MD Stem Cells purported Right To Try program

I’ve been concerned about a stem cell group called MD Stem Cells for many years. Now it comes under more scrutiny from a new must-read piece by Donna Young over at S&P Global. Young’s article reports a puzzling, purported right-to-try (RTT) program that MD Stem Cells appears to working on with Richard Garr. Note that Garr is the former CEO of Neuralstem (for reference, that company just renamed itself Seneca Biopharma) who now runs a CRO focused on RTT.

My SCOTS concerns

Two physicians Dr. Jeffrey Weiss and Steven Levy, are the leaders of MD Stem Cells. The firm has run an unusual stem cell clinical experiment of a sort called “SCOTS”. It’s not a standard IND-based, FDA-cleared trial. SCOTS has been going for a long time using investigational bone marrow stem cells for vision loss. How would bone marrow cells help vision loss? I don’t get it.

From all I’ve seen, patients/customers have to pay a very large fee to be part of SCOTS, which is concerning in my opinion. I’m also not aware of the firm having already conducted controlled pre-clinical studies in animals first or having placebo control subjects for SCOTS itself even after all this time.

Furthermore and more concerning, at some point SCOTS patients started publicly self-reporting adverse events (e.g. here) after getting the marrow cell preps injected in their eyes as part of SCOTS. MD Stem Cells has disputed links between SCOTS offerings and the patient claims of adverse events. However, despite some lack of clarity here, I tend to believe that multiple patients are unlikely to just make stuff up, and it’s risky in my view to just attribute possible negative outcomes to spontaneous loss of vision that supposedly was unrelated to the stem cell injections.

Over the years, I’ve tried to engage with Weiss and Levy to get more information and clarify things here for the wider stem cell community. We’ve emailed a bit, which was useful. Most recently I thought we were going to do an email Q&A here on The Niche in 2018, but my questions went unanswered. You can read the interview that never was here. It’s a good summary of my concerns about SCOTS.

NEST is next for MD Stem Cells

MD Stem Cells started running a second program called NEST, using investigational autologous bone marrow injections for a variety of neurological conditions. The full name is “Neurologic Stem Cell Treatment Study (NEST).” Unfortunately, NEST makes no more sense to me than SCOTS does, with the added concern that these two docs are not to my knowledge board-certified specialists in neurological diseases. I’m assuming they are working with one or more neurologists for NEST.

RTT?

Young’s new piece at S&P Global focuses on these neuro-related stem cell offerings by MD Stem Cells. She reports that these unproven offerings are represented with Garr’s consulting help for ALS and other neurological conditions under the umbrella of the national RTT law.

I did a recent interview with Garr about his RTT firm. I later wrote about what I see as a big potential right to try loophole. Note that some law and policy experts were writing about this and other potential loopholes back in 2018. For example, I recommend this piece from Beth Roxland and Elisa A. Hurley on the The PRIM&R blog, including this key section:

“This arguably provides some counterweight to the removal of federal protections included within the existing expanded access process. However, loose language and undefined terms in the RTT law provide a number of potential loopholes. These include the lack of any requirement that the entity providing access to the drug via the alternative route is the same one that is the sponsor of the filed investigational new drug application (IND), and of any independent third-party review (in lieu of the FDA, or even an IRB) to ensure a level of integrity or meaningfulness of the “completed” Phase 1 trial.”

They clearly saw this and other potential issues on the horizon.

Garr seems to believe this kind of RTT piggybacking (as I call it) can be done and that as long as some physician somewhere has got an IND and Phase I data for a certain kind of product then anybody else can legally use something similar to that product under the national RTT law.

We urgently need more clarity here and I call for the FDA to quickly address it because things are too vague. Young’s new piece has this quote from FDA CBER Director Peter Marks:

“Patients should be able to find out from the company if the product is under an IND, and those entities “should hopefully be able to truthfully answer that,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

“If it’s a stem cell product, it should be under IND with the agency,” Marks told S&P Global Market Intelligence. “If it’s not, that’s probably a red flag. It doesn’t absolutely mean there’s something wrong. But it may mean there’s something wrong.”

However, I’m not sure what this means for RTT cases. I also don’t know if MD Stem Cells has an IND for their neurological stem cell work, but I haven’t seen any evidence that they do.

If this is indeed a “piggybacking” situation, I don’t see how that’s in the best interest of patients.

MD Stem Cell disclaimer and looking ahead

Can we learn anything from this firm’s website? Here’s their disclaimer:

“The stem cell studies available through MD Stem Cells are Institutional Review Board (IRB)  approved and FDA compliant. They are open label, non-randomized efficacy studies and no guarantees of specific results or improvements to participating patients are being made.  Any medical procedure carries risks as well as potential benefits.  The studies have different treatment arms and our investigators assign patients to minimize risk and maximize potential benefit.  Depending on the arm chosen the risk of potential complications has been calculated to be from approximately 0.0008% to 5%.”

In my opinion that claim about FDA compliance is hard to judge as to accuracy, but if the FDA continues to do nothing then the claim seems to stand practically speaking. I also wonder if bone marrow stem cells injected into eyes or the nervous system constitutes non-homologous use, which would make them a drug product.

I’d be curious to know how the firm calculated the risk odds they state as well and whether some patients such as those claiming lost vision were somehow excluded.

While the FDA apparently would not say to Young whether the agency is looking into MD Stem Cells, I hope somehow we can all get more clarity on what’s going on with this situation.