In a stem cell trial by Vertex Pharmaceuticals of Boston, 10 out of 12 patients suffering from severe Type 1 Diabetes have been found cured of the illness. Just around a year ago to today, 10 of the patients no longer needed any insulin, whilst 2 need much less than before.
The study included transforming stem cells into pancreatic islets, and injecting them into the liver, where they take residence, and can produce insulin on their own. Now a year later, 83% of people subjected to the treatment, no longer need any additional insulin according to Vertex.
How does it work? The injected, lab-grown islets cells attach themselves to a liver vein. Which provides many advantages over the traditional under-the-skin injection. That is because switching off excessive liver glucose production is the main function of glucose. With this new treatment, they are targeted faster, and close to where they are created, therefore better correcting sugar levels.
The treatment has been improving, and within the first three months glucose levels were already well under control. Ever since then, throughout the course of a year most participants were able to decrease their insulin intake lower and lower until they no longer needed any.
Although this outlook seems hopeful, there is a downside; participants will need to take immunosuppressants for the rest of their lives. This stops their bodies from attacking the transplanted cells. However, it puts them at an elevated risk of infections, diseases, and certain types of cancer. Other, less severe side effects, include upset stomachs, diarrhea, headaches, and nausea, most of which showed up in over half the participants. Two of the 14 original members of the trial died, though inspection showed it was unrelated to the trial.
It is difficult to estimate when, and even IF this treatment might become widespread and accessible to the public. Vertex, like other drug companies, declined to announce the treatment’s cost before the Food and Drug Administration approves it. Therefore, its accessibility is still unknown. A Vertex spokeswoman also mentioned that they only have data specific to the subjects they tried it on and cannot say if this treatment is viable for all Type 1 diabetics.
We can estimate it might be more accessible than current islet transplants from dead donors, at least price wise, and has shown similarity in effectiveness.
Even though we do not know the cost, we do know that in this current trial, after a failed test of an immunosuppressant-free version of the treatment, the company now plans to watch the subjects for 10 years total, separated into an initial five-year follow-up, then a five-year extension study.
This gives a better general idea of how long we may need to wait before knowing the true viability of the treatment.
While the uncertainty around it may sound negative, the science community is optimistic that this may mean that an insulin-free life may be possible for diabetics in the near future.