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The Possibility of a Pill to Repair the Brain: It's Not Magic Powder. The Key to the "Brain-Healing Pill" is "Pairing"

The Possibility of a Pill to Repair the Brain: It's Not Magic Powder. The Key to the "Brain-Healing Pill" is "Pairing"

2025年09月06日 09:47

The headline from The New York Times (NYT) on September 4 was, "A Pill to Heal the Brain Could Revolutionize Neuroscience." This article provides an overview of the forefront of research aimed at enhancing recovery through "oral treatment" for neurodegenerative diseases, stroke, and traumatic brain injury (TBI). In other words, it challenges the medical student axiom that "the brain does not regenerate." The article was also featured in UCLA's news roundup, highlighting the discovery that the brain can sprout axons in remote areas after injury (sprouting). UCLA



What's "new": The idea of reopening the "window" of plasticity with drugs

The key concept is neuroplasticity. After an injury, the brain often opens a window of plasticity, which, when combined with learning and training, accelerates functional recovery—efforts are underway to intentionally reopen this window with molecular targeted drugs. The UCLA team focused on the role of parvalbumin-interneurons responsible for gamma oscillations and reported that a compound called DDL-920 enhanced motor recovery in a mouse stroke model. Pharmacological priming as an "accelerator" for regenerative medicine and rehabilitation is beginning to appear on the list of candidates for clinical application. UCLA BSCRCUCLA Health



Clinical trials for TBI: A strategy of multi-arm platforms using already approved drugs

In the field of traumatic brain injury (TBI), a multi-arm, multi-stage (MAMS) adaptive platform trial is underway, designed to compare the repurposing of already approved drugs such as statins, ARBs, and tetracyclines in parallel. The UCSF program is at the stage of verifying whether drugs like atorvastatin, candesartan, and minocycline can improve outcomes through anti-inflammatory, vascular protective, and microglial modulation, while setting up a clinical system to quickly identify "effective candidates." San Francisco Chronicleclinicaltrials.ucsf.edu



Existing evidence base: Amantadine and the "speed of recovery"

There has long been evidence that amantadine can enhance the speed of recovery of higher functions such as thinking and attention in TBI. While not a panacea, it has a wide safety margin, and the possibility that early administration can help "kickstart" functional recovery has been reproduced. The evaluation of new compounds and repurposed drugs builds upon such existing knowledge. Brain Injury Association of America



An "alternative route" with psychedelics: Reports on ibogaine research

Meanwhile, psychedelics such as ibogaine have the potential to promote synapse formation and network rewiring, with reported symptom improvements in veterans with TBI and PTSD. Although not yet sufficiently tested in randomized controlled trials (RCTs), the unique profile of "long-term effects with few doses" holds appeal as a "plasticity modulator." Stanford MedicinearXiv



"Magic powder" does not exist: Drugs must be paired with rehabilitation

As emphasized in the NYT article, drugs alone do not create the ability to "do" actions. Simply enhancing plasticity does not determine "which circuits to strengthen," so it is only when paired with rehabilitation tasks and learning tasks that the effects are maximized. This is the design philosophy of "drugs × training" common to motor learning, language training, and cognitive rehabilitation, and can be considered the implementation theory that most influences the clinical field. UCLA



Reactions on social media: Expectations, caution, and a practical perspective—A three-way battle

The NYT headline quickly spread on social media. **Voices of "revolutionary" expectations, caution to curb hype and focus on RCTs, and a practical perspective of "pairing with rehabilitation for realistic solutions"** are in a three-way standoff.

  • Optimists: In the tech-optimistic community, the enthusiastic tone of "Is a 'penicillin era' coming for the brain?" was prominent (discussed on Hacker News). Hacker News

  • Cautious voices: On X (formerly Twitter), there were frequent remarks that "'cure' is an overstatement. Robust human trials are needed." X (formerly Twitter)+1

  • Practical voices: Rehabilitation practitioners and clinical researchers shared the implementation point that "correct timing alignment (titration) with training is key" (UCLA-related mentions were cited and discussed). UCLA

Overall, voices seeking a balance of **"hope and verification"** are predominant. While being swept away by the momentum of the headline, the atmosphere of prioritizing evidence prevails.



Points for practical application: Who, when, and how to use it?

1) Indications: The pharmacology differs between acute inflammation suppression/edema reduction and subacute to chronic learning promotion. It is necessary to optimize the combination of drug × task for each patient group (stroke/TBI/hypoxic brain injury, etc.). News-Medical


2) Timing: It makes sense to administer in alignment with the "window of plasticity", bringing peak effects just before to just after task practice. UCLA


3) Safety: Central stimulants and psychedelics have issues such as their impact on
sleep, mood, and anxiety
, as well as dependency and long-term exposure problems. Optimization of dosage, frequency, and combination is essential. PMC


4) System: To reach conclusions quickly while keeping trial costs down, expanding MAMS-type platform trials is important. Payment designs that bundle insurance reimbursement and medical and intervention (rehabilitation) are also key. clinicaltrials.ucsf.edu


5) Social implementation/ethics: The concept of **"brain capital"** merges with policy, and the boundary with enhancement is also a topic of discussion. Frameworks like "Neuroshield" to protect the information environment and brain health are also in view. Baker Institute



The etiquette of the "age of the brain": Handling hype and advancing reality in three principles

  1. Do not get intoxicated by headlines, evaluate by outcomes. (Focus on real-life indicators such as ADL/QoL and return-to-work rates)

  2. Drugs are "amplifiers of practice." Design the entire intervention, including task design, frequency, intensity, sleep, and nutrition.

  3. Trials should be fast, but not sloppy. Explore with adaptive designs while prioritizing reproducibility and safety.



Conclusion: Hope is becoming reality—though it requires planning

The "pill to heal the brain" is no longer science fiction in the sense of pharmacologically supporting recovery at the circuit level. However, it is not a miracle drug on its own. Only by using it with the right patient, at the right time, paired with the right training can the "revolution" become reality. In the coming year, attention will be on the initial results of MAMS trials, RCTs of psychedelics, and the emergence of human applications of DDL-920. San Francisco ChronicleStanford MedicineUCLA BSCRC


Reference articles

Is a drug to heal the brain possible?
Source: https://www.nytimes.com/2025/09/04/science/neuroscience-brain-injury-pill.html

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