In the whirlwind that is the stem cell and regenerative medicine world, there are many concerning things that need attention, but also good stuff happens too and this post focuses on the positive.
The Asterias spinal cord injury clinical trial, a phase 1/2a trial called SCiStar, continues to make encouraging newswith a clean safety profile and additional hints at possible positive indicators of efficacy. With the usual, important caveats such as that this is early and it is not an RCT, the SCiStar momentum is positive. I’m excited to see what the future holds for this one including from an RCT. You can read my interview from last month with Asterias leadership here.
I remain very enthusiastic about ViaCyte’s trial as well using a stem cell capsule product for treatment of Diabetes. Their joining forces with BetaLogics a year ago just made their position even stronger.
I’m going to do a post soon on an analysis on the total number of stem cell and regenerative medicine trials compared to historical data I collected. Stay tuned on that. I’m guessing it’ll be good news.
Recently, we also saw evidence of fast action from FDA in response to the 21st Century Cures Act in terms of providing a clear documenton Regenerative Advanced Therapy designations and applications. It’s still unclear how the Cures stem cell provisions will play out, but I consider quick, clear action from the FDA to be a positive. I wish they were this fast on other stuff like dealing with stem cell clinics marketing unapproved drug products.
There have been a number of cool papers recently that I recommend reading:
The 21st Century Cures Act specifies some new FDA rules about investigational stem cell-based regenerative medicine therapies and perhaps most importantly defined a new class of product called a “Regenerative Advanced Therapy” or RAT, which is entitled to an expedited review.
The FDA already has a new pageup on RATs, which specifies the specifics of RAT designation and how an entity may submit an application for RAT designation. This was remarkably fast of the FDA.
Some things on the RATs page stand out including that a request for a RAT depends on an IND. The IND must either be existing or submitted along with the RAT request. This is a major point as obtaining an IND requires rigorous, controlled data.
Stem cell businesses out to make a quick buck off of patients and potentially hypothetically interested in get RATs are going to be out of luck unless they change their world view to be data-centric and also go for INDs.
Also, the FDA is emphasizing that the condition in question must be “serious of life-threatening” and the FDA decides on what meets this criteria.
Oddly, the FDA will not require data be part of the RAT itself. I don’t quite get this, although perhaps their thinking is that the data requirement of the IND will suffice.
Last week I posted my list of 2017 predictions for the stem cell field. Today a couple of days into 2017 I’m more focused on hope than realism. What would I wish for in the stem cell and regenerative medicine arena in the coming year?
More stem cell clinical trial data posted and published. There are few things as exciting as stem cell and regenerative medicine clinical trials across the full spectrum of stem cell types including adult, embryonic, and IPSC. But we need to have actual trial data be peer reviewed and published or at least posted. Clinical trial updates only by press release are not helpful to patients or the field.
Clinicaltrials.gov adapts. This vital resource of trial listings adjusts to new realities. It either filters its listings to screen out for-profit entries that aren’t real trials or it provides more practically useful information such as at a minimum clear indications of whether a listing has an IND (this doesn’t need to violate any confidentiality rules) and whether the listing requires payment as an inclusion criteria. See my interview with the leader of Clinicaltrials.gov.
FDA speaks clearly. Whatever the FDA does or does not do in terms of actual stem cell & regenerative medicine-related actions in 2017, it is clear about it. This year I hope the FDA provides concrete, consistent explanations in the public domain that don’t require an FDA-ese jargon dictionary to try to understand.
FDA and its CBER center are consistentwith good & bad citizens of the stem cell arena. The FDA has a tough job overall and its CBER branch focusing on biologics including stem cells has its own specific challenging task set. However, for years CBER has held different players in the stem cell arena to different rules and expectations. Paradoxically, essentially the better a citizen you are, the more the FDA expects from you. On the flip side, if you are for instance a stem cell clinic with no intention of following the rules (no BLA, no IND, no pre-IND, no expertise in stem cells, no data, etc.) CBER has historically generally left you alone.
Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.
You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.