Photo Courtesy of Lisa Mastramico
Scott King was diagnosed with type 1 diabetes in 1978. He worked as an investment analyst specializing in biological and medical technology until 1986 when he became focused on finding a cure for type 1 diabetes. He has been working for over 20 years on encapsulating islets. His project, the Islet Sheet, is soon to go to be tested in dogs. His team and their efforts will be captured in a documentary, Patient 13.
In 1980 you wrote an industry review of diabetes entitled Prospects in Diabetes Therapy. What influence does your business background have on your analysis of the diabetes landscape today? What are the most significant changes in the type 1 landscape since 1980?
I am going to start by answering the second part of your question. The fundamentals of type 1 have remained the same. The primary intervention is still to test blood sugar and take insulin. What has changed is technology and the market. In 1980, the type 1 therapeutics market was relatively unknown and small, a couple hundred million. Insulin was effectively generic and pretty cheap. However, the market has dramatically expanded. Type 1 diabetes is big business now.
In regards to the first question, I was educated as an academic scientist. But, when I came to New York I was side tracked by Wall Street and became an analyst. My interest in science influenced the analysis I wrote. You don’t find too many Wall Street analysts who subscribe to science journals. I used both my academic and Wall Street perspectives to write the report. I saw that there was a possibility for money to be made and that finding grants might be better suited by investment ventures. My two interests turned into a hybrid- to find a new therapy for type 1 diabetes using investor money.
There seems to be a news story about a “cure” almost every other day, but nothing has come to fruition so far. What do you believe is the main reason for so many false hopes?
Well, I think that there are two main reasons. The first is that most scientists are engaged in molecular biology, which is focused on studying the interaction of small molecules to produce life. This type of science became the norm in the 1950s. In institutions that give out grants, this influences the main template for projects, which is to identify new molecular components or to expand upon existing ones. This is thought to be good science that expands knowledge. But, it poses a problem because this type of science loses touch with the experiential side. Molecular biology views diabetes as an engineering problem- that insulin is not produced by the body. This view loses the “biological logic” of the islets of langerhans. Islets are parsimonious and only release what is needed, whereas engineered solutions [therapeutics that are designed to act like a pancreas] are not as sensitive as islets and risk giving you too much insulin.
Second, the test tube for diabetes treatments is animals. Using large animals who physiology most closely resembles humans is expensive. So, most ideas are tested in rodents. This is valid science but the biology of diabetes is different between humans and rodents because of their size difference. It is like building the Golden Gate Bridge and beginning with a 10 inch model. You cannot say that you have overcome all of the engineering challenges in the Golden Gate Bridge simply with the 10 inch model. There are problems that bringing it to size will uncover. The media also overemphasizes these results and often says “there may be a cure in five years”, which we know has yet to happen.
The Islet Sheet is a thin sheet containing encapsulated human islets. Can you explain in basic terms (for us nonscientists) how this works?
The Islet Sheet works the same as islets in the pancreas do, except slower. In a healthy pancreas, blood is circulated to the islets and delivers oxygen and nutrients. The islets analyze the composition and release hormones to regulate normal functioning. The islet sheet works the same way except that there are no blood vessels. Our islets are contained in a sheet about as thick as a piece of paper. It responds to plasma and lets nutrients in. The islets then analyze the plasma and produce hormones that diffuse out. The delay of diffusing in and out of the Islet Sheet is probably about 5 minutes.
The most serious problem with the Islet Sheet is getting oxygen in. Oxygen is poorly soluble in water and thus is transported around the body in red blood cells. The red blood cells cannot permeate the sheet, so the islets need to be close to the surface to get oxygen. This is why our sheet is so thin; it is around 300 microns which is 1/3 of a millimeter. The sheet is almost like saran wrap.
Why do you believe this is the best technology for islet transplantation?
Transplanted islets have been shown to produce insulin. The problem is that harsh immunosuppressant drugs have been used to stop the body from rejecting and attacking the islets. So, the two improvements and alternatives are to make immunosuppressant drugs without side effects or to solve the autoimmune attack in another way. There are people working to create better immunosuppressant drugs, but I think that the encapsulated islets are the better alternative. The problem with encapsulation, as I said in the last question, is getting oxygen to the islets. But we have solved this problem by making the sheet as thin as possible.
Describe the surgical and recovery process for the Islet Sheet procedure? How frequently do you anticipate that the sheet may need to be replaced?
The surgery is a keyhole [or laparoscopic] surgery and does not require a long recovery process. We will probably monitor the patient overnight. The Islet Sheet should work pretty quickly, we anticipate within a few days during which we will taper off a person’s insulin use.
On our website, we provide our anticipation of what the Islet Surgery will be like in Theresa’s Story.
You are about to begin a study using the Islet Sheet on dogs. Can you share the salient features of this trial? What are the biggest challenges you anticipate?
The trial is a complicated procedure run by the best people in their fields. Our dog studies are being conducted in Cedars-Sinai. However, the challenge is that the team is new to type 1. They know the techniques from working with type 2. Our chief scientist, Dr. Lakey has not done dog research in five years. The pancreas of a dog clings to the intestines. So, dogs require a delicate procedure to make sure that the pancreas removed without damaging the intestines. The procedure takes 1-2 hours. Dr. Lakey will be doing a practice procedure this week to reacquaint himself with this technique.
There are always complications, but we have been working on this project for over 20 years. We have done all that we can to prepare and we’ll respond to any problems if they arise.
Do you believe the Islet Sheet research can meet the JDCA definition of a Practical Cure? Can it meet a 2025 timeframe? If not, when?
Yes, absolutely.
Patient 13 is a documentary that will follow you through dog and human trials for The Islet Sheet. What is your hope for this documentary (aside from showing successful results for your trial!)?
My hope is for it to show the sheer fun of being a scientist- enjoying the unknown, learning something from every mistake and ultimately succeeding. I am looking forward to the process and hope that it captures something important. Lisa and Guy are going to capture all the angles of type 1, our project, managing type 1 and what we all want- a cure. It’s going to be fun to have it all captured.
The documentary gets its name from your hope of being Patient 13 in your own trial. When do you think human trials will commence?
We hope to start human trials 18 months after our dog studies. If everything goes according to schedule, we should start the dog trials in early 2012 and start human trials in 2013. But, this all depends on the dogs. If something goes wrong, we will have to figure it out in the dogs since everything has worked in the rodents. The biggest challenge is measuring tissue responses. We don’t want a fibrotic capsule to encase the sheet because this will starve the Islet Sheet and stop nutrients from diffusing in. This will make the Islet Sheet ineffective. So, we want to know what’s going on around the Islet Sheet. But in order to make sure that fibrotic capsules don’t grow, nothing sticks to the sheet. So we can’t measure much using standard histology – because the sheet and the tissue separate during processing – and must rely on looking at a gross picture.
For more information about Scott and his work, visit The Hanuman Medical Foundation.
For more information about Patient 13, visit www.voxpopfilms.tv/patient13.
-Cara
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