CellCept (mycophenolate mofetil) is an oral immunosuppressant developed by Genentech, a member of the Roche group, and approved by the U.S. Food and Drug Administration (FDA) to prevent organ rejection in transplant patients.

The medicine is also being investigated as a treatment for patients with a type of Batten disease called juvenile Batten disease.

How CellCept works

Batten disease is an inherited neurodegenerative disorder that affects the normal function of lysosomes, which are responsible for clearing waste material from cells. When lysosomes do not work properly, waste products build inside cells, causing them to malfunction and eventually killing them.

But some studies suggest that certain symptoms of juvenile Batten disease could be caused by an autoimmune response, in which the patient’s immune system produces antibodies that mistakenly attack nerve cells, leading to neurodegeneration as the disease progresses.

CellCept is a therapy that suppresses the immune system and stops the formation of these antibodies. It directly interferes with DNA replication in immune cells, slowing the formation of new cells. In this way, it is expected that the medicine may reduce the levels of antibodies that attack nerve cells, slowing both disease progression and neurodegeneration.

CellCept in clinical trials for Batten disease

A study testing the effects of CellCept in a mouse model of juvenile Batten disease found that the medicine led to improvements in the animals’ motor function as well as a reduction in nerve cell death and circulating autoantibodies.

Based on these findings, the safety and tolerability of CellCept in treating 19 ambulatory juvenile Batten disease patients for eight weeks were evaluated in a Phase 2 placebo-controlled clinical trial (NCT01399047). The 22-week study was a cross-over study, meaning one group was treated for eight weeks while another was given placebo, then after a four-week washout, treatments were switched.

The study’s primary outcome was the tolerability of short-term treatment. Secondary objectives included signs of efficacy, as measured by the Unified Batten Disease rating scale (UBDRS), a scale developed to quantify the motor, behavioral, and functional capability of patients.

Trial results, published in the scientific journal JIMD Reports in 2018, showed that Cellcept was well-tolerated in the short-term, with no significant differences in tolerability rates between CellCept and placebo group and no serious side effects.

However, no definite effects on measures of autoimmunity or clinical outcomes in eight weeks of treatment were seen.The researchers recommended further and long-term treatment to determine the clinical benefit, as well as the safety, of CellCept in these patients.

Other details

Common side effects of CellCept include diarrhea; vomiting; stomach pain; swelling of the lower legs, ankles, and feet; and high blood pressure.

Because CellCept weakens the immune system, patients are at a higher risk of infections.


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