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Hopkins Health
Seeking a Safe Treatment for a Stubborn Disorder
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| Dermatologist Grant Anhalt, searching for an answer to the painful and persistent autoimmune disease pemphigus vulgaris. |
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Dermatologist Grant Anhalt, M.D., couldn’t have been happier two years ago when he saw the fantastic results the chemotherapy cyclophosphomide was having on his patients with the potentially fatal autoimmune skin disease pemphigus vulgaris.
All but one of them went into remission. In one patient, whose open blisters covering the inside of her mouth (a hallmark of the disease) had persisted two years, the sores began to heal within days of the treatment.
That was the good news. Now, the bad news. Although the patients who underwent the cyclophosphomide therapy experienced remissions of up to two years, they’ve all relapsed. Many of them are now being treated conventionally with corticosteroids, which carry serious side effects, including cataracts, hypertension and weight gain. Dermatologists like Anhalt were hoping that cyclophosphomide, which can have severe short-term side effects like hair loss and nausea, would avoid these long-term reactions.
The cyclophosphomide experience has pushed Anhalt back into the lab to search even harder for a therapy for this debilitating disease, which can cause severe pain, inability to eat, weight loss and emotional distress. Anhalt wants an effective treatment that is also less toxic. He’s now targeting two major immune regulation genes in pemphigus, called DR4 and DR6, predominant in Jewish and Asian populations, respectively. In animal models in other autoimmune diseases, Anhalt notes, flooding immune cells with a specific protein has been shown to shut the cells down.
“There’s good evidence that this peptide starts the whole process of antibody production, so it should down-regulate that process and improve the disease,” Anhalt says. “And because it’s just a piece of protein and not a drug, it should have no toxicity.”
While Anhalt awaits approval of a clinical trial to study the safety of the DR4 therapy, he continues to treat patients who have relapsed under cyclophosphomide. Some good news, he notes, is that the anti-cancer drug apparently does alter the disease in some way, making patients more responsive to conventional treatments.
“Some cyclophosphomide patients who have relapsed can now be managed with a little bit of prednisone, and others are responding to less-toxic drugs they failed before,” Anhalt says. “In some folks it really made the disease easier to manage.”
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