Stem Cell Blog
Употребата на матичните клетки од папочна врвца рапидно се зголемува. Пред 10 години крвта од папочна врвца можеше да лекува околу 40 состојби, но денес таа бројка е над 80. Со нетрпение очекуваме нови терапии за болести и нарушувања како што се дијабет, аутизам и мозочен удар, можете да бидете во тек со најновите случувања во регенеративната медицина на нашиот блог за матични клетки.
A new therapy, recently granted a UK Innovation Passport Designation, could help to improve treatment outcomes for Krabbe disease patients post-stem cell transplant.
What is Krabbe disease?
Krabbe disease is a rare genetic disorder affecting around 1 in every 100,000 births and is caused by a deficiency of the enzyme galactocerebrosidase (GALC), leading to the breakdown of the myelin in the nervous system.
The most common variant of Krabbe disease, Infant Krabbe Disease, affects children under the age of 1, and can cause muscle stiffness, seizures, and developmental delays. It is often fatal if untreated.
Approximately 85% of Krabbe disease cases are the infantile subtypes. [1]
What are the current treatment options for Krabbe disease?
Currently, the only effective treatment option for Krabbe disease is a transplant of haematopoietic stem cells (HSCs), which are found in bone marrow, peripheral blood, and umbilical cord blood. [2]
By transplanting HSCs from a donor to a Krabbe disease patient, the patient’s unhealthy cells lacking in the GALC enzyme are eventually replaced by healthy red blood cells, white blood cells and platelets derived from HSCs.
These healthy blood cells can then work to populate the brain with GALC enzyme activity, reducing the breakdown of myelin and thereby stabilise cognitive function. [3]
Stem cell transplants using haematopoietic stem cells from cord blood, specifically, have been shown to be highly effective in improving neurological outcomes if the transplant is performed before the development of symptoms. [4]
While HSCT remains the only viable treatment option for Krabbe disease, it is not a cure.
It also does not combat the peripheral neuropathy occasioned by Krabbe disease, a condition affecting the nerves beyond the brain and spinal cord, leading to decline in motor function. [5]
What is the new gene therapy and how does it improve stem cell transplant outcomes?
Developed by Forge Biologics, the FBX-101 therapy works by delivering a copy of the GALC gene to cells in the nervous system, which improves myelination (the process by which the myelin sheath forms) and, crucially, motor function.
It is designed to be administered intravenously after the current standard of care, a haematopoietic stem cell transplant.
In its early phase trial, REKLAIM, FBX-101 was shown to improve motor function in all five of the patients who underwent treatment.
Building on these promising results, the Innovation Passport designation means that FBX-101 will be able to enter the Innovative Licensing and Access Pathway (ILAP), which accelerates both market and regulatory access in the UK. [6]
Why is newborn screening for Krabbe disease so important?
Newborn screening for Krabbe disease is crucial because the condition progresses rapidly, especially in its infantile form, and early intervention is the key to preventing severe neurological damage.
Ideally, the patient would receive a transplant within 30 days in order to have the best chance of improved neurological and transplant outcomes. [7]
Symptoms of Krabbe disease often appear within the first few months of life, and once they start, the deterioration of the nervous system is fast and irreversible.
By the time symptoms are noticeable, significant damage has already occurred, limiting the effectiveness of available treatments.
Newborn screening allows for early diagnosis before symptoms develop, enabling early intervention through HSCT, which is at its most effective if administered before significant damage to the nervous system. [8]
Could cord blood banking help?
Umbilical cord blood is a vital source of haematopoietic stem cells which can differentiate into various kinds of blood cells. These cells are crucial in the treatment of Krabbe disease, but rely on finding a donor match in order for a transplant to be successful.
If Krabbe disease runs in your family, saving cord blood for every child is probably a worthwhile investment.
If your baby does end up developing Krabbe disease, having their sibling’s stem cells in storage could make the difference in being able to access a life-saving transplant as a sibling has a 75% chance of being a partial donor match.
For more information about how cord blood banking could help with the treatment of Krabbe disease, visit our Krabbe Disease and Stem Cells page, here.
If either you or a family member or someone you know is expecting, why not download our free Welcome Pack to learn more about the benefits of cord blood banking. Simply fill out the form below.
References
[1] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477
[2] Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar; Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood 2021; 137 (13): 1719–1730. doi: https://doi.org/10.1182/blood.2020005477
[3] (2022, August 13). Krabbe Disease (Globoid Cell Leukodystrophy). Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/6039-krabbe-disease-globoid-cell-leukodystrophy
[4] Wright, M. D., Poe, M. D., DeRenzo, A., Haldal, S., & Escolar, M. L. (2017). Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology, 89(13), 1365–1372. https://doi.org/10.1212/WNL.0000000000004418
[5] Beltran-Quintero, M.L., Bascou, N.A., Poe, M.D. et al. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis 14, 46 (2019). https://doi.org/10.1186/s13023-019-1018-4
[6] (2024, March 19). Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation. Forge Biologics. https://www.forgebiologics.com/forge-biologics-novel-aav-gene-therapy-fbx-101-for-patients-with-krabbe-disease-is-granted-uks-innovation-passport-designation/
[7] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094
[8] Page, K. M., Ream, M. A., Rangarajan, H. G., Galindo, R., Mian, A. Y., Ho, M. L., Provenzale, J., Gustafson, K. E., Rubin, J., Shenoy, S., & Kurtzberg, J. (2022). Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease. Blood advances, 6(9), 2947–2956. https://doi.org/10.1182/bloodadvances.2021006094
There’s exciting news from the University of Galway, where researchers are developing a new technique that they hope will improve the viability of stem cell treatments for Parkinson’s disease.
The pioneering method, which involves the use of a substance called hydrogel, could revolutionise the way stem cell treatments for Parkinson’s disease are carried out, making them more effective and less prone to failure.
Parkinson’s disease is a neurological condition affecting around 150,000 people in the UK. [1]
The condition, which is caused by a lack of dopamine in the brain, manifests in severe ways with symptoms including tremors, muscle rigidity and slowness of movement. [2]
Unfortunately, Parkinson’s is degenerative, meaning that it gets worse over time. It can make people who live with the condition more vulnerable to poor health and disability, which can end up having fatal repercussions in some cases.
The degeneration of nerve cells in the brain is what results in the underproduction of dopamine. The aim of recent stem cell research has been to find a way of repairing and replacing these cells through the use of induced stem cells.
These induced stem cells are harvested from different areas of the body, such as skin, and then reprogrammed to become the type of cells necessary for brain repair.
However, these cells require transplantation at a very early stage of their development into brain cells and once transplanted, many of them do not end up converting.
What researchers at the University of Galway have discovered is that by transplanting these induced stem cells in a collagen hydrogel, effectively a water-based scaffold, significantly improves the chances of the stem cells both surviving and then differentiating into the cells necessary for therapy. [3]
With funding from the Michael J. Fox Foundation for Parkinson’s Research (MJFF), the study’s findings were published in the Journal of Neural Engineering and have been met with widespread acclaim.
The research is ongoing, but the team at the University of Galway and MJFF hope that this new transplantation technique will significantly improve outcomes for sufferers of Parkinson’s disease.
If you want to learn more about how you could give your baby access to future stem cell therapies, download our FREE Welcome Pack below.
References
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