Zwelethu (Zweli) Bashman, has been appointed chairman of the Pharmaceutical Industry Association (PTG), and Dr Stavros Nicolaou as deputy chairman. Four pharmaceutical associations, representing more than 80% of the industry, comprise the membership of the PTG.
Bashman is president of the Innovative Pharmaceutical Association South Africa (IPASA), and managing director of MSD South Africa and sub-Saharan Africa.
“Our goal is to contribute towards an environment that promotes growth and investment in the South African pharmaceutical industry while aspiring to broaden access to medicines for all people living in South Africa,” said Bashman.
Stavros Nicolaou has assumed the role of deputy chairman after serving as chairman of the PTG for several years.
Promising trial results indicate that a new type of cell therapy could improve the prognosis of those who are critically ill with acute respiratory distress syndrome (ARDS) resulting from severe COVID.
Published in the journal Nature Communications, Professor Justin Stebbing of Anglia Ruskin University (ARU) is the joint senior author of the new study investigating the use of agenT-797, MiNK Therapeutic’s allogeneic, unmodified invariant natural killer T (iNKT) cell therapy.
The iNKT cell therapy has the effect of rescuing exhausted T cells and prompting an anti-inflammatory cytokine response, potentially activating anti-viral immunity to help these patients fight infection as well as to reduce severe, pathogenic inflammation of the lung.
The new research was carried out at three medical centres and found that agenT-797, which is also under investigation in cancer trials, could be manufactured rapidly, had a tolerable safety profile, and appeared to have a positive effect on mortality among critically unwell Covid-19 ARDS patients receiving intensive care.
The exploratory trial included 20 mechanically ventilated patients with severe ARDS secondary to Covid-19. Of the 20 patients in the trial, 14 survived (70%) at 30 days (compared to a control group of 10%), and there was an 80% lower occurrence of bacterial pneumonia amongst those who received the highest dosage of agenT-797, compared to those who received fewer cells.
Twenty-one patients were treated overall (the main trial, plus one under compassionate use), which included five who were also receiving veno-venous extracorporeal membrane oxygenation (VV-ECMO), known as ‘the most aggressive salvage therapy’ for critically ill patients with ARDS.
In VV-ECMO, deoxygenated blood is pumped through a membrane lung and returned to the body via a cannula. This trial is believed to be the first immune cell therapy of any type to be used in critically unwell patients undergoing VV-ECMO.
Survival of the VV-ECMO cohort was 80% after 30 and 90 days, and 60% after 120 days. This compares favourably to overall survival of 51% for patients with Covid-19 who were treated with just VV-ECMO at the same institution, during the same timeframe.
Joint senior author Justin Stebbing, Professor of Biomedical Sciences at Anglia Ruskin University (ARU) in Cambridge, England, said: “During this small, exploratory study we observed that MiNK’s iNKT cell treatment, which is also being advanced for people with cancer, triggered an anti-inflammatory response in ARDS patients.
“Despite a poor prognosis, critically ill patients treated with this therapy showed favourable mortality rates and those treated at the highest dose also had reduced rates of pneumonia, underscoring the potential application of iNKT cells, and agenT-797 in particular, in treating viral diseases and infections more broadly.
Amongst the intricacies of South Africa’s healthcare landscape, a silent but significant challenge lurks – the prevalence of rare diseases. Behind the curtain of mainstream medical discourse, millions grapple with the complexities of these often overlooked conditions, a stark reality often overshadowed by the glare of more prevalent health concerns.
With more than 7000 identified rare diseases to date, they affect as many as 4.2 million South Africans, of which 50 – 70% are children1. These conditions are more prevalent than predicted, each posing unique and often debilitating challenges for patients and families alike.
With 29 February commemorated as Rare Diseases Day, Rare Diseases South Africa (RDSA), is hosting its third biennial rare diseases conference, Rare-X 2024, at the Indaba Hotel in Fourways, from 14 to 17 February.
More than just a conference, Rare-X 2024 will focus on patient advocacy, education, policy reform, and improving equitable access to ensure better outcomes and support for individuals living with rare diseases.
As the first in-person conference since the COVID-19 pandemic, the event brings together patients, policymakers, academics, government and pharmaceutical companies to discuss the plight of rare diseases and find collaborative ways to improve patients’ lives and treatment efforts.
The conference will comprise several activities, including keynote speeches by renowned experts in rare diseases; interactive panel discussions; workshops and training sessions; scientific presentations; networking opportunities and policy roundtables.
Some of the renowned speakers to share their insights and global developments on rare diseases include Prof Alex van den Heever, Chair of Social Security Systems Administration and Management Studies at the Wits School of Governance; Professor Fatima Suleman, Professor in the School of Health Sciences at the University of KwaZulu-Natal; and Professor Chris Hendriksz, Global Clinical Development Lead for Rare Diseases at Nestle Health Science, amongst others. Bringing a wealth of practical experience following his work with health professionals, will be traditional health practitioners (THPs), Mr Elliot Makhathini and Dr Conradie from North-West University’s Centre for Human Metabolomics, to name a few.
A rare disease relates to a condition that is considered rare when it affects one person in 20002. Currently, South Africa does not have its own definition of a rare disease, which is one of the major issues that need to be addressed by the government3.
As a patient-focused non-profit organisation, RDSA was launched in 2013 by CEO and Rare-X Director, Kelly du Plessis. The mother of a child with a rare condition, du Plessis realised the dire need for support for a highly under-acknowledged community, with the organisation advocating that people living with rare diseases and congenital disorders experience greater recognition, support, improved health services, and overall, a better quality of life.
“Despite the need for increased representation, the rare diseases community remains vulnerable from a medical and policy perspective,” says du Plessis. “As part of our mandate, RDSA brings together international best practice and local medical innovation, driving a collective voice and playing a fundamental role in bridging the gap between vulnerable communities and medical advancement.”
To date, RDSA has successfully launched initiatives that have positively impacted the lives of over 6500 patients including engaging with various governmental departments, organs of state, industry players and strategic stakeholders to raise awareness and move rare disease policy forward.
For more information on the Rare-X conference, kindly visit www.rare-x.co.za
A new method has been developed that could non-invasively ease pain, avoiding the side effects of pain medication and the addiction problems associated with current opioid pain relievers.
This new study by Wynn Legon, assistant professor at the Fralin Biomedical Research Institute at Virginia Tech, and his team targets the insula, the location for pain reception deep within the brain. Their study, published in the journal PAIN, found that soundwaves from low-intensity focused ultrasound aimed at this spot can reduce both the perception of pain and other effects of pain, such as heart rate changes.
“This is a proof-of-principle study,” Legon said. “Can we get the focused ultrasound energy to that part of the brain, and does it do anything? Does it change the body’s reaction to a painful stimulus to reduce your perception of pain?”
Unlike ultrasound scans, focused ultrasound delivers a narrow band of sound waves to a tiny point. At high intensity, ultrasound can ablate tissue. At low-intensity, it can cause gentler, transient biological effects, such as altering nerve cell electrical activity
Neuroscientists have long studied how non-surgical techniques, such as transcranial magnetic stimulation, might be used to treat depression and other issues. Legon’s study, however, is the first to target the insula and show that focused ultrasound can reach deep into the brain to ease pain.
The study involved 23 healthy human participants. Heat was applied to the backs of their hands to induce pain. At the same time, they wore a device that delivered focused ultrasound waves to a spot in their brain guided by magnetic resonance imaging (MRI).
Participants rated their pain perception in each application on a scale of zero to nine. Participants reported an average reduction in pain of three-fourths of a point.
“That might seem like a small amount, but once you get to a full point, it verges on being clinically meaningful,” said Legon, also an assistant professor in the School of Neuroscience in Virginia Tech’s College of Science.
“It could make a significant difference in quality of life, or being able to manage chronic pain with over-the-counter medicines instead of prescription opioids.”
Researchers also monitored each participant’s heart rate and heart rate variability as a means to discern how ultrasound to the brain also affects the body’s reaction to a painful stimulus.
The study also found the ultrasound application reduced physical responses to the stress of pain – heart rate and heart rate variability, which are associated with better overall health.
“Your heart is not a metronome. The time between your heart beats is irregular, and that’s a good thing,” Legon said.
“Increasing the body’s ability to deal with and respond to pain may be an important means of reducing disease burden.”
The effect of focused ultrasound on those factors suggests a future direction for the Legon lab’s research – to explore the heart-brain axis, or how the heart and brain influence each other, and whether pain can be mitigated by reducing its cardiovascular stress effects.
The period of austerity politics from 2012 to 2018 was associated with steeper increases in frailty with age compared to pre-austerity between 2002 and 2010, according to a new study published February 7th in the open-access journal PLOS ONE by Carys Pugh of the University of Edinburgh, UK, and colleagues.
Previous research has linked a stalling in life expectancy growth to austerity politics implemented in response to the 2008–2009 financial crisis. However, the mechanism through which public spending cuts are associated with decreased life expectancy has been unclear.
In the new study, researchers analysed data on the frailty index, which captures broad age-related declines in functional ability and physical and mental health. The analysis included 16 410 people enrolled in the English Longitudinal Study of Ageing 2002-2018 with a mean age of 67 years and a mean frailty index score of 0.15 (on a scale of 0 to 1, with 1 being maximum frailty).
The study found that frailty index scores increased more rapidly with age after implementation of austerity policies. This was the case for all population groups, across genders and socioeconomic groups, but was particularly so for the oldest people. For instance, among the poorest women aged 85+, the frailty index increased an average of 0.013 per year in the eight-year pre-austerity period (2002-2010) but increased by 0.021 per year, two thirds as much again, in the six-year austerity period (2012-2018).
The additional increase in frailty for all ages from 2012 to 2018 was similar in magnitude to the difference in mean frailty score between people five years apart – those aged 65-69 and those aged 70-74.
The authors conclude that frailty may be one mechanism through which public spending reductions have negative impacts on health and mortality, particularly among the oldest people in society. New austerity measures, they write, need careful consideration given their potential impact on long-term health.
The authors add: “Compared to the 2000s, we found that older people in England were becoming more frail, more quickly during the 2010s, and that frailty was particularly common in the poorest in society. Our work is another warning about the possible impacts of austerity on the health of an ageing nation.”
