XIT Foundation

🧬 ALL | ONE INFUSION. THE BODY BECOMES THE FACTORY.

Early human gene-therapy data: 3/3 MRD-negative complete responses

This one is fascinating.

VNX-101 is an experimental gene-delivery therapy being studied in people with relapsed or refractory CD19-positive blood cancers, including B-cell acute lymphoblastic leukemia (ALL).

And instead of repeatedly giving a cancer-fighting drug, researchers are trying something very different:

Give the body the genetic instructions once — and let the body keep making the therapy itself.

🔬 WHAT HAPPENED?

Among the first 3 evaluable patients with relapsed/refractory ALL who were negative for antibodies against the AAV vector before treatment:

3/3 achieved complete remission.

3/3 became MRD-negative.

All three MRD-negative results were confirmed using clonoSEQ, a highly sensitive test capable of detecting tiny amounts of residual leukemia.

One patient also had extensive leukemia outside the bone marrow and achieved a complete response at all disease sites on PET imaging by Day 28.

🧬 XIT DECIPHERS THE SCIENCE: HOW DOES ONE DOSE KEEP WORKING?

VNX-101 uses an engineered AAV — adeno-associated virus — as a delivery vehicle.

Think of the AAV as a microscopic delivery package.

Inside are genetic instructions telling primarily the patient’s liver cells how to manufacture a therapeutic protein called GP101.

GP101 is a CD19 × CD3 T-cell engager.

One end recognizes CD19 on leukemia cells.
The other recognizes CD3 on the patient’s T cells.

It essentially brings the patient’s own immune cells face-to-face with the leukemia and tells them:

“This is the cell to attack.”

But here’s the part we’re particularly interested in:

The liver keeps producing the cancer-targeting protein after the original infusion.

The first reported patient remained MRD-negative through Day 260 and then proceeded to stem-cell transplantation while still in MRD-negative remission.

Even after transplant, GP101 remained detectable at therapeutic levels at least 9 months after the original single VNX-101 infusion.

So rather than:

drug → drug disappears → give another dose

the concept is:

ONE INFUSION

↓
deliver genetic instructions
↓
the body becomes a long-lasting “factory”
↓
continuously produces the T-cell engager
↓
keeps recruiting T cells against CD19+ leukemia

That is why we’re watching this.

🎗️ WHAT ABOUT CHILDREN?

The 3 patients in this new report were adults — these are NOT pediatric results.

The current SENTRY-CD19 trial is a Phase 1/2 first-in-human study.

Part 1: ages 18–90

Part 2: the protocol allows expansion to patients 13–90 years old after dose selection and safety review.

VNX-101 has received FDA Rare Pediatric Disease, Fast Track and Orphan Drug designations. Those designations can facilitate development, but they do not mean the therapy is FDA-approved or currently available to children.

⚠️ WHAT FAMILIES SHOULD KNOW

This is very early evidence.

Three out of three MRD-negative complete responses is an exciting signal, but three patients cannot tell us what the eventual response rate will be.

These results were announced by the company and have not yet been published as peer-reviewed clinical results.

The first patient also proceeded to stem-cell transplantation. We therefore cannot say VNX-101 produced a cure or attribute subsequent long-term disease control solely to the gene therapy.

And because this is AAV-based gene delivery, patients must also be screened for antibodies against the AAV capsid. The current protocol requires an AAV antibody level below 1:400.

The trial includes long-term safety monitoring for 6–15 years, which is appropriate for a gene-delivery therapy designed to produce prolonged biological effects.

🔭 WHY XIT IS WATCHING

We’re interested in more than the 3/3.

We’re watching the platform.

If researchers can safely give the body genetic instructions that allow it to manufacture a therapeutic protein for months — rather than manufacturing personalized cells outside the body or repeatedly administering a drug — that could represent an entirely different way of delivering cancer immunotherapy.

And the concept may eventually extend beyond CD19 and ALL.

The underlying scientific question is enormous:

Can we program the body once and turn it into its own long-lasting medicine factory?

For now, we have an intriguing first human signal.

One infusion.
Three ALL patients.
Three MRD-negative complete remissions.
Therapeutic protein detected 9+ months after treatment in the first reported patient.

Now we need more patients, longer follow-up, detailed safety data, peer-reviewed clinical results — and pediatric data.

🧬FAMILIES INTERESTED IN THE TRIAL:

1. SENTRY-CD19 clinical trial — https://clinicaltrials.gov/study/NCT06533579

2. Peer-reviewed VNX-101 platform paper — https://pubmed.ncbi.nlm.nih.gov/40799205/?utm_source=chatgpt.com

3. September 21 VNX-101 clinical-results announcement⁠ — https://vironexis.com/vironexis-announces-complete-and-durable-responses-in-relapsed-refractory-all-patients-treated-with-vnx-101/

EVIDENCE LEVEL: EARLY HUMAN • PHASE 1/2 • n=3 ALL • ADULT DATA • CLINICAL RESULTS NOT YET PEER-REVIEWED

XIT Research Review: For educational and informational purposes only. This is not medical advice. Clinical-trial eligibility and treatment decisions should be discussed with the treating oncology team and study investigators.

🧬 Very Exciting ALL News — ANNOUNCED TODAY

ONE IV DOSE → 3/3 MRD-NEGATIVE COMPLETE RESPONSES

September 21, 2026: New early human results were announced today from VNX-101, an investigational gene-delivered immunotherapy being studied in patients with relapsed/refractory acute lymphoblastic leukemia (ALL).

The first ALL results are small — but noteworthy:

3 patients treated.
3 MRD-negative complete responses.
All 3 confirmed by clonoSEQ.

One patient with extensive disease outside the bone marrow also achieved a complete response at all disease sites on PET by Day 28.

The first treated patient remained MRD-negative through Day 260 and then proceeded to stem-cell transplant while still in complete remission.

🧬 WHAT MAKES THIS DIFFERENT?

VNX-101 isn’t traditional CAR-T.

It uses an AAV vector to deliver genetic instructions in vivo. After a single IV administration, the patient’s liver is designed to continuously produce a CD19×CD3 T-cell engager, which brings the patient’s own T cells into contact with CD19-positive cancer cells.

In simple terms-

Instead of manufacturing cancer-fighting cells outside the body, this approach gives the body genetic instructions to continuously make a cancer-targeting therapy itself.

The platform is designed to avoid leukapheresis, ex-vivo CAR-T manufacturing and repeat therapeutic infusions.

🎗️ WHAT ABOUT CHILDREN WITH ALL?

This is particularly interesting for pediatric cancer because VNX-101 has already received FDA Rare Pediatric Disease designation, as well as Fast Track and Orphan Drug designations.

However, there is an important distinction for families:

Vironexis currently describes the actively enrolling VNX-101 trial as enrolling adults.

So this is not currently a treatment that every child with ALL can sign up to receive.

For families with a child or adolescent with relapsed/refractory CD19-positive ALL, it may still be worthwhile to have the treating oncologist contact the study team to ask about future pediatric enrollment and upcoming cohorts.

🔎 HOW TO FIND THE TRIAL

https://clinicaltrials.gov/study/NCT06533579

Families should contact the trial through the study contact information listed on ClinicalTrials.gov or have their oncologist contact the nearest participating center. The study team performs the actual prescreening and determines eligibility.

⚠️ IMPORTANT CONTEXT

3/3 MRD-negative complete responses is an encouraging early human signal — but three patients is still only three patients.

This is an early Phase 1/2 study, not evidence of a 100% response rate in a larger population and not evidence of a cure rate.

One of the patients subsequently underwent stem-cell transplantation, which also means longer-term remission after transplant cannot be attributed to VNX-101 alone.

Why XIT is watching 🧬

The concept is what makes this particularly compelling:

One IV administration → genetic instructions delivered inside the body → the patient’s own cells continuously manufacture a cancer-targeting immunotherapy.

If these early responses prove reproducible and durable in larger studies, this could represent an important new way to deliver cancer immunotherapy.

XIT Research Review: For educational and informational purposes only. This is not medical advice. Clinical-trial eligibility and treatment decisions should be discussed with the treating oncology team and study investigators.



https://allsci.com/news/clinical-trials/vnx-101-gene-therapy-all-achieves-mrd-negative/

Vironexis reports early durable ALL responses with one-time AAV therapy Vironexis' VNX-101 AAV gene therapy produces MRD-negative responses in relapsed/refractory ALL patients. Single-dose treatment shows durable results. Read more.

🧬 GPC3-POSITIVE SOLID TUMOR NEWS

AFTER 2 DOSES OF ENGINEERED CAR-T, NO CANCER DETECTED

(A 3-year-old with chemotherapy-resistant metastatic hepatoblastoma achieved complete regression after two CAR-T infusions, with the response ongoing for 12+ months.)

The trial is currently recruiting. See below.

Researchers have reported in the New England Journal of Medicine the case of a 3-year-old with metastatic hepatoblastoma whose cancer had stopped responding to chemotherapy.

Before entering the trial, he had already received:

3 lines of chemotherapy
Removal of the primary liver tumor
2 surgeries for lung metastases

The cancer still returned.

He then entered the CARE Phase 1 trial — NCT04715191 — testing an unusually engineered form of CAR-T therapy.

The researchers took the child’s own T cells and genetically modified them with:

GPC3 CAR → designed to recognize the tumor

IL-15 + IL-21 → engineered into the cells to help support their activity and persistence

Inducible caspase-9 → a built-in safety switch that can be used to eliminate the engineered cells if necessary

The child received two outpatient infusions eight weeks apart.

After the first:

PARTIAL RESPONSE.

After the second:

COMPLETE REGRESSION.

And at the time of publication:

NO DETECTABLE DISEASE FOR AT LEAST 12 MONTHS.

Investigators reported no cytokine-release syndrome and no dose-limiting toxicity in this child.

For a chemotherapy-resistant metastatic pediatric solid tumor, that is an extraordinary clinical observation.

We do not yet know whether other children will have the same response or whether this remission will remain durable for many years.

The Phase 1 CARE trial is recruiting children and young adults with qualifying GPC3-positive solid tumors.

🎗️ Cancers listed in the trial include:
Liver cancers (including hepatoblastoma) • Rhabdomyosarcoma • Malignant rhabdoid tumor • Liposarcoma • Wilms tumor • Yolk sac tumor

Ages 1–21 • Texas Children’s Hospital • Houston, TX

Clinical Trial Link:
https://clinicaltrials.gov/study/NCT04715191

Contacts
Texas Children’s Hospital
David Steffin, MD
[email protected]

GPC3-POSITIVE SOLID TUMOR NEWS 🧬

AFTER 2 DOSES OF ENGINEERED CAR-T, NO CANCER DETECTED

(A 3-year-old with chemotherapy-resistant metastatic hepatoblastoma achieved complete regression after two CAR-T infusions, with the response ongoing for 12+ months.)

The trial is currently recruiting. See below.

Researchers have reported in the New England Journal of Medicine the case of a 3-year-old with metastatic hepatoblastoma whose cancer had stopped responding to chemotherapy.

Before entering the trial, he had already received:

3 lines of chemotherapy
Removal of the primary liver tumor
2 surgeries for lung metastases

The cancer still returned.

He then entered the CARE Phase 1 trial — NCT04715191 — testing an unusually engineered form of CAR-T therapy.

The researchers took the child’s own T cells and genetically modified them with:

GPC3 CAR → designed to recognize the tumor

IL-15 + IL-21 → engineered into the cells to help support their activity and persistence

Inducible caspase-9 → a built-in safety switch that can be used to eliminate the engineered cells if necessary

The child received two outpatient infusions eight weeks apart.

After the first: PARTIAL RESPONSE.

After the second: COMPLETE REGRESSION.

And at the time of publication:

NO DETECTABLE DISEASE FOR AT LEAST 12 MONTHS.

Investigators reported no cytokine-release syndrome and no dose-limiting toxicity in this child.

For a chemotherapy-resistant metastatic pediatric solid tumor, that is an extraordinary clinical observation.

We do not yet know whether other children will have the same response or whether this remission will remain durable for many years.

The Phase 1 CARE trial is recruiting children and young adults with qualifying GPC3-positive solid tumors.

🎗️ Cancers listed in the trial include:
Liver cancers (including hepatoblastoma) • Rhabdomyosarcoma • Malignant rhabdoid tumor • Liposarcoma • Wilms tumor • Yolk sac tumor

Ages 1–21 • Texas Children’s Hospital • Houston, TX

Clinical Trial Link:
https://clinicaltrials.gov/study/NCT04715191

Contacts
Texas Children's Hospital
David Steffin, MD
[email protected]

Source: Steffin D, Courtney AN, Choe M, et al. New England Journal of Medicine. September 9, 2026. 395:1029–1032. DOI: 10.1056/NEJMc2605958. CARE trial: NCT04715191. (New England Journal of Medicine)

Human Phase 1 case report. One patient. Complete regression lasting at least 12 months does not establish a cure rate or prove efficacy in other patients. Investigational therapy; educational information only, not medical advice.



https://www.texaschildrens.org/content/news-release/care-study-reports-complete-regression-liver-cancer-child-treated-with-novel

/CARE study reports complete regression of liver cancer in a child treated with novel immunotherapy A new report in the New England Journal of Medicine by researchers at Texas Children’s, Baylor College of Medicine and Seattle Children’s describes a complete regression of hepatoblastoma, the most common pediatric liver cancer, in a 3-year-old treated with a novel immunotherapy. The patient is ...

🎗️ XIT RESEARCH REVIEW: Mistletoe Extract + Osteosarcoma

This one has moved beyond the laboratory.

A pharmaceutical preparation derived from European mistletoe (Viscum album) is now being studied in a U.S. Phase 2 trial for children and young adults with relapsed osteosarcoma.

But this is important: researchers aren’t studying mistletoe tea or an over-the-counter supplement.

They are studying Iscador® P, a standardized, fermented aqueous extract made from the leaves, stems and berries of European mistletoe grown on pine trees. Its constituents include mistletoe lectins, viscotoxins and other biologically active compounds.

🧫 WHAT HAPPENED IN THE LAB?

In preclinical research, Viscum album preparations have shown several potential anticancer mechanisms, including induction of apoptosis (programmed cell death), inhibition of proliferation and effects on immune signaling and the tumor microenvironment. Laboratory studies have reported cytotoxic effects in osteosarcoma, Ewing sarcoma and rhabdomyosarcoma cell lines.

In osteosarcoma cells specifically, mistletoe extract altered pro- and anti-apoptotic proteins in a direction associated with increased cancer-cell death. Researchers have also investigated a mistletoe-derived microRNA called val-miR218, which inhibited osteosarcoma-cell proliferation in vitro and affected pathways involving the cell cycle and DNA replication.

👥 WHAT HAPPENED IN HUMANS?

This is where the finding becomes particularly interesting.

A very small randomized study enrolled 20 patients with osteosarcoma who were disease-free following surgery after a second metastatic relapse. Nine received subcutaneous Viscum album extract and 11 received oral etoposide (chemo).

At 12 months:

Viscum album: 55.5% post-relapse event-free survival
Etoposide: 27.3%

With longer follow-up, 5 of the 9 mistletoe-treated patients had never relapsed, whereas all 11 patients assigned to oral etoposide had relapsed. Median follow-up in the later report was 83 months (range 3–144 months).

The NCI’s independent review summarizes the long-term comparison as a 71% lower hazard of relapse with Iscador P versus oral etoposide (HR 0.287; 95% CI 0.076–0.884; P=.03).

⚠️ This does NOT establish that mistletoe prevents osteosarcoma relapse.

Twenty patients—and particularly nine patients in the mistletoe arm—is far too small a study to establish efficacy reliably. The findings need confirmation in a substantially larger prospective trial.

🇺🇸 NOW IT’S BEING TESTED IN A U.S. PHASE 2 TRIAL

That confirmation is now being attempted.

Clinical Trial Link: https://clinicaltrials.gov/study/NCT05726383

Locations:
Texas- M.D. Anderson Children's Cancer Hospital
Oncologist Email: [email protected]

New Jersey- Hackensack University Medical Center
Nurse Email: [email protected]

MISTOSUS — NCT05726383 is a multicenter Phase 2 study testing Iscador P after complete surgical removal of recurrent pulmonary osteosarcoma.

The trial plans approximately 32 patients, ages 8–30. Its primary endpoint is 12-month post-relapse event-free survival. The investigators plan to compare the results with historical outcomes rather than a randomized concurrent control group.

Current sources list the study as recruiting/active, including at MD Anderson Children’s Cancer Hospital in Houston and Hackensack University Medical Center in New Jersey.

There are no Phase 2 efficacy results yet.

⚠️ WHAT FAMILIES SHOULD KNOW

This research does not mean commercially available mistletoe supplements have been shown to treat osteosarcoma.

The clinical research uses a specific pharmaceutical preparation, Iscador P, administered subcutaneously under a defined clinical protocol. Different mistletoe extracts can have different chemical compositions depending on the host tree and manufacturing process.

The NCI notes that the overall mistletoe literature has important methodological weaknesses and currently states that mistletoe cannot be recommended as a cancer treatment outside well-designed clinical trials.

In the earlier osteosarcoma study, reported adverse effects in the mistletoe arm included local injection-site redness and hypotension in one patient. More broadly, injection-site reactions and temporary flu-like symptoms are among reported adverse effects.

🔬 WHY XIT IS WATCHING THIS

The interesting story is that a naturally derived, standardized pharmaceutical preparation produced enough preliminary biological and human evidence to justify a prospective Phase 2 osteosarcoma trial.

And now science gets to answer the question that matters:

Does the signal hold up in a larger group of patients?

We’ll be watching the data.

**Research update—not medical advice. Supplements can interact with cancer treatment. Discuss any supplement with your child’s oncology team.**

📚 PRIMARY SOURCES

2026 MISTOSUS Phase 2 paper — Future Oncology / PubMed Central⁠
DOI: 10.1080/14796694.2026.2638986

https://www.cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2023-08825

MD Anderson — Iscador P osteosarcoma trial⁠
https://www.mdanderson.org/patients-family/diagnosis-treatment/clinical-trials/clinical-trials-index/clinical-trials-detail.ID2022-0877.html?utm_source=chatgpt.com

Clinical trial: NCT05726383

Care enough that someday, somewhere, something is different because you were here.

We don’t get to choose how much time we’re given.
But we do get to choose what we do with it.

Sasha died from cancer,
But.
She is still changing rooms she never entered.

On walls. On signs.
In conversations about cures.
In rooms filled with people who refuse to accept that the next child has to share her fate.

Thank you, Beat Childhood Cancer Foundation & Sarah Detling Bartosz , for helping her remind people that this fight is far from over.

None of us gets to decide how long we are here.

But while we are here, we do get to decide what we move.
What we question.
What we build.
What we refuse to accept.
Who we fight for.
What becomes different because we existed.

Sasha was just a little girl.
She didn’t have decades to build a legacy.

And yet her name is now woven into a movement fighting for a future she never got to have.

So maybe a meaningful life isn’t measured in years.
Maybe it’s measured in what keeps moving after we are gone.

There are diseases waiting for cures.
People waiting for someone to care.
Problems everyone has learned to live with.
Things in this world that are still broken because too many of us assume someone else will fix them.

Pick something.

We all leave eventually.
The question is what refuses to leave with us.

Sasha picked to fight for a cure.
And we are just getting started. 🎗️
XIT.org

🧬 FOR DIPG

September 8, 2026: BrainChild Bio formally announced that it had initiated the Phase 2 ILLUMINATE trial and publicly launched the program.

This is a major step forward in pediatric brain-cancer research.

A genetically engineered CAR-T therapy originally developed at Seattle Children’s has now entered a pivotal Phase 2 clinical trial for newly diagnosed DIPG.

The trial is called :
ILLUMINATE -
https://clinicaltrials.gov/study/NCT07680439

Announcement Details Sept 8,2026:https://brainchildbio.com/wp-content/uploads/2026/09/BCBio_Phase2InitiationPR_FINAL_09-08-26.pdf

Researchers take a child’s own T cells and genetically engineer them to recognize:

B7-H3 🎯

—a protein found on DIPG cells.

But there is another important difference.

Instead of giving the CAR-T cells through an IV and asking them to cross the blood-brain barrier, researchers deliver them directly into the cerebrospinal fluid surrounding the brain through an implanted reservoir.

Children can receive:
UP TO 15 CAR-T INFUSIONS
approximately every 2 weeks
over 7–8 months

And this time the therapy is being studied soon after initial radiation—not only after multiple relapses.

The Phase 2 trial plans to enroll:
75 PATIENTS
AGES 1–26
across six U.S. pediatric cancer centers.

Why did researchers move this therapy forward?

In the Phase 1 BrainChild-03 trial:
21 patients with DIPG received CAR-T
and investigators administered:
253 CAR-T DOSES

Median survival from diagnosis was:
19.8 MONTHS

And three patients were still alive at approximately:
44.6 months
45.6 months
52.5 months
after diagnosis.

That is notable in a disease where historical median survival is approximately 11 months.

But this needs to be said clearly:
Those numbers do NOT prove CAR-T cures DIPG.

The earlier study was Phase 1, small and nonrandomized, and several factors can affect comparisons with historical survival.

Among 18 patients evaluable by MRI, researchers saw:
1 partial response
15 stable diseases
2 progressive diseases

The primary endpoint of ILLUMINATE is overall survival.

And the trial was designed in alignment with the FDA as a pivotal registrational study, meaning positive results could potentially form the basis of an application for approval. BCB-276 has also received FDA expedited-development designations during its development.

For educational purposes only. This information is not medical advice and should not replace guidance from your child’s treating oncology team.

XIT. org | Let’s X-IT Childhood Cancer



https://brainchildbio.com/

BrainChild Bio, Inc. - A kids-first, clinical-stage biotechnology company BrainChild Bio is a kids-first, clinical-stage biotechnology company harnessing the power of CAR-T cell technology for tumors in the central nervous system (CNS), prioritizing pediatrics with plans to expand our technology platform to adult indications.

🧬 A PERSONALIZED mRNA CANCER VACCINE IS NOW RECRUITING CHILDREN WITH HIGH-GRADE BRAIN TUMORS

What if instead of choosing one cancer target…scientists could use the genetic information from a child’s own tumor to teach the immune system what that cancer looks like?

That is now being tested in children.

This trial is especially interesting because researchers are testing the approach in newly diagnosed pediatric high-grade glioma after surgery and radiation — when the amount of cancer remaining may be much smaller.

Instead of waiting for enormous treatment-resistant relapse: they are attacking minimal residual disease.

Researchers at the University of Florida and Pediatric Neuro-Oncology Consortium are studying a personalized RNA-lipid-particle vaccine called PNOC020.

Here is how it works:

🧠 Tumor tissue is collected during surgery.

🧬 Researchers extract and amplify RNA from that individual child’s cancer.

🔬 The tumor RNA is packaged inside tiny lipid particles.

💉 The RNA vaccine is given intravenously.

🎯 The goal is to activate antigen-presenting cells and train the patient’s immune system to recognize many tumor antigens at once.

The pediatric high-grade glioma study is currently recruiting: PNOC020 — NCT04573140

Children receive:

💉 3 RNA-LP vaccines approximately every 2 weeks

followed by

💉 12 monthly vaccines

for approximately 15 total doses.

And there is now a separate first-in-human PNOC020 study for: RECURRENT / PROGRESSIVE MEDULLOBLASTOMA — NCT07492316

That study also manufactures the RNA vaccine using tumor material from the individual patient.

The long-term scientific question :
Can a personalized tumor-RNA vaccine extend progression-free survival?

The PNOC program plans to evaluate 12-month PFS in pediatric high-grade glioma as the platform advances.

⚠️There are NO pediatric efficacy results from these trials yet. This is early Phase 1 human research.

No complete-remission rate.
No PFS result.
No survival advantage has yet been demonstrated.

But this is no longer simply a mouse experiment.

Children are now entering clinical studies in which genetic material from their own tumors is being turned into personalized RNA cancer vaccines.

That is a major translational step. 🧬🎗️

XIT.org

🔗 CLINICAL TRIAL + RESEARCH LINKS

Newly diagnosed pediatric high-grade glioma — PNOC020
NCT04573140
https://clinicaltrials.gov/study/NCT04573140

Recurrent/progressive medulloblastoma — PNOC020 rMB
NCT07492316
https://clinicaltrials.gov/study/NCT07492316

Evidence level: Human Phase 1 • Recruiting • Personalized tumor-RNA vaccine • No efficacy results yet • Experimental therapy • Educational only — not medical advice.