Yelling From the Rooftop - Part 2

Continued from Part 1

The CHILD Trial Phase 1 had 3 documented funding sources: the Marcus Foundation, NIH, and the Maryland Stem Cell Research Fund — MSCRF.

MSCRF is a state fund and exists to support stem cell research conducted in Maryland. The money comes from a check-off on Maryland marriage licenses and from state appropriations — and it is, by statute, restricted to Maryland-based investigators and institutions. If you’re not in Maryland, MSCRF money cannot reach you.

Dr Kaushal’s lab was at the University of Maryland when Phase 1 was designed and launched. He had come there from Northwestern/Lurie Children’s in Chicago, where he’d been since 2007. Then he left Maryland, returned briefly to Lurie Children’s, and ultimately landed at UNLV where he is currently building Nevada’s pediatric cardiac surgery program while simultaneously trying to get CHILD Phase 2 off the ground.

So here is the geometry of what I think might have happened:

• MSCRF funded Phase 1 because Dr Kaushal was theirs — a Maryland researcher, doing Maryland research.

• But when he left, the money could not follow; not because anyone decided to abandon him, but because the law says it can’t cross state lines. MSCRF’s mandate ends at the Maryland border.

Remember when Dr Kaushal said that he’d been funded before and was now in a ‘position to look for funding’ for Phase 2? I saw his face crack – not by much mind you (he’s used to keeping his cards close to the vest and I could tell). And right up until that moment, I’d been talking to a man who’d been giving me his research/funding speech; rattling it off by rote, not even stopping to take a breath lest I should butt in and disrupt his memorized flow. But in that next moment… I saw a man who not only cared, but cared a great deal. And I found myself thinking about what being in this kind of conundrum must be like.

I mean think about it: you’ve spent years building the scientific case, brick by careful, meticulous brick. Then you finally go on to clinical trial, Phase 1, and it delivers promising results — the secondary endpoints, the parent-reported quality of life, the glimpses of something real. And then the moment you have the most compelling argument for Phase 2 … the funding geography has shifted, right out from under your feet. The foundation didn’t judge the science and find it wanting; it simply couldn’t follow you out of state. But the experience of it, for a researcher who has been building toward this for seventeen years? That’s gotta be devastating.

But – he didn’t say ‘devastating’ to me. What he actually said was “they balked.” That, ladies and gentlemen, is a word choice. That is deliberate. And it made my spidey senses tingle all over.

Before I go on: this is inference on my part, not confirmed fact. I didn’t ask him directly who the funder was, and he didn’t say. The 990-PFs tell me who wasn’t the defector; they don’t tell me who actually was. But the MSCRF theory so far seems to best fit the timeline, the geography, and the mechanism — and it reframes the abandonment as structural rather than judgmental; which frankly might be both more accurate and more painful.

I’m gonna be precise about the structural problem here because it isn’t an accident; it’s a design.

The beauty of autologous cell therapy (therapy made from a person’s own cells) is that every person’s therapy is unique, harvested from their body, grown in a specialized lab, returned specifically to them. And that is also precisely why it is inherently difficult to fund through commercial channels because you can’t manufacture it at scale, can’t bottle it, and can’t ship it to a pharmacy. In other words, there is:

• NO commercial product,

• NO IPO at the end of the pipeline, and

• NO transferable financial instrument that makes the investment attractive to capital markets.

(ELPIS, by contrast, uses allogeneic cells — donor cells, standardized, manufactured at scale by the company Longeveron. There’s actually a commercial product that’s out: laromestrocel, branded Lomecel-B. The FDA granted ELPIS II a Rare Pediatric Disease designation, which comes with a Priority Review Voucher upon approval — a transferable certificate worth, on the current market, somewhere between $100 and $150 million. A certificate like that… you can sell it to another pharmaceutical company that wants to fast-track their own drug. That’s what makes allogeneic therapy fundable through federal mechanisms. And that’s the financial incentive the autologous approach simply doesn’t have.)

None of this means ELPIS is wrong, mind you; it may well work, and the field genuinely needs both answers. But the commercial architecture rewards allogeneic therapy in ways it structurally cannot reward autologous therapy -- regardless of which approach ultimately produces better outcomes for patients.

And philanthropic funding (the realistic alternative for autologous approaches) is vulnerable to headlines. To reputational contagion. To a Harvard researcher’s fraud casting a shadow over an entire category of science that shares 3 words (”cardiac stem cells”) with his discredited work.

When I pushed Dr Kaushal on the naysayers, this is what he said: “The stem cell field has been tarnished. From some of the adult experiences... a critical investigator was challenged and found to have some fraudulent data that was published. And that really precipitated a blackening — a mark on the stem cell field.”

He didn’t say Anversa’s name. Researchers in this field tend not to (is this giving Voldemort vibes to anybody else here? No? Just me, then? Okay cool). But his is the only name that fits. “To rise from there, out of that mess,” Dr Kaushal went on to say, “is challenging. We’re persistent. Because we base all our studies on mechanism.”

Dr Kaushal made one reference that I’ve kept turning over.

When I asked what he wished existed for CHD families — what was missing, what he’d change — he talked about the DMD community (Duchenne Muscular Dystrophy). I looked it up and it’s a truly devastating progressive disease; fatal if untreated, and it affects roughly 15,000 people (the vast majority of which are boys and young men) in the US and over 800 in Canada.

“They have developed a great community,” he said. “They voice it all the way up to the FDA and all the way up to the NIH. So it’d be nice to have a community like that for single ventricle patients. Advocating for therapies.”

So 15,000 Americans and nearly 1,000 Canadians live with DMD. Now stack that up against about 1,000 American babies plus around another hundred from Canada born with HLHS every year. Now add ALL single ventricle conditions together — tricuspid atresia, pulmonary atresia, heterotaxy, the rest of the univentricular family — and you’re still talking about a population that medicine classifies, without irony, as too small to matter commercially (check out my DEBUNKED article on that particular takedown).

Nevertheless, the DMD community built infrastructure:

• Patient advocacy organizations with scientific advisory boards,

• Direct lines to FDA patient engagement programs,

• Foundations that fund research specifically because the commercial market won’t.

They made noise — organized, sustained, strategic noise — until someone listened.

CHD at large has 40,000 new diagnoses per year in the US and another 4,400 in Canada. We have the numbers. What we don’t yet have — and I want to be honest about this because I think it’s one of the most important gaps in our community — is the collective understanding of what’s actually at stake in the funding rooms we’re not in.

I’m working on that. In fact, so many of us out there are working on that! And it’s a big part of why this piece exists.

I have to tell you about the moment in the interview when the script cracked.

For most of our call — he had allotted me 20mins, I was able to stretch it to something like 32mins, and he still mostly used it himself — Dr Kaushal was in prepared-pitch mode. And, really, the pitch is good; polished, clear, scientifically precise… but it also came across to me like armour. That much was clear. I was treated to the version of the story that is safe to tell, doesn’t expose the wound, and doesn’t say anything a funder might find uncomfortable.

I had my mouth open more than once trying to get a word in – to the point that I remember reflecting to myself, Jesus, Marie-Jo, you look like a fish out of water in your zoom screen. I know he saw it (it was kinda hard to miss), but he plowed on anyway. (I’ve been in enough high-stakes meetings in my career to recognize when someone is running out the clock on purpose, even if the purpose is self-protective rather than rude.)

And then I threw him something of a curve ball. Deliberately, because if I wanted a chance to have any kind of meaningful dialogue in the few short moments I had left, I had to throw him off script once and for all. I said something like: CHD isn’t exactly a ‘sexy’ topic in medicine, is it.

And he caught it. He blinked. I saw it. I had caught him off guard, and something shifted.

After some hesitation, he said “Pediatrics is not on the forefront of research… It’s not on the forefront of everyone’s agenda. These are very small patient populations — niches — when you compare it to adults with obesity, or heart failure...”

He didn’t finish the sentence. He didn’t have to.

And that’s when he threw in, almost immediately after, “The sponsor who paid... who funded the Phase 1 and they balked on funding the Phase 2. So now we’re in a position where we have to look for funding.”

He picked up the metaphorical ball I’d thrown him and he had slammed it down. And in doing so gave me the thing I’d been trying to understand the whole conversation: not the spiel, but the humanity underneath it. He revealed the specific anguish of someone who had spent 17 long years doing everything right, by every measure of scientific rigour, and yet still found himself yelling from a rooftop in Las Vegas because a funding decision made in a boardroom somewhere (for reasons that have nothing to do with the quality of his science) almost ended one of the most promising therapies for HLHS babies in the world. I could see how much it cost him to say this, out loud.

I didn’t press him on who the sponsor was. I was out of time. It would have been incredibly naïve of me to think he would have revealed it to a stranger (a mom with a Substack, as far as he was concerned) on a recorded zoom call, and I wasn’t going to put him in an uncomfortable position with a potential future funder. But I went looking anyway, in the 990-PFs. As you’ve now read.

Here is where the story changes register — because it doesn’t end on that metaphorical rooftop.

CHILD Phase 2 is coming. Enrollment is expected to begin in summer 2026, across multiple centers. The Phase 2 design addresses the limitations of Phase 1 directly: larger cohort, properly powered, pre-specified efficacy endpoints. And a surprisingly fundamentally different delivery approach. Because Dr Kaushal and his team learned something from Phase 1: delivering cells once, surgically, into the heart wall is probably not the most effective way to drive sustained remodelling, which was in fact hypothesized in the Phase 1 paper. So now, the new approach will give multiple IV doses every couple of months for the duration of the study — kind of like a maintenance regimen rather than a one-time repair; a course of treatment rather than a single intervention.

The FDA, for its part, has been listening in ways that surprised even Dr Kaushal. Recent conversations have raised the possibility that the Phase 2 study could serve as an approval study — and that’s telling on just how important and viable CHILD is because it means potentially bypassing the usual requirement for a separate Phase 3. “The FDA understands the gravity of the patient population,” he said. “… sympathizes that we can’t do another 5-year study.” You know what I heard when he said that?

Somewhere in the machinery, someone is listening.

And this: Dr Kaushal mentioned (almost in passing, but I caught it) that these cells have potential applications far beyond HLHS. The secretome mechanism works through inflammation suppression; inflammation, he pointed out, is a driver of heart failure, of Alzheimer’s, of kidney disease, of liver disease.

The niche-that’s-too-small-to-matter argument may look very shortsighted in hindsight. (Shameless promo — that argument no longer stands: I did the math)

He has founded a company — Secretome Therapeutics — to begin building the commercial infrastructure that autologous therapy doesn’t otherwise have. Whether that changes the funding calculus remains to be seen; but the fact that he’s building it says something about how clearly he sees the structural problem.

Near the end of our call, I mentioned the Japanese 8-year follow-up data on cardiosphere-derived cells in single ventricle CHD. Durable benefit; longest follow-up in the field, published in 2024.

He hadn’t seen it.

Wait, whaaaaaat???

A pediatric cardiac surgeon, one of the most active researchers in cell therapy for CHD, hadn’t seen the 8-year Japanese follow-up! I had found it while doing my own background research to prepare for my conversation with him, on the field of stem cell therapy studies for people with complex CHD. I offered to send it to him.

“I’d love to see that,” he said.

I’ve thought about that a lot since. About what it means to be so deep in the work of doing the science — raising the money, moving cities, managing FDA conversations, running trials across multiple centers — that the literature is potentially getting ahead of you. About a researcher who is simultaneously trying to save a therapy and build a company and relocate twice and file grant applications and talk to CHD moms who have their mouths half open trying to get a word in.

The researchers who are yelling from the rooftops are also the ones doing all the other work. The yelling is not their full-time job; it’s what they do with the scraps of time left over from the actual work.

And I thought about the parent who commented on my HLHS CHILD Trial article, the one whose child was in the stem cell group and was doing amazingly well (hand to heart, I welled up when I saw her post).

She knows something is working. She can’t read it in a grant application or a 990-PF. She can only feel it in the fact that her child is here, thriving, in a way the statistics said was not likely.

Seventeen years of bricks. A funding architecture that wasn’t built for children who can’t be scaled. A fraud that poisoned a well. A private foundation that flinched (for reasons that may have had nothing to do with the science) at the moment the science needed them most.

And a researcher in Las Vegas, yelling from a rooftop, with Phase 2 enrollment opening this summer.

Well I’m listening, Dr Kaushal.

We all are.

Take heart,

Marie-Jo 💕

The IRS Form 990-PF filings referenced in this article (Marcus Foundation, EIN 58-1815651, filing years 2019–2024) are publicly available through ProPublica’s Nonprofit Explorer. I reviewed the full itemized grant schedules for each year. No grants to Longeveron, University of Maryland, or any ELPIS-related entity appear in any filing year reviewed.

The Piero Anversa fraud case: Harvard Medical School and Brigham and Women’s Hospital called for retraction of 31 papers in October 2018. Brigham and Women’s settled with the U.S. Department of Justice for $10 million in 2017 over federally-funded research fraud. Full documentation at STAT News and Retraction Watch.

Dr Kaushal’s quotes are drawn from a recorded Zoom interview conducted in March/April 2026. Dr Kaushal was informed that an article would be written from this conversation and agreed to the interview on that basis.

CHILD Trial

• Kaushal, S., Hare, J. M., Mahle, W. T., Khan, A., Ohye, R. G., Slesnick, T. C., Chai, P. J., Shashidharan, S., Robinson, J. D., Jone, P.-N., Doman, T., Si, M.-S., Lu, J. C., Bacallao, K., Nettina, A. E., Lamazares, R., Saltzman, R. G., Simpson, L. M., Li, R., … Davis, M. E. (2026). Phase I randomized study of cardiac stem cells in patients with hypoplastic left heart syndrome: The CHILD trial. JACC: Heart Failure, 14(1), 102723. https://doi.org/10.1016/j.jchf.2025.102723

• Kaushal, S., Hare, J. M., Shah, A. M., Pietris, N. P., Bettencourt, J. L., Piller, L. B., Khan, A., Snyder, A., Boyd, R. M., Abdullah, M., Mishra, R., Sharma, S., Slesnick, T. C., Si, M.-S., Chai, P. J., Davis, B. R., Lai, D., Davis, M. E., & Mahle, W. T. (2022). Autologous cardiac stem cell injection in patients with hypoplastic left heart syndrome (CHILD study). Pediatric Cardiology, 43(7), 1481–1493. https://doi.org/10.1007/s00246-022-02872-6

Anversa Fraud & Field Impact

• Oransky, I., & Marcus, A. (2018, October 14). Harvard calls for retraction of dozens of papers by former professor. STAT News. https://www.statnews.com/2018/10/14/harvard-brigham-retractions-stem-cell/

• Servick, K. (2018, October 15). Retract cardiac stem cell papers, Harvard Medical School says. Science. https://www.science.org/content/article/retract-cardiac-stem-cell-papers-harvard-medical-school-says

• O’Riordan, M. (2018). NIH presses pause on cardiac stem cell trial, further muddying a murky field. TCTMD. https://www.tctmd.com/news/nih-presses-pause-cardiac-stem-cell-trial-further-muddying-murky-field

• O’Riordan, M. (2018). Stem cell research “shattered” after fabrication scandal, needs rebuild, says EHJ editor. TCTMD. https://www.tctmd.com/news/stem-cell-research-shattered-after-fabrication-scandal-needs-rebuild-says-ehj-editor

8-Year Japanese Follow-Up (CDC Outcomes)

• Ishigami, S., Ohtsuki, S., Tarui, S., Ousaka, D., Eitoku, T., Kondo, M., Okuyama, M., Kobayashi, J., Baba, K., Arai, S., Kawabata, T., Fujii, K., Masumoto, H., Kato, H., Sakata, K., Ohtani, H., Sano, S., & Oh, H. (2024). Eight-year follow-up of cardiac function and quality of life in patients with single ventricle physiology after cardiosphere-derived cell infusion. Journal of the American Heart Association, 13, e034956. https://doi.org/10.1161/JAHA.124.034956

• Longeveron Inc. (2026, January). FDA Type C meeting announcement re: ELPIS II (laromestrocel for HLHS). https://www.theglobeandmail.com/investing/markets/markets-news/GlobeNewswire/36466953/longeveron-announces-fda-grants-type-c-meeting-ahead-of-data-readout-for-pivotal-phase-2-clinical-trial-elpis-ii-evaluating-treatment-for-hypoplastic-left-heart-syndrome-hlhs/. Top-line data expected Q3 2026.

• ELPIS II clinical trial: NCT05962658. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT05962658

990-PF Filings

The Marcus Foundation (EIN 58-1815651). IRS Form 990-PF filings, 2019–2024. ProPublica Nonprofit Explorer. https://projects.propublica.org/nonprofits/organizations/581815651

Dr. Kaushal Research Timeline

• Compiled from PubMed (

https://pubmed.ncbi.nlm.nih.gov/?term=kaushal+s+cardiac+stem+cell

• ) and NIH Reporter (https://reporter.nih.gov/).

Interview

• Kaushal, S. (2026, March). Personal interview via Zoom [recorded with consent]. Conducted by M.-J. Herard Furey for Between the Beats.

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