Friends,

Saddle up because off we go again with a whirlwind of hope and news. Hydrate and read on because this issue is a head full and a mouthful.

Three months ago the question was: "Does CAR-T even work for rare neuro?"

Today it's: "How do I get it, and will insurance cover it?"

The conversation shifted. The timeline compressed. This week: five fast stories that matter RIGHT NOW, plus three questions to bring to your next appointment.

We are NOT recommending anything or any changes in your health strategy. We want to show options and help educate not replace your healthcare team!

✅ Stem cells show measurable drops in brain inflammation on MRI
✅ NIH opens ultra-rare gene therapy pathway (URGenT Network)
✅ Fanconi anemia survivors need neuro screening as they live longer
✅ San Antonio testing new flu antivirals (matters for GBS/CIDP)
✅ Three questions to ask your neurologist before New Year's

⏱ Read time: 4 minutes

Longeveron's mesenchymal stem cell therapy showed measurable drops in MRI markers of brain inflammation in Alzheimer's patients versus placebo. FDA already gave it fast-track designation.

Why rare neuro should care: This proves cell therapies can move from "maybe" to "measurable MRI changes" in the brain. That helps every inflammatory neuro disease make its case.

Your action: Email your neurologist: "Are stem-cell or other cell-based approaches being studied for our disease? Who should I be following?"

The URGenT (Ultra-rare Gene-based Therapy) Network exists specifically to move gene or RNA therapies for tiny patient populations from late-stage lab work into first-in-human trials. Two-stage application process with trial infrastructure support.

Translation: If your disease affects 50 people worldwide and someone has a gene therapy idea, URGenT is the front door.

Your action: Google "URGenT Network NINDS" plus your disease name. If nothing shows up, ask your specialist: "Is anyone thinking about URGenT for our condition?"

Adults with Fanconi anemia are living longer thanks to better transplants. Now some are showing balance changes, cognitive issues, and neurologic symptoms that older studies never tracked. University of Rhode Island just got NIH funding to study this.

Bigger picture: When a "blood disease" or "immune disease" finally comes under control, it's time to ask if long-term follow-up should include neurology or cognitive screening.

Your action: Ask clinic: "Now that my primary condition looks better, should long-term follow-up include neurology or neuropsych testing?"

Salarius Pharmaceuticals and Texas Biomedical Research Institute are testing computer-designed antivirals against H5N1 and other flu strains in San Antonio. Still early lab work, not people yet.

Why this matters: Severe infections trigger GBS, CIDP relapses, and autoimmune encephalitis crises. Anything cutting serious flu risk could mean fewer neuro crashes.

Your action: Ask your doctor: "Given my condition, what's the current plan to reduce infection-triggered flares—vaccines, early antivirals, anything new?"

CAR-T "immune reset" is in early trials for myasthenia gravis and other immune diseases. Mesenchymal stem cells are being tested for neuroinflammation. Gene therapy and antisense drugs are moving in parallel for genetic neuromuscular conditions.

Honest truth: Most of this is still Phase 1-2. Access is through tightly controlled trials, not everyday practice. But you're not "too early" to ask—you're right on time.

Print this. Stick it in your bag. Bring it to your next appointment.

Question 1:
"Between CAR-T, stem-cell therapy, antisense drugs, and gene therapy, which (if any) are being studied for my specific disease right now—and where are those trials?"

Why it matters: Your neurologist may not have the full list. You're asking what exists so you can follow up.

Question 2:
"If I'm not sick enough—or I'm too complicated—for current trials, what would need to happen for me to become eligible in the next 2-3 years?"

Why it matters: This gets you a realistic roadmap. Know whether to watch closely now or think in five-year terms.

Question 3:
"Is there a patient registry, natural-history study, or biobank for our disease? If not, would you help connect me with researchers who might start one?"

Why it matters: Registries and biobanks are how ultra-rare groups become visible to networks like URGenT. This is how your disease gets on the system's radar.

You might be thinking: "This is a lot. I'm exhausted already."

That reaction is normal.

So pick ONE thing. Not all five.

• If you have a blood or immune disease that's finally stable, ask about long-term neuro screening
• If you have an ultra-rare genetic condition, Google "URGenT Network" plus your disease name
• If you have autoimmune neuro, ask where your disease sits in the CAR-T/stem-cell picture
• If infections knock you down, ask about prevention plans

If you're overwhelmed, choose the question that scares you most and ask it anyway. That's the action.

UT Southwestern Neuromuscular Center
ALS, CIDP, MG – emerging therapies
(214) 645-8800

Houston Methodist Neuromuscular Clinic
CIDP, MG, NMOSD – second opinions, trials
(713) 441-3760

Texas Children's Cell & Gene Therapy
Pediatric rare neuro – 10,000+ patients treated
(832) 824-1000

GBS/CIDP Foundation Clinical Trial Hotline
National trial matching (business hours)
1-866-224-3301

We hope this issue moved fast. That was intentional.

The rare-neuro field is accelerating. CAR-T, stem cells, antivirals, gene networks—they're all real. They're all happening. And the families who get access first aren't always the sickest. They're usually the ones who asked the clearest questions, built relationships with research centers, and didn't wait for permission.

You don't need to be a researcher. You don't need a medical degree. You need to be awake, curious, and willing to ask one hard question this week.

Do that. Then email us: What question did you ask? What answer surprised you? (anonymously, of course—we're not trying to doxx anybody.)

We are not doctors. Everything here is verified against FDA, NIH, ClinicalTrials.gov, and peer-reviewed sources. If your condition matches something we mentioned, talk to your clinician first, then come back here with what you learned. Feedback keeps us honest. When in doubt, talk to your doc—then swap stories with us!

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