which has figured out a way to produce human insulin-producing beta cells on a large scale.<\/a> The next task is figuring out how to get the cells through the FDA approval process and to market. In late September, I went to a panel at Harvard about the infrastructure Melton and his colleagues have created to push that process along. There, it became clear that like Boston University biomedical engineer Ed Damiano, who is seeking FDA approval for the bionic pancreas, Melton has decided to break the type 1 diabetes problem into two parts, planning to seek FDA approval for the stem-cell derived beta cells, and later to seek approval for the encapsulation technology necessary to protect them from the autoimmune attack that characterizes type 1. (The bionic pancreas is designed to be a dual-hormone pump\u2014insulin to bring blood sugar down, and glucagon to keep blood sugar from going too low; but Damiano and his company Beta Bionics are seeking FDA approval for an insulin-only pump first, and later will seek approval for a dual-hormone pump.)<\/p>\nThe HSCI has now joined with others from the medical field to form the Boston Autologous Islet Replacement Program (BAIRT). The moderator of the panel called it \u201cthe full spectrum of the Boston biotech ecosystem.\u201d Aside from HSCI, the program includes Semma Therapeutics, a biotech company named after Melton\u2019s children, Sam and Emma, who both have type 1; Joslin Diabetes Center, Brigham and Women\u2019s Hospital, which will be involved in recruiting patients and transplanting beta cells; and the Dana-Farber Cancer Institute, which already has in place a cell production facility.<\/p>\n
Melton started off the panel by saying that the basic goal of his work is to replace insulin injections with stem cell injections\u2014\u201cproviding nature\u2019s own solution\u201d to the problem of diabetes. The difference between a healthy beta cell\u2019s ability to measure sugar and the best constant glucose sensing technology is vast: \u201cBeta cells read sugar a thousand times a second, a glucometer reads it every 5 minutes. They have a long way to go to catch up with millions of years of evolution.\u201d<\/p>\n
While Melton and his colleagues seem to have solved the problem of how to turn induced pluripotent stem cells into beta cells in the lab, he admits that he needs help translating the work to people. \u201cAs my children would say, \u2018We don\u2019t care about making more rats non diabetic.\u2019 But I\u2019m a basic research scientist; I couldn\u2019t do this on my own.\u201d<\/p>\n
That\u2019s where the rest of the BAIRT team comes in.<\/p>\n
Melton\u2019s lab licensed its beta cell technology to Semma Therapeutics, which is working to bring the technology from the lab to the clinic. Semma\u2019s CEO and co-founder, Robert Millman, was cautious about the project\u2019s timeline: \u201cThere are a lot of scientific steps that have to be gone through and a lot of regulatory steps. The FDA is working with us very early in the process\u2014that\u2019s encouraging. I would love to say we\u2019ll be able to treat Doug\u2019s kids; however, how long it will take us to get through the process is unclear.\u201d<\/p>\n
The initial study, which will start enrolling next year, will involve about ten patients, who will have the stem-cell derived beta cells injected into their forearms, in the hopes that the cells will start producing insulin within the body. These patients will be people who have become diabetic because their pancreases were removed due to pancreatitis\u2014not type 1s whose ability to create beta cells has been curtailed by autoimmune attack. In other words, this study is only about the efficacy of the cells themselves. In order for the cells to get approved, they will have to pass a number of hurdles. As Millman explained: \u201cWe have to show the FDA that we have a product that won\u2019t do something bad to the body. We have to show that these cells are committed to being beta cells; and we have to show that they are regulated and functioning properly, that they don\u2019t secrete too much insulin.\u201d<\/p>\n
In terms of protecting the cells from autoimmune attack, BAIRT is working on a couple of different initiatives, both short term and long term. A short term solution would be to protect the cells by a physical barrier, which would nevertheless allow the cells to release insulin into the body in response to glucose signals. Melton described this physical barrier as \u201clike a tea bag.\u201d The sugar is like the water coming into the bag; the insulin released from the beta cells is like the tea that floats out. But the long-term goal, he said, \u201cis to understand why the immune system is making this mistake, and then kill off this immune response.\u201d Having an unlimited supply of beta cells will help in this process. \u201cOnce we have a patient\u2019s own beta cells you\u2019ll be able to interrogate the immune system in a way you haven\u2019t been able to,\u201d Melton said. His lab is also looking to the field of cancer immunotherapy, and ways to adjust the immune system so that it would no longer attack beta cells.<\/p>\n
When asked about how much it would cost to bring beta cells to market as a treatment, Millman first said \u201clots,\u201d and then added that he thought the whole program would cost about $30 million. But, as Melton pointed out: \u201c<\/strong>This year 22 billion dollars of insulin will be injected into people. We are talking about millions of dollars to replace that.\u201d<\/p>\nGiven the large amount of money that pharmaceutical companies make from selling insulin, audience members pointed out that Big Pharma is unlikely to support this technology\u2014the goal of which is to let people with type 1 diabetes live free of insulin injections. Melton pointed to another potential source of support: insurance companies. \u201cThis is a lifelong disease that costs thousands of dollars a year to treat. So it seems to me that insurance companies should also be interested in this. Treatment is expensive over a lifetime.\u201d<\/p>\n
I talked with Melton a bit after the event, and he made it clear that his goal is to come up with a treatment that changes the face of diabetes\u2014essentially a cure, drawing upon the human body\u2019s own perfectly calibrated system\u2014and in so doing that pulls the whole multi-billion dollar diabetes market along with it. \u201cIt\u2019s like Tesla\u2014GM and Chrysler weren\u2019t going to develop the electric car until Tesla came along and proved it could be done, and that people wanted to buy the cars. Now all those companies are jumping in to be part of that market, for economic reasons.\u201d But of course Melton, with his two children with type 1, is not doing this for economic reasons. \u201cThis treatment could improve people\u2019s lives. You can\u2019t put a number on that.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"
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