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Cure

A cure is a substance, , or that completely resolves a medical condition, such as a , by eliminating its cause, symptoms, and any risk of recurrence, thereby restoring the individual to full . This contrasts with a , which may manage or alleviate symptoms but does not necessarily eradicate the underlying condition. In , cures are achieved through methods like medications, surgeries, changes, or vaccinations, and their feasibility varies by —many infectious diseases are curable, while some conditions like certain cancers may only be manageable. In statistical and epidemiological contexts, particularly for curable diseases, the concept of a "" refers to the proportion of patients who achieve long-term equivalent to the general population, indicating they are . Cure models, such as mixture and non-mixture approaches, are used to estimate this from population-based data, aiding in monitoring treatment efficacy and disease trends. The pursuit of has driven medical advancements, though challenges persist for incurable diseases, as explored in subsequent sections.

Core Concepts

Definition

In medicine, a is defined as a therapeutic that completely eradicates a or medical condition, resulting in the restoration of normal without any expectation of recurrence or need for ongoing . This contrasts with partial or management strategies, which may alleviate symptoms or slow progression but fail to address the underlying cause, thereby requiring continuous to maintain . Examples of curable conditions include bacterial infections, such as strep throat or urinary tract infections, which can be fully resolved through antibiotics that target and eliminate the infecting pathogens. Similarly, nutritional deficiencies like , caused by deficiency, are cured by supplementation with ascorbic acid, leading to rapid reversal of symptoms and prevention of recurrence upon adequate intake. The concept of a cure has evolved significantly from ancient practices, where remedies often relied on humoral balance, interventions, or explanations for resolution, to modern that emphasizes empirical validation through clinical trials and targeted therapies. In contemporary contexts, the cure fraction represents the proportion of patients who achieve this complete resolution following .

Cure Fraction

The cure fraction refers to the proportion of patients in a who are effectively cured of their , meaning they achieve long-term survival without recurrence, often represented as the plateau level in a survival curve where the hazard rate approaches that of the general . In , this metric quantifies the subset of individuals who no longer experience -related mortality, distinguishing them from those susceptible to eventual failure. The Kaplan-Meier estimator can provide a non-parametric approximation of this plateau in survival data. The cure rate, closely related to the cure fraction, denotes the probability that a will be cured following a specific , typically contrasted with disease-free rates that may include temporary remissions. Unlike overall , which captures all causes of , the cure rate focuses on the treatment's ability to eradicate the disease permanently in a given . This probability is particularly relevant in curable cancers, where it serves as a for therapeutic success beyond mere prolongation of life. Several factors influence the cure fraction, including of at , which determines the extent of tumor burden and metastatic potential. Treatment efficacy, such as the use of targeted therapies or approaches, directly impacts the proportion achieving cure by enhancing tumor control. demographics, including , , , and , also modulate outcomes, with younger patients and those from higher socioeconomic backgrounds often showing higher cure fractions due to better access to care and physiological resilience. In chronic conditions like cancer, the psychological of cure plays a significant role, as patients may hesitate to embrace the "cured" label due to fears of recurrence or lingering . This hesitation can stem from misperceptions about treatment goals, where some individuals with incurable disease incorrectly believe their therapy aims for cure, affecting emotional adjustment and . Such perceptions highlight the need for clear communication in clinical settings to align expectations with statistical realities of the cure fraction.

Statistical Approaches

Estimation Methods

The Kaplan-Meier estimator provides a non-parametric approach to estimating the from right-censored data, enabling the visualization of survival probabilities over time without assuming a specific underlying distribution. In estimating cure probabilities, this method generates survival curves that may exhibit a plateau at extended follow-up periods, where the survival probability levels off; the height of this plateau serves as an indicator of the cure fraction, representing the proportion of individuals unlikely to experience the event of interest. The estimator handles censoring by incorporating only observed events up to each time point, ensuring unbiased estimates under the assumption of independent censoring. To assess treatment effects on cure, Kaplan-Meier curves of disease-free survival are commonly compared between treated and control groups, often using log-rank tests to evaluate differences in curve shapes and plateau levels. A visibly higher plateau in the treated group's disease-free survival curve relative to the control implies an increased cure rate attributable to the , providing intuitive evidence of long-term efficacy without parametric assumptions. Despite these advantages, estimating probabilities encounters significant challenges, including variability in follow-up durations among patients, which can distort plateau detection if short-term observations dominate the data. Right-censoring further complicates matters, as censored cases—arising from loss to follow-up or study termination—may include both cured and uncured individuals, leading to potential underestimation of the fraction unless follow-up is sufficiently prolonged to separate these groups. Limited follow-up exacerbates bias in plateau-based inferences, particularly when the true rate is modest. Practical implementation of these estimation methods is supported by statistical software; in , the survival package's survfit function computes Kaplan-Meier estimates and facilitates group comparisons, while PROC LIFETEST offers analogous tools for generating curves, testing differences, and managing censored data.

Key Models

Key models in the statistical analysis of cure rates primarily revolve around parametric frameworks that explicitly account for the presence of a cured subpopulation within data. The foundational approach is the mixture cure model introduced by Berkson and Gage in , which posits that the observed population consists of two distinct subpopulations: a cured group that experiences no further events (such as disease recurrence or death due to the condition) and a susceptible (uncured) group that follows a standard distribution. This model separates the overall into contributions from these groups, enabling estimation of the cure fraction even when long-term follow-up data may be limited. In parametric cure models, the population is formally expressed as S(t) = \pi + (1 - \pi) S_u(t), where \pi denotes the (the proportion of individuals who are cured and thus have infinite time with respect to the event of interest), and S_u(t) represents the for the uncured subpopulation, typically parameterized using distributions such as Weibull or log-logistic to capture the hazard dynamics. These models extend the Berkson-Gage framework by incorporating covariates into both \pi (often via ) and S_u(t) (via proportional hazards or accelerated failure time structures), allowing for personalized predictions of long-term outcomes. A core assumption of these models is homogeneity within the cured subpopulation, meaning all cured individuals share the same infinite profile with respect to the disease-specific event, though they may still face general mortality risks in population-based analyses. However, poses significant limitations, particularly with short-term follow-up data, where the survival curve's plateau (indicative of a cure fraction) can be confounded with a slowly declining tail in the uncured , leading to non-unique parameter estimates without sufficient long-term observations. This issue is exacerbated in datasets with heavy censoring, requiring careful model diagnostics and analyses to ensure reliable . These parametric frameworks find prominent applications in fitting data from cancer clinical trials, where they facilitate long-term by estimating cure fractions and projecting outcomes beyond observed follow-up periods, as demonstrated in analyses of and trials. For instance, in settings, they have been used to quantify the proportion of patients achieving durable remission after treatments like or , informing trial design and regulatory decisions. Unlike non-parametric precursors such as the Kaplan-Meier , which can suggest a plateau but lack explicit parameterization of the cure mechanism, these models provide interpretable components for hypothesis testing and covariate effects. In addition to mixture cure models, other key classes include non-mixture models such as the promotion time cure model (PTM), introduced by Chen, Ibrahim, and Sinha in 1999. The PTM assumes that the time to event for uncured individuals arises from the minimum promotion time among a random number of latent "competing risks" or cells, leading to a population survival function of the form S(t) = \int_0^\infty [S_u(t)]^k \, dF(k), where k is the random number of latent cells (often Poisson-distributed) and S_u(t) is the survival function for a single cell's promotion time. This approach differs from the mixture model by not assuming a distinct cured subgroup but rather deriving the cure probability endogenously from the distribution of latent cells, offering advantages in scenarios with biological interpretations involving tumor initiation. Semi-parametric extensions, such as the Cox proportional hazards cure model, combine the Cox model for the latency part with a logistic model for the incidence (cure fraction), allowing flexible hazard modeling without specifying S_u(t) fully. Recent developments as of have expanded cure modeling capabilities, including the integration of methods to enhance mixture cure models through improved variable selection, prediction accuracy, and handling of high-dimensional , as seen in applications using elastic net penalties or extensions for covariate effects. Additionally, new software tools like the curesurv facilitate the estimation of cure indicators in mixture and non-mixture (promotion time) models, particularly for cancer with complex censoring patterns, while joint models incorporating longitudinal and flexible hazard ratios address limitations in traditional frameworks. These advances enable more robust analyses in modern clinical trials with interval-censored or high-dimensional .

Remission and Recovery

Remission refers to the temporary absence or significant reduction of of a , distinguishing it from a permanent cure. In medical contexts, remission is classified as complete when all detectable evidence of the disease is eliminated, such as no leukemia cells in the and normalization of blood counts in (AML), or partial when symptoms and disease markers decrease but persist to some degree. For instance, in , complete remission is achieved when shows fewer than 5% blasts and peripheral blood counts return to normal following treatment, though this state does not guarantee the disease will not return. Recovery, in contrast, denotes the restoration of normal health or function after illness, often without fully resolving the underlying pathological process. This can occur after acute infections where the primary pathogen is cleared, but residual effects linger, as seen in post-viral fatigue syndromes following infections like Epstein-Barr virus or , where patients regain some daily functioning despite ongoing exhaustion and cognitive issues. Unlike remission, which focuses on activity, recovery emphasizes functional , though it may not address root causes such as immune dysregulation in post-viral conditions. The key distinctions between remission, recovery, and cure lie in their duration and reliability: remission and carry a substantial risk of , whereas a implies permanent eradication of the with no possibility of recurrence. In remission, disease signs may re-emerge due to dormant cells or triggers, as in cancer where can recur after initial complete remission; similarly, from viral illnesses can be incomplete if persists indefinitely. This contrasts with as the permanent endpoint, where the is fully eliminated. Clinically, these states necessitate vigilant monitoring to detect potential recurrence early, improving outcomes through timely intervention. For patients in remission, such as those with , regular follow-up includes blood tests, imaging, and biopsies to identify rising disease markers before symptoms return. In recovery phases like post-viral syndromes, ongoing assessment of symptoms and function helps manage lingering effects and prevents misattribution to new conditions. This surveillance underscores the provisional nature of remission and recovery, guiding strategies.

Response and Therapy

In medical contexts, particularly , a treatment response refers to a partial reduction in following , often quantified by at least a 20-50% shrinkage in tumor size or alleviation of symptoms such as or functional . This assessment, typically evaluated via imaging or clinical metrics like RECIST criteria, indicates the therapy's immediate impact without implying complete eradication. For instance, patients may experience symptom relief within 2-3 weeks of initiating , reflecting early biological activity against the disease. Therapies are broadly classified by intent: curative approaches aim to eliminate the entirely, while palliative ones focus on symptom management and quality-of-life improvement without targeting cure. Curative therapies, such as surgical resection for early-stage cancers like non-small cell or , remove localized tumors to achieve complete remission when the disease has not metastasized. exemplifies curative intent in certain lymphomas; for early-stage , regimens like yield cure rates exceeding 90%. In contrast, palliative controls advanced disease progression but does not pursue eradication. Within the pathway to a potential cure, an initial positive response serves as a key prognostic indicator, correlating with improved long-term outcomes and higher likelihood of sustained remission. Early tumor shrinkage, for example, identifies patients more likely to derive durable benefits from ongoing , guiding decisions on continuation or intensification. This predictive role underscores response evaluation as a dynamic tool in curative strategies, potentially leading to as an outcome. Preventive medicine complements therapeutic efforts by averting disease onset, contrasting with treatments for established conditions. Vaccinations, such as the administered before exposure, prevent up to 90-99% of cervical cancers caused by high-risk strains, thereby eliminating the need for curative interventions in many cases. This primary prevention shifts the focus from response and to avoidance, highlighting a proactive dimension in the broader cure paradigm.

Historical Developments

Pre-20th Century

In ancient and medieval periods, attempts to cure infections primarily involved herbal remedies derived from plants, which were applied topically or ingested to treat wounds and fevers, though these were frequently ineffective against underlying microbial causes. For instance, substances like for venomous bites and as an for open wounds were commonly used in monastic , reflecting a reliance on empirical observations passed down through texts such as those by and . Despite occasional palliative benefits, such remedies often failed to prevent or systemic spread, contributing to high fatality rates from even minor injuries. A landmark nutritional cure emerged in the mid-18th century with the treatment of scurvy, a debilitating disease plaguing long sea voyages. In 1747, Scottish naval surgeon James Lind conducted one of the earliest controlled clinical trials aboard the HMS Salisbury, assigning 12 scurvy patients to different dietary interventions and observing that two who received citrus fruits—oranges and lemons—recovered swiftly within six days, while others on vinegar, cider, or seawater showed no improvement. Lind's findings, detailed in his 1753 Treatise on the Scurvy, established citrus as an effective remedy, later understood to supply vitamin C, though adoption by the British Royal Navy lagged until the late 18th century. The late 19th century saw the advent of immunological cures for bacterial toxins, pioneered by Emil von Behring. In 1890, von Behring and Shibasaburo Kitasato developed the first antitoxin serum against tetanus by immunizing animals with sublethal toxin doses and harvesting protective antibodies from their blood, which neutralized the disease in experimental models. Building on this, von Behring extended the approach to diphtheria in 1891, producing a serum that significantly reduced child mortality rates from over 50% to around 15-20% in early clinical use during the 1890s. These serums represented a shift toward targeted therapies based on emerging understandings of immunity. Despite these breakthroughs, pre-20th century curative efforts were severely limited by the absence of germ theory, which was not widely accepted until the 1880s–1890s through work by and . Without recognizing pathogens as disease agents, treatments often addressed symptoms via humoral imbalances or miasmas, resulting in persistent high mortality—global life expectancy hovered around 30–40 years, largely due to uncontrolled infections. Even successful interventions like Lind's or von Behring's were hampered by inconsistent production, contamination risks, and incomplete disease models, underscoring the era's rudimentary scientific framework.

Early 20th Century

In the early 1920s, the discovery of insulin marked a pivotal advancement in treating , transforming it from a fatal condition to a manageable one. Canadian researchers and Charles Best, working at the , successfully isolated insulin from pancreatic extracts in experiments beginning in 1921, with the first successful injection in a human patient occurring in January 1922. This breakthrough, refined with the assistance of to produce a purer form, dramatically reduced mortality rates among children with the disease, enabling many to live productive lives beyond what was previously possible. Although insulin therapy requires lifelong administration and does not eliminate the underlying autoimmune process, it effectively reversed the immediate life-threatening in numerous cases, earning and John Macleod the 1923 Nobel Prize in Physiology or Medicine. Parallel developments in refined techniques, building on Jenner's 18th-century method to enhance efficacy and safety during the early 1900s. By the 1910s and 1920s, production shifted from risky arm-to-arm human transfers to more standardized methods using calf lymph or other animal sources, which reduced contamination risks and improved potency. These improvements, advocated by organizations like the League of Nations Health Organization, facilitated widespread campaigns in and the Americas, significantly lowering incidence rates during the 1930s and 1940s through campaigns, including mandatory school programs, with reported cases decreasing from over 42,000 in 1930 to about 2,800 by 1940 and the last endemic case occurring in 1949. Such refinements laid the groundwork for the global eradication efforts that would culminate decades later, demonstrating 's potential as a curative preventive measure against infectious diseases. The 1930s introduced the era of synthetic antibacterial agents with the discovery of sulfonamides, offering the first reliable chemical treatments for systemic bacterial infections. In 1932, German pathologist at identified —a red containing a group—as highly effective against streptococcal infections in mice, following tests that showed it protected against otherwise lethal doses of . Clinical trials soon confirmed its efficacy in humans, notably saving a young girl from a severe infection in 1935, and it rapidly became the standard for treating puerperal fever, , and other bacterial ailments. Domagk's work, which earned him the 1939 in or (though initially suppressed by the Nazi regime), spurred the development of sulfa drugs like , revolutionizing infectious disease management before natural antibiotics gained prominence. Despite these triumphs, the pre-penicillin transition to antibiotics in the faced significant challenges, including and emerging that limited sulfonamides' long-term utility. Early sulfa drugs often caused severe side effects such as kidney damage, , and allergic reactions, necessitating careful dosing and monitoring, while their bacteriostatic mechanism—merely inhibiting bacterial growth rather than killing—proved ineffective against certain pathogens like staphylococci. developed quickly in clinical settings, as adapted through mutations in synthesis pathways, foreshadowing broader challenges. These hurdles underscored the need for broader-spectrum agents, setting the stage for penicillin's wartime refinement, though sulfonamides remained a cornerstone therapy until the .

Mid-to-Late 20th Century

The mid-to-late marked a transformative era in , characterized by the development and mass deployment of antibiotics and vaccines that enabled cures on a global scale, dramatically reducing mortality from infectious diseases. The advent of penicillin, discovered by in 1928 but not widely utilized until the , revolutionized treatment for bacterial infections such as , , and wound infections. During , U.S. and Allied efforts led to industrial-scale production, with output increasing from mere milligrams to billions of units annually by 1944, allowing penicillin to save countless lives among soldiers and civilians alike. By the late , its availability had expanded commercially, transforming previously fatal bacterial illnesses into curable conditions and laying the foundation for the antibiotic era. Vaccines emerged as another pillar of mass-scale cures, with Jonas Salk's inactivated , licensed in 1955 after extensive field trials, proving pivotal in combating paralytic . Administered via injection, the demonstrated high efficacy in preventing infection, contributing to a more than 99% reduction in cases worldwide from the 1950s onward through widespread campaigns. This success exemplified how vaccination could achieve near-elimination of a crippling disease, shifting from an threat to a rarity in vaccinated populations. Similarly, the , first licensed in 1981 as a plasma-derived , targeted prevention of , including and , by inducing immunity against the . By the 1980s, technology enabled safer production, leading to routine childhood that averted millions of chronic infections globally. A crowning achievement of this period was the eradication of , declared by the in 1980 after a coordinated global campaign launched in 1967. Employing a strategy of ring —targeting contacts of cases rather than mass —the effort eliminated the variola from reservoirs, with the last natural case reported in 1977. This success, the first for any infectious disease, prevented an estimated 2-3 million deaths annually and demonstrated the feasibility of total through international collaboration and deployment. Retrospectively, such achieved cure fractions approaching 100% in preventing disease transmission in susceptible populations.

21st Century

The has marked significant progress in achieving cures for previously intractable viral and chronic diseases, driven by advances in antiviral therapies, transplantation, and editing technologies. These developments have shifted the from lifelong management to potential eradication of pathogens or correction of genetic defects, particularly post-2010, with high success rates in targeted populations. While challenges remain in and , these breakthroughs have demonstrated curative potential for conditions like hepatitis C and certain genetic disorders. In the , the introduction of direct-acting antivirals revolutionized hepatitis C treatment, with sofosbuvir-based regimens achieving sustained virologic response () rates exceeding 95% across most s, effectively curing chronic infection in the majority of patients. For instance, combined with velpatasvir in a single-tablet regimen yielded SVR12 rates over 97% in clinical trials for genotypes 1-6, excluding genotype 3 where rates were around 94.7%, allowing for shorter 12-week courses without . These oral therapies have led to over 90% cure rates in real-world settings, transforming hepatitis C from a lifelong condition to a curable one for millions globally. Functional cures for HIV have been achieved through in select cases, highlighting the role of donor cells with the CCR5Δ32 mutation that confers resistance to the virus. The , , received such a transplant in 2008 for and remained in remission for over 12 years until his from unrelated causes in 2020, marking the first documented HIV cure. Similarly, the London Patient, treated in 2016 and reported in 2019, has achieved sustained HIV-1 remission for over 9 years as of 2025 without antiretroviral therapy, with no detectable replication-competent virus in blood, , or tissues. These rare successes, now involving at least 10 cases by late 2025, underscore the curative potential of this approach but its limitations due to high risks and donor scarcity. Gene therapies using CRISPR-Cas9 have provided curative options for genetic disorders, exemplified by the 2023 FDA approval of Casgevy (exagamglogene autotemcel) for in patients 12 years and older with recurrent vaso-occlusive crises. This edits the patient's hematopoietic stem cells to boost production, addressing the root genetic and leading to sustained symptom resolution in clinical trials, where 94% of patients were free from severe crises for at least 12 months post-infusion. Casgevy represents the first CRISPR-based approved for a genetic disease, extending curative potential to conditions like beta-thalassemia as well. Insights from the have advanced preventive strategies, with mRNA vaccines like BNT162b2 (Pfizer-BioNTech) and mRNA-1273 () demonstrating over 90% efficacy in preventing severe disease, hospitalization, and death, though they do not cure active infections. Real-world data confirmed these vaccines reduced severe outcomes by 88-96% against variants like and , averting millions of deaths globally without addressing established infections directly.

Modern Challenges

Incurable Diseases

Incurable diseases are for which no exists to achieve a complete and permanent elimination of the underlying , distinguishing them from diseases that can enter remission or be managed toward . These conditions often involve irreversible to critical biological systems, such as neuronal in neurodegenerative disorders or autoimmune destruction of cells. Despite advances in , they remain resistant to curative interventions, shifting focus to symptom control and quality-of-life preservation. Alzheimer's disease exemplifies an incurable neurodegenerative condition, characterized by progressive cognitive decline due to the accumulation of and tangles that destroy brain cells. Type 1 diabetes is an autoimmune disorder where the permanently destroys insulin-producing beta cells in the , rendering natural insulin regulation impossible without external support. Pancreatic cancer, particularly , is largely incurable because it typically metastasizes rapidly, with tumors often ensconced in dense stromal tissue that hinders drug penetration and surgical resection. The incurability of these diseases stems from multiple barriers, including genetic complexity where hundreds of variants interact unpredictably, multifactorial etiologies combining genetic predispositions with environmental triggers, and late-stage diagnosis that allows irreversible progression before intervention. In Alzheimer's, the destruction of intricate neuronal circuits occurs gradually, complicating reversal once symptoms manifest. For , the autoimmune attack is self-perpetuating without a means to reliably regenerate beta cells or halt immunity long-term. Pancreatic cancer's and desmoplastic further evade targeted therapies, often evading detection until advanced stages. Management strategies for incurable diseases emphasize lifelong therapies to mitigate symptoms and extend survival, rather than eradication of the root cause. Patients with rely on exogenous insulin administration via injections or pumps to regulate blood glucose, preventing acute complications like while enabling near-normal with vigilant monitoring. Alzheimer's management includes cholinesterase inhibitors like donepezil to temporarily alleviate cognitive symptoms, alongside nonpharmacological approaches such as cognitive training and social engagement to slow functional decline. For , when cure is unattainable, chemotherapy regimens like combined with aim to shrink tumors and alleviate pain, potentially extending median survival from months to over a year in advanced cases. In scenarios where full cure eludes treatment, achieving partial remission—temporary symptom relief without disease eradication—serves as a realistic goal to enhance . Ethical considerations in addressing incurable diseases revolve around balancing hope with realism, particularly in allocating funding amid limited resources. Overemphasizing speculative cures can foster false hope, potentially diverting funds from proven management strategies and burdening patients with unfulfilled expectations, as seen in prolonged trials for Alzheimer's therapies that yield marginal benefits. Conversely, realistic communication about incurability encourages informed , yet funding bodies must prioritize high-impact on multifactorial without abandoning ethical imperatives to support palliative innovations. This tension underscores the need for transparent , ensuring resources address both immediate needs and long-term scientific inquiry without exploiting vulnerability.

Emerging Therapies

Immunotherapies represent a frontier in achieving cures for hematologic malignancies, particularly through chimeric antigen receptor () T-cell therapies targeting on B cells. In patients with refractory large B-cell lymphoma, CAR-T therapies such as have demonstrated complete response rates of 50-60%, with durable remissions in over 40% of responders at five years, indicating potential curative outcomes in a subset of cases previously unresponsive to standard treatments. Similarly, has shown complete remission rates approaching 80% in pediatric refractory B-cell acute lymphoblastic leukemia, with many patients remaining disease-free long-term. These successes stem from engineering patient T cells to recognize and eliminate cancer cells, though challenges like persist. Gene editing technologies, notably CRISPR-Cas9, have advanced toward curative interventions for genetic disorders. In 2023, the FDA approved Casgevy (exagamglogene autotemcel), a CRISPR-based that edits the BCL11A gene in hematopoietic cells to boost production, enabling transfusion independence in over 90% of patients with transfusion-dependent beta-thalassemia after one year. For , a phase I/II trial by Excision BioTherapeutics using (EBT-101) to excise proviral DNA from infected cells, completed in 2025, demonstrated safe delivery and targeted editing without off-target effects but did not prevent viral rebound during treatment interruptions, though preclinical models showed potential for viral control. These approvals and trials highlight CRISPR's precision in addressing monogenic causes of disease, though scalability and long-term durability remain under evaluation. Personalized medicine is leveraging artificial intelligence (AI) to accelerate drug discovery for rare diseases, where traditional approaches falter due to small patient populations. AI algorithms analyze genomic, proteomic, and clinical datasets to repurpose existing drugs or design novel ones tailored to individual mutations; for instance, the 2024 TxGNN model from Harvard identified repurposing candidates for more than 7,000 diseases, outperforming prior methods by up to 49% in prediction accuracy for indications. In a notable example, Insilico Medicine's AI platform generated a novel inhibitor for idiopathic pulmonary fibrosis—a rare fibrotic lung disease—in under 18 months, advancing to phase II trials by 2024. Such tools enhance precision by integrating multi-omics data, reducing development timelines from years to months while prioritizing therapies for underserved conditions. In , a November 2025 study reported a combination blood and pancreatic islet cell transplant that resets the , stopping disease progression in early-stage patients and enabling insulin independence in some, as a promising step toward functional cure. Looking ahead, emerging therapies hold promise for curing neurodegenerative diseases like Alzheimer's and Parkinson's by the 2030s through advanced gene editing and delivery systems, potentially restoring neuronal function via targeted corrections of genetic risks such as or mutations. However, ethical hurdles, including off-target editing that could induce unintended genomic alterations or oncogenic risks, necessitate rigorous preclinical validation and equitable access frameworks to mitigate disparities in therapy availability. Cure rate estimation in these trials will be crucial for assessing long-term efficacy, often defined as sustained functional recovery without relapse.