LEUKODYSTROPHY · MITOCHONDRIAL DISEASE · RETT SYNDROME · RARE GENETIC NEUROLOGY
A physician-led biological programme targeting the downstream neurological damage caused by inherited metabolic and mitochondrial conditions — neuroinflammation, demyelination, energy failure and progressive cellular loss. Cellular therapy does not correct the underlying genetic mutation. It addresses what that mutation does to the nervous system.
Request Medical ConsultationAbout the Condition
Genetic neurological disorders are a broad group of inherited conditions in which a single gene mutation disrupts the normal development or maintenance of the nervous system. The mutation itself is present from birth, but its consequences — progressive demyelination, mitochondrial energy failure, enzyme deficiency, toxic metabolite accumulation — unfold over months or years, destroying neural tissue that was initially intact.
These conditions include leukodystrophies (metachromatic leukodystrophy, Krabbe disease, adrenoleukodystrophy), mitochondrial disorders (MELAS, Leigh syndrome), Rett syndrome and a range of other rare inherited neurological diseases. Most are diagnosed in childhood. Many follow a trajectory of progressive neurological decline with limited pharmacological options.
Current standard of care focuses on symptom management, seizure control and supportive rehabilitation. For a small number of conditions, gene therapy or enzyme replacement therapy is available — but for the majority, no disease-modifying treatment exists. Our programme targets the biological damage downstream of the genetic defect: the neuroinflammation, demyelination and metabolic failure that drive clinical deterioration.
Leukodystrophies
A family of inherited disorders affecting the white matter of the brain and spinal cord. Metachromatic leukodystrophy (MLD), Krabbe disease (globoid cell leukodystrophy) and X-linked adrenoleukodystrophy (X-ALD) are the most common forms. The underlying defect — typically an enzyme deficiency — leads to progressive demyelination, loss of motor and cognitive function, and in many cases seizures. Onset ranges from infancy to early adulthood depending on the specific mutation.
Mitochondrial Disorders
Conditions caused by mutations affecting mitochondrial function — the energy-producing machinery within every cell. MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes) and Leigh syndrome are among the most recognised forms. Symptoms include developmental regression, seizures, muscle weakness, lactic acidosis and stroke-like episodes. The nervous system is disproportionately affected because neurons have exceptionally high energy demands.
Rett Syndrome
A neurodevelopmental disorder caused primarily by mutations in the MECP2 gene. Almost exclusively affects females. After a period of apparently normal early development, affected children lose acquired motor and language skills, develop stereotypic hand movements and often experience seizures. The condition is not neurodegenerative in the classical sense — the neurons remain present but function abnormally. This distinction is clinically relevant to our therapeutic approach.
Other Rare Genetic Neurological Conditions
A heterogeneous group including neuronal ceroid lipofuscinoses (Batten disease), giant axonal neuropathy, Pelizaeus-Merzbacher disease and other ultra-rare inherited conditions affecting the central or peripheral nervous system. Each has a distinct genetic mechanism, but the downstream pathology — chronic neuroinflammation, metabolic failure, progressive neural loss — shares common biological features that our programme is designed to address.
Our program is individually adapted for all subtypes and all stages of progression.
Important: Each patient is accepted into the programme only after a comprehensive individual medical assessment. For paediatric patients, this includes detailed review of genetic diagnosis, current developmental status, seizure history, neuroimaging and metabolic profile.
We do not claim to correct the underlying genetic mutation. Our programme targets the downstream biological mechanisms — neuroinflammation, demyelination, mitochondrial dysfunction and metabolic waste accumulation — that drive progressive neurological damage. The clinical objective is to slow decline, stabilise function and, where biologically possible, support partial recovery of compromised neural pathways.
Clinical Outcomes
The following data are derived from structured observational analysis of patients with genetic neurological disorders treated at BioCells Medical between 2016 and 2025. Given the rarity and heterogeneity of these conditions, patient numbers are smaller than for more common diagnoses. All figures represent aggregated clinical registry outcomes with longitudinal follow-up. These are observational results — not randomised controlled trial data — and do not constitute a guarantee of therapeutic effect.
18
patients with genetic neurological disorders treated
66%
demonstrated measurable stabilisation on condition-specific validated scales within 3–6 months
44%
showed improvement in at least one functional domain (motor, cognitive or seizure frequency)
72%
parents and caregivers reported improved alertness, engagement or responsiveness
39%
demonstrated reduction in seizure frequency or severity during follow-up period
Improved visual tracking and environmental awareness
61%
Increased head control and postural stability (paediatric GMFCS assessment)
44%
Reduced frequency or intensity of seizure episodes
39%
Improved feeding tolerance and oral motor coordination
50%
Enhanced interactive behaviour and caregiver-reported alertness (Bayley-III subscales)
56%
3–8 weeks
Initial functional response
3–6 months
Clinically meaningful change
1–2 years under continued monitoring
Stabilisation phase
Important: Outcomes depend on the specific genetic diagnosis, age at treatment, disease stage, baseline neurological function and individual biological response. Genetic neurological disorders are inherently heterogeneous — results vary significantly between conditions and between patients with the same condition. These figures should be interpreted with caution given the small cohort size.
Find out if our program can help in your specific case. The initial medical consultation is free and carries no obligation.
Request ConsultationPatient Stories
“Our daughter has metachromatic leukodystrophy. By the time we found BioCells she had lost the ability to sit unsupported and her speech was almost gone. Four months after treatment, the decline stopped. She has not regained what was lost, but she has not lost anything further in over a year. Her neurologist confirmed it on MRI: the demyelination has not progressed. For a condition where everything moves in one direction, standing still is significant.”
Patient's mother
Metachromatic Leukodystrophy · Germany
Every case is assessed individually by our physician team. Request a consultation to discuss your specific situation with our physician team.
Request ConsultationThe BioCells Program
Our programme for genetic neurological disorders combines five biological components into a single personalised protocol. The programme does not target the genetic mutation itself — it targets the downstream damage that the mutation causes to the nervous system. No two protocols are identical — each is constructed following a detailed medical evaluation of the patient's genetic diagnosis, disease stage, metabolic profile and clinical priorities.
No surgery required
Treatment is delivered by intravenous infusion or targeted local injection — not surgical instruments. Particularly important for paediatric patients where minimising invasive procedures is a priority.
No general anaesthesia
Avoids the neurological and metabolic risks of general anaesthesia, which can be significant in patients with mitochondrial disorders or compromised respiratory function.
No risk of immune rejection — autologous option
Where clinically appropriate, we use the patient's own cells. For young paediatric patients where autologous collection is not feasible, carefully matched allogeneic sourcing is used with comprehensive safety protocols.
Targets downstream damage, not the gene itself
Our protocol does not attempt to correct the genetic mutation. It addresses the neuroinflammation, demyelination, mitochondrial dysfunction and metabolic waste that the mutation causes — the biological processes that drive progressive clinical deterioration.
Complements existing medical management
Our programme is compatible with antiepileptic medication, enzyme replacement therapy and other current treatments. Patients do not need to discontinue existing therapy before commencing our protocol.
Treatment at your location worldwide
Our medical team is available to conduct treatment at our Warsaw clinic or to travel to the patient's location anywhere in the world. For families with severely affected children where travel is difficult, this removes a major barrier to accessing care.
What It Is
MSCs are multipotent regenerative cells with well-documented immunomodulatory and neuroprotective properties. In the context of genetic neurological disorders, their primary therapeutic role is suppression of neuroinflammation and metabolic support of compromised neural tissue — not correction of the underlying genetic defect.
How It Is Done
Cells are collected from the patient's own bone marrow (autologous) or sourced from a certified donor (allogeneic), depending on the patient's age, condition and clinical indications. For young paediatric patients, allogeneic sourcing is often preferred. All cells are expanded, quality-controlled and tested in our certified Warsaw laboratory before administration.
Biological Mechanisms
How This Helps in What Are Genetic Neurological Disorders
In genetic neurological disorders, the primary genetic defect triggers a cascade of secondary damage — chronic neuroinflammation, oxidative stress and progressive loss of healthy neural tissue that was not directly affected by the original mutation. MSCs address this secondary cascade, creating a more stable neurological environment and reducing the rate at which collateral damage accumulates.
Your Medical Board
The exact combination, dosage, sequencing and delivery method of all five components is determined individually by our medical board for each patient. Protocols for paediatric patients are designed with particular attention to age-appropriate dosing, minimally invasive delivery and the specific metabolic profile of each genetic condition. No two treatment protocols are identical.
Your protocol is designed individually. Speak with our medical team to understand what your personalised program would include.
Request ConsultationPatient Journey
Your case is reviewed remotely by our physician team. We assess the genetic diagnosis, current neurological status, medical history and treatment goals. For paediatric patients, we work directly with parents and the child's existing medical team. This consultation is free and carries no obligation.
A detailed review of all medical documentation including genetic testing, neuroimaging, metabolic panels and developmental assessments. Our medical board evaluates eligibility, confirms safety parameters and designs the personalised therapeutic protocol.
Cells are collected (where autologous sourcing is appropriate), isolated, expanded and quality-tested in our certified Warsaw laboratory. For paediatric patients where allogeneic sourcing is indicated, donor cells are prepared and verified. Each batch receives a full traceability certificate. This stage typically takes 2–3 weeks.
Cells are delivered by intravenous infusion or targeted local administration — no surgery, no general anaesthesia. Treatment is available at our Warsaw clinic or with our medical team at your location worldwide. Airport transfers, accommodation and visa support are included in the programme. Where clinically appropriate, our medical board may approve a travelling treatment programme — our medical team flies directly to the patient.
Structured rehabilitation sessions with our specialist, adapted to the patient's age, current functional level and specific condition. For paediatric patients, rehabilitation protocols integrate with developmental therapy and are coordinated with the child's existing therapy team. Available at our clinic or remotely coordinated with your local medical team.
Your dedicated coordinator monitors neurological status, developmental progress and overall well-being. Clinical guidance is adjusted based on follow-up data. For paediatric patients, regular developmental reassessment is part of the monitoring protocol. A medical-grade wearable bracelet supports continuous health tracking regardless of your location.
The first step is free. Request a medical consultation and our medical consultant will contact you within 24 hours.
Request ConsultationSafety Profile
Cellular therapy is considered safe when delivered under proper medical supervision and according to validated protocols. In our practice, the procedure is well-tolerated by the majority of patients, including paediatric patients with complex genetic conditions.
Temporary mild reactions — such as transient local discomfort at the infusion site, slight fatigue or low-grade temperature — may occur in a minority of patients. These are typically short-lived and indicate active immune engagement.
A final medical assessment is performed on-site before every treatment session. If a patient's status has changed — including metabolic destabilisation, seizure escalation or intercurrent illness — the programme may be temporarily modified or postponed for safety reasons.
All contraindications are evaluated individually. A contraindication in one clinical context does not necessarily preclude treatment in a different context — this is always determined by physician assessment. For paediatric patients, additional safety monitoring is standard throughout the treatment period.
Standard Contraindications
Active acute infection or fever
Active malignancy or ongoing chemotherapy / radiotherapy
Severe decompensated cardiac or renal failure
Acute metabolic crisis (treatment is deferred until stabilisation)
Pregnancy
Post-Treatment
Dedicated rehabilitation specialist
monitors neurological function, developmental progress and overall well-being
Personalised rehabilitation programme
adapted to the patient's age, condition and current functional capacity
Medical-grade wearable monitoring
continuous physiological data collection supporting clinical decision-making
Long-term coordinator support
proactive check-ins, clinical guidance and response to any changes in neurological status
Continued clinical access
our medical team remains available for ongoing reassessment and protocol adjustment
In genetic neurological conditions, biological response develops gradually and requires sustained clinical monitoring. Families should expect a structured follow-up period during which our medical team tracks neurological status, adjusts rehabilitation recommendations and evaluates whether additional treatment cycles are clinically indicated. The post-treatment period is integral to the programme — not an afterthought.
Get Started
If your child or family member has been diagnosed with a genetic neurological condition, our medical team is available for a free, no-obligation medical consultation — based on the specific diagnosis, current neurological status and individual clinical profile.
We review every inquiry personally. You will speak with a physician, not an administrator.
Submit your case online or by phone
Our medical consultant contacts you to review your documents
The medical board presents your personalised treatment plan
Request a Consultation
Tell us about your condition. Our medical consultant will contact you within 24 hours to review your documents.
Open Consultation FormMultilingual coordination — English, Italian, French, Russian, Polish
Evidence Base
Our clinical approach is informed by and consistent with published research in the field of regenerative medicine.
