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Genetic-Targeting Drugs Offer New Hope for Brain Cancer Patients

some targeted drug strategies in action

In recent years, significant breakthroughs have occurred in relation to various types of cancer treatments. Those targeting breast and lung cancers have notably made progress in reducing morbidity and mortality. Likewise, many of these new therapies are better tolerated with fewer side effects. Many of these treatments involve precision techniques or personalized medicine approaches. Some attack highly specific targets designed to eliminate inherited defects and/or their effects using targeted drug strategies. But despite these advances, progress in the field of brain cancer treatments have been more limited. Fortunately, however, recent research offers some notable hope for those suffering from these types of tumors.

someone getting targeted drug strategies in an MRI machine
Targeted drug strategies are a new, innovative weapon the battle against brain cancer.

(Multiple myeloma is toast–or may soon be. Read more in this Bold story.)

In a recent study conducted globally, a newly developed brain cancer treatment has shown significant promise. This new medication not only delays tumor recurrence after surgery but also the need for adjunctive treatments. The participants in the study all had low-grade gliomas, a common type of brain cancer with a poor long-term prognosis. Therefore, it’s understandable why there is considerable excitement surrounding the research results. Named Vorasidenib, this medication works through targeted drug strategies against a mutant protein that causes rapid tumor growth. Based on the success of this study, it’s likely this treatment will be soon approved. And it’s even more probable similar medications for a variety of illnesses will follow.

“[A low-grade gliomas is] a very difficult disease without much success. Seeing these patients do well on the trial has been really gratifying.”  – Dr. Patrick Wen, Director of the Center for Neuro-oncology at Dana-Farber Cancer Institute in Boston

An Overview of Brain Gliomas

When it comes to cancer in general, brain tumors are nowhere near the top of the list. But what they don’t have in frequency, they make up for in morbidity and mortality. Overall, there are around 25,000 people diagnosed with brain and spinal cord cancers each year. Of these, about a third are gliomas, which are the most common type of brain cancer. Some gliomas are low grade, slowly advancing over several years. Higher grade tumors like glioblastomas are much more aggressive and can cause death within months. In both cases, however, brain cancer treatment tends to be limited, often only delaying the inevitable. As a result, researchers have been busy trying to develop more targeted drug strategies in hopes of having better outcomes.

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When it comes to low-grade gliomas, the primary treatment is resection of as much of the tumor as possible. Depending on the site of the lesion, this can leave notable deficits and trigger seizures and other complications. After surgery, the site is monitored through imaging studies with most patients showing recurrence within a year or more. At that point, they receive brain cancer treatment regimens using chemotherapy, radiation therapy, or both. Naturally, these therapies do not use a targeted drug strategy and thus have many side effects. Therefore, any new intervention that delays recurrence and the use of these therapies is preferred. This is precisely what the latest research seems to support.

A doctor staring at his collection of brain pics
Targeting the genes to take out glioblastoma may prove to be a game-changer–and save a lot of lives.

Recent Research Evidence

The current study was conducted by researchers involving 77 different cancer centers in 10 different A total of 331 patients with low grade brain gliomas were enrolled, which represents a sizable number. One of the challenges in testing new brain cancer treatment drugs is the small number of patients often accessible. This explains why so many centers were utilized. Of the 331 participants, half received Vorasidenib, which is taken as a daily pill. The other half received a placebo as the control group. Notably, after two years of observation of the targeted drug strategies of Vorasidenib, it was evident that clear benefits were present. It was at that time that those taking placebo treatments were also offered the medication.

The statistical results of the study were quite impressive. In the placebo group, the average time after surgery where no tumor progression was seen was 11 months. However, in the Vorasidenib brain cancer treatment group, this figure was 28 months. In addition, those who delayed chemotherapy or radiation therapy at two years post-surgery also varied between groups. Only 27% of the placebo group made it past two years while 83% of the treatment group did. These findings indicate that the targeted drug strategies of Vorasidenib have significant benefits in deterring tumor growth and recurrence.

Using Targeted Drug Strategies

a doctor outlining a new brain cancer treatment
A new brain cancer treatment is always welcome!

Increasingly, advances in scientific and genetic research are allowing greater precision and personalized treatment approaches. In the case of many cancers, aberrant proteins created by DNA mutations play a role. If such aberrations can be identified well, then targeted drug strategies can be used to focus on the key issue. This is exactly how the brain cancer treatment Vorasidenib works. In roughly 80% of low-grade gliomas, an IDH mutated protein is present that accelerates tumor growth. However, Vorasidenib  targets this area and inhibits the protein, resulting in slower tumor growth. In theory, this explains the findings in the most recent study.

In addition to focusing on specific mutations and aberrations, these targeted drug strategies have other benefits as well. Because they are more focused, many have fewer side effects and adverse reactions. Other brain cancer treatment options like chemotherapy and radiation therapy are less precise and commonly cause other symptoms. But targeted therapies tend to have smaller side effect profile. In fact, Vorasidenib only was noted to cause elevated liver enzymes in some patients. In terms of brain cancer treatment regimens, this is much more favorable when compared to traditional protocols.

Future Research for Glioma Treatment

Based on the study’s findings, Servier Pharmaceuticals hopes to gain FDA approval for Vorasidenib use sometime in 2024. At the same time, the company is also planning to move forward with additional trials involving Vorasidenib. These include trials that combine its targeted drug strategies as an adjunct to chemotherapy or radiation therapy. They also plan to combine it with immunotherapy as a brain cancer treatment. While the outcomes of such an approach are not known, the current study supports further exploration in these directions. At a minimum, such treatments could significantly extend quality and quantity of life in these patients. And hopefully, such a combination could provide additive benefits that may lead to longer periods of remission.


Targeted medicine and treatments are the future–read all about in this Bold story.

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