Mister SASB is happy.

It isn’t often I get to say that we are closing in on a cure for a major disease. Much less on two. But if you or someone you care about has either diabetes or muscular sclerosis, I can tell you things are looking a heck of a lot better.

Let’s start with Diabetes.

BLOOD VESSELS KEY TO STOPPING DIABETES

In type 1 diabetes your body  can’t make its own insulin; in  type 2 it can but the insulin doesn’t get metabolized right  so your blood sugar goes out of control.  Neither one is great but, for my money, I would take my chances with type 2. I just would not like to live my life with type 1 diabetes.

Well guess what? Habib Zaghouani, PhD, J. Lavenia Edwards (Pediatrics at MU School of Medicine) has been working on a cure. He (and his colleagues) already figured  out how to get the immune system to stop destroying the islet cells in the pancreas that makes insulin.

The idea worked. But it didn’t work.

Yes, they were able to get the immune system to stop attacking islet cells, but, somehow, that wasn’t enough. Something else was killing those cells. After some deep thinking, Dr. Zaghouani realized that the immune system wasn’t attacking just the islet cells. It was also attacking their blood supply. Without a supply of blood, there can be no islet cells and the patient is still hosed.

Too bad, huh?

So Dr. Zaghouani tried injecting adult stem cells from the patient’s bone marrow into the pancreas. This stopped the immune system from attacking he blood vessels – an utterly amazing and flabbergasting result, don’t you think? Even Dr. Z was surprised but he didn’t seem too sad about it either. He’s off to the patent office with an application on the process.

Great, great news.

MULTIPLE SCLEROSIS DRUG LOOKS G-R-E-A-T

Here’s what’s happening.

Your nerves are protected by a sleeve or “sheath” made out of myelin which insulates it so it won’t “short out” and malfunction. If your immune system is confused enough to attack this sheath, you have  multiple sclerosis.  “Sclerosis” means the tissue is changing – becoming “sclerotic” or hardened. People with MS have trouble controlling their muscles or may have trouble with their senses. If things get bad, it can also affect the heart or lungs. It would be good if the immune system didn’t do that, right?

Dr. Stephen Miller (Northwestern University Feinberg School of Medicine and one of the senior authors of the article in Science Translational Medicine) thinks so too. His team has developed a treatment which “resets” the immune system. At the end of a “phase 1 clinical trial”, the results looked startling. The drug seems to be safe and well tolerated (the main purpose of phase 1 drug trials is to figure out if there will be side effects) and the drug cut down the immune systems tendency to attack the nerves dramatically. In the next trial – phase II – for which they are currently trying to raise money now, the goal is to stop the progress of MS in its tracks.

What does MISTER ScienceAintSoBad think of this? He thinks it’s cool beans.  I don’t know the total enrollment for the first human trials but Phase I trials aren’t usually huge so we’ll have to hope problems don’t crop up as more people are pulled into the studies. But this is great work.

ScienceAintSoBadRating = 10 for both of  these good applications of medical science.

Yay!

Note: This isn’t the last time I will write about huge progress about either of these diseases. Other researchers are taking different and equally interesting tacks.

I’ll keep on it, okay?

– – – – – – –

Drawing by me

SOMETHING FOR TYPE 1?

Sometimes my job sucks.

Occasionally, I hear from readers who say they’re sick.  “Science,” they say, “pah! Who CARES about ‘fascinating’ papers by science geeks ? I need a cure. Not a rat model.”

And- you know what? –  MISTER ScienceAintSoBad gets it.  He does. He knows “interesting” has to take a back seat to “it’s killin’ me.” How often do I find you a nice little pill to cure cancer? I don’t even have anything much for diarrhea.  I read through all kinds of crap about “breakthroughs”. Hah! Some breakthroughs! The mice feel better.  For humans? We’ll get back to you on that.

What a job!

Could be things are getting better. My last article, Alzheimer’s, Things Are Starting To Look Good, had little glitter balls of hope stuck all over it. And now  something about type 1 diabetes that could please you and add to my own optimism about less sick people to drag down Obamacare.  Seriously! It could happen faster than anyone thinks.

The Type 1 vs Type 2 thing

The thing about type 1 is that you can’t make insulin. The cells in the pancreas that  do this job get destroyed by the immune system  Without insulin your blood sugar climbs. If you don’t get yourself stabilized with supplementary insulin,  you won’t last. Even if you are a very good patient, you may have to work very hard at keeping your sugar level in a good range. If you are  lucky you may avoid some of the crappy complications but it won’t be easy. Type 2 usually comes on later in life and is because your body loses its ability to efficiently use the insulin that it produces. It’s not a lack of insulin; just can’t seem to use the stuff right. You wind up needing extra. You don’t want either type of diabetes but individuals with  type 1  often have a tougher challenge.  

What I saw was published in the journal Diabetes. (Dr. Jeffrey Bluestone, PhD and Dr. A.W. and Mary Clausen).  It’s about a drug called teplizumab which, in a very sophisticated way, keeps T-cells (the business end of the immune system) from going after the insulin producing cells on the pancreas. The strength of the results even surprised the scientists conducting the study; they were  “very excited by the efficacy”.

THE CATCH?

Well, here’s the thing. This IS exciting. (That’s what they said in the press release, right?) Great news. BUT these are only phase II trials on 52 kids under 15 years old. Half the kids did great. That’s why so much excitement. But what about the other kids? They didn’t do as well. The study says this might be because it works best where the insulin producing cells are still in fairly good shape. In other words,  where the disease hasn’t been active that long. But, come ON! These were teenagers with newly diagnosed disease. Obviously this part needs to be cleared up. Still. Such great results do justify moving right along to phase III. Great potential here.

By the way, this isn’t the only hope. There’s other research attacking the disease from other directions. That’s good. We don’t want to kick people under the bus just because they’ve been sick for a long time.

Vanquished!

Diabetes: cure.

INSULIN PRODUCING BETA CELLS PROTECTED IN FOUR YEAR STUDY

Let’s see what’s new.

Ah.  Here’s a study from the Juvenile Diabetes Research Foundation about a cure for diabetes.

Now I KNOW I’m dreaming!

A new drug, otelixizumab, is in phase III trials. (Phase III trials are the randomized, controlled, multicenter trials where you figure out if the drug really works).

Forty eight  months into the study, the insulin making cells of the eighty “type 1” patients in the study are still OK.

How’re you doin’? Hair standing up on the back of yer neck? Hands shakin’? If not you don’t get it. This is an atomic bomb! This is a breakthrough of a breakthrough! This is.. well… ScienceAintSoBadRating = 10.

MISTERScienceAintSoBad SHOULD know better than to give away 10’s like this. Something this important’s gotta be tested on more than 80 patients. Maybe the head’ll fall off of the 81st one (considered a setback). But DAMN this is neat!

Maybe there IS something to all this science stuff!

THAT'S one. (A pancreas.)

image from Creative Commons

Medicine: Type 1 Diabetes.

An artificial pancreas really is just over SOME horizon now. An announcement from Cambridge (article in the Lancet) describes the fine work of Dr Roman Hovorka at the University of Cambridge, working with a group of seventeen diabetic kids.

A cure for diabetes would be nice, of course. And there are some intriguing hints but, for now, an artificial pancreas would be stunning enough.

If it works out (and if it is widely accepted), this development has the potential to greatly reduce the complications of the disease, ease peoples’ lives, and reduce health care costs significantly (Wouldn’t THAT be nice?).

Back to which horizon this is over.

It’s hard for MISTER ScienceAintSoBad to say this but, once again, this is only a tantalizing possibility of something that is badly needed. We ARE a lot closer, thanks to the great work being done. Maybe as little are three, four or five years.

Karen Addington (Juvenile Diabetes Research Foundation) says this is a “proof of principle” and that we “need to redouble our efforts.”

I’m not sure that’s what we want to hear. But reality IS so darn real, isn’t it?

ScienceAintSoBadRating = 9

– – – – – – – – – Postscript – – – – – – – – –

Karen Addington was nice enough (and ubiquitous enough) to leave a comment which I am duplicating below since it addresses the question of when this device might really hit the road. Notice that her motivation is personal.

Her comment:

Thanks for picking up on this new research.

We know that developments like the artificial pancrease can’t come quickly enough for people living with type 1 diabetes, and their families. That’s why we’re working really hard to make sure that the artificial pancreas becomes a reality as soon as possible. In January my colleagues at JDRF International (based in the USA) announced a partnership with Animas (a Johnson & Johnson company that manufactures pumps) to develop a first generation artificial pancreas.

The goal is to develop a system that can prevent the extremes of both hypoglycaemia and hyperglycaemia automatically – but will still need some input from the wearer, such as informing the system of meal times and periods of exercise.

This partnership is planned for four years and by the end we hope to reach the point where it will ready to go forward for approval by regulatory bodies like the US Food and Drug Administration (FDA) and the European Medicines Agency (EMEA), and from there to people with type 1.

I have had type 1 diabetes for 30 years, and I’m really excited about what this research could soon mean for me, and everyone else with type 1.

Progress

Medicine: Reversing Diabetes.

Remember the difference between Type 1 Diabetes and Type 2?

Type 1’s the type where the pancreas just can’t produce insulin, period.

Without insulin you die.

So. If you’re a “Type 1” you can plan on a regimented life of becoming an expert on your own body, carefully monitoring your glucose levels, eating with scrupulous exactitude, and self administering calibrated amounts of insulin.

With motivation, discipline, and luck, Type 1’s can have great lives. Like our new Supreme Court Justice.

But who wants to be a poster child?

Better to cure the disease.

Step by step, this thing’s going away. I really believe that.

But what if you’re waiting for a cure, watching the damage accumulate in parts of your body? Well, good news! A monkey in Virginia has seen his Type 1 diabetes reversed for a year.

Not a thousand monkey. Not a hundred monkey. Not replicated by other labs (yet). And, most certainly, not in humans. But, according to the article in the Roanoke Times, we may be a few years away from human trials.

MISTER ScienceAintSoBad is ‘sposed to love science.

He does. He does.

But it can be so SLOWWWWW.

This good work was done (mainly) at Revivicor, a small private company in Virginia. To Revivicor and a monkey that’s feeling so much better, ScienceAintSoBadRating = 9 .

(Hey. I’ll bump you up to 10 for a cure.)