55 million Africans predicted to suffer from diabetes by 2045

By World Health Organisation

Africa is expected to experience the highest increase in diabetes globally. The number of people suffering from the disease is predicted to rise to 55 million by 2045—a 134% spike compared with 2021. 

At 70%, the continent also has the world’s highest number of people who do not know they have diabetes.

Dr Bernadette Adeyileka-Tracz, Founder and Chief Operating Officer of Diabetes Africa, a non-profit organization working to improve the health of Africans living with diabetes, explains the challenges in diabetes prevention and care in the continent.

What are the challenges a person living with diabetes faces in Africa? 

 

The number one challenge for a person living with type 1 or type 2 diabetes in Africa is diagnosis: people living with diabetes may not know they have the condition until serious complications develop. That’s because “looking healthy” is not the same as “being healthy.” 

People with excess blood sugar, particularly in the case of type 2 diabetes, can live without experiencing complications for a long time. However, when these complications do manifest, they present huge problems to livelihoods and add to the burden of already-strained health systems. Blindness, amputation, heart emergencies are not light complications.

In many places across the continent, this challenge is compounded by a lack of testing facilities or training among health workers that would enable them to identify risks early on. The earlier Type 2 diabetes or pre-diabetes can be diagnosed and treated, the better for the person and for the community at large.

The cost and access to medication is of course an acute challenge in Africa. We can add that managing blood glucose levels requires testing blood sugar levels regularly, and this presents challenges on its own: people living with diabetes should receive adequate training on the topic, but also be able to afford it: how can we expect people to test regularly if they must choose between feeding their families and buying test strips?

Diagnosis and testing are the first of a series of cascading challenges. Experts often use what they call the “rule of halves” to describe them: of 100 people living with diabetes, only 50 of them will have been diagnosed. The rest will live with the condition and its consequences without being aware of it. Of the 50 that have been diagnosed, 25 will receive care. 

The others are unable to seek care, for personal or economic reasons. Finally, among the 25 who receive adequate care, only 12 or 13 will meet their targets in terms of readings or measurements. But in the end, only six of them will have a positive health outcome.

This shows the scale of the challenge in front of us. It’s also possible that in remote and rural areas in Africa a rule of thirds or a rule of fourths applies.

How has COVID-19 made their situation worse? 

 

COVID-19 has certainly increased risks for people living with diabetes. People living with diabetes are more likely to have more severe symptoms of COVID-19. Research is still ongoing, but anecdotal evidence coming from Africa tends to corroborate research done in the United Kingdom and in the United States of America. 

In the Democratic Republic of the Congo, for example, the analysis of a small sample of 215 people who died of COVID-19 showed that 30% of them had diabetes.

The public response to the pandemic has also had an impact on people living with diabetes. An initial reaction to the pandemic has been to encourage people to stay at home and reduce visits to the hospital. People living with diabetes also chose to stay at home for fear of catching the virus. With the pandemic stretching into 2021, this did not prove to be a sustainable solution.

The sudden global focus on health may have encouraged some people to control their diabetes better and make an extra effort, but in general, COVID-19 and its associated restrictions has meant that people became more sedentary, experienced stress and anxiety and had more difficulties keeping a healthy diet and controlling their diabetes.

Luckily, in most instances, hospitals and healthcare providers acknowledged these challenges and made a special effort to allow people living with chronic conditions to receive face-to-face care. Telemedicine has helped in countries where phone calls and video calls were a workable option, for example in Nigeria and Kenya. However, in other countries, such as Uganda for example, the cost and technical challenges of telemedicine were often prohibitive.

COVID-19 has also been the catalyst for positive changes. Civil society organizations have had an active role in encouraging governments to tackle non-communicable diseases like diabetes to lighten the burden on health systems. In Kenya, the Noncommunicable Diseases Alliance has been actively advocating for essential diabetes medicines to be covered by the National Health Insurance Fund.

 

How can countries innovate and manage diabetes in a low-resource setting? 

 

It’s difficult to look at diabetes in isolation and ask how we could do more with less. Adequate healthcare demands a basic level of resources and given their impact on the overall system, noncommunicable diseases such as diabetes remain largely underfunded.

This is regrettable because managing health in a low-resource setting would benefit from actions that reduce costly complications, such as early diagnosis and prevention of diabetes. People who stay in the hospital with COVID-19 often have comorbidities. Can we not address these comorbidities at the source?

One of the greatest innovations in a low-resource setting would be to change mindsets: consider investing in keeping people healthy, rather than fixing problems. There will always be emergencies and costly operations that cannot be avoided. But diabetes need not be the cause of them.

This involves an effort to train healthcare professionals, inform and train people who may be at risk, sharing knowledge at a much greater scale, which is what Diabetes Africa aims to do. This would also entail working with commercial organizations, in particular in the food and beverage industry to label products and help people make informed decisions.

Study: Access to a simple breathing monitor could prevent 11,000 fatal anaesthetic accidents per year in sub-Saharan Africa

A study, the Global Capnography Project (GCAP), published 26 September 2018, in the journal Anaesthesia, clearly identified the need for middle and low-income countries to have access to a simple and life-saving method of monitoring a patient’s breathing, called capnography, in operating theatres and in intensive care units.

Capnography is an essential monitor for safe anaesthesia and is widely used in high-income countries as part of major improvements in the safety of anaesthesia care. 

The study called: ‘Global Capnography Project (GCAP): implementation of capnography in Malawi - an international anaesthesia quality improvement project’ and will be published in the journal Anaesthesia in September 2018 https://www.onlinelibrary.wiley.com/doi/10.1111/anae.14426 

A landmark study in 2011, the 4th National Audit Project (NAP4), further confirmed the importance of capnography both in the operating theatre and especially in the intensive care unit. Despite this, the device is hardly ever available in low-income countries.

The GCAP team set out to measure the availability of capnography and determine whether its introduction was feasible and could improve patient care in a low-income country. 

A pilot site was identified in Malawi and forty capnographs were donated by Medtronic to eight hospitals in the southern part of the country. In addition, thirty-two anaesthesia providers at these hospitals received a one-day capnography training course.

Prior to the pilot project, there was only one capnograph in the southern region of Malawi, across eight hospitals. During the six-month pilot, 699 episodes of monitoring with the capnographs were recorded and 90% of the anaesthesia providers trained believed that the use of capnography had saved lives. They reported that a minimum of 57 lives had been saved during the six months of use. 

Oesophageal intubation (accidently placing a tracheal tube in the patient’s oesophagus) and breathing circuit disconnections are two of the most important incidents in operating theatres and intensive care units that capnography monitoring can detect. Both of these can lead to significant patient harm and mortality if not identified early and corrected quickly.

During the six-month pilot, 44 oesophageal intubations were detected by capnography. From this, the GCAP team made an estimation that with a population of 7.5 million in Southern Malawi there is a rate of 11.7 oesophageal intubations per million population per year.

Assuming Southern Malawi is representative of sub-Saharan Africa as a whole (with a population of 1,022 million), the team then went on to estimate that over 11,000 oesophageal intubations could occur per year in the region.

These incidents pose a very significant patient safety risk that could be prevented by access to capnography. The GCAP team estimated that there are at least 70,000 operating theatres in the world
without Capnography.

Prof Ellen O’Sullivan from GCAP Team, said: “We believe that this is one of the most important projects in anaesthesia safety in the last decade. Our research clearly shows that lives are at risk in low-income countries due to an absence of a simple method of monitoring breathing, called capnography. 

This is despite international standards recommending its use. We are calling for the development of an international project to make global capnography to become a reality, so that like pulse oximetry, it can be included the World Health Organisation (WHO) surgical safety checklist to help improve patient safety worldwide All relevant organisations should consider taking this forward.”

 “These results show that capnography is desperately needed to help save patients’ lives in our operating theatres and ICUs. We found the equipment robust and easy to use. We plead with the international anaesthesia community to support the dissemination of the GCAP project throughout Malawi and other sub-Saharan African countries,”Dr Delia Mabedi from Zomba Hospital, Malawi commented.

"Medtronic is grateful for the opportunity to participate in the GCAP project that demonstrated the major clinical benefits of monitoring patients breathing with capnography. We look forward to working with the clinical community to address the global need for this technology and to keep patients safe worldwide,” said Vafa Jamali, senior vice president and president of Respiratory, Gastrointestinal and Informatics, which is part of the Minimally Invasive Therapies Group at Medtronic.

-Ends-
REF: https://www.onlinelibrary.wiley.com/doi/10.1111/anae.14426

Study finds blocking or hiding facial movement can affect sharing of emotions and social interaction

Hiding the bottom half of the face with a mask could have a detrimental effect on our ability to socially interact and share other people’s emotions, new research suggests.

A study, which was led by Cardiff University and involved researchers from Queen’s University Belfast, found that people with facial paralysis, people seeing others who wear face masks, or even children sucking on dummies, could struggle to show empathy or detect positive social cues.

The research is published in the journal Cognitive, Affective, and Behavioral Neuroscience.

Lead author Dr Ross Vanderwert, from Cardiff University’s School of Psychology, says: “People tend to automatically imitate others’ facial expressions of emotion when looking at them, whether that be a smile, a frown, or a smirk. 

This facial mimicry – where the brain recreates and mirrors the emotional experience of the other person – affects how we empathise with others and interact socially.

“Our study suggests that when the movements of the lower part of the face are disrupted or hidden, this can be problematic, particularly for positive social interactions and the ability to share emotions.

“Wearing a face mask continues to be vital to protect ourselves and others during the COVID-19 pandemic – but our research suggests this may have important implications for the way we communicate and interact.”

Dr Magdalena Rychlowska, from Queen’s University Belfast’s School of Psychology, was second author on the paper. She comments: “Our findings suggest that processing faces is a very challenging task and that the brain may need more support from, and rely more heavily on, our own faces to support the visual system for understanding others' emotions.

“This mirroring or simulation of another person’s emotions may enable empathy; however, up until now the neural mechanisms that underline this kind of emotion communication have been unclear.”

The scientists recorded the brain activity of 38 individuals via an electroencephalogram whilst they watched videos of fearful, happy, and angry expressions, as well as a collection of inanimate everyday objects, as a control.

Study participants were asked to watch the videos whilst holding a pen between their teeth for half the videos and without the pen for the remaining videos.

The researchers were investigating, for the first time, the effect this had on a process known as neural mirroring – activity in the motor system for our own actions that is also active observing other’s actions. Neural mirroring facilitates simple tasks such as hand-eye coordination and more complex tasks like understanding the emotions of others.

The results revealed that participants who could freely move their face showed significant neural mirroring when observing the emotional expressions, but not the everyday objects.

While the pen was held in their teeth, no neural mirroring was observed when looking at the happy and angry expressions – but it did show neural mirroring when looking at fearful expressions.

Dr Rychlowska adds: “For emotions that are more heavily expressed by the eyes, for example fear, blocking the information provided by the mouth doesn’t seem to affect our brain’s response to those emotions. But for expressions that depend on the mouth, like a friendly smile, the blocking had more of an effect.”

Cervical cancer rates reduced by 87% in women vaccinated against HPV, English study confirms

Cervical cancer rates are 87% lower in women who were offered vaccination against human papillomavirus (HPV) when they were between the ages of 12-13 than in previous generations confirms a new study published in The Lancet. 

The researchers also found reductions in cervical cancer rates of 62% in women offered vaccination between the ages of 14-16, and 34% in women aged of 16-18 when vaccination was introduced. This is the first direct evidence of prevention of cervical cancer using the bivalent vaccine, Cervarix.

HPV vaccination has been introduced in 100 countries as part of efforts by the World Health Organization (WHO) to eliminate cervical cancer. England initially used a bivalent vaccine which protects against the two most common types of HPV, responsible for approximately 70-80% of all cervical cancers. 

The English HPV vaccination programme was introduced in 2008, with vaccines given to women between 12-13 years old and “catch-up” vaccinations offered to older age groups up to the age of 18.

“Although previous studies have shown the usefulness of HPV vaccination in preventing HPV infection in England, direct evidence on cervical cancer prevention was limited,” says Professor Peter Sasieni, King’s College London, one of the authors of the paper “Early modelling studies suggested that the impact of the vaccination programme on cervical cancer rates would be substantial in women aged 20-29 by the end of 2019. Our new study aims to quantify this early impact. The observed impact is even greater than the models predicted.”

The study looked at population-based cancer registry data between January 2006 and June 2019 for seven cohorts of women who were between the ages of 20-64 at the end of 2019. Three of these cohorts formed the vaccinated population, where women were vaccinated with Cervarix between the ages of 12-13, 14-16 and 16-18 respectively. Incidences of cervical cancer and non-invasive cervical carcinoma (CIN3) in the seven populations were recorded separately.

During the study period, 28,000 diagnoses of cervical cancer and 300,000 diagnoses of CIN3 were recorded in England. In the three vaccinated cohorts there were around 450 fewer cases of cervical cancers and 17,200 fewer cases of CIN3 than expected in a non-vaccinated population. 

The research found reductions in cervical cancer rates of 87% (with a confidence interval of 72-94%) in women targeted between the ages of 12-13 (89% of whom received at least one dose of the HPV vaccine and 85% of whom had received three jabs and were fully vaccinated), 62% (CI: 52-71%) in women potentially vaccinated between the ages of 14-16, and 34% (CI: 25-41%) in those eligible for vaccination between the ages of 16-18 (60% of whom received at least one dose and 45% of whom were fully vaccinated). 

The corresponding reductions in CIN3 rates were 97% in women vaccinated between the ages of 12-13, 75% in women vaccinated between the ages of 14-16 and 39% in women vaccinated between the ages of 16-18.

“This study provides the first direct evidence of the impact of the UK HPV vaccination campaign on cervical cancer incidence, showing a large reduction in cervical cancer rates in vaccinated cohorts. As expected, vaccination against HPV was most effective in the cohorts vaccinated at ages 12-13 amongst whom the uptake was greatest and prior infection least likely,” says Dr Kate Soldan from the UK Health Security Agency and co-author. 

“This represents an important step forwards in cervical cancer prevention. We hope that these new results encourage uptake as the success of the vaccination programme relies not only on the efficacy of the vaccine but also the proportion of the population vaccinated.”

Lucy Elliss-Brookes, Associate Director for Data Curation at NHS Digital and one of the authors of the paper said: “The findings of this study are hugely important in encouraging those eligible to take up the vaccine, but also in demonstrating the power of data [2] in helping medical researchers and the NHS to understand what causes cancer and how best to diagnose, prevent and treat it.”

The authors acknowledge some limitations with the study, principally that cervical cancer diagnosis is rare in young women. In addition, the number of registered cases of cervical cancer is impacted by the age at which women are screened; most of the follow-up for women in vaccinated cohorts occurred under the age of 25 and small differences in the age of first screening can have a large impact on registered cervical cancer cases in women below this age. 

Because the vaccinated populations are still young, the authors stress that this means that it is still too early to assess the full impact of HPV immunisation on cervical cancer rates. However, it is important to note that the two most common HPV infections which the bivalent vaccine protects against are present in as many as 92% of women diagnosed with cervical cancer before the age of 30.

It should also be noted that the bivalent vaccine Cervarix used in the UK from 2008-2012. Since September 2012, the quadrivalent vaccine Gardasil has been used instead.

Writing in a linked comment, Professor Maggie Cruickshank from the University of Aberdeen (UK), who was not involved in the study, says: “The scale of HPV vaccination effect reported by this study should stimulate vaccination programmes in low and middle-income countries where the problem of cervical cancer is a far greater public health issue than those with well-established systems of vaccination and screening."

"The most important issue, besides the availability of the vaccine (related to the decision-makers in the health policy), is the education of the population to accept the vaccination, as an increase in the rate of immunization is a key element of success.”

Infertility Treatments Raise No Insurmountable Theological or Legal Problems for Muslim Scholars, says WISH 2020 Report

By Esther Nakkazi

Women and men who are infertile and fail to have children face discrimination, stigma, and ostracism in many cultures worldwide and Assisted Reproductive Technologies (ARTs), more commonly known as fertility treatments are often morally questioned. 

In the Islamic world moral tradition is characterized by diversity of opinions on many of the questions raised by ARTs. 

This gives healthcare professionals, their patients, and policymakers greater freedom in formulating governing policies, regulations, and in drafting laws says a report released during the 2020 World Innovation Summit for Health (WISH) is collaborating with the Research Center for Islamic Legislation & Ethics (CILE) at the College of Islamic Studies, Hamad Bin Khalifa University (HBKU).

Infertility affects around 10-15% of couples globally. ARTs have provided solutions that have benefited many couples and while the biomedical scientists have been working to improve the safety and efficacy of these new technologies, ethicists and religious experts have simultaneously been studying the related moral issues. 

This is so couples that undergo treatment do not compromise their moral or religious values and principles. The observation is pertinent for Qatar and other Muslim-majority countries, where a legal vacuum in this field persists.

The research report Islamic Ethics and Infertility Treatment presented at the WISH 2020 biennial conference provided healthcare professionals and policymakers, with informative and practical guide to better understanding how ARTs intersect with the moral world in the Islamic tradition.

Maha El Akoum, Head of Content at WISH and a contributor to the report, said a meeting of religious and biomedical scholars on this subject is crucial, and this seminal report is an extension of WISH’s long-term interest in examining the interplay between biomedical sciences and Islamic ethics.

"It is an honor to be drawing on the widely-respected scholarship of CIS’ research center at HBKU, to provide informed and evidence-based policy recommendations from the perspective of Islamic ethics.”

But miscarriage prevention remains a low public health priority in many low- and middle-income countries, where there are many competing health care priorities and services for women can be especially limited.

There needs to be a minimum service available globally for women with recurrent miscarriage, and in low- and middle-income countries this service should include tests to check for diabetes, anaemia, thyroid abnormalities, and antiphospholipid syndrome when indicated, with appropriate treatment based on the results. 

There also needs to be a focus on providing pre-pregnancy counselling and psychological support to women with repeated miscarriages. This will require investment in early pregnancy care in low- and middle-income countries, and we recommend increasing provision of medication and equipment, training in scanning and surgical procedures, and creating dedicated early pregnancy units to ensure high-quality care. Awareness-raising programmes to encourage women to seek care is also needed.” [1]

QF’s global health initiative collaborates with HBKU’s College of Islamic Studies ahead of 2020 health summit this November.

Qatar launched its first IVF center in April 1993, and the Assisted Reproductive Center (ARC) established at HMC, the country’s largest governmental healthcare provider, currently performs over 1,500 cycles a year for female and male infertility, with a success rate comparable to that of international centers.

Maha El Akoum, Head of Content at WISH and a contributor to the report, said: “Infertility affects around 10-15% of couples globally. ARTs have provided solutions that have benefited many couples and while the biomedical scientists have been working to improve the safety and efficacy of these new technologies, ethicists and religious experts have simultaneously been studying the related moral issues. This is so couples that undergo treatment do not compromise their moral or religious values and principles.

Some of those issues revolve around the risks of such therapies, while others focus on the definitions of what constitutes a marriage or family. The financial aspect is another cause of ethical concern, particularly around the inequitable access to care, given the high-cost of these therapies. Government-aided funding for ART varies widely among different countries.

In responding to the complex and multidimensional questions arising from the use of ARTs, Muslim religious scholars have sometimes formally collaborated with biomedical scientists to understand some of the scientific and biomedical aspects of ARTs. 

As explained in previous studies published by WISH, the collaboration between religious scholars and biomedical scientists was facilitated through the mechanism of collective religio-moral reasoning.

The World Innovation Summit for Health (WISH) is a global healthcare community dedicated to capturing and disseminating the best evidence-based ideas and practices. WISH is an initiative of Qatar Foundation for Education, Science and Community Development (QF) and is under the patronage of Her Highness Sheikha Moza bint Nasser, its Chairperson.

The inaugural WISH Summit took place in Doha in 2013 and convened more than 1,000 global healthcare leaders. Through international summits and a range of ongoing initiatives, WISH is creating a global community of leading innovators in healthcare policy, research, and industry.

Together, they are harnessing the power of innovation to overcome the world’s most urgent healthcare challenges and inspire other stakeholders to action.

Qatar Foundation for Education, Science and Community Development (QF) is a non-profit organization that supports Qatar on its journey to becoming a diversified and sustainable economy. QF strives to serve the people of Qatar and beyond by providing specialized programs across its innovation-focused ecosystem of education, science and research, and community development.

QF was founded in 1995 by His Highness Sheikh Hamad bin Khalifa Al Thani, the Father Emir, and Her Highness Sheikha Moza bint Nasser, who shared the vision to provide Qatar with quality education. Today, QF’s world-class education system offers lifelong learning opportunities to community members as young as six months through to doctoral level, enabling graduates to thrive in a global environment and contribute to the nation’s development.

QF is also creating a multidisciplinary innovation hub in Qatar, where homegrown researchers are working to address local and global challenges. By promoting a culture of lifelong learning and fostering social engagement through programs that embody Qatari culture, QF is committed to empowering the local community and contributing to a better world for all.

New study: How UNESCO's World Heritage forests play a vital role in mitigating climate change

Paris, 28 October – The first ever scientific assessment of the amounts of greenhouse gases emitted from and absorbed by forests in UNESCO World Heritage sites has found that forests in World Heritage sites play a vital role in mitigating climate change by absorbing 190 million tons of CO2 from the atmosphere each year. However, ten forestsreleased more carbon than they sequestered due to pressure from human activity and climate change, which is alarming.

World Heritage forests absorb 190m tons of CO2 each year

By combining satellite-derived data with monitoring information at the site level, researchers at UNESCO, World Resources Institute (WRI) and the International Union for Conservation of Nature (IUCN) were able to estimate the gross and net carbon absorbed and emitted by UNESCO World Heritage forests between 2001 and 2020 and determine the causes of some emissions.

The research found that, as a whole, UNESCO World Heritage forests in 257 separate sites, absorbed the equivalent of approximately 190 million tons of CO2 from the atmosphere each year, comparable to roughly half the United Kingdom’s annual CO2 emissions from fossil fuels. “We now have the most detailed picture to date of the vital role that forests in World Heritage sites play in mitigating climate change,” said Tales Carvalho Resende, from UNESCO who co-authored the report.

World Heritage forests, whose combined area of 69 million hectares is roughly twice the size of Germany, are biodiversity-rich ecosystems. In addition to absorbing CO2 from the atmosphere they also store substantial amounts of carbon. Carbon sequestration by these forests over long periods has led to total carbon storage of approximately 13 billion tons of carbon, which is more than the carbon in Kuwait’s proven oil reserves. If all this stored carbon were to be released into the atmosphere as CO2, it would be akin to emitting 1.3 times the world’s total annual CO2 emissions from fossil fuels.

Findings from 10 World Heritage forests are cause for concern

However, given that World Heritage sites are highly prized and protected, the fact that 10 of 257 forests emitted more carbon than they captured between 2001 and 2020 due to different anthropogenic disturbances and pressures is alarming.

At some sites the clearance of land for agriculture caused emissions to be greater than sequestration. The increasing scale and severity of wildfires, often linked to severe periods of drought, is also a predominant factor in several cases. Other extreme weather phenomena, such as hurricanes, contributed at certain sites.

“All forests should be assets in the fight against climate change. Our report’s finding that even some of the most iconic and best protected forests such as those found in World Heritage sites can actually contribute to climate change is alarming and brings to light evidence of the severity of this climate emergency”, said Tales Carvalho Resende.

In the coming years, ongoing sequestration and carbon sinks are likely to be affected at a growing number of sites worldwide as a result of increasingly fragmented and degraded landscapes, and more frequent and intense climate-related events.

Better management of sites can yield results

The report urges strong and sustained protection of UNESCO World Heritage sites and their surrounding landscapes to ensure their forests can continue to act as strong carbon sinks and stores for future generations. To achieve this, the report recommends rapidly responding to climate-related events, as well as maintaining and strengthening ecological connectivity through improved landscape management.

For example, in Indonesia, government agencies have been using near real-time fire alert systems to significantly reduce their average fire response time. Rapid response is integral to preventing fires from developing into destructive conflagrations that produce extensive CO2 emissions.

At the Sangha Trinational World Heritage site, located within Cameroon, the Central African Republic and the Republic of Congo, the creation of a buffer zone around the site has kept some human activity farther from this important carbon sink.

The report also recommends integrating the continued protection of UNESCO World Heritage sites into international, national and local climate, biodiversity and sustainable development strategies in line with the Paris climate agreement, the Post-2020 Global Biodiversity Framework and the Sustainable Development Goals.

“This analysis of iconic World Heritage sites shows that combining satellite data with on-the-ground information can improve local decision-making and strengthen accountability, thereby helping forests, climate, and people,” said David Gibbs, WRI Research Associate and co-author of the report.

“Protecting World Heritage sites from increasing fragmentation and escalating threats will be central to our collective ability to address climate change and biodiversity loss,” added Tim Badman, Director of IUCN’s World Heritage Programme.

New biotech firm launched to develop ‘revolutionary’ treatment for one of world’s rarest neurodegenerative diseases

A new biotech business SynaptixBio has been launched to tackle one of the world’s rarest neurodegenerative diseases.

SynaptixBio is working to develop the world’s first disease-modifying treatment for TUBB4a leukodystrophy, including H-ABC - a debilitating and potentially life-limiting condition.

Founded by a worldwide team of leading medical and pharmaceutical experts, the Oxford-based research company has entered into a sponsored research agreement with Children’s Hospital of Philadelphia (CHOP) in the US related to a new method for treating TUBB4a leukodystrophy.

SynaptixBio also has an option to exclusively license CHOP research related to this project. Antisense Oligonucleotides (ASOs) therapy, which has previously been used to treat conditions such as Duchenne muscular dystrophy and spinal muscular atrophy, is also hoped to dramatically improve the quality of - and extend – the lives of leukodystrophy patients.

Dr Dan Willams, CEO and co-founder of SynaptixBio, said the treatment had the potential to “modify the disease, increase survival and significantly improve motor skills development.

“The new approach provides the potential to stabilise, improve quality of life and extend life expectancy in children suffering from the condition.“Successful prevention of leukodystrophy progression would be a revolutionary life-saving and life-enriching treatment.”

TUBB4a leukodystrophy causes hypomyelination in the brain, which can lead to developmental delays in motor skills such as walking, sitting, speaking and swallowing; learning difficulties, seizures, paralysis and an early death.

Research has already begun, with the firm aiming to launch clinical trials in 2024.“This project will change people’s lives,” Williams said. “The research and development of a clinically-proven treatment for TUBB4a would be a real game-changer for patients and their families.

“There is a real chance to improve the lives of leukodystrophy sufferers. We want to ensure that dream becomes a reality.”