"Thank you for keeping my family complete," said Alysia Padilla-Vaccaro, her voice cracking as she spoke to the governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known.
Her daughter, Evangelina, also whispered a soft "thank you"to the board and CIRM staff at the Oakland meeting. She had suffered from what is known as the "bubble baby syndrome."
Evangelina was one of two persons cured by treatments developed with the help of financing from the stem cell agency, created by voters in 2004 and supported by state bonds.
Brenden Whittaker, 22, was the second person. He was near death twice and lost parts of his lungs and liver as the result of another, rare, immune deficiency disease. He said,
"My experience has truly been life-changing...I hope you continue to push the boundaries and help as many people as you can."Randy Mills, president of the agency, presented Whittaker and Evangelina to the board during a review of the progress of the agency. He said they were "the first two patients to be cured by CIRM programs." Mills also introduced two other patients who have been helped by the agency's research: Jake Javier, who suffered a paralyzing spinal cord injury, and Karl Trede, a cancer patient. The agency has not developed a therapy for widespread use.
Mills hailed their courage and the courage of their families for taking part in the risky clinical trials. Mills told the 29-member CIRM board that in pursuit of stem cell cures that the agency had to be "Jake-strong," a term originating with Javier's mother after his injury.
The stem cell agency estimates it has $692 million left for new awards between now and 2020, when it expects to run out of cash. The research budget for next year was approved at $328 million with most of it going for clinical programs. Clinical trials represent the last stage for a proposed therapy before it reaches the market.
The CIRM board has approved a goal of 40 more clinical trials between now and 2020. Currently it has 22 underway. Only one out of 10 conventional treatments that begin trials wind up as commercial products. Often the trial process can take many years even with conventional therapies. No stem cell treatments have yet been approved by the Food and Drug Administration in this country.
Here is more on the patients featured at today's meeting:
Evangelina, of Corona, Ca., was diagnosed shortly after birth with "bubble baby" disease and no functioning immune system. Her blood cells were genetically altered to create a new blood and immune system. The therapy was developed by Donald Kohn of UCLA. CIRM has backed his work, which goes beyond the bubble baby affliction, with nearly $52 million. (See here, here and here.)
Whittaker, of Columbus, Ohio, almost died twice as a result of a rare immune system disease. Parts of his lung and liver were removed due to repeated infection. His blood stem cells were genetically modified to create a new blood system and a healthy immune system. The treatment was also developed by Kohn at UCLA.
|Karl Trede, CIRM graphic|
(For the text of CIRM summaries on the four, see here.)
(Editor's note: The earlier graphics in this item were replaced later with the ones that actually were displayed the day of the meeting. An earlier version also misstated Evangelina's age.)