New Targeted Cancer Therapies Prove Successful When Chemo Does Not

3-D Illustration of dabrafenib

3-D Illustration of dabrafenib (brand name: TAFINLAR). 

Being diagnosed with cancer can be a life-changing event for any person, even more so in the case of a child. Sky-high medical bills, multiple hospital visits, and aggressive therapies can take their toll. In the past, chemotherapy was automatically considered as a first tier treatment for cancer patients, but a new era of innovative therapies may soon outperform the seemingly randomized successes of chemo.

In a special report published by Reuters last month, Michele Gershberg describes the painstaking medical journey of her 4-year-old son. “It was March 2017. Over the previous year, the signs had mounted that something was wrong. First, Natan’s voice weakened. He repeatedly fell ill with lung infections. Each passing week seemed to bring a new warning. He choked when eating or drinking. His left foot dragged. He would trip and fall at play. His eyes moved rapidly from side-to-side. When we spoke to our bright boy, it was harder to connect, as if a heavy fog had settled between him and the world,” she described.

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After years of illnesses and symptoms, Natan was diagnosed with a brain tumor. “Like many people facing a crisis, we called on every resource we had at our disposal. We were fortunate to have access to excellent doctors and world-renowned hospitals close to home. The health insurance provided through my employer covered nearly all our expenses,” Gershberg informed.

“Two weeks after we received the diagnosis, Natan endured a six-hour operation at New York-Presbyterian/Weill Cornell Medical Center. Dr. Mark Souweidane, director of pediatric neurological surgery at Weill Cornell and Memorial Sloan Kettering Cancer Center, removed about 20 percent of the tumor, most of it in the cervical spine. Venturing farther into the brainstem, where the tumor was harder to distinguish from healthy tissue, would have been too dangerous.”

Though some of Natan’s symptoms were curbed from the surgery, his treatment was far from over. Dr. Matthais Karajannis, Sloan Kettering's chief of pediatric neuro-oncology, recommended that Gershberg and her husband try a drug called dabrafenib, which Novartis (NOVN.S) sells under the brand name Tafinlar.

According to the drug’s website, “TAFINLAR and MEKINIST are prescription medicines that can be used in combination to help prevent melanoma that has a certain type of abnormal “BRAF” gene from coming back after the cancer has been removed by surgery. TAFINLAR, in combination with MEKINIST, is a prescription medication used to treat a type of lung cancer called non-small cell lung cancer (NSCLC) that has spread to other parts of the body (metastatic NSCLC), and that has a certain type of abnormal “BRAF V600E” gene . . . It is not known if TAFINLAR with MEKINIST is safe and effective in children.”

Research on targeted cancer therapies such as Tafinlar reveals interesting results. Mark Kieran, MD, Ph.D., Director of Pediatric Medical Neuro-Oncology at Dana-Farber Cancer Institute, Boston was the lead investigator in a preliminary study on dabrafenib's effect on pediatric low-grade glioma.  "Response rates were encouraging. Among 29 evaluable patients, there was 1 complete response, 11 partial responses, 14 with stable disease, and 3 with progressive disease. The clinical benefit rate was 75% (responses plus stable disease)," quotes a report on the study by Alice Goodman.

After noting critical tumor shrinkage in patients throughout the study, Dr. Kieran disclosed that "we want to make the response rate with dabrafenib even higher by combining it with a MEK inhibitor since that works in adults. Adding two drugs together normally produces twice as much toxicity. But much of the toxicity from the BRAF drug is inhibited by the MEK drug, so the combination is less toxic than either drug alone, which is unusual . . . The finding that dabrafenib can shrink tumours or stop them growing is exciting and has led to trials with a MEK inhibitor and now the combination of drugs. This combined therapy may completely change the way we treat low-grade gliomas in children with this mutation. The caveat is that these targeted personalised drugs are relatively new so we need to make sure that they don’t have any long-term developmental toxicities in children.”

After making the difficult decision to attempt use of Tafinlar, the Gershbergs experienced miraculous results. "Natan’s next MRI, in October 2017, showed a result far beyond what we had allowed ourselves to hope: Nearly all his detectable tumor was gone . . . It’s been more than a year since Natan started treatment. He began to run again, and jump, and swim, and climb. He’s now in first grade, excited to get to school each day and see his friends," Gershberg expressed. 'Natan now swallows his capsule with a sip of water, flashing a thumbs-up each time. He has traveled on vacation, even overseas, without mishap. On our last trip, he picked out a new T-shirt for himself. On the front, it says: “Never give up.”'

Though not every case can divulge such a happy ending as Natan’s, innovative cancer therapies are becoming more popular by the day. With continued successes involving drugs such as Tafinlar, the future of cancer treatment may improve greatly in the coming years.

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