An experimental pill has achieved the complete remission of cancer in 18 near-terminal patients with aggressive tumors that did not respond to treatments. The illness, acute myeloid leukemia, is the most common blood cancer in adults, accounting for 120,000 cases each year. The three-year survival rate is just 25%. The new drug, called revumenib, has completely eliminated cancer in a third of the participants in a long-awaited clinical study in the United States. The results are preliminary and do not suggest a definitive cure, but the authors of the experiment are optimistic. “We think this pharmaceutical is extraordinarily effective, and we hope for it to be accessible to everyone who needs it,” says Dr. Ghayas Issa from the MD Anderson Cancer Center at the University of Texas.
Acute myeloid leukemia attacks bone marrow, where blood cells are produced, and causes the uncontrolled production of defective cells. That’s what happened to the 23-year-old Lithuanian architect Algimante Daugeliate. She had received two bone marrow transplants from her sister. All other treatments had failed. Her doctors had begun thinking about palliative care to alleviate her suffering. “I was desperate. It was like living through a horrible movie. I felt like death was imminent, and I was just 21 years old,” she recalls. Two years ago, she started to take revumenib pills. She finished college, and now she works at an architecture studio in Copenhagen.
The drug does not work for all patients. Researchers have focused on two genetic subtypes, in which a protein called menin allows leukemia to progress. Revumenib attaches to the protein and inhibits it, thanks to its complex chemical recipe: 32 carbon atoms, 47 of hydrogen, one fluoride, six nitrogen, four oxygen and one of silver. That formula, C32H47FN6O4S, has saved 18 lives. The promising results were published on Wednesday in Nature.
I felt like death was imminent and I was only 21 years old
Algimante Daugelaite, architect
Hematologist Pau Montesinos, coordinator of the Spanish Group of Acute Myeloid Leukemia, believes that the new information is “fairly hopeful,” but she emphasizes the caveats: revumenib still must be tested on hundreds of people to confirm its safety and effectiveness. Montesinos’s team, the Leukemia Unit at the La Fe hospital in Valencia, will participate in the next international trials of the pill, developed by the American company Syndax Pharmaceuticals.
Montesinos also adds that the pharmaceutical alone is not a panacea. “In the vast majority of cases, these targeted therapies, on their own, can revert leukemia, but rarely cure it,” the hematologist explains. “The strategy is to combine these new pharmaceuticals with classic chemotherapy or other approaches.” Montesinos recalls the case of another pill, quizartinib, an experimental treatment by the Japanese pharmaceutical company Daiichi Sankyo, which inhibits a different protein involved in acute myeloid leukemia. Adding quizartinib to chemotherapy increased remission from almost 40% to almost 50%, according to the preliminary results of a trial with 500 patients who suffer from another subtype. “For us, increasing survival by 10 percentage points is a lot,” the Spanish doctor says.
Revumenib’s mechanism of action – the inhibition of the menin protein – is new. Half a dozen pharmaceutical companies are developing substances with the same tactics. Revumenib’s success is good news for them. The oncologist Ghayas Issa calculates that the new pills could benefit almost 400,000 people with acute leukemias that are resistant to other treatments, including myeloid and the strain most common in children, called lymphocytic.
These targeted therapies, on their own, can revert leukemia, but rarely cure it
Pau Montesinos, hematologist
Issa and his colleagues agree that economic factors will be key if the pill is approved. The prices of the latest oral pharmaceuticals against cancer tend to be over $235,000 per patient in the US, according to a report by Democratic congressperson Katie Porter.
Revumenib has one other weak point, as pointed out by hematologist Eytan Stein of the Memorial Sloan Kettering Cancer Center in New York, who led the trials. “The main Achilles heel seems to be the development of mutations at the drug’s fusion site, which causes resistance,” the researcher explains. Revumenib had some positive effect on half of the 60 participants in the clinical trial. In some of the patients, though, the menin protein changed slightly, causing resistance to the treatment, similar to how bacteria mutate to become antibiotic-resistant.
“This demonstrates that we’re on the right path, and that the target of the drug [the menin protein] is critical for the development of leukemia with these genetic subtypes,” Stein says. To avoid such resistance mutations, the authors propose combining pharmaceuticals with different mechanisms of action. As Ghayas Issa and his colleague Eytan Stein observe, menin inhibitors “will definitely be part of a treatment for these leukemias.” The architect Algimante Daugelaite is grateful that she was part of the trial, saying that science has given her “another opportunity to study, work, travel, see the world, and, most importantly, live.”
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FAQs
The architect Algimante Daugelaite has seen the complete remission of her aggressive leukemia thanks to the pharmaceutical revumenib. An experimental pill has achieved the complete remission of cancer in 18 near-terminal patients with aggressive tumors that did not respond to treatments.
Is there a pill for leukemia remission? ›
The drug, revumenib, is part of a group, or class, of drugs known as menin inhibitors. In an early-phase clinical trial called AUGMENT-101, treatment with revumenib caused about one-third of study participants' cancers to completely disappear, known as a complete remission.
What is the experimental pill for leukemia? ›
Revumenib is a new class of targeted therapy for acute leukaemia that inhibits a specific protein called menin. The drug works by reprogramming leukaemia cells back into normal cells.
What is the cancer pill for leukemia? ›
BCL-2 inhibitor
Venetoclax (Venclexta) targets BCL-2, a protein in cancer cells that helps them live longer than they should. This drug can be used with chemo in people newly diagnosed with AML who are 75 years or older, or who are not healthy enough to tolerate strong chemo. It's taken by mouth once a day.
What is the new pill for leukemia? ›
Rye Brook, N.Y., December 8, 2023 – The U.S. Food and Drug Administration has approved pirtobrutinib (Jaypirca™) for the treatment of adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) that has worsened or returned despite at least two earlier forms of treatment.
Can leukemia be treated with a pill? ›
Depending on your diagnosis, you may be prescribed a targeted therapy drug like BCR-ABL1 inhibitors, BTK inhibitors, or BCL2 inhibitors. These have been shown to be very effective, but successful treatment means you may have to take a pill a day for the rest of your life.
How long can you live with leukemia in remission? ›
About 80 percent who go into remission will do so within 1 month of therapy. In some people, however, the disease will return, lowering the cure rate. Chronic lymphocytic leukemia (CLL): On average, people with this cancer survive 9 years, although some have lived for decades, cancer always comes back at some point.
What is the newest treatment for leukemia? ›
CAR T cell therapy is one of the newest, most advanced ways to treat certain types of leukemia. We take T cells (immune cells) from your blood, then engineer them to fight leukemia. We infuse the engineered cells back into your body.
Has anyone been cured from leukemia? ›
While leukemia is currently not curable, it is possible to treat the cancer to help improve outlook. For children with acute lymphocytic leukemia (ALL), the 5-year survival rate is now around 90%, according to the American Cancer Society .
What is the new drug for leukaemia? ›
The drug, which is known as NX-5948, and so new it doesn't have a name, is for the treatment of patients with CLL (chronic lymphocytic leukaemia) whose disease has come back after previous treatment or the cancer has stopped responding to treatment. CLL is a type of cancer of the blood and bone marrow.
A medicine called Folotyn, approved earlier this year for patients with a rare form of lymphoma, costs $30,000 per month, the New York Times reports. The drug hasn't been proven to extend patients' lives; in a study cited by the FDA, tumors shrank in 27% of patients who took the drug.
What is the miracle drug for cancer treatment? ›
The drug, dostarlimab, targets a specific variant of colorectal cancer. While it is still being clinically trialled, it is already showing remarkable results – avoiding the need for surgery, radiotherapy or chemotherapy. Carrie Downey was diagnosed a year ago and given dostarlimab infusions for six months.
Is there a pill that stops cancer? ›
mTOR blockers (inhibitors) can stop the growth of some types of cancer. mTOR inhibitors include: temsirolimus (Torisel) everolimus (Afinitor)
How much does the leukemia pill cost? ›
Treating leukemia can be expensive. A study from 2012 found that some prescription drugs for leukemia had a total one-year cost that ranged from $70,000 to $120,000. Cost is a major reason for nonadherence to treatment for leukemia and other cancers.
What is the hardest leukemia to treat? ›
Acute myeloid leukemia (AML) is the most common type of acute leukemia, though it is rare, accounting for only about 1% of all cancers overall. AML is also the most fatal type of leukemia. The five-year survival rate for AML, that is, how many people will be alive 5 years after diagnosis, is 29.5%.
Can leukemia be cured in adults? ›
There isn't a cure for leukemia, but this doesn't mean some people don't achieve long-term remission.
What is the oral pill for leukemia? ›
VENCLEXTA is a prescription medicine used: to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who: are 75 years of age or older, or.
What is the remission therapy for leukemia? ›
Your effort to get into remission starts with the first phase of AML treatment, called remission-induction therapy. You get high-dose chemotherapy to kill as many leukemia cells as possible in your blood and bone marrow -- the spongy place inside your bones that makes blood cells.
How to keep leukemia in remission? ›
In some situations, a third drug might be added as well to try to improve the chances of putting the leukemia into remission: For people whose leukemia cells have an FLT3 gene mutation, a targeted therapy drug such as midostaurin (Rydapt) or quizartinib (Vanflyta) might be given along with chemo.
What is the daily chemo pill for leukemia? ›
The chemo drug hydroxyurea (Hydrea®) is taken as a pill, and can help quickly lower very high white blood cell counts and shrink an enlarged spleen. Other drugs sometimes used include cytarabine (Ara-C), busulfan, cyclophosphamide (Cytoxan®), and vincristine (Oncovin®).
