A "T-cell vaccine" to treat multiple sclerosis (MS) will enterpivotal Phase II/III clinical trials this summer in Belgium, withmajor funding from Cel-Sci Corp., the company announcedTuesday.
Cel-Sci of Alexandria, Va., signed a letter of intent over theweekend with the Dr. L. Willems Institute of Diepenbeek,Belgium (about 40 miles east of Brussels), for an exclusiveworldwide license to the vaccine. The institute, an independentpublic-service facility affiliated with Limburg University, isBelgium's leading MS treatment and research center.
In Phase I trials at Willems last year, nine MS patients receivedthree subcutaneous injections of their own selectively cloned Tcells activated by cells presenting myelin basic protein (MBP)antigen. This dosage and toxicity study showed the vaccinationto be non-toxic in humans. A bonus finding revealed that thelevel of MBP-reactive T cells PP prime suspects in the etiologyof MS PP had dropped to zero in all nine patients.
Science published a paper reporting this human experimentlast September. BioWorld also reported it in an articleheadlined, "MS Vaccine Trial Asks Questions, ProvidesAnswers," describing the subcutaneous inoculations developedby Willems neuroimmunologist Jingwu Zhang as consisting of"self" -- T cells programmed to destroy the other T cells thatattack myelin tissue, and so bring on MS. To counterattack themyelin-munching autoreactive T cells turned on by MBPantigen, Zhang's system immunized MS patients against thesesingle-target autoimmune T lymphocytes.
A few days after this story appeared, Cel-Sci's chief operatingofficer, Geert Kersten, called BioWorld to obtain the WillemsInstitute's telephone number. "We had looked at Zhang's paperin Science and put it aside until the article in BioWorldreminded us of this," he recalled. "We called them, double-checked all of the data that you had written about, had ourscientists go there and found everything 100 percent top-notch."
While the Belgians were visiting Alexandria to sign the letter ofintent, they also worked out tentative details of the majorclinical trial, Kersten added, "which is bigger than anything Cel-Sci has ever been involved in. We will try to design it as apivotal Phase II/III trial for which we will file an IND. Thatway the FDA gets a chance to review the protocol and makesuggestions so the results are acceptable in the U.S."
The single-center, double-blinded, placebo-controlled studywill take place at Willems, enrolling about 100remitting/relapsing MS patients to receive the vaccine and anequal number of controls. It should start this summer andcontinue for three or four years, Kersten said, noting that they"haven't decided yet on the trial's endpoints."
Under the terms of its impending license agreement, to befinalized contractually in two months, Cel-Sci will underwritethe costs of the trial and associated research. Kersten did notdisclose the amount of funding involved but described the sumas "substantial." As part of the overall contract, Cel-Sci getsrights of first refusal on other projects the institute undertakes,past and future. "We found all kinds of very interesting workthat they are doing in MS diagnostics and various other areas,such as T cell vaccination for rheumatoid arthritis," saidKersten.
He added that "MS diagnosis is still in the Stone Age. If you candefinitely diagnose somebody early on, you'll be able to savepeople not only many years of agony but also incredibleamounts of money. You'll be able to start the T cell treatmentmuch earlier, to halt the disease more effectively."
Chiron Corp.'s recently approved MS therapeutic, Betaseron,Kersten observed, "while very good, has side effects, requiresshots every other day, at a cost of $10,000 a year, and eventhen only reduces the MS exacerbation rate by 30 percent."
By contrast, he explained, Willems' T cell vaccination is a one-time treatment, with three injections spaced six months apart,and is non-toxic because it puts the patient's own cells backafter cloning and irradiating them.
-- David N. Leff Science Editor
(c) 1997 American Health Consultants. All rights reserved.