PARKINSON’S PROJECT AIMS FOR 2014 APPROVAL
By Bradley J. Fikes • U-T DEC. 15, 2013
For eight local Parkinson’s patients seeking treatment with stem cell technology, 2014 could bring the milestone they’ve been anticipating.
If all goes well, the U.S. Food and Drug Administration will approve an attempt to replace the brain cells destroyed by Parkinson’s. The new cells, grown from each patient’s own skin cells, are expected to restore normal movement in the patients.
Because the new brain cells are made from the patients’ own cells, immunosuppressive drugs shouldn’t be needed. Ideally, patients could stop taking their medications and resume normal activities for many years, or even the rest of their lives.
The project, Summit4StemCell.org, is a collaboration between three nonprofits. The Scripps Research Institute handles the science; Scripps Clinic takes care of the medical side; and the Parkinson’s Association of San Diego helps to raise money for the self-funded project.
Since 2011, the focus has been at the institute, where scientists led by Jeanne Loring have made the artificial embryonic stem cells, called induced pluripotent stem cells, and turned them into the needed brain cells. Now Scripps Clinic is assuming a more prominent role to prepare for treating the patients.
A study in rats began in early December; results are expected by April. The animal study is meant to assess safety, although researchers will also look for signs of effectiveness.
In January, scientists will visit the FDA to lay the groundwork for a formal application, said Scripps Clinic neurologist Melissa Houser, who treats all eight patients.
Success in the animal study will likely result in a go-ahead, Houser said. If the animal trial fails, “it’s back to the drawing board.”
FDA approval could come in the fall, Houser said. Approval will also be needed from Scripps Clinic. Scripps will appoint an institutional review board to examine the proposed line of treatment to make sure patients are protected.
Assuming success in the pilot study, Houser said there will be a strong case for performing a conventional clinical trial with more people.
The process is expensive, although numbers at this state are still imprecise. About $900,000 has been raised so far, according to the group. This has financed research up to the animal study phase. Getting to the FDA application stage will take an additional $3.1 million, and then $1 million to conduct the procedure and provide follow-up. That’s a total of $5 million.
However, Houser says more conservative figures point to a total amount of from $7 million to as much as $10 million. The additional $2 million to $5 million constitutes a reserve to care for patients just in case the treatment goes wrong.
That’s a lot of money to just eight people. But it’s also a proof of concept for a treatment that could be applied to other Parkinson’s patients. And it could save money down the road in avoided costs of years of medical treatment.
Parkinson’s destroys brain cells that make dopamine, a neurotransmitter that enables movement. Medications can restore movement but tend to produce uncontrollable abnormal movements and tics called dyskinesias.
A study led by Sanford-Burnham scientists indicates a mutation predisposes some to the incurable disease. Parkinson’s is triggered by exposure to certain pesticides or the herbicide Paraquat, according to the study. But no cause has been found for Parkinson’s in the majority of patients.
Loring said it’s likely that these new dopamine-producing neurons will also be destroyed by the disease. However, the new cells could last for 10 to 15 years, perhaps longer, considering the disease’s slow progress. So the research team decided to ignore the question of how the original cells were destroyed and concentrate on replacing them. Loring and patient Ed Fitzpatrick discussed the project’s status at the World Stem Cell Summit this month in San Diego.
Earlier attempts to treat Parkinson’s with a stem cell-like therapy mostly failed because of difficulties in quality control of the source, brain cells from aborted fetuses, Loring said. But some patients gained lasting improvement, a hint that the trials were on the right track.
Fitzpatrick said he’s eager to go ahead.
“I am 66 years old and growing very impatient waiting for something that can permit me to go from a sitting position on the floor to a full upright stance in less than eight minutes and 20 seconds, and without holding on to a dog’s tail and yelling, ‘Sic ’em,’ ” Fitzpatrick said.
By the standards of traditional research, Summit4StemCell is progressing extremely quickly. The approach being studied wasn’t even possible until 2006, when Japanese researcher Shinya Yamanaka first produced induced pluripotent stem cells from animals. In 2007, Yamanaka made them from human cells. For his discovery, Yamanaka shared the Nobel Prize in Physiology or Medicine in 2012.
Scripps Clinic and TSRI will hold a public forum on the project’s status from 6 to 8 p.m. Jan. 29 at Scripps Green Hospital, 10666 N. Torrey Pines Road in La Jolla, at the Tamkin Amphitheatre. To register, call (800) 727-4777.
By Bradley J. Fikes • U-T DEC. 15, 2013
For eight local Parkinson’s patients seeking treatment with stem cell technology, 2014 could bring the milestone they’ve been anticipating.
If all goes well, the U.S. Food and Drug Administration will approve an attempt to replace the brain cells destroyed by Parkinson’s. The new cells, grown from each patient’s own skin cells, are expected to restore normal movement in the patients.
Because the new brain cells are made from the patients’ own cells, immunosuppressive drugs shouldn’t be needed. Ideally, patients could stop taking their medications and resume normal activities for many years, or even the rest of their lives.
The project, Summit4StemCell.org, is a collaboration between three nonprofits. The Scripps Research Institute handles the science; Scripps Clinic takes care of the medical side; and the Parkinson’s Association of San Diego helps to raise money for the self-funded project.
Since 2011, the focus has been at the institute, where scientists led by Jeanne Loring have made the artificial embryonic stem cells, called induced pluripotent stem cells, and turned them into the needed brain cells. Now Scripps Clinic is assuming a more prominent role to prepare for treating the patients.
A study in rats began in early December; results are expected by April. The animal study is meant to assess safety, although researchers will also look for signs of effectiveness.
In January, scientists will visit the FDA to lay the groundwork for a formal application, said Scripps Clinic neurologist Melissa Houser, who treats all eight patients.
Success in the animal study will likely result in a go-ahead, Houser said. If the animal trial fails, “it’s back to the drawing board.”
FDA approval could come in the fall, Houser said. Approval will also be needed from Scripps Clinic. Scripps will appoint an institutional review board to examine the proposed line of treatment to make sure patients are protected.
Assuming success in the pilot study, Houser said there will be a strong case for performing a conventional clinical trial with more people.
The process is expensive, although numbers at this state are still imprecise. About $900,000 has been raised so far, according to the group. This has financed research up to the animal study phase. Getting to the FDA application stage will take an additional $3.1 million, and then $1 million to conduct the procedure and provide follow-up. That’s a total of $5 million.
However, Houser says more conservative figures point to a total amount of from $7 million to as much as $10 million. The additional $2 million to $5 million constitutes a reserve to care for patients just in case the treatment goes wrong.
That’s a lot of money to just eight people. But it’s also a proof of concept for a treatment that could be applied to other Parkinson’s patients. And it could save money down the road in avoided costs of years of medical treatment.
Parkinson’s destroys brain cells that make dopamine, a neurotransmitter that enables movement. Medications can restore movement but tend to produce uncontrollable abnormal movements and tics called dyskinesias.
A study led by Sanford-Burnham scientists indicates a mutation predisposes some to the incurable disease. Parkinson’s is triggered by exposure to certain pesticides or the herbicide Paraquat, according to the study. But no cause has been found for Parkinson’s in the majority of patients.
Loring said it’s likely that these new dopamine-producing neurons will also be destroyed by the disease. However, the new cells could last for 10 to 15 years, perhaps longer, considering the disease’s slow progress. So the research team decided to ignore the question of how the original cells were destroyed and concentrate on replacing them. Loring and patient Ed Fitzpatrick discussed the project’s status at the World Stem Cell Summit this month in San Diego.
Earlier attempts to treat Parkinson’s with a stem cell-like therapy mostly failed because of difficulties in quality control of the source, brain cells from aborted fetuses, Loring said. But some patients gained lasting improvement, a hint that the trials were on the right track.
Fitzpatrick said he’s eager to go ahead.
“I am 66 years old and growing very impatient waiting for something that can permit me to go from a sitting position on the floor to a full upright stance in less than eight minutes and 20 seconds, and without holding on to a dog’s tail and yelling, ‘Sic ’em,’ ” Fitzpatrick said.
By the standards of traditional research, Summit4StemCell is progressing extremely quickly. The approach being studied wasn’t even possible until 2006, when Japanese researcher Shinya Yamanaka first produced induced pluripotent stem cells from animals. In 2007, Yamanaka made them from human cells. For his discovery, Yamanaka shared the Nobel Prize in Physiology or Medicine in 2012.
Scripps Clinic and TSRI will hold a public forum on the project’s status from 6 to 8 p.m. Jan. 29 at Scripps Green Hospital, 10666 N. Torrey Pines Road in La Jolla, at the Tamkin Amphitheatre. To register, call (800) 727-4777.