Αρχική World News Targeted Drug Approved for Acute Myeloid Leukemia with IDH1 Gene Mutations

Targeted Drug Approved for Acute Myeloid Leukemia with IDH1 Gene Mutations

August 13, 2018, by NCI Staff

Approximately 6-10% of people with AML have a mutation in the IDH1 gene.

Credit: Adapted from PLOS One Sept. 15, 2015. https://doi.org/10.1371/journal.pone.0133813. CC BY 4.0

UPDATE: On May 2, 2019, FDA expanded its approval of ivosidenib (Tibsovo) for adult patients with acute myeloid leukemia (AML) that has a mutation in the IDH1 gene. Under this expanded approval, ivosidenib can be used to treat newly diagnosed patients age 75 or older or those with other health conditions, or comorbidities, that would preclude them from receiving the intensive chemotherapy that is a standard initial treatment for AML.

The new approval is based on results from a phase 1 clinical trial. In the trial, approximately 40% of participants saw all signs of their disease disappear, and approximately 40% of patients who entered the trial dependent on blood transfusions no longer needed transfusions for at least 8 weeks.

Side effects in newly diagnosed patients were similar to those in patients with relapsed or refractory disease.

On July 20, the Food and Drug Administration (FDA) approved ivosidenib (Tibsovo) for the treatment of adults with acute myeloid leukemia (AML) that has a specific mutation in a gene called IDH1. The approval covers patients whose AML has returned after earlier treatment (relapsed) or has not responded to standard therapies (refractory).

Ivosidenib is a tablet that patients take orally once a day.

FDA also approved a companion diagnostic test called the Abbott RealTime IDH1 assay, which can identify patients with IDH1 mutations who may benefit from ivosidenib. Approximately 6% to 10% of patients with AML have the specific IDH1 mutation.

Ivosidenib is the first FDA-approved treatment that inhibits abnormal proteins produced by mutated IDH1 genes. Blocking these proteins may help keep cancer cells from growing.

Last year, FDA approved an oral drug that inhibits abnormal forms of the IDH2 protein in patients with AML. The drug, enasidenib (Idhifa), was the first IDH2 inhibitor approved for AML, and there is a companion diagnostic test to identify specific mutations in the IDH2 gene.

Both enasidenib and ivosidenib are molecularly targeted therapies for AML that were approved based on the results of single-arm phase 1 studies, noted Roland Walter, M.D., Ph.D., an AML specialist at Fred Hutchinson Cancer Research Center in Seattle.

In these trials, the effectiveness of the drugs and their side effect profiles were “virtually identical,” Dr. Walter said. He noted that the effectiveness of the drugs was measured by the proportion of patients achieving morphologically-defined remission, which means they had less than 5% myeloblasts in the bone marrow and either a full or partial recovery of blood counts, and the durations of those remissions.

Dr. Walter cautioned, however, that more research is needed to compare enasidenib and ivosidenib with salvage therapy (treatments that are used when other therapies for AML fail).

“There are currently no comparative data available, and it is not clear whether—and to what degree—treatment outcomes are better with these molecularly targeted therapies compared with conventional salvage therapy,” he added.

Testing an IDH1 Inhibitor in Patients with AML

FDA’s approval of ivosidenib was based on results from 174 patients with relapsed or refractory disease in a clinical trial for adults with AML whose blood or bone marrow had a mutation in the IDH1 gene. Agios Pharmaceuticals, the maker of ivosidenib, funded the trial.

With a median follow-up of 8.3 months, following treatment with ivosidenib, nearly a third of the patients (32.8%) experienced complete remissions. The median duration of response was 8.2 months.

Of the 110 patients who needed transfusions of blood or platelets when they began the study because of their AML, 37.3% went at least 56 days without requiring a transfusion after treatment with ivosidenib, according to FDA.

Additional results from the trial appeared in the New England Journal of Medicine on June 21.

Common side effects of ivosidenib included fatigue, increase in white blood cells, joint pain, diarrhea, shortness of breath, and swelling in the arms or legs. Approximately 19% of patients experienced a side effect called differentiation syndrome, which includes fever and respiratory distress and can be fatal if not treated.

Physicians will have to become familiar with the side effects of this drug, such as differentiation syndrome, noted Dr. Walter. “There is still much to learn about ivosidenib and enasidenib, including how best to combine these drugs with others,” he added.

Source

NEWSLETTER

Συμπληρώστε το email σας για να λαμβάνετε τις σημαντικότερες ειδήσεις από το ogkologos.com

Βρείτε μας

2,449ΥποστηρικτέςΚάντε Like
57ΑκόλουθοιΑκολουθήστε

Διαβαστε Επίσης

ΕΞΕΛΙΞΕΙΣ ΣΤΗ ΘΕΡΑΠΕΙΑ ΤΟΥ ΜΗ-ΜΙΚΡΟΚΥΤΤΑΡΙΚΟΥ ΚΑΡΚΙΝΟΥ ΤΟΥ ΠΝΕΥΜΟΝΑ (ΜΜΚΠ)

ΕΞΕΛΙΞΕΙΣ ΣΤΗ ΘΕΡΑΠΕΙΑ ΤΟΥ ΜΗ-ΜΙΚΡΟΚΥΤΤΑΡΙΚΟΥ ΚΑΡΚΙΝΟΥ ΤΟΥ ΠΝΕΥΜΟΝΑ (ΜΜΚΠ) Γράφει ο Δρ Παπαδούρης Σάββας, Παθόλογος-Ογκολόγος   Ο ΜΜΚΠ βρίσκεται αναλογικά στο 80% και πλέον του συνολικού...

Διατρέχουν όντως οι καρκινοπαθείς μεγαλύτερο κίνδυνο λόγω κοροναϊού;

Σε πρακτικό επίπεδο, τα δεδομένα των σχετικών μελετών υποδηλώνουν ότι η χημειοθεραπεία ή οι άλλες αντι-νεοπλασματικές θεραπείες δεν αυξάνουν σημαντικά τον κίνδυνο θνησιμότητας από...

FDA: Η ακτινοβολία των smartphones δεν προκαλεί καρκίνο

Σε μια νέα έκθεσή της, η Υπηρεσία Τροφίμων και Φαρμάκων (FDA) των ΗΠΑ αναφέρει ότι επανεξέτασε τις σχετικές επιστημονικές έρευνες που δημοσιεύθηκαν τα τελευταία...

Νέα ανακάλυψη, νέα ελπίδα για τον καρκίνο

Ένα νεοανακαλυφθέν τμήμα του ανοσοποιητικού μας συστήματος θα μπορούσε να αξιοποιηθεί για την αντιμετώπιση όλων των ειδών καρκίνου, σύμφωνα με επιστήμονες του πανεπιστημίου Cardiff...

Καρκίνος: Ευεργετική για τους καρκινοπαθείς η άσκηση

Σημαντικά ωφέλη προσφέρει η άσκηση, ακόμη και σε ασθενείς με καρκίνο. Οι περισσότεροι ασθενείς αγνοούν τα οφέλη που μπορεί να έχει γι’ αυτούς η συχνή...

Prostate Cancer

The article is provided by Scientific Communications Officer of NIPD Genetics, Ms Marina Charitou (MSc) Prostate Cancer Awareness Prostate cancer is the second most common cancer...
- Advertisment -

Ροή Ειδήσεων

Targeted Therapy Based on Molecular Profiling of Malignant Mesothelioma Tumours Is Feasible

Targeted therapy recommendations can be made based on the molecular profiles of tumour samples from patients with metastatic malignant mesothelioma (MM) according to findings...

News digest – targeted radiation beam, precision medicine and our early detection roadmap

<img width="620" height="348" src="https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1.jpg" class="attachment-hero size-hero" alt srcset="https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1.jpg 620w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-300x168.jpg 300w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-220x123.jpg 220w, https://zniup3zx6m0ydqfpv9y6sgtf-wpengine.netdna-ssl.com/wp-content/uploads/2020/10/Radiation_hero-1-150x84.jpg 150w" sizes="(max-width: 620px) 100vw, 620px" data-attachment-id="18059" data-permalink="https://scienceblog.cancerresearchuk.org/2020/10/17/news-digest-targeted-radiation-beam-precision-medicine-and-our-early-detection-roadmap/radiation_hero-3/" data-orig-file="https://scienceblog.cancerresearchuk.org/wp-content/uploads/2020/10/Radiation_hero-1.jpg" data-orig-size="620,348" data-comments-opened="1"...

A Pet Pug Helped Two Sisters Realize They Both Had Breast Cancer

Sisters Amanda Tasca and Amy Niosi are grateful for one very perceptive pug. Amanda’s dog Daisy, a gift from her husband Steve and one...

Woman with Breast Cancer May Have Saved Her Twin’s Life by Revealing Her Lumpectomy Scar

When Meagan McCallum was diagnosed with breast cancer, she had no idea that her experience might actually save her sister’s life. Meagan underwent a...

Lo que debe saber sobre el cáncer de pulmón

La Dra. Jyoti D. Patel es profesora de Medicina y directora de Oncología Torácica de la Universidad de Chicago y es editora asociada para...

Alternatives for Outdoor Foods

Autumn has arrived, but that doesn’t mean outdoor dining has to end! You might be wondering what foods you could prepare that are healthy...