News & numbers "This is a moral indictment of the state of our world. It is an obscenity.
We passed the science test. But we are getting an F in Ethics." Antonio Guterres, UN secretary-general
Targeting amyloid in Alzheimer’s patients
Eli Lilly is the latest drug maker to seek approval for an Alzheimer’s treatment that targets amyloid plaques in the brain. It announced its intention to seek the same accelerated approval given to aducanumab earlier this year in its third-quarter earnings report published in October. The FDA shocked the pharmaceutical and medical communities when it approved the drug in June, making it the first Alzheimer’s pharmaceutical to be approved since 2003. Importantly, the approval, made on the condition that the drug’s maker Biogen conducts a phase 4 trial to prove clinical benefit, also leant regulatory credibility to the theory that reducing amyloid is an effective way to treat cognitive decline. Eli Lilly is seeking the same pathway to market for its own drug Donanemab, which has amyloid as its therapeutic target. The
company also stated plans in its earnings report to run a study pitting the treatment against Biogen’s aducanumab. There’s been no shortage of scepticism from the medical community since its approval, and that’s evident from the fact Biogen made $1.6m in the first few weeks after its US launch – around half the amount expected. Critics also centred their arguments on the fact amyloid-related imaging abnormalities have been observed at high doses of the drug, the main being swelling inside the brain. Several medical experts, like UCL Professor of Old Age Psychiatry Robert Howard, have been outspoken in their public criticism of the FDA decision, insisting Biogen’s submission for approval in EU countries should be rejected. “The FDA’s accelerated approval of aducanumab, solely on the grounds that it was reasonable to
expect that reduction in amyloid would lead to improvement in the course of Alzheimer’s disease, despite all the evidence indicating no meaningful correlation between amyloid reduction and symptom improvement, has been highly controversial and has called into question the impartiality of the FDA and its Staff,” he said.
“I would anticipate that EMA (European Medicines Agency), when they meet in December, will not grant a license to aducanumab. Aducanumab is a treatment without convincing efficacy, with serious associated adverse effects and a high financial cost. On the basis of the available evidence and in the best interests of people with Alzheimer’s disease, their families and those who care for them, EMA and MHRA should not approve a license for aducanumab.”
ALS therapy should target the brain $2,100
The brain could be a viable target for medical treatments given to patients with amyotrophic lateral sclerosis (ALS), according to scientists at Northwestern University, Illinois.
The current understanding of the disease’s pathophysiology is that the degeneration of brain motor neurons is a by-product of spinal motor neuron degeneration. But Northwestern researchers believe a recent study they carried out reveals brain motor neurons don’t degenerate as a consequence of spinal motor neuron degeneration, but independently of it. “We have discovered that the brain degenerates early in diseases like ALS, sends us warning signals and shows defects very early in the disease,” said lead study author Hande Ozdinler, an associate professor of neurology at Northwestern University Feinberg School of Medicine. “Therefore, we need to repair the brain motor neurons if we want long-term and effective treatment strategies. The brain is important in ALS.” The researchers also believe their study is the first to show that the gene UCHL1 is
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important for maintaining the health of brain motor neurons that are diseased due to two independent underlying causes: The accumulation of badly folded proteins and the accumulation of sticky protein clumps inside the cells. These issues are observed in more than 90% of all ALS cases and in other cases of upper motor neuron diseases. “Our findings not only give legitimacy for targeting brain motor neuron health in ALS as a therapeutic intervention, it also reveals the first target gene that can help these neurons be revitalised,” Ozdinler said. This study, published in the journal Gene Therapy, reveals the importance and significance of treating upper motor neurons in ALS and identifies the first genetic target. The researchers said the next step is to determine the best dose and the best site of injection with respect to improvement of movement and reduction of disease conditions in at least two different ALS disease models. After preclinical toxicology studies, scientists will move to translate these results into a clinical trial, a process that likely will take several years.
US bets on Thor’s hammer to
smash Covid-19 An FDA advisory panel has voted 13 to ten in favour of the regulator granting emergency authorisation to Merck’s molnupiravir – a Covid-19 antiviral named after Thor’s hammer Mjölnir. The US government has already pre-ordered 3.1 million courses of the drug in anticipation of its approval and based on data that showed a 30% reduction in hospital admissions. The country has already approved three monoclonal antibody (mAb) treatments that demonstrated more than double this reduction, but Merck’s drug comes in at a third of the price of the most used mAb REGEN-COV and can be taken at home as a course of pills.
Clinical Trials Insight /
www.worldpharmaceuticals.net
Cost per dose in the US of REGEN- COV, the most commonly used monoclonal antibody for Covid-19. MSF Access Campaign
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