Chiba University Hospital announced that it has received approval from the Ministry of Health, Labor and Welfare for conducting a first-class regenerative medicine clinical study (*) targeting "familial LCAT deficiency," which is the world's first gene therapy research.In collaboration with CellgenTech, Inc., a bio-venture from Chiba University, we will proceed with clinical trials targeting patients.
LCAT is an enzyme present in plasma.Familial LCAT deficiency is a hereditary disease in which the gene that produces this LCAT becomes abnormal and the LACT protein cannot be produced. When LACT stops working, good cholesterol (HDL) in the blood decreases significantly, and excess cholesterol accumulates in the kidneys and eyes, causing disorders such as renal dysfunction, corneal opacity, and hemolytic anemia.It is a relatively rare genetic disease and no cure has been established, so it was designated as an intractable disease in July 2015.
Adipocytes have a particularly long lifespan among human cells, and are unlikely to undergo changes such as canceration.Focusing on this point, Chiba University Hospital has been conducting research on the practical application of "therapeutic gene-introduced human adipocytes" processed to secrete proteins in order to treat intractable diseases.The treatment method used in the clinical study this time is to culture fat cells collected from the adipose tissue of patients with familial LCAT deficiency in vitro, introduce the genetically deficient LCAT gene, and produce LCAT protein. After being processed into a drug, it is transplanted again to the patient to continuously replenish the normal LCAT protein into the body.
Until now, there was no radical cure for LCAT deficiency, but this time, it is said that clinical trials for the practical application of this treatment can take a big step.In the future, it is expected to be a technology that will lead to the development of treatments for many intractable diseases such as hemophilia and lysosomal storage diseases as well as familial LCAT deficiency.
* Type 26 regenerative medicine clinical research Approved by the Specified Certified Regenerative Medicine Committee and the Ministry of Health, Labor and Welfare as "Type 11" with the highest risk in the Regenerative Medicine Safety Assurance Act that came into effect on November 25, XNUMX. It refers to clinical research on regenerative medicine that meets strict standards.
