Scott and Bennet’s JDRF Adventure

I have written periodically about JDRF’s IDDP program. Occasionally I have had conversations either via email exchanges on on the phone with JDRF’s communications staff about IDDP. For those less interested in alphabet soup, IDDP stands for Industrial Discovery and Development Partnerships. This is a smaller portion of JDRF’s activities as measured in dollar terms, maybe 10% of their funding budget. The goal of IDDP is to help transition discoveries from the lab to therapeutic options in the clinic.

I think the IDDP program is valuable. I am added a link in my sidebar with the Newbie link and Teens that can help anyone interested find more about what I have said about IDDP. (I realize that is probably a very small group.) In time I’ll ad a post that includes other good pieces on the projects.

There is a wide series of gaps between the discoveries we read about in laboratories and the options our care team can prescribe. Each gap being another step along a regulatory process from lab to pharmacy. This path will be followed by better care options and it will be true of any incremental steps to a cure.

Here is what those gaps cumulatively look like.  Bringing a pharmaceutical treatment to market is estimated in one research study cost $1.3 billion, yes with a “B” and take 7.9 years.¹

IDDP helps bridge gaps at strategic points. These may be points where industry is less inclined to do take some of the steps along that 1.3 billion dollar process or possibly steps where a type 1 application for a product is not seen by a pharmaceutical company. You can think of it as JDRF helping to push the processes along at key sticking points.

Even representing a fraction of JDRF’s funding budget the amounts involved are substantial. So I believe there is a need for clear communications on what these partnerships entail. I think this is one way that bloggers can serve our community. (This post is about Scott and Bennet's excellent JDRF  adventure, for a primer on IDDP here is a link.)

Last week Scott Strumello and I had the opportunity to to meet with JDRF in their New York offices for an extended conversation on IDDP. I was anxious to have Scott participate. He and I share an interest in writing about the business side of diabetes. We have both written about the IDDP process.

I think he writes in deeper detail while I write try to translate business writing into a more common vernacular. Somewhere in there, between the two of us, I hope folks get an insight they may not otherwise have. I respect and admire Scott’s writing. I consider him a friends and am proud to join him in writing about IDDP. In fact I would not have scheduled a meeting at JDRF without him as every Andy Sipowicz needs a Bobby Simone. I am not sure who is which.

Scott and I with Rachel Steingardt, Joana Casas and Gary Feit of JRFD’s communications team first. We talked about social media and more transparent means of JDRF sharing information in general and the IDDP program specifically.

We were then joined by Richard Insel, MD, JDRF’s Chief Scientific Officer and Karin Hehenberger, MD, Ph.D. Senior Vice President of Strategic Alliances. Jeffery Brewer JDRF’s CEO was going to join us but he was ill. (Insert your joke about me making people sick here.) Jeffery has since followed up with an email which was very gracious. I greatly appreciate that as sign of his openness. My good friend Kelly Kunik speaks well of him and that is high praise in my book.

I had scores of questions going in but the question that troubled me the most was this; how could I approach JDRF with detailed and potentially tough questions about IDDP projects and be seen as an advocate of people living with type 1 and not simply a critic? In fact that I think was the real goal. To open lines of communications that could facilitate better communications about these projects. To be able to learn on an ongoing basis share what we learn with the diabetes online community. I think Scott and I were received with respect and developed a level of rapport that will facilitate our future writing on IDDP.

I will close with one point that Dr. Hehenberger made very clear in our conversations. The IDDP program isn’t just about financial investments in specific projects with for profit businesses. More significantly is a sharing and networking of JDRF contacts in a wide variety of labs with a similarly wide set of contacts in industry to help bring the two together to get promising research out of the lab.

Hopefully our meeting was a similar opportunity to bring transparency to IDDP efforts. I hope we can help those of us who Walk with JDRF for a Cure to see IDDP projects as a positive step in the mix of efforts JDRF facilitates.

¹ Kaitin K, Clinical Pharmacology & Therapeutics (2010) 87 3, 356–361. doi:10.1038/clpt.2009.293

Somewhere in the Pump Market

Sometimes I wonder if folks remember the stuff they write. Wait I better be careful, that is a standard that I could be guilty of not living up to too. My Ramblings May Vary.

That said I was interested by articles at massdevice.com. This week there was some gloom and doom about Medtronic restructuring and their commitment to diabetes devices. It quotes David Kliff of diabetic Investor saying "Looking ahead, it will be interesting to see whether the company is serious about change, or will they once again become bogged down in the Medtronic corporate meat grinder," Kliff wrote. "The folks at Medtronic corporate in Minneapolis view the diabetes unit as a cash cow and see little reason to invest precious dollars upgrading the Paradigm line or getting things right with the patch pump.”

I tend to be skeptical of just about everything so it isn’t real hard to think, maybe there is something to this. On the other hand there hasn’t been anything exactly innovative in the pump market in the last few years in the US since Medtronic integrated CGM into the pump. Sure Animas hooked up a meter remote but that isn’t exactly earth shattering. Despite rumors of Accu-Chek / Solo and Medtronic working on patch pumps nobody else had a pod like product on the market. So it easy to think, “Hey these guys (and gals) are getting lazy.”

Maybe but maybe not.

Last April the same blog, massdevice.com wrote that, “The Food & Drug Administration launches an initiative to institute stricter pre-market requirements infusion pumps, issuing new guidance and advising manufacturers they'll likely be required to "conduct additional risk assessments to support clearance of new or modified pumps."”

In Europe Medtronic has a pump / CGM combination available that shuts of insulin in hypos. Animas is expected to have a pump / CGM combination product for sale in Europe with Dexcom shortly. Even massdevice.com reports that Insulet has the much talked about Pod 2.0 into the FDA. Solo is, well, somewhere. So is Cellnovo.

Somewhere could be a lot of places. It could be back burnered as Kliff seems to suggest. There is a song about somewhere being over the rainbow. Somewhere is also, and I hope this is the case, being in with the FDA. Funny thing when stuff is into the FDA folks follow the rules, at least to some extent, and shut up.

Shutting up isn’t so great for bloggers. Well OK, in all honesty, there are a lot of folks at J&J, Medtronic, Roche, WaveSense, Sanofi, JDRF and the like that would probably think it would be GREAT to see me shut up. What I mean to suggest is that when the industry shuts up, news letters and blogs don’t have as much fodder to write about (but we as in I, Kliff and Massdevice.com keep writing anyway.)

I will continue to advocate for innovation. I have and will carp on about the pace on innovation and less than ideal products. I would love to see Mac OS software, data standards, devices that work together, the death of proprietary cables, respect for customer loyalty and a lot of other stuff that doesn’t seem all that hard.

However when the noise about a innovative product stops, does that mean it is dead or into the FDA for review? Honestly we don’t know.

Massdevice.com also noted that reported that Medtronic “diabetes sales were up 8 percent to $979 million during the first nine months of its fiscal year.” Not bad in this economy. So I emailed Medtronic about the news. I know they can’t talk about stuff that is into the FDA but they sent an official reply about restructuring. Here is their reply, Your Diabetes News May Vary.

“Yes, as Medtronic previously announced, we are globally restructuring our business to align with our customer needs and the current business environment. We are continuing to invest where business is growing and see strong growth in the diabetes division. However, we are restructuring the diabetes business in order to increasing operating efficiencies, while creating the infrastructure to grow and serve more customers.
 
“For example, we decided that a single facility focused solely on customer experience excellence will best meet the needs of our customers, so that means moving some positions from Northridge, CA to our Customer Care Center in San Antonio, TX. 
 
“Our customers can rest assured that we're committed to continuing to advance technology and innovation, while leading the industry in education, service and support to improve the lives of people with diabetes around the world.”

FTNW: JDRF and Amylin

From the News Wire: JDRF and Amylin Partner to IDDP to Investigate Co-Formulating Two Hormones for Treatment of Type 1 Diabetes

NEW YORK and SAN DIEGO, May 10, 2011 /PRNewswire/ -- The Juvenile Diabetes Research Foundation (JDRF) and Amylin Pharmaceuticals, Inc. (Nasdaq: AMLN) announced today that they have entered into a research collaboration agreement to provide financial support for a series of clinical studies to investigate the feasibility of mixing pramlintide, an analog of the human hormone amylin, with insulin to treat type 1 diabetes. Pramlintide, marketed by Amylin as SYMLIN® (pramlintide acetate) injection, is approved for use as an adjunct treatment in patients with diabetes who use mealtime insulin therapy and who have failed to achieve desired glucose control despite optimal insulin therapy. SYMLIN and insulin are currently not approved to be mixed and must be administered as separate injections.

Read the full press release here: http://www.prnewswire.com/news-releases/jdrf-and-amylin-partner-to-investigate-co-formulating-two-hormones-for-treatment-of-type-1-diabetes-121555613.html

Peeping in on Dexcom and the FDA

Dexcom released an earnings call yesterday.

Here is my voyeur's disclaimer: I listen to Dexcom’s earning calls more as Peeping Tom looking in a window into what seems to me to be strange courtship of of industry and the FDA around integrating of sensor and pumps. That is more interesting to me than the perspective of a Dexcom shareholder. I think my bias as a parent of diabetic kids is more pronounced than my vested interest in a few Dexcom shares. That said, as you read this, know I write from personal interests and make no bones about them.

This peep and tell used to be a lot easier when the call was available transcribed on Seeking Alpha. I recommend to anyone foolish enough to take my interpretation of the call as fair and balanced to re-think that and listen to the call themselves. They can find it here:
http://investor.shareholder.com/dexcom/events.cfm
While many, if not most folks may find earnings calls only slightly less dull than watching paint dry, listening for yourself will free you from my slant.

The call starts with new board members and a new president as symbols of plans to grow, whoopee. Wait I own shares that should be: Whoopee!

Somewhere in the call was a mention, in passing, of a type 2 CGM study. I opened another window to peep through and found a link and have posed an abstract of that study.

Dexcom talked about working with the FDA collaboratively to define what they need to do to get trials done for the gen 4 sensor. It sounds like Dexcom is hoping to get clarity from the FDA and have trials and the PMA for the 4th generation product started this year. This is significant because gen 4 is this product that will be paired up with pumps from Animas and Omni Pod. As I understood the call the gen 4 product studies are expected to take 60 to 90, under the heroic assumption that Dexcom has a clue about what the FDA wants. (Sorry, was that too sarcastic about FDA intent?) Dexcom is hoping to have a PMA supplement to the FDA by late summer.

Dexcom said on the call, they continue to expect the Animas / Dex integrated product to be introduced in Europe before it comes to market in the USA. In fact they said they expect the combo product to be marketed before the end of this quarter in Europe.

The Pod product is expected to be the new Pod and the gen 4 sensor. Neither of which as far as I can tell FDA blessed. So while both apparently are in works that sounds a bit further down the road. (Insert you favorite piece of FDA timing skepticism here.)

Dexcom seem to think that US products are in the time frame of end of this year or beginning of next year. If I am not mistaken that has been the expected time frame on each call back over a number of years. (Insert you favorite piece of product timing skepticism here. Which is why parents shouldn’t speculate on this stuff - but hey these tools are possibly a means to better lives for our kids - I mentioned we’re parents right?)

Hold not thy breath, lest ye die.


In the ‘interesting conversation’ side bar realm one of the issues the FDA may worry about is the risk of dosing insulin based on a device that isn’t indicated for that use and may not be sufficiently accurate. {Cough: meters} Far be it from me to point out that meter accuracy has been something that advocates have been calling for an improvement in since the late 80s.  Before the DCCT - the bench mark study on tight control taht got the world chasing tight control with meters that were not designed for that.

Granted meters may be more accurate than sensing but the simple fact of living with type 1 is that it is a balancing act. Part of that balance is coping with less than perfect sources of information. Sensing can ad trend to the over all mix of information. Trend can inform urgency. More importantly studies show sensing makes for better results.

So FDA repeat after me: "Perfect is not be the enemy of better."

I feel a number of my typical rants coming on and am looking around to borrow Glen Bech chalk board. If that isn’t a sign I have carried on too long I don’t know what is.

Diabetes Data Standards - Scholar's Edition

In the Journal of Diabetes Science and Technology Volume 5, Issue 3, May 2011

Rachel L. Richesson, Ph.D., M.P.H.

Abstract
Widespread adoption of electronic health records (EHRs) and expansion of patient registries present opportunities to improve patient care and population health and advance translational research. However, optimal integration of patient registries with EHR functions and aggregation of regional registries to support national or global analyses will require the use of standards. Currently, there are no standards for patient registries and no content standards for health care data collection or clinical research, including diabetes research. Data standards can facilitate new registry development by supporting reuse of well-defined data elements and data collection systems, and they can enable data aggregation for future research and discovery. This article introduces standardization topics relevant to diabetes patient registries, addresses issues related to the quality and use of registries and their integration with primary EHR data collection systems, and proposes strategies for implementation of data standards in diabetes research and management.
J Diabetes Sci Technol 2011;5(3):476-485

http://www.journalofdst.org/May2011/PDF/Abstract/VOL-5-3-SYM1-RICHESSON-ABSTRACT.pdf

CGM in Non Insulin Using T2

There is a study in the May 2011 issue of Journal of Diabetes Science and Technology.

Conclusions:
Real-time continuous glucose monitoring significantly improves A1C compared with SMBG in patients with T2DM not taking prandial insulin. This technology might benefit a wider population of people with diabetes than previously thought.

You can read an abstract at:

http://www.journalofdst.org/May2011/PDF/Abstract/VOL-5-3-ORG1-EHRHARDT-ABSTRACT.pdf

Gary Scheiner's Free Pizza Sampler!

Gary, Think Like a Pancreas, Scheiner as a short class on diabetes and pizza as a sample of the way classes at his Type 1 University work. Pizza is a challenge for a lot of folks so why not have Gary help demystify it.

Phil Southerland - "Not Dead Yet"

In the preface to his book, “Not Dead Yet” Phil Southerland asks, “Who am I to write a memoir when I am still in my twenties?”

'Cause you’re an aggressive, competitive, sometimes cocky, heck of an inspiration. Philbert.

This is the story of an ordinary kid. A kid with some passion and personal ambition who chased a dream and found some unexpected consequences while making other plans. I am not even sure that it is extraordinary that this kid brought type 1 diabetes, literally, along for the ride.

I am a parent to two type 1 diabetics. I find the most amazing thing about kids and adults successfully living with type 1 is their ability to put diabetes in its place. Second, close, but not first. It gets attention but not the title. Diabetes comes second to living life. These folks drag diabetes, kicking and screaming, into their dreams of the future. Dreams that diabetes at first seems to deny, yet in many enables. Dreams of acting, competing, writing, parenting, contributing and yes even professional bike racing. 

Phil’s is one of these story.  Diabetes, his family was told, would close off personal dreams and aspirations with blindness and early death. Ultimately it facilitated greater opportunities.  That the story unfolds in this book.

It is not the story of a master plan but a process of growth to a life beyond, in spite of and because of diabetes. It is a story with a positive supporting cast of halloween candy, Snickers, Oreo Blizzards, Super Big Gulps, burritos and characters that come with them. I encourage you to discover and experience them yourself. They brought me a lot of big smiles. 

As a type 1 parent I was particularly interested in reading how Phil viewed his mom’s roll in his life and with diabetes. I carefully make a distinction as we have rolls as parents and in diabetes management but they aren't always the same things. I see type 1 parents’ goal as raising kids to be creative, individuals actively purse their dreams as moral, useful contributors to society bringing diabetes along. That process isn’t flipping a switch and saying “Done!” it is gradual evolution. Seeing that process unfold in Phil’s life from the child, teen and adult’s perspectives is a uniquely valuable view for parents regardless of diabetes.

Phil’s story reinforces my belief that parenting diabetic kids isn’t about numbers. It is about the values that make for creative, active, useful, participant in life. On the rare occasion that Phil’s story talks about numbers it is to make points beyond them. About self care needed to facilitate the dreams. That Phil’s journey isn’t about numbers and is about discovering and channeling passions is a wonderful affirmation of brilliant parenting.

To a dad of two type 1 kids and two kids with their own challenging opportunities Phil hits real close to home when he writes about the risk of the non diabetic siblings becoming the others. That is something the diabetes parenting community doesn’t talk enough about. I worry that I am guilty of falling down in this area but take comfort in the idea that I fall down and stand up for the diabetics too. I hope my lack of parental perfection evens itself out.

A lack of perfection is one of the false gods of diabetes. We make idols to it and pray to those idols seeking comfort in numbers. In doing so we elevate numbers to goals and risk a myopia that totally misses the idea that variation can be part of a healthy learning process. Phil ranged about seeking  adventures on a bike. Numbers went along not in the pursuit of perfection but in the support of the freedom to roam.

One of my favorite lines from the book anticipates being less than perfect. It is brilliant, sage and simple advice, “A key part of being a diabetic is choosing trustworthy friends, reliable friends that you can depend on in a crisis.” I submit that is equally true if you take out ‘being a diabetic’ and insert ‘life’ but possibly more true for diabetics.

I think that a similar substitution is true of the book as a whole. It isn’t about diabetes it is about life. As such “Not Dead Yet” is great read for anyone whether they live with diabetes or not.

Through this book Phil becomes a friend. It is clear that, as a regular guy, he expresses friendship through good natured ribbing and honest, brief, airing of differences. So here friend, three hundred odd pages and other than a typo on page 272 (that is OK, I am good for a typo every sentence or two) I loved all but three words. The title.

To me type 1 isn’t about death it is about living. That I have such a different attitude from our family’s experiences than he does from his says a lot about the changes in expectations from Phil’s diagnosis in 1982 to our first twenty years later. So I affectionately recommend to Junior a better title, from last three words, of the first paragraph, on his second to last page.

“I will Live”

The problem with my suggestion is to see that title’s real value, you need to have traveled the previous three hundred and seven pages. Those pages share Phil’s story of how to really live. It is a great ride. I recommend it highly.

I was given an review copy. I intended, after reading, to raffle it off. Having finished it, I want the kids to read it. So sorry you will have to buy a copy.

I understand the book goes on sale Tuesday May 10. There is a Pre-Order link right up there in the spiffy banner the PR folks let me use.

I look forward to sharing comments and spoilers as folks get their own copies. 

What's In a Number?

I just read a comment from a parent who was disappointed by an A1C, in a kids full into puberty. It had gone up a half a point to the lower 8s. The parent was upset. Here is my reply:


This is the problem with numbers. They miss the point.


If there had been no number, you may well have been pleased to hear the doctor say that you, both of you, are coping with the swings of puberty as well as your endo could expect. That you are adapting and making changes appropriately. You could take pride in hearing nobody could have managed the diabetes better. You could have felt great about an active life style, that while it adds to the challenges of blood sugar management now, is a great life style habit that most Amarican's lack and will contribute greatly to years of healthy living.

I say it all the time, our goal as parents is to raise kids to be creative, actively purse their dreams as moral, useful contributors to their communities bringing diabetes along for the ride. None of that is about any one BG or A1C number. It is all about long term focus most of which has little to do with diabetes per se. All the diabetes management is a means to an end and that end isn't numbers it is quality of life that can't be expressed in a number.

Numbers aren't the point.

Raising kids to become great young adults is the goal.

Numbers, at best, are a means along the path to that end.

Be proud of every step to the real goal. You're doing fine.

Type 2 Autoimmune - Say What?

A Standford / U of Toronto study suggest that type 2 may have roots in an autoimmune process.

That link is http://med.stanford.edu/ism/2011/april/engleman.html and in part it says:

Nearly all type-2 diabetes drugs marketed today are designed to control a patient’s high blood sugar levels — a symptom of the body’s inability to respond properly to insulin. However, the researchers found that anti-CD20, which targets and eliminates mature B cells, could completely head off the development of type-2 diabetes in laboratory mice prone to the disorder and restore their blood sugar levels to normal.

As I understand this release, and let me be clear - I don't understand it, T cells and B cells inflame fatty tissues. This results in fat cells growing so rapidly that they exceed their blood supply and begin to die. To clean up the dieing cells the immune system creates macrophages. This process of inflammation, dieing and clean up inhibits the remaining fat cells ability to respond to insulin.

The significance is that this suggest a new process for type 2, an autoimmune process. The study suggest treatments based on this autoimmune reaction that are new ways of addressing type 2, specifically an antibody called anti-CD20. This antibody is already approved for use in humans to treat some blood cancers and autoimmune diseases.

Daniel Winer, DM of Stanford says, “We are in the process of redefining one of the most common diseases in America as an autoimmune disease, rather than a purely metabolic disease.”

As regular readers may have guessed,  mice were involved in the studies. How this translates to humans is an open issue. This statement warented it own paragraph in the relaease: "Despite the treatment’s effectiveness in mice, the researchers caution against assuming rituximab will work in humans with established type-2 diabetes."

More Studies are require.

YDMV.