Updated List of Practical Cure Research in Human Clinical Trials

Practical Cure projects in human trials as of June 2015 This is the semi-annual review of T1D research in human clinical trials. The review identified 361 open trials that focus on T1D and only 11 initiatives have the potential to deliver a Practical Cure. Over the three years that we have been tracking T1D projects in human trials, the story has remained more or less unchanged: there are very few Practical Cure projects. We strongly believe that more qualifying projects in human trials would significantly increase the chances of a successful outcome. Since the last update in November 2014, there has been only small changes in the Practical Cure project list. The number of qualifying initiatives has moved from 8 to 11, with the addition of two new projects and one returning. The returning project, ATG/GCSF,recently started Phase II trials, after over two years of inactivity. No project has been removed since November. The chart below lists the Practical Cure projects in human trials as of June 2015. A more detailed record of the Practical Cure research in human trials can be located on the clinicaltrials.gov website using the NCT numbers listed in the chart.

Glu : Lilly Plans to Take Locemia Solution’s Needle-free Glucagon to Market

Glu : Lilly Plans to Take Locemia Solution’s Needle-free Glucagon to Market

How we—you and T1D Exchange—played a role in advancing intranasal glucagon

We want to congratulate two of our member companies, Lilly and Locemia Solutions, on some exciting news!

Lilly has acquired Phase III Intranasal Glucagon from Locemia Solutions. Intranasal glucagon is a potential treatment for severe hypoglycemia in people with diabetes who use insulin and could be the first needle-free rescue treatment for severe hypoglycemia. (Learn more about severe hypoglycemia and intranasal glucagon below.)

This announcement highlights the important collaborations that are happening every day to improve outcomes in type 1 diabetes.

And you are part of this exciting collaboration. How? Our T1D Exchange community participated in advancing intranasal glucagon in the following ways:

Question of the Day

• In 2013, the Glu community answered a Question of the Day about the complexities of using injectable rescue glucagon and about severe hypoglycemia preparedness. Our community’s real-world experiences helped Locemia Solutions define the challenges of injectable glucagon in discussions with the FDA, which ultimately led to clinical studies of an intranasal form of glucagon.

T1D Exchange Clinic Expertise

• Locemia Solutions partnered with T1D Exchange to:
  • Design clinical protocols and conduct two multi-site clinical studies. Our highly qualified clinical investigators conducted these studies.
  • Recruit participants from the T1D Exchange Clinic Registry to participate in clinical studies.

“When you’re looking for that special population of people with diabetes, access to the expertise, and the investigators who can run the special procedures like that, T1D Exchange offers something that just is unavailable anywhere [else] in the world…” —Dr. Claude Piché, CEO of Locemia Solutions

 Collaborating for Improved Outcomes: A Case Study

The potential of bringing intranasal glucagon to market to relieve the burden of severe hypoglycemia has been a case study in partnership and collaboration, including:

• Initial funding from The Leona M. and Harry B. Helmsley Charitable Trust and Locemia Solutions
• Real-world evidence about complexities of the glucagon rescue kit from the Glu community.
• T1D Exchange works with Locemia Solutions to design and run phase III clinical studies and recruits participants from its Clinic Registry
• Lilly is now leveraging its global expertise and heritage in diabetes to bring this product to market.

Read more about this announcement from Lilly.

Background on Severe Hypo and Intranasal Glucagon

In type 1 diabetes, a severe low can cause confusion, seizures, or unconsciousness. It requires medical intervention right away from someone other than the person with T1D. It might be a spouse, parent, or friend. It can be a teacher, coach, or babysitter—most often, it’s from someone who has no medical training.

Today’s life-saving hypoglycemia rescue kit is complex. It contains a powdered form of glucagon that needs to be reconstituted into a liquid, drawn into a needle and injected in the person’s thigh. No easy task under the best of circumstances—and certainly not while a loved one or friend is seizing or unconscious.

Intranasal glucagon could be the first needle-free rescue treatment for severe hypoglycemia.

Intranasal glucagon uses a proprietary glucagon nasal powder formulation that is delivered in an emergency situation using a single-use, ready-to-use device. The caregiver presses a small plunger on the bottom of the device to release the glucagon as a puff in the nose, where the glucagon is absorbed in the nasal passages.

Congratulations to our members, Lilly and Locemia Solutions, and to the entire T1D Exchange patient community!

—Dana Ball
Co-founder and Executive Director, T1D Exchange

Diabetes News: First Patient in BioHub Pilot Trial No Longer Requires Insulin

Diabetes News: First Patient in BioHub Pilot Trial No Longer Requires Insulin

New Transplant Technique Restores Natural Insulin Production in Type 1 Diabetes

MIAMI, FL – September 9, 2015 – The Diabetes Research Institute (DRI), a Center of Excellence at the University of Miami Miller School of Medicine, announced today that the first patient in its clinical trial has been free from insulin injections in record time following the implantation of islet cells within a biological scaffold. The patient, Wendy Peacock, 43, from San Antonio, TX, underwent the minimally invasive procedure on August 18, 2015, and is now producing her own insulin naturally for the first time since being diagnosed with type 1 diabetes at age 17. In this pilot study, DRI researchers are testing a new transplant technique for insulin-producing cells, building upon decades of progress in clinical islet transplantation. This trial is an important first step toward the development of the DRI BioHub, a bioengineered mini-organ that mimics the native pancreas to restore natural insulin production in people with type 1 diabetes. 

“The first subject in our Phase I/II pilot BioHub trial is now completely off insulin with an excellent glucose profile. These are the best post-transplant results we've seen in an islet recipient," said Camillo Ricordi, MD, director of the DRI and the Stacy Joy Goodman Professor of Surgery, Distinguished Professor of Medicine, Professor of Biomedical Engineering, Microbiology and Immunology at the University of Miami Miller School. Dr. Ricordi also serves as director of the DRI’s Cell Transplant Center. “This was the first tissue engineered islet transplant using a ‘biodegradable scaffold’ implanted on the surface of the omentum. The technique has been designed to minimize the inflammatory reaction that is normally observed when islets are implanted in the liver or in other sites with immediate contact to the blood. If these results can be confirmed, this can be the beginning of a new era in islet transplantation. Our ultimate goal is to include additional technologies to prevent the need for life-long anti-rejection therapy," Dr. Ricordi added.

Diabetes tipo 1: identificado tratamento promissor | ALERT® ONLINE - PT

Diabetes tipo 1: identificado tratamento promissor

Estudo publicado na revista “Scientific Reports”

03 agosto 2015

A utilização de uma citoquina anti-inflamatória mostrou-se eficaz no tratamento da diabetes tipo 1, dá conta um estudo publicado na revista “Scientific Reports”.

A diabetes tipo 1 é uma doença crónica, na qual os pacientes necessitam de injeções diárias de insulina ao longo da vida. A insulina é uma hormona produzida pelas células beta do pâncreas e é necessária para evitar um aumento prejudicial do nível de glucose no sangue.

A causa exata da diabetes tipo 1 ainda é desconhecida, contudo, esta é considerada uma doença autoimune, uma condição que ocorre quando o sistema imunológico por engano ataca e destrói as células saudáveis do organismo. Na diabetes tipo 1, uma infeção ou outros fatores desconhecidos desencadeiam um ataque imune, que conduz a uma produção insuficiente de insulina.

Neste estudo, os investigadores da Universidade de Uppsala, na Suécia, decidiram estudar a ação das chamadas células T reguladoras em modelos de ratinhos para a diabetes tipo 1. O estudo apurou que na diabetes tipo 1 este tipo de linfócitos alteram a sua função produzindo proteínas pró-inflamatórias em vez de anti-inflamatórias, como é o caso da interleuquina 35 (IL-35).

Os investigadores também constaram que, comparativamente com os indivíduos saudáveis, a concentração da IL-35 era mais baixa nos pacientes com diabetes tipo 1. Estes achados sugerem que esta interleuquina pode desempenhar um papel importante na doença. Constatou-se ainda que as células T reguladoras alteravam o seu destino na presença da diabetes tipo 1.

Os investigadores testaram se a IL-35 poderia impedir o desenvolvimento da condição e reverter a diabetes tipo 1. De forma a induzir a doença nos ratinhos foi injetado um composto químico denominado streptozotocina. Os animais desenvolveram sinais de diabetes tipo 1 e constatou-se um aumento dos níveis de glucose no sangue semelhante ao observado nos humanos com a doença. Surpreendentemente, as injeções de IL-35 aos ratinhos diabéticos normalizaram as concentrações de glucose, impedindo o desenvolvimento da diabetes tipo 1.

O estudo também investigou o papel da IL-35 num outro modelo de ratinhos, denominado ratinhos diabéticos não obesos. Observou-se que a interrupção do tratamento não resultou no reaparecimento da diabetes em nenhum dos modelos dos animais.

Estes achados encorajam investigações futuras a utilizar a IL-35 no tratamento da diabetes tipo 1 e oferece novas pistas sobre o motivo pelo qual as células T reguladoras não são capazes de combater a diabetes tipo 1.

ALERT Life Sciences Computing, S.A

Para contar hidratos de carbono...

Ficam os links de 2 tabelas - uma Portuguesa e outra Espanhola!

Tabela da Composição de Alimentos (TCA)


Fundación para la diabetes - Tabla de raciones de hidratos de carbono


Divirtam-se... :s 

Vacina BCG pode reverter diabetes tipo 1? | ALERT® ONLINE - PT

Vacina BCG pode reverter diabetes tipo 1? | ALERT® ONLINE - PT

Teste aprovado pela Food and Drug Administration

11 junho 2015

Um ensaio clínico de fase II para testar a capacidade da vacina genérica do bacilo Calmette-Guérin (BCG) reverter a diabetes tipo 1 foi aprovado pela Food and Drug Administration (FDA).

O ensaio apresentado recentemente nas sessões científicas da Associação Americana de Diabetes investigará se a vacinação repetida com BCG pode melhorar clinicamente a diabetes tipo 1 em adultos entre os 18 e os 60 anos cujos níveis de secreção de insulina são baixos, mas detetáveis.

Os investigadores, liderados por Denise Faustman, foram os primeiros a documentar a reversão da diabetes tipo 1 em ratinhos e a completar com sucesso um ensaio clínico de fase I em humanos com a vacina BCG.

A vacina é utilizada na clínica há mais de 90 anos tendo sido também aprovada pela FDA para a vacinação contra a tuberculose e tratamento do cancro da bexiga. A vacina é conhecida por aumentar os níveis de um modulador do sistema imunitário, o fator de necrose tumoral (TNF). Estudos anteriores, conduzidos pelos mesmos investigadores, demonstraram que o TNF é capaz de temporariamente eliminar, em humanos e ratinhos, os leucócitos responsáveis pela diabetes tipo 1. Níveis aumentados de TNF também estimulam a produção de linfócitos T reguladores.

Na fase I dos ensaios clínicos, publicados na revista “PLOS Medicine”, verificou-se que a administração de duas injeções de BCG, espaçadas de quatro semanas, conduziu à eliminação temporária dos linfócitos T causadores da diabetes e forneceu evidência de uma pequena alteração transitória da secreção da insulina.

O ensaio clínico de fase II irá incluir uma dose mais frequente ao longo de um período mais longo, com intuito de determinar o potencial da repetição da vacinação BCG na melhoria do estado autoimune e de parâmetros clínicos como o HbA1c, um marcador que indica o nível médio de açúcar no sangue.

No novo ensaio, 150 pacientes com diabetes irão receber aleatoriamente duas injeções de BCG ou um placebo, com quatro semanas de intervalo . Posteriormente será administrada uma única dose anual da vacina ao longo de quatro anos. Os pacientes vão ser acompanhados ao longo dos cinco anos do estudo onde serão medidas possíveis melhorias nos resultados dos testes da HbA1c.

“Na fase I dos ensaios clínicos demonstrámos uma resposta estatisticamente significativa para o BCG, mas nosso objetivo na fase II é criar uma resposta terapêutica duradoura. Vamos estar novamente a trabalhar com pessoas que tiveram diabetes tipo 1 por muitos anos. Este não é um teste de prevenção; em vez disso, estamos a tentar criar um regime que irá tratar a doença, mesmo que avançada”, conclui Denise Faustman.

ALERT Life Sciences Computing, S.A.

A chip placed under the skin for more precise medicine

A chip placed under the skin for more precise medicine



27.05.15 - It’s only a centimetre long, it’s placed under your skin, it’s powered by a patch on the surface of your skin and it communicates with your mobile phone. The new biosensor chip developed at EPFL is capable of simultaneously monitoring the concentration of a number of molecules, such as glucose and cholesterol, and certain drugs.

“This is the world’s first chip capable of measuring not just pH and temperature, but also metabolism-related molecules like glucose, lactate and cholesterol, as well as drugs,” said Dr Carrara. A group of electrochemical sensors works with or without enzymes, which means the device can react to a wide range of compounds, and it can do so for several days or even weeks.

This one-centimetre square device contains three main components: a circuit with six sensors, a control unit that analyses incoming signals, and a radio transmission module. It also has an induction coil that draws power from an external battery attached to the skin by a patch. “A simple plaster holds together the battery, the coil and a Bluetooth module used to send the results immediately to a mobile phone,” said Dr Carrara.

Dr. Denise Faustman Ready for Next Phase of Diabetes Vaccine Research

Dr. Denise Faustman Ready for Next Phase of Diabetes Vaccine Research
(...)
'People with type 1 diabetes, multiple sclerosis (MS), and other diseases had a relative deficiency in a hormone known as TNF, and with a natural vaccine boosting TNF, you could get rid of bad T-cells and boost T-regs, and the pancreas regenerates. We decided to use a safe, 100-year-old vaccine to make this happen and we've found that it works.
(...)
In early data, we showed that in long-term diabetics, indeed these T-regs were boosted and we could see the targeted death of bad T-cells. We also the beginnings of pancreas regeneration. Sure, no one was throwing insulin syringes away yet, because it was just the beginning... but it showed that this could be done. And importantly, this was in long-term type 1s with 15 to 20 years -- that rattled a lot of people. This was a totally unique patient population, and not how most research was done in newly diagnosed individuals.
(...)
The people in the trial had diabetes for an average of 15 years, and this showed that we could restore insulin production, at least briefly, for people who had type 1 for many years.
(...)
We are going to look at how much BCG is needed, and how frequently. That's the key, the secret: knowing how much to dose. With Phase II-a, I'll try to perfectly match those results in Phase I, with longstanding type 1s who still make a little C-peptide. Then it will be Phase II-B where there isn't C-peptide in longtime type 1s. And after each part, we need to follow these people for five more years.'

PLOS ONE: Covariation of the Incidence of Type 1 Diabetes with Country Characteristics Available in Public Databases

PLOS ONE: Covariation of the Incidence of Type 1 Diabetes with Country Characteristics Available in Public Databases


Aqui fica a introdução de um estudo que me parece bastante interessante. Leia tudo no link acima.

'Introduction

It has long been noticed that the incidence of Type 1 Diabetes (T1D) is highly variable from one country to another. For example, the 62.42/100.000 persons/year incidence found in Finland [1] was 780-fold larger than the 0.08/100.000 persons/year incidence in Papua New Guinea [2]; differences in T1D incidence are also observed between countries where the health care systems are comparable. The variability of T1D incidence is even visible within countries; for example, in Italy, T1D incidence varied between 54.4/100.000 persons/year in Sardinia [3] and 4.4/100.000 persons/year in Lombardia [4]. The reason for these differences is not precisely known, but is most unlikely due to classification bias, as the disease cannot go untreated, and the diagnosis is relative easy to perform in children [5]. The country-to-country T1D variability is known to be partly explained by genetic variations. Indeed, HLA (human leukocyte antigen) and 33 other genes are associated with elevated risk of T1D (T1Dbase Version 4.18 updated on 30/9/2014 available at http://www.t1dbase.org) [5–11]. The genetic characteristics of several populations have been found to—at least partially—explain the level of their T1D incidence [12]. For example, the low incidence in Japan, and more generally in southeast Asia, was strongly associated with the absence of highly susceptible haplotypes, such as DRB1*03-DQB1*0201 and DRB1*04-DQB1*0302 found in Caucasian populations [13] or DRB1*030101-DQB1*0201 [14] found in Arab populations (Bahrainis, Lebanese, and Tunisians). Instead, the major susceptible HLA haplotypes in the Japanese and Korean populations were DRB1*0405-DQB1*0401 and DRB1*0901-DQB1*0303 [15].

Another peculiarity of T1D epidemiology is that a dramatic increase of the incidence (on average 3% per year [16]) was observed over the last decades in many countries, in particular European countries with previously low incidences. This increase cannot be explained by genetic factors, since the genetic structure of these countries cannot have varied greatly over such a short period of time. The reasons are more likely to be found in environmental factors (taking here environment broadly, as encompassing physical, chemical, social and life-style factors). However, no single environmental factor, or configuration of factors, that could explain the patterns of differences has ever been identified. More likely, there are complex networks of environmental causes, and of gene-environmental causes that remain to be discovered.

The search of genetic factors of T1D was facilitated during the last 10 years by the GWAS (genome-wide association studies) technology that replaced the gene candidate approaches and instead scanned the entire genome to find SNPs (Single-Nucleotide Polymorphisms) that were significantly associated with T1D [17]. The discovery of an SNP was not the discovery of a “gene”, but was a marker leading to the possible discovery of a gene. Here, we translate this data-driven approach to search for environmental markers related to variations of T1D incidence that might eventually lead to environmental causes, possibly in interaction with genetic factors. One could indeed expect that, in this age of information, plenty of environmental characteristics could be readily available, insofar as local and global organizations collect such data, and provide them free to researchers, with easy interface on the Internet. A limitation commonly advocated is that country statistics are in many cases of too low quality. However, for two related reasons, this argument does not hold, or will not hold for long: a) why collect, maintain and publish such statistics if they cannot be used by researchers? and b) how can one encourage a better quality for these statistics—meaning more resources devoted to them—if they are never used?

Here, we present the attempt we made to use open public data to identify climate and environmental, demographic, economic, and health characteristics correlated with variations in T1D incidence between countries.'

Diabetes tipo 1: desenvolvida insulina inteligente | ALERT® ONLINE - PT

Diabetes tipo 1: desenvolvida insulina inteligente | ALERT® ONLINE - PT

'Diabetes tipo 1: desenvolvida insulina inteligente

Estudo publicado na “Proceedings of the National Academy of Sciences”

12 fevereiro 2015

Investigadores americanos desenvolveram uma insulina de longa duração, a Ins-PBA-F, que se auto-ativa quando os níveis de glucose aumentam, dá conta um estudo publicado na revista “Proceedings of the National Academy of Sciences”.

Os investigadores da Universidade de Utah, nos EUA, verificaram em modelos de ratinhos que os efeitos de uma injeção deste tipo de insulina duram pelo menos 14 horas, período durante o qual pode reduzir repetidamente e automaticamente os níveis de glucose no sangue após a administração de quantidades de glucose comparáveis às ingeridas na alimentação.

Os pacientes com diabetes tipo 1 têm de monitorizar os seus níveis de açúcar várias vezes ao dia e determinar quando e quanta insulina devem administrar. Um erro de cálculo ou um lapso na administração de insulina pode fazer com que os níveis de glucose atinjam níveis demasiado elevados (hiperglicemia), conduzindo possivelmente a doenças cardíacas, cegueira ou outras complicações a longo prazo. Por outro lado, se os níveis de glucose atingirem níveis muito baixos (hipoglicemia) pode induzir o coma ou levar mesmo à morte.

Apesar dos avanços em torno do tratamento da diabetes, como bombas de insulina e o desenvolvimento de quatro tipos de insulina, os pacientes continuam a ter que ajustar manualmente a quantidade de insulina administrada diariamente. Os níveis de glucose variam bastante em função de vários fatores, incluindo alimentação e prática de exercício físico.

Deste modo, uma insulina sensível à glucose que é ativada automaticamente quando os níveis de açúcar no sangue são muito elevados, eliminaria a necessidade de injeções adicionais e reduziria os perigos associados a uma quantificação incorreta. Apesar de estarem em desenvolvimento várias insulinas “inteligentes”, muitas delas apresentam efeitos secundários indesejáveis.

Esta nova insulina inteligente, a Ins-PBA-F, foi criada através da modificação química da insulina. Esta insulina consiste num derivado da insulina de ação prolongada que tem um motivo químico, o ácido fenilborónico, num dos extremos. Em condições normais a Ins-PBA-F liga-se a proteínas séricas que circulam na corrente sanguínea, bloqueando a sua atividade. Quando os níveis de glucose são demasiado elevados estes ligam-se ao ácido fenilborónico fazendo com que a Ins-PBA-F seja libertada.

O estudo apurou ainda que a Ins-PBA-F é mais rápida a diminuir os níveis de glucose do que a insulina de ação prolongada detemir.

De acordo com um dos autores do estudo, Danny Chou, como a Ins-PBA-F é uma versão quimicamente modificada de uma hormona natural, é muito provável que seja segura o suficiente para ser utilizada numa base diária, de um modo semelhante a outros derivados de insulina que já estão atualmente no mercado.'

ALERT Life Sciences Computing, S.A.

Re-educating the Immune System in T1D | JDRF: Improving Lives. Curing Type 1 Diabetes

Re-educating the Immune System in T1D | JDRF: Improving Lives. Curing Type 1 Diabetes

"In a recent publication in the prestigious scientific journal, Proceedings of the National Academy of Sciences, JDRF partner Selecta published the details of a novel technology that is being used to develop a vaccine for the induction of immune tolerance in people with T1D. Their technique involves creating tiny packages called nanoparticles containing the T1D antigens and immunomodulatory agents that ultimately reach the key cells of the immune system and trigger the re-education process. Selecta is one of the leaders in the development of antigen-specific immune tolerance therapies and their approach holds great promise for T1D. With recently renewed support from a JDRF-Sanofi partnership, Selecta is set to begin testing this antigen-specific immune therapy in mouse models of T1D. If successful, this could pave the way for pilot clinical studies and ultimately a means to restore normal immune function and halt the T1D disease process.

For more information or to support the JDRF restoration research program, please click here."

Abbott’s FreeStyle Libre – Transforming Glucose Monitoring Through Utter Simplicity, Fingersticks Aside! | diaTribe

Abbott’s FreeStyle Libre – Transforming Glucose Monitoring Through Utter Simplicity, Fingersticks Aside! | diaTribe


'In October, Abbott launched its highly awaited FreeStyle Libre Flash Glucose Monitoring system in Europe. The unique product is intended as a replacement for blood glucose meters, while giving patients many of the benefits of continuous glucose monitoring (CGM), including real-time glucose values, trend information and comprehensive reports. Though it is not yet approved in the US, we were able to test the product over the past month (the device can only be ordered online from websites in Europe). 

Given what we had heard from so many European bloggers, we had high expectations going into our test, and FreeStyle Libre absolutely met them at every step – the system was  easy to setup and use (a major win for healthcare providers); discreet to wear on the upper arm; accurate enough from which to dose insulin, with performance similar to Dexcom’s G4 Platinum CGM (though no fingersticks were required); and it gave an excellent picture of glucose trends through real-time and on-device reports. In short, it is transformative compared to the limited information provided by traditional blood glucose meters, all in a package anyone can pick up and learn to use. We give FreeStyle Libre an emphatic thumbs up and would recommend it to nearly anyone with diabetes, especially those on insulin who test their blood glucose frequently and want more actionable information than fingersticks alone can provide. One key point of difference from CGM is that FreeStyle Libre does not have high or low alarms, meaning it is not as ideal for those with lots of hypoglycemia or hypoglycemia unawareness.'

FreeStyle® Libre