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Edwin Kaswalder describes his thoughts and experiences during his child's participation in the Protégé clinical trial
In April of 2008, our healthy nine-year-old son, Gaspar, was diagnosed with type 1 diabetes. After his two days in the ICU and a week in the hospital, a new life began for all of us. Although we couldn't immediately grasp all its implications and were simultaneously dealing with our shaken world, we gave the situation a "think outside the box" approach. When the endocrinologist told us, "That's the way it is. Just focus on the controls and all will be fine," we asked whether the condition might be cured or attenuated if we acted quickly at the beginning. We were met with the usual answer: "There's nothing you can do. Just focus on the controls."
Although we are very happy with our endocrinologist, we did some research on our own to learn about the leading edge of diabetes research. Much to our surprise, there were hundreds of research investigations into alleviating diabetes or making controls more comfortable. Only a few, however, focused on the root cause of the problem and were about to start phase II or phase III of a clinical trial. These were the trials for teplizumab (Macrogenics Protégé-still recruiting, and Abate-no longer enrolling), otelixizumab (from Tolerx), and Diamyd. All of them aim to stop the self-immune attack, although by different means, and to preserve the remaining islet cells that are still producing insulin.
The age criteria left Gaspar outside the otelixizumab trial. His age was fine for the Diamyd trial, which was in a phase II in Sweden, but our inquiries were never answered. Then we came across a registered nurse testimonial for teplizumab on DiabetesHealth.com. Because Gaspar qualified by age, we deepened our research, looking for the original papers in medical journals and other testimonials. Eventually we set up briefing and screening meetings for our son.
We learned that some rash, nausea, and other minor nuisances may occur in some patients throughout the infusion itself, but that patients are carefully monitored day by day. If anything seems out of line, the treatment is interrupted. We found no reports of long-term horrible effects (not even in patients who were treated from 2000 to 2001).
Knowing the damage that uncontrolled diabetes can do, we decided to go ahead. We believe our son will stand a better chance for any future treatment with some of his islets preserved, rather than none.
The treatment started at the Research Institute in Dallas, Texas, where Gaspar had two sets of infusions of teplizumab, six months apart. We felt lucky to be there because the staff went out of their way to make us feel completely comfortable and to keep us well informed. They answered our questions (the tasteful and the distasteful ones) and volunteered information that we did not know to ask about. Throughout the briefings, infusions, and controls, Dr. Aronoff and his staff showed us first-hand what compassionate care looks like.
We felt that the nurses and other staff were truly concerned about the discomfort our son felt when pricked. After all, he was not even three months out of the ICU. They worked hard to make Gaspar feel more comfortable, bringing him DVD movies and sugar-free candies, letting him surf the Web, and more. It was as if Gaspar had gained a group of additional grandmothers: Tanya, Liz, Terry, Gayle, Dion, Gracie, Lisa, and Marcus (well, that would be grandpa) were wonderful. And yet, we wondered constantly whether it was worth it to have our son go through all that. Now, a year after the diagnosis, I'll let the numbers answer that question.
When Gaspar was diagnosed with type 1, his A1c was 12.1%. We put him on a very tough diet with minimum carbs, and two months later (just before the first infusion), his A1c was down to 8.5%. But the pediatrician told us that without carbs, his growth might suffer. So we prioritized food over figures (as long as they didn't go too wrong) and developed another plan.
We allow Gaspar to eat anything he wants, although with a few limitations. Among them, he needs to eat every three hours, after making a control and a fast insulin adjustment for what he plans to eat next, taking into account his last meal if necessary. We do not limit the number of grams of carbs per day, but we give him nutritious carbs, not plain sugars. With no other dietary precautions than those, his A1c was down to 7.1% three months after the first set of infusions. Three months after that, it was down to 6.5%.
The second set of infusions was followed by a long vacation. I am sorry to report that none of us ate well during that trip-we all ate a lot of junk food. We worried that Gaspar's A1c would go through the roof, but two months after the second set of infusions, it was only 6.7%.
Back at home, we returned to the original meal plan. Now Gaspar's 30-day blood glucose average is 126. His 90-day average is 139 (which equates to an A1c of 6.6%), with no sweat. During the last 90 days, we averaged 4.3 BG tests, two basal insulin shots, and 2.5 bolus insulin shots per day. We tested Gaspar's A1c in mid-June, and it was 6.8% (against the 6.6% that the 90-day BG average would give). This is well within the ADA recommended A1c of 8% for children from ages six to 12, and it was achieved with no food restrictions!
And what about lows? We haven't exactly relaxed our guard, but we have sort of forgotten about them. Before the treatment, our son's school called an average of twice a week to report that he was low. But after the first set of infusions, they never had to call again. On only one occasion, during our vacation, was there a very, very bad low (BG of 26 mg/dl). It was a day with lots of activity, some food, and overlaps of slow and fast insulin. We decided that from then on, when more than 10 units of fast acting are needed, we will give it in two stages; for example, six or seven units now and the remainder one hour later. Other than that one instance, however, lows have disappeared off the radar.
Our son's insulin dosing has stayed the same for the last seven or eight months: 22 to 24 units of slow acting NPH per day and the normal fast-acting correction (1 unit for every 15 grams of carbs to lower his BG to 50 mg/dl). To monitor BG peaks, we measure an average of four to five times a day. Over the past three months, totaling 382 measurements, we had 62 in the 200 to 300 mg/dl range, 15 in the 300 to 400 range, and six above 400, topping out at 440. For lows, 19 measurements were below 50 (normally before a meal), but during only two of them did our son feel a bit shaky. We give him additional glucose on those occasions.
Is there a downside? Is it just a honeymoon?
We cannot help worrying about the potential for unforeseen effects from the treatment. Is our son's immune system weaker? So far, he has gotten only one huge cold. Like most kids, he resents having to wear a jacket in winter, plays with cats and dogs, and crawls in the mud. None of it seems to make a difference in his health.
What will happen in the future? I monitor the news each and every day, afraid I'll learn some unpleasant news, but it hasn't happened so far. I haven't heard of anyone having a really bad -and permanent- outcome from teplizumab. But I will probably have these fears for years to come, no matter what happens.
Is our son honeymooning? Although we don't have any experience with honeymoons, some people describe the treatment as an "extended honeymoon." If that's the case, we can only hope that it lasts as long as possible.
Questions I can't answer
Is this the way to go? Should other parents do it? That's a decision that each person must make individually. The factors that I've weighted as important may be the very ones that someone else might see as unimportant. And the worst part, when you are an adult making this decision for a child, is that you are not the one deprived of the benefits or facing the consequences of participating in a research trial
All I can say is that we try to keep our hopes realistic and just be thankful for each day that things go well. When we hear about other parents' difficulty keeping their children's numbers down, we know that we are blessed. We have a very easy time controlling Gaspar's blood glucose. He has a decent A1c and BG average, and we don't have to worry much about him going hypoglycemic. Best of all, we don't have to curtail his activities or his food intake. He is allowed to have a childhood.
http://clinicaltrials.gov/ct2/show/NCT00378508 - 8 to 30 years of age, 4 to 12 months after diagnosis.
http://clinicaltrials.gov/ct2/show/NCT00385697 - 8 to 35 years of age, up to 12 weeks after diagnosis.
http://www.protegediabetes.org/ - Protégé trial website.
http://content.nejm.org/cgi/content/full/346/22/1692 - New England Journal of Medicine Teplizumab article, year 2002.
http://diabetes.diabetesjournals.org/cgi/content/full/54/6/1763 - NCBI Teplizumab article, year 2005.
http://www.researchdallas.com/ - Research Institute of Dallas. Yes, I'm happy with them.
http://www.diabeteshealth.com/read/2008/07/31/5849/letter-of-the-week-any-more-honeymooners-out-there/ - RN Denise testimonial II.
http://www.abatetrial.org/ - Abate trial website - No longer enrolling.
http://talk.news-medical.net/profiles/blogs/the-release-of-drugs-to-treat - Denise's blog with some updates on how her son's doing.
Edwin Kaswalder is a consultant for several oil services companies. He and his wife, Veronica, have two children. Until recently, when the family settled in South America, they lived in several countries. Edwin blogs about his son's diabetes and treatments at teplizumabdallas.blogspot.com.
Edwin Kaswalder is not a medical doctor. This article neither advises a particular course of action nor endorses any drug or institution.
8 comments - Aug 3, 2009