Dr. Lenny Bell - A man on a mission
By: Marilyn Moss, Special to the Bulletin
03/29/2007
WOODBRIDGE - Dr. Lenny Bell and his company, Alexion Pharmaceuticals, have broken new ground in the biotechnology industry. On March 16, the FDA approved an innovative new drug developed by Alexion. The drug, eculizumab or Soliris, is an infusible monoclonal antibody for treating a rare blood disorder, paroxysmal nocturnal hemoglobinuria or PNH. Until now, there has been no way to treat this debilitating and life-threatening disease.
PNH is an uncommon disease, affecting less than 10,000 people in the U.S. and Western Europe. Yet those who suffer from PNH can be incapacitated. The average life span following diagnosis is 10 years, and those years can be limited by anemia, pain, and excessive fatigue. PNH patients can also develop blood clots or thromboses, which is the leading cause of death in those with the disease.
PNH results from an acquired genetic mutation to bone marrow stem cells.
This mutation leads to the production of red blood cells with abnormal cell membranes, leaving them vulnerable to destruction by the body's ever-vigilant immune system. These abnormal blood cells are attacked by complement proteins, which are part of the body's immune system. This results in the destruction of the mutated red blood cells or hemolysis. Soliris blocks this hemolysis by blocking part of the protective complement cascade.
Until now, treatment for patients was limited to blood transfusions to counteract the anemia resulting from the hemolysis. Other complications required additional interventions, such as anticoagulant therapy for those who developed blood clots.
With the advent of Soliris, PNH patients face a different future. Studies involving the use of Soliris have shown a 100 percent observable beneficial effect in PNH patients. In clinical studies, after PNH patients received Soliris, their need for transfusions was dramatically reduced. In fact, 50 percent of patients required no transfusions after treatment began.
Bell said, "Hemolysis was decreased in every single patient."
Blocking the actions of the complement proteins required a delicate balance, since complement is the body's first line of defense against infections. Alexion had to develop an antibody to block the complement cascade after the point at which it succeeded in dispatching infections but before it could destroy blood cells. The developed antibody accomplishes this, except for one particular type of bacteria, meningococcal infection. In light of the potential risk from meningococcal infection, patients must be appropriately vaccinated before beginning treatment, and they must be carefully monitored for signs of infection.
Alexion projects that Soliris will be available for treatment in the U.S. before the end of the month. To aid patients and physicians, Alexion is offering a program called Soliris OneSource to deal with some of the issues related to this innovative treatment. This program provides trained case managers to deal with education and facilitation of treatment. Information for patients is available through www.soliris.net or by phone at 888-765-4747.
Bell said that this was a monumental accomplishment for Alexion. Bell said, "The number of companies that come out with a successful drug is low."
But Alexion has more plans for Soliris. In particular, the company will be looking at the effectiveness of Soliris treatment in regards to decreasing other PNH interventions, such as the use of anticoagulants. According to the studies, use of Soliris resulted in a reduction of thrombotic events in PNH patients. Bell said, "We will continue to look at the withdrawal of anticoagulants in PNH patients."
In addition, Alexion will continue to investigate the effectiveness of Soliris in other complement-mediated diseases, such as rheumatoid arthritis or myasthenia gravis.
Bell, a Woodbridge resident, followed his dream to establish a biotechnology company to offer therapies for clinical treatment. Bell, along with several colleagues, started Alexion in the early 1990s. The company, which was originally located in Science Park in New Haven, relocated to Cheshire in 2000. The facility now employs over 200 individuals, and Alexion has facilities in Rhode Island and Paris, as well.
The road for Bell has been long and arduous, with unexpected turns and unforeseen obstacles. But Bell and his colleagues stayed the course. These efforts led to a much-needed therapy for PNH patients. "Treatment with Soliris has transformed patients' lives," said Bell.
Bell attributed his success to passion and perseverance. Those are inherent qualities that Bell brings to all his endeavors. Some residents may remember Bell's instrumental leadership with the Amity Board of Education as well as in modulating the planned electrical upgrade that affected Woodbridge, as well as other local communities. Bell successfully coerced the utility companies to make modifications to their electrical upgrade plans to minimize potential health and safety risks to residents.
Bell compared his success with the electrical companies to those in the biotechnology industry. "You succeed because you have passion and persistence. It all hangs together pretty well."
zwire.com |
