NEW ORLEANS - She was only in kindergarten when doctors gave her family the bad news.
Now she's one of the first in Louisiana to try a new treatment for people who get gravely ill after a bone marrow transplant.
The last three years of Sami Smith's life have been physically and emotionally painful.
"I literally, they try to scare me and they can't, because I've been through the scariest thing that you can," said Smith, 9, of Ponchatoula.
Her mother noticed she was napping more and bruising. Doctors diagnosed AML, a type of leukemia or blood cancer. Had she not gotten to the doctor then, she would not have made it much longer. A Child's Wish sent her to Disney World. The good news, one of her teen sisters Mary Hannah, 13, was a good bone marrow match. The transplant worked and Sami was cancer free.
Then devastating news. Sami got a condition called GvHD (Graft-versus-host disease) where the new marrow launches a painful attack on the recipient's body. It's the leading cause of transplant-related death.
"She was in horrible pain. She didn't want lights. She didn't want sound. We'd just sit for days on end in silence," her mother, Gabriella Smith remembers sadly.
Doctors say that 15 to 50 percent of the children who get a bone marrow transplant end up with GvHD and there's even a higher risk in adult patients who get a bone marrow transplant.
Every treatment Sami tried failed. Then as a last hope, doctors at Children's Hospital tried a new type of cellular therapy called Prochymal (remestemcel-L). It's the world's first approved stem cell treatment. Sami got 12 infusions. It worked.
"Yes, it worked and it was amazing. It took her about two infusions before we started seeing that her diarrhea subsided," said Dr. Lolie Yu, the director and division chief of Pediatric Hematology and Oncology at LSU Health Sciences Center and the stem cell transplant program at Children's Hospital and LSUHSC.. "After the fourth infusion she was able to eat and feel a little bit better. It's very, very optimistic for her as far as her future is concerned."
Sami and her family were invited by Osiris Therapeutics to Washington DC to meet the scientists and see those microscopic special stem cells.
"It was kind of cool because I got to like see what saved my life. It was kind of like, 'Oh my God, these things are so small and they saved my life.' And I was just thankful that that drug was there at the time," said Sami.
Sami is still swollen from the years of steroids. She hopes to go back to school in her home in Ponchatoula. Home school teachers say she tests at advanced levels. Sami is not only book smart but also wise beyond her years. Her mother heard her prayer after her new friend on the ward, a little boy with cancer, lost his fight.
"She said, 'Thank you for giving me cancer and leading me down this path. It's allowed me to meet all of these people that's been brought into my life,' " said Gabriella.
The new stem cell therapy can be used in any person.
It is approved in Canada but still being tested in the U.S.
Doctors hope it will be approved by the FDA by the end of the year.
GvHD represents a major unmet medical need with no approved treatment until Prochymal. GvHD is the leading cause of transplant related mortality, in which immune cells contained within the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure. Severe GvHD is extremely painful and fatal in up to 80 percent of cases. Currently, steroids are used as first-line therapy with a success rate of only 30-50 percent. When steroids fail, treatment options are limited to immunosuppressive agents used off-label with little benefit and significant toxicities.
About Prochymal (remestemcel-L):
Prochymal is the world's first approved drug which has a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is currently approved in Canada for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Program. Prochymal is currently in Phase 3 trials for refractory Crohn's disease and also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.
About Osiris Therapeutics
Osiris Therapeutics, Inc. is the leading stem cell company, having developed the world's first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic areas and wound healing areas. Osiris currently markets Prochymal for refractory GvHD, Grafix® for burns and chronic wounds, and Ovation® for orthopedic applications. The company's pipeline of internally developed biologic drug candidates under evaluation includes Prochymal for inflammatory, autoimmune and cardiovascular indications, as well as Chondrogen for arthritis in the knee. Osiris is a fully integrated company with capabilities in research, development, manufacturing and distribution of stem cell products. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign patents.
Osiris, Prochymal, Grafix and Ovation are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, www.Osiris.com. (OSIR-G)