The immune cells of the brain could treat neurodegenerative diseases

Human behavior relies on the stimulation and inhibition of neurons, and scientists have long considered that neurons are mainly responsible for maintaining this circuit of activity in balance. However, now, a research team led by the Icahn School of Medicine at Mount Sinai has found that the brain’s immune cells also play a vital role in regulating behavior.

These immune cells, known as microglia, are scavengers that eliminate dying cells. In a new study published in Nature, the team showed similar to inhibitory neurons; microglia can also sense and decrease excessive neuronal activation. The results could have implications for treating behavioral problems associated with neurodegenerative diseases, as the researchers argued.

The study’s senior author Schaefer and colleagues lowered the microglia population in mice by using drugs to block the protein receptor CSF1R. Doing so made the animals supersensitive to neuro stimulants. When they gave neuro stimulants to the altered mice, 90% of the animals developed long-lasting epileptic seizures, indicating overexcited neurons. Only 11% of the control animals have experienced seizures.

The researchers moreover recognized the process behind neuron-microglia communication. When neurons are active, they release ATP, which proffers the energy to living cells for performing activities. Microglia can find out and respond to ATP released from the synaptic junctions between neurons.

Moderna’s COVID-19 vaccine induces an immune response in older adults

Moderna has published data from adults aged 56 years and older who received its COVID-19 vaccine. The results from the small cohort hint the vaccine triggers comparable immune responses in younger and older adults.

The investigators running the phase 1 trial of mRNA vaccine mRNA-1273 described the study’s expansion to include 40 older adults, as written in The New England Journal of Medicine. Age is associated with worse results in COVID-19 patients, but vaccines can flunk to confer immunity to older people, raising eyebrows that prophylactics will fail the demographic that most needs protecting.

The expanded trial enrolled 20 adults aged 56 to 70 years and 20 adults aged 71 years and older. Ten subjects from each age group received a 25-μg dose, and 10 received the 100-μg dose Moderna took into phase 3.

After the second dose, levels of binding antibodies in the participants entered the upper quarter of the responses seen in former COVID-19 patients who donated plasma. The link between antibody levels and immunity remains vague, and the small size of the trial makes it difficult to draw firm conclusions. However, the increase in antibody levels is an encouraging early sign of effectiveness.

Pfizer funnels USD 200 Million into CStone

CStone Pharmaceuticals has nabbed a USD 200 million investment from U.S. Big Pharma Pfizer and received a China license for its PD-L1 asset sugemalimab.

The company is working in five areas of interest: oncology, cardiovascular diseases, rheumatoid arthritis, hematology, and autoimmune diseases, focusing on immuno-oncology. Since its inception, CStone has worked on a pipeline of more than a dozen candidates, including treatments targeting PD-L1, IDH1, and CTLA-4.

Two years later, the Suzhou, China-based biotech, raised another USD 260 million in its B round, which helped progress its clinical-stage assets, including lead program CS1001, aka sugemalimab, an anti-PD-L1 antibody. The proceeds were also tapped for extending CStone’s pipeline both organically through in-house R&D and partnerships.

The relugolix of Myovant falls short in metastatic prostate cancer with FDA verdict

Myovant’s prostate cancer drug outshone the standard of care at keeping patients’ testosterone levels down, a phase 3 displaying that teed up an FDA filing. As the agency contemplates the case, the company reveals the data for patients with metastatic disease, and they are not so good.

The company pitted the drug, relugolix, against the standard of care, leuprolide, in 934 patients with advanced prostate cancer. Nearly all patients taking relugolix (97%) kept their testosterone levels down over 48 weeks, compared with 89% of patients taking leuprolide reported by Myovant. However, the drug staved off cancer progression at about the same rate as the incumbent in a subgroup of patients whose cancer had spread beyond the prostate, as the new data show.

After 48 months, 74% of metastatic patients on relugolix were castration resistance-free, compared with 75% of leuprolide patients. Castration-resistant prostate cancer keeps growing even when testosterone in the patient’s body is at deficient levels. According to the National Cancer Institute, many early-stage prostate cancers need testosterone to grow, but castration-resistant cancers do not.

Despite the disappointing showing in patients with metastatic disease, Myovant hopes that the relugolix could still unseat leuprolide in the broader population.

Alnylam phase 3 links lumasiran to clinically significant improvement in infants

A phase 3 trial has linked the lumasiran of Alnylam to a clinically significant decline in a substance that drives negative results in children with rare kidney disease. The top-line readout comes before the FDA is due to rule on whether to nod the RNAi therapeutic in older patients.

Lumasiran aims glycolate oxidase to counter the over-production of oxalate, leading to kidney damage in patients with primary hyperoxaluria type 1 (PH1). Alnylam displayed data from a phase 3 trial of the drug in PH1 patients aged six years and older late last year, setting it up to secure approval in the U.S. population by the end of the year,2020.

In parallel, Alnylam has worked to display whether lumasiran works in PH1 patients aged under six years. Alnylam showed top-line findings from the phase 3 trial in infants and young children.

The investigators enrolled 18 subjects aged 3 to 72 months and gave them subcutaneous injections of lumasiran. After six months, investigators evaluated whether lumasiran had driven a reduction in urinary oxalate excretion from baseline.