Gene theraphy for Spinal Muscular Atrophy approved by FDA

Dr. Kouzi

 

A new drug to treat spinal muscular atrophy (SMA) approved by the Food and Drug Administration (FDA) on Friday will come with a price tag of $2.1 million, making it the most expensive medicine in the world.

The drug, called Zolgensma, treats SMA, a genetic disease that causes debilitating muscle weakness and paralysis, and is a leading cause of infant mortality.

The high price tag is the latest illustration of how innovative drugs, while holding immense promise for improving the lives of the patients who need them, also pose a financial burden on the health care system through their cost.

The maker of the drug, Novartis, argued on Friday that the cost is still less than what it costs to treat people with SMA, which the company said was $4.1 million over 10 years.

"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” said Vas Narasimhan, Novartis’s CEO. “Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system.”

A top FDA official said the drug provides the opportunity for improved care for people with SMA.

“Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival.”

The drug will treat children under the age of two.

https://thehill.com/policy/healthcare/445451-fda-approves-worlds-most-expensive-drug-at-%242.1M

Treatment of Multiple Sclerosis with hematopoietic stem cells

Dr. Kouzi

https://jamanetwork.com/journals/jama/article-abstract/2720728

 

Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis  A Randomized Clinical Trial

Richard K. Burt, MD¹; Roumen Balabanov, MD²; Joachim Burman, MD³; et alBasil Sharrack, MD⁴; John A. Snowden, MD⁵; Maria Carolina Oliveira, MD⁶; Jan Fagius, MD³; John Rose, MD⁷; Flavia Nelson, MD⁸; Amilton Antunes Barreira, MD⁹; Kristina Carlson, MD¹⁰; Xiaoqiang Han, MD¹; Daniela Moraes, MD⁶; Amy Morgan, APRN¹; Kathleen Quigley, RN¹; Kimberly Yaung, RN¹; Regan Buckley, RN¹; Carri Alldredge, RN¹; Allison Clendenan, APRN¹; Michelle A. Calvario, APRN¹; Jacquelyn Henry, APRN¹; Borko Jovanovic, PhD¹¹; Irene B. Helenowski, PhD¹¹

JAMA. 2019;321(2):165-174. doi:10.1001/jama.2018.18743

 

Question  Is nonmyeloablative autologous hematopoietic stem cell transplantation (HSCT) more effective than disease-modifying therapy for patients with highly active relapsing-remitting multiple sclerosis (MS)?

Findings  In this randomized clinical trial that included 110 patients with relapsing-remitting MS, treatment with nonmyeloablative HSCT compared with disease-modifying therapy resulted in a significantly prolonged time to disease progression (hazard ratio, 0.07).

Meaning  In this preliminary study, nonmyeloablative HSCT was more effective than disease-modifying therapy for patients with relapsing-remitting MS.

Abstract

Importance  Hematopoietic stem cell transplantation (HSCT) represents a potentially useful approach to slow or prevent progressive disability in relapsing-remitting multiple sclerosis (MS).

Objective  To compare the effect of nonmyeloablative HSCT vs disease-modifying therapy (DMT) on disease progression.

Design, Setting, and Participants  Between September 20, 2005, and July 7, 2016, a total of 110 patients with relapsing-remitting MS, at least 2 relapses while receiving DMT in the prior year, and an Expanded Disability Status Scale (EDSS; score range, 0-10 [10 = worst neurologic disability]) score of 2.0 to 6.0 were randomized at 4 US, European, and South American centers. Final follow-up occurred in January 2018 and database lock in February 2018.

Interventions  Patients were randomized to receive HSCT along with cyclophosphamide (200 mg/kg) and antithymocyte globulin (6 mg/kg) (n = 55) or DMT of higher efficacy or a different class than DMT taken during the previous year (n = 55).

Main Outcomes and Measures  The primary end point was disease progression, defined as an EDSS score increase after at least 1 year of 1.0 point or more (minimal clinically important difference, 0.5) on 2 evaluations 6 months apart, with differences in time to progression estimated as hazard ratios.

Results  Among 110 randomized patients (73 [66%] women; mean age, 36 [SD, 8.6] years), 103 remained in the trial, with 98 evaluated at 1 year and 23 evaluated yearly for 5 years (median follow-up, 2 years; mean, 2.8 years). Disease progression occurred in 3 patients in the HSCT group and 34 patients in the DMT group. Median time to progression could not be calculated in the HSCT group because of too few events; it was 24 months (interquartile range, 18-48 months) in the DMT group (hazard ratio, 0.07; 95% CI, 0.02-0.24; P < .001). During the first year, mean EDSS scores decreased (improved) from 3.38 to 2.36 in the HSCT group and increased (worsened) from 3.31 to 3.98 in the DMT group (between-group mean difference, −1.7; 95% CI, −2.03 to −1.29; P < .001). There were no deaths and no patients who received HSCT developed nonhematopoietic grade 4 toxicities (such as myocardial infarction, sepsis, or other disabling or potential life-threatening events).

Conclusions and Relevance  In this preliminary study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT, resulted in prolonged time to disease progression. Further research is needed to replicate these findings and to assess long-term outcomes and safety.

Trial Registration  ClinicalTrials.gov Identifier: NCT00273364

Editorial

Stem Cell Transplantation to Treat Multiple Sclerosis

 

 

Cerebral palsy treatment with cord blood stem cells

Dr. Kouzi

Ashton’s Cord Blood Story April 2019 A ViaCord Family Story - by Ashton’s parents, Alisa & Mayo   “We’re not scientists, doctors, or researchers.

Parent’s Guide to Cord blood

By Frances Verter

” Alisa: “My husband and I went for fertility for three years, and we had our first son Preston. He was healthy, and wonderful, and we were so happy. And then Ashton came along.” Mayo: “I didn’t know how I was going to feel about it, because we worked so hard to get our first son. I thought it was awesome. And we had two sons.” Alisa: “So we were just ecstatic. Another baby. Mayo: “Ashton was born natural and normal. And then 6 weeks later he gets sick.” Alisa: “At 6 weeks old, Ashton became sick one night with RSV and pneumonia and we had him rushed into the hospital. His blood oxygen level went from 100% to zero.” Mayo: “I was more shocked than anything else: didn’t know what to do, didn’t know what to think. How could this happen to my son?” Alisa: “The doctors kept working with him, and finally got his heart back.” Mayo: “Unfortunately, because of what happened to him, with the lack of oxygen to his brain, that’s what causes cerebral palsy, and that’s what he has. Alisa: “We had to look up what cerebral palsy was, we had no idea, you know, how bad it was. They really did extensive testing and said ‘there’s not much hope for activities in the future for him, physically or mentally’.  And we didn’t have total belief that Ashton’s body was going to ever either walk or be strong enough to balance.” Mayo: “It’s hard on a parent to have your son go through all that.” Alisa: “We needed a lot of physical help, because he’s getting bigger and heavier and it’s a lot of lifting on us. So we were looking for any type of help physically, for his physical condition, that we could get.” Mayo: “Before Ashton was born my wife brought this idea of saving his cord blood. And so she kept poking at me, poking at me, and she says, ‘You know, we should save it, you never know when he might need it.’” Alisa: “I brought it to my husband’s attention after reading all the positive testing they’ve done with stem cells, and I thought ‘Hey, why not?’.  Never once in my mind did I think my new baby would ever be ill enough to use his own stem cells.” Alisa: “Five years into Ashton’s life, we get paperwork saying they’re doing a study on children like Ashton who have cerebral palsy, using stem cells. So we decided to go ahead with it, and go ahead do it, and have Ashton infused with his own stem cells that we banked and see what the results would be. They ended up infusing him in his foot, through an IV, and the process didn’t take 20 minutes, if that.” Alisa: “Five months into it, we noticed Ashton regaining some strength and balance in his body. Then we noticed Ashton cognitively making his wants and needs clearly known to us. Mayo: “We go to his parent-teacher conference to find out how his progress is in school, and they’re telling us how he’s doing in school. I said ‘That’s all fine and dandy. How’s he doing with walking?’  Because I want to know if he’s walking.  Oh, his therapists look at each other and just laugh and they say ‘He’s walking every day here.’” Alisa: “So we saw balance, the school saw balance, and Ashton started walking and balancing. Ashton can walk.” Mayo: “He adapts and overcomes his disability. That’s what makes him a warrior, I believe. There’s nothing that he won’t do or try. He’ll play lacrosse with his brother. His brother is his best friend. He plays basketball. He’ll ride a bike. He drives his little power wheel around. You know what, he likes to swim.” Alisa: “Ashton was on a wheelchair bus for quite a long time. He fussed, and he cried, and he didn’t feel like the other kids because he was the only one on the wheelchair. Seeing him walk off that school bus and not be in a wheelchair means everything. Now he can walk into any facility with his head up. He smiles, he gives people high fives, he says ‘Hey, what’s up?’.  You know, he feels like one of the gang.” Mayo: “Don’t quit. You adapt and you overcome. My hero is my son. We received that letter from Viacord; I think it’s the best thing for my son. To see him accomplishing everything that he’s doing now, I attribute to the stem cell. I think everybody should look into it more often. If it’s done this for my son, it could do it for everybody else. Look at him today.”

 

Biohellenika today received the renewed certificate of AABB

Dr. Kouzi

Biohellenika today received the renewed certificate of accreditation from the American Association of Blood Banks (AABB). Accreditation is valid until September 30, 2020.

Accreditation from AABB classifies Biohellenika among the most trusted companies in the industry internationally and enables parents to use their stem cells in all hospitals in Greece and abroad.