Menin inhibitors as a cancer treatment

Greenberger said there were multiple new and encouraging treatment modalities for leukemia at ASH 2022.

One of the most promising, he said, are menin inhibitors, which are targeted therapies for various types of acute myeloid leukemia (AML), a difficult-to-treat type of cancer that starts in the bone marrow but usually moves into the blood.

AML can spread to other parts of the body, including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles, according to the American Cancer Society.

One of the menin inhibitors showing promise is ziftomenib from Kura Oncology.

“I expect this treatment to be available for people within two to five years, depending on how future clinical trials go,” Greenberger said.

In a phase 1 study, 30% of people with NPM1-mutant AML exhibited complete remissions, complete disappearance of leukemia, and restoration of normal blood function when treated with ziftomenib.

Based on the data presented at ASH 2022, company officials said they will begin a phase 2 study to seek approval from the Food and Drug Administration (FDA).

Troy Wilson, Ph.D., the chairman and chief executive officer of Kura, told Healthline that ziftomenib has the potential to address approximately 35% of acute myeloid leukemia cases, including NPM1-mutant AML and KMT2A-rearranged AML.

“These are areas of significant unmet need for cancer patients since no approved targeted therapies currently exist,” Wilson said.

“We also believe that by combining ziftomenib with other cancer therapies, in an effort to minimize or prevent treatment resistance, we may be able to expand the patient population by potentially addressing up to 50 percent of acute leukemias,” he added.

Premature aging from cancer treatments

A broad spectrum of consumer issues was addressed at ASH this year, from the quality of life after treatment to maternal health, economic and racial inequities, and the potential harms of blood cancer treatments in young people.

One study that used long-term data from St. Jude Children’s Hospital showed that treatment for pediatric Hodgkin’s lymphoma can negatively affect patients’ neurocognitive function over their lifetime and even cause premature death.

The findings, which haven’t been published in a peer-reviewed journal yet, were presented at the ASH conference.

Annalynn M. Williams, Ph.D., an epidemiologist at Wilmot Cancer Institute at the University of Rochester in New York, who focuses on neuropsychological and psychosocial issues in adolescent and young adult cancer survivors, said she recently discovered something that did not make sense to her.

“It was nagging at my brain that there are a group of patients who don’t receive any treatment that are known to harm the central nervous system but were still having cognitive problems earlier than their peers,” she told Healthline.

Her new study of 215 survivors of pediatric Hodgkin’s lymphoma showed that long-term survivors are at elevated risk for cardiopulmonary morbidity, cognitive impairment, early onset of dementia, and premature death.

“The treatment causes epigenetic changes and over time they persist and accumulate because of what they are exposed to in daily life,” said Williams.

Lance Kawaguchi, a global venture philanthropist for children, teens, and young adults with cancer, told Healthline, “According to a 2015 global analysis, the most common cancers diagnosed in children were leukemia, non-Hodgkin’s lymphoma (NHL), brain and nervous system cancers, as well as other neoplasms. Many children who survive these cancers initially, in particular, NHL, progress to later stages and suffer long-term complications. This is simply devastating. After already enduring so much, they deserve better.”

Two cancer treatments are compared

One of the most anticipated studies at ASH was the head-to-head comparison between cancer treatments zanubrutinib and ibrutinib.

Zanubrutinib, the targeted drug from BeiGene that is sold under the brand name Brukinsa, showed better efficacy with fewer side effects than ibrutinib in a study.

The findings, which haven’t been published yet in a peer-reviewed journal, were presented at the ASH conference.

Ibrutinib is manufactured by Pharmacyclics, an AbbVie company, and Janssen Biotech. It is sold under the brand name Imbruvica.

It was the first head-to-head comparison between the two drugs among people with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

Ibrutinib, currently a standard therapy for CLL and SLL, was the first Bruton tyrosine kinase (BTK) inhibitor to gain FDA approval.

The newer drug, zanubrutinib, is a BTK inhibitor that has received FDA approval for treating several types of cancer.

BTK inhibitors as a cancer treatment

BTK inhibitors are given orally and work by interfering with a key signaling pathway in cancer cells.

Dr. Jennifer R. Brown, a medical oncology specialist at the Dana-Farber Cancer Institute, said in an ASH press release that “zanubrutinib not only improves the response rate, it also improves progression-free survival compared to ibrutinib, including in our highest risk patients.”

She added that progression-free survival “is pretty much our gold standard for efficacy, so our data suggest that zanubrutinib should really become the standard of care in this setting.”

At two years, 79% of people taking zanubrutinib and 67% of those taking ibrutinib were still alive without evidence of their cancer returning, according to the ASH press announcement.

Dr. Mehrdad Mobasher, the chief medical officer of hematology at BeiGene, told Healthline that the company believes these results could help this treatment emerge as a new standard of care in the treatment of CLL.

“We are awaiting an FDA decision on our submission for Brukinsa (zanubrutinib) to treat adult patients with CLL”, with a target date of January 20, he said.