The global cost of diabetes is skyrocketing, with some experts estimating it could reach $2.5 trillion by 2030 or more than 2% of total GDP. So, it’s no surprise that clinical trial results involving just one patient who has been ‘cured’ of the disease is making headlines.
Vertex Pharmaceuticals has announced 90-day results from its phase 1/2 trial for VX-880, a stem-cell-derived therapy that restores insulin production. The 40-year-old patient with type 1 diabetes – who was insulin-dependent and had experienced five life-threatening hypos in the past year – has seen a 91% reduction in his daily insulin requirement and robust improvement in glucose control.
VX-880 is a stem-cell-derived, fully differentiated pancreatic islet cell replacement therapy that is delivered as a single infusion. The experimental treatment builds on experience gained from transplants of pancreatic islet cells harvested from cadavers.
“As a surgeon who has worked in the field of islet cell transplantation for decades, this approach, which obviates the need for an organ donor, could be a game changer,” said Dr James Markmann, professor of surgery at Massachusetts General Hospital. “We are excited to progress this unique and potentially transformative medicine through clinical trials and to patients.”
One major drawback of VX-880 is that patients need to also take immunosuppressive drugs to prevent their own immune systems from destroying the implanted pancreatic islets cells. However, Vertex aims to avoid this problem by encapsulating the pancreatic cells to shield them from immune attack.
“These results from the first patient treated with VX-880 are unprecedented,” said Vertex’s head of cell and genetic therapies, Bastiano Sanna. “While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression.”
The therapy was first created by Harvard University’s Doug Melton, who said: “More than a decade ago our lab had a vision for developing an islet cell replacement therapy to provide a functional cure to people suffering from T1D. These promising results bring great hope that stem cell-derived, fully differentiated islet cells could deliver a life-changing therapy for people who suffer from the relentless life-long burden of T1D.”
Vertex has competition in its bid to develop a curative treatment from San Diego-based ViaCyte, which has announced positive results from its cell-based therapy.
In addition to the current phase 1/2 programme for VX-880, Vertex plans to file an IND for its encapsulated islet cell programme in 2022.