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In the current issue of Journal of American Medical Association (JAMA. 2007;297) Voltarelli and colleagues publish promising findings in a small group of patients of sample size 15, who underwent autologous hematopoietic stem cell transplantation (AHSCT) within 6 weeks of receiving a type 1 DM diagnosis. The authors report that patients who underwent AHSCT had increased beta cell function as evidenced by an increment in C-peptide levels and by low levels of hemoglobin A1C despite very low doses or frank discontinuation of insulin therapy.

Type 1 diabetes is caused by selective immunological destruction of pancreatic beta cells which results in insulin deficiency. Type 1 DM warrants regular and strict monitoring of blood glucose and mainstay of treatment being externally administered insulin.



This new study has been conducted by Northwestern university, Chicago and designed by Dr Richard Burt. Here they used patients own stem cells through AHSCT with high dose immunosuppression in newly diagnosed Type 1 DM. This study was carried out by Voltarelli and colleagues in University of Sao Paulo, Brazil. The results were provocative in the sense they concluded, High-dose immunosuppression and AHST were performed with acceptable toxicity. With AHST, beta cell function was increased in all but 1 patient and induced prolonged insulin independence in the majority of the patients.

One limitation of the study is an absence of a control group. There need to be few more controlled studies in this direction.

Ethical Objections :

This study has also spurred ethical objections for putting Brazilian children under risk.

The risks of the protocol were high. The risks involved with the new technique were more when weighed against the benefits. Currently the Type-1 diabetics lead a normal life with the long-term insulin therapy and regular monitoring. So, is it justifiable to go on with the study before checking on the unnecessary risks? Mind that we are not treating an incurable cancer here.

When adult patients were available why the researchers included pediatric age group (age group limits 14 to 31) is uncertain. The original protocol had the cut off age of 18.

Why the study was carried out in Brazil rather than in US, when plenty of diabetic experts are available in US itself. Was it because it was difficult to get the permissions to carry out this risky protocol?

Another minor concern is “what was the need to take newly diagnosed Type 1 diabetics”. An editorial in JAMA same issue by Jay S Skyler has given explanation for enrolling newly diagnosed patients. Because when the target of therapy with AHSCT is the type 1 DM disease process leading to beta cell destruction, this intervention should be applied when sufficient beta cells are available for salvage (i.e, relatively early in the course of the disease).



The article link Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Newly Diagnosed Type 1 Diabetes Mellitus



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