Stem cell transplant offers Jake a glimpse of hope

Jake

Jake Javier surrounded by friends; Photo courtesy Julie Haener KTVU

On Thursday, July 7th, Jake Javier became the latest member of a very select group. Jake underwent a stem cell transplant for a spinal cord injury at Santa Clara Valley Medical Center here in the San Francisco Bay Area.

The therapy is part of the CIRM-funded clinical trial run by Asterias Biotherapeutics. For Asterias it meant it had hit a significant milestone (more on that later). But for Jake, it was something far more important. It was the start of a whole new phase in his life.

Jake seriously injured his spinal cord in a freak accident after diving into a swimming pool just one day before he was due to graduate from San Ramon Valley high school. Thanks, in part, to the efforts of the tireless patient advocate and stem cell champion Roman Reed, Jake was able to enroll in the Asterias trial.

astopc1The goal of the trial is to test the safety of transplanting three escalating doses of AST-OPC1 cells. These are a form of cell called oligodendrocyte progenitors, which are capable of becoming several different kinds of brain cells, some of which play a supporting role and help protect nerve cells in the central nervous system – the area damaged in spinal cord injury.

To be eligible, individuals have to have experienced a severe neck injury in the last 30 days, one that has left them with no sensation or movement below the level of their injury, and that means they have typically lost all lower limb function and most hand and arm function.

The first group of three patients was completed in August of last year. This group was primarily to test for safety, to make sure this approach was not going to cause any harm to patients. That’s why the individuals enrolled were given the relatively small dose of 2 million cells. So far none of the patients have experienced any serious side effects, and some have even shown some small improvements.

In contrast, the group Jake is in were given 10 million cells each. Jake was the fifth person treated in this group. That means Asterias can now start assessing the safety data from this group and, if there are no problems, can plan on enrolling people for group 3 in about two months. That group of patients will get 20 million cells.

It’s these two groups, Jakes and group 3, that are getting enough cells that it’s hoped they will see some therapeutic benefits.

In a news release, Steve Cartt, President and CEO of Asterias, said they are encouraged by the progress of the trial so far:

“Successful completion of enrollment and dosing of our first efficacy cohort receiving 10 million cells in our ongoing Phase 1/2a clinical study represents a critically important milestone in our AST-OPC1 clinical program for patients with complete cervical spinal cord injuries. In addition, while it is still very early in the development process and the patient numbers are quite small, we are encouraged by the upper extremity motor function improvements we have observed so far in patients previously enrolled and dosed in the very low dose two million cell cohort that had been designed purely to evaluate safety.”

 

jake and familyJake and his family are well aware that this treatment is not going to be a cure, that he won’t suddenly get up and walk again. But it could help him in other, important ways, such as possibly getting back some ability to move his hands.

The latest news is that Jake is doing well, that he experienced some minor problems after the surgery but is bouncing back and is in good spirits.

Jake’s mother Isabelle said this has been an overwhelming experience for the family, but they are getting through it thanks to the love and support of everyone who hears Jake’s story. She told CIRM:

 “We are all beyond thrilled to have an opportunity of this magnitude. Just the thought of Jake potentially getting the use of his hands back gives him massive hope. Jake has a strong desire to recover to the highest possible level. He is focused and dedicated to this process. You have done well to choose him for your research. He will make you proud.”

He already has.

Jake and Brady gear

New England Patriots star quarterback Tom Brady signed a ball and jersey for Jake after hearing about the accident


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6 thoughts on “Stem cell transplant offers Jake a glimpse of hope

  1. What a difference between what was told to my son Roman Reed when he was first injured. No hope was given him at all.
    But now the question of cure is not if, but when.
    Long life to CIRM, Asterias,Jake, and of course Roman Reed.

  2. Dear Sir,
    Saw and read wonderful news a big development.
    We at this third world can not imagine we have many cases like this and many more blood disorder children need help treatment and many more.
    Regards

  3. Pingback: July 2016 eNews - Nebraska Coalition for Lifesaving Cures

  4. In 2004 the PBS program Innovation featured the work of Dr. Carlos Lima of Portugal who had restored sensation and movement in spinal injuries well past the 30-day period. He used adult stem cells from olfactory tissue. Other than brief discussion on George Noory’s radio program, I never saw any follow-up on (the late) Dr. Lima’s work. I recall reports about the first CRM trial using embryonics being abruptly suspended without explanation apparently due to issues regarding the private funding involved with the company associated with the research. Nothing I can find on this site acknowledges any of this.

    For those of us concerned with war veterans care this is most frustrating. There are thousands of injured vets who would gladly participate in a study like this; how would you like to be a quadriplegic and find out there was a possible therapy and you were denied access to it for what looks like maybe political reasons–or “safety” issues, if I were a quad I would say “what have I got to lose?”

  5. Dear Mark, thanks for the comment. I am familiar with Dr. Lima’s work, it was, in many ways, pioneering and helped get us to where we are today. Sadly Dr. Lima died in 2012 but the results of his work were published in a scientific journal in 2006 – https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1864811/

    The first CIRM trial using embryonic stem cells for spinal cord injury was with Geron. That was halted in 2011 for business reasons. Geron got a new CEO and he decided to focus the company’s efforts on other areas. However, the approach that Geron used was revived by Asterias and that is the trial we are funding now, the one we hope will help Jake Javier and others.

    I can understand your sense of frustration at the slow pace of progress but science doesn’t always move foward as fast as we would like. We are working as hard as we can to make sure whatever therapies we test in clinical trials are safe for people. The last thing anyone wants to do is cause more pain to people whose lives have already been impacted by injury. We also don’t want to raise false hope by saying something could help someone when we don’t know if it can.

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