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‘Bubble baby’ treatment shifted to stem cell agency, UCLA

Evangelina Padilla-Vaccaro’s medical team gathers around her on the day she received her gene-therapy stem-cell transplant. (Photo: Padilla-Vaccaro family, via UCLA Health)

A London-based biotech firm has given up its life-saving treatment for the bubble baby disease and turned it over to California’s $12 billion stem cell agency and UCLA, where it was developed with tens of millions of taxpayer dollars.  

The families of children suffering from the rare affliction hailed the decision, saying that they were relieved, grateful and hopeful that it would soon mean treatments for their children.

The agency was created 16 years ago by voters who were led to believe during the ballot initiative campaign that miracle therapies were right around the corner.

“Waiting was never the worst part,” said Shayla Sulack of Tehachapi, whose daughter was in line to be treated.  “It was wondering if the waiting would ever end, or was this (treatment) on hold indefinitely. This is movement, at least, in the forward direction.” (See the full texts of the parents’ comments here.)

Orchard Therapeutics PLC had declined to provide compassionate use of the treatment (OTL-101) to 20 children, whose immune systems were crippled by the rare genetic mutation called ADA-SCID. Orchard’s actions disturbed the parents of the children, who deplored the company’s moves as placing profits over saving lives. The treatment has already saved the lives of 50 other children, according to UCLA.

The success so far of the OTL-101 treatment has been a hallmark for the agency, which was created 16 years ago by voters who were led to believe during the ballot initiative campaign that miracle therapies were right around the corner.

Known officially as the California Institute for Regenerative Medicine (CIRM), the program was set to expire last year. Voters, however, refinanced it with $5.5 billion more in state bonds and vastly expanded its scope. Nonetheless, funding will run out in roughly another decade unless additional sources of cash are found.  CIRM has yet to produce a stem cell therapy that is widely available to the public.

“The news that Orchard Therapeutics is returning OTL-101 … is wonderful news for families who have children with ADA-SCID.” — Maria T. Millan

The stem cell agency supported the OTL-101 research with $42 million at UCLA, which licensed it to Orchard. Twelve months ago, money-losing Orchard put the treatment on a financial back burner in favor of potential therapies that may generate more profits. And it suspended the clinical trial that was being financed by California taxpayers.

Maria T. Millan, president and CEO of the stem cell agency, had an optimistic perspective. 

“The news that Orchard Therapeutics is returning OTL-101 …is wonderful news for families who have children with ADA-SCID,” she said in a statement.

 “This means that UCLA will now be able to begin the process of applying to the Food and Drug Administration (FDA) for permission to start an expanded use protocol to treat patients.”

(See her full text here.)

In a statement, Orchard said it could not overcome “technical issues” dealing with manufacturing the treatment.

The announcement raised a number of questions. They include whether Orchard would supply any cash in the deal or what financial contributions would be forthcoming from UCLA or CIRM.

“We have encountered technical issues specific to the commercial-grade manufacturing processes for this particular therapy that must be addressed…” — Orchard Therapeutics PLC

UCLA said, “The decision by Orchard Therapeutics is good news for patients and their families. UCLA will work closely with Orchard and CIRM on the many details necessary, including FDA permission, to make the treatment available to patients through an expanded access program as soon as practical. 

“Given the complexities of clinical trials and regulatory processes, we are unable to offer a timetable. Beyond that, UCLA and our collaborator in developing this therapy, University College London, will look for a new commercial partner who can seek full regulatory approval and make this treatment available long-term.” 

(See full text of UCLA statement here.)

Orchard said it would “support” UCLA with “financial and material” resources for the treatment and elaborated on its decision to shut down the treatment, which was close to reaching commercialization. 

“Although the clinical data for investigational OTL-101 are very encouraging,” Orchard said, “we have encountered technical issues specific to the commercial-grade manufacturing processes for this particular therapy that must be addressed before we, or any other entity, could progress the program toward a regulatory submission. Without the ability to reliably manufacture OTL-101 at a commercial standard there is no way to receive FDA approval for the gene therapy.

“We have actively worked with CIRM, UCLA, UCL (University College London) and our external manufacturing partners to determine potential solutions to address these manufacturing requirements. In addition, we have sought partners willing to invest to help us take this work forward, but we have not identified a viable path forward on either front.”

Orchard continued, “We understand the importance of OTL-101 to the ADA-SCID community, and we are proud of the progress we have made in advancing OTL-101 to date. However, we also recognize and wish to respect the community’s strong preference that we return the program to our academic partners at this time. For these reasons, we have made the difficult decision to inform our collaborators at UCLA and UCL of our intent to terminate our license agreement and facilitate a smooth transfer of OTL-101 back to them.”

“Despite our decision to return the license, we will continue to stand by the commitment we made to our academic partners to support them with financial and material resources to seek to treat ADA-SCID patients under a separate compassionate use program that would be led and administered by their institutions.”

Editor’s Note: David Jensen is a retired newsman who has followed the affairs of the $12 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report. His recently published book, “California’s Great Stem Cell Experiment,” explores the pluses and minuses of the agency’s work. 

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