A promising cure for a currently incurable disease - type 1 diabetes - is being developed at City of Hope, and patients can now apply for the Phase 1 trial.
“There are only a handful of clinical trials in the world right now aiming for a functional cure, and this is one of them,” said Ping H. Wang, M.D., professor and chair of the Department of Diabetes, Endocrinology & Metabolism. “If you have been recently diagnosed, this is an opportunity to be involved in a trial that might eventually lead to a cure.”
At the present time, the main treatment for people with type 1 diabetes is insulin, which can control blood sugar levels and improve quality of life and longevity. However, the disease remains and can progress, sometimes leading to serious complications, such as nerve damage, blindness, heart disease and kidney failure.
This trial takes a completely different approach with a personalized immunotherapy called PlpepTolDC, designed to address the underlying cause of the disease.
An Autoimmune Disease
Unlike type 2 diabetes, which may be caused by lifestyle issues, such as obesity and inactivity, type 1 diabetes is an autoimmune disease.
Our immune systems are designed to attack invading organisms, such as bacteria and viruses. But in an autoimmune disease, something goes wrong and the body attacks its own cells.
“In the early stages, when the pancreatic islets are not totally gone, you still have a reasonable number of insulin-producing cells left,” said Wang. “And if we can then stop the immune attack, we have a chance to preserve the remaining functioning beta cells and may even allow some of those cells to grow back.”
The trial treatment, PlpepTolDC, is designed to stop the autoimmune attack on the pancreatic islets, and should be more effective for those recently diagnosed. If successful, progression of the disease will be halted, the remaining beta cells will produce insulin and the course of disease can be reversed.
A 'Reverse' Vaccine
PlpepTolDC is a type of “reverse” vaccine. Unlike, say, the COVID-19 vaccine, which teaches the immune system to destroy a particular invader, PLpepTolDC does the opposite — it trains the immune system to stop destroying beta cells.
“There’s a specialized part of the immune system called the dendritic cells, which play a key role in triggering the attack on the beta cells,” explained Wang. “We reengineer these cells in the laboratory to make them immune-tolerant so that they can coexist in peace with the islet cells.”
This highly personalized form of immunotherapy is made from the patient’s own cells. In a type of blood draw called leukapheresis, the white cells are harvested and the blood reinfused into the body. The process, which is similar to the way platelets are donated, takes about four hours — and during that time you can binge on a favorite TV show, watch a movie or listen to music.
Reengineering the cells, which involves combining them with vitamin D3 and a beta cell protein, takes about two months. Once the PlpepTolDC is ready, the patient will receive two doses, separated by at least 28 days. The health of each patient and the efficacy of the treatment will be monitored for two years — every three months during the first year and every six months during the second.
Patients will continue to take insulin during the trial. Depending on their response to the treatment, the insulin injections may be reduced or even eliminated.
Qualifying for the Trial
Only six people will be accepted into the City of Hope trial. You may qualify if you:
• have been diagnosed with type 1 diabetes within the last one to four years
• are between the ages of 18 and 45
• have no other diabetes-related conditions
• use insulin
If you are interested in taking part in the trial, you can check the full list of inclusion criteria. For more information, contact a study coordinator at 866-44-ISLET (47538) or DL-TolDC@coh.org.
It is reassuring to note that this trial is built on the work of a study conducted in the Netherlands several years ago, which showed PlpepTolDC to be safe and well tolerated, with few side effects.
The Netherlands trial also showed some efficacy, though some subjects had been diagnosed as long as 10 years earlier and would have had fewer surviving beta cells than the patients who will be chosen for City of Hope's study.
The trial is sponsored by The Wanek Family Project for Type 1 Diabetes, and though it is limited to six subjects, there’s a huge team of City of Hope physicians and scientists behind it, including specialists in cell therapy preparation, quality control and patient monitoring.
“For this kind of trial,” said Wang, “it takes a village to move mountains, and we would like to invite qualified patients to join our village.”