{"id":13522,"date":"2018-10-23T14:52:54","date_gmt":"2018-10-23T14:52:54","guid":{"rendered":"https:\/\/cells4life.com\/?p=13522"},"modified":"2023-11-15T17:32:04","modified_gmt":"2023-11-15T17:32:04","slug":"how-cord-blood-transfusions-can-treat-krabbe-disease","status":"publish","type":"post","link":"https:\/\/cells4life.com\/us\/2018\/10\/how-cord-blood-transfusions-can-treat-krabbe-disease\/","title":{"rendered":"How Cord Blood Transfusions Could Treat Krabbe Disease"},"content":{"rendered":"[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; hover_enabled=&#8221;0&#8243; global_colors_info=&#8221;{}&#8221; sticky_enabled=&#8221;0&#8243;]A new study published by the University of Pittsburgh School of Medicine suggests <a href=\"https:\/\/cells4life.com\/cord-blood-banking-overview\/\" target=\"_blank\" rel=\"noopener\">cord blood<\/a> could extend the lives of children with <a href=\"https:\/\/cells4life.com\/krabbe-disease-and-stem-cells\/\">Krabbe disease<\/a>.<\/p>\n<p>Research, led by Dr Maria Escolar, showed that cord blood transfusions not only improved life expectancy for patients, but also increased quality of life.<\/p>\n<h3><strong>What is Krabbe disease?<\/strong><\/h3>\n<p>Krabbe disease is a rare and deadly disorder of the nervous system. Patients with the condition have a defective gene in Chromosome 14, which results in a lack of protein called galactosylceramidase (GALC).<\/p>\n<p>Normally, GALC helps to clear unneeded molecules out of cells and creates myelin. Myelin is used to coat and protect nerve fibres and without it, nerves and cells in the brain die \u2013 causing other parts of the body to cease working.<\/p>\n<p>It is a genetic disorder inherited from parents and unfortunately, there is no cure.<\/p>\n<p><strong>There are two types of Krabbe disease:<\/strong><br \/>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221; column_structure=&#8221;1_2,1_2&#8243;][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_color=&#8221;#009fc4&#8243; background_layout=&#8221;dark&#8221; custom_padding=&#8221;15px|15px|0px|15px&#8221; global_colors_info=&#8221;{}&#8221;]\n<h4><strong>Early onset Krabbe Disease <\/strong><\/h4>\n<p>This form of the condition is diagnosed within the first six months of the baby\u2019s life.<\/p>\n<p><strong>Symptoms include:<\/strong><\/p>\n<ul>\n<li>Feeding problems<\/li>\n<li>Loss of head control<\/li>\n<li>Fevers<\/li>\n<li>Irritability and excessive crying<\/li>\n<li>Persistent vomiting<\/li>\n<li>Poor coordination<\/li>\n<li>Stiffness<\/li>\n<li>Seizures<\/li>\n<li>Muscle spasms<\/li>\n<li>Muscle tone changes<\/li>\n<li>Deterioration of mental\/motor function<\/li>\n<li>Deafness\/blindness<\/li>\n<\/ul>\n[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_color=&#8221;#bb1350&#8243; background_layout=&#8221;dark&#8221; custom_padding=&#8221;15px|15px|0px|15px&#8221; global_colors_info=&#8221;{}&#8221;]\n<h4><strong>Late onset Krabbe Disease <\/strong><\/h4>\n<p>This form of the condition is diagnosed when the child is between six months and three years old.<\/p>\n<p><strong>Symptoms include:<\/strong><\/p>\n<ul>\n<li>Progressive loss of vision, leading to blindness<\/li>\n<li>Difficulty walking (ataxia)<\/li>\n<li>Poor hand coordination<\/li>\n<li>Muscle weakness\/rigid muscles<\/li>\n<li>As well as many of the symptoms of early onset Krabbe Disease<\/li>\n<\/ul>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;]\n<h3><strong>What treatment options are available?<\/strong><\/h3>\n<p>Treatment of Krabbe disease involves transplanting cells into the patient in order to compensate for the lack of GALC protein. Usually, this is done in one of two ways:<br \/>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221; column_structure=&#8221;1_2,1_2&#8243;][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_color=&#8221;#009fc4&#8243; background_layout=&#8221;dark&#8221; custom_padding=&#8221;15px|15px|15px|15px&#8221; global_colors_info=&#8221;{}&#8221;]\n<h4><strong>Bone marrow transplantation<\/strong><\/h4>\n<p>Bone marrow transplantation replaces the patient\u2019s bone marrow with a donor, in order to introduce healthy cells into the body which can cure the deficiencies which cause the disease.<\/p>\n<p>These bone marrow cells are <a href=\"https:\/\/cells4life.com\/cord-blood-banking-overview\/about-umbilical-cord-blood\/\" target=\"_blank\" rel=\"noopener\">haematopoietic<\/a>, which means they are blood forming. It is possible to reconstitute an entire blood system using bone marrow cells.<\/p>\n<p>In the past, the best results have been seen in patients with late-onset Krabbe disease, before their more severe symptoms develop.<br \/>\n[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_color=&#8221;#bb1350&#8243; background_layout=&#8221;dark&#8221; custom_padding=&#8221;15px|15px|15px|15px&#8221; global_colors_info=&#8221;{}&#8221;]\n<h4><strong>Cord blood transfusion<\/strong><\/h4>\n<p>Cord blood transfusions use blood collected from the umbilical cord at the time of birth to introduce healthy cells into the patient\u2019s blood system.<\/p>\n<p>Unlike bone marrow transplantation, cord blood transfusions are more successful when they are delivered before symptoms appear.<br \/>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221; column_structure=&#8221;1_3,2_3&#8243;][et_pb_column type=&#8221;1_3&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_color=&#8221;#009fc4&#8243; background_layout=&#8221;dark&#8221; custom_padding=&#8221;15px|15px|15px|15px&#8221; global_colors_info=&#8221;{}&#8221;]\n<h5>\u201cOur study provides evidence that the youngest babies who are at risk of developing the most severe form of Krabbe disease should be strongly considered for HSCT as soon as possible. HSCT, like all surgical procedures, does carry some risks so parents may be inclined to wait until Krabbe symptoms develop, but our findings suggest that may be too late for the best outcomes.\u201d<\/h5>\n<p><strong>Dr Maria Escolar\u00a0<\/strong><br \/>\n[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;2_3&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;]\n<h3><strong>What did the trial involve?<\/strong><\/h3>\n<p>Dr Escolar\u2019s study treated 18 children with cord blood transfusions within 7 weeks of their birth.<\/p>\n<p>Children were chosen based on a high risk of developing early-onset Krabbe disease, based on family history and genetic screening.<\/p>\n<p>After transfusion, researchers followed the children\u2019s progress for up to 15 years. This involved comprehensive annual evaluations of cognitive, speech and motor function, tests to determine the speed and strength of electrical signals from peripheral nerves, as well as brain imaging.<\/p>\n<p>Intervention is most effective when done before symptoms appear and may help children live at least til their teens<\/p>\n<h3><strong>What were the results?<\/strong><\/h3>\n<p>In 2005, Dr Escolar\u2019s research showed that haematopoietic stem cell transplants had at least three years of benefits.<\/p>\n<p>This most recent study showed that early treatment of Krabbe disease with cord blood could improve quality of life and lead to longer lifespans.<\/p>\n<p>The majority of the 18 children saw continued improvement in their motor, cognitive and language skills \u2013 even though these would usually degrade as the disease progresses.<\/p>\n<p>10 of 18 children can now walk &#8211; two of whom can walk independently.<br \/>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_image src=&#8221;https:\/\/cells4life.com\/us\/wp-content\/uploads\/2018\/10\/Krabbe_disease_infographic.jpg&#8221; align_tablet=&#8221;center&#8221; align_last_edited=&#8221;on|desktop&#8221; _builder_version=&#8221;4.16&#8243; always_center_on_mobile=&#8221;on&#8221; global_colors_info=&#8221;{}&#8221; align_phone=&#8221;center&#8221;][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;4.16&#8243; background_color=&#8221;#bd144f&#8221; custom_padding=&#8221;0px|||&#8221; global_colors_info=&#8221;{}&#8221; theme_builder_area=&#8221;post_content&#8221;][et_pb_row padding_mobile=&#8221;on&#8221; column_padding_mobile=&#8221;on&#8221; _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; custom_padding=&#8221;0px|20px&#8221; global_colors_info=&#8221;{}&#8221; theme_builder_area=&#8221;post_content&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221; theme_builder_area=&#8221;post_content&#8221;][et_pb_image src=&#8221;https:\/\/cells4life.com\/us\/wp-content\/uploads\/2015\/09\/section-divider11.png&#8221; align=&#8221;center&#8221; align_tablet=&#8221;center&#8221; align_last_edited=&#8221;on|desktop&#8221; _builder_version=&#8221;4.16&#8243; animation_style=&#8221;slide&#8221; animation_direction=&#8221;top&#8221; animation_duration=&#8221;500ms&#8221; animation_intensity_slide=&#8221;10%&#8221; use_border_color=&#8221;off&#8221; border_color=&#8221;#ffffff&#8221; border_style=&#8221;solid&#8221; animation=&#8221;top&#8221; sticky=&#8221;off&#8221; global_colors_info=&#8221;{}&#8221; theme_builder_area=&#8221;post_content&#8221; align_phone=&#8221;center&#8221;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221; theme_builder_area=&#8221;post_content&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221; theme_builder_area=&#8221;post_content&#8221;][et_pb_text admin_label=&#8221;RWP&#8221; _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; background_layout=&#8221;dark&#8221; use_border_color=&#8221;off&#8221; global_colors_info=&#8221;{}&#8221; theme_builder_area=&#8221;post_content&#8221;]\n<h3>FIND OUT MORE, REQUEST YOUR WELCOME PACK TODAY<\/h3>\n<p>All you need to know to make an informed decision.<\/p>\n<p>Provide your contact details to request:<\/p>\n<p>&#8211; Complete Welcome Pack and Parent\u2019s Guide<br \/>\n&#8211; Information via email<br \/>\n&#8211; Contact from our specialist advisors<\/p>\n<p>&nbsp;<\/p>\n\n<div class=\"wpcf7 no-js\" id=\"wpcf7-f6967-o1\" lang=\"en-US\" dir=\"ltr\" data-wpcf7-id=\"6967\">\n<div class=\"screen-reader-response\"><p role=\"status\" aria-live=\"polite\" aria-atomic=\"true\"><\/p> <ul><\/ul><\/div>\n<form action=\"\/us\/wp-json\/wp\/v2\/posts\/13522#wpcf7-f6967-o1\" method=\"post\" class=\"wpcf7-form init\" aria-label=\"Contact form\" novalidate=\"novalidate\" data-status=\"init\">\n<fieldset class=\"hidden-fields-container\"><input type=\"hidden\" name=\"_wpcf7\" value=\"6967\" \/><input type=\"hidden\" name=\"_wpcf7_version\" value=\"6.1.5\" \/><input type=\"hidden\" name=\"_wpcf7_locale\" value=\"en_US\" \/><input type=\"hidden\" name=\"_wpcf7_unit_tag\" value=\"wpcf7-f6967-o1\" \/><input type=\"hidden\" name=\"_wpcf7_container_post\" value=\"0\" \/><input type=\"hidden\" name=\"_wpcf7_posted_data_hash\" value=\"\" \/>\n<\/fieldset>\n<p>Title* <span class=\"wpcf7-form-control-wrap\" data-name=\"salutation\"><select class=\"wpcf7-form-control wpcf7-select wpcf7-validates-as-required\" aria-required=\"true\" aria-invalid=\"false\" name=\"salutation\"><option value=\"- Select -\">- Select -<\/option><option value=\"Mr\">Mr<\/option><option value=\"Mrs\">Mrs<\/option><option value=\"Miss\">Miss<\/option><option value=\"Ms\">Ms<\/option><option value=\"Dr\">Dr<\/option><option value=\"Prof\">Prof<\/option><option value=\"Sir\">Sir<\/option><option value=\"Madam\">Madam<\/option><option value=\"Other\">Other<\/option><\/select><\/span>\n<\/p>\n<p><span class=\"wpcf7-form-control-wrap\" data-name=\"first_name\"><input size=\"40\" maxlength=\"400\" class=\"wpcf7-form-control wpcf7-text wpcf7-validates-as-required c4l-capitalize\" aria-required=\"true\" aria-invalid=\"false\" placeholder=\"First Name\" value=\"\" type=\"text\" name=\"first_name\" \/><\/span>\n<\/p>\n<p><span class=\"wpcf7-form-control-wrap\" data-name=\"last_name\"><input size=\"40\" maxlength=\"400\" class=\"wpcf7-form-control wpcf7-text wpcf7-validates-as-required c4l-capitalize\" aria-required=\"true\" aria-invalid=\"false\" placeholder=\"Surname\" value=\"\" type=\"text\" name=\"last_name\" \/><\/span>\n<\/p>\n<p><span class=\"wpcf7-form-control-wrap\" data-name=\"email1\"><input size=\"40\" maxlength=\"400\" class=\"wpcf7-form-control wpcf7-email wpcf7-validates-as-required wpcf7-text wpcf7-validates-as-email email1_3838\" aria-required=\"true\" aria-invalid=\"false\" placeholder=\"Email Address\" 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size=\"40\" maxlength=\"400\" class=\"wpcf7-form-control wpcf7-text wpcf7-validates-as-required c4l-capitalize\" aria-required=\"true\" aria-invalid=\"false\" placeholder=\"Hospital\" value=\"\" type=\"text\" name=\"webform_hospital_c\" \/><\/span>\n<\/p>\n<p>By post?<span class=\"wpcf7-form-control-wrap\" data-name=\"welcome_pack_by_post_c\"><span class=\"wpcf7-form-control wpcf7-checkbox\" id=\"cf7tosugar_by_post\"><span class=\"wpcf7-list-item first last\"><input type=\"checkbox\" name=\"welcome_pack_by_post_c[]\" value=\"Yes\" \/><span class=\"wpcf7-list-item-label\">Yes<\/span><\/span><\/span><\/span>\n<\/p>\n<p class=\"cf7tosugar_address\"><span class=\"wpcf7-form-control-wrap\" data-name=\"primary_address_street\"><input size=\"40\" maxlength=\"400\" class=\"wpcf7-form-control wpcf7-text c4l-capitalize\" aria-invalid=\"false\" placeholder=\"Address 1\" value=\"\" type=\"text\" name=\"primary_address_street\" \/><\/span>\n<\/p>\n<p class=\"cf7tosugar_address\"><span 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value='{\"closepopup\":false,\"closedelay\":0,\"openpopup\":false,\"openpopup_id\":0}' \/><div class=\"wpcf7-response-output\" aria-hidden=\"true\"><\/div>\n<\/form>\n<\/div>\n[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]\n","protected":false},"excerpt":{"rendered":"<p>A new study published by the University of Pittsburgh School of Medicine suggests cord blood could extend the lives of children with Krabbe disease. Research, led by Dr Maria Escolar, showed that cord blood transfusions not only improved life expectancy for patients, but also increased quality of life. What is Krabbe disease? 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