The Human Trial–must watch

If there is one video that I could show that captures the daily agony … and hope … that I feel as a parent of a child with Type 1, this is it. Please watch. And if you can, donate, not just to the documentary, but to Viacyte. This is the game changer the T1D community has been waiting for.

The Big Business of a Cure

The diabetes pharmaceutical industry is big business, and by “big business” I mean “big money.” Every year, Americans spend billions of dollars on insulin, test strips, syringes, meters, etc. I am fortunate to get my son’s insulin and testing supplies free of charge through our insurance company (because as much as insulin and test strips cost, they are still cheaper than hospitalization for diabetic ketoacidosis caused by uncontrolled diabetes). Without insurance, however, our monthly pharmaceutical costs would be in the $3000 range–that is how much the insurance company pays Lilly and Roche for our supplies; costs to us, lowly consumers, would possibly be more.

Given how profitable diabetes is for pharmaceutical companies, many in the Type 1 community doubt the earnestness of those researching a cure. They believe finding a cure would not be in pharmaceutical companies best interests because a cure would cut into profits. So I wasn’t surprised by the many, many negative comments that accompanied an article that showed up in my Facebook feed about Johnson & Johnson collaborating with small, private company Viacyte on its stem cell-based cure. One reader wrote “Hopeful but skeptical. It doesn’t pay to find a cure…” Another wrote “Taking bets that Johnson and Johnson has ulterior motives and will stall the research now that it is involved with Viacyte.”

Honestly, all that pessimism was disheartening and, as much as I hate to say it, made me feel a bit empathetic toward J & J. (That’s something because I am still reeling from Martin Shkreli‘s 5,000% price hike when his company Turing Pharmaceuticals acquired the patent for Daraprim, a drug that used in malaria, HIV, and cancer treatments).

Here are FIVE reasons we should all take heart and think positively about the Johnson & Johnson and Viacyte partnership:

  1. Johnson & Johnson was already a financial supporter of Viacyte, committing $20 million in August 2014 for the future right to evaluate transactions related to its stem cell trials (guess they exercised that right this week!)
  2. Viacyte comes out on top here, acquiring 145 patents and 565 pending patent applications belonging to Johnson & Johnson.
  3. Type 1 is personal for Johnson & Johnson. T1D runs in the Johnson family and Casey Johnson, great granddaughter of founder Robert Woods Johnson I, died of diabetic ketoacidosis (the result of blood glucose levels that are too high) in 2010. She stopped taking insulin.
  4. VC-01, Viacyte’s encapsulated stem-cell replacement technology, is already in phase 1 human trials and it is showing positive results. The FDA has expressed a willingness to fast track approval if all phases are as successful as anticipated.
  5. The VC-01 trial is being filmed for a documentary titled The Human Trial. Viacyte is giving filmmakers unprecedented access to trial participants, the lab, and the company’s research records. The film is backed by celebrities such as R&B artist Usher, and director/producer Lisa Hepner has plans to follow VC-01’s progress to its approval by the FDA. Lisa has lived with Type 1 for 22 years.

Cure for T1D becomes a reality

beta cell replacement iconSixteen months ago, when my son was diagnosed with Type 1 Diabetes, I was told by doctors there is no cure. In the hours/days/weeks/months following the diagnosis, I searched frantically trying to prove them wrong. I discovered a lot of research that made me hopeful for the future–a researcher at Johns Hopkins who was giving newly diagnosed T1D patients who were in the honeymoon stage a pill that stopped the auto-immune response (too late for my son); a Harvard researcher who was injecting lab-produced beta cells in the gut of T1D patients every two-three weeks to simulate the production of insulin (see my previous post); a scientist in Denmark that was able to regenerate pancreatic beta cells in lab rats. But these were just possibilities that promised a future cure but could not deliver it in the short term. Today, that has changed.

ViaCyte, a private medical research company in California, has successfully implanted embryonic stem cells into a human being. Unlike other stem cell therapies being tested around the globe, the cells in this therapy have been programmed so that they are protected not only from the body’s natural instinct to reject them, but  also against the autoimmune response that killed the test subject’s pancreatic cells leading to T1D. In previously published research on stem cell therapies conducted by other entities, the implanted stem cells have all succumbed to the on-going autoimmune response that causes T1D. ViaCyte has succeeded where many others have not.

Over the next few weeks, the implanted cells are expected to mature into Islet cells and to start producing insulin (hormone necessary to deliver sugar to the body’s cells to use for energy), glucagon (the body’s natural hormone that responds to and corrects low blood sugar), somatostatin (the pancreatic hormone that naturally stops the release of insulin and glucagon as needed) and amylin (another hormone that regulates secretion of glucagon) permanently. If all goes well, Viacyte will recruit 40 patients to participate in a first stage clinical trial at the beginning of 2015.

This is incredible news. I never imagined sixteen months ago that we’d be so close to a cure in such a short time. You can read more about ViaCyte’s success here.