Cell- and gene-based therapies can potentially transform the lives of those with life-threatening diseases and are a robust example of precision medicine.
A single dose is all it takes to cure blood cancers, which previously had high mortality rates.
“We’re starting to see cure rates of 70 to 80% for leukaemia,” says Professor Michael Pepper, founder of the Institute for Cellular and Molecular Medicine at the University of Pretoria (UP). This had not been possible previously.
The first paediatric patient in the experimental T-cell therapy clinical trial for advanced B cell leukaemias and lymphomas in the United States recently celebrated a decade of being cancer-free.
Regarded as one of the most exciting and rapidly growing areas in the practice of medicine, cell- and gene-based therapeutic techniques have been limited in South Africa.
Until recently, virtually nothing had been done to explore the potential offered by these technologies to address the high disease burden in the country.
Broadly speaking, advanced cellular and gene therapeutic technologies can be grouped under regenerative and adoptive strategies.
Regenerative therapies focus on repairing or replacing damaged or diseased tissues and organs. They stimulate the body’s natural ability to heal itself by promoting tissue regeneration and repairing damaged cells. These therapies involve the use of stem cells, growth factors and other biological agents to promote tissue repair and regeneration.
Adoptive therapies, on the other hand, involve modifying or manipulating the cells from a patient’s immune system outside the body, then infusing them back into the patient. These therapies enhance immune response against particular diseases such as cancer, infections or autoimmune disorders.
Both therapies are being investigated at UP’s Institute for Cellular and Molecular Medicine.
Advanced therapy for all
It is imperative that these advanced therapies not be limited to the privileged few, nor for rare diseases only, Prof Pepper says. The institute’s research targets several diseases that contribute to the high disease burden in South Africa.
These include communicable diseases such as HIV, non-communicable diseases such as obesity and cancer, and diseases responsible for infant morbidity and mortality.
“These forward-looking projects contribute to the development of novel regenerative and adoptive cell therapies that will address a global need, particularly in lower- and middle-income countries,” Prof Pepper says.
There are benefits beyond the knowledge shared by north-south collaborations with international institutions where these therapies are being manufactured and administered.
According to a paper co-authored by Prof Pepper and published in the journal Nature, the genetic diversity of Southern Africa, which includes some of the oldest living human populations, is making an important Global South contribution to productive research collaborations.
The Global North, in turn, provides much-needed technological expertise for the analysis of this rich source of information.
The economics of health
Novel therapies require investment: in people, manufacturing infrastructure and long-term thinking.
“It is important to weigh up the cost of an expensive once-off treatment against the lifetime costs of continued treatment, morbidity and mortality,” Prof Pepper says.
Novel therapies also require appropriate, accurate and unambiguous legislation. South African law has not kept pace with the advances in technology, and legislation governing the manufacture or import of cell and gene therapies is vague.
According to Prof Pepper, to meet the South African National Development Plan’s goal of “universal, equitable, efficient and quality healthcare”, all members of society should benefit from advances in medical science.
South African legal deficiencies should not delay matters.
Advanced cell and gene therapies will have a major impact on medicine in the years to come.
Although South Africa has yet to establish a platform for the delivery of these technologies to its population, work by the Institute for Cellular and Molecular Medicine and others is paving the way for affordable, accessible therapies that will benefit a wide range of patients with a multitude of diseases.
“We cannot deny South Africans the benefit of these therapies,” Prof Pepper says.
Why this research matters
Advances in the field of genome engineering mean that in our lifetimes, patients with incurable diseases are likely to benefit from a cure from a single-application procedure.
According to a paper published in Nature and co-authored by Prof Pepper, this is an unprecedented advancement in medicine that will increase substantially in the next few years.
Professor Michael Pepper is a co-founder of Antion Biosciences, a cell and gene engineering company that is based in Switzerland, which is ranked as the top country globally in terms of research per capita.
The company’s proprietary technologies take multiplex cell engineering beyond the current capabilities of gene editing with the aim of enabling off-the-shelf, efficient, scalable cellular immunotherapies.
The intellectual property of these technologies is managed by the University of Geneva, with benefits that will flow back to UP as valuable third- stream income.
Established in 2011 with the intention of curing HIV, the company took the same technology and pivoted to cancer treatment.
It has since progressed from autologous cancer therapies to allogeneic therapies: autologous therapies are manufactured as a single lot from the patient being treated; allogeneic therapies are manufactured in large batches from cells from an unrelated donor.
Researcher: Professor Michael Pepper, Director of the Institute for Cellular and Molecular Medicine