New Drug May Improve Red Blood Cell Count in Patients with Rare Anemia

A group of physician-scientists conducted a study on the safety and efficacy of a new drug for patients with pyruvate kinase deficiency. This disorder is a rare type of anemia caused by a lack of pyruvate kinase, a key enzyme in red blood cell production and survival. According to the Dana-Farber Cancer Institute, there have been no disease-modifying treatments for the deficiency since the discovery of the disorder in the 1960s. Patients with pyruvate kinase
deficiency could only be treated for the symptoms of the disorder, most often with a splenectomy, cholecystectomy, and/or blood transfusions.

A study published in the New England Journal of Medicine has shown that the new oral drug, mitapivat, can raise hemoglobin levels in about half of study participants with pyruvate kinase deficiency. In a typical patient with this disorder, red blood cells break down in just a few weeks due to a lack of pyruvate kinase. Mitapivat works by activating this enzyme in red blood cells,
which helps prevent premature breakdown of the hemoglobin-carrying cells.

The breakdown of red blood cells and subsequent hemolytic anemia causes some patients to face severe complications, including gallstones, pulmonary hypertension, and cirrhosis. In addition, the medical procedures completed to relieve symptoms, such as splenectomies, have their own list of potential complications. Mitapivat provides an alternative by modifying the disease directly, rather than the symptoms of the anemia.

While mitapivat represents an important step forward in the treatment methodology for this rare genetic disease, further research needs to be conducted to determine the safety of the drug long-term and for children. Mitapivat also saw improvement in only half of participants, so continuing research is needed for a drug for those who do not respond to mitapivat.

Is Interventional Oncology the Future of Cancer Treatment?

While medical, surgical and radiation oncology have all been accepted in the medical community as effective cancer treatments, interventional oncology has begun to pick up steam as a potential alternative treatment with fewer side effects. Surgery, radiation, chemotherapy, and other traditional cancer therapies have an array of complications and side effects, including pain and risk of death. Using imaging and a catheter-based approach to cancer treatment, interventional oncology seeks to maximize the effectiveness of the treatment without disrupting the remainder of the body.

Interventional oncology actually finds its roots in interventional radiography, which is a subspecialty of radiology that uses imaging coupled with interventional procedures to diagnose and treat diseases. One of the goals of interventional radiography is to use the least invasive method possible to treat a disease, and interventional oncology is guided by the same overarching principle.

One of the most valuable assets that interventional oncology can offer is its ability to aid in both cancer therapy and symptom palliation. Interventional oncology can be used to deliver cancer therapeutic agents directly to a tumor, and can provide the imaging necessary for precise treatment. Interventional oncology can also treat pain through neurolysis, ablation, and bone augmentation. According to Yale Medicine, interventional oncology can be used to treat liver, colorectal, lung, bone/soft tissue, kidney, and metastatic cancers.

The clear advantage to interventional oncology is its ability to treat tumors or symptoms as
effectively as traditional cancer therapies, but without the organ damage, side effects and complications of a traditional cancer treatment plan. As interventional oncology continues to gain authority in the rapidly evolving oncology space, there is potential for this minimally-invasive treatment method to become the future of fighting cancer.