The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the appropriate agencies. Due to the length of this process, many treatments take years until they reach the market. Fortunately, OTL-200 is able to shorten at least one part of this process, as it has been granted accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). OTL-200 is used for the treatment of metachromatic leukodystrophy (MLD) and was developed by Orchard Therapeutics.
About Metachromatic Leukodystrophy (MLD)
MLD is an autosomal recessive disorder, meaning that both parents must pass down the gene in order for a child to have it. It is caused by a mutation in the ARSA gene, which is responsible for the instructions to make the enzyme arylsulfatase A, which resides within lysosomes and helps to break down sulfatides. Because these sulfatides are allowed to build up, they become toxic to the nervous system. The accumulation of sulfatides destroys white matter throughout the central and peripheral nervous system. This destruction results in the symptoms of the disorder, which are characterized by the deterioration of motor skills and intellectual functions. These symptoms include the loss of sensation in their extremities, seizures, incontinence, paralysis, blindness, and an inability to hear and speak. As the disorder progresses, people will lose sense of their surroundings and become unresponsive. MLD is present in one of every 40,000 to 160,000 people throughout the world.
It is usually isolated in specific populations, such as Arab groups in Israel, a Western portion of the Navajo nation, and Habbanites, who are a group of Jews who emigrated from southern Arabia. About 50-60% of people who have this disorder have the late infantile form, meaning that it appears in the second year of life. Those who have this form will lose any speech that they have developed, have trouble walking, and lose their muscle tone. Other forms of the disorder include the juvenile form, which occurs after age four but during adolescence, and the adult form, which sees the onset of symptoms during teenage years. There are treatments available for MLD, which include bone marrow transplants, gene therapies, and enzyme-replacement therapy.
About Orchard Therapeutics and OTL-200
OTL-200 is a stem cell based gene therapy. It is ex-vivo, autologous, and hematopoietic. It started from a collaboration between two organizations, GSK and Hospital San Raffaele, who began working on it in 2010. It was acquired by Orchard Therapeutics in 2018, which is a “fully integrated commercial-stage biopharmaceutical company.” They work with gene therapies in order to treat rare diseases. They have a history of developing ex-vivo, autologous, hematopoietic, stem cell based gene therapies, such as Strimvelis, which treats severe combined immune deficiencies. They have offices in the United Kingdom, Boston, and San Francisco.
The work of Orchard Therapeutics and other companies who focus on treatments for rare diseases point towards developments of cures for diseases that have had little to no treatment for a long time. As technology advances, hopefully more developments will come and bring new treatments.
Click here for the original article.