First Patient Dosed in Phase 1 Clinical Trial for T1D

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There’s some good news for a company and a therapeutic approach that CIRM has been supporting for many years.

In September 2018, CRISPR Theraputics and ViaCyte entered a partnership to discover, develop and market gene-edited stem cell-derived therapies to treat type 1 diabetes (T1D). Today, they may stand one step closer to their goal. 

Last week the companies jointly announced that they have dosed the first subject in the Phase 1 clinical trial of VCTX210 for the treatment of T1D. VCTX210 is an investigational stem cell-based therapy. It was developed combining CRISPR’s gene-editing technology with ViaCyte’s stem cell expertise to generate pancreatic beta cells that can evade the immune system.

ViaCyte, a regenerative medicine company long backed by CIRM, has developed an implantable device which contains pancreatic endoderm cells that mature over a few months and turn into insulin-producing pancreatic islet cells, the kind destroyed by T1D. 

ViaCyte’s implantable stem cell pouch

Using CRISPR technology, the genetic code of the implanted cells is modified to create beta cells that avoid all recognition by the immune system. This collaboration aims to eliminate the requirement of patients taking daily immunosuppressants to stop the immune system from attacking the implanted cells. 

The first phase of the VCTX210 clinical trial will assess the safety, tolerability, and immune evasion in patients with T1D. 

“We are excited to work with CRISPR Therapeutics and ViaCyte to carry out this historic, first-in-human transplant of gene-edited, stem cell-derived pancreatic cells for the treatment of diabetes designed to eliminate the need for immune suppression,” said James Shapiro, a clinical investigator in the trial. “If this approach is successful, it will be a transformative treatment for patients with all insulin-requiring forms of diabetes.”

CIRM has been a big investor in ViaCyte’s work for many years and has invested more than $72 million in nine different awards.  

Why “Right to Try” laws are more feel good than do good

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L to R: Don Gibbons, CIRM; Jeanne Loring; Beth Roxland; Aaron Levine

In the last few years some 24 states have approved so-called “Right to Try” laws. These are intended to give terminally ill patients faster and easier access to experimental therapies. But a panel of experts at the World Stem Cell Summit in Atlanta today said they are more symbolic than anything and do little to actually help patients get much-needed therapies.

The Right to Try laws are modeled after a federal law that allows “compassionate use” of experimental medications and lets doctors prescribe investigational medicines being safely used in early stage clinical trials.

Beth Roxland, a bioethicist with Johnson & Johnson, says the name of the law is misleading:

“If you look at the actual text of these laws they only say you have the right to “ask” for these drugs. That right already exists in federal law but neither federal law nor these Right to Try laws say you have the right to access.”

Aaron Levine from Georgia Tech says it’s also misleading to assume that just because a state passes a Right to Try law that it has any legal impact. He says state laws don’t over rule the Food and Drug Administration’s (FDA) regulation of this area and so the federal government would still have the authority to stop this kind of access.

But Levine says these laws are interesting in that they are indicative of the growing determination of patients and patient advocates to work around obstacles to access and have a bigger say in their own care.

One of the audience members, William Decker from Baylor College of Medicine, says that in Texas a law was recently crafted saying that as long as a potential therapy had gone through a Phase 1 safety trial it should be offered to the public and the public should be able to pay for it.

“If you know how clinical trials work you know you can get almost any schlock through a Phase 1 trial and the kinds of things that you can get to the public without any idea if they work often turn out to not be very useful. We saw this as an avenue to promote fraud, and the last thing you should be doing to a dying patient is take their money or divert their attention away from something that might help them.”

Decker and his colleagues argued before the Texas Legislature that potential therapies should at least have to go through a Phase 2 trial to make sure they were not only safe but also showed some benefit for patients. In the end Texas lawmakers rejected the Phase 2 idea but did say patients could not be charged for the therapy, and there could be no compensation from insurers or anyone else for the manufacturer of the therapy.

He says removing the financial benefits and incentives pretty much ensured that no company would offer patients a therapy under this law.

Jeanne Loring, a CIRM grantee from the Scripps Research Institute, says that likely won’t stop other clinics in other states:

“Some stem cell clinics are using adipose (stem cells derived from fat) therapy as an option for every disease imaginable and I’m sure some will take advantage of these laws to say it gives them the right to offer these to patients and the patients will pay for them directly. “

Roxland says that may already be happening:

“I think there is some evidence on the stem cell side that companies have popped up in states that have these laws, to make it easier to offer their therapies to patients.”

The panel agreed that in most cases these laws don’t give patients any rights they don’t already have, but do give the appearance of making access easier. They said it’s feel-good legislation, allowing people to feel they are doing something without actually doing anything.

Aaron Levine said that while some companies may try to take advantage of these laws, the most serious ones won’t:

 “Almost any legitimate company that wants an FDA approved product wouldn’t want to take advantage of these laws. It could put their product at risk. Most companies that need to work with the FDA have no incentive to go this route.”