Alpha-1 Antitrypsin Deficiency — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Second-Generation Antipsychotics for Depression in Serious Illness: A First-Line Augmentation Strategy.
Robbins-Welty Gregg et al. — Journal of pain and symptom management (21 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41871641/
- 2.
The NAD-brain pharmacokinetic study of NAD augmentation in blood and brain using oral precursor supplementation.
Berven Haakon et al. — iScience (20 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41858901/
- 3.
Regenerative Nodule as a Diagnostic Dilemma in the Neonate With Acute Liver Failure and a Focal Liver Lesion.
Castaneda Claire et al. — Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society (19 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41858078/
- 4.
[Sequential surgery following conversion therapy based on the combination of immune checkpoint inhibitors and antiangiogenic targeted drugs alters the treatment landscape and outcomes for advanced hepatocellular carcinoma].
Tang H W et al. — Zhonghua yi xue za zhi (17 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41820050/
- 5.
Original research: Amplification of genetic and metabolic factors in alpha-1 antitrypsin deficiency.
Schrader Christina et al. — Hepatology (Baltimore, Md.) (11 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41812027/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Recruiting — Phase 1 — Wave Life Sciences Ltd.
https://clinicaltrials.gov/study/NCT06405633
- 2.
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
Recruiting — Phase 3 — Takeda
https://clinicaltrials.gov/study/NCT05677971
- 3.
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Recruiting — Phase 1 — Beam Therapeutics Inc.
https://clinicaltrials.gov/study/NCT06389877
- 4.
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Recruiting — Phase 1 — AIRNA Corporation
https://clinicaltrials.gov/study/NCT07431112
- 5.
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Recruiting — Phase 2 — Sanofi
https://clinicaltrials.gov/study/NCT05897424
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency is an inherited condition where the body does not produce enough of a protective protein for the lungs, leading to early-onset emphysema and liver disease. It is significantly underdiagnosed, often mistaken for COPD or asthma. Gene and RNA editing therapies in clinical trials offer hope for a future cure.
Most Recent Research
Depression in serious illness is common, disabling, and often requires rapid improvement. Traditional antidepressants may take weeks to work, whereas second-generation antipsychotics (SGAs) have evidence for faster onset and robust augmentation effects in general psychiatric populations. In this Palliative Care Rounds, we review the general psychiatric and serious illness-specific evidence for the use of SGAs as monotherapy and augmentation therapy for depression. In the psychiatric literature, SGA augmentation improves response and remission rates (ORs 1.34-2.93; NNT 7-13), with onset of improvement within 1-2 weeks. Monotherapy is less well tolerated and not guideline-recommended. No RCTs have evaluated SGAs specifically for depression in serious illness, but numerous cancer trials support their safety for nausea, appetite, and other symptoms. Despite the absence of serious illness-specific psychiatric trials, SGAs have the strongest evidence base among augmentation options and may offer meaningful benefits when prognosis or symptom severity necessitates rapid improvement. Low-dose augmentation should be considered early, rather than only after multiple failed antidepressants, particularly when SGAs can also target co-occurring physical symptoms relevant to palliative care.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.