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FDA Signs Off on Treatment for Rare Adrenal Gland Tumors

August 6, 2018

The FDA has approved the injectable drug Azedra for rare cancers of the adrenal glands — the first ever non-surgical therapy OK’d for these tumors. Azedra (iobenguane I131) is a radiotherapy drug that attacks tumors with a high, specifically targeted dose. It’s designed to treat adults and children (12 and older) with inoperable locally advanced or metastic cancers called phenochromocytoma and paraganglioma. Pheochromocytoma forms inside and paraganglioma grows outside the adrenal gland(s). Both tumor types release hormones that can cause symptoms including high blood pressure, rapid heartbeat and anxiety. University of Pennsylvania researchers gave 68 patients in a trial at least one therapeutic dose of Azedra. The results: 25 percent who received at least one dose and 32 percent of those who received two doses saw their blood pressure drop enough to cut their hypertension meds in half. “This is a true breakthrough. Until today, there were no anti-tumor therapies available for patients with these tumors who were not candidates for surgery,” said principal investigator Daniel Pryma, an associate professor of Radiology and Radiation Oncology. “This therapy not only controls the tumors but also the debilitating symptoms caused by their excess hormone production, meaning it provides dual benefit to patients,“ added Pryma, also chief of Penn’s Nuclear Medicine and Clinical Molecular Imaging. Reported side effects include low white blood cell and platelet counts, fatigue and anemia. The FDA gave Azedra an Orphan Drug designation, Fast Track status and Breakthrough Therapy designation in the U.S. Its license is held by Progenics Pharmaceuticals.

Japan Greenlights Parkinson’s Trial Using Adult Stem Cells
In the first trial of its kind, Kyoto University scientists have won approval from Japanese regulators to test adult stem cells as a possible treatment for Parkinson’s disease. Induced pluriopotent stem cells (iPS) are derived from skin or blood cells and induced back into an embryonic-like pluripotent state that can divide into more stem cells or become any type of cell in the body, leading to a potentially unlimited source of any type of human cell needed for therapeutic purposes. They’re considered promising for regenerative research because they can become different human cells and, also, avoid controversy surrounding stem cells from human embryos. Researchers plan to transplant iPS cells into the brains of Parkinson’s patients in the hope they will help repair or replace damaged nerve cells. This is the first trial of its kind to use iPS cells. Scientists say they’re cautiously optimistic after testing the process in monkeys, who showed improvement and didn’t develop brain tumors — a much-feared potential side effect.

ALS Trial: Enrollment Complete
Researchers have attained full enrollment in a trial testing whether the anti-inflammatory drug ibudilast can help protect patients from the ravages of ALS. MediciNova wants to see if ibudilast can help prevent microglia — immune-triggering cells in the brain and spinal cord — from targeting healthy nerve cells as Amyotropic Lateral Schlerosis progresses. ALS, also known as Lou Gehrig’s disease (after the baseball legend who died from the condition), is a progressive neurodegenerative disorder. There’s no known cure and patients typically die within five years of diagnosis. Researchers plan to give the 35 ALS patients (in their ongoing 2a trial) 50 mg of ibudilast twice a day for 36 weeks. They’ll monitor participants’ physical function and scan brain activity to see if the drug helps slow disease progression. The trial is being led by Nazem Atassi, associate director of Massachusetts General Hospital’s Neurological Clinical Research Institute.

Antioxidant May Help Prevent Hearing Loss in Kids in Chemo
The antioxidant sodium thiosulfate may help prevent hearing loss in children being treated with the chemotherapy drug cisplatin, researchers have found. Drugs such as cisplatin have offered near-miraculous results — more than 80 percent of children with cancer treated with it live at least five years after their diagnoses. But 60 percent to 80 percent of those given cisplatin suffer significant hearing loss. Children with operable liver cancer are often treated with cisplatin before and after surgery. In 2015, researchers recruited 109 kids for a trial who had been diagnosed with primary liver cancer. Participants went through six chemotherapy sessions — four pre- and two post-surgery; 57 were also given sodium thiosulfate and the remaining 52 a placebo within hours of their cisplatin-chemo cocktails. More than half of the children in the placebo group suffered hearing loss compared to 29 percent of those given sodium thiosulfate, according to the study, published in the New England Journal of Medicine. The antioxidant didn’t appear to have any other serious side effects, the researchers said.

New Low Blood Sugar Marker
Researchers at Louisiana State University say they may have found a new way to determine whether someone is suffering from dangerously low blood sugar. They believe that analyzing the way brains use acetate may be an indication of hypoglycemia-associated autonomic failure (HAAF), a potentially deadly condition in diabetics whose blood sugar levels crash so often they stop experiencing symptoms. The body often looks for alternate sources of energy like acetate when it doesn’t have enough glucose or sugar. So researchers wondered if spikes in acetate levels might be triggered by or correspond with low blood sugar. In a small trial, LSU researchers had six healthy men check into the hospital for continuous glucose monitoring over four days. They then had participants fast for 72 hours — offering them only water — before rescanning their acetate levels. The results showed fasting reduced their blood-sugar levels and also increased their acetate concentrations. Those with higher baseline acetate concentrates suffered from longer periods of hypoglycemia, the team found. Researchers hope their findings will bring them closer to HAAF prevention or treatment.

Team Finds Ebola Antibodies
A team of Vanderbilt University researchers say they’ve identified two antibodies that appear to neutralize three different strains of the deadly Ebola virus. Ebola kills about half the people it infects, according to the World Health Organization (WHO). Researchers report in the July 17 issue of the journal Immunity that they isolated the antibodies EBOV-515 and EBOV-520 in blood plasma samples from 17 survivors. Researchers said the findings are a “promising” step in the search for a vaccine against the disease, which claimed at least 11,000 lives between 2014 and 2016 in West Africa. That was the largest and most complex Ebola outbreak since the virus was first discovered in 1976, according to WHO.

Lung Cancer Drug Shows Promise
Alunbrig may help extend patient life spans, according to early results from Phase III trials of the lung cancer drug. Takeda’s Alunbrig (brigatinib) is a small-molecule targeted cancer therapy designed as a tumor inhibitor. Researchers are testing the drug to see if it can improve progression-free survival in lung cancer patients better than Merck’s Keytruba (crizotnib), another targeted therapy. About half the participants in the ALTAL 1L trials were given 180 mg of Alunbrig once a day; the rest were given 250 mg of crizotnib twice daily. According to results released last week, trial participants who took Alunbrig showed “a statistically significant” improvement in progression-free survival rates. Alunbrig is awaiting FDA approval as a frontline treatment for lung cancer.

Reefer Badness: Cannabis Flunks COPD Trial
A small new trial found cannabis did not help relieve COPD symptoms. Studies have shown cannabis may help improve lung function in people with asthma. So McGill University scientists wanted to see if it could also help boost it in adults with advanced chronic obstructive pulmonary disease. To find out, they gave 35 mg of vaporized cannabis to 16 COPD patients in two separate doses, at least five days apart. The results, published in the current issue of the Annals of American Thoracic Society: Cannabis “had no clinically meaningful positive or negative effect on airway function, exertional breathlessness and exercise endurance in adults with advanced COPD.” Researchers acknowledged their sample size was small and the trial period short. But they said they’re planning larger trials, which they hope will yield more promising results.The study was prompted in part by anectotal reports from COPD patients who told researchers marijuana seemed to ease their symptoms.