Tiptoeing Toward A Cure For Type 1 Diabetes

By Ben Comer, Chief Editor, Life Science Leader

As seasoned investors know well, early-stage research and drug development can be deceptively compelling. Excitement around a new discovery, followed by promising results in animal models (or in modeling software), big valuations, or successful fundraising rounds can promote a sense of inevitability, perhaps especially among the scientists doing the work, and the patients who stand to benefit.

And yet, exceedingly few new drug development programs follow a straight line to market. In working on this month’s cover story, which features Imagine Pharma, a pre-clinical stage biotech based in Pittsburgh, it was hard not to get swept up in the enthusiasm and promise of the company’s autologous cell therapy treatment for Type 1 diabetes (T1D). In visiting Imagine Pharma’s lab and meeting its leadership and chief scientists in person for interviews, I was impressed by the intelligence, ingenuity, and humility on display. Surely this group of exceptionally talented and committed individuals, I felt, would be capable of accomplishing great things, perhaps even curing a disease that causes so many problems and complications, and can cost so much to effectively manage.

Imagine Pharma is only one of the hundreds of other plucky biotechs to have emerged over the past five years, all of which believe they are on a path to a breakthrough. The unfortunate truth is that most of these companies will fail; in some cases, because the science itself fails or hits a dead end, and in others, the reasons are more pedestrian — inexperienced leadership, poor fundraising, or unforeseen headwinds in the market and competitive landscape. In a disease area like T1D, where insulin prices are a constant political football, and where the promise of cell therapies rendering patients insulin-independent have been very slow to progress, a healthy degree of skepticism regarding preclinical and early-phase research, no matter how spectacular, is certainly warranted.

Following two patients with T1D as they navigate the first human clinical trials of ViaCyte’s stem cell transplant therapy, writer/ director Lisa Hepner’s excellent film The Human Trial is a bracing reminder of the stakes of clinical research, and the often disappointing results, even when they move the science forward. For both of the trial participants followed by Hepner, herself a T1D patient, the treatment didn’t work; the transplanted cells didn’t produce insulin, and didn’t help the participants. Parts of the film were heartbreaking to watch. Hepner notes that one of the chief ironies of T1D is that she herself, to all outward appearances, looks healthy. But she is not; nerve damage, due to elevated blood glucose levels over time, has started to creep in, and worse complications are likely on the horizon.

And yet, the film strives to end on a modestly upbeat note, championing the trial participants’ willingness to participate in an experimental, as yet untested cellular transplant, not just for themselves, but with the hope that their participation will eventually lead to successful therapy for other T1D patients. And indeed, it might; Vertex Pharma’s $320 million acquisition of ViaCyte in July will provide the company with additional resources and runway, and ViaCyte’s ongoing collaboration with CRISPR Therapeutics, aimed at creating an “immune-evasive” stem cell therapy, could potentially eliminate the need for the immunosuppressant drugs that must accompany most donor-sourced transplants. More than a dozen companies are now exploring cell therapies for the treatment of T1D; with those improved odds, maybe a cure isn’t as far off as it seems.