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They use the word "c". Shares of Mustang Bio surged more than 400% on Wednesday after normal market hours by announcing that a licensed gene therapy delivered by the St. Jude Children's Research Hospital had cured eight infants with diabetes. A severe combined immunodeficiency linked to X (X-SCID), also known as "bubble boy".
The treatment, called MB-107, uses a lentiviral vector to deliver a working copy of the IL2RG gene to babies with X-SCID. The disease is caused by mutations in the IL2RG gene, which encodes a protein that the immune system needs to function properly. Babies with X-SCID have white blood cells called lymphocytes that do not develop and function normally, which prevents them from fighting infections.
MB-107 is based on hematopoietic – hematogenous stem cells – taken from the patient's bone marrow. An artificial lentivirus is used to deliver a normal copy of IL2RG into these cells, which are then reinjected into the patient after receiving a low dose of busulfan, an anticancer drug. The role of Busulfan is to help the gene-modified stem cells to take root in the patient's bone marrow and to start making new blood cells.
The half-life study, published in the New England Journal of Medicine, showed that MB-107 restored immune function in seven out of eight infants in three to four months. The researchers found that the treatment increased the number of natural killer cells, B cells and T cells – all types of lymphocytes – in these infants to normal levels. The eighth child initially lagged behind the number of T cells, but developed them after receiving a second dose of gene-modified stem cells. The children were followed for an average of 16.4 months.
The results are based on data presented at the annual meeting of the American Society for Gene and Cell Therapy a year ago. At that time, six of the eight patients had "achieved their reconstituted immune system" within three to four months of treatment. Mustang authorized the treatment three months later.
RELATED: Mustang Bio embarks on St. Jude bubble boy gene therapy
"The results have been very good so far. We have managed to quickly restore a complete immune system, "said the study leader, Ewelina Mamcarz, M.D. in a statement. "All these patients managed to get out of their isolation and returned home with a fully functioning immune system. Patients suffering from very serious infections came to see us and they eliminated this newly developed immune system. "
"We are excited to continue working with St. Jude to evaluate MB-107 in this clinical trial. We are looking forward to transferring the IND to Mustang by the end of the year, after which the patient cells of the three participating clinical trial sites will be treated in our Worcester facility, in Massachusetts, said Mustang Medical Director Martina Sersch, MD, Ph.D., in her statement.
At the close of Thursday, Mustang shares stabilized at $ 5.64, or 112% of their price before the news the day before.
The company could seek FDA approval by the end of 2021, said Bloomberg, Manny Litchman, CEO of Mustang.
The current standard of care for babies with X-SCID is a bone marrow transplant from a sibling or genetically matched sister. But as one might imagine, not all patients have a brother or sister, let alone a compatible donor. Some patients end up having the bone marrow of a parent or other donor, but this type of treatment only partially restores immunity, the National Institutes of Health (NIH) said. One of the study sites, in a statement. Other sites were St. Jude and UCSF Benioff Hospitals.
"These patients require lifelong treatment and may continue to experience complex medical problems, including chronic infections," the agency said.
If the MB-107 ultimately turns out to be a cure, it would offer infants who tend to succumb to infections in the first or first two years of life "To quote Anthony Fauci, MD, director from the National Institute of Allergy and Infectious Diseases of NIH.
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