to remove them without harming healthy tissue.

In the study, published this month in the journal Naturethe researchers focused on leukemias triggered by mutations in the NPM1 and KMT2A genes.

This represents about 40 percent of leukemia cases in the United States. Up to 80 percent of people diagnosed with these mutations in their leukemia die within five years.

In leukemia with these mutations, a protein called menin binds to genes that keep cancer cells growing and dividing.

To break this cycle, revumenib uses a molecule that binds to menin, preventing it from attaching to genes and thus activating them.

in his studio, the scientists administered the drug to 68 patients who had relapsed leukemia or leukemia that did not respond to treatment. Almost all had leukemia with NPM1 or KMT2A mutations and were around 42 years old.

Patients were asked to take the drug, administered as a capsule, twice a day, with each dose 12 hours apart.

They were tracked for about a year on average.

The results showed that a total of 18 patients (30 percent) had a complete remission of their cancer during the study, meaning that all signs and symptoms of the cancer had disappeared.

The average time to achieve complete remission was two months, the scientists said.

Another 32 patients (53 percent) were also shown to have a partial remission, or a decrease in the size of their tumors or the extent of cancer in the body.

They remained in partial remission for nine months.

In general, the patients survived for about seven months after the start of the study.

Almost all patients experienced side effects, the most common being irregular heartbeat and nausea.

Seven people had to withdraw from the study due to a severe reaction to the drug.

However, there was evidence in some participants that cancer cells had developed resistance to the drug.

Dr Armstrong, who led the study, said: “For patients with acute leukemia who have undergone several prior treatments, this is a very encouraging result.

“However, after the second treatment cycle, some patients developed resistance to revumenib.”

Phase one trials are designed to test the safety and optimal dosage of an experimental treatment.

Currently, people with leukemia mutated in NPM1 or mutations in KMT2A are offered chemotherapy to fight the disease.

Surgery is rarely used to treat acute myeloid leukemia. Radiation therapy may be used in some cases where cancer has spread outside of the bone marrow and blood.

About two out of three of these patients will go into remission, says the American Cancer Society.

Doctors say that people who have NPM1 leukemia tend to respond better to chemotherapy treatments.


Leukemia is a cancer that begins in blood-forming tissue, usually the bone marrow.

It leads to the excessive production of abnormal white blood cells, which fight infection.

But a higher number of white blood cells means there is “less room” for other cells, including red blood cells, which carry oxygen throughout the body, and platelets, which cause blood to clot when the skin is cut.

There are many different types of leukemia, defined by the immune cells they affect and how the disease progresses.

For all types combined, 9,900 people in the UK were diagnosed with leukemia in 2015, Cancer Research UK statistics reveal.

And in the US, about 60,300 people were told they had the disease last year, according to the National Cancer Institute.

Most cases have no obvious cause, since cancer is not contagious or hereditary.

Leukemia generally becomes more common with age, with the exception of acute lymphoblastic leukemia, which peaks in children.

Other risk factors include being male, being exposed to certain chemicals or radiation, and some bone marrow disorders.

The symptoms are generally vague and get worse over time.

These may include:

  • Fatigue
  • frequent infections
  • sweats
  • bruises
  • Heavy periods, nosebleeds, or bleeding gums
  • palpitations
  • Difficulty breathing

Acute leukemia, which progresses rapidly and aggressively, is often curable with chemotherapy, radiation therapy, or stem cell transplantation.

Chronic forms of the disease, which generally progress slowly, tend to be incurable; however, these patients can often live with the disease.

Fountain: leukemia care

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