The conference officially ended with a farewell breakfast on Sunday morning. We extended our stay two more nights at the same conference discounted rate. I admit I loved being on site and able to ride the buses and enjoy the extra magic hours!
Sunday, we headed to Magic Kingdom as soon as we finished breakfast. We met my aunt & cousin too. We got on the Splash Mountain & Big Thunder Mountain first before the crowds were too long. We spent a few hours at the park and then left for a break. Ben and I headed back to the hotel for nap, but the older kids went to the Contemporary with my aunt to check out their pool since it was closer. My aunt had made reservations for the girls to go to tea, so the boys headed back to our hotel anyway to rest until the heat let up.
When we got back, the line at Space Mountain was too long. Members at CWD had recommended getting a Guest Assistance Card (GAC) for our diabetic children in the heat of summer. The heat alone does crazy things to their blood sugar & they may have to stop to deal with highs & lows & extra restroom breaks, etc. Disney agreed and had someone at the Coronado on Wednesday to help us with them. Our GAC allowed Andrew to wait in a cool place or use an alternate entrance. Roller coasters were an easy place for me to justify using the card. If he has to unplug his pump and leave it behind to ride the ride, he didn't need to spend forever standing around with no insulin. We only used it on roller coasters, but it really made a huge difference!
Saturday night, Ben made it until almost 9:00 & then started saying, "I want to go home (meaning the hotel)." Ryan had been out until midnight the night before so he decided to return to the hotel with me. The three of us and a stroller got to right in front of Cinderella's castle just in time for the Wishes Nighttime Spectacular. The lights in the park went down & there was literally no way we could make it through the crowd, so we just watched. It was an amazing light show that turned the castle different colors. Ryan is old enough to appreciate how difficult that was to do, and he was mesmerized! It's harder to have magical moments with kids as they get older, but, despite the fact that Ben was still crying to go home, that was my magical moment with Ryan. When the light show was over, we did try to get out of the park before the actual fireworks. We weren't entirely successful, so when we got into the bus poor Ben uttered, "Now we're safe." We tried to take him a store or restaurant to hide for any fireworks after that. The rest of the family enjoyed the Electrical Parade and stayed until midnight on Saturday. Sunday night, Ben made it until almost midnight, and the older kids enjoyed extra magic hours until nearly 3 am!
Sunday, July 31, 2011
Family Day at the Coronado
Saturday was Family Day at the Coronado Resort. We slept in a bit in the morning, checked out the FFL scavenger hunt, and then had a character lunch catered at the convention center. Mickey, Minney, Goofy, Donald, Woody, Jesse, and Chip and Dale were all there! The lunch was complete with great food, good photo ops, hula hoops, a DJ and dance floor, and lots of new friends!
After lunch, I took Ben back to the room for a nap, and Gene took the kids to ride the surrey! They had a wonderful time as you can see!
We could have spent the entire day just enjoying the resort. In the evening though, we couldn't resist the chance to see the magic at Magic Kingdom!
Saturday, July 30, 2011
Can We Prevent Type 1 Diabetes w/ Teplizumab?
Dr. Jay Skyler is the chairman of Type 1 Diabetes Trialnet, an international network conducting clinical trials to prevent type 1 diabetes or interdict the type 1 diabetes disease process. Kaitlyn is involved in Trialnet. Dr. Skyler did a great job teaching in this session. It was one of the hardest for me because it reminds me not of what Andrew goes through every day, but what most likely lies ahead for Kaitlyn.
He went through the process: genetic predisposition, trigger, autoimmunity antibodies, loss of first phase insulin response only detectable by IVGTT, glucose intolerance detectable by oral glucose tolerance test (OGTT), and diabetes. Once you hit OGTT glucose intolerance or dysglycemia, you have a 75 - 80% chance of developing type 1 within the next five years. That counter started ticking for Kaitlyn in December 2008.
Last summer, when Kaitlyn's numbers came back so high (194 at 2 hours where >200 is diabetes), I did quite a bit of research on Teplizumab and Diamyd. I read medical journals & did my best to decipher what it all meant even though much of it was over my head. My conclusions, for what it's worth, was that teplizumab did have an effect in preserving c-peptide, but the idea of cytokine release scared me. Diamyd was safer, but didn't seem to have much effect. Well, in December of last year, both drugs failed their clinical trials. I wasn't that surprised about Diamyd, but Teplizumab? Others online asked, "How can a drug prove effective in phase 2 trials and fail miserably in phase 3?"
Dr. Skyler answered these questions. I've lost my notes on this section, but here is what I remember. Diamyd failed, plain and simple. Teplizumab on the other hand faced two problems. The first was a poor choice in endpoints. Every trial has to preselect how success will be measured. That trial defined success as A1Cs under 6.5 and using less than typical amounts of insulin. The average A1Cs were 7.1, which though still good fails by the endpoint. The best thing Teplizumab does is preserve c-peptide. C-peptide is a measure of how much insulin you make yourself. Studies show that c-peptide > .2 puts a patient at reduced risk for hypoglycemia as well as eye and kidney problems. So a new study with sustained c-peptide as the end point could be successful.
The second challenge of the Teplizumab trial came from the company & FDA themselves. They wanted to reduce the effects of the cytokine release - headache, nausea, and other flu-like symptoms. So they reduced the dosage (again, this is from memory, but I'm fairly certain) from 45 mg in the effective phase 2 study to ...wait for it... 3 mg. Guess what? 3 mg didn't do much.
Also interesting was Dr. Skyler's comments on the cytokine release symptoms. He didn't see them as side effects since it isn't anything permanent. It was an expected intolerance of the body to the drug and proof it was doing what it was supposed to. I talked to Dr. Skyler afterwards and told him about Kaitlyn and asked him if he had any safety concerns about teplizumab and whether or not he would allow his daughter to take it. He is convinced it is safe. They have seen it preserve c-peptide in newly diagnosed patients, and it is most effective in the subgroup of children and those closest to diagnosis. For example, it worked better on those 6 weeks from diagnosis than those 12 weeks after diagnosis. The hope is what if we could stop the autoimmune attack right now when she does nothing and her A1C is 4.8? That would be wonderful.
You might worry that Dr. Skyler would only tell me what I wanted to hear. He didn't. I shared with him last year's results to her OGTT, but how this year's were close to normal. It hurt when he looked me in the eye and said, "Yes, it can fluctuate. However, at this point, we would consider your daughter at a 80-95% risk of developing type 1 in the next five years." Ouch!
Whether or not to do teplizumab is a decision we will likely have to make. Trialnet is doing a study trying to prevent diabetes altogether using teplizumab on people like Kaitlyn. The FDA requires a phase in of ages. They have to prove safety in the 18 and up population before taking 12 and up, and then 8 and up. Dr. Skyler has already requested from the FDA to take the younger crowd. Once the FDA approves 12 and up, he says that Kaitlyn's name will automatically show up to be contacted. It is being offered at Vanderbilt.
Please pray with us that we will know what to do. I know the decision is probably coming, but I'm sure we will be blind-sided. It will require daily infusions for a two week period. It is hard to know what side effects are really out there. I'm not a fan of drugs, especially experimental ones. On the other hand, every story I've read has been positive. Some are still making c-peptide 9 years later! I would love Kaitlyn to be spared this horrible disease. She is doing well right now. She started and ended her last OGTT in range, but did hit 208 in between. Her A1C is 4.8. I think it is miraculous that she is doing as well as she is. I believe it is the restraining hand of God on her life. Please pray that it continues to be so!
He went through the process: genetic predisposition, trigger, autoimmunity antibodies, loss of first phase insulin response only detectable by IVGTT, glucose intolerance detectable by oral glucose tolerance test (OGTT), and diabetes. Once you hit OGTT glucose intolerance or dysglycemia, you have a 75 - 80% chance of developing type 1 within the next five years. That counter started ticking for Kaitlyn in December 2008.
Last summer, when Kaitlyn's numbers came back so high (194 at 2 hours where >200 is diabetes), I did quite a bit of research on Teplizumab and Diamyd. I read medical journals & did my best to decipher what it all meant even though much of it was over my head. My conclusions, for what it's worth, was that teplizumab did have an effect in preserving c-peptide, but the idea of cytokine release scared me. Diamyd was safer, but didn't seem to have much effect. Well, in December of last year, both drugs failed their clinical trials. I wasn't that surprised about Diamyd, but Teplizumab? Others online asked, "How can a drug prove effective in phase 2 trials and fail miserably in phase 3?"
Dr. Skyler answered these questions. I've lost my notes on this section, but here is what I remember. Diamyd failed, plain and simple. Teplizumab on the other hand faced two problems. The first was a poor choice in endpoints. Every trial has to preselect how success will be measured. That trial defined success as A1Cs under 6.5 and using less than typical amounts of insulin. The average A1Cs were 7.1, which though still good fails by the endpoint. The best thing Teplizumab does is preserve c-peptide. C-peptide is a measure of how much insulin you make yourself. Studies show that c-peptide > .2 puts a patient at reduced risk for hypoglycemia as well as eye and kidney problems. So a new study with sustained c-peptide as the end point could be successful.
The second challenge of the Teplizumab trial came from the company & FDA themselves. They wanted to reduce the effects of the cytokine release - headache, nausea, and other flu-like symptoms. So they reduced the dosage (again, this is from memory, but I'm fairly certain) from 45 mg in the effective phase 2 study to ...wait for it... 3 mg. Guess what? 3 mg didn't do much.
Also interesting was Dr. Skyler's comments on the cytokine release symptoms. He didn't see them as side effects since it isn't anything permanent. It was an expected intolerance of the body to the drug and proof it was doing what it was supposed to. I talked to Dr. Skyler afterwards and told him about Kaitlyn and asked him if he had any safety concerns about teplizumab and whether or not he would allow his daughter to take it. He is convinced it is safe. They have seen it preserve c-peptide in newly diagnosed patients, and it is most effective in the subgroup of children and those closest to diagnosis. For example, it worked better on those 6 weeks from diagnosis than those 12 weeks after diagnosis. The hope is what if we could stop the autoimmune attack right now when she does nothing and her A1C is 4.8? That would be wonderful.
You might worry that Dr. Skyler would only tell me what I wanted to hear. He didn't. I shared with him last year's results to her OGTT, but how this year's were close to normal. It hurt when he looked me in the eye and said, "Yes, it can fluctuate. However, at this point, we would consider your daughter at a 80-95% risk of developing type 1 in the next five years." Ouch!
Whether or not to do teplizumab is a decision we will likely have to make. Trialnet is doing a study trying to prevent diabetes altogether using teplizumab on people like Kaitlyn. The FDA requires a phase in of ages. They have to prove safety in the 18 and up population before taking 12 and up, and then 8 and up. Dr. Skyler has already requested from the FDA to take the younger crowd. Once the FDA approves 12 and up, he says that Kaitlyn's name will automatically show up to be contacted. It is being offered at Vanderbilt.
Please pray with us that we will know what to do. I know the decision is probably coming, but I'm sure we will be blind-sided. It will require daily infusions for a two week period. It is hard to know what side effects are really out there. I'm not a fan of drugs, especially experimental ones. On the other hand, every story I've read has been positive. Some are still making c-peptide 9 years later! I would love Kaitlyn to be spared this horrible disease. She is doing well right now. She started and ended her last OGTT in range, but did hit 208 in between. Her A1C is 4.8. I think it is miraculous that she is doing as well as she is. I believe it is the restraining hand of God on her life. Please pray that it continues to be so!
Friday
Friday morning started with a breakfast buffet. Then Gene and I attended "Making Sense of Sensor Data" with Gary Schneiner. I was so excited to meet this author of Think Like A Pancreas! After a break, we attended "Can We Prevent Type 1 Diabetes" with Jay Skyler. That one will need its own post. Then we had lunch and one last trip to the Exhibit Hall. Then Gene and I attended "Advanced Pumping Strategies That Work" with John Walsh, author of Pumping Insulin. It was amazing! It included lots of tips like a 1% rise in TDD (total daily dose) will drop the average (which correlates to A1C) 4 points. Your TDD is too high if you have 2 low to high swings in a day. His book is also full of great formulas like this. Whenever I need to just start over for Andrew, I pull out his book. An example of this will be in a few weeks. Andrew starts baseball practices Monday. Then he goes back to school in a couple weeks. He will run high the first week because of the excitement and nerves of something new. Then, he will settle into the routine of higher activity. At least I think so - there isn't much recess in 5th grade. This will require more than small tweaking. I'll sit down with the formulas and his schedule (P.E. is after lunch this year) and essentially start over. My job will be easier because of the teaching of our great nurse at Vanderbilt and John Walsh. After this session was a break and then the Closing Keynote by Jay Hewitt. He is an Ironman Triathlete who began training for the Ironman after he was diagnosed with type 1 diabetes. He was a very inspiring man and speaker!
Ryan and the other teens spent the day at Hollywood Studios. They were well supervised and had a great time. They were back by the time we got out of the keynote, so we all went to the pool for a little while. The teens had a dinner dance from 8 until midnight, and the tweens had a tween social from 8 until 11. Andrew ate the ice cream and left. Kaitlyn and Ryan had fun and stayed until the bitter end! Again, I didn't have that much to do, but I was tired waiting up for my teen and pre-teen. There are definitely scary days ahead!
Ryan and the other teens spent the day at Hollywood Studios. They were well supervised and had a great time. They were back by the time we got out of the keynote, so we all went to the pool for a little while. The teens had a dinner dance from 8 until midnight, and the tweens had a tween social from 8 until 11. Andrew ate the ice cream and left. Kaitlyn and Ryan had fun and stayed until the bitter end! Again, I didn't have that much to do, but I was tired waiting up for my teen and pre-teen. There are definitely scary days ahead!
Epcot Thursday Night
After Test Track, we headed to the countries. Then Andrew's pump alarm went off. Out of insulin. Yes, there had been a warning. I think it was at breakfast. We had plenty left at the time. We just forgot to put more in before heading to Epcot. For dessert. His blood sugar was around 150. It was already evening and they closed at 9:00. I offered to take just Andrew back to the hotel, but Gene wanted everyone to stay together & all stay or all leave. I chose to stay. Even though the pump said it was empty, there is still some insulin in the bottom of the cartridge. I gave Andrew the option of taking a shot or saving his dessert until he got back to the room. He chose waiting until the dessert was staring him in the face. Then he wanted the shot.
Ben loved the movie in France. I thought he might be bored, so I started quietly pointing things out to him in the opening scenes. He was so fascinated! I loved watching him watch the movie! Then we headed over to the American movie, which was to get out right at 8:45 in time for the IllumiNations show. When we got out, though, it was POURING down rain. Not raining. Pouring! Ben was tired. Andrew's blood sugar was high. Ben didn't like fireworks, so we just ran for the exit. From the back of the park. With one umbrella, five kids, and a stroller. Worried about whether Andrew should be exercising at all. Was he making ketones? The ending was rough, but we had enjoyed most of Epcot up to that point. Once we got back to the room, all was well. Andrew's blood sugar came down fine as soon as he got a new site and insulin. And, no. No ketones this time.
The Artificial Pancreas
diaTribe talked quite a bit about the Artificial Pancreas on Wednesday and so did Stu Weinzimer & Ed Damiano from JDRF on Thursday. I skipped Wednesday's session because of arriving late & missed the Thursday session because of Will's eye, but Gene attended both. This post comes from my understanding, Gene's handouts, and the FDA's website.
An artificial pancreas pairs the continuous glucose monitor & insulin pump like the ones that Andrew already wears with a control algorithm that makes changes to his insulin delivery as the data comes in. We already have experimental versions of this. Many trials ask the patient to control their diabetes with set portion sizes and activity one day and then allows the computer to control insulin delivery with the same food & activity the next. The computer algorithm consistently outperforms people. However, the trials are still highly supervised in a hospital setting. They may be moving to a clinical setting, and we will know we are making progress when they get approved for home trial.
There is some danger in giving technology control over insulin delivery when too much insulin or too little insulin can be so dangerous so quickly. The FDA in June 2011 issued a statement of support of developing an artificial pancreas and provided guidelines to companies seeking to develop one. There will be several types of artificial pancreas device systems. The first is a reactive low glucose suspend system. When the cgm detects very low blood sugars and no response from the user, it suspends insulin delivery. It's primary function is to save lives of people who are really low and asleep or passed out and unable to respond to the low. Since we have a system in place to wake me when Andrew is low, I don't expect this first stage to help Andrew's glucose control at all. It will be an important first step in beginning to trust the cgm technology and will mean the next few steps are on the way. A predictive low glucose suspend system will antipate lows and temporarily suspend insulin to prevent lows. Since low blood sugars often rebound to highs, preventing lows would improve patient control and take away some of the fear of embarassing low blood sugars.
I'm even more excited about the treat-to-range or treat-to-target systems. The treat-to-range system system not only prevents lows but prevents high blood sugars as well. You specify the blood sugar range you wish to stay between and the pump dispenses more or less insulin as you approach the high or low end of your range. According to the FDA descriptions, these pumps would still require the patient to check blood sugars & bolus for meals. This sounds to me like what we once told would be a "minimizer" pump, but I don't really hear that terminology much anymore. The treat-to-target systems would be fully automated, requiring no boluses or carb counting. You would calibrate the CGM twice a day with a finger prick & then go about your day staying in target. Is this possible? Yes! Currently, trials are achieving 70%+ time in target with often 90%+ overnight control. The data I saw (See the Future of the Cure Post) showed the patient going slightly over 200 after eating & not bolusing and hitting 68 after exercising and only eating salad for lunch. That was still better than what he did on his own under the same conditions.
The FDA already envisions three types of treat-to-target systems. The first is insulin only, which just adjusts insulin amounts to achieve the results. This is working now in trials & would work even better with faster acting insulin. However, our bodies use a combination of insulin and glucagon to control our glucose levels, so scientists are beginning to conceive of bi-hormonal pumps that give insulin to treat high glucose levels and faster-acting glucagon or similar hormone to raise low glucose. It will take longer to approve through the FDA since there is no currently approved glucagon pump. The last treat-to-target system is a hybrid system that while still fully automated, allows patients to supplement insulin pre-meal to prevent post-meal spikes. I like this idea. Currently, cgms measure interstitial fluid which lags behind blood glucose by about 10 minutes. Then boluses of insulin take about 15 minutes to absorb and start working. So, in an automated system, the insulin would begin working about 25 minutes after ingesting the carbs. Pre-bolusing is important now, so I like the idea of keeping the capability.
Rumor has it that Dexcom has a new CGM that is smaller & more accurate that is pending FDA approval. Insulin pumps have already been approved, so that isn't an obstacle. I don't see anything new coming out until we see the new, more accurate sensors approved. I expect to see low glucose suspend systems next, etc.
Is the artificial pancreas a cure? NO!!! Andrew already wears a pump and a cgm, and I have seen both fail. Being connected to life support is not a cure. I have even seen angry posts from patients toward JDRF for investing so much into the artificial pancreas. I don't agree with them either. I want a cure! In the meantime, however, God has allowed Andrew to have type 1 diabetes. We have to do the best we can to manage it. Andrew already wears the pump and the cgm. Would I like them to talk to each other and prevent those high and low blood sugars that take their toll on his little body? YES! Insulin isn't a cure, but a treatment, but I'm so glad to have it and to see Andrew alive and healthy. The artificial pancreas won't be a cure, but I'd be so glad to have it and to see Andrew alive, healthy, in range, not afraid of lows, and at less risk for the complications of diabetes that come from high blood sugar! So, in my opinion, bring on the artificial pancreas!
An artificial pancreas pairs the continuous glucose monitor & insulin pump like the ones that Andrew already wears with a control algorithm that makes changes to his insulin delivery as the data comes in. We already have experimental versions of this. Many trials ask the patient to control their diabetes with set portion sizes and activity one day and then allows the computer to control insulin delivery with the same food & activity the next. The computer algorithm consistently outperforms people. However, the trials are still highly supervised in a hospital setting. They may be moving to a clinical setting, and we will know we are making progress when they get approved for home trial.
There is some danger in giving technology control over insulin delivery when too much insulin or too little insulin can be so dangerous so quickly. The FDA in June 2011 issued a statement of support of developing an artificial pancreas and provided guidelines to companies seeking to develop one. There will be several types of artificial pancreas device systems. The first is a reactive low glucose suspend system. When the cgm detects very low blood sugars and no response from the user, it suspends insulin delivery. It's primary function is to save lives of people who are really low and asleep or passed out and unable to respond to the low. Since we have a system in place to wake me when Andrew is low, I don't expect this first stage to help Andrew's glucose control at all. It will be an important first step in beginning to trust the cgm technology and will mean the next few steps are on the way. A predictive low glucose suspend system will antipate lows and temporarily suspend insulin to prevent lows. Since low blood sugars often rebound to highs, preventing lows would improve patient control and take away some of the fear of embarassing low blood sugars.
I'm even more excited about the treat-to-range or treat-to-target systems. The treat-to-range system system not only prevents lows but prevents high blood sugars as well. You specify the blood sugar range you wish to stay between and the pump dispenses more or less insulin as you approach the high or low end of your range. According to the FDA descriptions, these pumps would still require the patient to check blood sugars & bolus for meals. This sounds to me like what we once told would be a "minimizer" pump, but I don't really hear that terminology much anymore. The treat-to-target systems would be fully automated, requiring no boluses or carb counting. You would calibrate the CGM twice a day with a finger prick & then go about your day staying in target. Is this possible? Yes! Currently, trials are achieving 70%+ time in target with often 90%+ overnight control. The data I saw (See the Future of the Cure Post) showed the patient going slightly over 200 after eating & not bolusing and hitting 68 after exercising and only eating salad for lunch. That was still better than what he did on his own under the same conditions.
The FDA already envisions three types of treat-to-target systems. The first is insulin only, which just adjusts insulin amounts to achieve the results. This is working now in trials & would work even better with faster acting insulin. However, our bodies use a combination of insulin and glucagon to control our glucose levels, so scientists are beginning to conceive of bi-hormonal pumps that give insulin to treat high glucose levels and faster-acting glucagon or similar hormone to raise low glucose. It will take longer to approve through the FDA since there is no currently approved glucagon pump. The last treat-to-target system is a hybrid system that while still fully automated, allows patients to supplement insulin pre-meal to prevent post-meal spikes. I like this idea. Currently, cgms measure interstitial fluid which lags behind blood glucose by about 10 minutes. Then boluses of insulin take about 15 minutes to absorb and start working. So, in an automated system, the insulin would begin working about 25 minutes after ingesting the carbs. Pre-bolusing is important now, so I like the idea of keeping the capability.
Rumor has it that Dexcom has a new CGM that is smaller & more accurate that is pending FDA approval. Insulin pumps have already been approved, so that isn't an obstacle. I don't see anything new coming out until we see the new, more accurate sensors approved. I expect to see low glucose suspend systems next, etc.
Is the artificial pancreas a cure? NO!!! Andrew already wears a pump and a cgm, and I have seen both fail. Being connected to life support is not a cure. I have even seen angry posts from patients toward JDRF for investing so much into the artificial pancreas. I don't agree with them either. I want a cure! In the meantime, however, God has allowed Andrew to have type 1 diabetes. We have to do the best we can to manage it. Andrew already wears the pump and the cgm. Would I like them to talk to each other and prevent those high and low blood sugars that take their toll on his little body? YES! Insulin isn't a cure, but a treatment, but I'm so glad to have it and to see Andrew alive and healthy. The artificial pancreas won't be a cure, but I'd be so glad to have it and to see Andrew alive, healthy, in range, not afraid of lows, and at less risk for the complications of diabetes that come from high blood sugar! So, in my opinion, bring on the artificial pancreas!
Friday, July 29, 2011
What We Learned From the Stem Cell Session
Disclaimer: I took notes & have studied, so I believe all of these facts to be accurate. However, I am a mom not a doctor or scientist!
Islet cell translants have been very successful. Over 1000 transplants have been done and >90% are insulin free after one year. Sounds great, right? The problem is we have no way to get enough islets to help everyone. The natural leap is to look to stem cells to become islet cells.
There are several types of stem cells. Mesenchymal stem cells (MSC) can develop into tissue and can be found in bone marrow & dental pulp. Hematopoietic stem cells (HSC) can develop into all the blood cell types and are found in cord blood and also in bone marrow. There are also liver stem cells which are similar to pancreatic cells. In mice, human c-peptide has been detected from transplanted liver cells. Embryonic Stem Cells (ES) are very powerful, but the moral issues there are huge and it simply isn't necessary to use them at all. Scientists have found that they can reprogram skin cells by releasing genes and essentially taking them back in time. This creates cells similar to embryonic stem cells, but you can get these cells from the patient!
To do this, we have to consider genes. Our DNA has thousands of genes. When proteins are needed, genes are transcribed into RNA, and the proteins are built based on the code in the RNA. In the past, genes have been shuttled with viruses. However, if you split a cancer preventing gene, you will get cancer. You really don't want to use viruses! So, they tried to use the proteins themselves, but they can't get inside the cells. They program a key (amino acid) that can get into a cell and, once inside, turn the cell into whatever they need it to be. Scientists use this protein technology to create stem cells, educate stem cells, and then reprogram adult cells into islets.
This research still isn't in patients yet. It isn't safe yet. You want to teach cells to replicate & create lots of islet cells, but if you have out of control growth, it becomes cancer. They are working on a shut off switch too. Only weeks ago, a group found a way to send Messenger RNA to tell cells to shut down.
They have also learned that islet cells make up 2% of the pancreas (98% makes digestive juices), and yet islet cells get 1/4 of the oxygen supply of the entire pancreas! In isolating islet cells, they found that islets die without high levels of oxygen. This seems really interesting to me, and seems worth pondering.
I was probably speculating what causes reduced oxygen in the pancreas, when I missed the full quote, but he mentioned something about still having a 20% incidence of benigh tumors. I think he was talking about in the mice trials. So, would I trust stem cell research done in other countries? No! There is still too much to learn, but they are learning quickly. Perhaps the day will come though, that doctors can take Andrew's skin cells, reprogram them in a lab to become islet cells, allow them to replicate, insert the killer RNA to shut down anything other than healthy islet cells, and safely transplant those cells without need for rejection medication. Those cells could provide enough insulin for him to be insulin free for at least up to a year. Even without fixing the autoimmune attack, if healthy insulin secretion could be attained in a once a year, safe, out-patient procedure, that would be pretty close to a cure. Better yet, would be shutting off the autoimmune attack and having a once-for-all procedure to restore insulin production and cure diabetes.
Islet cell translants have been very successful. Over 1000 transplants have been done and >90% are insulin free after one year. Sounds great, right? The problem is we have no way to get enough islets to help everyone. The natural leap is to look to stem cells to become islet cells.
There are several types of stem cells. Mesenchymal stem cells (MSC) can develop into tissue and can be found in bone marrow & dental pulp. Hematopoietic stem cells (HSC) can develop into all the blood cell types and are found in cord blood and also in bone marrow. There are also liver stem cells which are similar to pancreatic cells. In mice, human c-peptide has been detected from transplanted liver cells. Embryonic Stem Cells (ES) are very powerful, but the moral issues there are huge and it simply isn't necessary to use them at all. Scientists have found that they can reprogram skin cells by releasing genes and essentially taking them back in time. This creates cells similar to embryonic stem cells, but you can get these cells from the patient!
To do this, we have to consider genes. Our DNA has thousands of genes. When proteins are needed, genes are transcribed into RNA, and the proteins are built based on the code in the RNA. In the past, genes have been shuttled with viruses. However, if you split a cancer preventing gene, you will get cancer. You really don't want to use viruses! So, they tried to use the proteins themselves, but they can't get inside the cells. They program a key (amino acid) that can get into a cell and, once inside, turn the cell into whatever they need it to be. Scientists use this protein technology to create stem cells, educate stem cells, and then reprogram adult cells into islets.
This research still isn't in patients yet. It isn't safe yet. You want to teach cells to replicate & create lots of islet cells, but if you have out of control growth, it becomes cancer. They are working on a shut off switch too. Only weeks ago, a group found a way to send Messenger RNA to tell cells to shut down.
They have also learned that islet cells make up 2% of the pancreas (98% makes digestive juices), and yet islet cells get 1/4 of the oxygen supply of the entire pancreas! In isolating islet cells, they found that islets die without high levels of oxygen. This seems really interesting to me, and seems worth pondering.
I was probably speculating what causes reduced oxygen in the pancreas, when I missed the full quote, but he mentioned something about still having a 20% incidence of benigh tumors. I think he was talking about in the mice trials. So, would I trust stem cell research done in other countries? No! There is still too much to learn, but they are learning quickly. Perhaps the day will come though, that doctors can take Andrew's skin cells, reprogram them in a lab to become islet cells, allow them to replicate, insert the killer RNA to shut down anything other than healthy islet cells, and safely transplant those cells without need for rejection medication. Those cells could provide enough insulin for him to be insulin free for at least up to a year. Even without fixing the autoimmune attack, if healthy insulin secretion could be attained in a once a year, safe, out-patient procedure, that would be pretty close to a cure. Better yet, would be shutting off the autoimmune attack and having a once-for-all procedure to restore insulin production and cure diabetes.
Thursday's Schedule
8:00 Breakfast - We started Thursday morning with a wonderful breakfast. There were buffet lines in the hall & then large rooms with tables for 10 so that you could visit with other families as you ate. Of course, we take up 7 of the 10 chairs, so we always met couples with only one child. I enjoyed each and every conversation.
9:00 Opening Keynote Richard Rubin & Oliver Double on "The Importance of Humor". Oliver Double's song "Think Like A Pancreas" was hilarious & one of Andrew's favorite parts of the entire conference. I wish I could find it on youtube!
10:00 Break; Ben and Ryan had reported to classes at 9:00, but Kaitlyn, Andrew, and Will found their classes after the break. Kaitlyn & Andrew were Tweens & Will was an elementary student.
10:45 DRI Research Update 1: Stem Cells: New Sources, More Options
We could choose from lots of sessions. This is the one Gene and I both chose. I'll write about what I learned in this session in its own post.
During this session, Will was running with his nametag on, when it swung up and hit him in the ... EYE! It was really hurting, but I was hoping it would go away over lunch...
12:00 Lunch Buffet
After lunch, Will wasn't feeling any better. There was an eye doctor doing retinal screenings for the type 1's, so I took Will there. They checked Will's eye & said they didn't think he had scratched the retina, but that with it still bothering him, he should go rest in the dark.
1:30 Will & I go to the hotel room; Gene attends "The Artificial Pancreas Project."
2:45 Snack Break - Ben loved the Mickey Bars!
3:30 DRI Research Update 2: Tissue Engineering & Immunology: New Ways To Protect Islets. Will felt well enough to return to his class. Yeah!
6:30 Family & Friends Banquet with Crystal Bowersox - we didn't go! Tickets were $25 each for seven of us. We were already getting Disney tickets, so it was easier to add an extra ticket for everyone. So we headed to EPCOT!
9:00 Opening Keynote Richard Rubin & Oliver Double on "The Importance of Humor". Oliver Double's song "Think Like A Pancreas" was hilarious & one of Andrew's favorite parts of the entire conference. I wish I could find it on youtube!
10:00 Break; Ben and Ryan had reported to classes at 9:00, but Kaitlyn, Andrew, and Will found their classes after the break. Kaitlyn & Andrew were Tweens & Will was an elementary student.
10:45 DRI Research Update 1: Stem Cells: New Sources, More Options
We could choose from lots of sessions. This is the one Gene and I both chose. I'll write about what I learned in this session in its own post.
During this session, Will was running with his nametag on, when it swung up and hit him in the ... EYE! It was really hurting, but I was hoping it would go away over lunch...
12:00 Lunch Buffet
After lunch, Will wasn't feeling any better. There was an eye doctor doing retinal screenings for the type 1's, so I took Will there. They checked Will's eye & said they didn't think he had scratched the retina, but that with it still bothering him, he should go rest in the dark.
1:30 Will & I go to the hotel room; Gene attends "The Artificial Pancreas Project."
3:30 DRI Research Update 2: Tissue Engineering & Immunology: New Ways To Protect Islets. Will felt well enough to return to his class. Yeah!
6:30 Family & Friends Banquet with Crystal Bowersox - we didn't go! Tickets were $25 each for seven of us. We were already getting Disney tickets, so it was easier to add an extra ticket for everyone. So we headed to EPCOT!
Thursday, July 28, 2011
Wedneday at FFL (Friends For Life Conference)
Wednesday, we got up & packed the van again to drive back to the Coronado Resort & Convention Center at Disney. We got there around noon. The resort was beautiful!
Thursday and Friday were the main teaching days of the conference, but you could register for sessions on Wednesday as well. Gene and I had both signed up for a 1:00 session, so we had hoped to get there earlier, but it was hard to get going after such a long day the day before. Gene decided to go to the session (Diatribe- Targeting a Cure for Type 1 Diabetes: How Long Will We Have To Wait?) while I registered at the conference, got the GAC (again more later), met with Medtronic about evaluating their pump (Andrew still prefers Animas), and then unpacked in our room. At 3:00, we went to the first timers reception. We not only learned about the conference, but they split us up by region. We met another family from Alabama and then the former NFL football player Kendall Simmons joined us! He was so friendly and down to earth, and a great example to the kids!
That evening was the grand opening of the Exhibit Hall. You could visit every pump company out there. I also remember FRIO, Spi-belt, Trialnet, & Dex-4. I even saw one I hadn't considered before. The kids got books on diabetes, coloring books, t-shirts, toys, meters, etc. My favorite was the Nova MAX Plus ketone meter. It checks glucose or ketones & doesn't require any coding to go between them. I like it much better than the Precision Xtra. Blood ketones are so much more accurate than urine ketones, but the strips cost so much ($5 each, not covered by our insurance) that we only use them when Andrew is sick. Free strips are like gold! They had lots of games & goodies for the kids, so they all had a great time. The sugar-free sno-cones were Andrew's favorite. It was the best selection of flavors that he could have that he had ever seen!
Ryan, 13, had a teen ice breaker from 9 - 10 pm. The teens were going to Hollywood Studios on Friday and had to stay with a partner, so they needed to get started making friends! Ryan had a great time. I think it was my first experience staying awake waiting for my teenager to come home.
Thursday and Friday were the main teaching days of the conference, but you could register for sessions on Wednesday as well. Gene and I had both signed up for a 1:00 session, so we had hoped to get there earlier, but it was hard to get going after such a long day the day before. Gene decided to go to the session (Diatribe- Targeting a Cure for Type 1 Diabetes: How Long Will We Have To Wait?) while I registered at the conference, got the GAC (again more later), met with Medtronic about evaluating their pump (Andrew still prefers Animas), and then unpacked in our room. At 3:00, we went to the first timers reception. We not only learned about the conference, but they split us up by region. We met another family from Alabama and then the former NFL football player Kendall Simmons joined us! He was so friendly and down to earth, and a great example to the kids!
That evening was the grand opening of the Exhibit Hall. You could visit every pump company out there. I also remember FRIO, Spi-belt, Trialnet, & Dex-4. I even saw one I hadn't considered before. The kids got books on diabetes, coloring books, t-shirts, toys, meters, etc. My favorite was the Nova MAX Plus ketone meter. It checks glucose or ketones & doesn't require any coding to go between them. I like it much better than the Precision Xtra. Blood ketones are so much more accurate than urine ketones, but the strips cost so much ($5 each, not covered by our insurance) that we only use them when Andrew is sick. Free strips are like gold! They had lots of games & goodies for the kids, so they all had a great time. The sugar-free sno-cones were Andrew's favorite. It was the best selection of flavors that he could have that he had ever seen!
Ryan, 13, had a teen ice breaker from 9 - 10 pm. The teens were going to Hollywood Studios on Friday and had to stay with a partner, so they needed to get started making friends! Ryan had a great time. I think it was my first experience staying awake waiting for my teenager to come home.
Pre-Conference
We headed out on Sunday, July 3rd, and the kids travelled really well. We warned Ben that we drive from before the sun came up until after the sun went down. That worked out pretty well. He has driven me crazy asking about how much longer to get to the ball field before, but he didn't ask on the way to Florida. We occasionally commented about the sun still being up. We stopped for lunch, gas, dinner, and gas. We left around 6:30 a.m. Central time & arrived at my aunt's home in Vero Beach at 9:30 p.m. Eastern time. It was a long day, but well worth it!
July 4th, we headed to the beach, and all got horribly burnt. Yes, I put sunscreen on the kids. No, it wasn't enough. Ben didn't care much for the beach. He was quickly afraid of the waves & begged me to take him out. On the way, a wave surprised me and toppled me over. I knew I couldn't let him go under without him being traumatized, so I landed full-force on my knees to steady myself and hold his head above the water. I managed to scrape my knee enough for it to bleed pretty freely, so he was still traumatized. Aunt Estelle has convinced him that they "fixed the hole at the beach" and it is safe now.
Tuesday, our family, my aunt, and her granddaughter (I'll call her K) drove two hours up to Orlando for a day at Animal Kingdom. I had never been. We had such a great time!!! K led us straight to the back of the park and we went on the Kilimanjaro Safari first. The animals were all out & the sight was spectacular! Then we headed over to Expedition Everest, which had only a 15 minute wait! Gene had taken the tickets to get fastpasses, so Andrew didn't disconnect his pump until we were ready to ride the roller coaster. Pumps aren't supposed to go on roller coasters because of magnetic forces. I had heard/read that Andrew could give the $7000 pump to one of the cast members & pick it up after the ride. They said NO! I was so upset! I could have just held the pump & missed the ride, but it was the first real roller coaster for my kids & I hated to miss it. Finally, another cast member agreed to leave it at the exit, even though "they couldn't be responsible for it." That was good enough for me, and we were off! We grabbed the pump at the end, gave it to Gene & rode it again with still very little wait. We never asked anyone to hold the pump again. Gene isn't fond of roller coasters & someone had to stay with Ben anyway. Usually, Andrew gave the pump to Gene before getting in line. I'll explain the GAC later, but for those who already know about it, we did not have one this day. We enjoyed our day at Animal Kingdom. I think because it was first, and because we were all relatively rested, it was one of the most magical days for me! It was also the day that Ben met Mickey Mouse for the very first time!
July 4th, we headed to the beach, and all got horribly burnt. Yes, I put sunscreen on the kids. No, it wasn't enough. Ben didn't care much for the beach. He was quickly afraid of the waves & begged me to take him out. On the way, a wave surprised me and toppled me over. I knew I couldn't let him go under without him being traumatized, so I landed full-force on my knees to steady myself and hold his head above the water. I managed to scrape my knee enough for it to bleed pretty freely, so he was still traumatized. Aunt Estelle has convinced him that they "fixed the hole at the beach" and it is safe now.
Tuesday, our family, my aunt, and her granddaughter (I'll call her K) drove two hours up to Orlando for a day at Animal Kingdom. I had never been. We had such a great time!!! K led us straight to the back of the park and we went on the Kilimanjaro Safari first. The animals were all out & the sight was spectacular! Then we headed over to Expedition Everest, which had only a 15 minute wait! Gene had taken the tickets to get fastpasses, so Andrew didn't disconnect his pump until we were ready to ride the roller coaster. Pumps aren't supposed to go on roller coasters because of magnetic forces. I had heard/read that Andrew could give the $7000 pump to one of the cast members & pick it up after the ride. They said NO! I was so upset! I could have just held the pump & missed the ride, but it was the first real roller coaster for my kids & I hated to miss it. Finally, another cast member agreed to leave it at the exit, even though "they couldn't be responsible for it." That was good enough for me, and we were off! We grabbed the pump at the end, gave it to Gene & rode it again with still very little wait. We never asked anyone to hold the pump again. Gene isn't fond of roller coasters & someone had to stay with Ben anyway. Usually, Andrew gave the pump to Gene before getting in line. I'll explain the GAC later, but for those who already know about it, we did not have one this day. We enjoyed our day at Animal Kingdom. I think because it was first, and because we were all relatively rested, it was one of the most magical days for me! It was also the day that Ben met Mickey Mouse for the very first time!
Monday, July 25, 2011
Ben & the conference?
My aunt lives a couple hours from Orlando. She argued that she should keep Ben during the conference since he is too young (he was three in May) to get much out of it. He could stay on a routine, take naps, etc. We were going to sneak in a little Disney "big kid style" before bringing Ben back to see Magic Kingdom and Animal Kingdom. We told Ben that he would stay with her while we went to diabetes meetings, but then we would come back & take him to see Mickey Mouse. However, my aunt started having horrible migraines in May and realized that she might not be able to care for Ben. We signed up Ben for childcare on the last possible day. CWD was so wonderful about adding Ben. My aunt is doing better, but Ben was ready to come now! We ended up sticking with the plan to see some of Disney after the conference, return to my aunt's home to rest, and then travel back for a couple more days at Disney World.
Sunday, July 24, 2011
Looking for a new family camp
I've wanted to write this blog since we missed a week of school due to snow in January! I had promised the kids that we would go back to family camp that was supposed to be right after school got out. In January, we had a wonderful snow. I enjoyed it, but, since I work in a different system, every snow day pushed my school year further and further into family camp. I got out of school in time for the last day of family camp. I searched the internet for family diabetes camps and realized that we have something very special and family camps are rare. I did, however, find a conference we could all attend ... in Disney World! It was the Friends For Life International Children With Diabetes Conference on July 5-10, 2011. I'll take a few posts to tell the story, but we had a wonderful time!
Friday, July 1, 2011
Dr. Faustman Interview
Dr. Faustman believes that if in the presence of TNF (tumor necrosis factor), the bad T cells that attack the pancreas will die. She is attempting to raise TNF with a safe BCG vaccine used for tuberculosis. Her sample size was incredibly small and her patients didn't require less insulin or go into remission. Still, I think her study may change the course of diabetes research. She is one of the few researchers to attempt to work with patients who have had diabetes for a long period of time. She showed two significant things with her patients. First, the "bad" t-cells died, and, more importantly, the c-peptide levels went up. When insulin is created, it has a part that is broken off called c-peptide. Measuring c-peptide then becomes the gold standard for how much insulin the body creates. Most people have given up on the idea of patients who have had type 1 for more than a year ever producing more insulin than they currently make.
I'm still cautiously hopeful. Most of the therapies I looked at last summer when Kaitlyn's test came back so poorly have failed. Lots of drugs seem to improve c-peptide for awhile, but then they all trail off. Even those that help c-peptide might not improve A1c or reduce insulin use. I think this is still just a small piece of the puzzle. It is a far cry from a cure. However, I'm thankful for every little piece.
I'm still cautiously hopeful. Most of the therapies I looked at last summer when Kaitlyn's test came back so poorly have failed. Lots of drugs seem to improve c-peptide for awhile, but then they all trail off. Even those that help c-peptide might not improve A1c or reduce insulin use. I think this is still just a small piece of the puzzle. It is a far cry from a cure. However, I'm thankful for every little piece.
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