BHT-3009, a myelin basic protein-encoding plasmid for the treatment of multiple sclerosis.
Even though the etiology of multiple sclerosis (MS) remains largely unknown, research data support the hypothesis that autoimmunity plays a major role in disease development. Several disease-modifying agents have been approved for the treatment of MS; however, there is still a need for antigen-specific treatments that combine efficacy and safety. DNA vaccination represents a new therapeutic alternative in this respect. Preclinical studies in different models of autoimmunity have demonstrated that injection of plasmid DNA encoding a self-antigen in mice restores self-tolerance, leaving immunity against infectious and tumor antigens intact. Based on this evidence, the first DNA vaccine for MS has been created. Bayhill Therapeutic Inc's BHT-3009 encodes full-length, human myelin basic protein (MBP), and has recently been evaluated in a phase I/II and a phase II clinical trial. BHT-3009 was safe and well tolerated in both trials, inducing immune tolerance that extended beyond MBP to other myelin antigens. In addition, a reduction in the number of active lesions was observed, which was accompanied by a decrease in clinical relapse rates, particularly in patients with high immunological activity at baseline. BHT-3009 appears to be a promising new approach for the treatment of MS, although further clinical trials are warranted to confirm the early findings.
Correale
Jorge
J
Raúl Carrea Institute for Neurological Research (FLENI), Department of Neurology, Buenos Aires, Argentina. jcorreale@fleni.org.ar
Fiol
Marcela
M
D002986:Clinical Trials as Topic; D000075202:Contraindications; D004353:Drug Evaluation, Preclinical; D006801:Humans; D009103:Multiple Sclerosis / Q000188:drug therapy*; D004676:Myelin Basic Protein / Q000378:metabolism*; D010330:Patents as Topic; D010957:Plasmids / Q000235:genetics*; D013329:Structure-Activity Relationship; D019444:Vaccines, DNA / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*
C544595:BHT 3009; D004676:Myelin Basic Protein; D019444:Vaccines, DNA