Friday, July 27, 2012

Patient advocates speak in support of disease teams, new therapies

Huntington's disease advocates speaking to the Governing Board
Yesterday’s governing board meeting should have been a happy one. The agency awarded $150 million dollars to fantastic teams of researchers whose work could result in therapies for some of the worst diseases – Huntington’s disease, Lou Gehrig’s disease, melanoma and heart disease, to name a few.

But that happiness was bittersweet. The reason we need to fund research is because there are currently no cures for those diseases. And for the people living with the diseases, the results of the research we funded might come too late.

For me, the hardest part of the day by far was hearing from those families whose children—almost exclusively boys—have muscular dystrophy. These are parents who saw their happy, healthy, active boys start to weaken at around age five. These parents will inevitably lose their children to the disease unless there is a cure.

My own happy, healthy, active boys are five and seven, exactly the ages many of those parents described. As those parents were before the diagnosis, I’m looking forward to seeing my kids grow up, have fun, find careers and start families. Instead, those families bought their kids wheelchairs and have watched them grow weaker and more dependent.

Later in the meeting a woman in a wheelchair spoke about raising her three children on her own and being diagnosed with Lou Gehrig’s disease (properly known as ALS). That disease is also inevitably fatal.

One woman who is going blind with retinitis pigmentosa spoke about wanting to see her husband’s face.

Things most of us take for granted—seeing our loved ones, hugging our kids—are the source of dreams for those who spoke at yesterday’s meeting.

Perhaps the most visible group of patient advocates at the meeting were the Huntington’s disease families who showed up en masse to support an application from Vicki Wheelock of the University of California, Davis. That community is so involved in driving new research that many have become old friends. They threw what might be the first confetti at a public state meeting when our governing board voted to approve funding for that award.

Their excitement wasn’t for a cure—even if Wheelock’s work is successful it will be many years before a treatment is widely available. It was for hope.

Speaking before the board, Katie Jackson, whose husband has Huntington’s disease said:
"When Mike was diagnosed, I couldn't believe there was nothing they could do for him. We need hope more than we can explain to you."
CIRM can’t know in advance which research projects are going to be successful. Some will inevitably not result in the hoped-for clinical trials. That’s the process of science—trying different approaches until you find one that works. All we can do is try to pick the best science and then do what we can help those teams succeed in turning the hope into a reality.

Here's our press release about the awards



  1. "All we can do is try to pick the best science and then do what we can help those teams succeed in turning the hope into a reality"

    Then can you explain why 10+ applications from the only company currently conducting hesc trials has been denied over and over again?

    Geron award CIRM left 50 Million on the Table. (funding round was a complete failure)
    This round you left 100 Million on the Table.

    How hard is it to give out money to the most promising research actually showing positive results in humans?

  2. Thank you for highlighting the plight of boys and young men with Duchenne muscular dystrophy. We are excited about the progress that potential CIRM funding can generate.

  3. So what is a patient to do...go to Israel and attain "Compassion​ate Use" designation?

    Israeli Channel 2 reports: Dramatic Change in Patient with ALS and MG
    following Compassionate Use of BrainStorm's NurOwn(TM) Cell Therapy

    PETACH TIKVA, Israel, Jul 02, 2012 (BUSINESS WIRE) -- BrainStorm Cell
    ; Israeli Channel 2 TV interviewed
    today a patient suffering from Myasthenia Gravis (MG) and recently diagnosed with ALS. The patient reported that he has experienced
    visible improvement in his speech, walking, balance, posture, muscle strength, appetite, digestion, and weight gain following compassionate
    treatment with BrainStorm's NurOwn cell therapy.

    "Due to the rare disease combination of MG and ALS, this patient was approved for compassionate treatment with BrainStorm's NurOwn cell
    therapy," says Professor Dimitrios Karussis of the Neurology Department at The Hadassah Medical Center in Jerusalem, and Principal Investigator of BrainStorm's current Phase I/II clinical trial.
    "Within a few weeks following injection with NurOwn cells, the patient showed dramatic improvement in a variety of functions including
    breathing, speech, walking, muscular strength, and overall well-being. While we cannot draw scientific conclusions based on the outcome of an
    individual patient, these results are extremely encouraging."


  5. FDA Gives Miami Project to Cure Paralysis Green Light to Begin Human Trial

    Led by W. Dalton Dietrich, Ph.D., the Schwann cell clinical trial team at The Miami Project is composed of a multi-disciplinary group of basic science and clinical faculty members, scientific staff, and regulatory personnel focused on advancing the trial.
    “Obtaining clearance from the FDA to initiate this important Phase I clinical trial is a vital step for the field of SCI research, and for the Miami Project team that has been working diligently on this therapeutic concept for more than a quarter of a century. This trial, when completed successfully, will lay the critical foundation for future cell-based therapies we plan to target SCI,” said Dr. Dietrich, Scientific Director of The Miami Project.
    The clinical trial will enroll eight participants with an acute thoracic SCI. Newly injured patients brought to the trauma center would have to meet the stringent criteria and agree to participate in further screening within five days of their injury. At that point, the participant will undergo a biopsy of a sensory nerve in one leg to obtain his or her own Schwann cells. The Schwann cells will then be grown in a culturing facility for three to five weeks to generate the number of cells necessary for transplantation, and to undergo the strict purification process. By the time the Schwann cells are surgically transplanted into the injury site, participants will be 26-40 days post-injury.