Breakthrough T1D Research
in General
In the News
April 2013
Breakthrough T1D is the only global organization with a strategic plan to deliver a continuous flow of life-changing therapies and, ultimately, a cure for T1D. Past Breakthrough T1D investments have yielded significant advances and taught us that progress towards our goals will be made incrementally over time. These advances and breakthroughs are what move scientific discoveries from the laboratory to clinical trials and ultimately into the hands of patients. As the leader in global T1D research and development, Breakthrough T1D-supported T1D research progress is routinely reported in the media. Here is a summary of Breakthrough T1D-supported T1D research progress reported by the media in April 2013.
Breakthrough T1D Research and Development Strategy
Foundations Fuel Biotech Ventures
Non-profit, patient-focused foundations are becoming an increasingly visible force in early-stage biotech research and development. Many are no longer just handing out grants to academic scientists and hoping the discoveries they fund someday make it into clinical practice. Today, many are being far more proactive, providing capital for entrepreneurs to take discoveries out of academia and translate them into commercial opportunities. At the forefront of this trend since launching its industry-partnering program in 2004, Breakthrough T1D has funded $110 million at more than 40 partnered companies to translate early-stage science in T1D development opportunities. Now other foundations are following Breakthrough T1D’s lead recognizing the benefit of accelerating potential products into the hands of the patients they serve.
Breakthrough T1D Immune Therapies Research
Immunomodulatory Drugs Ineffective for New-Onset T1D
Reports were recently published on the findings of two independent phase 2 randomized, placebo-controlled clinical trials, supported in part by Breakthrough T1D, that assessed the efficacy of interleukin-1 beta inhibition as a means to halt deterioration of beta-cell function after the onset of T1D. The inhibition of interleukin-1 beta with either canakinumab (Ilaris, Novartis) or anakinra (Kineret, Sobi/Savient Pharmaceuticals) did not slow the decline of beta-cell function in new-onset T1D, according to data published in The Lancet. Canakinumab and anakinra were safe but were not effective as single immunomodulatory drugs in recent-onset T1D. Interleukin-1 blockade might be more effective in combination with treatments that target adaptive immunity in organ-specific autoimmune disorders.
New Study Investigates Cancer Drug as a Treatment for T1D
A new study funded by Breakthrough T1D will carry out research to see if an existing medication that is used to treat cancer may help to prevent cells in the pancreas from being destroyed in newly diagnosed patients with T1D. The drug which will be tested is called aldesleukin (Proleukin, Prometheus Therapeutics). The drug is a man-made form of a protein, which is produced by our body, called interleukin-2. Aldesleukin is currently used as a chemotherapy treatment for specific types of kidney and skin cancers. The research will be carried out at Addenbrooke’s Hospital, Cambridge, UK. Forty newly diagnosed patients with T1D that have agreed to take part in the trial will take a much smaller doses of the medication than is used for chemotherapy. The researchers hope that a small dose of the drug will help the immune system to regulate rogue T-cells and prevent them from incorrectly targeting and attacking beta cells in the pancreas.
Breakthrough T1D Beta Cell Encapsulation Research
BioHub Encapsulation Project Launched for T1D
JDRF-funded researchers at the University of Miami’s Diabetes Research Institute announced the launch of a project to develop a biologically-engineered, implantable encapsulated beta cell source that would mimic the pancreas in creating the precise amount of insulin the body would need at any given moment, thereby controlling an individual’s blood sugar levels. This project is one of several concepts that Breakthrough T1D is supporting to develop implantable, encapsulated beta cells as a cure for individuals with T1D.
Breakthrough T1D Artificial Pancreas Project Research
Companies Racing Towards Artificial Pancreas
Despite all the advances in medical devices for individuals with T1D, nothing resembling an artificial pancreas has made its way to the market stateside, but Breakthrough T1D-supported companies like Medtronic, Johnson & Johnson (Animas) and Tandem are working to push the technology forward. And while those devices promise to ease the lives of individuals with T1D, the gold standard remains a full-fledged artificial pancreas: an interconnected system of devices that can continuously monitor a person’s blood sugar levels and dispense insulin as needed – 24/7. Animas leads the way on that front, working through a second feasibility study of its external device, but Tandem and Medtronic have also partnered with Breakthrough T1D to advance their technologies toward realizing a fully automated monitor/pump combo.
Workshop Advances Innovation Towards an Artificial Pancreas
JDRF, NIH, and FDA recently convened a workshop with experts in the field of artificial pancreas research and development to review progress and address hurdles to continued innovation. One roundtable discussion to conclude the meeting was notable, featuring many of the biggest names in AP research debating what the future holds for closed-loop delivery. Breakthrough T1D’s Dr. Aaron Kowalski “threw down the gauntlet” and urged all of industry to get involved in this arena and conveyed his hopes for more rapid progress, including on the regulatory front. Indeed, this is a major funding initiative for Breakthrough T1D this year, lucky for individuals with T1D. Dr. Kowalski put forward hope that any company that currently sells a pump will make a firm commitment to commercializing it with a predictive low glucose suspend feature in the immediate future.
Breakthrough T1D Glucose Control Drugs Research
Diabetes Research at University of Buffalo Boosted by $2.1M
Two new grants totaling $2.1 million will advance research into T1D at the University at Buffalo. Dr. Paresh Dandona, a SUNY distinguished professor of medicine, received the grants to further his work into “liraglutide,” a drug to be tested in addition to insulin for treating T1D. The research is exploring how the addition of liraglutide (also known as Victoza, Novo Nordisk, and currently sold for treating T2D) might change the course of treatment for T1D. Dondona received a five-year, $1.4 million grant from the National Institutes for Health (NIH); and a $712,000 grant from Breakthrough T1D. Dondona and his team will use the funds to conduct the first placebo-controlled, prospective, randomized study to determine the effect of adding liraglutide to insulin. The 56-week study of 90 people with T1D also will assess whether the drug suppresses the blood-sugar-raising hormone glucagon following meals and helps improve overall blood glucose control.
Breakthrough T1D Prevention Research
Size of an Individual’s Pancreas Linked to T1D Risk
According to a Breakthrough T1D-funded study at the University of Florida, the number of insulin-producing beta cells in the pancreas could indicate a risk of diabetes. Dr. Martha Campbell-Thompson, Professor of Pathology at the University of Florida, weighed 164 pancreases taken from deceased donors as part of the Breakthrough T1D nPOD program (Network for Pancreatic Organ Donors with Diabetes). She found that those at a high risk of developing T1D weighed 25 percent less than normal. “This implies that even before one becomes diagnosed with T1D, you may have fewer insulin producing beta cells,” Campbell-Thompson said. “This could be happening many years before signs of diabetes occur.” To learn more about nPOD, go to: www.jdrfnpod.org.
Research Hope for T1D
Gemma Morton, aged 21, from Wincobank in the UK, decided to take part in the new diabetes research program at the city’s Northern General Hospital after seeing her dad Ian, aged 53, suffer from hypoglycemic episodes, brought on by T1D. The study, called TrialNet, is partly funded by Breakthrough T1D and involves relatives of people who have the condition undergoing a blood test and also providing some family history. Doctors hope the results could protect future generations by letting people know they are at risk of T1D years before symptoms appear. Gemma said: “Diabetes can be pretty scary and it’s only through my own knowledge of my dad’s condition that I’ve been able to pull him through some life-threatening situations. Once a person’s risk for T1D is determined, they may be eligible for various clinical studies investigating ways to prevent people at risk from progressing to a clinical diagnosis of T1D. To learn more about TrialNet, go to: www.diabetestrialnet.org.