A glioma is a type of brain tumor that begins to multiply in the cells of the brain or spinal cord.. The cells of these gliomas are very similar to healthy brain neurons (glial cells) that surround nerve cells and help them to function. Not all gliomas are considered cancerous and grow slowly. If they are malignant, they can invade healthy tissues and grow quickly. On the other hand, there are gliomas that affect adults and others that affect children.
A very common type of tumor
According to the Mayo Clinic on its website, gliomas account for up to 33 percent of brain tumors. This type of tumor is known as intra-axial because it grows within the white matter of the brain. They are also classified, according to the same source, into astrocytomas, brainstem gliomas, ependymomas, mixed gliomas (or oligogastrocytomas), oligodendrogliomas and optic nerve gliomas.
Depending on the part of the central nervous system where they are found, symptoms vary, from headache and nausea to convulsions, loss of energy or numbness. Some gliomas, however, have no symptoms. To determine this, magnetic resonance imaging is used and then a biopsy will provide the definitive diagnosis.
A promising drug against glioma
With the technical name of LAM561 (2-hydroxyoleic acid) Laminar Pharma has created a synthetic derivative of oleic acid that can be taken orally and reach brain cells by crossing the blood-brain barrier. As they explain on their website “This drug changes the composition of the plasma membrane of cancer cells, reducing the signaling activity of membrane-associated proteins that are known to drive the growth of tumor. “The administration of LAM561 shows promising results in the treatment of aggressive brain tumors, glioblastomas.”
The drug LAM561 and the rare childhood disease
Recently, the US Food & Drug Administration (FDA) granted Laminar Pharma Rare Pediatric Disease (RPD) designation to the investigational drug LAM561. Targeted at patients with pediatric diffuse high-grade glioma.
the Dr. Victoria Lladó, scientific director of the US subsidiary, ‘Laminar Pharma Inc’, assures that the children who develop it “face limited treatment options and have different characteristics than adult patients.” For the doctor, this designation as RPD leads to serious manifestations and danger that the disease brings to the life of the child population that suffers from it. Therefore, he says that the appointment represents further support for Laminar Pharma’s commitment to “provide LAM561 as a potential new treatment option for our patients.”
Pediatric patients who develop it “face limited treatment options and have different characteristics than adult patients.”
Finding treatments for rare childhood illnesses
The goal of the RPD program is nothing but promoting the development of new drugs for the treatment of rare childhood diseases. So much so, that the designation is given by the FDA to those diseases that bring a risk to life, affecting less than 200,000 people in the United States and where serious manifestations occur mainly in an age group under 18 years of age.
In this scenario, Laminar Pharma will be eligible for priority review of any subsequent marketing application if, in the future, the FDA approves a new drug application for the drug the company is developing. A drug that, in the past, the FDA and the European Medicines Agency They have already granted orphan medicinal product nomenclature for the treatment of malignant glioma or called Fast-Track in 2022 to treat glioblastoma.
Clinical development of LAM561 includes two successfully completed studies to determine its safety and tolerability in adult patients. One of them in patients with advanced solid tumors and the other in newly diagnosed patients in combination with standard treatment. In addition, they have two more clinical trials currently underway. One for the treatment of newly diagnosed adult patients and in combination with European standard care; and another whose objective is to find safety, tolerability and potential clinical activity in pediatric patients with advanced solid tumors in the United States.
This information was prepared from a statement from Laminar Pharma, Johns Hopkins University and the Mayo Clinic. Please always consult your doctor.