PROJECTS
Ultra-rare WOREE syndrome responds to “Extinct” Generic Drug Amlexanox©
An Australian parent whose daughter was diagnosed with WOREE syndrome, posited that a readthrough therapy for the nonsense mutation might treat her symptoms, and explored many agents for treating PTC. He eventually discovered Amlexanox©, which was preclinically shown to have readthrough effect and inhibit nonsense mediated decay and convinced his daughter’s neurologist to prescribe the drug in an N=1 experiment, as it was approved for chronic use in adults and children. The immediate effect was a remarkable improvement in leg movement, vocalizing and interacting, which had been absent prior to the drug treatment. Improvement lasted four months until they ran out of drug, as it was no longer marketed. After treatment stopped the patient regressed and returned to baseline.Host-Directed Therapies for Tuberculosis to Address Post TB Lung Sequelae
Tuberculosis (TB) remains a leading cause of global morbidity and mortality. Current treatments are complex, often poorly tolerated, and leave 30-40% of patients with permanent lung damage, notably decreased FEV1% (forced expiratory volume in one second), which is linked to increased long-term mortality risks, especially in low-income countries. Addressing post-TB lung disease is a global priority.
TB and its long-term lung consequences pose significant financial challenges, with projected global TB-related costs of $17.5 trillion and annual productivity losses in India reaching $340 million. Addressing these issues can alleviate substantial economic burdens.
To evaluate the efficacy, safety, and tolerability of Metformin and N-Acetyl Cysteine (NAC) alongside standard TB treatment (HREZ) in promoting lung function recovery post-TB, we are planning to conduct a randomized, open-label, multi-center phase 3 trial.
Metformin, a widely used diabetes medication, enhances TB intracellular killing by modulating AMPK, ROS, and autophagy whereas NAC, an amino acid derivative with mucolytic and hepatoprotective properties, has been shown to improve lung function impaired by TB. Studies suggest small reductions in FEV1 correlate with higher mortality risks, highlighting the need for trials like this.
By integrating Metformin and NAC into TB treatment, this study aims to improve post-TB lung function and reduce the socioeconomic impacts of the disease.
The trial will be sponsored by the Open Source Pharma Foundation (OSPF) and conducted at ICMR-National Institute for Research in Tuberculosis. Collaborators include NewFound, the Global Alliance for TB, Aurum Institute (South Africa), the Government of Brazil’s FioCruz, and A*STAR (Singapore), with potential additional global sites.
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