Tagged: Faustman

Dear JDCA (Juvenile #Diabetes Cure Alliance) Part Two

hands-talking-1311915-640x480If you have no idea what’s going on, please read Dear JDCA Part One.

It will bring you up to speed (as quickly as 2500 words can), giving you the information regarding what the Juvenile Diabetes Cure Alliance thinks are the most likely “practical cures” for people with Type 1 diabetes and why their petition demanding JDRF and ADA to commit 30% of donations to “cure research” isn’t doing anything to help the diabetes community except cause deep fractures amongst us.

Part One covered the transplantation “practical cures” that JDCA believes we should all support with the intent to have these cures available by 2025.

Part Two below covers the devices and the immune system manipulation options they highlight.

Quick Recap

The Juvenile Diabetes Care Alliance states that they want a “practical cure” by 2025. JDCA’s Four categories of a “practical cure”:

  • Islet cell transplantation
  • A device that mimics the pancreas
  • Glucose-responsive insulin (“smart insulin”) ***Please note that none of the potential practical cures are of this type.***
  • Modification of the immune system (blocking, balancing, and/or retraining)

Every year, JDCA issues a report that tells potential donors which “practical cures” are more likely to pull ahead. Here are the 2014 “Potential Practical Cure Solutions,” found in the JDCA State of the Cure report. (2015 hasn’t been published yet, but expect it in the fall as in previous years).

For each,  I will provide you the basic info into what it is, where this is in terms of “potential” release into the community and who is funding it.

JDCA’s 2014 Potential Practical Cure Solutions (Part 2)

Device

Bionic Pancreas (iLet)

Boston University

– Boston, MA

What it is: 

Engineers from Boston University have developed a bionic pancreas system that uses continuous glucose monitoring along with subcutaneous delivery of both rapid-acting insulin (to lower blood glucose) and glucagon (to raise blood glucose) as directed by a computer algorithm. The bionic pancreas automatically makes a new decision about insulin and glucagon dosing every five minutes; that’s 288 decisions per day, 7 days per week, 365 days per year. (Artificialpancreas.org)

Where it is in the pipeline of “practical cure”:

According to Clinical Trials.gov, Phase III clinical trials will begin soon, with an estimated completion date of August, 2016. 80 participants will be in this trial. All clinical trials to this date have had favorable results.

If the pipeline is followed, commercialization is to be expected by 2018. The major issues will be funding and the FDA approval of stable glucagon provided by Xeris Pharmaceuticals.

 

Who is funding this “practical cure”?

Drs. Damiano and Russell do not work for a privately funded company. They work for universities. Funding is obtained through NIH grants and generous donations from private donors. JDRF did fund a portion of this research. 

There is currently no investment funding for future commercial agreement. Funding is being obtained through donations made through Boston University and Massachusetts General Hospital, private grants, and potentially NIH.

This is one “potential cure” that is fueled by direct donations from the general public.

If you are interested in donating to the Bionic Pancreas (iLet), you can do so here. 

If you want to learn more about the iLet, click here.


Modification of the immune system

BCG

Faustman Labs (Massachusetts General Hospital)

-Boston, MA

What it is: 

The BCG Human Clinical Trial Program is testing Bacillus Calmette-Guérin (BCG), an inexpensive generic drug, as a treatment for advanced type 1 diabetes.

…current research focuses on discovering and developing new treatments for type 1 diabetes and other autoimmune diseases, including Crohn’s disease, lupus, scleroderma, rheumatoid arthritis, Sjögren’s syndrome, and multiple sclerosis. (Faustman Lab website)

Where it is in the pipeline of “practical cure”:

A Phase II clinical trial which will last five years is being launched.

In the phase I clinical trial, which was published in the August 8, 2012, issue of PLOS Medicine, two injections of BCG spaced four weeks apart led to temporary elimination of diabetes-causing T cells and provided evidence of a small, transient return of insulin secretion. The phase II clinical study will include more frequent dosing over a longer time period to determine the potential of repeat BCG vaccination to ameliorate the autoimmune state and improve clinical parameters such as HbA1c, a marker of average blood sugar control. (Eureka Alert)

Here is the Clinical Trials.gov posting for the Phase II Clinical Trial. Please note that this is a double blinded trial. Neither the investigator nor the participant will know if they are being administered the BCG vaccine or saline injections over 5 years. 150 participants will be selected.

Earliest results will be in 2020. Please also be aware that a Phase III Clinical Trial must be conducted after successful results are shown, which can delay commercialization by several years if the Phase II Clinical Trial results show promise.

Who is funding this “practical cure”?

The Lee Iacocca Foundation gave Faustman Labs $10 million dollars initial funding for her Phase I trials and has also committed funding to Phase II trials. Faustman Labs estimates that Phase II trials will cost $25.2 million dollars.

This is one “potential cure” that is fueled by direct donations from the general public.

If you are interested in donating to Faustman Labs, click here. 

Note: Mike Hoskins of DiabetesMine/Healthline did a recent interview with Dr. Denise Faustman in March, 2015. It’s an important read.  During the interview, Dr. Faustman mentions that neither JDRF or Helmsley Charitable Trust has funded her, but that NIH, private supporters, Lee Ioacocca and others have chosen her research as their “practical cure.”


TOL-3021

Tolerion, Inc.

-Portola Valley, CA

What it is: 

TOL-3021, is a novel reverse vaccine that induces tolerance to the type 1 diabetes specific auto antigen proinsulin and thereby reduces disease activity.

Unlike conventional vaccines, which act to stimulate the immune system, the reverse vaccine TOL-3021 is designed to selectively suppress specific elements of the immune system that are inappropriately activated in type 1 diabetes. TOL-3021 contains an engineered DNA plasmid that expresses proinsulin, which is associated with the autoimmune-caused destruction characterizing type 1 diabetes.(Tolerion website)

Where it is in the pipeline of “practical cure”:

In 2013, a press release regarding TOL-3021 stated:

The Phase 2 study results reported in today’s edition of Science Translational Medicine1 demonstrated that TOL-3021 preserved pancreatic beta-cell function while reducing destructive disease-specific T-cell activity in patients with type 1 diabetes.

These promising Phase 2 data indicate that TOL-3021 may stop the destruction of pancreatic beta cells and improve the long-term outlook for patients with type 1 diabetes, even in adults with long-established disease. Based on these results, we are eager to test TOL-3021 in a larger trial with longer dosing beyond 12 weeks, and to assess whether it might slow or stop disease progression entirely in younger patients when administered before large numbers of beta calls have been destroyed.

Nothing in regards to clinical trials has been published since 2013. In 2013, an article regarding the Stanford researchers who conducted this trial stated:

There are caveats with the trial. For one, the vaccine must be studied in more humans and is years away from being considered for Food and Drug Administration approval. What’s more, in the study, the vaccine’s benefits tailed off a few weeks after its 12-week dosing schedule was stopped.

I have yet to find another announcement regarding a Phase III clinical trial or anything regarding this “practical cure” since 2013. 

Who is funding this “practical cure”?

Dr. Lawrence Steinman, the Stanford researcher who, along with other researchers involved with this project, founded Tolerion. Here’s what he had to say regarding funding:

I can’t give enough praise to Bayhill’s investors, the JDRF (formerly the Juvenile Diabetes Research Foundation) and well-known VCs on Sand Hill Road and the Bay Area.

(When I dug a little deeper, I found that the original name of this drug was BHT-3021 and the trial was funded by… JDRF in collaboration with Bayhill Therapeutics.)

Rights to the reverse vaccine technology and associated product pipeline have been licensed to Tolerion by Stanford University.

There is no public donation funding being requested.


Cyclosporine Omeprazole/Lansoprazole

Perle Bioscience

-Charleston, NC

What it is: 

Cyclosporine is a well-known immunosuppressant given as an anti-rejection medication after organ transplantation and for treatment of RA (rheumatoid arthritis) or psoriasis.

Lansoprazole is a proton-pump inhibitor. You might know it by its brand name: Prevacid.Omeprazole is also a proton-pump inhibitor. (Brand name: Prilosec.) It decreases the amount of acid in the stomach and is commonly used in treating heartburn and GERD (reflux).

In 2013, Chris Leach of Insulin Nation wrote an incredibly informative article about Perle Bioscience and their “practical cure”.  He gave a better overview than I ever could about this so I encourage you to read his work.

Where it is in the pipeline of “practical cure”:

On June 23, 2015, Perle Bioscience announced that a Stage 3 clinical trial was beginning with their drug combination – PRL001. Except…

This Phase III clinical trial is not for individuals with long-standing Type 1 diabetes. According to the ClinicalTrial.gov information, this trial is for newly diagnosed Type 1 diabetics aged 10 – 20 years old. The trials will not be conducted in the U.S.

For those with diabetes diagnosed more than 12 weeks ago, the only clinical trial data listed on ClinicalTrial.gov, there is simply a “This study is not yet open for participant recruitment.” It’s last update was October 3, 2013.

Perle Bioscience’s website gives this information regarding its pipeline:

PRL001

PRL001 consists of the combination of two previously approved therapeutic agents, each yielding their specific response on the body. In Vivo animal studies, the first product inside of PRL001 causes the regeneration of the patients own insulin producing pancreatic beta cells to start growing again. The second part of PRL001 lowers the body ability from re-attacking the newly formed insulin producing cells to essentially put the pancreas back to how it was functioning prior to diabetes. Both agents are taken orally and no injections are needed for this product. Perle Bioscience, Inc. holds the issued and pending US and IPC patents (see below for patents) for the new use of these agents in treating diabetes. PRL001 is starting multi-center Phase 3 human trials in early 2015.

PRL002

PRL002 is a novel peptide developed by Perle Bioscience, Inc., where in current in vitro and in vivo studies, is showing signs of high levels of regeneration of insulin producing pancreatic cells. PRL002 is made up of our novel Beta Regeneration Agent for Diabetes (BRAD) peptides. Currently PRL002 is in animal trials with the hope to have an IND application to the FDA in early 2016. Our hope is that PRL002 will be used for both type 1 and type 2 diabetes. Please sign up for our newsletter to stay up to date on our progress (signup from the bottom of any page).

There is no mention of the Phase III clinical trial for individuals with “existing Type 1 diabetes” on Perle Bioscience’s website. 

Who is funding this “practical cure”?

According to Motley Fool:

Perle is a privately held company, so no investments can be made here as of yet.


Tianhe Stem Cell Biotechnologies

Stem Cell Educator Therapy

-China

What it is: 

“Stem Cell Educator therapy” is the innovative technology developed by Dr. Yong Zhao that uses stem cells drawn from human cord blood to targets autoimmune diseases. Currently, Tianhe is focusing on the application of Stem Cell Educator therapy in diabetes. Our clinical data provide powerful evidence that Stem Cell Educator therapy can balance the immune system and lead to the regeneration of islet beta cells and improve metabolic control in long-standing diabetic subjects. This groundbreaking technology is taking steps towards the ultimate cure of diabetes and revolutionizing the treatment of other autoimmune-related diseases.(Tianhe website.)

Where it is in the pipeline of “practical cure”:

Clinical Trials.gov gives this information: Stem Cell Educator Therapy in Type 1 Diabetes was last updated in November, 2013 and is still stating that it is recruiting participants in China and Spain. Expected completion was September, 2014.

No additional information regarding trials and a “practical cure” has been listed on the Tianhe website. The latest clinical trial information discusses Stem Cell Educator Therapy for hair regrowth in Alopecia Areata patients. (April 22, 2015)

Who is funding this “practical cure”?

The Chinese government, according to the sponsorship information provided on Clinical Trials.gov. The Tianhe website is looking for investors, stating this as an enticement:

A huge marketing opportunity due to the global prevalence of diabetes: For instance, the total number of Americans living with diabetes will increase by 64% in 2025. Annual Medicare cost will increase by 72%, with $514 billion/year (72nd ADA report).

There is no request from the general public for donations.


Previous JDCA “Practical Cures”

file-1-3-1237622-640x640JDCA has published three reports, talking about the “cure” and which projects they believe fall under the guidelines. I’ve focused on the 2014 report (which is the latest), but what about 2013? 2012? (They began in 2011.)

2013

In 2013, here’s what they said were practical cures paths, because they were in human trials:

Still going…

Diabecell (Phase II) – I’ve listed the latest in Part 1.

Tianhe Stem Cell Educator Therapy (Phase II) – I’ve listed the latest above.

BCG (Phase II recruiting) – I’ve listed the latest above.

ViaCyte (Phase I) – I’ve listed the latest in Part 1.

Off the list in 2014…

Sitagliptin/Lansoprazole (Phase II) Note: collaborator listed on ClinicalTrials.gov is JDRF. Status listed now as “The recruitment status of this study is unknown because the information has not been verified recently. )

Monolayer Cellular Device (Phase 1) According to ClinicalTrials.gov, this study is still recruiting patients for one location in Belgium, but JDCA has dropped it from the 2014 list of practical cures.

2012

In 2012, here’s what they said were practical cures paths:

Still going…

Diabecell (Phase II) – I’ve listed the latest in Part 1.

BCG (Phase II recruiting) – I’ve listed the latest above.

Off the list in 2013 and 2014…

Sitagliptin/Lansoprazole

Monolayer Cellular Device

ATG/GCSF – This Phase II clinical trial  is no longer recruiting patients but is active (meaning they got enough participants). The work is still ongoing, but if you read the criteria, it was modified to say that the participants must have been diagnosed between 1 and 2 years before the start of the study, taking out the possibility for those with long-standing Type 1 to participate. The trial is called: Reversing Type 1 Diabetes After it is Established and is being run by University of Florida with grants from the Helmsley Charitable Trust and Genzyme.


My Scorecard for 2014 Practical Cures

Based on what JDCA says are “practical cures” and what I’ve researched, here is the likelihood that they will become commercially available by JDCA’s 2025 “deadline.”

Transplantation

VIACYTE – Phase I/II clinical trial right now. Estimated commercialization date of this product if Phase I/II is successful, a Phase III trial is conducted and successful and proceeds to FDA approval? Unlikely by 2025.  
DIABECELL – Phase I/IIa completed. No Phase III clinical trials listed in ClinicalTrials.gov. Estimated commercialization date of this product if there is a Phase III trial and it proceeds to FDA approval? Unlikely by 2025.
ßAIR BIO-ARTIFICIAL PANCREAS – Phase I/II clinical trial right now. Estimated commercialization date of this product if Phase I/II is successful, Phase III happens and is successful and proceeds to FDA approval? Unlikely by 2025.

Device

BIONIC PANCREAS (iLET) – Phase I/II completed and Phase III beginning soon. Estimated commercialization date of this product if  Phase III proceeds to FDA approval and Xeris Pharmaceuticals gets FDA approval for stable glucagon? Likely by 2018 (2019 if you’re hedging bets.).

Modification of the Immune System

BCG – Phase II clinical trial being launched, lasting five years. Estimated commercialization date of this product if Phase II and Phase III is successful, proceeding to FDA approval? Unlikely by 2025.
TOL-3021 – Phase II clinical trial completed, with results showing the benefits did not last longer than the 12 week dosing period. There are no Phase III clinical trials list. Unlikely by 2025.
CYCLOSPORINE/LANSOPRAZOLE – Phase II completed. No clinical Phase III clinical trial listed on clinicaltrials.gov for patients with existing Type 1 diabetes. Unlikely by 2025.
STEM CELL EDUCATOR THERAPY – Phase II clinical trial listed in 2013 and still recruiting. Estimated commercialization date of this product if Phase I/II is successful, a Phase III trial is conducted and successful and proceeds to FDA approval? Unlikely by 2025.  

Conclusion to this LONG Part 2…

paper-numbers-1236363-639x426What one organization thinks is a  cure isn’t always a cure to you. It’s easy for JDCA call out an organization for not doing enough for what they think is “cure research.”
It’s much more difficult to do so when you realize that everyone has a different (and in JDCA’s reports, showing an ever changing) idea of what a “practical cure” would mean. 
What is practical in 2012, 2013, and 2014 may not be practical in 2015. JDCA kept mentioning DRI’s BioHub, but never put it on the top list of their “practical cures,” but yesterday’s announcement that the first human subject to be implanted with the BioHub is off insulin. (Time will tell is this is successful in the long-term, but JDCA didn’t consider this a donation priority.)
What about “smart insulin?” What about the other closed loop AP trials? What about…. something we haven’t thought of yet? We don’t know what the young researchers in five years will present to major diabetes organizations (or the general public). Seed money comes from these large diabetes non-profits to new researchers who seek out grants to get them off the ground. What happens to these researchers if they don’t get the seed money?
Nothing. Literally. 
JDCA is cherry-picking (and finger pointing, but to what end remains to be seen). It’s their right and I’ve learned a lot about the different avenues to a “practical cure. ” (Hat tip to JDCA for sending me down the rabbit hole. I’m certainly better educated for it.)
We are ALL cherry-picking. It’s our right. But do it by doing the research and making smart, educated choices.
We are all in this together, despite differences in opinions about what a cure entails, who is going to provide it, and who should fund it. And most importantly, who speaks for you. (Here’s a hint… it’s not me or JDCA.) It’s my hope that you learned something about “cure research” and where YOU can get your information about cure pipelines so you can speak for yourself.
Thank you for reading all the way to the end. 

7 Noteworthy Diabetes Clinical Trials Recruiting NOW…

elixir-1312949-639x468Clinical trials are the only way that we are going to get better treatments, better devices, and better…cures.

Here are seven noteworthy diabetes clinical trials recruiting now that you might want to look into and see if you (or anyone you know) might be eligible to volunteer.

Click on the titles of each trial to get more info straight from the ClinicalTrials.gov website. 

(Remember… some clinical trials may have you take a placebo in lieu of the investigational drug. Some clinical trials may require extra visits, invasive testing, and travel. You need to think about what the benefits and risks are for trial participation. That being said… nothing ventured, nothing gained.)

Repeat BCG Vaccinations for the Treatment of Established Type 1 Diabetes

The purpose of this study is to see if repeat bacillus Calmette-Guérin (BCG) vaccinations can confer a beneficial immune and metabolic effect on Type 1 diabetes. Published Phase I data on repeat BCG vaccinations in long term diabetics showed specific death of some of the disease causing bad white blood cells and also showed a short and small pancreas effect of restored insulin secretion. In this Phase II study, the investigators will attempt to vaccinate more frequently to see if these desirable effects can be more sustained.

Eligible volunteers will either be vaccinated with BCG in a repeat fashion over a period of four years or receive a placebo treatment. The investigators hypothesize that each BCG vaccination will eliminate more and more of the disease causing white blood cells that could offer relief to the pancreas for increased survival and restoration of insulin secretion from the pancreas.

If you’re interested and meet the criteria (and the location, as the trial is being conducted in Boston and requires weekly injections for the first year… don’t know if you can do this at home…), you should send an email to: diabetestrial@partners.org

This is Dr. Denise Faustman’s lab and website. Check out the details and what she’s doing.

Multiple Daily Injections and Continuous Glucose Monitoring in Diabetes (DIaMonD)

Evaluate if addition and use of real time continuous glucose monitoring (RT-CGM) improves glycemic outcome of patients using multiple daily injections (MDI) and self monitoring blood glucose (SMBG) testing, who are not at target glycemic control.

If you are on multiple daily injections, this might be a great opportunity to participate in a really interesting study if you are willing to wear a continuous glucose monitor (CGM) and possibly a pump. Check out the inclusion/exclusion criteria and locations, then send an email or call to either:

Eileen Casal, RN, MSN 858-875-9774 ecasal@dexcom.com
 David Price, MD 858-875-9525 dprice@dexcom.com

A Trial Comparing Continuous Glucose Monitoring With and Without Routine Blood Glucose Monitoring in Adults With Type 1 Diabetes (REPLACE-BG)

The primary objective of the study is to determine whether the routine use of Continuous Glucose Monitoring (CGM) without Blood Glucose Monitoring (BGM) confirmation is as safe and effective as CGM used as an adjunct to BGM.

This study will determine if we can actually make treatment decisions based on our CGM alone when we feel it is accurate, not verifying it with a finger-stick blood glucose check.

This. Is. Huge.

Why? Because one of the reasons why Medicare, Medicaid, and some insurance companies refuse to pay for a continuous glucose monitor, claiming it’s just an adjunct to a blood glucose meter and we still have to check to make treatment decisions. (And we know better, don’t we?) This trial has a lot of inclusion and exclusion criteria, but seriously… if you can do this, you will help the entire T1 diabetes community get access to this device.

Contact either person for more info:

 Katrina Ruedy, MSPH 813-975-8690 kruedy@jaeb.org
 Nhung “Leena” Nguyen, MPH, CCRP 813-975-8690 nnguyen@jaeb.org

Glucose Variability Pilot Study for the Abbott Sensor Based Glucose Monitoring System-Professional

This is to trial the Abbot Libre system, which is a sensor with “flash monitoring” for individuals with Type 2 diabetes. How cool is that? They currently need participants in the following locations: San Diego, Detroit, Kansas City, MO and Pearland, TX. If you meet the criteria, shoot Dr. Karinka an email for more info and get enrolled.

Shridhara Alva Karinka, Ph.D. 510-749-6393 shridhara.alva@abbott.com

A Study To Assess The Safety Of PF-06342674 In Adults With Type 1 Diabetes

If you are a newly diagnosed (within the last two years) adult (over 18), you can participate in a Phase 1 clinical trial for a biological drug, examining safety issues. Again, look at the criteria and locations, then if you are interested, call:

Pfizer CT.gov Call Center 1-800-718-1021

Please refer to this study by its ClinicalTrials.gov identifier: NCT02038764

In-Clinic Evaluation of the Predictive Low Glucose Management (PLGM) System in Adult and Pediatric Insulin Requiring Patients With Diabetes Using the Enlite 3 Sensor

This is a Medtronic study for their next step in the artificial pancreas technology pathway. (And hello… “Enlite 3 sensor!”)

All subjects will undergo hypoglycemic induction at Visit 2 with target set to 65 mg/dL using the rate of change basal increase algorithm. Low Limit setting when PLGM ON is 65 mg/dL.

The more patients willing to participate in artificial pancreas technology trials, the faster this technology will become available! Take a look at the locations and criteria and if you’re able to do this trial, contact:

Julie Sekella (818) 576-5171 julie.sekella@medtronic.com

Along those same lines…

Hybrid Closed Loop Pivotal Trial in Type 1 Diabetes

This is a BIG. DEAL. for people with diabetes in the United States. If on this trial, you get to wear the MMT-670G insulin pump, using it with the closed loop algorithm.

Closed Loop. Closed Loop. Need we say more?

Contact: Thomas P Troub(818) 576-3142 thomas.troub@medtronic.com to get involved.

There are so many studies out there that need our help. We help ourselves AND all people with diabetes. Do what you can. If you can’t participate, share this post with someone who might be able to volunteer.