By Julia Bayly, BDN Staff
Posted May 16, 2015
Click for video:
http://bangordailynews.com/2015/05/16/health/a-promise-of-hope-bangor-boy-to-take-part-in-experimental-stem-cell-therapy-to-treat-his-autism/
BANGOR, Maine — Among all places a mom and 12-year-old son might hang out, Bangor International Airport is perhaps the least likely. For Connor and Rachel White, though, the airport has become a regular stop as they prepare for a trip of a lifetime.
On June 7, the Whites will leave Bangor en route to Panama so Connor can take part in an exciting, but highly experimental, stem cell therapy to treat his autism.
That treatment, said Rachel, involves doctors from the U.S. and around the world, and it will hopefully help lessen Connor’s symptoms. Connor is nonverbal — he does not speak — and his mother describes him as “challenged with sensory processing.”
Imagine, she said, sitting in a seat and someone is reading a story to you in a loud voice, touching you and making you smell intense smells all at the same time.
“That is what it is like for people with autism like Connor all the time,” she said. “Every sound — traffic, the fridge starting up, dogs barking — come at him with equal intensity, [and] there is no filtering system.”
This makes preparing for the upcoming flights so critical. Through repeated airport visits, Rachel wants to establish a frequent-flyer routine for Connor before they take off.
They have watched videos of a plane during a flight, created stories about Connor’s upcoming flying adventures and made numerous trips to the federal building in downtown Bangor just to use the restroom.
“To use the bathroom you have to go through security, [and] now he knows what that is like,” she said. “The guards there have been wonderful.”
For Rachel, the upcoming flight is the culmination of a five-year effort, which started when she saw a news piece on stem cell therapy.
“When I started looking into it I saw a promise of hope that, prior to that, we did not have,” she said. “I was so excited about seeing it and maybe Connor being a part of how the United States will move forward in making the treatment available.”
The treatment Connor will receive in Panama, however, is not approved by the Food and Drug Administration, nor sanctioned by the Autism Science Foundation , which posts this warning on its website: “There is no evidence that the treatment is safe or effective for autism, and no guarantee that the stem cells used in these countries are even human.”
Rachel and Connor are optimistic and say they have the support of their physicians.
“Connor’s doctors all know that I have dedicated my life to researching treatments for autism,” said Rachel. “His pediatrician and his pediatric nurse have been nothing but supportive through every prior step that has brought us to today, [and] they understand how much this means to me and what this could mean for Connor.”
Promise, but risks
It’s a long way from Bangor to the Stem Cell Institute in Panama City, where Connor will go for treatment.
Rachel first applied to be part of the trial’s Phase 1 study in 2013 but was not accepted. A month ago, the family was contacted by the center and asked, based on their 2013 application, if they would like to re-apply.
Now Connor is among 20 children between the ages of 6 and 16 taking part in Phase 2 of the Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism clinical trial. In the first phase of clinical trials, researchers mainly evaluate a drug or therapy’s safety.
The second phase involves a larger study group and further tests safety and whether the treatment is effective. New medications and therapies must then pass a third phase before being sold, and many never make it to market.
The institute says its adult stem cells used in the trial come from donated umbilical cord tissue. Its research says stem cells can regulate the immune system, and it is thought they may help reverse some of the conditions associated with autism.
Rachel calls what the stem cells will do “a biochemical reboot of the brain.”
The treatment will take place during four five-day sessions over the coming year, she said, all in Panama, where the stem cells will be administered to Connor intravenously much like chemotherapy treatments.
The regulatory authority overseeing the trial is Panamanian, since stem cell therapy for autism is not approved by the FDA, said Laura Keylon, patient coordinator at the Stem Cell Institute.
“The trial is approved under the Panamanian food and drug regulations,” she said. “But we are getting closer to having trials approved in the U.S.”
The first stem cell trial for autism in this country is being conducted by the Sutter Health network and is about halfway through its course, according to Scientific American. It differs from the Panama trials in that children between 2 and 7 with autism received a single infusion of their own umbilical cord blood.
Last July, Duke University was reportedly getting ready to launch its own clinical trial exploring stem cells from umbilical cord blood as a treatment for autism. But experts also have cautioned that the Duke trial is premature.
It is this situation — and the easier availability of treatment abroad — that has many Americans with many maladies going overseas for health care.
In most cases, however, those trips fall into the category of medical tourism — electing foreign treatment for cost purposes, said Gordon Smith, executive vice president of the Maine Medical Association. This is not the case for the Whites.
“Their situation is truly different,” Smith said. “They are going out of the country because the treatment is not available here because it has not been approved here.”
That move is not always supported by American doctors.
“Most health professionals in this country are not comfortable with [traveling for non-FDA- approved treatments] because we rely on the FDA and regulators to require that the manufacturing of devices, procedures and drugs show ethics and safety,” Smith said.
There also is a risk that patients and their families could be vulnerable to scams or fraudulent practices in foreign countries, by unscrupulous facilities that offer false hope instead of groundbreaking treatments.
However, Smith did say that since Connor’s study is listed on the U.S. National Institutes of Health-maintained website, it lends it great credibility, as does the fact it is in the second phase of trials. He wishes them well but also urges caution.
“I certainly hope the best for them,” he said. “But this is not something the Maine Medical Association would ever recommend due to the lack of [FDA] safety regulations, and we are a pretty progressive organization.”
Optimism, realism
Connor is a sixth-grader at William S. Cohen School, where he takes part in its aspirations program. He also is a regular at the Bangor YMCA pool and participates in Special Olympics.
He lives on his routine, Rachel said, so any disruption can be emotionally catastrophic.
“Thank God,” she said, “there is a direct flight from Boston to Panama City.”
Because he is part of a clinical trial, all costs associated with obtaining, administering and tracking the stem cell treatments have been waived for Connor. Still, the costs of four trips to Panama are up to the family, which Rachel estimates will be around $17,000.
She has set up a crowdfunding campaign, and a fundraising spaghetti dinner and silent auction is planned for 5-9 p.m. May 20 at Hermon High School.
Rachel and Connor will fly from Bangor to Boston on an Angel Flight Northeast, which provides free air travel for individuals seeking medical attention far from home.
For travel days, Rachel had special T-shirts made for Connor that say, “This is What Autism Looks Like” and “Please be Patient.” En route, he will have activities to keep him occupied.
“He travels well as long as he has pistachios to open, [and] he can chew gum to help with changes in air pressure,” she said. “He’ll have his iPad and will sit next to the window so he can look out.”
All of these activities combine to make air travel part of Connor’s routine and increase the odds for a calm flight.
“Through my experience of being his mom for a dozen years, I have learned how to speak the language of autism,” Rachel said. “You want to be three steps ahead and always backward planning for what we may face that day.”
After his year of treatments in Panama, Connor will continue to be tracked by the medical staff at the Stem Cell Institute for a full year. The study is due to end in 2017.
With takeoff just weeks away, Rachel is optimistic — and realistic.
“I am not delusional and thinking he will be cured 100 percent, because the reality is Connor will still be autistic at the end of the trial,” she said. “If what we are doing is a step forward in curing autism, that is very exciting.”
Posted May 16, 2015
Click for video:
http://bangordailynews.com/2015/05/16/health/a-promise-of-hope-bangor-boy-to-take-part-in-experimental-stem-cell-therapy-to-treat-his-autism/
BANGOR, Maine — Among all places a mom and 12-year-old son might hang out, Bangor International Airport is perhaps the least likely. For Connor and Rachel White, though, the airport has become a regular stop as they prepare for a trip of a lifetime.
On June 7, the Whites will leave Bangor en route to Panama so Connor can take part in an exciting, but highly experimental, stem cell therapy to treat his autism.
That treatment, said Rachel, involves doctors from the U.S. and around the world, and it will hopefully help lessen Connor’s symptoms. Connor is nonverbal — he does not speak — and his mother describes him as “challenged with sensory processing.”
Imagine, she said, sitting in a seat and someone is reading a story to you in a loud voice, touching you and making you smell intense smells all at the same time.
“That is what it is like for people with autism like Connor all the time,” she said. “Every sound — traffic, the fridge starting up, dogs barking — come at him with equal intensity, [and] there is no filtering system.”
This makes preparing for the upcoming flights so critical. Through repeated airport visits, Rachel wants to establish a frequent-flyer routine for Connor before they take off.
They have watched videos of a plane during a flight, created stories about Connor’s upcoming flying adventures and made numerous trips to the federal building in downtown Bangor just to use the restroom.
“To use the bathroom you have to go through security, [and] now he knows what that is like,” she said. “The guards there have been wonderful.”
For Rachel, the upcoming flight is the culmination of a five-year effort, which started when she saw a news piece on stem cell therapy.
“When I started looking into it I saw a promise of hope that, prior to that, we did not have,” she said. “I was so excited about seeing it and maybe Connor being a part of how the United States will move forward in making the treatment available.”
The treatment Connor will receive in Panama, however, is not approved by the Food and Drug Administration, nor sanctioned by the Autism Science Foundation , which posts this warning on its website: “There is no evidence that the treatment is safe or effective for autism, and no guarantee that the stem cells used in these countries are even human.”
Rachel and Connor are optimistic and say they have the support of their physicians.
“Connor’s doctors all know that I have dedicated my life to researching treatments for autism,” said Rachel. “His pediatrician and his pediatric nurse have been nothing but supportive through every prior step that has brought us to today, [and] they understand how much this means to me and what this could mean for Connor.”
Promise, but risks
It’s a long way from Bangor to the Stem Cell Institute in Panama City, where Connor will go for treatment.
Rachel first applied to be part of the trial’s Phase 1 study in 2013 but was not accepted. A month ago, the family was contacted by the center and asked, based on their 2013 application, if they would like to re-apply.
Now Connor is among 20 children between the ages of 6 and 16 taking part in Phase 2 of the Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism clinical trial. In the first phase of clinical trials, researchers mainly evaluate a drug or therapy’s safety.
The second phase involves a larger study group and further tests safety and whether the treatment is effective. New medications and therapies must then pass a third phase before being sold, and many never make it to market.
The institute says its adult stem cells used in the trial come from donated umbilical cord tissue. Its research says stem cells can regulate the immune system, and it is thought they may help reverse some of the conditions associated with autism.
Rachel calls what the stem cells will do “a biochemical reboot of the brain.”
The treatment will take place during four five-day sessions over the coming year, she said, all in Panama, where the stem cells will be administered to Connor intravenously much like chemotherapy treatments.
The regulatory authority overseeing the trial is Panamanian, since stem cell therapy for autism is not approved by the FDA, said Laura Keylon, patient coordinator at the Stem Cell Institute.
“The trial is approved under the Panamanian food and drug regulations,” she said. “But we are getting closer to having trials approved in the U.S.”
The first stem cell trial for autism in this country is being conducted by the Sutter Health network and is about halfway through its course, according to Scientific American. It differs from the Panama trials in that children between 2 and 7 with autism received a single infusion of their own umbilical cord blood.
Last July, Duke University was reportedly getting ready to launch its own clinical trial exploring stem cells from umbilical cord blood as a treatment for autism. But experts also have cautioned that the Duke trial is premature.
It is this situation — and the easier availability of treatment abroad — that has many Americans with many maladies going overseas for health care.
In most cases, however, those trips fall into the category of medical tourism — electing foreign treatment for cost purposes, said Gordon Smith, executive vice president of the Maine Medical Association. This is not the case for the Whites.
“Their situation is truly different,” Smith said. “They are going out of the country because the treatment is not available here because it has not been approved here.”
That move is not always supported by American doctors.
“Most health professionals in this country are not comfortable with [traveling for non-FDA- approved treatments] because we rely on the FDA and regulators to require that the manufacturing of devices, procedures and drugs show ethics and safety,” Smith said.
There also is a risk that patients and their families could be vulnerable to scams or fraudulent practices in foreign countries, by unscrupulous facilities that offer false hope instead of groundbreaking treatments.
However, Smith did say that since Connor’s study is listed on the U.S. National Institutes of Health-maintained website, it lends it great credibility, as does the fact it is in the second phase of trials. He wishes them well but also urges caution.
“I certainly hope the best for them,” he said. “But this is not something the Maine Medical Association would ever recommend due to the lack of [FDA] safety regulations, and we are a pretty progressive organization.”
Optimism, realism
Connor is a sixth-grader at William S. Cohen School, where he takes part in its aspirations program. He also is a regular at the Bangor YMCA pool and participates in Special Olympics.
He lives on his routine, Rachel said, so any disruption can be emotionally catastrophic.
“Thank God,” she said, “there is a direct flight from Boston to Panama City.”
Because he is part of a clinical trial, all costs associated with obtaining, administering and tracking the stem cell treatments have been waived for Connor. Still, the costs of four trips to Panama are up to the family, which Rachel estimates will be around $17,000.
She has set up a crowdfunding campaign, and a fundraising spaghetti dinner and silent auction is planned for 5-9 p.m. May 20 at Hermon High School.
Rachel and Connor will fly from Bangor to Boston on an Angel Flight Northeast, which provides free air travel for individuals seeking medical attention far from home.
For travel days, Rachel had special T-shirts made for Connor that say, “This is What Autism Looks Like” and “Please be Patient.” En route, he will have activities to keep him occupied.
“He travels well as long as he has pistachios to open, [and] he can chew gum to help with changes in air pressure,” she said. “He’ll have his iPad and will sit next to the window so he can look out.”
All of these activities combine to make air travel part of Connor’s routine and increase the odds for a calm flight.
“Through my experience of being his mom for a dozen years, I have learned how to speak the language of autism,” Rachel said. “You want to be three steps ahead and always backward planning for what we may face that day.”
After his year of treatments in Panama, Connor will continue to be tracked by the medical staff at the Stem Cell Institute for a full year. The study is due to end in 2017.
With takeoff just weeks away, Rachel is optimistic — and realistic.
“I am not delusional and thinking he will be cured 100 percent, because the reality is Connor will still be autistic at the end of the trial,” she said. “If what we are doing is a step forward in curing autism, that is very exciting.”