Thursday, March 05, 2015

Transcontinental Diabetes Duel: The Search for a Stem Cell 'Cure'

Paul Laikind
Doug Melton,













One might call it a California-Massachusetts stem cell face-off. The tussle is over a stem cell cure of sorts for diabetes.

The players are Doug Melton of Harvard and Paul Laikind, CEO of ViaCyte in San Diego.  
Recently in separate forums, the men critiqued each other’s approaches to diabetes.

Most recently it was Laikind three days ago on the blog, ipscell.com, of UC Davis researcher Paul Knoepfler. Laikind was responding in a Q&A carried by Knoepfler.

Knoepfler asked about Melton’s comments in the MIT Technology Review last month. Melton was described in an article as being “worried” that ViaCyte’s technology, now in a first stage clinical trial, would not work. The California stem cell agency has invested $55 million in the firm's approach.

Knoepfler continued,
“(Melton) raised concerns more specifically about the Encaptra capsule, for example, functionally becoming fibrotic and mentioned worries about your cells being immature and taking a long time to mature. Any response on capsule and cells? He also has suggested that his beta cells will be a better option.” 
Laikind replied,  
“Dr. Melton’s work on the beta cell is very interesting. As to the cells, we made the choice to use the pancreatic progenitor cells. An important consideration is that when you first put in cells, they are in a hypoxic environment. Beta cells are sensitive to low oxygen levels, which can negatively affect their survival and function. Beta cells typically exist in a mature highly vascularized organ. The pancreatic progenitor cells that we use undergo an organogenesis-like process, more similar to how they behave in nature, and thus we believe they should be better able to handle low oxygen. They also are believed to release angiogenic and other factors to promote vascularization.
“In regards to the capsule, we do expect there to be a foreign body reaction in patients after implantation, which will generate a fibrotic capsule. In fact, we see a thin fibrotic capsule around the device in mice. But in the mouse model this capsule around the device is very well vascularized. The vasculature is right up against the device membrane on the outside, allowing for oxygen and nutrient diffusion to the cells inside.”
Knoepfler also asked about the diabetes effort in Canada involving BetaLogics Venture, a subsidiary of Johnson & Johnson, which also made a $20 million investment in ViaCyte last summer.

Laikind said situation involving Melton and BetaLogic was “healthy competition.”

He continued,
“There’s room in this area for multiple efforts and we aren’t especially concerned with competition. Yet we do feel we are ahead of others and we have substantial intellectual property that they will need to navigate (~50 patents issued in the United States, and a couple hundred pending patent applications, including international). At ViaCyte we view the real competition as the biology rather than with the efforts of others as we seek to cure this devastating disease.” 
Laikin had more to say about his firm’s product and Melton’s comments.  He also discussed ViaCyte’s clinical trial, which now has four patients with a goal of 40. He said the initial evaluation of efficacy could occur by late 2016. Within five years, he hopes to see “success” with the product and “be moving to market.”

Responding to a question about “product placement,” Laikin said the firm is currently inserting its tiny device in the lower back of patients. Laikin said,
“The reason for that placement is that while the device can withstand the impact of a 60 mph baseball (based on cadaver testing), a needle could go right through it, so we want to put it where patients don’t typically inject insulin.” 
Concerning Melton’s views on ViaCyte, the Feb. 12 MIT Technology Review piece said,
“Douglas Melton, a biologist at Harvard University who has two children with type 1 diabetes, worries that the ViaCyte system may not work. He thinks deposits of fibrotic, scarlike tissue will glom onto the capsules, starving the cells inside of oxygen and blocking their ability to sense sugar and release insulin. Melton also thinks it might take immature cells up to three months to become fully functional. And many won’t become beta cells, winding up as other types of pancreatic cells instead.
“Melton says the ‘inefficiency’ of the system means the company ‘would need a device about the size of a DVD player’ to have enough beta cells to effectively treat diabetes. ViaCyte says it thinks 300 million of its cells, or about eight of its capsules, would be enough. (Each capsule holds a volume of cells smaller than one M&M candy.)    Last October, Melton’s group announced it had managed to grow fully mature, functional beta cells in the lab, a scientific first that took more than 10 years of trial-and-error research. Melton thinks implanting mature cells would allow a bioartificial pancreas to start working right away.”
The Web site, diaTribe, last fall carried an analysis of all three approaches.
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2 comments:

  1. A reporter called me when Doug Melton's paper was published- the reporter had not heard of Viacyte's FDA approval for a diabetes therapy. This gave me a sense of unreality that you're probably familiar with, David. The big news for patients is a clinical trial, not a lab experiment. Why wasn't that the big news? One publicity machine trumped the other.

    Anyone care to comment?

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  2. Re Jeanne Loring and the recent diabetes item, she is right. Clinical trials generally should be bigger news than a lab experiment, particularly for patients. However, I suspect that the news coverage on Melton's research last fall reflected a certain reality in the way the media functions. Based on decades of experience in the news business, it is safe to say that an East Coast tilt generally exists in mainstream news coverage.. It has to do with the location on the East Coast of the headquarters of key, historically important news organizations (NYtimes, The Associated Press, the now all but defunct UPI, Washington Post, WSJ, etc.). After beginning this blog 10 years ago, I was surprised to see a similar tilt in science, which presumably is driven by comparable forces, such as the clout (perceived and real) exercised by such enterprises as Harvard and Yale. Plus there is simply the skill of the PR folks who are involved, some of whom really know how to peddle a story, not to mention scientists who cultivate various reporters and editors. Then there is old-fashioned luck. A slow news day can elevate a routine story into something much greater.

    Of course, many businesses and institutions simply do not know how to handle the media and thus their announcements are disposed of summarily. I worked for United Press International in Los Angeles for four years in the 1960s-70s. The bureau was a big producer of national and international stories(the Manson killings, Sirhan trial, Nixon's Western White House, Santa Barbara spill and so forth). The bureau chief there would deal with the hundred or so of press releases that came in daily with great dispatch. He would grab a handful of the envelopes, glance at the return address of each and, one by one, would flip nearly all of them into a nearby wastebasket.

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