Recent regulatory actions and clinical studies highlight major shifts in neurology and child health, led by the FDA’s approval of Lantheus’s tau PET imaging agent Tauklarify for Alzheimer’s disease and the NIH appointment of pediatric neurologist John Gaitanis, MD. According to recent reports from Science, JAMA Network Open, and the FDA, these developments coincide with new safety trials for gene therapies and insights into long-term neurological risks.
Leadership Changes and Regulatory Approvals in Pediatric and Adult Neurology
The National Institutes of Health named John Gaitanis, MD, a pediatric neurologist with controversial views on autism, to lead the agency’s child health institute, according to reporting by Science.
Concurrently, the FDA approved florquinitau F 18 injection (marketed as Tauklarify), a new tau PET imaging agent designed for Alzheimer’s disease, according to an announcement by Lantheus. This diagnostic approval expands the toolkit available for clinicians evaluating cognitive impairment.
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Clinical Trials Face Regulatory Hurdles in Rare Disease Research
While diagnostic tools advance, therapeutic pipelines face strict regulatory oversight. The FDA placed a clinical hold on a trial investigating the gene therapy clemidsogene lanparvovec (RGX-121) for mucopolysaccharidosis (MPS) type II, also known as Hunter syndrome, according to a statement from Regenxbio. The agency enacted the pause after asymptomatic small masses were identified on the spines of five patients.
Despite setbacks in clinical gene therapy trials, computational methods offer new avenues for discovery. Drug screening and machine learning successfully predicted neuroprotective agents in a preclinical model of MPS type IIIA, or Sanfilippo syndrome, a form of childhood dementia, as detailed in Nature Communications.
Long-Term Risks and Diagnostic Discoveries in Stroke and Dementia
New data from JAMA Neurology reveals that young survivors of aneurysmal subarachnoid hemorrhage face substantial lifelong risks of developing new aneurysms. A prospective study showed no plateau in risk over four decades of follow-up.
In the Alzheimer’s treatment space, Eisai announced that the once-weekly lecanemab subcutaneous injection (Leqembi Iqlik) is now available as initiation therapy for adults with mild cognitive impairment or mild dementia due to Alzheimer’s. This follows the July approval of the at-home starter dose.
Other recent findings address maternal and paternal health factors in developmental disorders:
- Valproate Exposure: Data from Taiwan published in Neurology showed that fathers who used the antiseizure drug valproate before conceiving were no more likely to have children with autism or attention deficit-hyperactivity disorder than other fathers.
- Brain Volume Trajectory: Age at menopause was associated with brain volume trajectory in a cohort study of older women published in JAMA Network Open.
- Tuberous Sclerosis: A longitudinal study published in Epilepsia suggested distinct developmental pathways to autism in children with tuberous sclerosis complex.
- Inflight Seizure Management: Experts arguing in JAMA Neurology maintained that benzodiazepines are essential for managing inflight seizures and should be standard in airline emergency medical kits.
Frequently Asked Questions
What is Tauklarify used for?
According to Lantheus and FDA announcements, Tauklarify (florquinitau F 18 injection) is a tau PET imaging agent approved to assist in the evaluation of Alzheimer’s disease.
Why was the RGX-121 gene therapy trial placed on clinical hold?
According to Regenxbio, the FDA placed a clinical hold on the clemidsogene lanparvovec trial for Hunter syndrome after asymptomatic small masses were found on the spines of five patients.
Does paternal valproate use increase autism risk?
Data from Taiwan published in Neurology indicated that fathers taking the antiseizure drug valproate prior to conception showed no increased likelihood of having children with autism or ADHD.
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