Bedside to bench: research by new institute at UP driven by patients in need

28 April 2015 by Department of University Relations

“There are more than 6 million HIV+ people living in our region, and some would say we are the 3^rd most obese nation in the world,” says Prof Michael Pepper, Director of the recently launched Institute for Cellular and Molecular Medicine (ICMM) at the University of Pretoria (UP).

Add high incidences of tuberculosis (TB), cancer, cardiovascular disease, domestic violence, car accidents, mother and child deaths and depression, and South Africa’s health burden feels heavy indeed.

Diagnosis can also intensify the problem – take cystic fibrosis (CF) for instance, which is caused by a mutation in a single gene. “In many black African and mixed-race patients we can’t make a molecular diagnosis because the tools we have were designed for mutations in Caucasians,” says Prof Pepper.

Now however, the ICMM has sequenced the genes of southern Africans, and found new mutations that have never been described before. Says Prof Pepper, “in the case of CF this has allowed us to reduce the 50% of people we couldn’t diagnose using molecular tools before to just 5%.”

An accurate molecular diagnosis is important to manage a CF patient’s treatment properly, especially since the high incidence of TB in South Africa means that CF and TB, which both affect the lungs, can be confused.

The high genetic diversity of southern Africans (they have been on the planet the longest, so have had the most time to accumulate genetic variations), together with the high disease burden gives scientists a unique opportunity for niche research that will benefit patients in southern Africa, Africa and the rest of the developing world: the more patients there are, the more material (cells, DNA samples, tissue samples, medical histories etc.) becomes available for studies aimed at finding cures, treatments and vaccines.

In 2008, with these challenges and opportunities in mind, Pepper and a few colleagues began what is now the ICMM. They started with a laptop and an idea and were allocated a gutted laboratory. That room today houses the core of the ICMM – a globally-competitive facility with high-tech infrastructure and skilled scientists.

Today, the ICMM draws on seven of the nine faculties at UP, with 20 cross-disciplinary research projects across four streams – stem cells, genomics, neuroscience and infectious diseases – and a pool of legal, ethical and entrepreneurship expertise.

“The question was, ‘how do you improve the quality of patients’ lives?’”, he explains. “First you do the hard science to get good data and develop novel diagnostics and therapeutics. You do this in a setting that is legally and ethically sound, and you must have an entrepreneurial component, otherwise these products will never get to market. The patient is our market.”

In the process, new career scientists are trained; top researchers are retained and attracted from around the globe; small-to-medium enterprises are grown; intellectual property, patents and peer-reviewed publications are generated; and the public, doctors and research ethics committees are educated about new developments. In other words, this market-driven, bedside-to-bench approach to research doesn’t only contribute to improved health, it also boosts the knowledge and bio-economy.

A testament to the ICMM’s success so far, the Medical Research Council recently awarded it an Extramural Research Unit for Stem Cell Research and Therapy, which now hosts one of the institute’s major projects: finding a gene therapy cure for HIV.

“We are one of the most active stem cell research groups in the country, and this is our flagship research,” says Prof Pepper. As long as this stream of research is sufficiently productive, funding from the MRC should continue for the next 15 years.

The CF research described earlier falls under the genomics research stream, and Prof Pepper describes those results, which are currently being patented, as “fantastic”.

As part of the infectious diseases stream, several projects and collaborations focussing on HIV and TB are underway.

Lastly, Pepper notes that mental health has been severely neglected on the African continent, and that globally, depression is set to become one of the most widespread diseases by 2030 (according to WHO). This is why the ICMM will also focus on neuroscience, and Pepper is excited that neuroendocrinology (the study of how the brain and hormones interact) will soon become a major theme in the Faculty of Health Sciences at UP.

Researchers

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  Professor Michael Pepper

  Professor Michael S Pepper is Director of the Institute for Cellular and Molecular Medicine at the University of Pretoria (UP) and Director of the South African Medical Research Council’s Extramural Unit for Stem Cell Research and Therapy.
  
  His research goal is to understand disease pathogenesis and to develop cell and gene therapies for selected diseases. Prof Pepper has been conducting research at UP since 2005, and through his work, aims to improve the quality of life of South Africans and reduce the heavy disease burden in the country.
  
  The professor’s research group has identified key areas that contribute to the burden of disease in South Africa, including communicable diseases such as HIV and COVID-19, non-communicable diseases like obesity or cancer, and maternal and child health issues such as birth asphyxia.
  
  The group’s birth asphyxia project recently received funding from the Bill & Melinda Gates Foundation, and focuses on the identification of biomarkers (molecular and imaging) for the condition. This is important in understanding disease pathogenesis and for the design of an interventional clinical trial aimed at assessing the efficacy of one or more neuroprotective agents.
  
  Over the past 18 months, Prof Pepper’s research group has also started two new COVID-19 projects: genetic determinants of susceptibility and severity as well as novel therapies for acute respiratory distress syndrome.
  
  Prof Pepper is working with Prof Fourie Joubert of the University’s Faculty of Natural and Agricultural Sciences and Prof Pieter de Villiers of the Faculty of Engineering, Built Environment and Information Technology in transdisciplinary cross-faculty research. Prof Pepper says the work they are doing is inspired by a need to address the needs of South Africans and to ensure that taxpayers’ money is being spent responsibly. Significant advances are being made, and medical research in selected fields in South Africa is being conducted at the highest level globally.
  
  Prof Pepper’s advice to prospective and undergraduate students who are interested in his field is to find their passion and not to be driven by a desire for fame or material possessions. They should be prepared to work hard and be ready to adapt to the rapidly changing environment in which we live.
  
  He enjoys the theatre and horse riding.
  

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  Bedside to bench: research by new institute at UP driven by patients in need