The race to unlock new treatments just hit a milestone. Montana’s bold new law is shaking up access to experimental drugs. Meanwhile, cutting-edge therapies for rare genetic disorders and neurodegenerative diseases are moving from the lab to clinical trials. The future of medicine is arriving fast—and it’s packed with hope.
Montana’s Expanded Right to Try Law Opens Doors
Montana took a giant leap forward. Since 2015, the state had a “right to try” law for terminally ill patients. Now, in 2023, it expanded access to all patients—not just the terminally ill. Anyone who gives informed consent and can pay can try experimental drugs. This could change lives.
The Montana Department of Health and Human Services finalized strict rules for clinics offering these drugs. They set up an experimental treatment review board, or ETRB. This board will review applications for access to unproven and unapproved medicines. The first two applications will be reviewed in the coming weeks.
Matt Kaeberlein, a longevity researcher and ETRB member, promises rigorous oversight. “It will be done in a very rigorous way, with qualified medical professionals and appropriate oversight,” he said. Bioethicist Jessica Flanigan and Montana-licensed doctor James Burke also join the board, ensuring patients get safe, transparent access to experimental treatments.
For families like Kris DeVault’s, this law means hope. Kris fights for access to an experimental drug for his son Brody, who has creatine transporter deficiency. Kris said, “He’s got no words, really. He wants to communicate more than he’s able to … which then turns into frustration.” “That is my one shot for Brody,” Kris said.
CAMP4’s Clinical Trial for SYNGAP1 Disorder Gets Green Light
On the other side of the world, CAMP4 Therapeutics just hit a major regulatory milestone. Australian authorities approved their Phase 1/2 clinical trial for CMP-002, a therapy for SYNGAP1-related disorder. This disorder affects over 10,000 Americans and causes intellectual disability in every patient.
Symptoms are severe:
- Epilepsy in roughly 85% of patients
- Severe behavioral problems in around 70%
- Sleep problems in about 60%
- Nearly 30% of patients are non-verbal
CMP-002 works by increasing SYNGAP1 gene expression. It binds to a specific regulatory RNA targeting SYNGAP1. In lab tests, the drug boosted SYNGAP protein levels in patient neurons. It reversed behavioral problems and reduced seizures in mouse models.
This Australian clearance lets CAMP4 raise up to $50 million through a private placement closing expected within five business days. CAMP4’s trial site in Australia will be one of the first to enroll patients. CEO Josh Mandel-Brehm is pushing forward to give hope to families facing this rare disorder.
New Pill Shows Promise Against Neurodegenerative Diseases
Could a simple pill slow down brain diseases? Researchers say yes. A new pill combines two drugs—osivelotor and PT2399—to mimic low oxygen conditions in mice. This “hypoxia pill” more than doubled survival for mice with Leigh syndrome, extending life from 62 days to 158 days.
It also stopped declines in strength and coordination in mice with Friedreich’s ataxia. Parkinson’s model mice improved coordination and balance after treatment. Low oxygen may boost brain growth factors and protect cells from oxygen damage.
Researcher Vamsi Mootha leads this work. Gerontologist Jamie Justice praised the potential, saying, “I saw an opportunity to help build a safe, transparent, and scientifically rigorous process.” Kamilla Miskowiak, a neuroscientist, said, “Taking a pill is much more feasible and much more something that could be implemented as a treatment. I’m quite excited about it.”
Still, questions remain. Researcher Gordon Mitchell asked, “How much of the benefit was simply that everything slowed down?” This will guide future studies.
The Road Ahead Is Bright
Montana’s law, CAMP4’s clinical trial, and the new hypoxia pill show a powerful trend. Patients are getting earlier access to experimental treatments. Rare diseases and neurodegenerative disorders are finally seeing breakthroughs.
The momentum is building. Clinics will soon review applications for unapproved drugs in Montana. CAMP4’s trial enrollment will open soon in Australia. And the hypoxia pill could revolutionize treatment for multiple brain diseases.
The future is closer than ever. Patients, families, and researchers are pushing the boundaries. The new era of medicine is here. And it’s filled with hope, science, and a hunger to heal.
Based on
- Montana’s new “right to try” law can’t come soon enough for some — technologyreview.com
- Montana’s plan to become an experimental medical hub just pushed forward | MIT Technology Review — technologyreview.com
- CAMP4 Therapeutics Secures Australian Regulatory Clearance to Initiate First-in-Human Clinical Trial of CMP-002 in Patients With SYNGAP1-Related Disorder | Markets Insider — markets.businessinsider.com
- Pill that lowers oxygen levels in brain may ease Parkinson’s symptoms | New Scientist — newscientist.com
