Breast Cancer Trial Resumes

Mersana says it has worked out a new agreement with the FDA to resume recruiting patients for a clinical trial of its breast cancer drug months after one of the participants died.

Under the altered trial design, Mersana will now exclude patients who show signs of liver damage. Researchers will also offer a once-every-four-weeks dose to compare safety and efficacy.

The Phase I trial, which began in November 2016 and is scheduled to run through November 2019, had hoped to enroll 120 patients with advanced breast cancer and offer them IV infusions of the experimental drug XMT-1522 once every three weeks.

But in July, Mersana announced that it was putting a partial hold on the trial after one of the patients in it died and researchers couldn’t rule out XMT-1522 as a contributing factor.

At the time, the company agreed to temporarily halt recruiting new patients, but kept those already enrolled in the trial on their current dose levels.

COPD Therapy has Early Success

A new treatment significantly reduced symptoms in clinical trial participants with chronic obstructive pulmonary disease (COPD).

The AIRFLOW 2 Phase II clinical trials are testing whether (Minneapolis-based) Nuvaira’s targeted lung denervation (TLD) system can help improve breathing in COPD patients by sending an electrical charge through a catheter to the nerves outside their damaged airways to relax and widen them.

Researchers are trying the device on 82 patients at six clinical trial sites in Europe. Half of them received the treatment and the rest received a “sham” procedure in which a catheter was inserted but no radiofrequency sent. Participants didn’t know whether they were receiving the real or fake therapy.

Six months later, 71 percent of the patients in the sham group suffered a serious COPD flare, compared to only 32 percent of those who received the treatment.

The findings were presented last week at the European Respiratory Society International Congress in Paris.

Researchers say they’re planning to test the treatment in a much larger Phase III trial next year.

More than 16 million Americans have been diagnosed with COPD but it’s estimated millions more may suffer from the disease and not know it.

$3.5M for Yellow Fever Trials

The federal government has given another $3.5 million to help BioCryst conduct clinical trials of a potential treatment for yellow fever.

Galidesivir is a broad-spectrum antiviral drug being developed for treatment of the Marburg and Ebola viruses — and the company hopes it will also be effective treating yellow fever, a disease the World Health Organization reports kills an estimated 60,000 people a year.

A two-year Phase I randomized clinical trial, which included 94 healthy volunteers, found that galidesivir was safe and well-tolerated.

Most of the development funding has come from the National Institute of Allergy and Infectious Diseases and the Biomedical Advanced Research and Development Authority at Health and Human Services.

The new infusion of funds increases the research pot to more than $43 million.

New UK Virtual Cancer Center

Five British research institutes are pooling their resources to create a virtual medical center thanks to an $18.4 million grant from the nonprofit group Cancer Research UK.

The new Cancer Research UK City of London Centre will be staffed by researchers from University College London, King’s College London, Queen Mary University of London and the Francis Crick Institute.

Organizers say the goal is to provide some 14 million people in and around London with access to clinical trials and cutting-edge advanced cancer treatments such as CAR-T cell therapy.

Marijuana OK’d for Clinical Trial

The Drug Enforcement Agency (DEA) has agreed to allow researchers at the University of California, San Diego, to import Canadian marijuana for a clinical trial.

In a rare move, the DEA signed a waiver permitting the university’s Center for Medicinal Cannabis Research to import a cannaboid capsule manufactured by British Columbia-based Tilray Inc. for a Phase I/II trial involving patients suffering from Essential Tremor. ET is a neurological disorder that affects an estimated 7 million Americans.

Canada recently legalized marijuana, but federal law here still bans it. Tilray’s capsules contain cannabidiol and THC, both extracted from the marijuana plant.

Researchers plan to enroll at least 16 ET patients in the trial set to begin early next year. Half of the participants will be put on a dose escalation schedule for a year.

Researchers Identify Target for Alzheimer’s Clinical Trials

Researchers at Kings College London say they may have found a new target for Alzheimer’s clinical trials: a protein called Dkk1, which they believe could be the key to a dementia “feedback loop” that has vexed drug development for decades.

Scientists have long suspected that Alzheimer’s and other dementias are related to the buildup of beta-amyloids, which attack synapses connecting brain nerve cells.

The team says it has determined that once nerve cells are destroyed, they create even more beta-amyloids, leading to a vicious cycle. The beta-amyloids are provoked by Dkk1, which tends to accumulate as humans age.

The findings, published last week in the journal Transitional Psychiatry, suggest that Alzheimer’s clinical trials have failed so far because they focus on the beta-amyloids rather than on their underlying cause.

“We think that once this feedback loop gets out of control, it is too late for drugs which target beta-amyloid to be effective,” lead researcher Richard Killick said.

Researchers conducted their Dkk1 experiments on mice, but are now seeking funding for clinical trials to determine whether an experimental drug called fasudil — already approved as a human treatment for strokes in China and Japan and showing promising results for reducing beta-amyloids in animals — could be an effective treatment for early-stage dementia.

CHMP Recommends First Treatment for Rare Eye Disease after Successful Clinical Trials

The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization in the EU for Spark Therapeutics’ Luxturna (voretigene neparvovec) gene therapy for treatment of inherited retinal dystrophy caused by RPE65 gene mutations.

The treatment – the first to be recommended for the rare genetic disease – is intended for patients who have inherited the mutation from both parents and who have enough viable retinal cells.

CHMP’s decision was based on an assessment by the EMA’s Committee for Advanced Therapies. The main clinical trial supporting approval showed a significant improvement of patient night vision after one year, while no improvement was seen in the trial’s control group. Impaired night vision is a typical symptom of the disease.

“Given the novelty of the treatment and the limited number of treated patients, the CHMP requires the company to ensure the long-term follow-up of patients to confirm Luxturna’s continuing efficacy and safety,” the committee said.

Spark agreed to conduct follow-up studies, including a post-authorization safety study based on a disease registry in patients who have experienced vision loss due to the disease – which causes a progressive loss of vision that often ends with blindness. Most young patients lose their sight completely before they become adults and there is currently no approved treatment.

“We are encouraged by today’s decision, which further affirms our pioneering clinical program and brings Luxturna one step closer to patients with inherited retinal disease (IRD) caused by mutations in both copies of the RPE65 gene in the European Union,” Spark Therapeutics said.