When 16-year-old Samantha Coon started losing her appetite a few months ago, no one realized how serious it would become.
"I just went downhill fast."
"She just got sicker and sicker, and we couldn't figure out exactly what was going wrong."
Samantha now knows she has type-one diabetes -- T-cells in the pancreas destroy beta cells that produce insulin.
"They told me I would be on, taking shots, pretty much for the rest of my life. I'm not afraid of needles, so shooting myself with needles isn't that hard," said Samantha Coon.
Now, in a clinical trial, patients in their first 100 days of type one diabetes -- who are still producing some insulin -- get different kinds of shots -- intramuscular injections of a drug called Alefacept
"What we're trying to do is see if we can delay the progression of type 1 diabetes."
Alefacept has been approved to reduce destructive
T-cell activity in another auto-immune disorder called plaque psoriasis. Researchers believe it could have a similar effect on diabetes.
"If we can provide a medication or some kind of intervention that will prevent those cells from being destroyed, then the hope is that these patients will continue being able to produce insulin on their own and not have diabetes," said Eric Felner, M.D., M.S.C.R., Associate Professor of Pediatrics Emory University School of Medicine.
For Samantha, it's too soon to know if the treatment's working, but she knows no matter what, she'll be OK.
"I'm still me."
For more information on other series produced by Ivanhoe Broadcast News contact John Cherry at (407) 691-1500, firstname.lastname@example.org.
MEDICAL BREAKTHROUGHS - RESEARCH SUMMARY:
BACKGROUND: Type 1 diabetes, once known as juvenile diabetes or insulin-dependent diabetes, is a chronic condition in which the pancreas does not produce insulin -- a hormone needed to allow sugar (glucose) to enter cells to produce energy. When first diagnosed, it is believed that some cells in the pancreas are still viable and continue to produce insulin, but production lessens over time. Various factors may contribute to type 1 diabetes including genetics and exposure to certain viruses. Although type 1 diabetes typically appears during adolescence, it can develop at any age. Despite active research, type 1 diabetes has no cure, although it can be managed. With proper treatment, people who have type 1 diabetes can expect to live longer, healthier lives than in the past. (SOURCE: Mayo Clinic, www.choa.org)
SYMPTOMS: Type 1 diabetes signs and symptoms can come on quickly and may include:
• Increased thirst and frequent urination: As excess sugar builds up in the bloodstream, fluid is pulled from tissues. This may leave you thirsty. As a result, you may drink and urinate more than usual.
• Extreme hunger: Without enough insulin to move sugar into your cells, your muscles and organs become depleted of energy. This triggers intense hunger that may persist even after you eat.
• Weight loss: Despite eating more than usual to relieve hunger, you may lose weight -- sometimes rapidly.
• Fatigue: If your cells are deprived of sugar, you may become tired and irritable.
• Blurred vision: If your blood sugar level is too high, fluid may be pulled from your tissues -- including the lenses of your eyes. This may affect your ability to focus clearly. (SOURCE: Mayo Clinic)
ALEFACEPT: Alefacept is a protein that reduces specific actions of the immune system and may increase risks of developing or reactivating a chronic infection. In a clinical trial, patients in their first 100 days of having type 1 diabetes receive injections of the drug. Alefacept reduces levels of white blood cells in the body. If the levels fall too low, treatment may need to be withheld or discontinued. Alefacept may increase the risk of developing cancer. Patients should contact their doctors if symptoms of an infection develop such as fever, chills, sore throat, coughing, or burning with urination. They should not use alefacept if they had a previous allergic reaction to it; have heart or blood vessel problems; have cancer or a history of cancer; have an infection or history of chronic infection; are HIV positive; or are taking immunosuppressive medications or receiving phototherapy. (SOURCE: www.drugs.com)
FOR MORE INFORMATION, PLEASE CONTACT:
Assistant Director, Media Relations
Emory Health Sciences Communications
THE FOLLOWING IS AN IN-DEPTH INTERVIEW WITH THE DOCTOR FROM THE STORY ABOVE:
Eric Felner, MD, MSCR, Associate Professor of Pediatrics at Emory University School of Medicine, talks about a new therapy being used for type 1 diabetes.
Tell me about this trial, what are you trying to do with this particular drug?
Dr. Felner: What we’re trying to do is see if we can delay the progression of Type I diabetes. Type 1 Diabetes is a progressive, autoimmune destruction of the islet cells resulting in failure of the body to produce insulin. If we can provide a medication or some kind of intervention that will prevent those cells from being destroyed then hopefully, these patients will have the ability to produce their own insulin and not progress to complete insulin deficiency.
What does the drug involve and what does it do?
Dr. Felner: Alefacept is currently approved for use in plaque psoriasis and it seems to be effective for that condition. Psoriasis is thought to be an autoimmune disorder in which excessive reproduction of skin cells is a result of inappropriate activation of infection-fighting T-cells. The fact that Alefecept works in Psoriasis, possibly an autoimmune-related disorder involving T-cells, the hypothesis is that Alefecept, may also work in Type 1 Diabetes. Alefecept has two likely mechanisms of action. The first is that it inhibits a subset of T-cels known as CD-4 (helper) and CD-8 (killer) T-cells. By inhibiting these abnormal, dysregulated T-cells, which are likely causing the destruction of the islet cells in those with Type 1 Diabetes, in theory, the islet cell destruction can be prevented, or at least limited. This is probably the main mechanism. The other possible mechanism is it Alefecept may simply cause T-cell death.
They used to call it juvenile diabetes but it’s really just Type I now isn’t is, or is it?
Dr. Felner: It was probably a bad name way back when when they started using it but Type I diabetes is an autoimmune destruction of the islet cells that can occur at any age. For the majority of patients with Type I diabetes they’re less than twenty years of age but there are a small percentage of Type 1 diabetics who are diagnosed in their twenties, thirties and even possibly older. In general, it’s considered a juvenile cause because prior to this obesity epidemic more than 95% of children with newly diagnosed diabetes had Type I diabetes.
What happens in Type I and how devastating can it be?
Dr. Felner: What happens is this autoimmune destruction occurs where these islet cells are no longer able to produce insulin. The body requires insulin to utilize glucose (or carbohydrate) for energy. If you are unable to utilize carbohydrates as an energy source, you will have to use an alternative energy source such as fats. Using fats as your primary energy source will ultimately cause a person to develop significant breakdown products of fats (known as ketones) which will result in a significant drop in the body’s pH (acidosis). Acidosis, if untreated with insulin, will ultimately result in multi-organ failure and death.
Has the approach changed to treating these kids, you guys have a couple of different drug trials going? Did it used to be to try to control the disease and now you’re trying to stop it?
Dr. Felner: That’s the main idea behind all of these studies. Improved methods for caring for those with diabetes has improved tremendously over the past 20 years with technology, smaller syringes, insulin pumps, and different insulin preparations. Prior to these studies on immunotherapy, islet cell transplantation was felt to be the key to eliminating this disease. Those studies are still on-going, and some have been successful, but it has not proven to be definitive. With these immunomodulation studies we are hopeful that we can determine if a single drug, or combination of drugs can prevent the autoimmune destruction of islet cells.
For patients like Sam what could this mean, if this works the way you are hoping what could this particular drug trial do or mean for her?
Dr. Felner: If this single drug were to work all by itself the thought and the hope is that her cells will continue to be able to produce insulin and she would not require insulin for treatment for diabetes. Her body, specifically her own islet cells would be capable of producing enough insulin to prevent hyperglycemia.
Is it too soon to tell or do you think it’s going to take a combination of drugs in addition to this one?
Dr. Felner: Previous human and animal studies with other immunomodulating drugs would suggest that a single or multiple drug regimen may prevent the islet cell destruction in these newly diagnosed patients with diabetes. We are not informed of the results until all subjects in the country have completed the study and their data evaluated by the monitoring board. Therefore, it appears too spoon to tell what the results will be.
How many different drugs are you working on, I know there are other trials with different types of drugs.
Dr. Felner: This is the third drug that we’ve been involved in. The other two drugs are: anti-thymoglobulin and alpha-1-antitrypsin. The alpha-1 study (known as RETAIN) is closed as the study group monitors the safety and efficacy data in both the adult and pediatric subjects who received the medications. Assuming there are no safety concerns, the trial will open in the next 2-3 months as a randomized trial similar to the Alefecept (T1DAL) trial. The antithymoglobulin (START) trial has been closed since the summer but follow-up will continue for these subjects for 2 years after receiving the initial treatment.
Do you envision maybe within your lifetime that you might unlock the mystery and be able to solve this? I understand you have a personal interest in this as well.
Dr. Felner: I have a personal interest because I have had autoimmune type 1 diabetes for almost thirty five years. I have witnessed not only through the patients I have cared for but also for myself how difficult it is to manage diabetes as a child, adolescent, young adult, and adult on a daily basis. I am excited to be a part of this research endeavor and would be thrilled to think our research team was involved in discovering a cure. Despite the fact that my diabetes, at least at this point, appears irreversible, I am encouraged by our patients and families to look for a cure.
Does your gut tell you that this might be one that has some real potential?
Dr. Felner: I think the way that it works for psoriasis and the similar mechanisms with the T-cells make it a very favorable drug. Other immunomodulating drugs have been studied and we have not seen an overwhelming amount of success in significantly preventing islet cell destruction. With the mechanism of action and effect in plaque Psoriasis I believe it’s the most promising drug available right now.
How far out is this trial going to go?
Dr. Felner: The patients receive the medication for twelve weeks, get a twelve week break, and then receive it for another twelve weeks. We will follow the patients for up to two years after the initial injection, and measure how well their own islet cells can produce insulin.