News & numbers
“This acquisition represents a unique opportunity to bring innovative science and leading experts in T-cell receptor biology and cell therapy manufacturing together
with our internal oncology cell therapy team, unlocking new ways to target cancer.” Susan Galbraith, executive vice-president, AstraZeneca oncology R&D on the acquisition of Neogene.
Hope with sleeping sickness drug
A single-dose oral treatment has raised hope for the elimination of sleeping sickness in Africa after positive results were seen from a phase II/III study. The Drugs for Neglected Diseases initiative (DNDi) and Sanofi announced treatment success rates of up to 95% from the study investigating the safety and efficacy of single dose acoziborole, a potentially transformative investigational treatment for sleeping sickness. Transmitted by the bite of an infected Tsetse fly, Gambiense Human African Trypanosomiasis (g-HAT), known more commonly as sleeping sickness, is fatal without treatment. In the early stage of the disease, people suffer from headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing neuropsychiatric symptoms such as sleep disruption, confusion, lethargy, convulsions – and ultimately, death.
“Sleeping sickness is a nightmare disease that affects patients in some of the most remote settings in West and Central Africa,” said Dr Victor Kande, former neglected tropical diseases expert advisor at the Ministry of Health of the Democratic Republic of the Congo, principal investigator of the trial and lead author on the study. “We are now on the cusp of a potential treatment that can be given in one day.” The number of reported cases of sleeping sickness has fallen in the past 20 years, from almost 40,000 reported cases in 1998 (with estimates of over 300,000 undiagnosed cases) to less than 1,000 in 2020. Though an encouraging trend, the researchers noted that it should not be a reason for complacency.
“By simplifying the treatment paradigm, acoziborole would be an innovation that enables a sustainable response to sleeping sickness for health systems,” said Dr Antoine Tarral, head of the sleeping sickness programme at DNDi and co-author of the study. “With these new data, we have hope that we may be able to finally eliminate the disease, once and for all, by opening the door to a ‘screen-and-treat' approach at the village level.”
Acoziborole is under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
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Media coverage slows take- up of aducanumab trial
The drug aducanumab received accelerated approval as a treatment for Alzheimer’s disease from the US Food and Drug Administration (FDA). It is the first FDA-approved therapy to address the underlying biology of Alzheimer’s disease. It is the first therapy to demonstrate that removing beta-amyloid, one of the hallmarks of Alzheimer’s disease, from the brain is reasonably likely to reduce cognitive and functional decline in people living with early Alzheimer’s. However, a new survey has found news coverage of the FDA’s decision to approve controversial Alzheimer’s disease drug aducanumab made the public less willing to volunteer for clinical trials on drugs with the same target. The drug was approved in June 2021 despite experts advising against it because its ability to decrease plaques hadn’t shown an impact on the disease’s clinical progression. The agency’s controversial go-ahead and further disaccord over the drug’s labelling and price captured widespread media attention and researchers from the University of California, Irvine, Institute for Memory Impairments and Neurological Disorders (UCI MIND) designed a survey to evaluate the impact of this coverage
The team performed the survey in tandem with the FDA’s spring 2021 consideration of aducanumab among people aged 50–79 who had expressed willingness to take part in drug research. Two weeks before the FDA’s decision, UCI MIND asked respondents if they would be interested in enrolling in a hypothetical four-year study of a plaque- reducing monoclonal antibody and a plaque-preventing drug known as a BACE inhibitor. Eight days after the FDA gave aducanumab the green light, UCI MIND sent survey participants a similar questionnaire with a new
section about the monoclonal antibody and its approval. “We found those who had heard about the FDA decision before our follow up became less willing to take part in a drug trial,” said neurobiology and behaviour graduate student Marina Ritchie, first and corresponding author of the paper. “The people who learned about it from our materials demonstrated absolutely no change in their willingness.” UCI MIND director, Professor Joshua Grill added: “This is surprising because it goes against some of our previous data showing people are generally more willing to take part in studies involving approved drugs compared to investigational ones. We believe it could be evidence of the powerful influence of media coverage of science.”
The researchers believe the survey’s findings may offer important insights for Alzheimer’s disease researchers. “Alzheimer’s is the most important medical condition society faces and we need an army of citizen volunteers to participate in drug trials,” Professor Grill said. “Anything that diminishes credibility in scientific research impedes our progress. Media coverage has the potential to influence people’s choices. That can hold us back or push us forward.”
Funding for the project was provided by the UCI Alzheimer’s Disease Research Center.
1in9
Americans aged 65 or older have Alzheimer’s disease, or 10.7% of the population . Cohort
Clinical Trials Insight /
www.worldpharmaceuticals.net
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