Can the leaders of the $12 billion California stem cell agency “march in” and force the use of a genetic treatment to save the lives of children with a rare, fatal affliction known as the bubble baby disease?
They can try.
But the battle could be long and the outcome in considerable doubt. At stake, nonetheless, are the babies born with a genetic defect that is medically described as ADA-SCID.
Children with the defect usually die by the age of two if left untreated. Their immune systems are broken. They suffer from serious lung infections and chronic diarrhea. “They have trouble gaining weight and do not grow very well. Other symptoms include skin rashes, absent tonsils and lymph nodes, bone abnormalities and developmental delay,” says the National Institutes of Health(NIH).
About 100 children are born annually in the United States with SCID, based on a rate of about one case among 40,000 newborns.
The question of the babies’ survival involves a publicly traded British company called Orchard Therapeutics PLC and its clinical trial for the genetic therapy. The trial is being financed by the state stem cell agency, officially known as the California Institute for Regenerative Medicine(CIRM).
CIRM has backed the bubble baby research with more than $40 million. The therapy was developed at UCLA and licensed exclusively to Orchard, which last October suspended the CIRM-backed trial in favor of developing other, more profitable therapies.
That means that children who are not already involved in the trial do not have access to the treatment, which UCLA says has saved the lives of more than 50 persons.
It would seem to be a case where “marching in” to save the babies might naturally come into play. But the march-in story is complex and goes to such matters as patents, intellectual property, the survival of a business, ethics and the appropriate role of government funding agencies -- not to mention life and death.
March-in rights, as they are known, were conceived decades ago under federal law to prevent private companies from shelving effective treatments for financial reasons. Federal march-in rights, however, have never been exercised. The California state agency has its own version of march-in rights, but it too has never exercised them.
They are not without controversy. The rights are attached usually to early stage research that is funded by the government. In the case of CIRM, California taxpayers will pony up $12 billion for all its efforts, based on current estimates.
A CIRM document dealing with march-in says CIRM “may intercede in certain circumstances on behalf of the state and grant exclusive or nonexclusive licenses if the awardee/exclusive licensee:
“Fails to make reasonable efforts to achieve practical application of the invention or research data;
“Fails to comply with the access plan;
“Fails to use the data or invention to address a public health emergency declared by the Governor.”
A company has at least a year to comply, except in the case of a gubernatorial emergency, and, of course, could litigate beyond that period.
In 2012, the National Academy of Medicine (formerly the Institute of Medicine) performed a study of CIRM (at a cost of $700,00 to the agency) that included a look at its march-in provisions.
The study cited criticism of the provisions by organizations that have representation on the CIRM governing board, including the University of California and BIOCOM, an industry group. “Overly broad” and “chilling” were two comments. The study said the provisions “raise considerable uncertainty about the future implementation of rights that may outlive CIRM itself.”
CIRM told the California Stem Cell Report last week that it “reserves” the possibility of using its march-in rights. “CIRM is committed to its mission of doing everything we can to help patients in need and so we are working with all of our stakeholders in moving this project forward,” the statement said.
The agency has been mum on the subject since then. In response to questions, Orchard has released only a brief statement and declined to go beyond that. However, the company said in a separate financial filing last week that it has already saved $2.5 million by shelving the bubble baby treatment and plans to continue to deprioritize the cure.
Here are links to earlier articles on the Orchard/CIRM/UCLA trial.
California Stem Cell Agency Pledges to Do "Everything" to Salvage Cure for Bubble Baby Disease -- Orchard Therapeutics Says, However, It Will Continue to Deprioritize the Therapy and Has Already Saved $2.5 Million