While type 2 diabetes is largely preventable, type 1 diabetes is a destructive autoimmune disease that was once thought to be incurable. “Once” is the key word here, because a new and indisputable form of stem cell therapy should be able to cure the disease once and for all.
Based on turning small pieces of mature skin tissue into beta cells in the pancreas — which produce the illusory hormone insulin in people with diabetes — the treatment bypasses genetic mutations that cause the immune system to attack these cells and cause disease.
Diabetes, especially type 1, severely affects quality of life and, if not treated with care, can lead to serious complications such as leg amputation and premature death.
In addition, the US healthcare industry costs approximately $85,000 per patient for lifetime care, a tremendous burden that, if eliminated, could save the entire sector hundreds of millions.
Researchers at the Salk Institute for Biological Research, pioneers in all forms of biology, have pioneered a new way to make pancreatic beta cells for this type 1 treatment – and now the only thing preventing it from being in hospitals setting is safety try outs on humans.
“Stem cells are a very promising approach for developing many cell therapies, including better management of type 1 diabetes,” Salk professor Juan Carlos Ispiua Belmonte, lead author of the article, told Salk Press.
“This method of producing large numbers of safe and functional beta cells is an important step forward.”
This reservation is very important because, although the concept of stem cell therapy for the treatment of diabetes was mentioned in an early 2007 report, the challenges with pancreatic beta-cell production have not been overcome.
Growing a cure.
Prior to using stem cells, patients could receive islet cells transplanted from a donor with a fully functional insulin system, but the small number of donors would make this treatment burn slowly, even if it worked.
Existing stem cell methods are successful in converting only about 10-40% of human pluripotent stem cells (PSCs) into pancreatic beta cells. Moreover, the pancreatic beta cells of PSCs tend to be heterogeneous and contain undesired cell types that can interfere with or impair the maturation and desired function of pancreatic beta cells.
Previous methods of using PSCs to generate beta cells have also sometimes led to dysfunction or, in some cases, formation of (sometimes large) teratomas or cysts.
“For beta cell therapy to be a viable option for patients, it is important to facilitate the production of these cells,” said co-author Haisong Liu, a former member of Dr. Belmonte, across from Salk. “We have to find a way to optimize the process.”
And that’s what they do. The use of three-dimensional Petri dishes allowed cells to interact and grow in an environment similar to their natural environment, and within two weeks of being transplanted into diabetic mice, their blood sugar dropped to normal levels, as seen in non-diabetics mice.
To test the safety and effectiveness of the resulting beta cells, the team transplanted them at various stages and monitored their effects. At the highest growth stage, the cells did not form cysts even 20 weeks after implantation and showed considerable functionality in vivo and in vitro.
Three-dimensional stem cell production methods are being further refined at Salk with the only study remaining on disabled people and FDA approval. At least one pharmaceutical company has started testing stem cell therapy for type 1 diabetes this year, so the days of the disease are numbered.