A breakthrough treatment has given a family of a teenager diagnosed with a rare brain tumor much to be grateful for. Lucas was six years old when he was diagnosed with an aggressive brain tumor with a 10-percent chance of survival. Most children diagnosed live less than a year after their diagnosis. However, Lucas is defying the odds thanks to treatment in a clinical trial that offered him hope.
Lucas was diagnosed with diffuse intrinsic pontine glioma (DIPG). This aggressive form of cancer occurs in children and forms in the brainstem. Most patients have a “less than 10%” chance of surviving beyond two years after diagnosis, the National Cancer Institute (NCI) says.
DIPG is primarily treated with radiation therapy, which only slows the tumor’s progression. “Surgery is not possible due to the invasive nature of the disease in a critical area of the brain; only radiotherapy has shown a transient palliative effect on disease progression, and no adjuvant therapy has proven to increase disease control,” the Journal of Clinical Oncology says. Lucas’ doctor, Jacques Grill, is the head of the brain tumor program at the Gustave Roussy Cancer Center in Paris. At the time of initial treatment, Grill says he gave Lucas’ parents a worrisome message.
“I told his parents that we would try to contain the disease for as long as possible (via radiation therapy) but that we would not be able to cure him,” Grill explained to French news outlet “20 Minutes.”
Then, Lucas found hope in a clinical trial called the BIOMEDE trial for diffusing intrinsic pontine glioma.
The Clinical Trial that Gave Lucas and Others Hope
Clinical trials help doctors better understand cancer and discover more effective treatment methods. They also allow patients to try a treatment before it’s approved by a regulating authority such as the U.S. Food and Drug Administration (FDA) in the United States.
BIOMEDE trial tests new drug treatments for DIPG. Lucas was given everolimus, which is a targeted therapy cancer drug. Targeted therapy drugs target proteins that control how cancer cells grow. Everolimus may appear under the brand names Afinitor, Votubia, and Zortress in the United States. The FDA has approved it for some breast, pancreatic, gastrointestinal, lung, and kidney cancers.
Lucas responded well to the treatment.
“Over a series of MRI scans, I watched as the tumor completely disappeared,” Grill told Agence France-Presse.
Doctors believe due to Lucas’ tumor mutation or “biological particularities,” the drug treatment responded well.
“Lucas beat all the odds,” Grill said as he’s now in remission. Seven other children who participated in the clinical trial also survived well beyond the typical life expectancy for DIPG. Still, Lucas was the only one whose tumor no longer appeared in scans.
Researchers continue to study why Lucas’ tumor type responded so well to the treatment.
Expert Resources on Clinical Trials
- Clinical Trials Can be Life-Saving for Some
- Clinical Trials Need More Minority Patients
- Clinical Trials Test State-of-the-Art Cancer Treatments
- Clinical Trials Using CAR-T Cells Are Extremely Promising
- Clinical Trials and What Statistics Really Mean
Why Clinical Trials Offer Hope
Within the U.S., all new drugs must go through clinical trials before the FDA approves them. Although the rewards of clinical trials can be great, they also come with risks. Talking to your doctor about this before enrolling in a trial is important. Some risks to consider include:
- The risk of harm and/or side effects due to experimental treatments
- Researchers may be unaware of some potential side effects of experimental treatments
- The treatment may not work for you, even if it has worked for others
Dr. Beth Karlanis a gynecologic oncologist at UCLA Health. She says the goal with clinical trials is to advance cancer research to a point where the disease becomes akin to diabetes, where it becomes a manageable condition.
“Clinical trials hopefully can benefit you, but they also provide vital information to the whole scientific community about the effectiveness of these treatments,” Dr. Karlan said.
“They can be life-saving. We’ve seen many in the last few years of children and adults who have participated in trials and have had miraculous results,” Dr. Karlan continued.
WATCH: Clinical trials can be life-saving.
Before you enroll in a trial, you must be allowed to read the consent documents thoroughly and to ask any questions you may have. The documents will likely contain the following:
- The purpose of the research
- Any risks and benefits expected from the research
- Information about procedures that may cause discomfort (like frequent blood tests)
- Any alternative procedures the patient might consider instead
- How the patient’s information will be kept private
- How long the study is expected to take
- A form confirming you are participating in research voluntarily
- Whether any compensation or additional medical care is available if some sort of injury occurs
- The patient’s rights (like the right to stop research in the middle of the trial)
- Contacts for any patient questions
Patients are allowed to walk away at any time during the trial. Understanding your rights as a voluntary patient is important before you participate in a clinical trial, and understanding that the treatment may not work is also crucial.
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Kavontae Smalls is a writer and reporter for SurvivorNet. Read More
