This study, if it is far from perfect, nevertheless brings us closer to the day when these patients will no longer need insulin doses to control their blood sugar, commented Doctor Rémi Rabasa-Lhoret, an expert in this disease at the Montreal Clinical Research Institute who has gone to a hair to participate in these works.
“It’s a big step in the right direction,” he said.
Fourteen patients were recruited for the purpose of this study. Two died along the way, one of cryptococcal meningitis and the other of pre-existing dementia. The need for exogenous insulin was reduced or eliminated in the remaining twelve subjects, to the point where 83 % of them no longer needed insulin twelve months after the start of treatment.
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“Although the elimination of the need for exogenous insulin is desirable, the results of this study show the clinical advantages of restoration of the function of pancreatic islets, even in the absence of complete elimination of insulin treatment,” write the authors.
There has also been a complete absence of serious hypoglycemic events in the twelve participants who received a complete dose of therapy and completed a follow -up of at least twelve months.
We always keep this hope, said Doctor Rabasa-Lhoret, “to succeed in healing type 1 diabetes by allowing people to find the cells that produce insulin”. For the moment, this option is only accessible to a tiny number of patients each year and implies a transplant either of the entire pancreas, or cells producing insulin – which, of course, requires the recent death of a compatible donor.
The patient will then have to be tied to an immunosuppression regime to prevent their immune system from rejecting the transplant.
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The new study comes up against the same obstacle, since the stem cells used came from foreign donors and not from the patient himself. On this subject, most of the side effects observed have been light or moderate, ensure the authors of the study, and were essentially attributable to the required immunosuppressive therapy.
“The main drawback (stem cell therapy) is that an anti-rejection treatment must still be taken,” said Doctor Rabasa-Lhoret. And for type 1 diabetes, we have other solutions. ”
We must keep in mind, he recalled, that an immunosuppression treatment is not commonplace since it has a significant risk of certain infections and even certain cancers. Thus, even if this is not mentioned in the study, it cannot be excluded that cryptococcal meningitis which has won one of the participants is associated with this immunosuppression, added Doctor Rabasa-Lhoret.
That being said, the new study takes “an absolutely essential step to go towards something without immunosuppression,” he said.
A possible treatment without immunosuppression will involve the transformation of stem cells withdrawn into the patients into cells capable of producing insulin.
However, it is easier to say than to do, warned Doctor Rabasa-Lhoret, and the feat has now been carried out only in a single patient, as part of a study whose results have not yet been published.
When you take the stem cells of a patient to transform them into cells that produce insulin, he explained, “we can introduce small differences that will make the organism begin not to recognize them”, which would cause an undesirable and potentially dangerous immune reaction.
We will also have to ensure that these stem cells do not cause other medium or long-term problems, for example by turning into cancer cells, he said.
“The Holy Grail that patients are looking for is a healing,” said Doctor Rabasa-Lhoret.
But with the solutions that we have at the moment to manage type 1 diabetes, he continued, the disadvantages of immunosuppression become “not bad less interesting”.
There will necessarily be people who will not be able to take their insulin or who will have other problems, recalled Doctor Rabasa-Lhoret, “and there will be a small market with these people, but the next step is really the absence of immunosuppression”.
“We now know that it is doable,” he concluded. It is not in mice, it is not in rats, it is not in pig, it is in humans. We know that it is feasible to graft cells and produce insulin in patients, which is quite magical anyway. ”
The treatment under trial within the framework of this phase 1/2 study was developed by the Vertex biotechnology company, which has already been talked about after putting a treatment on the market – also based on stem cells – against cystic fibrosis and which, for the first time, was attacked in the deep causes of the disease and not only to its symptoms.
